Disease-modifying Therapies (DMT) Treatment Outcomes in Patients With Spinal Muscular Atrophy (SMA) - A Real-world Study Using the Komodo Claims Data
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 4,805
- 试验地点
- 1
- 主要终点
- Number of Patients by SMA-related Complication, Procedure, and Durable Medical Equipment (DME) Before Receiving the First DMT (Period I)
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
- •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.
排除标准
- •Patients with SMA type
- •Patients with claims of multiple DMTs in their birth year.
研究组 & 干预措施
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.
DMT Cohort D2
A sub-group of DMT Cohort D who started the first DMT between 2022 and 2024.
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.
结局指标
主要结局
Number of Patients by SMA-related Complication, Procedure, and Durable Medical Equipment (DME) Before Receiving the First DMT (Period I)
时间窗: 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)
时间窗: 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)
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: Up to approximately 1 year
Inpatient Visit Length of Stay PPPY During Period II
时间窗: Up to an average of approximately 2 years
Inpatient Visit Length of Stay PPPY During Period III
时间窗: Up to an average of approximately 2 years
次要结局
- 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)