Summary

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• This summary is based on the review of one systematic review/meta-analysis. ^[1]
• Motor and Cognitive Function: In late infantile (LI) and early juvenile (EJ) patients, atidarsagene autotemcel (arsa-cel) treatment resulted in normal development or slower progression of gross motor and cognitive functions compared to the rapid decline seen in natural history patients. Allogeneic hematopoietic stem cell transplantation (alloHSCT) showed no benefits for motor and cognitive function in LI patients and mixed results in EJ patients, with some stabilization in pre-symptomatic or minimally-symptomatic individuals.
• Survival: Arsa-cel provided a survival benefit for LI patients compared to both natural history patients and those treated with alloHSCT. In contrast, alloHSCT did not improve survival in either LI or EJ patients, with survival outcomes similar to the natural history group.
• Comparison: Arsa-cel outperformed alloHSCT in terms of gross motor function, cognitive function, and survival in LI patients. For EJ patients, arsa-cel demonstrated better motor and cognitive outcomes than alloHSCT, which had limited efficacy in this population. No data was available for late juvenile (LJ) patients treated with arsa-cel.
• Atidarsagene Autotemcel (arsa-cel): No specific safety outcomes were detailed in the provided study for arsa-cel, implying no major safety concerns were highlighted.
• Allogeneic Hematopoietic Stem Cell Transplantation (alloHSCT): Associated with significant safety risks, including treatment-related mortality, graft-versus-host disease, and the need for re-transplantation in both late infantile (LI) and EJ patients. These complications were not mentioned for arsa-cel.
• There is no population types or subgroups information available in the reviewed studies.