BACKGROUND: Although there is a body of literature on the use of risdiplam (EVRYSDI®) in adult patients from clinical trials, including SUNFISH (NCT02908685; 2–25 years) and JEWELFISH (NCT03032172; 1–60 years), available real-world evidence on the use of risdiplam is limited in adult patients.
OBJECTIVE: This is a report of a multicenter retrospective chart review that aims to provide information on patient and disease characteristics, outcomes (e.g. motor function or healthcare resource utilization) and treatment patterns of patients who were first written an order for risdiplam treatment aged ≥25 years in a real-world practice setting.
METHODS: The analysis will include data from patients with a confirmed spinal muscular atrophy (SMA) diagnosis, at least two clinic visits for SMA-related care with at least 12 months of follow-up after the first order for risdiplam treatment. Interim analysis will include data from up to four centers. Patient and clinical characteristics will be summarized. Descriptive results will be presented by subgroups of interest (e.g. prior SMA treatment, survival of motor neuron 2 copy number, disease duration, and ambulatory status), and reported as available. Outcomes will include survival and motor function (including the Hammersmith Functional Motor Scale – Expanded, Revised Upper Limb Module, and healthcare resource utilization [e.g. hospitalization, including length of stay, and emergency room or urgent care visits]). Continuous measures will be described using means, medians and standard deviations. Categorical measures will be summarized using counts and percentages.
RESULTS: Data on patient and clinical characteristics, treatment patterns, and the outcomes available will be presented.
CONCLUSIONS: To supplement existing clinical data for adults with SMA treated with risdiplam, real-world data are needed. This study will help to fill a need for descriptive information on the treatment, management and medical burden associated with SMA in patients aged ≥25 years treated with risdiplam.
