Clinical Features, Cancer Biology, Transplant Approach and Other Integrated Management Strategies for Wiskott–Aldrich Syndrome

Abstract

Smitha Hosahalli Vasanna, Maria A Pereda, Jignesh Dalal Department of Pediatrics, Division of Pediatric Hematology Oncology, Rainbow Babies and Children’s Hospital, University Hospitals, Cleveland, OH, USACorrespondence: Jignesh DalalDepartment of Pediatrics, Division of Pediatric Hematology Oncology, Rainbow Babies and Children’s Hospital, University Hospitals, 11100 Euclid Ave, Cleveland, OH, 44106, USATel [phone]Fax [phone]Email [email protected]: Wiskott–Aldrich syndrome (WAS) is a rare X-linked recessive inborn error of immunity (IEI) first described in 1937. Classic WAS is characterized by the triad of thrombocytopenia with small platelets, recurrent infections due to combined immunodeficiency, and eczema. Hematopoietic stem cell transplantation (HSCT) was the only curative option available for five decades, with excellent outcomes reported for matched sibling donors (MSD) and matched unrelated donors (MUD). More recently, alternative donor transplants such as umbilical cord blood (UCB) and haploidentical transplant have emerged as viable options due to improvements in better graft selection, cell dosing, and effective allograft manipulation measures. Gene therapy is another potential curative option with promising results, yet currently is offered only as part of a clinical trial.Keywords: Wiskott–Aldrich syndrome, X-linked thrombocytopenia, supportive care, hematopoietic stem cell transplantation, gene therapy

Keywords

• wiskott-aldrich syndrome
• x-linked thrombocytopenia
• supportive care
• hematopoietic stem cell transplantation
• gene therapy