Contributions
Abstract: P585
Type: Poster Sessions
Abstract Category: Therapy - Neuroprotection and Repair
Background: The efficacy and safety of MD1003 (high dose Pharmaceutical grade Biotin) for the treatment of progressive MS (PMS) was recently examined in the MS-SPI study (Tourbah et al., 2016). MD1003 led to a reversal of disability in 12.6% of patients (pts) with PMS (vs. 0% of pts receiving placebo) and was well tolerated. MD1003 is available in France through an expanded access programme.
Objective: To evaluate the effectiveness and safety of MD1003 for the treatment of PMS in real-world clinical practice.
Methods: This is a prospective, observational, single-centre (CHU de Reims, France) study of MD1003. Pts with primary or secondary PMS (PPMS; SPMS), any baseline Expanded Disability Status Score (EDSS), and without clinical or radiological evidence of inflammatory activity within the previous year (“not active”) were treated with MD1003 300 mg/day. Disease progression was assessed using EDSS, timed 25-foot walk (TW25), walking distance, and Clinical Global Impression (CGI). Safety outcomes, including the occurrence of relapses, were also recorded.
Results: As of May 2018, 71 pts have been treated with MD1003, of which 36 were followed up to 1 year. Of these 36 pts, 53% were female, 53% were diagnosed with SPMS, mean age was 54 years (SD=9.4), and mean disease duration since diagnosis was 17.3 years (SD=8.4). At month (M) 12, 25 (69.5%) pts improved in at least one of the 3 parameters measuring disease progression (EDSS, TW25 ≤20%, or walking distance ≥20%). Conversely, 3 (8.3%) pts worsened at M12. Prior to initiating MD1003 treatment, mean EDSS change from baseline showed worsening of the disease (-0.22); this worsening was reversed from M0 to M12 (0 to -0.22) under MD1003 treatment. In addition, between M0 and M12, mean TW25 and mean walking distance improved with a decrease in TW25 (-14.1%) and an increase in mean walking distance of 167.4 meters. CGI confirmed the effectiveness of MD1003 in PMS, with 25 (69.4%) pts demonstrating improvement. Adverse events were in line with results from previous studies. Additional data at M12 in a larger cohort of pts and complementary sub-group analysis will be presented.
Conclusions: MD1003 was effective in a real-world clinical setting in France, with > 2/3 of pts with PMS experiencing improvement in at least one of 3 commonly used disease activity measures (EDSS, TW25, and walking distance) at M12. MD1003 was also well-tolerated, with safety outcomes in line with previous studies.
Disclosure: Justine Faure, Joelle Jauffret, Christophe Carreau, Pauline Legrain, Cedric Castexhave nothing to disclose
Ayman Tourbah has received consulting and lecturing fees, travel grants and research support from Medday, Biogen, Sanofi-Genzyme, Novartis, Merck Serono, Teva Pharma, and Roche
Abstract: P585
Type: Poster Sessions
Abstract Category: Therapy - Neuroprotection and Repair
Background: The efficacy and safety of MD1003 (high dose Pharmaceutical grade Biotin) for the treatment of progressive MS (PMS) was recently examined in the MS-SPI study (Tourbah et al., 2016). MD1003 led to a reversal of disability in 12.6% of patients (pts) with PMS (vs. 0% of pts receiving placebo) and was well tolerated. MD1003 is available in France through an expanded access programme.
Objective: To evaluate the effectiveness and safety of MD1003 for the treatment of PMS in real-world clinical practice.
Methods: This is a prospective, observational, single-centre (CHU de Reims, France) study of MD1003. Pts with primary or secondary PMS (PPMS; SPMS), any baseline Expanded Disability Status Score (EDSS), and without clinical or radiological evidence of inflammatory activity within the previous year (“not active”) were treated with MD1003 300 mg/day. Disease progression was assessed using EDSS, timed 25-foot walk (TW25), walking distance, and Clinical Global Impression (CGI). Safety outcomes, including the occurrence of relapses, were also recorded.
Results: As of May 2018, 71 pts have been treated with MD1003, of which 36 were followed up to 1 year. Of these 36 pts, 53% were female, 53% were diagnosed with SPMS, mean age was 54 years (SD=9.4), and mean disease duration since diagnosis was 17.3 years (SD=8.4). At month (M) 12, 25 (69.5%) pts improved in at least one of the 3 parameters measuring disease progression (EDSS, TW25 ≤20%, or walking distance ≥20%). Conversely, 3 (8.3%) pts worsened at M12. Prior to initiating MD1003 treatment, mean EDSS change from baseline showed worsening of the disease (-0.22); this worsening was reversed from M0 to M12 (0 to -0.22) under MD1003 treatment. In addition, between M0 and M12, mean TW25 and mean walking distance improved with a decrease in TW25 (-14.1%) and an increase in mean walking distance of 167.4 meters. CGI confirmed the effectiveness of MD1003 in PMS, with 25 (69.4%) pts demonstrating improvement. Adverse events were in line with results from previous studies. Additional data at M12 in a larger cohort of pts and complementary sub-group analysis will be presented.
Conclusions: MD1003 was effective in a real-world clinical setting in France, with > 2/3 of pts with PMS experiencing improvement in at least one of 3 commonly used disease activity measures (EDSS, TW25, and walking distance) at M12. MD1003 was also well-tolerated, with safety outcomes in line with previous studies.
Disclosure: Justine Faure, Joelle Jauffret, Christophe Carreau, Pauline Legrain, Cedric Castexhave nothing to disclose
Ayman Tourbah has received consulting and lecturing fees, travel grants and research support from Medday, Biogen, Sanofi-Genzyme, Novartis, Merck Serono, Teva Pharma, and Roche