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

Novartis Pharmaceuticals4,805 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

4,805

Conditions

Muscular Atrophy, Spinal

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (1)

Novartis

East Hanover, New Jersey, United States

Outcomes

Primary Outcomes

Number of Patients by SMA-related Complication, Procedure, and Durable Medical Equipment (DME) Before Receiving the First DMT (Period I)

DME is medical equipment that assists patients with their medical condition, can be used at home, and lasts a long time.

Time frame: Up to approximately 1 year

Number of Patients by SMA-related Complication, Procedure, and DME After Receiving the First DMT and Before the Second DMT (Period II)

DME is medical equipment that assists patients with their medical condition, can be used at home, and lasts a long time.

Time frame: Up to an average of approximately 2 years

Number of Patients by SMA-related Complication, Procedure, and DME After Receiving the Second DMT and Before the Third DMT (Period III)

DME is medical equipment that assists patients with their medical condition, can be used at home, and lasts a long time.

Time frame: Up to an average of approximately 2 years

Number of Patients by All-cause Medical Encounters During Period I

Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits.

Time frame: Up to approximately 1 year

Number of Patients by All-cause Medical Encounters During Period II

Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits.

Time frame: Up to an average of approximately 2 years

Number of Patients by All-cause Medical Encounters During Period III

Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits.

Time frame: Up to an average of approximately 2 years

Data from ClinicalTrials.gov

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Secondary Outcomes

Number of Patients who Received Multiple DMTs by Order in Which DMTs were Received

Time frame: Up to approximately 5 years and 5 months

Time Between the First and Second DMT Initiation Among Patients Treated With Multiple DMTs

Time frame: Up to an average of approximately 2 years

Time Between the Second and Third DMT Initiation Among Patients Treated With Multiple DMTs

Time frame: Up to an average of approximately 2 years

Time on the First DMT During Period II Among Patients Treated With Multiple DMTs

Time frame: Up to an average of approximately 2 years

Time on the First DMT During Period III Among Patients Treated With Multiple DMTs

Time frame: Up to an average of approximately 2 years

Time on the Second DMT During Period III Among Patients Treated With Multiple DMTs

Time frame: Up to an average of approximately 2 years

Number of Patients who Started a Second DMT

Time frame: Up to approximately 5 years and 5 months

Duration of Continuous Enrollment for Patients Treated With at Least 1 DMT

Time frame: Up to approximately 5 years and 5 months