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Age does not influence cladribine’s efficacy in MS
SEATTLE – In addition, age does not affect the likelihood that a patient who receives cladribine will achieve no evidence of disease activity (NEDA), according to a study presented at the annual meeting of the Consortium of Multiple Sclerosis Centers.
In the phase 3 CLARITY study, a cumulative dose of 3.5 mg/kg of cladribine over 2 years was associated with significantly reduced relapse rate and disability progression and improved MRI outcomes, compared with placebo. The drug’s efficacy persisted in patients who were switched to placebo in a 96-week extension study.
A post hoc analysis
A 2017 study by Weideman et al. suggested that disease-modifying treatment (DMT) is less effective in older patients. For this reason, Gavin Giovannoni, MBBCh, PhD, professor of neurology at Queen Mary University of London, and colleagues decided to investigate the effect of age on the efficacy of treatment with 3.5 mg/kg of cladribine. The investigators performed a post hoc analysis of the CLARITY and CLARITY extension studies of patients with relapsing-remitting MS. They categorized patients as older than 45 years or age 45 years or younger.
Patients enrolled in CLARITY were between ages 18 years and 65 years. They underwent MRI at pretrial assessment and at weeks 24, 48, and 96 or early termination. The investigators defined a qualifying relapse as one associated with changes in Kurtzke Functional Systems score and other specified clinical parameters. Qualifying relapses were confirmed by an independent evaluating physician who was blinded to treatment assignment.
In the CLARITY extension study, 98 participants who had received cladribine tablets 3.5 mg/kg in CLARITY were randomized to placebo for 2 additional years. Participants who continued on placebo in the CLARITY extension were evaluated according to age at entry into CLARITY.
Dr. Giovannoni and colleagues performed efficacy analyses for qualifying relapses; all relapses; and mean and cumulative numbers of new T1 gadolinium-enhancing lesions, active T2 lesions, and combined unique lesions. They defined NEDA as freedom from qualifying relapses, 6-month confirmed disability progression (as measured by the Expanded Disability Status Scale [EDSS] score), T1 gadolinium-enhancing lesions, and active T2 lesions. The investigators performed equivalent analyses for patients who received placebo in the CLARITY extension.
Age did not influence efficacy
Within each age group, participants in both treatment arms had similar baseline demographic and disease characteristics. In CLARITY, 221 patients were older than 45 years, and 649 were age 45 years or younger. In the CLARITY extension, 22 patients were older than 45 years, and 76 were age 45 years or younger. In CLARITY, but not the extension, the proportion of women was higher in the older group than in the younger group (77.7% vs. 66.1%). In CLARITY, patients aged 45 years or younger had a higher number of T1 gadolinium-enhancing lesions at baseline, compared with older patients.
At week 96 in CLARITY, the annual rate of qualifying relapses among patients older than 45 years was 0.14 for cladribine and 0.28 for placebo. Among patients aged 45 or younger, the annual rate of qualifying relapses was 0.15 for cladribine and 0.37 for placebo. For patients older than 45 years, the annual rate of all relapses was 0.28 for cladribine and 0.55 for placebo. For patients aged 45 years or younger, the annual rate of all relapses was 0.26 for cladribine and 0.65 for placebo. The treatment effect of cladribine, compared with placebo, on qualifying relapses and all relapses was similar for both age groups. In the CLARITY extension, the annualized relapse rate (ARR) was 0.17 in patients aged 45 years or younger and 0.05 in patients older than 45 years.
The mean number of new T1 gadolinium-enhancing lesions and cumulative new T1 gadolinium-enhancing lesions was reduced with cladribine, compared with placebo, in both age groups at week 96 in CLARITY. The mean number of active T2 lesions per patient per scan also was significantly reduced with cladribine, compared with placebo, in both age groups. The reduction was 0.167 in patients older than 45 years and 0.667 in patients aged 45 years and younger. In addition, the mean number of combined unique lesions per patient per scan also was significantly reduced with cladribine, compared with placebo, in both age groups. The reduction was 0.333 in patients older than 45 years and 0.667 in patients aged 45 years or younger.
The proportion of participants who achieved NEDA in CLARITY was 55.6% among patients older than 45 years who received cladribine, 39.6% among patients aged 45 years or younger who received cladribine, 28.2% among patients older than 45 years who received placebo, and 9.5% of patients aged 45 years or younger who received placebo. The odds ratio for achieving NEDA was significantly more favorable for the cladribine group, compared with the placebo group, in both age groups (3.19 for patients older than 45 years and 6.23 for patients aged 45 years or younger). In the CLARITY extension, the proportion of participants who achieved NEDA was 40.9% among patients older than 45 years and 28.2% among patients aged 45 years or younger.
“These data are consistent with previous analyses from CLARITY using a different age cutoff and with results from the overall study population,” said Dr. Giovannoni and colleagues.
Merck KGaA, which manufactures and markets cladribine, supported the study. Dr. Giovannoni and several of his coinvestigators have received speaker honoraria, consulting fees, or other funding from companies including Merck KGaA.
SEATTLE – In addition, age does not affect the likelihood that a patient who receives cladribine will achieve no evidence of disease activity (NEDA), according to a study presented at the annual meeting of the Consortium of Multiple Sclerosis Centers.
In the phase 3 CLARITY study, a cumulative dose of 3.5 mg/kg of cladribine over 2 years was associated with significantly reduced relapse rate and disability progression and improved MRI outcomes, compared with placebo. The drug’s efficacy persisted in patients who were switched to placebo in a 96-week extension study.
A post hoc analysis
A 2017 study by Weideman et al. suggested that disease-modifying treatment (DMT) is less effective in older patients. For this reason, Gavin Giovannoni, MBBCh, PhD, professor of neurology at Queen Mary University of London, and colleagues decided to investigate the effect of age on the efficacy of treatment with 3.5 mg/kg of cladribine. The investigators performed a post hoc analysis of the CLARITY and CLARITY extension studies of patients with relapsing-remitting MS. They categorized patients as older than 45 years or age 45 years or younger.
Patients enrolled in CLARITY were between ages 18 years and 65 years. They underwent MRI at pretrial assessment and at weeks 24, 48, and 96 or early termination. The investigators defined a qualifying relapse as one associated with changes in Kurtzke Functional Systems score and other specified clinical parameters. Qualifying relapses were confirmed by an independent evaluating physician who was blinded to treatment assignment.
In the CLARITY extension study, 98 participants who had received cladribine tablets 3.5 mg/kg in CLARITY were randomized to placebo for 2 additional years. Participants who continued on placebo in the CLARITY extension were evaluated according to age at entry into CLARITY.
Dr. Giovannoni and colleagues performed efficacy analyses for qualifying relapses; all relapses; and mean and cumulative numbers of new T1 gadolinium-enhancing lesions, active T2 lesions, and combined unique lesions. They defined NEDA as freedom from qualifying relapses, 6-month confirmed disability progression (as measured by the Expanded Disability Status Scale [EDSS] score), T1 gadolinium-enhancing lesions, and active T2 lesions. The investigators performed equivalent analyses for patients who received placebo in the CLARITY extension.
Age did not influence efficacy
Within each age group, participants in both treatment arms had similar baseline demographic and disease characteristics. In CLARITY, 221 patients were older than 45 years, and 649 were age 45 years or younger. In the CLARITY extension, 22 patients were older than 45 years, and 76 were age 45 years or younger. In CLARITY, but not the extension, the proportion of women was higher in the older group than in the younger group (77.7% vs. 66.1%). In CLARITY, patients aged 45 years or younger had a higher number of T1 gadolinium-enhancing lesions at baseline, compared with older patients.
At week 96 in CLARITY, the annual rate of qualifying relapses among patients older than 45 years was 0.14 for cladribine and 0.28 for placebo. Among patients aged 45 or younger, the annual rate of qualifying relapses was 0.15 for cladribine and 0.37 for placebo. For patients older than 45 years, the annual rate of all relapses was 0.28 for cladribine and 0.55 for placebo. For patients aged 45 years or younger, the annual rate of all relapses was 0.26 for cladribine and 0.65 for placebo. The treatment effect of cladribine, compared with placebo, on qualifying relapses and all relapses was similar for both age groups. In the CLARITY extension, the annualized relapse rate (ARR) was 0.17 in patients aged 45 years or younger and 0.05 in patients older than 45 years.
The mean number of new T1 gadolinium-enhancing lesions and cumulative new T1 gadolinium-enhancing lesions was reduced with cladribine, compared with placebo, in both age groups at week 96 in CLARITY. The mean number of active T2 lesions per patient per scan also was significantly reduced with cladribine, compared with placebo, in both age groups. The reduction was 0.167 in patients older than 45 years and 0.667 in patients aged 45 years and younger. In addition, the mean number of combined unique lesions per patient per scan also was significantly reduced with cladribine, compared with placebo, in both age groups. The reduction was 0.333 in patients older than 45 years and 0.667 in patients aged 45 years or younger.
The proportion of participants who achieved NEDA in CLARITY was 55.6% among patients older than 45 years who received cladribine, 39.6% among patients aged 45 years or younger who received cladribine, 28.2% among patients older than 45 years who received placebo, and 9.5% of patients aged 45 years or younger who received placebo. The odds ratio for achieving NEDA was significantly more favorable for the cladribine group, compared with the placebo group, in both age groups (3.19 for patients older than 45 years and 6.23 for patients aged 45 years or younger). In the CLARITY extension, the proportion of participants who achieved NEDA was 40.9% among patients older than 45 years and 28.2% among patients aged 45 years or younger.
“These data are consistent with previous analyses from CLARITY using a different age cutoff and with results from the overall study population,” said Dr. Giovannoni and colleagues.
Merck KGaA, which manufactures and markets cladribine, supported the study. Dr. Giovannoni and several of his coinvestigators have received speaker honoraria, consulting fees, or other funding from companies including Merck KGaA.
SEATTLE – In addition, age does not affect the likelihood that a patient who receives cladribine will achieve no evidence of disease activity (NEDA), according to a study presented at the annual meeting of the Consortium of Multiple Sclerosis Centers.
In the phase 3 CLARITY study, a cumulative dose of 3.5 mg/kg of cladribine over 2 years was associated with significantly reduced relapse rate and disability progression and improved MRI outcomes, compared with placebo. The drug’s efficacy persisted in patients who were switched to placebo in a 96-week extension study.
A post hoc analysis
A 2017 study by Weideman et al. suggested that disease-modifying treatment (DMT) is less effective in older patients. For this reason, Gavin Giovannoni, MBBCh, PhD, professor of neurology at Queen Mary University of London, and colleagues decided to investigate the effect of age on the efficacy of treatment with 3.5 mg/kg of cladribine. The investigators performed a post hoc analysis of the CLARITY and CLARITY extension studies of patients with relapsing-remitting MS. They categorized patients as older than 45 years or age 45 years or younger.
Patients enrolled in CLARITY were between ages 18 years and 65 years. They underwent MRI at pretrial assessment and at weeks 24, 48, and 96 or early termination. The investigators defined a qualifying relapse as one associated with changes in Kurtzke Functional Systems score and other specified clinical parameters. Qualifying relapses were confirmed by an independent evaluating physician who was blinded to treatment assignment.
In the CLARITY extension study, 98 participants who had received cladribine tablets 3.5 mg/kg in CLARITY were randomized to placebo for 2 additional years. Participants who continued on placebo in the CLARITY extension were evaluated according to age at entry into CLARITY.
Dr. Giovannoni and colleagues performed efficacy analyses for qualifying relapses; all relapses; and mean and cumulative numbers of new T1 gadolinium-enhancing lesions, active T2 lesions, and combined unique lesions. They defined NEDA as freedom from qualifying relapses, 6-month confirmed disability progression (as measured by the Expanded Disability Status Scale [EDSS] score), T1 gadolinium-enhancing lesions, and active T2 lesions. The investigators performed equivalent analyses for patients who received placebo in the CLARITY extension.
Age did not influence efficacy
Within each age group, participants in both treatment arms had similar baseline demographic and disease characteristics. In CLARITY, 221 patients were older than 45 years, and 649 were age 45 years or younger. In the CLARITY extension, 22 patients were older than 45 years, and 76 were age 45 years or younger. In CLARITY, but not the extension, the proportion of women was higher in the older group than in the younger group (77.7% vs. 66.1%). In CLARITY, patients aged 45 years or younger had a higher number of T1 gadolinium-enhancing lesions at baseline, compared with older patients.
At week 96 in CLARITY, the annual rate of qualifying relapses among patients older than 45 years was 0.14 for cladribine and 0.28 for placebo. Among patients aged 45 or younger, the annual rate of qualifying relapses was 0.15 for cladribine and 0.37 for placebo. For patients older than 45 years, the annual rate of all relapses was 0.28 for cladribine and 0.55 for placebo. For patients aged 45 years or younger, the annual rate of all relapses was 0.26 for cladribine and 0.65 for placebo. The treatment effect of cladribine, compared with placebo, on qualifying relapses and all relapses was similar for both age groups. In the CLARITY extension, the annualized relapse rate (ARR) was 0.17 in patients aged 45 years or younger and 0.05 in patients older than 45 years.
The mean number of new T1 gadolinium-enhancing lesions and cumulative new T1 gadolinium-enhancing lesions was reduced with cladribine, compared with placebo, in both age groups at week 96 in CLARITY. The mean number of active T2 lesions per patient per scan also was significantly reduced with cladribine, compared with placebo, in both age groups. The reduction was 0.167 in patients older than 45 years and 0.667 in patients aged 45 years and younger. In addition, the mean number of combined unique lesions per patient per scan also was significantly reduced with cladribine, compared with placebo, in both age groups. The reduction was 0.333 in patients older than 45 years and 0.667 in patients aged 45 years or younger.
The proportion of participants who achieved NEDA in CLARITY was 55.6% among patients older than 45 years who received cladribine, 39.6% among patients aged 45 years or younger who received cladribine, 28.2% among patients older than 45 years who received placebo, and 9.5% of patients aged 45 years or younger who received placebo. The odds ratio for achieving NEDA was significantly more favorable for the cladribine group, compared with the placebo group, in both age groups (3.19 for patients older than 45 years and 6.23 for patients aged 45 years or younger). In the CLARITY extension, the proportion of participants who achieved NEDA was 40.9% among patients older than 45 years and 28.2% among patients aged 45 years or younger.
“These data are consistent with previous analyses from CLARITY using a different age cutoff and with results from the overall study population,” said Dr. Giovannoni and colleagues.
Merck KGaA, which manufactures and markets cladribine, supported the study. Dr. Giovannoni and several of his coinvestigators have received speaker honoraria, consulting fees, or other funding from companies including Merck KGaA.
REPORTING FROM CMSC 2019
Ibudilast’s Efficacy Differs in Primary and Secondary Progressive MS
Key clinical point: Ibudilast’s treatment effect in a phase 2 trial for progressive multiple sclerosis (MS) primarily was driven by patients with primary progressive MS.
Major finding: The rate of brain atrophy for untreated patients with primary progressive MS was about twice as fast as that for those with secondary progressive MS.
Study details: The randomized, placebo-controlled phase 2 SPRINT-MS trial of ibudilast included 134 patients with primary progressive MS and 121 with secondary progressive MS.
Disclosures: The SPRINT-MS trial was funded by the National Institute of Neurological Disorders and Stroke. The National Multiple Sclerosis Society and MediciNova also supported the study. Dr. Goodman reported receiving research support from pharmaceutical companies, as well as personal compensation from companies for consulting, serving on a scientific advisory board, and speaking.
Citation: Goodman A et al. AAN 2019, Abstract S12.007.
Key clinical point: Ibudilast’s treatment effect in a phase 2 trial for progressive multiple sclerosis (MS) primarily was driven by patients with primary progressive MS.
Major finding: The rate of brain atrophy for untreated patients with primary progressive MS was about twice as fast as that for those with secondary progressive MS.
Study details: The randomized, placebo-controlled phase 2 SPRINT-MS trial of ibudilast included 134 patients with primary progressive MS and 121 with secondary progressive MS.
Disclosures: The SPRINT-MS trial was funded by the National Institute of Neurological Disorders and Stroke. The National Multiple Sclerosis Society and MediciNova also supported the study. Dr. Goodman reported receiving research support from pharmaceutical companies, as well as personal compensation from companies for consulting, serving on a scientific advisory board, and speaking.
Citation: Goodman A et al. AAN 2019, Abstract S12.007.
Key clinical point: Ibudilast’s treatment effect in a phase 2 trial for progressive multiple sclerosis (MS) primarily was driven by patients with primary progressive MS.
Major finding: The rate of brain atrophy for untreated patients with primary progressive MS was about twice as fast as that for those with secondary progressive MS.
Study details: The randomized, placebo-controlled phase 2 SPRINT-MS trial of ibudilast included 134 patients with primary progressive MS and 121 with secondary progressive MS.
Disclosures: The SPRINT-MS trial was funded by the National Institute of Neurological Disorders and Stroke. The National Multiple Sclerosis Society and MediciNova also supported the study. Dr. Goodman reported receiving research support from pharmaceutical companies, as well as personal compensation from companies for consulting, serving on a scientific advisory board, and speaking.
Citation: Goodman A et al. AAN 2019, Abstract S12.007.
Eculizumab Cuts Relapse Risk in NMO Spectrum Disorder
Key clinical point: Treatment with eculizumab substantially reduced risk of relapse versus placebo in patients with aquaporin-4 positive neuromyelitis optica spectrum disorder.
Major finding: Time to first adjudicated relapse on trial, the primary endpoint of the study, showed a significant (P less than .0001) effect in favor of monoclonal antibody treatment over placebo, with a 94.2% reduction in risk of relapse.
Study details: A phase 3, randomized, double-blind, placebo-controlled, multicenter trial (PREVENT) including 143 adult patients.
Disclosures: The study was supported by Alexion Pharmaceuticals. Dr. Pittock provided disclosures related to Alexion Pharmaceuticals, MedImmune, and Grifols, along with patents related to administration of eculizumab and cancer markers in neuromyelitis optica.
Citation: Pittock SJ et al. AAN 2019, Emerging Science Abstract 009.
Key clinical point: Treatment with eculizumab substantially reduced risk of relapse versus placebo in patients with aquaporin-4 positive neuromyelitis optica spectrum disorder.
Major finding: Time to first adjudicated relapse on trial, the primary endpoint of the study, showed a significant (P less than .0001) effect in favor of monoclonal antibody treatment over placebo, with a 94.2% reduction in risk of relapse.
Study details: A phase 3, randomized, double-blind, placebo-controlled, multicenter trial (PREVENT) including 143 adult patients.
Disclosures: The study was supported by Alexion Pharmaceuticals. Dr. Pittock provided disclosures related to Alexion Pharmaceuticals, MedImmune, and Grifols, along with patents related to administration of eculizumab and cancer markers in neuromyelitis optica.
Citation: Pittock SJ et al. AAN 2019, Emerging Science Abstract 009.
Key clinical point: Treatment with eculizumab substantially reduced risk of relapse versus placebo in patients with aquaporin-4 positive neuromyelitis optica spectrum disorder.
Major finding: Time to first adjudicated relapse on trial, the primary endpoint of the study, showed a significant (P less than .0001) effect in favor of monoclonal antibody treatment over placebo, with a 94.2% reduction in risk of relapse.
Study details: A phase 3, randomized, double-blind, placebo-controlled, multicenter trial (PREVENT) including 143 adult patients.
Disclosures: The study was supported by Alexion Pharmaceuticals. Dr. Pittock provided disclosures related to Alexion Pharmaceuticals, MedImmune, and Grifols, along with patents related to administration of eculizumab and cancer markers in neuromyelitis optica.
Citation: Pittock SJ et al. AAN 2019, Emerging Science Abstract 009.
Pediatric MS May Go Untreated in Year After Diagnosis
Key clinical point: Physicians face considerable uncertainty regarding how to treat pediatric patients with MS.
Major finding: About 65% of pediatric patients with multiple sclerosis do not receive disease-modifying therapy within 1 year of diagnosis.
Study details: Retrospective, observational study of claims data from 288 patients with pediatric MS.
Disclosures: Novartis funded the study, and Dr. Deshpande, who presented the findings, and a coauthor are employees of Novartis. Other coauthors reported consulting fees from Novartis, as well as consulting fees and grant funding from other pharmaceutical companies.
Citation: Greenberg B et al. CMSC 2019, Abstract DXM02.
Key clinical point: Physicians face considerable uncertainty regarding how to treat pediatric patients with MS.
Major finding: About 65% of pediatric patients with multiple sclerosis do not receive disease-modifying therapy within 1 year of diagnosis.
Study details: Retrospective, observational study of claims data from 288 patients with pediatric MS.
Disclosures: Novartis funded the study, and Dr. Deshpande, who presented the findings, and a coauthor are employees of Novartis. Other coauthors reported consulting fees from Novartis, as well as consulting fees and grant funding from other pharmaceutical companies.
Citation: Greenberg B et al. CMSC 2019, Abstract DXM02.
Key clinical point: Physicians face considerable uncertainty regarding how to treat pediatric patients with MS.
Major finding: About 65% of pediatric patients with multiple sclerosis do not receive disease-modifying therapy within 1 year of diagnosis.
Study details: Retrospective, observational study of claims data from 288 patients with pediatric MS.
Disclosures: Novartis funded the study, and Dr. Deshpande, who presented the findings, and a coauthor are employees of Novartis. Other coauthors reported consulting fees from Novartis, as well as consulting fees and grant funding from other pharmaceutical companies.
Citation: Greenberg B et al. CMSC 2019, Abstract DXM02.
Patient registry sheds light on the economic impact of MS
SEATTLE –
“MS seems to prevent people with MS from realizing their full potential at work or home,” said study coauthor Kottil Rammohan, MD, who summarized the study results in a video interview. Dr. Rammohan is professor of clinical neurology, director of the MS center of excellence, and chief of the multiple sclerosis division at the University of Miami. The study findings were presented at the annual meeting of the Consortium of Multiple Sclerosis Centers.
The North American Registry for Care and Research in Multiple Sclerosis (NARCRMS) prospectively collects information about the health care economics of patients with MS and its effects on daily life. In 2017, NARCRMS established the health care economics outcomes research (HEOR) advisory group. NARCRMS developed a Health-Related Productivity Questionnaire and Health Resource Utilization Questionnaire. The questionnaires were incorporated into the existing case report forms that are completed by patients at enrollment, annual, and exacerbation visits.
This analysis was based on 480 patients who had completed HEOR case report forms. Among those, 77% are employed either full or part time; however, of those 15% were underemployed, meaning they wanted to work more than their current work levels. About 13% are on disability.
“What we found was there was a significant impact at home as well,” said Dr. Rammohan. Patients reported that MS kept them from completing household chores. “MS is a disease that seems to impact not only the work environment, but also the home environment.”
When polled to determine the main reason why these MS patients are not able to function, “what we found was that it was not because of gait or immobility, it was difficulty related to fatigue,” Dr. Rammohan said. The second most common impairment was related to cognition.
“These are what we call the silent or the transparent symptoms of MS.”
Dr. Rammohan disclosed consulting fees from EMD Serono, Biogen, Sanofi-Aventis, Genzyme, Novartis, Teva Neurosciences, Acorda, and Roche/Genentech.
SEATTLE –
“MS seems to prevent people with MS from realizing their full potential at work or home,” said study coauthor Kottil Rammohan, MD, who summarized the study results in a video interview. Dr. Rammohan is professor of clinical neurology, director of the MS center of excellence, and chief of the multiple sclerosis division at the University of Miami. The study findings were presented at the annual meeting of the Consortium of Multiple Sclerosis Centers.
The North American Registry for Care and Research in Multiple Sclerosis (NARCRMS) prospectively collects information about the health care economics of patients with MS and its effects on daily life. In 2017, NARCRMS established the health care economics outcomes research (HEOR) advisory group. NARCRMS developed a Health-Related Productivity Questionnaire and Health Resource Utilization Questionnaire. The questionnaires were incorporated into the existing case report forms that are completed by patients at enrollment, annual, and exacerbation visits.
This analysis was based on 480 patients who had completed HEOR case report forms. Among those, 77% are employed either full or part time; however, of those 15% were underemployed, meaning they wanted to work more than their current work levels. About 13% are on disability.
“What we found was there was a significant impact at home as well,” said Dr. Rammohan. Patients reported that MS kept them from completing household chores. “MS is a disease that seems to impact not only the work environment, but also the home environment.”
When polled to determine the main reason why these MS patients are not able to function, “what we found was that it was not because of gait or immobility, it was difficulty related to fatigue,” Dr. Rammohan said. The second most common impairment was related to cognition.
“These are what we call the silent or the transparent symptoms of MS.”
Dr. Rammohan disclosed consulting fees from EMD Serono, Biogen, Sanofi-Aventis, Genzyme, Novartis, Teva Neurosciences, Acorda, and Roche/Genentech.
SEATTLE –
“MS seems to prevent people with MS from realizing their full potential at work or home,” said study coauthor Kottil Rammohan, MD, who summarized the study results in a video interview. Dr. Rammohan is professor of clinical neurology, director of the MS center of excellence, and chief of the multiple sclerosis division at the University of Miami. The study findings were presented at the annual meeting of the Consortium of Multiple Sclerosis Centers.
The North American Registry for Care and Research in Multiple Sclerosis (NARCRMS) prospectively collects information about the health care economics of patients with MS and its effects on daily life. In 2017, NARCRMS established the health care economics outcomes research (HEOR) advisory group. NARCRMS developed a Health-Related Productivity Questionnaire and Health Resource Utilization Questionnaire. The questionnaires were incorporated into the existing case report forms that are completed by patients at enrollment, annual, and exacerbation visits.
This analysis was based on 480 patients who had completed HEOR case report forms. Among those, 77% are employed either full or part time; however, of those 15% were underemployed, meaning they wanted to work more than their current work levels. About 13% are on disability.
“What we found was there was a significant impact at home as well,” said Dr. Rammohan. Patients reported that MS kept them from completing household chores. “MS is a disease that seems to impact not only the work environment, but also the home environment.”
When polled to determine the main reason why these MS patients are not able to function, “what we found was that it was not because of gait or immobility, it was difficulty related to fatigue,” Dr. Rammohan said. The second most common impairment was related to cognition.
“These are what we call the silent or the transparent symptoms of MS.”
Dr. Rammohan disclosed consulting fees from EMD Serono, Biogen, Sanofi-Aventis, Genzyme, Novartis, Teva Neurosciences, Acorda, and Roche/Genentech.
EXPERT ANALYSIS FROM CMSC 2019
Cannabis Misuse in MS Linked to Anxiety, Depression
Key clinical point: Providers should consider screening for hazardous cannabis use, especially if patients report depression, anxiety, or difficulty sleeping.
Major finding: Patients with MS who are depressed, anxious, or have poor sleep may be more likely to misuse or abuse cannabis.
Study details: 100 patients with a confirmed MS diagnosis who were receiving outpatient care in a university-affiliated MS center.
Disclosures: No study funding was reported and the authors report no relevant disclosures.
Citation: REPORTING FROM CMSC 2019
Key clinical point: Providers should consider screening for hazardous cannabis use, especially if patients report depression, anxiety, or difficulty sleeping.
Major finding: Patients with MS who are depressed, anxious, or have poor sleep may be more likely to misuse or abuse cannabis.
Study details: 100 patients with a confirmed MS diagnosis who were receiving outpatient care in a university-affiliated MS center.
Disclosures: No study funding was reported and the authors report no relevant disclosures.
Citation: REPORTING FROM CMSC 2019
Key clinical point: Providers should consider screening for hazardous cannabis use, especially if patients report depression, anxiety, or difficulty sleeping.
Major finding: Patients with MS who are depressed, anxious, or have poor sleep may be more likely to misuse or abuse cannabis.
Study details: 100 patients with a confirmed MS diagnosis who were receiving outpatient care in a university-affiliated MS center.
Disclosures: No study funding was reported and the authors report no relevant disclosures.
Citation: REPORTING FROM CMSC 2019
Lapses in DMT Increases Risk of Relapse in MS Patients
Key clinical point: Lapses in the use of MS disease-modifying oral therapy increases the risk for relapse, hospitalization, emergency department visits, and outpatient visits, and leads to higher healthcare costs.
Major finding: Over an 18-month follow-up period, those with drug lapses of more than 60 days had 28% more relapses than did the other subjects (mean 1.2 vs. 0.8; P less than .0001).
Study details: A claims database study of 8,779 patients with MS during 2011-2015
Disclosures: EMD Serono, a division of Merck KGaA, provided funding for the study. Dr. Nicholas disclosed grant support from EMD Serono, and two other study authors are employees of the company. Another two authors worked for a consulting firm that received funding from EMD Serono to conduct the study.
Citation: REPORTING FROM CMSC 2019
Key clinical point: Lapses in the use of MS disease-modifying oral therapy increases the risk for relapse, hospitalization, emergency department visits, and outpatient visits, and leads to higher healthcare costs.
Major finding: Over an 18-month follow-up period, those with drug lapses of more than 60 days had 28% more relapses than did the other subjects (mean 1.2 vs. 0.8; P less than .0001).
Study details: A claims database study of 8,779 patients with MS during 2011-2015
Disclosures: EMD Serono, a division of Merck KGaA, provided funding for the study. Dr. Nicholas disclosed grant support from EMD Serono, and two other study authors are employees of the company. Another two authors worked for a consulting firm that received funding from EMD Serono to conduct the study.
Citation: REPORTING FROM CMSC 2019
Key clinical point: Lapses in the use of MS disease-modifying oral therapy increases the risk for relapse, hospitalization, emergency department visits, and outpatient visits, and leads to higher healthcare costs.
Major finding: Over an 18-month follow-up period, those with drug lapses of more than 60 days had 28% more relapses than did the other subjects (mean 1.2 vs. 0.8; P less than .0001).
Study details: A claims database study of 8,779 patients with MS during 2011-2015
Disclosures: EMD Serono, a division of Merck KGaA, provided funding for the study. Dr. Nicholas disclosed grant support from EMD Serono, and two other study authors are employees of the company. Another two authors worked for a consulting firm that received funding from EMD Serono to conduct the study.
Citation: REPORTING FROM CMSC 2019
Adherence to Oral Treatments for MS is Poor
Key clinical point: Adherence to current oral therapies for multiple sclerosis (MS) is poor.
Major finding: Almost half of patients with MS discontinue their initial oral therapy.
Study details: A retrospective administrative claims study of 8,251 patients with MS.
Disclosures: The authors received no financial support for this study. Dr. Nicholas reported receiving grant support from EMD Serono.
Citation: Nicholas J et al. CMSC 2019. Abstract DXT34.
Key clinical point: Adherence to current oral therapies for multiple sclerosis (MS) is poor.
Major finding: Almost half of patients with MS discontinue their initial oral therapy.
Study details: A retrospective administrative claims study of 8,251 patients with MS.
Disclosures: The authors received no financial support for this study. Dr. Nicholas reported receiving grant support from EMD Serono.
Citation: Nicholas J et al. CMSC 2019. Abstract DXT34.
Key clinical point: Adherence to current oral therapies for multiple sclerosis (MS) is poor.
Major finding: Almost half of patients with MS discontinue their initial oral therapy.
Study details: A retrospective administrative claims study of 8,251 patients with MS.
Disclosures: The authors received no financial support for this study. Dr. Nicholas reported receiving grant support from EMD Serono.
Citation: Nicholas J et al. CMSC 2019. Abstract DXT34.
In MS, the challenges for women are unique
SEATTLE – Mitzi Joi Williams, MD.
About three in four people with MS are female – about 750,000 in the United States. And the risk and incidence may be highest in African American women.
In a presentation about the unique needs of women with MS, Dr. Williams, an assistant professor of internal medicine at the Morehouse School of Medicine in Atlanta, offered these tips at the annual meeting of the Consortium of Multiple Sclerosis Centers.
Pay attention to sexual dysfunction
Patients with MS often are ashamed to talk about sexual dysfunction, Dr. Williams said, but it is on many minds. “If I have a program on intimacy in MS, people are out the door.”
She urged colleagues to understand that MS can affect sexuality through three routes: primary, secondary, and tertiary dysfunction.
In primary sexual dysfunction, brain and spinal lesions directly related to MS can cause problems such as lack of sensation or abnormal sensations, decreased libido, vaginal dryness, and difficult orgasm.
Secondary sexual dysfunction refers to problems caused by symptoms of MS such as fatigue, which can worsen as the day progresses and affect nighttime intimacy, she said. Bladder dysfunction is another sensitive area in sexuality, with patients – especially women – “concerned that they will lose control of their bladder or they have already lost control.”
Cognitive dysfunction also can disrupt sexual function. “It is important to focus, and certain things cannot happen if you do not. If you are not able to focus and concentrate, it can affect interest,” Dr. Williams said.
Additionally, medications can improve some symptoms while making others worse. For example, a drug may relieve spasticity but boost fatigue. “We have to walk this tightrope,” she said. “But if we are not asking our patients, they may not volunteer this information.”
Finally, she said, MS can spark tertiary sexual dysfunction – poor body image, depression, anxiety, and disruptive changes in familial roles. For example, one partner may become a caregiver, and “it is hard to go from caregiving to sexy time.”
“It is something we have to acknowledge and find ways to deal with,” Dr. Williams said.
To address these issues, she pointed to strategies for symptomatic relief and disease-modifying therapy (DMT) and pinpointed several treatment options.
- Fatigue – stimulants, diet, exercise.
- Spasticity – muscle relaxants, exercise.
- Bladder dysfunction – fluid restriction, medication.
- Paresthesia – antidepressants, anticonvulsants.
- Numbness – vibrators, devices to increase stimulation.
Sexual therapy, couples therapy, and pelvic floor physical therapy also can be helpful.
Be aware of special needs during prepregnancy and pregnancy
“MS itself does not have a lot of effects on fertility, pregnancy, or pregnancy outcomes,” Dr. Williams said. However, “medications cause concern about how we manage pregnancy and fertility.”
In vitro fertilization may increase the risk of relapse, she added, and patients on dimethyl fumarate who experience vomiting or diarrhea may not be able to properly absorb oral contraceptives.
Women with MS may not need to go off DMT when they are trying to conceive, she said. “If patients have very aggressive disease, they may need to be on DMT through conception, through the first trimester, and even the entire pregnancy to prevent long-term disability.”
What about pregnancy itself? “An MS diagnosis alone does not mean that a pregnancy is high risk,” she said. “There are not necessarily additional tests and ultrasounds that are recommended for our patients based on MS diagnosis alone.”
Treatment discontinuation may be warranted during pregnancy, when MS generally improves. However, some MS symptoms – fatigue, bladder dysfunction, and balance – may increase. Corticosteroids can be appropriate if relapses occur during pregnancy.
Menopause and MS symptoms may overlap
Symptoms such as hot flashes, mood changes, sleep disturbance, bladder dysfunction, and decreased energy may be signs of MS, or they could indicate menopause, Dr. Williams said. “Sometimes patients come in and they are getting worse, and we look into it and discover they are premenopausal.”
A decline in estrogen during menopause may worsen MS symptoms, she added, and hormone therapy may be appropriate. A phase 2 study found a benefit in menopausal patients with MS for estriol in conjunction with a DMT, but more studies are needed.
Dr. Williams reported no relevant financial disclosures.
SEATTLE – Mitzi Joi Williams, MD.
About three in four people with MS are female – about 750,000 in the United States. And the risk and incidence may be highest in African American women.
In a presentation about the unique needs of women with MS, Dr. Williams, an assistant professor of internal medicine at the Morehouse School of Medicine in Atlanta, offered these tips at the annual meeting of the Consortium of Multiple Sclerosis Centers.
Pay attention to sexual dysfunction
Patients with MS often are ashamed to talk about sexual dysfunction, Dr. Williams said, but it is on many minds. “If I have a program on intimacy in MS, people are out the door.”
She urged colleagues to understand that MS can affect sexuality through three routes: primary, secondary, and tertiary dysfunction.
In primary sexual dysfunction, brain and spinal lesions directly related to MS can cause problems such as lack of sensation or abnormal sensations, decreased libido, vaginal dryness, and difficult orgasm.
Secondary sexual dysfunction refers to problems caused by symptoms of MS such as fatigue, which can worsen as the day progresses and affect nighttime intimacy, she said. Bladder dysfunction is another sensitive area in sexuality, with patients – especially women – “concerned that they will lose control of their bladder or they have already lost control.”
Cognitive dysfunction also can disrupt sexual function. “It is important to focus, and certain things cannot happen if you do not. If you are not able to focus and concentrate, it can affect interest,” Dr. Williams said.
Additionally, medications can improve some symptoms while making others worse. For example, a drug may relieve spasticity but boost fatigue. “We have to walk this tightrope,” she said. “But if we are not asking our patients, they may not volunteer this information.”
Finally, she said, MS can spark tertiary sexual dysfunction – poor body image, depression, anxiety, and disruptive changes in familial roles. For example, one partner may become a caregiver, and “it is hard to go from caregiving to sexy time.”
“It is something we have to acknowledge and find ways to deal with,” Dr. Williams said.
To address these issues, she pointed to strategies for symptomatic relief and disease-modifying therapy (DMT) and pinpointed several treatment options.
- Fatigue – stimulants, diet, exercise.
- Spasticity – muscle relaxants, exercise.
- Bladder dysfunction – fluid restriction, medication.
- Paresthesia – antidepressants, anticonvulsants.
- Numbness – vibrators, devices to increase stimulation.
Sexual therapy, couples therapy, and pelvic floor physical therapy also can be helpful.
Be aware of special needs during prepregnancy and pregnancy
“MS itself does not have a lot of effects on fertility, pregnancy, or pregnancy outcomes,” Dr. Williams said. However, “medications cause concern about how we manage pregnancy and fertility.”
In vitro fertilization may increase the risk of relapse, she added, and patients on dimethyl fumarate who experience vomiting or diarrhea may not be able to properly absorb oral contraceptives.
Women with MS may not need to go off DMT when they are trying to conceive, she said. “If patients have very aggressive disease, they may need to be on DMT through conception, through the first trimester, and even the entire pregnancy to prevent long-term disability.”
What about pregnancy itself? “An MS diagnosis alone does not mean that a pregnancy is high risk,” she said. “There are not necessarily additional tests and ultrasounds that are recommended for our patients based on MS diagnosis alone.”
Treatment discontinuation may be warranted during pregnancy, when MS generally improves. However, some MS symptoms – fatigue, bladder dysfunction, and balance – may increase. Corticosteroids can be appropriate if relapses occur during pregnancy.
Menopause and MS symptoms may overlap
Symptoms such as hot flashes, mood changes, sleep disturbance, bladder dysfunction, and decreased energy may be signs of MS, or they could indicate menopause, Dr. Williams said. “Sometimes patients come in and they are getting worse, and we look into it and discover they are premenopausal.”
A decline in estrogen during menopause may worsen MS symptoms, she added, and hormone therapy may be appropriate. A phase 2 study found a benefit in menopausal patients with MS for estriol in conjunction with a DMT, but more studies are needed.
Dr. Williams reported no relevant financial disclosures.
SEATTLE – Mitzi Joi Williams, MD.
About three in four people with MS are female – about 750,000 in the United States. And the risk and incidence may be highest in African American women.
In a presentation about the unique needs of women with MS, Dr. Williams, an assistant professor of internal medicine at the Morehouse School of Medicine in Atlanta, offered these tips at the annual meeting of the Consortium of Multiple Sclerosis Centers.
Pay attention to sexual dysfunction
Patients with MS often are ashamed to talk about sexual dysfunction, Dr. Williams said, but it is on many minds. “If I have a program on intimacy in MS, people are out the door.”
She urged colleagues to understand that MS can affect sexuality through three routes: primary, secondary, and tertiary dysfunction.
In primary sexual dysfunction, brain and spinal lesions directly related to MS can cause problems such as lack of sensation or abnormal sensations, decreased libido, vaginal dryness, and difficult orgasm.
Secondary sexual dysfunction refers to problems caused by symptoms of MS such as fatigue, which can worsen as the day progresses and affect nighttime intimacy, she said. Bladder dysfunction is another sensitive area in sexuality, with patients – especially women – “concerned that they will lose control of their bladder or they have already lost control.”
Cognitive dysfunction also can disrupt sexual function. “It is important to focus, and certain things cannot happen if you do not. If you are not able to focus and concentrate, it can affect interest,” Dr. Williams said.
Additionally, medications can improve some symptoms while making others worse. For example, a drug may relieve spasticity but boost fatigue. “We have to walk this tightrope,” she said. “But if we are not asking our patients, they may not volunteer this information.”
Finally, she said, MS can spark tertiary sexual dysfunction – poor body image, depression, anxiety, and disruptive changes in familial roles. For example, one partner may become a caregiver, and “it is hard to go from caregiving to sexy time.”
“It is something we have to acknowledge and find ways to deal with,” Dr. Williams said.
To address these issues, she pointed to strategies for symptomatic relief and disease-modifying therapy (DMT) and pinpointed several treatment options.
- Fatigue – stimulants, diet, exercise.
- Spasticity – muscle relaxants, exercise.
- Bladder dysfunction – fluid restriction, medication.
- Paresthesia – antidepressants, anticonvulsants.
- Numbness – vibrators, devices to increase stimulation.
Sexual therapy, couples therapy, and pelvic floor physical therapy also can be helpful.
Be aware of special needs during prepregnancy and pregnancy
“MS itself does not have a lot of effects on fertility, pregnancy, or pregnancy outcomes,” Dr. Williams said. However, “medications cause concern about how we manage pregnancy and fertility.”
In vitro fertilization may increase the risk of relapse, she added, and patients on dimethyl fumarate who experience vomiting or diarrhea may not be able to properly absorb oral contraceptives.
Women with MS may not need to go off DMT when they are trying to conceive, she said. “If patients have very aggressive disease, they may need to be on DMT through conception, through the first trimester, and even the entire pregnancy to prevent long-term disability.”
What about pregnancy itself? “An MS diagnosis alone does not mean that a pregnancy is high risk,” she said. “There are not necessarily additional tests and ultrasounds that are recommended for our patients based on MS diagnosis alone.”
Treatment discontinuation may be warranted during pregnancy, when MS generally improves. However, some MS symptoms – fatigue, bladder dysfunction, and balance – may increase. Corticosteroids can be appropriate if relapses occur during pregnancy.
Menopause and MS symptoms may overlap
Symptoms such as hot flashes, mood changes, sleep disturbance, bladder dysfunction, and decreased energy may be signs of MS, or they could indicate menopause, Dr. Williams said. “Sometimes patients come in and they are getting worse, and we look into it and discover they are premenopausal.”
A decline in estrogen during menopause may worsen MS symptoms, she added, and hormone therapy may be appropriate. A phase 2 study found a benefit in menopausal patients with MS for estriol in conjunction with a DMT, but more studies are needed.
Dr. Williams reported no relevant financial disclosures.
EXPERT ANALYSIS FROM CMSC 2019
Pediatric MS often goes untreated in the year after diagnosis
SEATTLE – Females may be more likely than males to receive DMT during this time, said Chinmay Deshpande, PhD, at the annual meeting of the Consortium of Multiple Sclerosis Centers.
Pediatric onset of MS occurs in 3-5% of patients with the disease, and the median age of pediatric onset is 15 years. This population tends to have a high relapse rate and may develop disability at a younger age, said Dr. Deshpande, associate director of health economics and outcomes research at Novartis. “There have been very few studies done on this population, especially in the clinical trial setting. ... Physicians face considerable uncertainty in how to treat these patients.”
Observational data
To assess the proportion of patients with pediatric MS who receive DMT in the year after diagnosis, Dr. Deshpande and colleagues analyzed retrospective observational data from the Truven Health Marketscan Commercial and Encounters administrative claims databases. They studied patients who received an MS diagnosis between Jan. 1, 2010, and Dec. 31, 2016. In addition, they examined which DMTs were used as first-line therapies, whether prescribing patterns changed between 2010 and 2017, and time to treatment discontinuation or switch.
The databases included data from more than 182,000 patients with two or more claims of MS diagnosis. After including only patients age 17 years or younger at the index diagnosis date who had insurance during the 6 months prior to the index date and 12 months after the index date and who did not use DMT during the 6 months prior to the index date, 288 patients remained in the analysis. Patients had an average age of about 14 years.
The primary outcome was the proportion of patients who started DMT in the year after MS diagnosis. “The proportion of untreated patients within their first year of diagnosis was around 65%,” said Dr. Deshpande. On average, treated patients were slightly older than untreated patients (15.0 years vs. 13.3 years). Among treated patients, 75% were female, and 25% were male. Overall, however, 61% were female and 39% were male. The difference in treatment by gender was surprising and the reason for it is not understood, Dr. Deshpande said. One possibility is that the difference relates to earlier maturation in females, but that is only a hypothesis, he said.
Glatiramer acetate and interferon beta-1a were first-line DMTs for 48% and 30.6% of the treated patients, respectively. Dimethyl fumarate (7.1%), natalizumab (5.1%), fingolimod (4.1%), interferon beta-1b (4.1%), and peginterferon beta-1a (1%) also were used as first-line therapy.
Twenty percent of patients who received DMT switched to another medication during the follow-up period. The median time of switching was within 6 months of starting first-line therapy. Most patients who discontinued DMT – that is, they did not have any DMT for 60 days after stopping their first DMT – did so within 10 months of diagnosis.
Use of newer medications
Overall, the use of glatiramer acetate and interferons has decreased over time, and while the use of newer DMTs has increased, the trend is not consistent. “With the growing uptake of newer oral and infusible DMTs over the recent years, there is a need to increase treatment awareness in the pediatric MS population and to inform currently approved treatment options to the prescribers,” Dr. Deshpande and colleagues said.
The claims database is generalizable and nationally representative, but it does not include clinical or MRI data. “It’s hard to understand the reasoning why they discontinued or why they are switching,” Dr. Deshpande said. In addition, the sample size was relatively small, and the results should be interpreted accordingly, he said.
Novartis funded the study, and Dr. Deshpande and a coauthor are employees of Novartis. Other coauthors reported consulting fees from Novartis, as well as consulting fees and grant funding from other pharmaceutical companies.
SOURCE: Greenberg B et al. CMSC 2019, Abstract DXM02.
SEATTLE – Females may be more likely than males to receive DMT during this time, said Chinmay Deshpande, PhD, at the annual meeting of the Consortium of Multiple Sclerosis Centers.
Pediatric onset of MS occurs in 3-5% of patients with the disease, and the median age of pediatric onset is 15 years. This population tends to have a high relapse rate and may develop disability at a younger age, said Dr. Deshpande, associate director of health economics and outcomes research at Novartis. “There have been very few studies done on this population, especially in the clinical trial setting. ... Physicians face considerable uncertainty in how to treat these patients.”
Observational data
To assess the proportion of patients with pediatric MS who receive DMT in the year after diagnosis, Dr. Deshpande and colleagues analyzed retrospective observational data from the Truven Health Marketscan Commercial and Encounters administrative claims databases. They studied patients who received an MS diagnosis between Jan. 1, 2010, and Dec. 31, 2016. In addition, they examined which DMTs were used as first-line therapies, whether prescribing patterns changed between 2010 and 2017, and time to treatment discontinuation or switch.
The databases included data from more than 182,000 patients with two or more claims of MS diagnosis. After including only patients age 17 years or younger at the index diagnosis date who had insurance during the 6 months prior to the index date and 12 months after the index date and who did not use DMT during the 6 months prior to the index date, 288 patients remained in the analysis. Patients had an average age of about 14 years.
The primary outcome was the proportion of patients who started DMT in the year after MS diagnosis. “The proportion of untreated patients within their first year of diagnosis was around 65%,” said Dr. Deshpande. On average, treated patients were slightly older than untreated patients (15.0 years vs. 13.3 years). Among treated patients, 75% were female, and 25% were male. Overall, however, 61% were female and 39% were male. The difference in treatment by gender was surprising and the reason for it is not understood, Dr. Deshpande said. One possibility is that the difference relates to earlier maturation in females, but that is only a hypothesis, he said.
Glatiramer acetate and interferon beta-1a were first-line DMTs for 48% and 30.6% of the treated patients, respectively. Dimethyl fumarate (7.1%), natalizumab (5.1%), fingolimod (4.1%), interferon beta-1b (4.1%), and peginterferon beta-1a (1%) also were used as first-line therapy.
Twenty percent of patients who received DMT switched to another medication during the follow-up period. The median time of switching was within 6 months of starting first-line therapy. Most patients who discontinued DMT – that is, they did not have any DMT for 60 days after stopping their first DMT – did so within 10 months of diagnosis.
Use of newer medications
Overall, the use of glatiramer acetate and interferons has decreased over time, and while the use of newer DMTs has increased, the trend is not consistent. “With the growing uptake of newer oral and infusible DMTs over the recent years, there is a need to increase treatment awareness in the pediatric MS population and to inform currently approved treatment options to the prescribers,” Dr. Deshpande and colleagues said.
The claims database is generalizable and nationally representative, but it does not include clinical or MRI data. “It’s hard to understand the reasoning why they discontinued or why they are switching,” Dr. Deshpande said. In addition, the sample size was relatively small, and the results should be interpreted accordingly, he said.
Novartis funded the study, and Dr. Deshpande and a coauthor are employees of Novartis. Other coauthors reported consulting fees from Novartis, as well as consulting fees and grant funding from other pharmaceutical companies.
SOURCE: Greenberg B et al. CMSC 2019, Abstract DXM02.
SEATTLE – Females may be more likely than males to receive DMT during this time, said Chinmay Deshpande, PhD, at the annual meeting of the Consortium of Multiple Sclerosis Centers.
Pediatric onset of MS occurs in 3-5% of patients with the disease, and the median age of pediatric onset is 15 years. This population tends to have a high relapse rate and may develop disability at a younger age, said Dr. Deshpande, associate director of health economics and outcomes research at Novartis. “There have been very few studies done on this population, especially in the clinical trial setting. ... Physicians face considerable uncertainty in how to treat these patients.”
Observational data
To assess the proportion of patients with pediatric MS who receive DMT in the year after diagnosis, Dr. Deshpande and colleagues analyzed retrospective observational data from the Truven Health Marketscan Commercial and Encounters administrative claims databases. They studied patients who received an MS diagnosis between Jan. 1, 2010, and Dec. 31, 2016. In addition, they examined which DMTs were used as first-line therapies, whether prescribing patterns changed between 2010 and 2017, and time to treatment discontinuation or switch.
The databases included data from more than 182,000 patients with two or more claims of MS diagnosis. After including only patients age 17 years or younger at the index diagnosis date who had insurance during the 6 months prior to the index date and 12 months after the index date and who did not use DMT during the 6 months prior to the index date, 288 patients remained in the analysis. Patients had an average age of about 14 years.
The primary outcome was the proportion of patients who started DMT in the year after MS diagnosis. “The proportion of untreated patients within their first year of diagnosis was around 65%,” said Dr. Deshpande. On average, treated patients were slightly older than untreated patients (15.0 years vs. 13.3 years). Among treated patients, 75% were female, and 25% were male. Overall, however, 61% were female and 39% were male. The difference in treatment by gender was surprising and the reason for it is not understood, Dr. Deshpande said. One possibility is that the difference relates to earlier maturation in females, but that is only a hypothesis, he said.
Glatiramer acetate and interferon beta-1a were first-line DMTs for 48% and 30.6% of the treated patients, respectively. Dimethyl fumarate (7.1%), natalizumab (5.1%), fingolimod (4.1%), interferon beta-1b (4.1%), and peginterferon beta-1a (1%) also were used as first-line therapy.
Twenty percent of patients who received DMT switched to another medication during the follow-up period. The median time of switching was within 6 months of starting first-line therapy. Most patients who discontinued DMT – that is, they did not have any DMT for 60 days after stopping their first DMT – did so within 10 months of diagnosis.
Use of newer medications
Overall, the use of glatiramer acetate and interferons has decreased over time, and while the use of newer DMTs has increased, the trend is not consistent. “With the growing uptake of newer oral and infusible DMTs over the recent years, there is a need to increase treatment awareness in the pediatric MS population and to inform currently approved treatment options to the prescribers,” Dr. Deshpande and colleagues said.
The claims database is generalizable and nationally representative, but it does not include clinical or MRI data. “It’s hard to understand the reasoning why they discontinued or why they are switching,” Dr. Deshpande said. In addition, the sample size was relatively small, and the results should be interpreted accordingly, he said.
Novartis funded the study, and Dr. Deshpande and a coauthor are employees of Novartis. Other coauthors reported consulting fees from Novartis, as well as consulting fees and grant funding from other pharmaceutical companies.
SOURCE: Greenberg B et al. CMSC 2019, Abstract DXM02.
REPORTING FROM CMSC 2019