Baseline characteristics of patients from ongoing post‐marketing surveillance, evaluating the real‐world efficacy and safety of edaravone, a novel, free radical scavenger, for amyotrophic lateral sclerosis patients in Japan (SUNRISE Japan)

Abstract Background The ongoing SUNRISE Japan post‐marketing surveillance is collecting real‐world data about the long‐term efficacy and safety of edaravone in amyotrophic lateral sclerosis (ALS) for up to 5 years. Aim To provide detailed methodologic information about SUNRISE Japan and report baseline data. Methods Survival time, time to invasive tracheal intubation, other clinical events, ALS Functional Rating Scale‐Revised [ALSFRS‐R] scores, and safety will be assessed. Results In total, 797 patients were included in the safety and efficacy analysis sets; 55.3% were male, two‐thirds of patients were aged ≥65 years, and 86.1% had a body mass index of <25 kg/m 2 . Disease duration was <1 year (37.3% of patients), or 1‐2 years (32.1%). Disease severity (Japan ALS severity classification) included grade 2 (42.0% of patients), grade 3 (28.5%), or grade 1 (15.7%). Most ALS cases were sporadic (96.4% of patients). The most common site of symptom onset was the upper limbs (43.5% of patients). ALS diagnosis (El Escorial revised Airlie House criteria) was definite (34.5% of patients), probable (36.1%), probable supported by laboratory data (19.4%), possible (6.4%), or suspected (3.5%). The mean ALSFRS‐R score was 38.5, and mean percent forced vital capacity, 83.6%. Over 60% of patients had been treated with riluzole, and over 60% were receiving other concomitant drugs. Complications were noted in two‐thirds of patients. Conclusion Data from the SUNRISE Japan study are expected to extend ALS knowledge in Japan and worldwide, and the study methodology may provide a model for other long‐term surveillance studies in neurodegenerative diseases.