Hematopoietic Cell Transplantation for Wiskott-Aldrich Syndrome: Advances in Biology and Future Directions for Treatment

The Wiskott-Aldrich syndrome (WAS) is an X-linked disorder characterized by a triad of diagnostic clinical elements: immunodeficiency, eczema, and hemorrhage caused by thrombocytopenia with small-sized platelets. Hematopoietic cell transplantation (HOT) can cure WAS, and is most often performed using myeloablative conditioning regimens. Our growing understanding of the biology of WASp has revealed cell-type specific functions that impact immune and hematopoietic outcome after HCT. Shortand long-term toxicities associated with HCT remain the main obstacle to safe and effective cure for every patient with WAS. Optimal management strategies for patients with milder forms of the disease (including X-linked thrombocytopenia) remain to be defined.