Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources.

ABSTRACT:

Introduction

Spinal muscular atrophy (SMA) is a rare neuromuscular disease characterized by progressive muscular atrophy and weakness. Nusinersen was the first treatment approved for SMA. Per the US label, the nusinersen administration schedule consists of three loading doses at 14-day intervals, a fourth loading dose 30 days later, and maintenance doses every 4 months thereafter. Using two large US databases, we evaluated real-world adherence to nusinersen with its unique dosing schedule among generalizable populations of patients with SMA.

Methods

Patients with SMA treated with nusinersen, likely to have complete information on date of treatment initiation, were identified in the Optum^® de-identified electronic health records (EHR) database (7/2017-9/2019), and

SUBMITTER: Youn B 

PROVIDER: S-EPMC9841927 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature