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. The formal proof that hematopoietic cell transplantation (HCT) could be used to cure WAS revealed a requirement for both immunosuppression and myelosuppression that still underlies the standard approach to curative therapy today. The current short- and long-term toxicities of HCT are the main stumbling block for the ability to cure every patient with WAS and X-linked thrombocytopenia, and much remains to be done.
Pai
Sung-Yun
SY
Division of Hematology-Oncology, Children's Hospital Boston, Karp Family Research Laboratories, 8th Floor, Room 8214, 1 Blackfan Circle, Boston, MA 02115, USA.
Notarangelo
Luigi D
LD
D000818:Animals; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D058846:Antibodies, Monoclonal, Murine-Derived; D019013:CD40 Antigens / Q000276:immunology; D017024:Chemotherapy, Adjuvant; D018380:Hematopoietic Stem Cell Transplantation; D006648:Histocompatibility; D006801:Humans; D007111:Immunity, Cellular / Q000187:drug effects; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D000069283:Rituximab; D055153:Stem Cell Niche; D014923:Wiskott-Aldrich Syndrome / Q000235:genetics / Q000276:immunology* / Q000628:therapy*
D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D019013:CD40 Antigens; D019653:Myeloablative Agonists; D000069283:Rituximab