This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.
This is a non-interventional study and does not impose a therapy protocol, diagnostic/therapeutic procedure, or a visit schedule. Patients will be treated according to the Japan package insert. Data will be collected using a case registration form (CRF) completed by the investigator at each study site.
None
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SMA patients administered onasemnogene abeparvovec by intrathecal injection in Japan.
Cohort
Prospective
Not listed
Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs)
Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications
Safety specifications include hepatotoxicity, transient thrombocytopenia, thrombotic microangiopathy, cardiac adverse events, dorsal root ganglia toxicity/peripheral sensory neuropathy, tumorigenicity due to chromosomal integration, and long-term monitoring of gene therapy.
Incidence of Serious Adverse Events and ADRs
Incidence based on the person-year method will be calculated.
Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
Incidence based on the person-year method will be calculated.
Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score
The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.
Change From Baseline in Revised Upper Limb Module (RULM) Score
RULM is a tool used to assess upper limb function in SMA patients. It consists of 20 items in total. The first entry item classifies overall functional ability and is not scored. The remaining 19 tasks are scored with 1 task scored as can/cannot (1 is the highest score) and 18 tasks rated on a 3-point scale as follows: * 0 - unable to perform the task * 1 - able to perform the task with some modification * 2 - able to perform the task with no difficulty The total RULM score ranges from 0 to 37 with higher scores indicating better upper limb function.
Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score
CHOP-INTEND is a tool used to measure the motor abilities in infants with SMA. It consists of 16 items scored on a scale from 0 (no response/unable to perform the movement) to 4 (complete response/able to perform the task). CHOP-Intend total score ranges from 0 to 64 with higher scores indicating higher motor function.
Change From Baseline in Clinical Global Impression-Severity (CGI-S) Score
The CGI-S scale is a physician-rated measure used to assess the severity of a patient's illness. Severity of illness is assessed with consideration for the overall clinical experience of patients of the same age. The CGI-S scores range from: * 0 = Not evaluated * 1 = Normal, not at all ill * 2 = Borderline normal and ill * 3 = Mildly ill * 4 = Moderately ill * 5 = Markedly ill * 6 = Severely ill * 7 = Among the most extremely ill
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