A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy

Disease-modifying Therapies (DMT) Treatment Outcomes in Patients With Spinal Muscular Atrophy (SMA) - A Real-world Study Using the Komodo Claims Data

The aim of this study was to evaluate treatment patterns, SMA-related complications and services, and all-cause medical encounters of patients with SMA receiving multiple DMTs in the real world. The DMTs included onasemnogene abeparvovec-xioi (OA), nusinersen, and risdiplam. This study was conducted using data from the Komodo Health Research Database (KRD+) between 01 January 2016 and 31 October 2024.

Inclusion criteria

• Patients with ≥1 SMA diagnosis at any time (International Classification of Disease, Tenth Revision, Clinical Modification [ICD-10-CM] codes: G12.0, G12.1, G12.9).
• Patients with ≥1 record of OA, nusinersen, or risdiplam based on relevant Healthcare Common Procedure Coding System (HCPCS) codes and National Drug Code (NDC).
• Patients who received at least 2 of the 3 DMTs, with the first DMT initiated on or after 26 May 2019, when at least 2 DMTs were available. The initiation of the first DMT was defined as the index date and the first DMT was defined as the index DMT.
• Patients with SMA types 1, 2, or 3.
• Patients with ≥1 quarter of clinical activities within 1 year prior to the index quarter (for patients with SMA type 2 and 3 only).
• Patients with ≥1 quarter of clinical activities any time after the index quarter, unless death occurred.

Exclusion criteria

• Patients with SMA type 4.
• Patients with claims of multiple DMTs in their birth year.