Contributions
Abstract: P1222
Type: Poster Sessions
Abstract Category: Therapy - Neuroprotection and Repair
Background: In the MS-SPI study, MD1003 (high dose Pharmaceutical grade Biotin)treatment was shown to be effective and well-toleratedin patients (pts) with progressive multiple sclerosis (PMS) (Tourbah et al., 2016). Notably, MD1003 reversed MS-related disease disability in 13% of pts with PMS. MD1003 is currently being prescribed to pts with PMS in France under an expanded access programme.
Objective: To determine the benefits, in terms of effectiveness and safety, of MD1003 in pts with primary PMS (PPMS) or secondary PMS (SPMS) in a real-world clinical centre.
Methods: MD1003 300mg/day (100mg TID) was prescribed to pts with PPMS or SPMS being cared for at a single centre in France (CHU-Toulouse) starting in January 2016.The following measures of effectiveness and safety are being collected: Expanded Disability Status Scale (EDSS), timed 25-foot walk (T25W), 9-hole peg test (9-HPT), number of relapses, and gadolinium (Gd)-enhancing lesions on T1-weighted images.
Results: To date (May 2018), a total of 220 pts have received MD1003; results presented here correspond to the first 91 pts that have 1 year of follow-up. At baseline (month [M] 0), mean age was 59.5 years (SD=9.3), 61.5% were females, 70.3% had SPMS, mean EDSS was 5.9 (SD=1.3), mean TW25 was 50.7 seconds (SD=69.3), 9-HPT in the dominant hand was 35.1 (SD=20.7), and the mean number of previous relapses was 5.1 (SD=4.8). After 1 year of treatment with MD1003, 19 (23%; n=83 with data) pts experienced improvement in EDSS and 15 (23%; n=66 with data) pts experienced ≥20% improvement in T25W. Active disease, a clinically-defined relapse and/or a Gd-enhancing T1 lesion, was observed in 9 (11%; n=79 with data) pts.
Conclusions: MD1003 300mg/day was effective in the treatment of PMS in a real-world clinical setting; at 1 year of treatment, 23% of pts experienced improvement in MS-related disease activity (as measured by EDSS or TW25). MD1003 was also well-tolerated. This real-world study supports the growing body of evidence that MD 1003 is an effective and safe treatment for PMS.
Disclosure: J. Ciron: nothing to disclose., B. Pignolet: nothing to disclose., F. Bucciarelli: nothing to disclose., F. Lerebours: nothing to disclose., G. Dorcet: nothing to disclose., C. Rabadeux: nothing to disclose., N. Freitas: nothing to disclose., F. Bonneville: nothing to disclose., D. Biotti received speaker honoraria from Teva, Merck, Roche, Biogen, Novartis., D. Brassat received research grant from EU FP7, 305477-2012, EU Marie Curie action 2008 (Co I) ABIRISK (co I), French ministery of health -2008 (PHRC), French MS society 2009, 2012, 2017. Lectures, congress invitation, board participation with Bayer, Biogen, Medday, Merck, Novartis, Roche, Sanofi-Genzyme and Teva
Abstract: P1222
Type: Poster Sessions
Abstract Category: Therapy - Neuroprotection and Repair
Background: In the MS-SPI study, MD1003 (high dose Pharmaceutical grade Biotin)treatment was shown to be effective and well-toleratedin patients (pts) with progressive multiple sclerosis (PMS) (Tourbah et al., 2016). Notably, MD1003 reversed MS-related disease disability in 13% of pts with PMS. MD1003 is currently being prescribed to pts with PMS in France under an expanded access programme.
Objective: To determine the benefits, in terms of effectiveness and safety, of MD1003 in pts with primary PMS (PPMS) or secondary PMS (SPMS) in a real-world clinical centre.
Methods: MD1003 300mg/day (100mg TID) was prescribed to pts with PPMS or SPMS being cared for at a single centre in France (CHU-Toulouse) starting in January 2016.The following measures of effectiveness and safety are being collected: Expanded Disability Status Scale (EDSS), timed 25-foot walk (T25W), 9-hole peg test (9-HPT), number of relapses, and gadolinium (Gd)-enhancing lesions on T1-weighted images.
Results: To date (May 2018), a total of 220 pts have received MD1003; results presented here correspond to the first 91 pts that have 1 year of follow-up. At baseline (month [M] 0), mean age was 59.5 years (SD=9.3), 61.5% were females, 70.3% had SPMS, mean EDSS was 5.9 (SD=1.3), mean TW25 was 50.7 seconds (SD=69.3), 9-HPT in the dominant hand was 35.1 (SD=20.7), and the mean number of previous relapses was 5.1 (SD=4.8). After 1 year of treatment with MD1003, 19 (23%; n=83 with data) pts experienced improvement in EDSS and 15 (23%; n=66 with data) pts experienced ≥20% improvement in T25W. Active disease, a clinically-defined relapse and/or a Gd-enhancing T1 lesion, was observed in 9 (11%; n=79 with data) pts.
Conclusions: MD1003 300mg/day was effective in the treatment of PMS in a real-world clinical setting; at 1 year of treatment, 23% of pts experienced improvement in MS-related disease activity (as measured by EDSS or TW25). MD1003 was also well-tolerated. This real-world study supports the growing body of evidence that MD 1003 is an effective and safe treatment for PMS.
Disclosure: J. Ciron: nothing to disclose., B. Pignolet: nothing to disclose., F. Bucciarelli: nothing to disclose., F. Lerebours: nothing to disclose., G. Dorcet: nothing to disclose., C. Rabadeux: nothing to disclose., N. Freitas: nothing to disclose., F. Bonneville: nothing to disclose., D. Biotti received speaker honoraria from Teva, Merck, Roche, Biogen, Novartis., D. Brassat received research grant from EU FP7, 305477-2012, EU Marie Curie action 2008 (Co I) ABIRISK (co I), French ministery of health -2008 (PHRC), French MS society 2009, 2012, 2017. Lectures, congress invitation, board participation with Bayer, Biogen, Medday, Merck, Novartis, Roche, Sanofi-Genzyme and Teva