DESTIN, FLA. – Rituximab has a place in the treatment of certain subsets of patients with lupus, but generally the biologic agent has proved disappointing for this disease, according to Dr. R. John Looney.

The chimeric monoclonal antibody against the protein CD20 has worked very well in certain autoimmune diseases, and it is approved for a number of indications including refractory rheumatoid arthritis and antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis.

Rituximab is commonly used in relapsing and remitting multiple sclerosis, and early trial data suggest that the agent may have promise in the management of type 1 diabetes, said Dr. Looney, a professor of medicine at the University of Rochester (N.Y.).

In the "amazingly negative" EXPLORER (A Study to Evaluate the Efficacy and Safety of Rituximab in Patients With Severe Systemic Lupus Erythematosus) trial, however, overall clinical response rates did not differ significantly in rituximab- and placebo-treated nonrenal lupus patients (Arthritis Rheum. 2010;62:222-33).

And in the LUNAR (A Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis) trial, patients with lupus nephritis who were treated with rituximab had a modest improvement in proteinuria, compared with those who received placebo, but only a small, non–statistically significant improvement in overall renal outcomes of about 11%, compared with those who received placebo, according to data reported at conferences, including the annual conference of the American College of Rheumatology in 2009, Dr. Looney said.

"So rituximab has been a remarkably effective drug in certain types of autoimmune disease, but it has been a relative failure in lupus," he added.

An exception is in patients with antibodies associated with neuromyelitis optica, he said.

Neuromyelitis optica (NMO), which can also present as a primary disease, is remarkably responsive to rituximab, and there is a subset of lupus patients who have the NMO IgG/aquaporin-4 antibody that is associated with the disease, he said.

"If you’re looking at a patient with lupus who develops optic neuritis or if they develop transverse myelitis, you have to think about whether they are in a subgroup with these NMO antibodies," he said, noting that these patients will tend to have longitudinally extensive transverse myelitis that extends over three vertebral bodies, and they will tend to be positive for aquaporin-4.

"In this subgroup, we have moved to using rituximab early in the course of disease if [these patients] have been very refractory to other treatments," he said.

Dr. Looney said he also continues to use rituximab in severe refractory systemic lupus erythematosus, particularly in patients with central nervous system disease or lupus nephritis, as well as in patients with refractory idiopathic thrombocytopenic purpura (ITP).

Although rituximab is not approved for ITP, it is widely used as a secondary therapy for this condition, he said.

Dr. Looney disclosed that he has been an advisor for Genentech.

DESTIN, FLA. – Rituximab has a place in the treatment of certain subsets of patients with lupus, but generally the biologic agent has proved disappointing for this disease, according to Dr. R. John Looney.

The chimeric monoclonal antibody against the protein CD20 has worked very well in certain autoimmune diseases, and it is approved for a number of indications including refractory rheumatoid arthritis and antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis.

Rituximab is commonly used in relapsing and remitting multiple sclerosis, and early trial data suggest that the agent may have promise in the management of type 1 diabetes, said Dr. Looney, a professor of medicine at the University of Rochester (N.Y.).

In the "amazingly negative" EXPLORER (A Study to Evaluate the Efficacy and Safety of Rituximab in Patients With Severe Systemic Lupus Erythematosus) trial, however, overall clinical response rates did not differ significantly in rituximab- and placebo-treated nonrenal lupus patients (Arthritis Rheum. 2010;62:222-33).

And in the LUNAR (A Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis) trial, patients with lupus nephritis who were treated with rituximab had a modest improvement in proteinuria, compared with those who received placebo, but only a small, non–statistically significant improvement in overall renal outcomes of about 11%, compared with those who received placebo, according to data reported at conferences, including the annual conference of the American College of Rheumatology in 2009, Dr. Looney said.

"So rituximab has been a remarkably effective drug in certain types of autoimmune disease, but it has been a relative failure in lupus," he added.

An exception is in patients with antibodies associated with neuromyelitis optica, he said.

Neuromyelitis optica (NMO), which can also present as a primary disease, is remarkably responsive to rituximab, and there is a subset of lupus patients who have the NMO IgG/aquaporin-4 antibody that is associated with the disease, he said.

"If you’re looking at a patient with lupus who develops optic neuritis or if they develop transverse myelitis, you have to think about whether they are in a subgroup with these NMO antibodies," he said, noting that these patients will tend to have longitudinally extensive transverse myelitis that extends over three vertebral bodies, and they will tend to be positive for aquaporin-4.

"In this subgroup, we have moved to using rituximab early in the course of disease if [these patients] have been very refractory to other treatments," he said.

Dr. Looney said he also continues to use rituximab in severe refractory systemic lupus erythematosus, particularly in patients with central nervous system disease or lupus nephritis, as well as in patients with refractory idiopathic thrombocytopenic purpura (ITP).

Although rituximab is not approved for ITP, it is widely used as a secondary therapy for this condition, he said.

Dr. Looney disclosed that he has been an advisor for Genentech.

DESTIN, FLA. – Rituximab has a place in the treatment of certain subsets of patients with lupus, but generally the biologic agent has proved disappointing for this disease, according to Dr. R. John Looney.

The chimeric monoclonal antibody against the protein CD20 has worked very well in certain autoimmune diseases, and it is approved for a number of indications including refractory rheumatoid arthritis and antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis.

Rituximab is commonly used in relapsing and remitting multiple sclerosis, and early trial data suggest that the agent may have promise in the management of type 1 diabetes, said Dr. Looney, a professor of medicine at the University of Rochester (N.Y.).

In the "amazingly negative" EXPLORER (A Study to Evaluate the Efficacy and Safety of Rituximab in Patients With Severe Systemic Lupus Erythematosus) trial, however, overall clinical response rates did not differ significantly in rituximab- and placebo-treated nonrenal lupus patients (Arthritis Rheum. 2010;62:222-33).

And in the LUNAR (A Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis) trial, patients with lupus nephritis who were treated with rituximab had a modest improvement in proteinuria, compared with those who received placebo, but only a small, non–statistically significant improvement in overall renal outcomes of about 11%, compared with those who received placebo, according to data reported at conferences, including the annual conference of the American College of Rheumatology in 2009, Dr. Looney said.

"So rituximab has been a remarkably effective drug in certain types of autoimmune disease, but it has been a relative failure in lupus," he added.

An exception is in patients with antibodies associated with neuromyelitis optica, he said.

Neuromyelitis optica (NMO), which can also present as a primary disease, is remarkably responsive to rituximab, and there is a subset of lupus patients who have the NMO IgG/aquaporin-4 antibody that is associated with the disease, he said.

"If you’re looking at a patient with lupus who develops optic neuritis or if they develop transverse myelitis, you have to think about whether they are in a subgroup with these NMO antibodies," he said, noting that these patients will tend to have longitudinally extensive transverse myelitis that extends over three vertebral bodies, and they will tend to be positive for aquaporin-4.

"In this subgroup, we have moved to using rituximab early in the course of disease if [these patients] have been very refractory to other treatments," he said.

Dr. Looney said he also continues to use rituximab in severe refractory systemic lupus erythematosus, particularly in patients with central nervous system disease or lupus nephritis, as well as in patients with refractory idiopathic thrombocytopenic purpura (ITP).

Although rituximab is not approved for ITP, it is widely used as a secondary therapy for this condition, he said.

Dr. Looney disclosed that he has been an advisor for Genentech.

DESTIN, FLA. – Rituximab has a place in the treatment of certain subsets of patients with lupus, but generally the biologic agent has proved disappointing for this disease, according to Dr. R. John Looney.

The chimeric monoclonal antibody against the protein CD20 has worked very well in certain autoimmune diseases, and it is approved for a number of indications including refractory rheumatoid arthritis and antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis.

Rituximab is commonly used in relapsing and remitting multiple sclerosis, and early trial data suggest that the agent may have promise in the management of type 1 diabetes, said Dr. Looney, a professor of medicine at the University of Rochester (N.Y.).

In the "amazingly negative" EXPLORER (A Study to Evaluate the Efficacy and Safety of Rituximab in Patients With Severe Systemic Lupus Erythematosus) trial, however, overall clinical response rates did not differ significantly in rituximab- and placebo-treated nonrenal lupus patients (Arthritis Rheum. 2010;62:222-33).

And in the LUNAR (A Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis) trial, patients with lupus nephritis who were treated with rituximab had a modest improvement in proteinuria, compared with those who received placebo, but only a small, non–statistically significant improvement in overall renal outcomes of about 11%, compared with those who received placebo, according to data reported at conferences, including the annual conference of the American College of Rheumatology in 2009, Dr. Looney said.

"So rituximab has been a remarkably effective drug in certain types of autoimmune disease, but it has been a relative failure in lupus," he added.

An exception is in patients with antibodies associated with neuromyelitis optica, he said.

Neuromyelitis optica (NMO), which can also present as a primary disease, is remarkably responsive to rituximab, and there is a subset of lupus patients who have the NMO IgG/aquaporin-4 antibody that is associated with the disease, he said.

"If you’re looking at a patient with lupus who develops optic neuritis or if they develop transverse myelitis, you have to think about whether they are in a subgroup with these NMO antibodies," he said, noting that these patients will tend to have longitudinally extensive transverse myelitis that extends over three vertebral bodies, and they will tend to be positive for aquaporin-4.

"In this subgroup, we have moved to using rituximab early in the course of disease if [these patients] have been very refractory to other treatments," he said.

Dr. Looney said he also continues to use rituximab in severe refractory systemic lupus erythematosus, particularly in patients with central nervous system disease or lupus nephritis, as well as in patients with refractory idiopathic thrombocytopenic purpura (ITP).

Although rituximab is not approved for ITP, it is widely used as a secondary therapy for this condition, he said.

Dr. Looney disclosed that he has been an advisor for Genentech.

DESTIN, FLA. – Rituximab has a place in the treatment of certain subsets of patients with lupus, but generally the biologic agent has proved disappointing for this disease, according to Dr. R. John Looney.

The chimeric monoclonal antibody against the protein CD20 has worked very well in certain autoimmune diseases, and it is approved for a number of indications including refractory rheumatoid arthritis and antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis.

Rituximab is commonly used in relapsing and remitting multiple sclerosis, and early trial data suggest that the agent may have promise in the management of type 1 diabetes, said Dr. Looney, a professor of medicine at the University of Rochester (N.Y.).

In the "amazingly negative" EXPLORER (A Study to Evaluate the Efficacy and Safety of Rituximab in Patients With Severe Systemic Lupus Erythematosus) trial, however, overall clinical response rates did not differ significantly in rituximab- and placebo-treated nonrenal lupus patients (Arthritis Rheum. 2010;62:222-33).

And in the LUNAR (A Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis) trial, patients with lupus nephritis who were treated with rituximab had a modest improvement in proteinuria, compared with those who received placebo, but only a small, non–statistically significant improvement in overall renal outcomes of about 11%, compared with those who received placebo, according to data reported at conferences, including the annual conference of the American College of Rheumatology in 2009, Dr. Looney said.

"So rituximab has been a remarkably effective drug in certain types of autoimmune disease, but it has been a relative failure in lupus," he added.

An exception is in patients with antibodies associated with neuromyelitis optica, he said.

Neuromyelitis optica (NMO), which can also present as a primary disease, is remarkably responsive to rituximab, and there is a subset of lupus patients who have the NMO IgG/aquaporin-4 antibody that is associated with the disease, he said.

"If you’re looking at a patient with lupus who develops optic neuritis or if they develop transverse myelitis, you have to think about whether they are in a subgroup with these NMO antibodies," he said, noting that these patients will tend to have longitudinally extensive transverse myelitis that extends over three vertebral bodies, and they will tend to be positive for aquaporin-4.

"In this subgroup, we have moved to using rituximab early in the course of disease if [these patients] have been very refractory to other treatments," he said.

Dr. Looney said he also continues to use rituximab in severe refractory systemic lupus erythematosus, particularly in patients with central nervous system disease or lupus nephritis, as well as in patients with refractory idiopathic thrombocytopenic purpura (ITP).

Although rituximab is not approved for ITP, it is widely used as a secondary therapy for this condition, he said.

Dr. Looney disclosed that he has been an advisor for Genentech.

DESTIN, FLA. – Rituximab has a place in the treatment of certain subsets of patients with lupus, but generally the biologic agent has proved disappointing for this disease, according to Dr. R. John Looney.

The chimeric monoclonal antibody against the protein CD20 has worked very well in certain autoimmune diseases, and it is approved for a number of indications including refractory rheumatoid arthritis and antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis.

Rituximab is commonly used in relapsing and remitting multiple sclerosis, and early trial data suggest that the agent may have promise in the management of type 1 diabetes, said Dr. Looney, a professor of medicine at the University of Rochester (N.Y.).

In the "amazingly negative" EXPLORER (A Study to Evaluate the Efficacy and Safety of Rituximab in Patients With Severe Systemic Lupus Erythematosus) trial, however, overall clinical response rates did not differ significantly in rituximab- and placebo-treated nonrenal lupus patients (Arthritis Rheum. 2010;62:222-33).

And in the LUNAR (A Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis) trial, patients with lupus nephritis who were treated with rituximab had a modest improvement in proteinuria, compared with those who received placebo, but only a small, non–statistically significant improvement in overall renal outcomes of about 11%, compared with those who received placebo, according to data reported at conferences, including the annual conference of the American College of Rheumatology in 2009, Dr. Looney said.

"So rituximab has been a remarkably effective drug in certain types of autoimmune disease, but it has been a relative failure in lupus," he added.

An exception is in patients with antibodies associated with neuromyelitis optica, he said.

Neuromyelitis optica (NMO), which can also present as a primary disease, is remarkably responsive to rituximab, and there is a subset of lupus patients who have the NMO IgG/aquaporin-4 antibody that is associated with the disease, he said.

"If you’re looking at a patient with lupus who develops optic neuritis or if they develop transverse myelitis, you have to think about whether they are in a subgroup with these NMO antibodies," he said, noting that these patients will tend to have longitudinally extensive transverse myelitis that extends over three vertebral bodies, and they will tend to be positive for aquaporin-4.

"In this subgroup, we have moved to using rituximab early in the course of disease if [these patients] have been very refractory to other treatments," he said.

Dr. Looney said he also continues to use rituximab in severe refractory systemic lupus erythematosus, particularly in patients with central nervous system disease or lupus nephritis, as well as in patients with refractory idiopathic thrombocytopenic purpura (ITP).

Although rituximab is not approved for ITP, it is widely used as a secondary therapy for this condition, he said.

Dr. Looney disclosed that he has been an advisor for Genentech.

DESTIN, FLA. – Rituximab has a place in the treatment of certain subsets of patients with lupus, but generally the biologic agent has proved disappointing for this disease, according to Dr. R. John Looney.

The chimeric monoclonal antibody against the protein CD20 has worked very well in certain autoimmune diseases, and it is approved for a number of indications including refractory rheumatoid arthritis and antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis.

Rituximab is commonly used in relapsing and remitting multiple sclerosis, and early trial data suggest that the agent may have promise in the management of type 1 diabetes, said Dr. Looney, a professor of medicine at the University of Rochester (N.Y.).

In the "amazingly negative" EXPLORER (A Study to Evaluate the Efficacy and Safety of Rituximab in Patients With Severe Systemic Lupus Erythematosus) trial, however, overall clinical response rates did not differ significantly in rituximab- and placebo-treated nonrenal lupus patients (Arthritis Rheum. 2010;62:222-33).

And in the LUNAR (A Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis) trial, patients with lupus nephritis who were treated with rituximab had a modest improvement in proteinuria, compared with those who received placebo, but only a small, non–statistically significant improvement in overall renal outcomes of about 11%, compared with those who received placebo, according to data reported at conferences, including the annual conference of the American College of Rheumatology in 2009, Dr. Looney said.

"So rituximab has been a remarkably effective drug in certain types of autoimmune disease, but it has been a relative failure in lupus," he added.

An exception is in patients with antibodies associated with neuromyelitis optica, he said.

Neuromyelitis optica (NMO), which can also present as a primary disease, is remarkably responsive to rituximab, and there is a subset of lupus patients who have the NMO IgG/aquaporin-4 antibody that is associated with the disease, he said.

"If you’re looking at a patient with lupus who develops optic neuritis or if they develop transverse myelitis, you have to think about whether they are in a subgroup with these NMO antibodies," he said, noting that these patients will tend to have longitudinally extensive transverse myelitis that extends over three vertebral bodies, and they will tend to be positive for aquaporin-4.

"In this subgroup, we have moved to using rituximab early in the course of disease if [these patients] have been very refractory to other treatments," he said.

Dr. Looney said he also continues to use rituximab in severe refractory systemic lupus erythematosus, particularly in patients with central nervous system disease or lupus nephritis, as well as in patients with refractory idiopathic thrombocytopenic purpura (ITP).

Although rituximab is not approved for ITP, it is widely used as a secondary therapy for this condition, he said.

Dr. Looney disclosed that he has been an advisor for Genentech.

DESTIN, FLA. – Rituximab has a place in the treatment of certain subsets of patients with lupus, but generally the biologic agent has proved disappointing for this disease, according to Dr. R. John Looney.

The chimeric monoclonal antibody against the protein CD20 has worked very well in certain autoimmune diseases, and it is approved for a number of indications including refractory rheumatoid arthritis and antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis.

Rituximab is commonly used in relapsing and remitting multiple sclerosis, and early trial data suggest that the agent may have promise in the management of type 1 diabetes, said Dr. Looney, a professor of medicine at the University of Rochester (N.Y.).

In the "amazingly negative" EXPLORER (A Study to Evaluate the Efficacy and Safety of Rituximab in Patients With Severe Systemic Lupus Erythematosus) trial, however, overall clinical response rates did not differ significantly in rituximab- and placebo-treated nonrenal lupus patients (Arthritis Rheum. 2010;62:222-33).

And in the LUNAR (A Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis) trial, patients with lupus nephritis who were treated with rituximab had a modest improvement in proteinuria, compared with those who received placebo, but only a small, non–statistically significant improvement in overall renal outcomes of about 11%, compared with those who received placebo, according to data reported at conferences, including the annual conference of the American College of Rheumatology in 2009, Dr. Looney said.

"So rituximab has been a remarkably effective drug in certain types of autoimmune disease, but it has been a relative failure in lupus," he added.

An exception is in patients with antibodies associated with neuromyelitis optica, he said.

Neuromyelitis optica (NMO), which can also present as a primary disease, is remarkably responsive to rituximab, and there is a subset of lupus patients who have the NMO IgG/aquaporin-4 antibody that is associated with the disease, he said.

"If you’re looking at a patient with lupus who develops optic neuritis or if they develop transverse myelitis, you have to think about whether they are in a subgroup with these NMO antibodies," he said, noting that these patients will tend to have longitudinally extensive transverse myelitis that extends over three vertebral bodies, and they will tend to be positive for aquaporin-4.

"In this subgroup, we have moved to using rituximab early in the course of disease if [these patients] have been very refractory to other treatments," he said.

Dr. Looney said he also continues to use rituximab in severe refractory systemic lupus erythematosus, particularly in patients with central nervous system disease or lupus nephritis, as well as in patients with refractory idiopathic thrombocytopenic purpura (ITP).

Although rituximab is not approved for ITP, it is widely used as a secondary therapy for this condition, he said.

Dr. Looney disclosed that he has been an advisor for Genentech.

DESTIN, FLA. – Rituximab has a place in the treatment of certain subsets of patients with lupus, but generally the biologic agent has proved disappointing for this disease, according to Dr. R. John Looney.

The chimeric monoclonal antibody against the protein CD20 has worked very well in certain autoimmune diseases, and it is approved for a number of indications including refractory rheumatoid arthritis and antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis.

Rituximab is commonly used in relapsing and remitting multiple sclerosis, and early trial data suggest that the agent may have promise in the management of type 1 diabetes, said Dr. Looney, a professor of medicine at the University of Rochester (N.Y.).

In the "amazingly negative" EXPLORER (A Study to Evaluate the Efficacy and Safety of Rituximab in Patients With Severe Systemic Lupus Erythematosus) trial, however, overall clinical response rates did not differ significantly in rituximab- and placebo-treated nonrenal lupus patients (Arthritis Rheum. 2010;62:222-33).

And in the LUNAR (A Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis) trial, patients with lupus nephritis who were treated with rituximab had a modest improvement in proteinuria, compared with those who received placebo, but only a small, non–statistically significant improvement in overall renal outcomes of about 11%, compared with those who received placebo, according to data reported at conferences, including the annual conference of the American College of Rheumatology in 2009, Dr. Looney said.

"So rituximab has been a remarkably effective drug in certain types of autoimmune disease, but it has been a relative failure in lupus," he added.

An exception is in patients with antibodies associated with neuromyelitis optica, he said.

Neuromyelitis optica (NMO), which can also present as a primary disease, is remarkably responsive to rituximab, and there is a subset of lupus patients who have the NMO IgG/aquaporin-4 antibody that is associated with the disease, he said.

"If you’re looking at a patient with lupus who develops optic neuritis or if they develop transverse myelitis, you have to think about whether they are in a subgroup with these NMO antibodies," he said, noting that these patients will tend to have longitudinally extensive transverse myelitis that extends over three vertebral bodies, and they will tend to be positive for aquaporin-4.

"In this subgroup, we have moved to using rituximab early in the course of disease if [these patients] have been very refractory to other treatments," he said.

Dr. Looney said he also continues to use rituximab in severe refractory systemic lupus erythematosus, particularly in patients with central nervous system disease or lupus nephritis, as well as in patients with refractory idiopathic thrombocytopenic purpura (ITP).

Although rituximab is not approved for ITP, it is widely used as a secondary therapy for this condition, he said.

Dr. Looney disclosed that he has been an advisor for Genentech.

DESTIN, FLA. – Bosentan, which has been shown to prevent digital ulcers in systemic sclerosis, also may have beneficial effects on skin fibrosis.

In a small, prospective, open-label study, treatment with the dual endothelin receptor antagonist was associated with a significant reduction in skin thickening as measured by the modified Rodnan skin score (mRSS), Dr. Annegret Kuhn said at the Congress of Clinical Rheumatology.

The mean change from baseline in the mRSS in the 10 systemic sclerosis patients in the study was 6.4 points on the 0-51 point scale, said Dr. Kuhn of Westfälische Wilhelms-Universität Münster (Germany).

Significant responses were seen in patients with both diffuse and limited disease, and there also was significant healing of digital ulcers. No differences were seen from baseline and week 24, however, on 20-MHz ultrasound, fist-closure evaluation, U.K. systemic sclerosis functional score, or the modified sclerosis health assessment questionnaire (SHAQ) and its visual analogue scale (Rheumatology [Oxford] 2010;49:1336-45).

Patients were treated with 62.5 mg of bosentan twice daily for 4 weeks, then with 125 mg twice daily for 20 weeks. This regimen is similar to the dosing used in the RAPIDS-1 and -2 (Randomized Placebo-Controlled Study on Prevention of Ischemic Digital Ulcers in Systemic Scleroderma–1 and –2) trials, which demonstrated the drug's efficacy for the prevention of digital ulcers. Bosentan is currently approved for use in the United States for pulmonary artery hypertension, and – based on the RAPIDS-1 trial (Arthritis Rheum. 2004;50:3985-93) – it was also approved in Europe in 2007 for prevention of digital ulcers.

The RAPIDS-2 trial that was published this year (Ann. Rheum. Dis. 2011;70:32-8), included treatment for at least 24 weeks, and confirmed the preventive effects seen in RAPIDS-1. Bosentan was not found in either trial to be associated with improved healing in existing ulcers, however.

"Iloprost remains the best treatment for digital ulcers in systemic scleroderma," Dr. Kuhn said,

But bosentan is recommended for prevention, and it may also improve fibrosis, she noted.

Guidelines that were developed this year by a group of German experts on scleroderma call for first-line treatment with iloprost for the healing of existing ulcers, with repeated infusions for long-term care. They also call for the use of bosentan for prevention, and note that current experimental treatments include sildenafil and atorvastatin, she said.

Treatment of underlying disease (such as Raynaud's phenomenon, vasculopathy, and the like) is also important, as are general preventive measures including nicotine abstinence, cold protection, physical treatments, lymphatic draining, elimination of sympathomimetics, and vasoconstrictors.

Topical treatments can include antiseptic agents, becaplermin gel, hydrocolloid membrane, topical lidocaine, and vitamin E gel, according to the guidelines.

Dr. Kuhn had no disclosures to report.

DESTIN, FLA. – Bosentan, which has been shown to prevent digital ulcers in systemic sclerosis, also may have beneficial effects on skin fibrosis.

In a small, prospective, open-label study, treatment with the dual endothelin receptor antagonist was associated with a significant reduction in skin thickening as measured by the modified Rodnan skin score (mRSS), Dr. Annegret Kuhn said at the Congress of Clinical Rheumatology.

The mean change from baseline in the mRSS in the 10 systemic sclerosis patients in the study was 6.4 points on the 0-51 point scale, said Dr. Kuhn of Westfälische Wilhelms-Universität Münster (Germany).

Significant responses were seen in patients with both diffuse and limited disease, and there also was significant healing of digital ulcers. No differences were seen from baseline and week 24, however, on 20-MHz ultrasound, fist-closure evaluation, U.K. systemic sclerosis functional score, or the modified sclerosis health assessment questionnaire (SHAQ) and its visual analogue scale (Rheumatology [Oxford] 2010;49:1336-45).

Patients were treated with 62.5 mg of bosentan twice daily for 4 weeks, then with 125 mg twice daily for 20 weeks. This regimen is similar to the dosing used in the RAPIDS-1 and -2 (Randomized Placebo-Controlled Study on Prevention of Ischemic Digital Ulcers in Systemic Scleroderma–1 and –2) trials, which demonstrated the drug's efficacy for the prevention of digital ulcers. Bosentan is currently approved for use in the United States for pulmonary artery hypertension, and – based on the RAPIDS-1 trial (Arthritis Rheum. 2004;50:3985-93) – it was also approved in Europe in 2007 for prevention of digital ulcers.

The RAPIDS-2 trial that was published this year (Ann. Rheum. Dis. 2011;70:32-8), included treatment for at least 24 weeks, and confirmed the preventive effects seen in RAPIDS-1. Bosentan was not found in either trial to be associated with improved healing in existing ulcers, however.

"Iloprost remains the best treatment for digital ulcers in systemic scleroderma," Dr. Kuhn said,

But bosentan is recommended for prevention, and it may also improve fibrosis, she noted.

Guidelines that were developed this year by a group of German experts on scleroderma call for first-line treatment with iloprost for the healing of existing ulcers, with repeated infusions for long-term care. They also call for the use of bosentan for prevention, and note that current experimental treatments include sildenafil and atorvastatin, she said.

Treatment of underlying disease (such as Raynaud's phenomenon, vasculopathy, and the like) is also important, as are general preventive measures including nicotine abstinence, cold protection, physical treatments, lymphatic draining, elimination of sympathomimetics, and vasoconstrictors.

Topical treatments can include antiseptic agents, becaplermin gel, hydrocolloid membrane, topical lidocaine, and vitamin E gel, according to the guidelines.

Dr. Kuhn had no disclosures to report.

DESTIN, FLA. – Bosentan, which has been shown to prevent digital ulcers in systemic sclerosis, also may have beneficial effects on skin fibrosis.

In a small, prospective, open-label study, treatment with the dual endothelin receptor antagonist was associated with a significant reduction in skin thickening as measured by the modified Rodnan skin score (mRSS), Dr. Annegret Kuhn said at the Congress of Clinical Rheumatology.

The mean change from baseline in the mRSS in the 10 systemic sclerosis patients in the study was 6.4 points on the 0-51 point scale, said Dr. Kuhn of Westfälische Wilhelms-Universität Münster (Germany).

Significant responses were seen in patients with both diffuse and limited disease, and there also was significant healing of digital ulcers. No differences were seen from baseline and week 24, however, on 20-MHz ultrasound, fist-closure evaluation, U.K. systemic sclerosis functional score, or the modified sclerosis health assessment questionnaire (SHAQ) and its visual analogue scale (Rheumatology [Oxford] 2010;49:1336-45).

Patients were treated with 62.5 mg of bosentan twice daily for 4 weeks, then with 125 mg twice daily for 20 weeks. This regimen is similar to the dosing used in the RAPIDS-1 and -2 (Randomized Placebo-Controlled Study on Prevention of Ischemic Digital Ulcers in Systemic Scleroderma–1 and –2) trials, which demonstrated the drug's efficacy for the prevention of digital ulcers. Bosentan is currently approved for use in the United States for pulmonary artery hypertension, and – based on the RAPIDS-1 trial (Arthritis Rheum. 2004;50:3985-93) – it was also approved in Europe in 2007 for prevention of digital ulcers.

The RAPIDS-2 trial that was published this year (Ann. Rheum. Dis. 2011;70:32-8), included treatment for at least 24 weeks, and confirmed the preventive effects seen in RAPIDS-1. Bosentan was not found in either trial to be associated with improved healing in existing ulcers, however.

"Iloprost remains the best treatment for digital ulcers in systemic scleroderma," Dr. Kuhn said,

But bosentan is recommended for prevention, and it may also improve fibrosis, she noted.

Guidelines that were developed this year by a group of German experts on scleroderma call for first-line treatment with iloprost for the healing of existing ulcers, with repeated infusions for long-term care. They also call for the use of bosentan for prevention, and note that current experimental treatments include sildenafil and atorvastatin, she said.

Treatment of underlying disease (such as Raynaud's phenomenon, vasculopathy, and the like) is also important, as are general preventive measures including nicotine abstinence, cold protection, physical treatments, lymphatic draining, elimination of sympathomimetics, and vasoconstrictors.

Topical treatments can include antiseptic agents, becaplermin gel, hydrocolloid membrane, topical lidocaine, and vitamin E gel, according to the guidelines.

Dr. Kuhn had no disclosures to report.

DESTIN, FLA. – Bosentan, which has been shown to prevent digital ulcers in systemic sclerosis, also may have beneficial effects on skin fibrosis.

In a small, prospective, open-label study, treatment with the dual endothelin receptor antagonist was associated with a significant reduction in skin thickening as measured by the modified Rodnan skin score (mRSS), Dr. Annegret Kuhn said at the Congress of Clinical Rheumatology.

The mean change from baseline in the mRSS in the 10 systemic sclerosis patients in the study was 6.4 points on the 0-51 point scale, said Dr. Kuhn of Westfälische Wilhelms-Universität Münster (Germany).

Significant responses were seen in patients with both diffuse and limited disease, and there also was significant healing of digital ulcers. No differences were seen from baseline and week 24, however, on 20-MHz ultrasound, fist-closure evaluation, U.K. systemic sclerosis functional score, or the modified sclerosis health assessment questionnaire (SHAQ) and its visual analogue scale (Rheumatology [Oxford] 2010;49:1336-45).

Patients were treated with 62.5 mg of bosentan twice daily for 4 weeks, then with 125 mg twice daily for 20 weeks. This regimen is similar to the dosing used in the RAPIDS-1 and -2 (Randomized Placebo-Controlled Study on Prevention of Ischemic Digital Ulcers in Systemic Scleroderma–1 and –2) trials, which demonstrated the drug’s efficacy for the prevention of digital ulcers. Bosentan is currently approved for use in the United States for pulmonary artery hypertension, and – based on the RAPIDS-1 trial (Arthritis Rheum. 2004;50:3985-93) – it was also approved in Europe in 2007 for prevention of digital ulcers.

The RAPIDS-2 trial that was published this year (Ann. Rheum. Dis. 2011;70:32-8), included treatment for at least 24 weeks, and confirmed the preventive effects seen in RAPIDS-1. Bosentan was not found in either trial to be associated with improved healing in existing ulcers, however.

"Iloprost remains the best treatment for digital ulcers in systemic scleroderma," Dr. Kuhn said,

But bosentan is recommended for prevention, and it may also improve fibrosis, she noted.

Guidelines that were developed this year by a group of German experts on scleroderma call for first-line treatment with iloprost for the healing of existing ulcers, with repeated infusions for long-term care. They also call for the use of bosentan for prevention, and note that current experimental treatments include sildenafil and atorvastatin, she said.

Treatment of underlying disease (such as Raynaud’s phenomenon, vasculopathy, and the like) is also important, as are general preventive measures including nicotine abstinence, cold protection, physical treatments, lymphatic draining, elimination of sympathomimetics, and vasoconstrictors.

Topical treatments can include antiseptic agents, becaplermin gel, hydrocolloid membrane, topical lidocaine, and vitamin E gel, according to the guidelines.

Dr. Kuhn had no disclosures to report.

DESTIN, FLA. – Bosentan, which has been shown to prevent digital ulcers in systemic sclerosis, also may have beneficial effects on skin fibrosis.

In a small, prospective, open-label study, treatment with the dual endothelin receptor antagonist was associated with a significant reduction in skin thickening as measured by the modified Rodnan skin score (mRSS), Dr. Annegret Kuhn said at the Congress of Clinical Rheumatology.

The mean change from baseline in the mRSS in the 10 systemic sclerosis patients in the study was 6.4 points on the 0-51 point scale, said Dr. Kuhn of Westfälische Wilhelms-Universität Münster (Germany).

Significant responses were seen in patients with both diffuse and limited disease, and there also was significant healing of digital ulcers. No differences were seen from baseline and week 24, however, on 20-MHz ultrasound, fist-closure evaluation, U.K. systemic sclerosis functional score, or the modified sclerosis health assessment questionnaire (SHAQ) and its visual analogue scale (Rheumatology [Oxford] 2010;49:1336-45).

Patients were treated with 62.5 mg of bosentan twice daily for 4 weeks, then with 125 mg twice daily for 20 weeks. This regimen is similar to the dosing used in the RAPIDS-1 and -2 (Randomized Placebo-Controlled Study on Prevention of Ischemic Digital Ulcers in Systemic Scleroderma–1 and –2) trials, which demonstrated the drug’s efficacy for the prevention of digital ulcers. Bosentan is currently approved for use in the United States for pulmonary artery hypertension, and – based on the RAPIDS-1 trial (Arthritis Rheum. 2004;50:3985-93) – it was also approved in Europe in 2007 for prevention of digital ulcers.

The RAPIDS-2 trial that was published this year (Ann. Rheum. Dis. 2011;70:32-8), included treatment for at least 24 weeks, and confirmed the preventive effects seen in RAPIDS-1. Bosentan was not found in either trial to be associated with improved healing in existing ulcers, however.

"Iloprost remains the best treatment for digital ulcers in systemic scleroderma," Dr. Kuhn said,

But bosentan is recommended for prevention, and it may also improve fibrosis, she noted.

Guidelines that were developed this year by a group of German experts on scleroderma call for first-line treatment with iloprost for the healing of existing ulcers, with repeated infusions for long-term care. They also call for the use of bosentan for prevention, and note that current experimental treatments include sildenafil and atorvastatin, she said.

Treatment of underlying disease (such as Raynaud’s phenomenon, vasculopathy, and the like) is also important, as are general preventive measures including nicotine abstinence, cold protection, physical treatments, lymphatic draining, elimination of sympathomimetics, and vasoconstrictors.

Topical treatments can include antiseptic agents, becaplermin gel, hydrocolloid membrane, topical lidocaine, and vitamin E gel, according to the guidelines.

Dr. Kuhn had no disclosures to report.

DESTIN, FLA. – Bosentan, which has been shown to prevent digital ulcers in systemic sclerosis, also may have beneficial effects on skin fibrosis.

In a small, prospective, open-label study, treatment with the dual endothelin receptor antagonist was associated with a significant reduction in skin thickening as measured by the modified Rodnan skin score (mRSS), Dr. Annegret Kuhn said at the Congress of Clinical Rheumatology.

The mean change from baseline in the mRSS in the 10 systemic sclerosis patients in the study was 6.4 points on the 0-51 point scale, said Dr. Kuhn of Westfälische Wilhelms-Universität Münster (Germany).

Significant responses were seen in patients with both diffuse and limited disease, and there also was significant healing of digital ulcers. No differences were seen from baseline and week 24, however, on 20-MHz ultrasound, fist-closure evaluation, U.K. systemic sclerosis functional score, or the modified sclerosis health assessment questionnaire (SHAQ) and its visual analogue scale (Rheumatology [Oxford] 2010;49:1336-45).

Patients were treated with 62.5 mg of bosentan twice daily for 4 weeks, then with 125 mg twice daily for 20 weeks. This regimen is similar to the dosing used in the RAPIDS-1 and -2 (Randomized Placebo-Controlled Study on Prevention of Ischemic Digital Ulcers in Systemic Scleroderma–1 and –2) trials, which demonstrated the drug’s efficacy for the prevention of digital ulcers. Bosentan is currently approved for use in the United States for pulmonary artery hypertension, and – based on the RAPIDS-1 trial (Arthritis Rheum. 2004;50:3985-93) – it was also approved in Europe in 2007 for prevention of digital ulcers.

The RAPIDS-2 trial that was published this year (Ann. Rheum. Dis. 2011;70:32-8), included treatment for at least 24 weeks, and confirmed the preventive effects seen in RAPIDS-1. Bosentan was not found in either trial to be associated with improved healing in existing ulcers, however.

"Iloprost remains the best treatment for digital ulcers in systemic scleroderma," Dr. Kuhn said,

But bosentan is recommended for prevention, and it may also improve fibrosis, she noted.

Guidelines that were developed this year by a group of German experts on scleroderma call for first-line treatment with iloprost for the healing of existing ulcers, with repeated infusions for long-term care. They also call for the use of bosentan for prevention, and note that current experimental treatments include sildenafil and atorvastatin, she said.

Treatment of underlying disease (such as Raynaud’s phenomenon, vasculopathy, and the like) is also important, as are general preventive measures including nicotine abstinence, cold protection, physical treatments, lymphatic draining, elimination of sympathomimetics, and vasoconstrictors.

Topical treatments can include antiseptic agents, becaplermin gel, hydrocolloid membrane, topical lidocaine, and vitamin E gel, according to the guidelines.

Dr. Kuhn had no disclosures to report.

DESTIN, FLA. – Bosentan, which has been shown to prevent digital ulcers in systemic sclerosis, also may have beneficial effects on skin fibrosis.

In a small, prospective, open-label study, treatment with the dual endothelin receptor antagonist was associated with a significant reduction in skin thickening as measured by the modified Rodnan skin score (mRSS), Dr. Annegret Kuhn said at the Congress of Clinical Rheumatology.

The mean change from baseline in the mRSS in the 10 systemic sclerosis patients in the study was 6.4 points on the 0-51 point scale, said Dr. Kuhn of Westfälische Wilhelms-Universität Münster (Germany).

Significant responses were seen in patients with both diffuse and limited disease, and there also was significant healing of digital ulcers. No differences were seen from baseline and week 24, however, on 20-MHz ultrasound, fist-closure evaluation, U.K. systemic sclerosis functional score, or the modified sclerosis health assessment questionnaire (SHAQ) and its visual analogue scale (Rheumatology [Oxford] 2010;49:1336-45).

Patients were treated with 62.5 mg of bosentan twice daily for 4 weeks, then with 125 mg twice daily for 20 weeks. This regimen is similar to the dosing used in the RAPIDS-1 and -2 (Randomized Placebo-Controlled Study on Prevention of Ischemic Digital Ulcers in Systemic Scleroderma–1 and –2) trials, which demonstrated the drug’s efficacy for the prevention of digital ulcers. Bosentan is currently approved for use in the United States for pulmonary artery hypertension, and – based on the RAPIDS-1 trial (Arthritis Rheum. 2004;50:3985-93) – it was also approved in Europe in 2007 for prevention of digital ulcers.

The RAPIDS-2 trial that was published this year (Ann. Rheum. Dis. 2011;70:32-8), included treatment for at least 24 weeks, and confirmed the preventive effects seen in RAPIDS-1. Bosentan was not found in either trial to be associated with improved healing in existing ulcers, however.

"Iloprost remains the best treatment for digital ulcers in systemic scleroderma," Dr. Kuhn said,

But bosentan is recommended for prevention, and it may also improve fibrosis, she noted.

Guidelines that were developed this year by a group of German experts on scleroderma call for first-line treatment with iloprost for the healing of existing ulcers, with repeated infusions for long-term care. They also call for the use of bosentan for prevention, and note that current experimental treatments include sildenafil and atorvastatin, she said.

Treatment of underlying disease (such as Raynaud’s phenomenon, vasculopathy, and the like) is also important, as are general preventive measures including nicotine abstinence, cold protection, physical treatments, lymphatic draining, elimination of sympathomimetics, and vasoconstrictors.

Topical treatments can include antiseptic agents, becaplermin gel, hydrocolloid membrane, topical lidocaine, and vitamin E gel, according to the guidelines.

Dr. Kuhn had no disclosures to report.

DESTIN, FLA. – Bosentan, which has been shown to prevent digital ulcers in systemic sclerosis, also may have beneficial effects on skin fibrosis.

In a small, prospective, open-label study, treatment with the dual endothelin receptor antagonist was associated with a significant reduction in skin thickening as measured by the modified Rodnan skin score (mRSS), Dr. Annegret Kuhn said at the Congress of Clinical Rheumatology.

The mean change from baseline in the mRSS in the 10 systemic sclerosis patients in the study was 6.4 points on the 0-51 point scale, said Dr. Kuhn of Westfälische Wilhelms-Universität Münster (Germany).

Significant responses were seen in patients with both diffuse and limited disease, and there also was significant healing of digital ulcers. No differences were seen from baseline and week 24, however, on 20-MHz ultrasound, fist-closure evaluation, U.K. systemic sclerosis functional score, or the modified sclerosis health assessment questionnaire (SHAQ) and its visual analogue scale (Rheumatology [Oxford] 2010;49:1336-45).

Patients were treated with 62.5 mg of bosentan twice daily for 4 weeks, then with 125 mg twice daily for 20 weeks. This regimen is similar to the dosing used in the RAPIDS-1 and -2 (Randomized Placebo-Controlled Study on Prevention of Ischemic Digital Ulcers in Systemic Scleroderma–1 and –2) trials, which demonstrated the drug’s efficacy for the prevention of digital ulcers. Bosentan is currently approved for use in the United States for pulmonary artery hypertension, and – based on the RAPIDS-1 trial (Arthritis Rheum. 2004;50:3985-93) – it was also approved in Europe in 2007 for prevention of digital ulcers.

The RAPIDS-2 trial that was published this year (Ann. Rheum. Dis. 2011;70:32-8), included treatment for at least 24 weeks, and confirmed the preventive effects seen in RAPIDS-1. Bosentan was not found in either trial to be associated with improved healing in existing ulcers, however.

"Iloprost remains the best treatment for digital ulcers in systemic scleroderma," Dr. Kuhn said,

But bosentan is recommended for prevention, and it may also improve fibrosis, she noted.

Guidelines that were developed this year by a group of German experts on scleroderma call for first-line treatment with iloprost for the healing of existing ulcers, with repeated infusions for long-term care. They also call for the use of bosentan for prevention, and note that current experimental treatments include sildenafil and atorvastatin, she said.

Treatment of underlying disease (such as Raynaud’s phenomenon, vasculopathy, and the like) is also important, as are general preventive measures including nicotine abstinence, cold protection, physical treatments, lymphatic draining, elimination of sympathomimetics, and vasoconstrictors.

Topical treatments can include antiseptic agents, becaplermin gel, hydrocolloid membrane, topical lidocaine, and vitamin E gel, according to the guidelines.

Dr. Kuhn had no disclosures to report.

DESTIN, FLA. – Bosentan, which has been shown to prevent digital ulcers in systemic sclerosis, also may have beneficial effects on skin fibrosis.

In a small, prospective, open-label study, treatment with the dual endothelin receptor antagonist was associated with a significant reduction in skin thickening as measured by the modified Rodnan skin score (mRSS), Dr. Annegret Kuhn said at the Congress of Clinical Rheumatology.

The mean change from baseline in the mRSS in the 10 systemic sclerosis patients in the study was 6.4 points on the 0-51 point scale, said Dr. Kuhn of Westfälische Wilhelms-Universität Münster (Germany).

Significant responses were seen in patients with both diffuse and limited disease, and there also was significant healing of digital ulcers. No differences were seen from baseline and week 24, however, on 20-MHz ultrasound, fist-closure evaluation, U.K. systemic sclerosis functional score, or the modified sclerosis health assessment questionnaire (SHAQ) and its visual analogue scale (Rheumatology [Oxford] 2010;49:1336-45).

Patients were treated with 62.5 mg of bosentan twice daily for 4 weeks, then with 125 mg twice daily for 20 weeks. This regimen is similar to the dosing used in the RAPIDS-1 and -2 (Randomized Placebo-Controlled Study on Prevention of Ischemic Digital Ulcers in Systemic Scleroderma–1 and –2) trials, which demonstrated the drug’s efficacy for the prevention of digital ulcers. Bosentan is currently approved for use in the United States for pulmonary artery hypertension, and – based on the RAPIDS-1 trial (Arthritis Rheum. 2004;50:3985-93) – it was also approved in Europe in 2007 for prevention of digital ulcers.

The RAPIDS-2 trial that was published this year (Ann. Rheum. Dis. 2011;70:32-8), included treatment for at least 24 weeks, and confirmed the preventive effects seen in RAPIDS-1. Bosentan was not found in either trial to be associated with improved healing in existing ulcers, however.

"Iloprost remains the best treatment for digital ulcers in systemic scleroderma," Dr. Kuhn said,

But bosentan is recommended for prevention, and it may also improve fibrosis, she noted.

Guidelines that were developed this year by a group of German experts on scleroderma call for first-line treatment with iloprost for the healing of existing ulcers, with repeated infusions for long-term care. They also call for the use of bosentan for prevention, and note that current experimental treatments include sildenafil and atorvastatin, she said.

Treatment of underlying disease (such as Raynaud’s phenomenon, vasculopathy, and the like) is also important, as are general preventive measures including nicotine abstinence, cold protection, physical treatments, lymphatic draining, elimination of sympathomimetics, and vasoconstrictors.

Topical treatments can include antiseptic agents, becaplermin gel, hydrocolloid membrane, topical lidocaine, and vitamin E gel, according to the guidelines.

Dr. Kuhn had no disclosures to report.

DESTIN, FLA. – For now, the lupus patients most likely to respond to recently approved belimumab may be those with moderate to severe disease without renal or central nervous system involvement who have failed to respond to standard therapy, according to Dr. R. John Looney.

More specific details on which patients are most likely to benefit will require further experience with the drug, he said at the Congress of Clinical Rheumatology.

Belimumab (Benlysta), which was approved in March, is the first new drug for systemic lupus erythematosus (SLE) in 50 years. It has a very good safety profile, although the effect size, while statistically significant, is relatively small, with only an average of about a 12 percentage point difference in the response rates between treatment and placebo in trials of the fully human monoclonal antibody that inhibits B-lymphocyte stimulator, said Dr. Looney, professor of medicine at the University of Rochester (N.Y.).

Still, that reflects improvement of about 25%-30%, he noted.

The standard dosage is 10 mg/kg, given intravenously every 2 weeks for three times, then every 4 weeks, in addition to standard care.

Keep in mind that the onset is somewhat slow, so this "may not be a drug you would use in a flaring patient," Dr. Looney said.

Also, it has not been studied in severe renal or central nervous system disease, nor in combination with cyclophosphamide, he noted.

Furthermore, no particular subgroup has yet been identified in whom the drug has particular benefits, so it is a bit of a challenge to determine who should be put on this drug.

"I would consider it in patients with moderate to severe, nonrenal, nonneurologic disease who have failed standard therapy," Dr. Looney said, explaining that he would consider those to be patients with continued disease activity or patients in whom steroids can’t be tapered or stopped despite treatment.

"So I look at this as a drug that might help me get my patients who are on hydroxychloroquine and let’s say CellCept [mycophenolate] or Imuran [azathioprine], down to a manageable dose of steroids," he added.

Determining how long to treat, however, is a challenge.

"We have no good way to look at an individual patient and say they are better and they are better because they are on belimumab," Dr. Looney said, noting that based on belimumab trial data, three out of four patients who were responders at 1 year would have been responders on placebo, so further study is needed to help guide treatment duration.

"I would also really like to see predictors of response," he said, noting that such predictors have thus far not been reported.

Further analysis of existing data may be useful for identifying subgroups who will respond best to belimumab. Additional studies in renal and CNS disease would also be useful.

"It may be that you add this drug to current therapies [in renal and CNS patients] and they do that much better," Dr. Looney said, noting that it would also be useful to look at belimumab treatment in conjunction with cyclophosphamide and rituximab as well "to see what happens when B cells are targeted in two different ways."

Dr. Looney disclosed that he has been an adviser for Genentech.

DESTIN, FLA. – For now, the lupus patients most likely to respond to recently approved belimumab may be those with moderate to severe disease without renal or central nervous system involvement who have failed to respond to standard therapy, according to Dr. R. John Looney.

More specific details on which patients are most likely to benefit will require further experience with the drug, he said at the Congress of Clinical Rheumatology.

Belimumab (Benlysta), which was approved in March, is the first new drug for systemic lupus erythematosus (SLE) in 50 years. It has a very good safety profile, although the effect size, while statistically significant, is relatively small, with only an average of about a 12 percentage point difference in the response rates between treatment and placebo in trials of the fully human monoclonal antibody that inhibits B-lymphocyte stimulator, said Dr. Looney, professor of medicine at the University of Rochester (N.Y.).

Still, that reflects improvement of about 25%-30%, he noted.

The standard dosage is 10 mg/kg, given intravenously every 2 weeks for three times, then every 4 weeks, in addition to standard care.

Keep in mind that the onset is somewhat slow, so this "may not be a drug you would use in a flaring patient," Dr. Looney said.

Also, it has not been studied in severe renal or central nervous system disease, nor in combination with cyclophosphamide, he noted.

Furthermore, no particular subgroup has yet been identified in whom the drug has particular benefits, so it is a bit of a challenge to determine who should be put on this drug.

"I would consider it in patients with moderate to severe, nonrenal, nonneurologic disease who have failed standard therapy," Dr. Looney said, explaining that he would consider those to be patients with continued disease activity or patients in whom steroids can’t be tapered or stopped despite treatment.

"So I look at this as a drug that might help me get my patients who are on hydroxychloroquine and let’s say CellCept [mycophenolate] or Imuran [azathioprine], down to a manageable dose of steroids," he added.

Determining how long to treat, however, is a challenge.

"We have no good way to look at an individual patient and say they are better and they are better because they are on belimumab," Dr. Looney said, noting that based on belimumab trial data, three out of four patients who were responders at 1 year would have been responders on placebo, so further study is needed to help guide treatment duration.

"I would also really like to see predictors of response," he said, noting that such predictors have thus far not been reported.

Further analysis of existing data may be useful for identifying subgroups who will respond best to belimumab. Additional studies in renal and CNS disease would also be useful.

"It may be that you add this drug to current therapies [in renal and CNS patients] and they do that much better," Dr. Looney said, noting that it would also be useful to look at belimumab treatment in conjunction with cyclophosphamide and rituximab as well "to see what happens when B cells are targeted in two different ways."

Dr. Looney disclosed that he has been an adviser for Genentech.

DESTIN, FLA. – For now, the lupus patients most likely to respond to recently approved belimumab may be those with moderate to severe disease without renal or central nervous system involvement who have failed to respond to standard therapy, according to Dr. R. John Looney.

More specific details on which patients are most likely to benefit will require further experience with the drug, he said at the Congress of Clinical Rheumatology.

Belimumab (Benlysta), which was approved in March, is the first new drug for systemic lupus erythematosus (SLE) in 50 years. It has a very good safety profile, although the effect size, while statistically significant, is relatively small, with only an average of about a 12 percentage point difference in the response rates between treatment and placebo in trials of the fully human monoclonal antibody that inhibits B-lymphocyte stimulator, said Dr. Looney, professor of medicine at the University of Rochester (N.Y.).

Still, that reflects improvement of about 25%-30%, he noted.

The standard dosage is 10 mg/kg, given intravenously every 2 weeks for three times, then every 4 weeks, in addition to standard care.

Keep in mind that the onset is somewhat slow, so this "may not be a drug you would use in a flaring patient," Dr. Looney said.

Also, it has not been studied in severe renal or central nervous system disease, nor in combination with cyclophosphamide, he noted.

Furthermore, no particular subgroup has yet been identified in whom the drug has particular benefits, so it is a bit of a challenge to determine who should be put on this drug.

"I would consider it in patients with moderate to severe, nonrenal, nonneurologic disease who have failed standard therapy," Dr. Looney said, explaining that he would consider those to be patients with continued disease activity or patients in whom steroids can’t be tapered or stopped despite treatment.

"So I look at this as a drug that might help me get my patients who are on hydroxychloroquine and let’s say CellCept [mycophenolate] or Imuran [azathioprine], down to a manageable dose of steroids," he added.

Determining how long to treat, however, is a challenge.

"We have no good way to look at an individual patient and say they are better and they are better because they are on belimumab," Dr. Looney said, noting that based on belimumab trial data, three out of four patients who were responders at 1 year would have been responders on placebo, so further study is needed to help guide treatment duration.

"I would also really like to see predictors of response," he said, noting that such predictors have thus far not been reported.

Further analysis of existing data may be useful for identifying subgroups who will respond best to belimumab. Additional studies in renal and CNS disease would also be useful.

"It may be that you add this drug to current therapies [in renal and CNS patients] and they do that much better," Dr. Looney said, noting that it would also be useful to look at belimumab treatment in conjunction with cyclophosphamide and rituximab as well "to see what happens when B cells are targeted in two different ways."

Dr. Looney disclosed that he has been an adviser for Genentech.

DESTIN, FLA. – For now, the lupus patients most likely to respond to recently approved belimumab may be those with moderate to severe disease without renal or central nervous system involvement who have failed to respond to standard therapy, according to Dr. R. John Looney.

More specific details on which patients are most likely to benefit will require further experience with the drug, he said at the Congress of Clinical Rheumatology.

Belimumab (Benlysta), which was approved in March, is the first new drug for systemic lupus erythematosus (SLE) in 50 years. It has a very good safety profile, although the effect size, while statistically significant, is relatively small, with only an average of about a 12 percentage point difference in the response rates between treatment and placebo in trials of the fully human monoclonal antibody that inhibits B-lymphocyte stimulator, said Dr. Looney, professor of medicine at the University of Rochester (N.Y.).

Still, that reflects improvement of about 25%-30%, he noted.

The standard dosage is 10 mg/kg, given intravenously every 2 weeks for three times, then every 4 weeks, in addition to standard care.

Keep in mind that the onset is somewhat slow, so this "may not be a drug you would use in a flaring patient," Dr. Looney said.

Also, it has not been studied in severe renal or central nervous system disease, nor in combination with cyclophosphamide, he noted.

Furthermore, no particular subgroup has yet been identified in whom the drug has particular benefits, so it is a bit of a challenge to determine who should be put on this drug.

"I would consider it in patients with moderate to severe, nonrenal, nonneurologic disease who have failed standard therapy," Dr. Looney said, explaining that he would consider those to be patients with continued disease activity or patients in whom steroids can’t be tapered or stopped despite treatment.

"So I look at this as a drug that might help me get my patients who are on hydroxychloroquine and let’s say CellCept [mycophenolate] or Imuran [azathioprine], down to a manageable dose of steroids," he added.

Determining how long to treat, however, is a challenge.

"We have no good way to look at an individual patient and say they are better and they are better because they are on belimumab," Dr. Looney said, noting that based on belimumab trial data, three out of four patients who were responders at 1 year would have been responders on placebo, so further study is needed to help guide treatment duration.

"I would also really like to see predictors of response," he said, noting that such predictors have thus far not been reported.

Further analysis of existing data may be useful for identifying subgroups who will respond best to belimumab. Additional studies in renal and CNS disease would also be useful.

"It may be that you add this drug to current therapies [in renal and CNS patients] and they do that much better," Dr. Looney said, noting that it would also be useful to look at belimumab treatment in conjunction with cyclophosphamide and rituximab as well "to see what happens when B cells are targeted in two different ways."

Dr. Looney disclosed that he has been an adviser for Genentech.

DESTIN, FLA. – For now, the lupus patients most likely to respond to recently approved belimumab may be those with moderate to severe disease without renal or central nervous system involvement who have failed to respond to standard therapy, according to Dr. R. John Looney.

More specific details on which patients are most likely to benefit will require further experience with the drug, he said at the Congress of Clinical Rheumatology.

Belimumab (Benlysta), which was approved in March, is the first new drug for systemic lupus erythematosus (SLE) in 50 years. It has a very good safety profile, although the effect size, while statistically significant, is relatively small, with only an average of about a 12 percentage point difference in the response rates between treatment and placebo in trials of the fully human monoclonal antibody that inhibits B-lymphocyte stimulator, said Dr. Looney, professor of medicine at the University of Rochester (N.Y.).

Still, that reflects improvement of about 25%-30%, he noted.

The standard dosage is 10 mg/kg, given intravenously every 2 weeks for three times, then every 4 weeks, in addition to standard care.

Keep in mind that the onset is somewhat slow, so this "may not be a drug you would use in a flaring patient," Dr. Looney said.

Also, it has not been studied in severe renal or central nervous system disease, nor in combination with cyclophosphamide, he noted.

Furthermore, no particular subgroup has yet been identified in whom the drug has particular benefits, so it is a bit of a challenge to determine who should be put on this drug.

"I would consider it in patients with moderate to severe, nonrenal, nonneurologic disease who have failed standard therapy," Dr. Looney said, explaining that he would consider those to be patients with continued disease activity or patients in whom steroids can’t be tapered or stopped despite treatment.

"So I look at this as a drug that might help me get my patients who are on hydroxychloroquine and let’s say CellCept [mycophenolate] or Imuran [azathioprine], down to a manageable dose of steroids," he added.

Determining how long to treat, however, is a challenge.

"We have no good way to look at an individual patient and say they are better and they are better because they are on belimumab," Dr. Looney said, noting that based on belimumab trial data, three out of four patients who were responders at 1 year would have been responders on placebo, so further study is needed to help guide treatment duration.

"I would also really like to see predictors of response," he said, noting that such predictors have thus far not been reported.

Further analysis of existing data may be useful for identifying subgroups who will respond best to belimumab. Additional studies in renal and CNS disease would also be useful.

"It may be that you add this drug to current therapies [in renal and CNS patients] and they do that much better," Dr. Looney said, noting that it would also be useful to look at belimumab treatment in conjunction with cyclophosphamide and rituximab as well "to see what happens when B cells are targeted in two different ways."

Dr. Looney disclosed that he has been an adviser for Genentech.

DESTIN, FLA. – For now, the lupus patients most likely to respond to recently approved belimumab may be those with moderate to severe disease without renal or central nervous system involvement who have failed to respond to standard therapy, according to Dr. R. John Looney.

More specific details on which patients are most likely to benefit will require further experience with the drug, he said at the Congress of Clinical Rheumatology.

Belimumab (Benlysta), which was approved in March, is the first new drug for systemic lupus erythematosus (SLE) in 50 years. It has a very good safety profile, although the effect size, while statistically significant, is relatively small, with only an average of about a 12 percentage point difference in the response rates between treatment and placebo in trials of the fully human monoclonal antibody that inhibits B-lymphocyte stimulator, said Dr. Looney, professor of medicine at the University of Rochester (N.Y.).

Still, that reflects improvement of about 25%-30%, he noted.

The standard dosage is 10 mg/kg, given intravenously every 2 weeks for three times, then every 4 weeks, in addition to standard care.

Keep in mind that the onset is somewhat slow, so this "may not be a drug you would use in a flaring patient," Dr. Looney said.

Also, it has not been studied in severe renal or central nervous system disease, nor in combination with cyclophosphamide, he noted.

Furthermore, no particular subgroup has yet been identified in whom the drug has particular benefits, so it is a bit of a challenge to determine who should be put on this drug.

"I would consider it in patients with moderate to severe, nonrenal, nonneurologic disease who have failed standard therapy," Dr. Looney said, explaining that he would consider those to be patients with continued disease activity or patients in whom steroids can’t be tapered or stopped despite treatment.

"So I look at this as a drug that might help me get my patients who are on hydroxychloroquine and let’s say CellCept [mycophenolate] or Imuran [azathioprine], down to a manageable dose of steroids," he added.

Determining how long to treat, however, is a challenge.

"We have no good way to look at an individual patient and say they are better and they are better because they are on belimumab," Dr. Looney said, noting that based on belimumab trial data, three out of four patients who were responders at 1 year would have been responders on placebo, so further study is needed to help guide treatment duration.

"I would also really like to see predictors of response," he said, noting that such predictors have thus far not been reported.

Further analysis of existing data may be useful for identifying subgroups who will respond best to belimumab. Additional studies in renal and CNS disease would also be useful.

"It may be that you add this drug to current therapies [in renal and CNS patients] and they do that much better," Dr. Looney said, noting that it would also be useful to look at belimumab treatment in conjunction with cyclophosphamide and rituximab as well "to see what happens when B cells are targeted in two different ways."

Dr. Looney disclosed that he has been an adviser for Genentech.

DESTIN, FLA. – For now, the lupus patients most likely to respond to recently approved belimumab may be those with moderate to severe disease without renal or central nervous system involvement who have failed to respond to standard therapy, according to Dr. R. John Looney.

More specific details on which patients are most likely to benefit will require further experience with the drug, he said at the Congress of Clinical Rheumatology.

Belimumab (Benlysta), which was approved in March, is the first new drug for systemic lupus erythematosus (SLE) in 50 years. It has a very good safety profile, although the effect size, while statistically significant, is relatively small, with only an average of about a 12 percentage point difference in the response rates between treatment and placebo in trials of the fully human monoclonal antibody that inhibits B-lymphocyte stimulator, said Dr. Looney, professor of medicine at the University of Rochester (N.Y.).

Still, that reflects improvement of about 25%-30%, he noted.

The standard dosage is 10 mg/kg, given intravenously every 2 weeks for three times, then every 4 weeks, in addition to standard care.

Keep in mind that the onset is somewhat slow, so this "may not be a drug you would use in a flaring patient," Dr. Looney said.

Also, it has not been studied in severe renal or central nervous system disease, nor in combination with cyclophosphamide, he noted.

Furthermore, no particular subgroup has yet been identified in whom the drug has particular benefits, so it is a bit of a challenge to determine who should be put on this drug.

"I would consider it in patients with moderate to severe, nonrenal, nonneurologic disease who have failed standard therapy," Dr. Looney said, explaining that he would consider those to be patients with continued disease activity or patients in whom steroids can’t be tapered or stopped despite treatment.

"So I look at this as a drug that might help me get my patients who are on hydroxychloroquine and let's say CellCept [mycophenolate] or Imuran [azathioprine], down to a manageable dose of steroids," he added.

Determining how long to treat, however, is a challenge.

"We have no good way to look at an individual patient and say they are better and they are better because they are on belimumab," Dr. Looney said, noting that based on belimumab trial data, three out of four patients who were responders at 1 year would have been responders on placebo, so further study is needed to help guide treatment duration.

"I would also really like to see predictors of response," he said, noting that such predictors have thus far not been reported.

Further analysis of existing data may be useful for identifying subgroups who will respond best to belimumab. Additional studies in renal and CNS disease would also be useful.

"It may be that you add this drug to current therapies [in renal and CNS patients] and they do that much better," Dr. Looney said, noting that it would also be useful to look at belimumab treatment in conjunction with cyclophosphamide and rituximab as well "to see what happens when B cells are targeted in two different ways."

Dr. Looney disclosed that he has been an adviser for Genentech.

DESTIN, FLA. – For now, the lupus patients most likely to respond to recently approved belimumab may be those with moderate to severe disease without renal or central nervous system involvement who have failed to respond to standard therapy, according to Dr. R. John Looney.

More specific details on which patients are most likely to benefit will require further experience with the drug, he said at the Congress of Clinical Rheumatology.

Belimumab (Benlysta), which was approved in March, is the first new drug for systemic lupus erythematosus (SLE) in 50 years. It has a very good safety profile, although the effect size, while statistically significant, is relatively small, with only an average of about a 12 percentage point difference in the response rates between treatment and placebo in trials of the fully human monoclonal antibody that inhibits B-lymphocyte stimulator, said Dr. Looney, professor of medicine at the University of Rochester (N.Y.).

Still, that reflects improvement of about 25%-30%, he noted.

The standard dosage is 10 mg/kg, given intravenously every 2 weeks for three times, then every 4 weeks, in addition to standard care.

Keep in mind that the onset is somewhat slow, so this "may not be a drug you would use in a flaring patient," Dr. Looney said.

Also, it has not been studied in severe renal or central nervous system disease, nor in combination with cyclophosphamide, he noted.

Furthermore, no particular subgroup has yet been identified in whom the drug has particular benefits, so it is a bit of a challenge to determine who should be put on this drug.

"I would consider it in patients with moderate to severe, nonrenal, nonneurologic disease who have failed standard therapy," Dr. Looney said, explaining that he would consider those to be patients with continued disease activity or patients in whom steroids can’t be tapered or stopped despite treatment.

"So I look at this as a drug that might help me get my patients who are on hydroxychloroquine and let's say CellCept [mycophenolate] or Imuran [azathioprine], down to a manageable dose of steroids," he added.

Determining how long to treat, however, is a challenge.

"We have no good way to look at an individual patient and say they are better and they are better because they are on belimumab," Dr. Looney said, noting that based on belimumab trial data, three out of four patients who were responders at 1 year would have been responders on placebo, so further study is needed to help guide treatment duration.

"I would also really like to see predictors of response," he said, noting that such predictors have thus far not been reported.

Further analysis of existing data may be useful for identifying subgroups who will respond best to belimumab. Additional studies in renal and CNS disease would also be useful.

"It may be that you add this drug to current therapies [in renal and CNS patients] and they do that much better," Dr. Looney said, noting that it would also be useful to look at belimumab treatment in conjunction with cyclophosphamide and rituximab as well "to see what happens when B cells are targeted in two different ways."

Dr. Looney disclosed that he has been an adviser for Genentech.

DESTIN, FLA. – For now, the lupus patients most likely to respond to recently approved belimumab may be those with moderate to severe disease without renal or central nervous system involvement who have failed to respond to standard therapy, according to Dr. R. John Looney.

More specific details on which patients are most likely to benefit will require further experience with the drug, he said at the Congress of Clinical Rheumatology.

Belimumab (Benlysta), which was approved in March, is the first new drug for systemic lupus erythematosus (SLE) in 50 years. It has a very good safety profile, although the effect size, while statistically significant, is relatively small, with only an average of about a 12 percentage point difference in the response rates between treatment and placebo in trials of the fully human monoclonal antibody that inhibits B-lymphocyte stimulator, said Dr. Looney, professor of medicine at the University of Rochester (N.Y.).

Still, that reflects improvement of about 25%-30%, he noted.

The standard dosage is 10 mg/kg, given intravenously every 2 weeks for three times, then every 4 weeks, in addition to standard care.

Keep in mind that the onset is somewhat slow, so this "may not be a drug you would use in a flaring patient," Dr. Looney said.

Also, it has not been studied in severe renal or central nervous system disease, nor in combination with cyclophosphamide, he noted.

Furthermore, no particular subgroup has yet been identified in whom the drug has particular benefits, so it is a bit of a challenge to determine who should be put on this drug.

"I would consider it in patients with moderate to severe, nonrenal, nonneurologic disease who have failed standard therapy," Dr. Looney said, explaining that he would consider those to be patients with continued disease activity or patients in whom steroids can’t be tapered or stopped despite treatment.

"So I look at this as a drug that might help me get my patients who are on hydroxychloroquine and let's say CellCept [mycophenolate] or Imuran [azathioprine], down to a manageable dose of steroids," he added.

Determining how long to treat, however, is a challenge.

"We have no good way to look at an individual patient and say they are better and they are better because they are on belimumab," Dr. Looney said, noting that based on belimumab trial data, three out of four patients who were responders at 1 year would have been responders on placebo, so further study is needed to help guide treatment duration.

"I would also really like to see predictors of response," he said, noting that such predictors have thus far not been reported.

Further analysis of existing data may be useful for identifying subgroups who will respond best to belimumab. Additional studies in renal and CNS disease would also be useful.

"It may be that you add this drug to current therapies [in renal and CNS patients] and they do that much better," Dr. Looney said, noting that it would also be useful to look at belimumab treatment in conjunction with cyclophosphamide and rituximab as well "to see what happens when B cells are targeted in two different ways."

Dr. Looney disclosed that he has been an adviser for Genentech.

CHICAGO – The list of off-label uses for biologic agents is growing, according to Dr. Eric Ruderman, who provided an update on dermatologic, ophthalmologic, and other uses at a symposium sponsored by the American College of Rheumatology.

There are scant data to support off-label uses, and these typically come from retrospective reports, small case series, or anecdotal experience, with a few exceptions, said Dr. Ruderman, a rheumatologist at Northwestern University, Chicago.

Dermatologic Uses

Infliximab is increasingly being used to treat hidradenitis suppurativa and neutrophilic dermatoses despite a lack of data from randomized controlled trials to support this practice. Findings from a small double-blind crossover trial involving 38 patients who were treated for 8 weeks, however, did suggest that infliximab improves pain, disease severity, and quality of life.

Some benefit was also shown with adalimumab in this disease in a recent case series, while benefits with etanercept were less clear in two open-label trials. Patients in the etanercept trials also had worsening of their condition after etanercept was discontinued.

As for neutrophilic dermatoses, infliximab appears to be useful for pyoderma gangrenosum and possibly for Sweet’s syndrome. In a double-blind study of 30 patients with pyoderma gangrenosum, a single dose of 5 mg/kg was associated with a 46% improvement, compared with 6% for placebo. Furthermore, nonresponders on placebo at 2 weeks were treated with infliximab with a 60% response rate, and 21% of treated patients achieved remission.

Case reports also show a benefit of infliximab in patients with Sweet's syndrome, but no controlled data are available for this condition, Dr. Ruderman said.

Ophthalmologic Uses

Biologic therapies being used for ophthalmologic conditions include infliximab, adalimumab, and/or daclizumab for childhood uveitis and other types of uveitis, he noted.

Retrospective case series have shown benefit with infliximab, adalimumab, and daclizumab in childhood uveitis, and an open-label prospective study comparing adalimumab with infliximab demonstrated greater benefit with adalimumab (60% vs. 20% remission at 40 months), he said.

Infliximab also may have some benefit in ocular sarcoidosis, with case reports and small open-label series demonstrating some benefit. Etanercept in this disease, however, appears to be no more effective than placebo, based on findings of a randomized study of 18 patients treated for 6 months.

In Behçet's uveitis, case reports and findings from two small open trials suggest some benefit with infliximab. In the open trials, 9 of 12 patients responded to a dose of 5 mg/kg, Dr. Ruderman said, adding that treatment helped reduce steroid use, but relapse was frequent after treatment discontinuation, suggesting that ongoing therapy is necessary.

Pulmonary Uses

Other conditions in which biologics are currently being used include pulmonary and extrapulmonary sarcoidosis, asthma, and chronic obstructive pulmonary disease (COPD).

In one "nicely done" randomized, controlled, multicenter study of 138 pulmonary sarcoidosis patients, infliximab was superior to placebo for improving the percent predicted forced vital capacity at 24 weeks, and also led to improvement in extrapulmonary disease, Dr. Ruderman said.

Improvements in scores on the extrapulmonary physician organ severity tool (ePOST) were better in the treatment versus placebo group at 24 weeks, even after adjusting for the number of organs involved, he said.

Findings from an open-label study of etanercept in this disease, however, showed that only 5 of 17 patients had "success," which was defined as lack of deterioration on x-rays or pulmonary function tests.

Case reports and retrospective data – but not randomized controlled data – have also shown some benefit of infliximab for cutaneous sarcoidosis. In 54 patients who received 116 courses of therapy, resolution or near resolution of lupus pernio was achieved in 77% of patients versus 11%-29% of those treated with steroids and other therapy. Some case reports show benefit in neurosarcoidosis, as well, but no solid data are available to back those up, Dr. Ruderman said.

For asthma, there may be a role for tumor necrosis factor inhibitors, although one randomized controlled trial showed no benefit with golimumab.

The Brigham RA Sequential Study, for example, demonstrated improvement with TNF inhibitors in nine patients with both rheumatoid arthritis (RA) and asthma, and findings from a small, controlled trial of etanercept showed some benefit on several measures of response. The benefits in that study, however, were not clear-cut, and two other etanercept trials, as well as one infliximab trial, failed to show any benefit in asthma.

Similarly, golimumab showed no benefit in a 52-week randomized, controlled trial of 231 patients with severe uncontrolled asthma. Patients in that trial had no improvements over placebo in forced expiratory volume in 1 second (FEV₁) or exacerbations, and they experienced frequent severe infections, Dr. Ruderman said.

Two studies found that infliximab therapy provided no benefit in the management of COPD. The first was a 24-week study of 3 mg/kg, 5 mg/kg, and placebo in 159 patients. The findings showed no improvement in health status or FEV₁, no lessening of exacerbations with treatment vs. placebo, and an increase in the risk of pneumonia in the treated group. The second study was an observational study involving a cohort of 15,771 patients with both RA and COPD. In this study, however, treatment with etanercept was associated with a 50% reduction in the rate of COPD hospitalization.

"I don’t know entirely what to make of that," Dr. Ruderman said, adding that it might be a result of improvement in the RA and thus overall health.

It's an interesting observation, but overall the findings don't say much about the off-label use of TNF inhibitors in COPD, he said.

Dr. Ruderman reported receiving consulting fees and/or research grants from Abbott, Allos, Amgen, Biogenidec, Celgene, Centocor, Crescendo, CVS/Caremark, Pfizer, and UCB.

CHICAGO – The list of off-label uses for biologic agents is growing, according to Dr. Eric Ruderman, who provided an update on dermatologic, ophthalmologic, and other uses at a symposium sponsored by the American College of Rheumatology.

There are scant data to support off-label uses, and these typically come from retrospective reports, small case series, or anecdotal experience, with a few exceptions, said Dr. Ruderman, a rheumatologist at Northwestern University, Chicago.

Dermatologic Uses

Infliximab is increasingly being used to treat hidradenitis suppurativa and neutrophilic dermatoses despite a lack of data from randomized controlled trials to support this practice. Findings from a small double-blind crossover trial involving 38 patients who were treated for 8 weeks, however, did suggest that infliximab improves pain, disease severity, and quality of life.

Some benefit was also shown with adalimumab in this disease in a recent case series, while benefits with etanercept were less clear in two open-label trials. Patients in the etanercept trials also had worsening of their condition after etanercept was discontinued.

As for neutrophilic dermatoses, infliximab appears to be useful for pyoderma gangrenosum and possibly for Sweet’s syndrome. In a double-blind study of 30 patients with pyoderma gangrenosum, a single dose of 5 mg/kg was associated with a 46% improvement, compared with 6% for placebo. Furthermore, nonresponders on placebo at 2 weeks were treated with infliximab with a 60% response rate, and 21% of treated patients achieved remission.

Case reports also show a benefit of infliximab in patients with Sweet's syndrome, but no controlled data are available for this condition, Dr. Ruderman said.

Ophthalmologic Uses

Biologic therapies being used for ophthalmologic conditions include infliximab, adalimumab, and/or daclizumab for childhood uveitis and other types of uveitis, he noted.

Retrospective case series have shown benefit with infliximab, adalimumab, and daclizumab in childhood uveitis, and an open-label prospective study comparing adalimumab with infliximab demonstrated greater benefit with adalimumab (60% vs. 20% remission at 40 months), he said.

Infliximab also may have some benefit in ocular sarcoidosis, with case reports and small open-label series demonstrating some benefit. Etanercept in this disease, however, appears to be no more effective than placebo, based on findings of a randomized study of 18 patients treated for 6 months.

In Behçet's uveitis, case reports and findings from two small open trials suggest some benefit with infliximab. In the open trials, 9 of 12 patients responded to a dose of 5 mg/kg, Dr. Ruderman said, adding that treatment helped reduce steroid use, but relapse was frequent after treatment discontinuation, suggesting that ongoing therapy is necessary.

Pulmonary Uses

Other conditions in which biologics are currently being used include pulmonary and extrapulmonary sarcoidosis, asthma, and chronic obstructive pulmonary disease (COPD).

In one "nicely done" randomized, controlled, multicenter study of 138 pulmonary sarcoidosis patients, infliximab was superior to placebo for improving the percent predicted forced vital capacity at 24 weeks, and also led to improvement in extrapulmonary disease, Dr. Ruderman said.

Improvements in scores on the extrapulmonary physician organ severity tool (ePOST) were better in the treatment versus placebo group at 24 weeks, even after adjusting for the number of organs involved, he said.

Findings from an open-label study of etanercept in this disease, however, showed that only 5 of 17 patients had "success," which was defined as lack of deterioration on x-rays or pulmonary function tests.

Case reports and retrospective data – but not randomized controlled data – have also shown some benefit of infliximab for cutaneous sarcoidosis. In 54 patients who received 116 courses of therapy, resolution or near resolution of lupus pernio was achieved in 77% of patients versus 11%-29% of those treated with steroids and other therapy. Some case reports show benefit in neurosarcoidosis, as well, but no solid data are available to back those up, Dr. Ruderman said.

For asthma, there may be a role for tumor necrosis factor inhibitors, although one randomized controlled trial showed no benefit with golimumab.

The Brigham RA Sequential Study, for example, demonstrated improvement with TNF inhibitors in nine patients with both rheumatoid arthritis (RA) and asthma, and findings from a small, controlled trial of etanercept showed some benefit on several measures of response. The benefits in that study, however, were not clear-cut, and two other etanercept trials, as well as one infliximab trial, failed to show any benefit in asthma.

Similarly, golimumab showed no benefit in a 52-week randomized, controlled trial of 231 patients with severe uncontrolled asthma. Patients in that trial had no improvements over placebo in forced expiratory volume in 1 second (FEV₁) or exacerbations, and they experienced frequent severe infections, Dr. Ruderman said.

Two studies found that infliximab therapy provided no benefit in the management of COPD. The first was a 24-week study of 3 mg/kg, 5 mg/kg, and placebo in 159 patients. The findings showed no improvement in health status or FEV₁, no lessening of exacerbations with treatment vs. placebo, and an increase in the risk of pneumonia in the treated group. The second study was an observational study involving a cohort of 15,771 patients with both RA and COPD. In this study, however, treatment with etanercept was associated with a 50% reduction in the rate of COPD hospitalization.

"I don’t know entirely what to make of that," Dr. Ruderman said, adding that it might be a result of improvement in the RA and thus overall health.

It's an interesting observation, but overall the findings don't say much about the off-label use of TNF inhibitors in COPD, he said.

Dr. Ruderman reported receiving consulting fees and/or research grants from Abbott, Allos, Amgen, Biogenidec, Celgene, Centocor, Crescendo, CVS/Caremark, Pfizer, and UCB.

CHICAGO – The list of off-label uses for biologic agents is growing, according to Dr. Eric Ruderman, who provided an update on dermatologic, ophthalmologic, and other uses at a symposium sponsored by the American College of Rheumatology.

There are scant data to support off-label uses, and these typically come from retrospective reports, small case series, or anecdotal experience, with a few exceptions, said Dr. Ruderman, a rheumatologist at Northwestern University, Chicago.

Dermatologic Uses

Infliximab is increasingly being used to treat hidradenitis suppurativa and neutrophilic dermatoses despite a lack of data from randomized controlled trials to support this practice. Findings from a small double-blind crossover trial involving 38 patients who were treated for 8 weeks, however, did suggest that infliximab improves pain, disease severity, and quality of life.

Some benefit was also shown with adalimumab in this disease in a recent case series, while benefits with etanercept were less clear in two open-label trials. Patients in the etanercept trials also had worsening of their condition after etanercept was discontinued.

As for neutrophilic dermatoses, infliximab appears to be useful for pyoderma gangrenosum and possibly for Sweet’s syndrome. In a double-blind study of 30 patients with pyoderma gangrenosum, a single dose of 5 mg/kg was associated with a 46% improvement, compared with 6% for placebo. Furthermore, nonresponders on placebo at 2 weeks were treated with infliximab with a 60% response rate, and 21% of treated patients achieved remission.

Case reports also show a benefit of infliximab in patients with Sweet's syndrome, but no controlled data are available for this condition, Dr. Ruderman said.

Ophthalmologic Uses

Biologic therapies being used for ophthalmologic conditions include infliximab, adalimumab, and/or daclizumab for childhood uveitis and other types of uveitis, he noted.

Retrospective case series have shown benefit with infliximab, adalimumab, and daclizumab in childhood uveitis, and an open-label prospective study comparing adalimumab with infliximab demonstrated greater benefit with adalimumab (60% vs. 20% remission at 40 months), he said.

Infliximab also may have some benefit in ocular sarcoidosis, with case reports and small open-label series demonstrating some benefit. Etanercept in this disease, however, appears to be no more effective than placebo, based on findings of a randomized study of 18 patients treated for 6 months.

In Behçet's uveitis, case reports and findings from two small open trials suggest some benefit with infliximab. In the open trials, 9 of 12 patients responded to a dose of 5 mg/kg, Dr. Ruderman said, adding that treatment helped reduce steroid use, but relapse was frequent after treatment discontinuation, suggesting that ongoing therapy is necessary.

Pulmonary Uses

Other conditions in which biologics are currently being used include pulmonary and extrapulmonary sarcoidosis, asthma, and chronic obstructive pulmonary disease (COPD).

In one "nicely done" randomized, controlled, multicenter study of 138 pulmonary sarcoidosis patients, infliximab was superior to placebo for improving the percent predicted forced vital capacity at 24 weeks, and also led to improvement in extrapulmonary disease, Dr. Ruderman said.

Improvements in scores on the extrapulmonary physician organ severity tool (ePOST) were better in the treatment versus placebo group at 24 weeks, even after adjusting for the number of organs involved, he said.

Findings from an open-label study of etanercept in this disease, however, showed that only 5 of 17 patients had "success," which was defined as lack of deterioration on x-rays or pulmonary function tests.

Case reports and retrospective data – but not randomized controlled data – have also shown some benefit of infliximab for cutaneous sarcoidosis. In 54 patients who received 116 courses of therapy, resolution or near resolution of lupus pernio was achieved in 77% of patients versus 11%-29% of those treated with steroids and other therapy. Some case reports show benefit in neurosarcoidosis, as well, but no solid data are available to back those up, Dr. Ruderman said.

For asthma, there may be a role for tumor necrosis factor inhibitors, although one randomized controlled trial showed no benefit with golimumab.

The Brigham RA Sequential Study, for example, demonstrated improvement with TNF inhibitors in nine patients with both rheumatoid arthritis (RA) and asthma, and findings from a small, controlled trial of etanercept showed some benefit on several measures of response. The benefits in that study, however, were not clear-cut, and two other etanercept trials, as well as one infliximab trial, failed to show any benefit in asthma.

Similarly, golimumab showed no benefit in a 52-week randomized, controlled trial of 231 patients with severe uncontrolled asthma. Patients in that trial had no improvements over placebo in forced expiratory volume in 1 second (FEV₁) or exacerbations, and they experienced frequent severe infections, Dr. Ruderman said.

Two studies found that infliximab therapy provided no benefit in the management of COPD. The first was a 24-week study of 3 mg/kg, 5 mg/kg, and placebo in 159 patients. The findings showed no improvement in health status or FEV₁, no lessening of exacerbations with treatment vs. placebo, and an increase in the risk of pneumonia in the treated group. The second study was an observational study involving a cohort of 15,771 patients with both RA and COPD. In this study, however, treatment with etanercept was associated with a 50% reduction in the rate of COPD hospitalization.

"I don’t know entirely what to make of that," Dr. Ruderman said, adding that it might be a result of improvement in the RA and thus overall health.

It's an interesting observation, but overall the findings don't say much about the off-label use of TNF inhibitors in COPD, he said.

Dr. Ruderman reported receiving consulting fees and/or research grants from Abbott, Allos, Amgen, Biogenidec, Celgene, Centocor, Crescendo, CVS/Caremark, Pfizer, and UCB.