Contributions
Abstract: P750
Type: Poster
Abstract Category: Therapy - disease modifying - 29 Risk management for disease modifying treatments
Background: Progressive multiple sclerosis (PMS) can be considered an orphan disease, because of lack of effective therapy currently available. Recently, oral high-dose biotin has been investigated as PMS treatment in a randomized clinical trial with promising results and without serious adverse events. Then, even though without indication by drug regulatory agencies, biotin has been prescribed by many clinicians in MS centres.
Aim: To report an unexpected increase of inflammatory activity in PMS patients treated with oral high-dose biotin.
Methods: We included all consecutive PMS patients who started biotin (300mg/die) in our centre, collecting clinical (relapses, EDSS score), brain and spinal MRI, tolerability and safety data.
Results: We included a total of 41 PMS patients (F 53.7%, mean age 54.3±9.82 years, mean EDSS 5.3±1.62, mean disease duration 15.8±9.28 years), with a primary progressive (PP) phenotype in 39.0% and secondary in 61.0% of cases. Mean treatment duration was 13.7±5.85 months. Annualized relapse rate increased from 0.10 in the previous year to 0.27 on treatment. Nine patients (22%), including 2 PP patients with no history of MS attacks, showed 12 relapses, 9 of them requiring steroid administration and 4 leaving residual disability. Seven patients (17%) showed MRI activity (new and/or enlarged T2 and/or Gd+ lesions), 3 of them with relapses. In 28 patients with treatment duration ≥ 12 months, EDSS score improved in 1, remained stable in 17 (60.7%), and worsened in 10 (35.7%) patients.
Conclusions: In our cohort of PMS patients treated with high-dose biotin we recorded an unexpected high rate, both clinical and radiological, of inflammatory activity. Clinicians should be very cautious when prescribing this drug until its efficacy and safety are definitely proven.
Disclosure:
F. Granella has received research grants for his Institution from Biogen; has served on scientific advisory boards for Biogen, Novartis, Sanofi Genzyme and Merck Serono; has received funding for travel from Biogen, Merck Serono and Sanofi Genzyme
E.Curti has served on scientific advisory boards for Merck Serono; has received funding for travel from Biogen, Merck Serono, Novartis and Sanofi Genzyme
E. Tsantes: nothing to disclose
E. Siena: nothing to disclose
Abstract: P750
Type: Poster
Abstract Category: Therapy - disease modifying - 29 Risk management for disease modifying treatments
Background: Progressive multiple sclerosis (PMS) can be considered an orphan disease, because of lack of effective therapy currently available. Recently, oral high-dose biotin has been investigated as PMS treatment in a randomized clinical trial with promising results and without serious adverse events. Then, even though without indication by drug regulatory agencies, biotin has been prescribed by many clinicians in MS centres.
Aim: To report an unexpected increase of inflammatory activity in PMS patients treated with oral high-dose biotin.
Methods: We included all consecutive PMS patients who started biotin (300mg/die) in our centre, collecting clinical (relapses, EDSS score), brain and spinal MRI, tolerability and safety data.
Results: We included a total of 41 PMS patients (F 53.7%, mean age 54.3±9.82 years, mean EDSS 5.3±1.62, mean disease duration 15.8±9.28 years), with a primary progressive (PP) phenotype in 39.0% and secondary in 61.0% of cases. Mean treatment duration was 13.7±5.85 months. Annualized relapse rate increased from 0.10 in the previous year to 0.27 on treatment. Nine patients (22%), including 2 PP patients with no history of MS attacks, showed 12 relapses, 9 of them requiring steroid administration and 4 leaving residual disability. Seven patients (17%) showed MRI activity (new and/or enlarged T2 and/or Gd+ lesions), 3 of them with relapses. In 28 patients with treatment duration ≥ 12 months, EDSS score improved in 1, remained stable in 17 (60.7%), and worsened in 10 (35.7%) patients.
Conclusions: In our cohort of PMS patients treated with high-dose biotin we recorded an unexpected high rate, both clinical and radiological, of inflammatory activity. Clinicians should be very cautious when prescribing this drug until its efficacy and safety are definitely proven.
Disclosure:
F. Granella has received research grants for his Institution from Biogen; has served on scientific advisory boards for Biogen, Novartis, Sanofi Genzyme and Merck Serono; has received funding for travel from Biogen, Merck Serono and Sanofi Genzyme
E.Curti has served on scientific advisory boards for Merck Serono; has received funding for travel from Biogen, Merck Serono, Novartis and Sanofi Genzyme
E. Tsantes: nothing to disclose
E. Siena: nothing to disclose