Muscular Atrophy, Spinal
Conditions
Keywords
Spinal muscular atrophy, Multiple disease modifying therapies, Real-world evidence, Treatment patterns
Brief summary
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.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients by SMA-related Complication, Procedure, and Durable Medical Equipment (DME) Before Receiving the First DMT (Period I) | Up to approximately 1 year | DME is medical equipment that assists patients with their medical condition, can be used at home, and lasts a long time. |
| Number of Patients by SMA-related Complication, Procedure, and DME After Receiving the First DMT and Before the Second DMT (Period II) | Up to an average of approximately 2 years | DME is medical equipment that assists patients with their medical condition, can be used at home, and lasts a long time. |
| Number of Patients by SMA-related Complication, Procedure, and DME After Receiving the Second DMT and Before the Third DMT (Period III) | Up to an average of approximately 2 years | DME is medical equipment that assists patients with their medical condition, can be used at home, and lasts a long time. |
| Number of Patients by All-cause Medical Encounters During Period I | Up to approximately 1 year | Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits. |
| Number of Patients by All-cause Medical Encounters During Period II | Up to an average of approximately 2 years | Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits. |
| Number of Patients by All-cause Medical Encounters During Period III | Up to an average of approximately 2 years | Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits. |
| Number of Medical Encounters per Patient per Year (PPPY) During Period I | Up to approximately 1 year | Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits. |
| Number of Medical Encounters PPPY During Period II | Up to an average of approximately 2 years | Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits. |
| Number of Medical Encounters PPPY During Period III | Up to an average of approximately 2 years | Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits. |
| Inpatient Visit Length of Stay PPPY During Period I | Up to approximately 1 year | — |
| Inpatient Visit Length of Stay PPPY During Period II | Up to an average of approximately 2 years | — |
| Inpatient Visit Length of Stay PPPY During Period III | Up to an average of approximately 2 years | — |
Secondary
| Measure | Time frame |
|---|---|
| Number of Patients who Received Multiple DMTs by Order in Which DMTs were Received | Up to approximately 5 years and 5 months |
| Time Between the First and Second DMT Initiation Among Patients Treated With Multiple DMTs | Up to an average of approximately 2 years |
| Time Between the Second and Third DMT Initiation Among Patients Treated With Multiple DMTs | Up to an average of approximately 2 years |
| Time on the First DMT During Period II Among Patients Treated With Multiple DMTs | Up to an average of approximately 2 years |
| Time on the First DMT During Period III Among Patients Treated With Multiple DMTs | Up to an average of approximately 2 years |
| Time on the Second DMT During Period III Among Patients Treated With Multiple DMTs | Up to an average of approximately 2 years |
| Number of Patients who Started a Second DMT | Up to approximately 5 years and 5 months |
| Duration of Continuous Enrollment for Patients Treated With at Least 1 DMT | Up to approximately 5 years and 5 months |
Countries
United States
Contacts
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed