Successful Haploidentical Stem Cell Transplant With Posttransplant Cyclophosphamide in Wiskott-Aldrich Syndrome With Myeloablative Conditioning

• Published in: Journal of pediatric hematology/oncology Vol. 43; no. 2; p. e230
• Main Authors: Sharma, Anil, Rastogi, Neha, Kapoor, Rohit, Chatterjee, Goutomi, Yadav, Satya P
• Format: Journal Article
• Language: English
• Published: United States 01.03.2021
• Subjects: Child, Preschool; Combined Modality Therapy; Cyclophosphamide - therapeutic use; Hematopoietic Stem Cell Transplantation - methods; Humans; Male; Myeloablative Agonists - therapeutic use; Prognosis; Tissue Donors; Transplantation Conditioning - methods; Transplantation, Haploidentical - methods; Wiskott-Aldrich Syndrome - pathology; Wiskott-Aldrich Syndrome - therapy
• ISSN: 1536-3678
• DOI: 10.1097/MPH.0000000000001841

Hematopoietic stem cell transplant (HSCT) is the only curative treatment modality for Wiskott-Aldrich syndrome. Haploidentical HSCT with posttransplant cyclophosphamide (PTCy) is an upcoming option in children with nonmalignant conditions. However, only few cases have been reported for Wiskott-Aldrich syndrome HSCT with PTCy approach. Here we report a 4-year-old boy, treated successfully by haploidentical HSCT with myeloablative conditioning (busulfan, fludarabine, and thiotepa) and PTCy. Posttransplant chimerism was fully donor. Of 13 cases (current case and other 12 published cases) 10 are alive and disease free after haploidentical HSCT with PTCy. Haploidentical HSCT with PTCy using myeloablative conditioning is feasible and safe.