Key result
Onasemnogene abeparvovec infusion improved health status in 7 out of 8 (87.5%) patients with Spinal Muscular Atrophy, with the greatest improvements seen when treated within 2 months of birth.
Why the study?
To verify whether therapy with onasemnogene abeparvovec led to clinical improvement measured by CHOP-INTEND scores and to identify any adverse reactions in treated patients with spinal muscular atrophy.
Does onasemnogene abeparvovec improve CHOP-INTEND scores in patients with Spinal Muscular Atrophy?
Observational (n=8)
No
Does onasemnogene abeparvovec improve CHOP-INTEND scores in patients with Spinal Muscular Atrophy?
Onasemnogene abeparvovec improved motor function in SMA patients, particularly when administered within 2 months of birth.
Real-world CHOP-INTEND gains with onasemnogene abeparvovec in SMA remain hypothesis-generating; prospective trials needed before practice change.
Objective: The term Spinal Muscular Atrophy (SMA) identifies a group of genetic disorders affecting spinal motor neurons. It is caused by the loss of the SMN1 gene, resulting in degeneration of spinal alpha motor neurons and muscle atrophy. This study is focused on innovative gene therapies with onasemnogene abeparvovec approved in Italy in March 2021 with full reimbursement by the National Health Service. The objective pursued is verify, by means of the CHOP-INTEND scores obtained, whether therapy with onasemnogene abeparvovec led to an improvement in the clinical picture of the treated subjects and any adverse reactions that occurred. Methods: this study was conducted by evaluating the scores in the different re-evaluations of individual patients treated in our hospital (Fondazione Policlinico Universitario Agostino Gemelli IRCCS – Rome) and comparing them with the results of the CL-303 study described in SPC (Summary of Product Characteristics). The data were extracted from the patients’ clinical records on the AIFA (Agenzia Italiana del Farmaco – Italian Medicines Agency) registries, also collecting information on any post-infusion ADRs. Everything was then represented graphically to have a clear comparison with the data from the study registered for drug approval. Results: from the data obtained, 7 out of 8 patients improved their health status post infusion with, in some cases, a significant increase in score. Conclusions: this result allows us to understand how crucial it is to start treatment as soon as possible after the diagnosis of the condition as the greatest improvements were seen in subjects who received treatment within 2 months of birth.
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Favia et al. (2023) conducted an observational in Spinal Muscular Atrophy (SMA) (n=8). Onasemnogene abeparvovec was evaluated on Improvement in clinical picture (CHOP-INTEND scores) and adverse reactions. Onasemnogene abeparvovec infusion improved health status in 7 out of 8 (87.5%) patients with Spinal Muscular Atrophy, with the greatest improvements seen when treated within 2 months of birth.
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