A retrospective review of radicava experience across us als centers

Objective: To examine the experience of US ALS Centers in the administration of Radicava Background: Radicava was approved by FDA based on a small (137 subjects) placebo controlled Phase 3 study in Japan using restrictive entry criteria that demonstrated a 33 percent reduction in the ALSFRSR decline over six months. There has been no study of the treatment in the US population Design/Methods: Participating centers retrospectively reviewed charts of Radicava treated ALS participants for demographic (gender, age of onset, onset site), symptom duration prior to diagnosis and treatment, duration of treatment, FVC and ALSFRSR scores pre and post treatment as available, riluzole useage, site of administration, and recorded complications. Data was combined in a single data base for analysis. Results: 29 centers participated and analysis has begun in 23 centers with 603 subjects (340 M, 216F). Average age of onset was 57.9Y (F58.9Y; M57.3Y). Onset was bulbar in 155 (25.7%) (F79, M76) with an average age of onset at 61.5Y (F62.3Y, M60.7Y). Limb onset was seen in 395 subjects (65.5%) (F137, M258) with an average age of onset 56.4Y (F57Y, M56.3Y). Respiratory onset occurred in 9 subjects (F2, M7) with an average age of onset at 60.3Y. The average time from diagnosis to treatment was 14.3 months. Adverse events were reported in 68 participants out of 410 with information, most commonly headaches (30), gait disturbance (11), fatigue (11), and line infections (27). Site of administration was home (246), infusion centers (118), and a combination (65). Analysis of the change in ALSFRSR and FVC pre and post-treatment is underway and full results will be presented. Conclusions: Preliminary examination of data from 29 US ALS centers suggests administration of Radicava is predominantly home administered and associated with an infected port rate of 6.5%. A complete analysis of the data will be presented.