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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DMT Cohort A | Patients with SMA who were treated with at least 1 DMT and had continuous health insurance coverage in KRD+ closed claims at the time of first DMT initiation. | ||
| DMT Cohort B | Patients with SMA who were treated with at least 1 DMT and received the first DMT at age 2 or younger. | ||
| DMT Cohort B1 | A sub-group of DMT Cohort B who started the first DMT between 2016 and 2021. | ||
| DMT Cohort B2 | A sub-group of DMT Cohort B who started the first DMT between 2022 and 2024. | ||
| DMT Cohort C | Patients with SMA types 1-3 who were treated with multiple DMTs. | ||
| DMT Cohort D | A subset of DMT Cohort C who received the first DMT at age two or younger. | ||
| DMT Cohort D1 | A sub-group of DMT Cohort D who started the first DMT between 2016 and 2021. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | 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. | 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. | 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. | 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. |
| Measure | Description | 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 |
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Inclusion criteria
Exclusion criteria
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SMA patients with data in the KRD+ between 01 January 2016 and 31 October 2024 who received OA, nusinersen, or risdiplam.
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis | East Hanover | New Jersey | 07936 | United States |
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| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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| DMT Cohort D2 | A sub-group of DMT Cohort D who started the first DMT between 2022 and 2024. |
| Up to an average of approximately 2 years |
| Number of Medical Encounters per Patient per Year (PPPY) During Period I | Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits. | Up to approximately 1 year |
| Number of Medical Encounters PPPY During Period II | Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits. | Up to an average of approximately 2 years |
| Number of Medical Encounters PPPY During Period III | Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits. | Up to an average of approximately 2 years |
| 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 |
| 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 |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |