Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.

ABSTRACT:

Importance

Wiskott-Aldrich syndrome is a rare primary immunodeficiency associated with severe microthrombocytopenia. Partially HLA antigen-matched allogeneic hematopoietic stem cell (HSC) transplantation is often curative but is associated with significant comorbidity.

Objective

To assess the outcomes and safety of autologous HSC gene therapy in Wiskott-Aldrich syndrome.

Design, setting, and participants

Gene-corrected autologous HSCs were infused in 7 consecutive patients with severe Wiskott-Aldrich syndrome lacking HLA antigen-matched related or unrelated HSC donors (age range, 0.8-15.5 years; mean, 7 years) following myeloablative conditioning. Patients were enrolled in France and England and treated between December 2010 and January 2014. Follow-up of patients in

SUBMITTER: Hacein-Bey Abina S 

PROVIDER: S-EPMC4942841 | biostudies-literature | 2015 Apr

REPOSITORIES: biostudies-literature