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Christopher Palmer has been an associate editor at MDedge News since 2017. When he's not tidying grammar, he writes short pieces about breaking FDA announcements and approvals, as well as journal articles. He proudly holds a BA in English and philosophy. Follow him on Twitter @cmacmpalm.
Subcutaneous Actemra approved for systemic JIA
The Food and Drug Administration has approved the subcutaneous formulation of Actemra (tocilizumab) for systemic juvenile idiopathic arthritis (SJIA) for patients aged 2 years and older, according to a press release from its developer, Genentech. The intravenous formulation was approved in 2011 for this indication.
The approval is based on data the JIGSAW-118 study. This 52-week, open-label, multicenter, phase 1b pharmacokinetic/pharmacodynamic bridging study was designed to determine the appropriate dosing regimen by treating 51 patients with SJIA according to body weight.
The safety profile of subcutaneous tocilizumab was similar to that seen with intravenous tocilizumab, although there were more injection-site reactions seen with the subcutaneous formulation. Its efficacy was extrapolated based on the drug’s pharmacokinetic profile seen with IV tocilizumab in SJIA patients and with subcutaneous tocilizumab in patients with rheumatoid arthritis.
SJIA is a rare disease with limited treatment options, according to the press release. In general, JIA affects almost 300,000 children in the United States, and about 10% of those cases are SJIA.
The Food and Drug Administration has approved the subcutaneous formulation of Actemra (tocilizumab) for systemic juvenile idiopathic arthritis (SJIA) for patients aged 2 years and older, according to a press release from its developer, Genentech. The intravenous formulation was approved in 2011 for this indication.
The approval is based on data the JIGSAW-118 study. This 52-week, open-label, multicenter, phase 1b pharmacokinetic/pharmacodynamic bridging study was designed to determine the appropriate dosing regimen by treating 51 patients with SJIA according to body weight.
The safety profile of subcutaneous tocilizumab was similar to that seen with intravenous tocilizumab, although there were more injection-site reactions seen with the subcutaneous formulation. Its efficacy was extrapolated based on the drug’s pharmacokinetic profile seen with IV tocilizumab in SJIA patients and with subcutaneous tocilizumab in patients with rheumatoid arthritis.
SJIA is a rare disease with limited treatment options, according to the press release. In general, JIA affects almost 300,000 children in the United States, and about 10% of those cases are SJIA.
The Food and Drug Administration has approved the subcutaneous formulation of Actemra (tocilizumab) for systemic juvenile idiopathic arthritis (SJIA) for patients aged 2 years and older, according to a press release from its developer, Genentech. The intravenous formulation was approved in 2011 for this indication.
The approval is based on data the JIGSAW-118 study. This 52-week, open-label, multicenter, phase 1b pharmacokinetic/pharmacodynamic bridging study was designed to determine the appropriate dosing regimen by treating 51 patients with SJIA according to body weight.
The safety profile of subcutaneous tocilizumab was similar to that seen with intravenous tocilizumab, although there were more injection-site reactions seen with the subcutaneous formulation. Its efficacy was extrapolated based on the drug’s pharmacokinetic profile seen with IV tocilizumab in SJIA patients and with subcutaneous tocilizumab in patients with rheumatoid arthritis.
SJIA is a rare disease with limited treatment options, according to the press release. In general, JIA affects almost 300,000 children in the United States, and about 10% of those cases are SJIA.
Dr. Hal C. Lawrence III retires from ACOG
The American College of Obstetricians and Gynecologists has announced that, after 11 years with ACOG, Hal C. Lawrence III, MD, is retiring from his positions as executive vice president and chief executive officer.
“It has been an honor to lead the association and our dedicated team of employees to support members and advance women’s health,” Dr. Lawrence said in a statement from ACOG. “I am proud of all that we have achieved together.”
Under his leadership, ACOG has seen the creation of the Council on Patient Safety in Women’s Health Care and the Alliance for Innovation on Maternal Health, development of ACOG’s Global Operations Advisory Group and immunization expert work group, and creation of the Women’s Preventive Services Initiative. He also helped to restructure and revitalize the ACOG annual meeting, which has been credited for increased member attendance and improved attendee satisfaction.
His retirement becomes effective Oct. 31, 2018.
Prior to his leadership roles at ACOG, Dr. Lawrence was a practicing ob.gyn. in Asheville, N.C., from 1979 to 2007 and was a member of the Ob.Gyn. News editorial advisory board.
The American College of Obstetricians and Gynecologists has announced that, after 11 years with ACOG, Hal C. Lawrence III, MD, is retiring from his positions as executive vice president and chief executive officer.
“It has been an honor to lead the association and our dedicated team of employees to support members and advance women’s health,” Dr. Lawrence said in a statement from ACOG. “I am proud of all that we have achieved together.”
Under his leadership, ACOG has seen the creation of the Council on Patient Safety in Women’s Health Care and the Alliance for Innovation on Maternal Health, development of ACOG’s Global Operations Advisory Group and immunization expert work group, and creation of the Women’s Preventive Services Initiative. He also helped to restructure and revitalize the ACOG annual meeting, which has been credited for increased member attendance and improved attendee satisfaction.
His retirement becomes effective Oct. 31, 2018.
Prior to his leadership roles at ACOG, Dr. Lawrence was a practicing ob.gyn. in Asheville, N.C., from 1979 to 2007 and was a member of the Ob.Gyn. News editorial advisory board.
The American College of Obstetricians and Gynecologists has announced that, after 11 years with ACOG, Hal C. Lawrence III, MD, is retiring from his positions as executive vice president and chief executive officer.
“It has been an honor to lead the association and our dedicated team of employees to support members and advance women’s health,” Dr. Lawrence said in a statement from ACOG. “I am proud of all that we have achieved together.”
Under his leadership, ACOG has seen the creation of the Council on Patient Safety in Women’s Health Care and the Alliance for Innovation on Maternal Health, development of ACOG’s Global Operations Advisory Group and immunization expert work group, and creation of the Women’s Preventive Services Initiative. He also helped to restructure and revitalize the ACOG annual meeting, which has been credited for increased member attendance and improved attendee satisfaction.
His retirement becomes effective Oct. 31, 2018.
Prior to his leadership roles at ACOG, Dr. Lawrence was a practicing ob.gyn. in Asheville, N.C., from 1979 to 2007 and was a member of the Ob.Gyn. News editorial advisory board.
Screen all infants exposed to Zika for eye abnormalities, study suggests
CNS abnormalities associated with antenatal Zika virus infection correlate strongly with opthalmic abnormalities, but there were cases of eye abnormalities in the absence of CNS abnormalities, which suggests a need for universal eye screening in endemic areas, according to a study published in Pediatrics.
Irena Tsui, MD, of the University of California, Los Angeles, and her associates examined 224 infants suspected of antenatal Zika virus infection for eye abnormalities between Jan. 2, 2016, and Feb. 28, 2017. They found that 40% had CNS abnormalities and 25% of all infants had eye abnormalities; of those 90 infants with CNS abnormalities, 54% had eye abnormalities, which makes for an odds ratio of 14.9 (P less than .0001). However, among the 134 infants without CNS abnormalities, 4% had eye abnormalities.
The study also investigated the existence of eye abnormalities among infants did not laboratory-confirmed diagnosis of Zika virus infection. To do so, they performed reverse transcriptase polymerase chain reaction (RT-PCR) testing on 189 infants. They found eye abnormalities among 22% of the 156 RT-PCR–positive infants and 38% of the 68 RT-PCR–unconfirmed infants. Among the 52% of infants with eye abnormalities who were reexamined, there were no signs of worsening, ongoing activity, or regression in their lesions.
The guidelines in Brazil, where the study was performed, recommend eye examinations only for infants with microcephaly, and the United States currently recommends it only at the discretion of the health care provider. The study investigators think that
“The early identification of eye abnormalities enables low-vision interventions to improve visual function with important repercussions for neurocognitive development,” they concluded.
SOURCE: Tsui I et al. Pediatrics. 2018 Oct;142(4):e20181104.
CNS abnormalities associated with antenatal Zika virus infection correlate strongly with opthalmic abnormalities, but there were cases of eye abnormalities in the absence of CNS abnormalities, which suggests a need for universal eye screening in endemic areas, according to a study published in Pediatrics.
Irena Tsui, MD, of the University of California, Los Angeles, and her associates examined 224 infants suspected of antenatal Zika virus infection for eye abnormalities between Jan. 2, 2016, and Feb. 28, 2017. They found that 40% had CNS abnormalities and 25% of all infants had eye abnormalities; of those 90 infants with CNS abnormalities, 54% had eye abnormalities, which makes for an odds ratio of 14.9 (P less than .0001). However, among the 134 infants without CNS abnormalities, 4% had eye abnormalities.
The study also investigated the existence of eye abnormalities among infants did not laboratory-confirmed diagnosis of Zika virus infection. To do so, they performed reverse transcriptase polymerase chain reaction (RT-PCR) testing on 189 infants. They found eye abnormalities among 22% of the 156 RT-PCR–positive infants and 38% of the 68 RT-PCR–unconfirmed infants. Among the 52% of infants with eye abnormalities who were reexamined, there were no signs of worsening, ongoing activity, or regression in their lesions.
The guidelines in Brazil, where the study was performed, recommend eye examinations only for infants with microcephaly, and the United States currently recommends it only at the discretion of the health care provider. The study investigators think that
“The early identification of eye abnormalities enables low-vision interventions to improve visual function with important repercussions for neurocognitive development,” they concluded.
SOURCE: Tsui I et al. Pediatrics. 2018 Oct;142(4):e20181104.
CNS abnormalities associated with antenatal Zika virus infection correlate strongly with opthalmic abnormalities, but there were cases of eye abnormalities in the absence of CNS abnormalities, which suggests a need for universal eye screening in endemic areas, according to a study published in Pediatrics.
Irena Tsui, MD, of the University of California, Los Angeles, and her associates examined 224 infants suspected of antenatal Zika virus infection for eye abnormalities between Jan. 2, 2016, and Feb. 28, 2017. They found that 40% had CNS abnormalities and 25% of all infants had eye abnormalities; of those 90 infants with CNS abnormalities, 54% had eye abnormalities, which makes for an odds ratio of 14.9 (P less than .0001). However, among the 134 infants without CNS abnormalities, 4% had eye abnormalities.
The study also investigated the existence of eye abnormalities among infants did not laboratory-confirmed diagnosis of Zika virus infection. To do so, they performed reverse transcriptase polymerase chain reaction (RT-PCR) testing on 189 infants. They found eye abnormalities among 22% of the 156 RT-PCR–positive infants and 38% of the 68 RT-PCR–unconfirmed infants. Among the 52% of infants with eye abnormalities who were reexamined, there were no signs of worsening, ongoing activity, or regression in their lesions.
The guidelines in Brazil, where the study was performed, recommend eye examinations only for infants with microcephaly, and the United States currently recommends it only at the discretion of the health care provider. The study investigators think that
“The early identification of eye abnormalities enables low-vision interventions to improve visual function with important repercussions for neurocognitive development,” they concluded.
SOURCE: Tsui I et al. Pediatrics. 2018 Oct;142(4):e20181104.
FROM PEDIATRICS
FDA warns kratom vendors about unproven claims
The Food and Drug Administration has issued letters of warning to – and therefore breaking federal law – according to a statement from FDA commissioner Scott Gottlieb, MD. These vendors both claimed that their kratom products could, among other things, “relieve” or “treat” opium/opioid withdrawal.
“To date, there have been no adequate and well-controlled studies involving the use of kratom as a treatment for opioid use withdrawal or other diseases in humans,” noted Dr. Gottlieb.
As Dr. Gottlieb pointed out in his statement, not only can fraudulent health claims pose direct health risks, they can also, in the case of kratom, deter or delay people who’re suffering from opioid use disorder from seeking FDA-approved treatments that have been demonstrated to be safe and effective.
Kratom, also known more formally as Mitragyna speciosa, is a plant native to Thailand, Malaysia, Indonesia, and Papua New Guinea. Some compounds in the plant are believed to be opioids, some of which may have the potential for abuse. As Dr. Gottlieb pointed out in his statement, the substance is illegal or controlled in several countries and banned in some states and municipalities in the United States.
Find out more in Dr. Gottlieb’s full statement on the FDA website.
The Food and Drug Administration has issued letters of warning to – and therefore breaking federal law – according to a statement from FDA commissioner Scott Gottlieb, MD. These vendors both claimed that their kratom products could, among other things, “relieve” or “treat” opium/opioid withdrawal.
“To date, there have been no adequate and well-controlled studies involving the use of kratom as a treatment for opioid use withdrawal or other diseases in humans,” noted Dr. Gottlieb.
As Dr. Gottlieb pointed out in his statement, not only can fraudulent health claims pose direct health risks, they can also, in the case of kratom, deter or delay people who’re suffering from opioid use disorder from seeking FDA-approved treatments that have been demonstrated to be safe and effective.
Kratom, also known more formally as Mitragyna speciosa, is a plant native to Thailand, Malaysia, Indonesia, and Papua New Guinea. Some compounds in the plant are believed to be opioids, some of which may have the potential for abuse. As Dr. Gottlieb pointed out in his statement, the substance is illegal or controlled in several countries and banned in some states and municipalities in the United States.
Find out more in Dr. Gottlieb’s full statement on the FDA website.
The Food and Drug Administration has issued letters of warning to – and therefore breaking federal law – according to a statement from FDA commissioner Scott Gottlieb, MD. These vendors both claimed that their kratom products could, among other things, “relieve” or “treat” opium/opioid withdrawal.
“To date, there have been no adequate and well-controlled studies involving the use of kratom as a treatment for opioid use withdrawal or other diseases in humans,” noted Dr. Gottlieb.
As Dr. Gottlieb pointed out in his statement, not only can fraudulent health claims pose direct health risks, they can also, in the case of kratom, deter or delay people who’re suffering from opioid use disorder from seeking FDA-approved treatments that have been demonstrated to be safe and effective.
Kratom, also known more formally as Mitragyna speciosa, is a plant native to Thailand, Malaysia, Indonesia, and Papua New Guinea. Some compounds in the plant are believed to be opioids, some of which may have the potential for abuse. As Dr. Gottlieb pointed out in his statement, the substance is illegal or controlled in several countries and banned in some states and municipalities in the United States.
Find out more in Dr. Gottlieb’s full statement on the FDA website.
Regimens produce similar results in FL
Rituximab plus lenalidomide had efficacy similar to that of rituximab plus chemotherapy in the treatment of follicular lymphoma (FL) in a phase 3 trial.
Patients with previously untreated FL had similar complete response (CR) rates and progression-free survival (PFS) rates whether they received rituximab-based chemotherapy or rituximab plus lenalidomide.
These results were published in The New England Journal of Medicine.
The trial, RELEVANCE, included 1,030 patients with previously untreated FL. They were randomized to receive rituximab plus chemotherapy (n=517) or rituximab plus lenalidomide (n=513) for 18 cycles.
Patients in the chemotherapy arm received one of three regimens—R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone), rituximab and bendamustine, or R-CVP (rituximab, cyclophosphamide, vincristine, and prednisone).
Patients in both treatment arms went on to receive rituximab maintenance every 8 weeks for 12 cycles. The total duration of treatment was 120 weeks. The median age of the combined groups was 59 years.
The coprimary endpoints were CR (confirmed or unconfirmed) and PFS. After a median follow-up of 37.9 months, the rates of coprimary endpoints were similar between the treatment arms.
CR was observed in 48% of the rituximab-lenalidomide arm and 53% of the rituximab-chemotherapy arm (P=0.13).
The interim 3-year PFS rate was 77% in the rituximab-lenalidomide arm and 78% in the rituximab-chemotherapy arm. The hazard ratio for progression or death from any cause was 1.10 (P=0.48).
The efficacy of rituximab plus chemotherapy was greater in low-risk patients (based on Follicular Lymphoma International Prognostic Index scores) and in patients whose disease was Ann Arbor stage I or II, whereas the efficacy of rituximab-lenalidomide was independent of prognostic factors.
Safety was the biggest area of difference, with some adverse events (AEs) being more common in one arm than the other.
AEs that were more common with rituximab-lenalidomide include cutaneous reactions (43% vs 24%), diarrhea (37% vs 19%), rash (29% vs 8%), abdominal pain (15% vs 9%), peripheral edema (14% vs 9%), muscle spasms (13% vs 4%), myalgia (14% vs 6%), and tumor flare reaction (6% vs <1%).
AEs that were more common with rituximab-chemotherapy were anemia (89% vs 66%), fatigue (29% vs 23%), nausea (42% vs 20%), vomiting (19% vs 7%), febrile neutropenia (7% vs 2%), leukopenia (10% vs 4%), and peripheral neuropathy (16% vs 7%).
Grade 3/4 cutaneous reactions were more common with rituximab-lenalidomide (7% vs 1%), and grade 3/4 neutropenia was more common with rituximab-chemotherapy (50% vs 32%).
The RELEVANCE trial was sponsored by Celgene and the Lymphoma Academic Research Organisation. The study authors reported various disclosures, including financial ties to Celgene.
Rituximab plus lenalidomide had efficacy similar to that of rituximab plus chemotherapy in the treatment of follicular lymphoma (FL) in a phase 3 trial.
Patients with previously untreated FL had similar complete response (CR) rates and progression-free survival (PFS) rates whether they received rituximab-based chemotherapy or rituximab plus lenalidomide.
These results were published in The New England Journal of Medicine.
The trial, RELEVANCE, included 1,030 patients with previously untreated FL. They were randomized to receive rituximab plus chemotherapy (n=517) or rituximab plus lenalidomide (n=513) for 18 cycles.
Patients in the chemotherapy arm received one of three regimens—R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone), rituximab and bendamustine, or R-CVP (rituximab, cyclophosphamide, vincristine, and prednisone).
Patients in both treatment arms went on to receive rituximab maintenance every 8 weeks for 12 cycles. The total duration of treatment was 120 weeks. The median age of the combined groups was 59 years.
The coprimary endpoints were CR (confirmed or unconfirmed) and PFS. After a median follow-up of 37.9 months, the rates of coprimary endpoints were similar between the treatment arms.
CR was observed in 48% of the rituximab-lenalidomide arm and 53% of the rituximab-chemotherapy arm (P=0.13).
The interim 3-year PFS rate was 77% in the rituximab-lenalidomide arm and 78% in the rituximab-chemotherapy arm. The hazard ratio for progression or death from any cause was 1.10 (P=0.48).
The efficacy of rituximab plus chemotherapy was greater in low-risk patients (based on Follicular Lymphoma International Prognostic Index scores) and in patients whose disease was Ann Arbor stage I or II, whereas the efficacy of rituximab-lenalidomide was independent of prognostic factors.
Safety was the biggest area of difference, with some adverse events (AEs) being more common in one arm than the other.
AEs that were more common with rituximab-lenalidomide include cutaneous reactions (43% vs 24%), diarrhea (37% vs 19%), rash (29% vs 8%), abdominal pain (15% vs 9%), peripheral edema (14% vs 9%), muscle spasms (13% vs 4%), myalgia (14% vs 6%), and tumor flare reaction (6% vs <1%).
AEs that were more common with rituximab-chemotherapy were anemia (89% vs 66%), fatigue (29% vs 23%), nausea (42% vs 20%), vomiting (19% vs 7%), febrile neutropenia (7% vs 2%), leukopenia (10% vs 4%), and peripheral neuropathy (16% vs 7%).
Grade 3/4 cutaneous reactions were more common with rituximab-lenalidomide (7% vs 1%), and grade 3/4 neutropenia was more common with rituximab-chemotherapy (50% vs 32%).
The RELEVANCE trial was sponsored by Celgene and the Lymphoma Academic Research Organisation. The study authors reported various disclosures, including financial ties to Celgene.
Rituximab plus lenalidomide had efficacy similar to that of rituximab plus chemotherapy in the treatment of follicular lymphoma (FL) in a phase 3 trial.
Patients with previously untreated FL had similar complete response (CR) rates and progression-free survival (PFS) rates whether they received rituximab-based chemotherapy or rituximab plus lenalidomide.
These results were published in The New England Journal of Medicine.
The trial, RELEVANCE, included 1,030 patients with previously untreated FL. They were randomized to receive rituximab plus chemotherapy (n=517) or rituximab plus lenalidomide (n=513) for 18 cycles.
Patients in the chemotherapy arm received one of three regimens—R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone), rituximab and bendamustine, or R-CVP (rituximab, cyclophosphamide, vincristine, and prednisone).
Patients in both treatment arms went on to receive rituximab maintenance every 8 weeks for 12 cycles. The total duration of treatment was 120 weeks. The median age of the combined groups was 59 years.
The coprimary endpoints were CR (confirmed or unconfirmed) and PFS. After a median follow-up of 37.9 months, the rates of coprimary endpoints were similar between the treatment arms.
CR was observed in 48% of the rituximab-lenalidomide arm and 53% of the rituximab-chemotherapy arm (P=0.13).
The interim 3-year PFS rate was 77% in the rituximab-lenalidomide arm and 78% in the rituximab-chemotherapy arm. The hazard ratio for progression or death from any cause was 1.10 (P=0.48).
The efficacy of rituximab plus chemotherapy was greater in low-risk patients (based on Follicular Lymphoma International Prognostic Index scores) and in patients whose disease was Ann Arbor stage I or II, whereas the efficacy of rituximab-lenalidomide was independent of prognostic factors.
Safety was the biggest area of difference, with some adverse events (AEs) being more common in one arm than the other.
AEs that were more common with rituximab-lenalidomide include cutaneous reactions (43% vs 24%), diarrhea (37% vs 19%), rash (29% vs 8%), abdominal pain (15% vs 9%), peripheral edema (14% vs 9%), muscle spasms (13% vs 4%), myalgia (14% vs 6%), and tumor flare reaction (6% vs <1%).
AEs that were more common with rituximab-chemotherapy were anemia (89% vs 66%), fatigue (29% vs 23%), nausea (42% vs 20%), vomiting (19% vs 7%), febrile neutropenia (7% vs 2%), leukopenia (10% vs 4%), and peripheral neuropathy (16% vs 7%).
Grade 3/4 cutaneous reactions were more common with rituximab-lenalidomide (7% vs 1%), and grade 3/4 neutropenia was more common with rituximab-chemotherapy (50% vs 32%).
The RELEVANCE trial was sponsored by Celgene and the Lymphoma Academic Research Organisation. The study authors reported various disclosures, including financial ties to Celgene.
Rituximab/lenalidomide similar to rituximab/chemotherapy for follicular lymphoma
Rituximab plus lenalidomide had efficacy similar to that of rituximab plus chemotherapy in treatment of follicular lymphoma, according to results from a phase 3 trial.
RELEVANCE (NCT01476787) was a multicenter, international, randomized, open-label trial designed to determine the superiority of rituximab/lenalidomide over rituximab/chemotherapy.
This trial randomized 1,030 patients with previously untreated follicular lymphoma to receive either rituximab plus lenalidomide (n = 513) or rituximab plus chemotherapy (n = 517) for 18 cycles; both groups then went on to receive rituximab maintenance therapy for 12 cycles. The total duration of treatment was 120 weeks. The median age of the combined groups was 59 years. The study was published in the New England Journal of Medicine.
One of the coprimary endpoints was complete response (confirmed or unconfirmed) by the end of the treatment period; the other was progression-free survival, which was planned to be assessed through three analyses, including two interim analyses, the first of which was reported in this study.
After a median follow-up of 37.9 months, the rates of coprimary endpoints were similar between the two groups. Complete response (confirmed or unconfirmed) was seen in 48% of the rituximab/lenalidomide group (95% confidence interval [CI], 44-53) and in 53% of the rituximab/chemotherapy group (95% CI, 49-57; P = .13). The hazard ratio for progression or death from any cause was 1.10 (95% CI, 0.85-1.43; P = .48).
In the subgroup analyses, the efficacy of rituximab plus chemotherapy was greater in low-risk patients (based on Follicular Lymphoma International Prognostic Index scores) and in patients whose follicular lymphoma was Ann Arbor stage I or II, whereas efficacy of rituximab/lenalidomide was independent of prognostic factors.
Safety was the biggest area of difference, with some events being more common in one group than in the other. For example, cutaneous reactions, diarrhea, rash, and myalgia were more common with rituximab/lenalidomide treatment, whereas anemia, fatigue, nausea, and febrile neutropenia were more common with rituximab/chemotherapy treatment. Among grade 3 or 4 events, cutaneous reactions were more common with rituximab/lenalidomide, and grade 3 or 4 neutropenia was more common with rituximab/chemotherapy.
“Overall, both treatment groups showed good outcomes, and a median has not yet been reached for either progression-free survival or overall survival,” the study authors wrote.
The RELEVANCE trial was sponsored by Celgene and the Lymphoma Academic Research Organisation. The study authors reported various disclosures, including financial ties to Celgene.
SOURCE: Morschhauser F et al. N Engl J Med. 2018;379:934-47.
Rituximab plus lenalidomide had efficacy similar to that of rituximab plus chemotherapy in treatment of follicular lymphoma, according to results from a phase 3 trial.
RELEVANCE (NCT01476787) was a multicenter, international, randomized, open-label trial designed to determine the superiority of rituximab/lenalidomide over rituximab/chemotherapy.
This trial randomized 1,030 patients with previously untreated follicular lymphoma to receive either rituximab plus lenalidomide (n = 513) or rituximab plus chemotherapy (n = 517) for 18 cycles; both groups then went on to receive rituximab maintenance therapy for 12 cycles. The total duration of treatment was 120 weeks. The median age of the combined groups was 59 years. The study was published in the New England Journal of Medicine.
One of the coprimary endpoints was complete response (confirmed or unconfirmed) by the end of the treatment period; the other was progression-free survival, which was planned to be assessed through three analyses, including two interim analyses, the first of which was reported in this study.
After a median follow-up of 37.9 months, the rates of coprimary endpoints were similar between the two groups. Complete response (confirmed or unconfirmed) was seen in 48% of the rituximab/lenalidomide group (95% confidence interval [CI], 44-53) and in 53% of the rituximab/chemotherapy group (95% CI, 49-57; P = .13). The hazard ratio for progression or death from any cause was 1.10 (95% CI, 0.85-1.43; P = .48).
In the subgroup analyses, the efficacy of rituximab plus chemotherapy was greater in low-risk patients (based on Follicular Lymphoma International Prognostic Index scores) and in patients whose follicular lymphoma was Ann Arbor stage I or II, whereas efficacy of rituximab/lenalidomide was independent of prognostic factors.
Safety was the biggest area of difference, with some events being more common in one group than in the other. For example, cutaneous reactions, diarrhea, rash, and myalgia were more common with rituximab/lenalidomide treatment, whereas anemia, fatigue, nausea, and febrile neutropenia were more common with rituximab/chemotherapy treatment. Among grade 3 or 4 events, cutaneous reactions were more common with rituximab/lenalidomide, and grade 3 or 4 neutropenia was more common with rituximab/chemotherapy.
“Overall, both treatment groups showed good outcomes, and a median has not yet been reached for either progression-free survival or overall survival,” the study authors wrote.
The RELEVANCE trial was sponsored by Celgene and the Lymphoma Academic Research Organisation. The study authors reported various disclosures, including financial ties to Celgene.
SOURCE: Morschhauser F et al. N Engl J Med. 2018;379:934-47.
Rituximab plus lenalidomide had efficacy similar to that of rituximab plus chemotherapy in treatment of follicular lymphoma, according to results from a phase 3 trial.
RELEVANCE (NCT01476787) was a multicenter, international, randomized, open-label trial designed to determine the superiority of rituximab/lenalidomide over rituximab/chemotherapy.
This trial randomized 1,030 patients with previously untreated follicular lymphoma to receive either rituximab plus lenalidomide (n = 513) or rituximab plus chemotherapy (n = 517) for 18 cycles; both groups then went on to receive rituximab maintenance therapy for 12 cycles. The total duration of treatment was 120 weeks. The median age of the combined groups was 59 years. The study was published in the New England Journal of Medicine.
One of the coprimary endpoints was complete response (confirmed or unconfirmed) by the end of the treatment period; the other was progression-free survival, which was planned to be assessed through three analyses, including two interim analyses, the first of which was reported in this study.
After a median follow-up of 37.9 months, the rates of coprimary endpoints were similar between the two groups. Complete response (confirmed or unconfirmed) was seen in 48% of the rituximab/lenalidomide group (95% confidence interval [CI], 44-53) and in 53% of the rituximab/chemotherapy group (95% CI, 49-57; P = .13). The hazard ratio for progression or death from any cause was 1.10 (95% CI, 0.85-1.43; P = .48).
In the subgroup analyses, the efficacy of rituximab plus chemotherapy was greater in low-risk patients (based on Follicular Lymphoma International Prognostic Index scores) and in patients whose follicular lymphoma was Ann Arbor stage I or II, whereas efficacy of rituximab/lenalidomide was independent of prognostic factors.
Safety was the biggest area of difference, with some events being more common in one group than in the other. For example, cutaneous reactions, diarrhea, rash, and myalgia were more common with rituximab/lenalidomide treatment, whereas anemia, fatigue, nausea, and febrile neutropenia were more common with rituximab/chemotherapy treatment. Among grade 3 or 4 events, cutaneous reactions were more common with rituximab/lenalidomide, and grade 3 or 4 neutropenia was more common with rituximab/chemotherapy.
“Overall, both treatment groups showed good outcomes, and a median has not yet been reached for either progression-free survival or overall survival,” the study authors wrote.
The RELEVANCE trial was sponsored by Celgene and the Lymphoma Academic Research Organisation. The study authors reported various disclosures, including financial ties to Celgene.
SOURCE: Morschhauser F et al. N Engl J Med. 2018;379:934-47.
FROM THE NEW ENGLAND JOURNAL OF MEDICINE
Key clinical point:
Major finding: Complete responses were seen in 48% of rituximab/lenalidomide patients versus 53% in the rituximab/chemotherapy patients (P = .13).
Study details: A phase 3 superiority trial of 1,030 patients with previously untreated follicular lymphoma.
Disclosures: Celgene and the Lymphoma Academic Research Organization funded the study. The authors reported various disclosures, including financial ties to Celgene.
Source: Morschhauser F et al. N Engl J Med. 2018;379:934-47.
Janssen submits NDA for esketamine nasal spray
Janssen has announced its submission of a new drug application to the Food and Drug Administration for esketamine nasal spray, which is intended for adult patients with treatment-resistant depression.
About 30% of people with depression do not respond to currently available interventions (Biol Psychiatry. 2016 Sep 15;80[6]:424-31). “This represents a major unmet public health need,” Mathai Mammen, MD, PhD, global head, Janssen Research & Development, said in a Sept. 4 press release announcing the NDA.
The application submitted by Janssen is based on data from five phase 3 trials, all of which demonstrated rapid reduction of depressive symptoms, as well as delayed time to relapse of symptoms, in patients with treatment-resistant depression, Janssen said in the release. Those studies compared treatment with esketamine plus a newly initiated oral antidepressant with that of placebo plus a newly initiated oral antidepressant. One of the studies evaluated long-term safety associated with esketamine treatment and found no new safety signals at 52 weeks of treatment, compared with those seen in short-term studies of the drug.
Previously, it received Breakthrough Therapy Designations for both treatment-resistant depression and major depressive disorder with imminent risk for suicide. Phase 3 clinical studies for the latter indication are ongoing. According to Janssen, the company plans to submit a Marketing Authorization Application to the European Medicines Agency later in 2018 for the treatment-resistant depression indication.
Janssen has announced its submission of a new drug application to the Food and Drug Administration for esketamine nasal spray, which is intended for adult patients with treatment-resistant depression.
About 30% of people with depression do not respond to currently available interventions (Biol Psychiatry. 2016 Sep 15;80[6]:424-31). “This represents a major unmet public health need,” Mathai Mammen, MD, PhD, global head, Janssen Research & Development, said in a Sept. 4 press release announcing the NDA.
The application submitted by Janssen is based on data from five phase 3 trials, all of which demonstrated rapid reduction of depressive symptoms, as well as delayed time to relapse of symptoms, in patients with treatment-resistant depression, Janssen said in the release. Those studies compared treatment with esketamine plus a newly initiated oral antidepressant with that of placebo plus a newly initiated oral antidepressant. One of the studies evaluated long-term safety associated with esketamine treatment and found no new safety signals at 52 weeks of treatment, compared with those seen in short-term studies of the drug.
Previously, it received Breakthrough Therapy Designations for both treatment-resistant depression and major depressive disorder with imminent risk for suicide. Phase 3 clinical studies for the latter indication are ongoing. According to Janssen, the company plans to submit a Marketing Authorization Application to the European Medicines Agency later in 2018 for the treatment-resistant depression indication.
Janssen has announced its submission of a new drug application to the Food and Drug Administration for esketamine nasal spray, which is intended for adult patients with treatment-resistant depression.
About 30% of people with depression do not respond to currently available interventions (Biol Psychiatry. 2016 Sep 15;80[6]:424-31). “This represents a major unmet public health need,” Mathai Mammen, MD, PhD, global head, Janssen Research & Development, said in a Sept. 4 press release announcing the NDA.
The application submitted by Janssen is based on data from five phase 3 trials, all of which demonstrated rapid reduction of depressive symptoms, as well as delayed time to relapse of symptoms, in patients with treatment-resistant depression, Janssen said in the release. Those studies compared treatment with esketamine plus a newly initiated oral antidepressant with that of placebo plus a newly initiated oral antidepressant. One of the studies evaluated long-term safety associated with esketamine treatment and found no new safety signals at 52 weeks of treatment, compared with those seen in short-term studies of the drug.
Previously, it received Breakthrough Therapy Designations for both treatment-resistant depression and major depressive disorder with imminent risk for suicide. Phase 3 clinical studies for the latter indication are ongoing. According to Janssen, the company plans to submit a Marketing Authorization Application to the European Medicines Agency later in 2018 for the treatment-resistant depression indication.
Risankizumab proves more effective in psoriasis than ustekinumab
, according to results of a pair of head-to-head trials published in the Lancet.
The replicate phase 3, randomized, double-blind, placebo- and active comparator–controlled trials, UltIMMa-1 (NCT02684370) and UltIMMa-2 (NCT02684375) altogether randomized 997 patients to risankizumab, ustekinumab, or placebo. The coprimary endpoints were the proportions of patients achieving 90% reduction in Psoriasis Area and Severity Index (PASI 90) at 16 weeks and a static Physician Global Assessment (sPGA) score of 0 or 1, and the 15 ranked secondary endpoints included proportions of those achieving PASI 100 or sPGA 0, both of which demonstrate total clearance of psoriasis, as well as measures of quality of life improvement.
Compared with those receiving either ustekinumab or placebo, a significantly higher proportion of patients receiving risankizumab achieved the coprimary endpoints, and all secondary endpoints were met. In UltIMMA-1, 75.3% of risankizumab patients achieved PASI 90, compared with 4.9% of placebo patients and 42% of ustekinumab patients (P less than .0001 when comparing it with both placebo and ustekinumab); sPGA of 0 or 1 was achieved by 87.8% of risankizumab patients and only 7.8% of placebo patients and 63% of ustekinumab patients (P less than .0001 when comparing it with both placebo and ustekinumab). Results were similar in UltIMMA-2: 74.8% of risankizumab patients achieved PASI 90, and 83.7% of them achieved sPGA 0 or 1 (P less than .0001 when comparing them with placebo and ustekinumab). According to results of the secondary endpoints, both studies also showed greater rates of clearance and improvements in quality of life among patients receiving risankizumab than among those receiving either placebo or ustekinumab.
The safety profiles across treatment groups were similar in both studies, with the most common adverse events including upper respiratory tract infection, headache, and diarrhea.
Risankizumab is a humanized IgG1 monoclonal antibody that targets the p19 subunit of only interleukin-23, unlike the studies’ active comparator, ustekinumab, which targets both interleukin-23 and interleukin-12. “Selectively blocking interleukin 23 with a p19 inhibitor appears to be one of the best ways to treat psoriasis,” commented Abigail Cline, MD, and Steven R. Feldman, MD, PhD, both of Wake Forest University, Winston-Salem, N.C., in an accompanying editorial (Lancet. 2018 Aug 7;392:616-71.).
The authors of the study reported relationships with various industry entities, including AbbVie, which sponsored the studies and developed risankizumab, and Boehringer Ingelheim, which collaborated in the studies. The authors of the editorial also disclosed relationships with entities, including AbbVie.
SOURCE: Gordon KB et al. Lancet. 2018 Aug 7;392:650-61.
, according to results of a pair of head-to-head trials published in the Lancet.
The replicate phase 3, randomized, double-blind, placebo- and active comparator–controlled trials, UltIMMa-1 (NCT02684370) and UltIMMa-2 (NCT02684375) altogether randomized 997 patients to risankizumab, ustekinumab, or placebo. The coprimary endpoints were the proportions of patients achieving 90% reduction in Psoriasis Area and Severity Index (PASI 90) at 16 weeks and a static Physician Global Assessment (sPGA) score of 0 or 1, and the 15 ranked secondary endpoints included proportions of those achieving PASI 100 or sPGA 0, both of which demonstrate total clearance of psoriasis, as well as measures of quality of life improvement.
Compared with those receiving either ustekinumab or placebo, a significantly higher proportion of patients receiving risankizumab achieved the coprimary endpoints, and all secondary endpoints were met. In UltIMMA-1, 75.3% of risankizumab patients achieved PASI 90, compared with 4.9% of placebo patients and 42% of ustekinumab patients (P less than .0001 when comparing it with both placebo and ustekinumab); sPGA of 0 or 1 was achieved by 87.8% of risankizumab patients and only 7.8% of placebo patients and 63% of ustekinumab patients (P less than .0001 when comparing it with both placebo and ustekinumab). Results were similar in UltIMMA-2: 74.8% of risankizumab patients achieved PASI 90, and 83.7% of them achieved sPGA 0 or 1 (P less than .0001 when comparing them with placebo and ustekinumab). According to results of the secondary endpoints, both studies also showed greater rates of clearance and improvements in quality of life among patients receiving risankizumab than among those receiving either placebo or ustekinumab.
The safety profiles across treatment groups were similar in both studies, with the most common adverse events including upper respiratory tract infection, headache, and diarrhea.
Risankizumab is a humanized IgG1 monoclonal antibody that targets the p19 subunit of only interleukin-23, unlike the studies’ active comparator, ustekinumab, which targets both interleukin-23 and interleukin-12. “Selectively blocking interleukin 23 with a p19 inhibitor appears to be one of the best ways to treat psoriasis,” commented Abigail Cline, MD, and Steven R. Feldman, MD, PhD, both of Wake Forest University, Winston-Salem, N.C., in an accompanying editorial (Lancet. 2018 Aug 7;392:616-71.).
The authors of the study reported relationships with various industry entities, including AbbVie, which sponsored the studies and developed risankizumab, and Boehringer Ingelheim, which collaborated in the studies. The authors of the editorial also disclosed relationships with entities, including AbbVie.
SOURCE: Gordon KB et al. Lancet. 2018 Aug 7;392:650-61.
, according to results of a pair of head-to-head trials published in the Lancet.
The replicate phase 3, randomized, double-blind, placebo- and active comparator–controlled trials, UltIMMa-1 (NCT02684370) and UltIMMa-2 (NCT02684375) altogether randomized 997 patients to risankizumab, ustekinumab, or placebo. The coprimary endpoints were the proportions of patients achieving 90% reduction in Psoriasis Area and Severity Index (PASI 90) at 16 weeks and a static Physician Global Assessment (sPGA) score of 0 or 1, and the 15 ranked secondary endpoints included proportions of those achieving PASI 100 or sPGA 0, both of which demonstrate total clearance of psoriasis, as well as measures of quality of life improvement.
Compared with those receiving either ustekinumab or placebo, a significantly higher proportion of patients receiving risankizumab achieved the coprimary endpoints, and all secondary endpoints were met. In UltIMMA-1, 75.3% of risankizumab patients achieved PASI 90, compared with 4.9% of placebo patients and 42% of ustekinumab patients (P less than .0001 when comparing it with both placebo and ustekinumab); sPGA of 0 or 1 was achieved by 87.8% of risankizumab patients and only 7.8% of placebo patients and 63% of ustekinumab patients (P less than .0001 when comparing it with both placebo and ustekinumab). Results were similar in UltIMMA-2: 74.8% of risankizumab patients achieved PASI 90, and 83.7% of them achieved sPGA 0 or 1 (P less than .0001 when comparing them with placebo and ustekinumab). According to results of the secondary endpoints, both studies also showed greater rates of clearance and improvements in quality of life among patients receiving risankizumab than among those receiving either placebo or ustekinumab.
The safety profiles across treatment groups were similar in both studies, with the most common adverse events including upper respiratory tract infection, headache, and diarrhea.
Risankizumab is a humanized IgG1 monoclonal antibody that targets the p19 subunit of only interleukin-23, unlike the studies’ active comparator, ustekinumab, which targets both interleukin-23 and interleukin-12. “Selectively blocking interleukin 23 with a p19 inhibitor appears to be one of the best ways to treat psoriasis,” commented Abigail Cline, MD, and Steven R. Feldman, MD, PhD, both of Wake Forest University, Winston-Salem, N.C., in an accompanying editorial (Lancet. 2018 Aug 7;392:616-71.).
The authors of the study reported relationships with various industry entities, including AbbVie, which sponsored the studies and developed risankizumab, and Boehringer Ingelheim, which collaborated in the studies. The authors of the editorial also disclosed relationships with entities, including AbbVie.
SOURCE: Gordon KB et al. Lancet. 2018 Aug 7;392:650-61.
FROM THE LANCET
USPSTF releases its first perinatal depression draft recommendations
It has given this draft recommendation a B grade, which means that there is moderate certainty of at least moderate net benefit. This is the first time the USPSTF has reviewed this topic.
It’s estimated that one in seven women are affected by perinatal depression, which can have negative effects on both the mothers affected and their children. In its review of evidence, the USPSTF found convincing evidence that counseling, particularly cognitive-behavioral and interpersonal therapies, can prevent perinatal depression.
The draft recommendation affects women who are pregnant or 1 year post partum; specifically, these women will not have a current diagnosis of depression but are at increased risk for it nonetheless. At this time, there is no accurate screening tool available to determine whether women have perinatal depression or are at increased risk. However, certain risk factors have been associated with it, including current stressful life events, personal or family history of depression, history of physical or sexual abuse, pregestational or gestational diabetes, complications during pregnancy such as hyperemesis or premature contractions, adolescent pregnancy, lack of social or financial support, and low socioeconomic status.
The USPSTF reviewed 50 trials considered to be of good or fair quality, with a mean participant age of 29 years; 26 included pregnant women, 22 included women post partum, and 2 included both. Counseling interventions were the most widely studied and “reduced the likelihood of the onset of perinatal depression by 39%” for a pooled risk ratio of 0.61 (95% confidence interval, 0.47-0.78), compared with control conditions. Two kinds of therapies stood out as especially effective: cognitive-behavioral and interpersonal. Cognitive-behavioral therapy focuses on negative thoughts, moods, behaviors, and attitudes and seeks to increase positive events and activities. Interpersonal therapy focuses on how interpersonal interactions and patterns can contribute to the development and maintenance of psychological issues and distress.
The comment period for these draft recommendations will continue through Sept. 24 at www.uspreventiveservicestaskforce.org/tfcomment.htm.
This article was updated 8/28/18.
It has given this draft recommendation a B grade, which means that there is moderate certainty of at least moderate net benefit. This is the first time the USPSTF has reviewed this topic.
It’s estimated that one in seven women are affected by perinatal depression, which can have negative effects on both the mothers affected and their children. In its review of evidence, the USPSTF found convincing evidence that counseling, particularly cognitive-behavioral and interpersonal therapies, can prevent perinatal depression.
The draft recommendation affects women who are pregnant or 1 year post partum; specifically, these women will not have a current diagnosis of depression but are at increased risk for it nonetheless. At this time, there is no accurate screening tool available to determine whether women have perinatal depression or are at increased risk. However, certain risk factors have been associated with it, including current stressful life events, personal or family history of depression, history of physical or sexual abuse, pregestational or gestational diabetes, complications during pregnancy such as hyperemesis or premature contractions, adolescent pregnancy, lack of social or financial support, and low socioeconomic status.
The USPSTF reviewed 50 trials considered to be of good or fair quality, with a mean participant age of 29 years; 26 included pregnant women, 22 included women post partum, and 2 included both. Counseling interventions were the most widely studied and “reduced the likelihood of the onset of perinatal depression by 39%” for a pooled risk ratio of 0.61 (95% confidence interval, 0.47-0.78), compared with control conditions. Two kinds of therapies stood out as especially effective: cognitive-behavioral and interpersonal. Cognitive-behavioral therapy focuses on negative thoughts, moods, behaviors, and attitudes and seeks to increase positive events and activities. Interpersonal therapy focuses on how interpersonal interactions and patterns can contribute to the development and maintenance of psychological issues and distress.
The comment period for these draft recommendations will continue through Sept. 24 at www.uspreventiveservicestaskforce.org/tfcomment.htm.
This article was updated 8/28/18.
It has given this draft recommendation a B grade, which means that there is moderate certainty of at least moderate net benefit. This is the first time the USPSTF has reviewed this topic.
It’s estimated that one in seven women are affected by perinatal depression, which can have negative effects on both the mothers affected and their children. In its review of evidence, the USPSTF found convincing evidence that counseling, particularly cognitive-behavioral and interpersonal therapies, can prevent perinatal depression.
The draft recommendation affects women who are pregnant or 1 year post partum; specifically, these women will not have a current diagnosis of depression but are at increased risk for it nonetheless. At this time, there is no accurate screening tool available to determine whether women have perinatal depression or are at increased risk. However, certain risk factors have been associated with it, including current stressful life events, personal or family history of depression, history of physical or sexual abuse, pregestational or gestational diabetes, complications during pregnancy such as hyperemesis or premature contractions, adolescent pregnancy, lack of social or financial support, and low socioeconomic status.
The USPSTF reviewed 50 trials considered to be of good or fair quality, with a mean participant age of 29 years; 26 included pregnant women, 22 included women post partum, and 2 included both. Counseling interventions were the most widely studied and “reduced the likelihood of the onset of perinatal depression by 39%” for a pooled risk ratio of 0.61 (95% confidence interval, 0.47-0.78), compared with control conditions. Two kinds of therapies stood out as especially effective: cognitive-behavioral and interpersonal. Cognitive-behavioral therapy focuses on negative thoughts, moods, behaviors, and attitudes and seeks to increase positive events and activities. Interpersonal therapy focuses on how interpersonal interactions and patterns can contribute to the development and maintenance of psychological issues and distress.
The comment period for these draft recommendations will continue through Sept. 24 at www.uspreventiveservicestaskforce.org/tfcomment.htm.
This article was updated 8/28/18.
FDA approves Diacomit for Dravet syndrome
The Food and Drug Administration has approved stiripentol (Diacomit) for the treatment of Dravet syndrome in patients aged 2 years and older who are taking clobazam.
Dravet syndrome is a rare form of epilepsy that presents within the first year as febrile seizures and then with other types of seizure later in life. Status epilepticus, a potentially life-threatening medical emergency, is a known risk. Also, developmental and interpersonal difficulties have been associated with this syndrome.
Because there are no clinical data for using stiripentol as monotherapy, it is indicated for use only in conjunction with clobazam. The recommended dosage for stiripentol is 50 mg/kg per day divided into either two or three doses, although the maximum recommended daily dose is 3,000 mg. It is available as either capsules or powder for oral solution, both of which come in either 250-mg or 500-mg forms.
Adverse reactions include somnolence, decreased appetite and weight, neutropenia and thrombocytopenia, tremor, nausea, and suicidal behavior and ideation. Because of the risk of neutropenia and thrombocytopenia, blood counts should be obtained via hematologic testing before starting stiripentol and again every 6 months. Also, as with most antiepileptic drugs, there is a risk of withdrawal symptoms, such as risk of increased seizure frequency and status epilepticus; as such, patients should not discontinue stiripentol without first consulting their health care professional.
Full prescribing information can be found on the FDA website.
The Food and Drug Administration has approved stiripentol (Diacomit) for the treatment of Dravet syndrome in patients aged 2 years and older who are taking clobazam.
Dravet syndrome is a rare form of epilepsy that presents within the first year as febrile seizures and then with other types of seizure later in life. Status epilepticus, a potentially life-threatening medical emergency, is a known risk. Also, developmental and interpersonal difficulties have been associated with this syndrome.
Because there are no clinical data for using stiripentol as monotherapy, it is indicated for use only in conjunction with clobazam. The recommended dosage for stiripentol is 50 mg/kg per day divided into either two or three doses, although the maximum recommended daily dose is 3,000 mg. It is available as either capsules or powder for oral solution, both of which come in either 250-mg or 500-mg forms.
Adverse reactions include somnolence, decreased appetite and weight, neutropenia and thrombocytopenia, tremor, nausea, and suicidal behavior and ideation. Because of the risk of neutropenia and thrombocytopenia, blood counts should be obtained via hematologic testing before starting stiripentol and again every 6 months. Also, as with most antiepileptic drugs, there is a risk of withdrawal symptoms, such as risk of increased seizure frequency and status epilepticus; as such, patients should not discontinue stiripentol without first consulting their health care professional.
Full prescribing information can be found on the FDA website.
The Food and Drug Administration has approved stiripentol (Diacomit) for the treatment of Dravet syndrome in patients aged 2 years and older who are taking clobazam.
Dravet syndrome is a rare form of epilepsy that presents within the first year as febrile seizures and then with other types of seizure later in life. Status epilepticus, a potentially life-threatening medical emergency, is a known risk. Also, developmental and interpersonal difficulties have been associated with this syndrome.
Because there are no clinical data for using stiripentol as monotherapy, it is indicated for use only in conjunction with clobazam. The recommended dosage for stiripentol is 50 mg/kg per day divided into either two or three doses, although the maximum recommended daily dose is 3,000 mg. It is available as either capsules or powder for oral solution, both of which come in either 250-mg or 500-mg forms.
Adverse reactions include somnolence, decreased appetite and weight, neutropenia and thrombocytopenia, tremor, nausea, and suicidal behavior and ideation. Because of the risk of neutropenia and thrombocytopenia, blood counts should be obtained via hematologic testing before starting stiripentol and again every 6 months. Also, as with most antiepileptic drugs, there is a risk of withdrawal symptoms, such as risk of increased seizure frequency and status epilepticus; as such, patients should not discontinue stiripentol without first consulting their health care professional.
Full prescribing information can be found on the FDA website.