Pooled data from two prospective studies and an expanded-access program (27 participants total) evaluated etuvetidigene autotemcel, an autologous gene therapy using lentiviral-transduced hematopoietic stem cells, in patients with Wiskott-Aldrich syndrome. Investigators reported 96% overall survival at 1 and 5 years, with severe infection rates falling from 2.00 to 0.15 per person-year and moderate-to-severe bleeding events falling from 2.00 to 0.80 per person-year after treatment; no insertional oncogenesis was observed, and the most common grade 3 or higher adverse event was central venous catheter-related infection. The authors concluded the findings are consistent with sustained clinical benefit over a median follow-up of 5.7 years.
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