Case Report: Haploidentical Bone Marrow Transplantation in Two Brothers With Wiskott-Aldrich Syndrome Using Their Father as the Donor
Wiskott-Aldrich syndrome (WAS) is an X-linked genetic disorder with a variable phenotypic expression that includes thrombocytopenia, eczema, and immunodeficiency. Some patients may also exhibit autoimmune manifestations. Patients with WAS are at increased risk of developing malignancies such as lymp...
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Published in | Frontiers in pediatrics Vol. 9; p. 647505 |
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Main Authors | , , , , |
Format | Journal Article |
Language | English |
Published |
Switzerland
Frontiers Media S.A
27.10.2021
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Subjects | |
Online Access | Get full text |
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Summary: | Wiskott-Aldrich syndrome (WAS) is an X-linked genetic disorder with a variable phenotypic expression that includes thrombocytopenia, eczema, and immunodeficiency. Some patients may also exhibit autoimmune manifestations. Patients with WAS are at increased risk of developing malignancies such as lymphoma. Allogeneic hematopoietic cell transplantation remains the only curative treatment. Haploidentical bone marrow transplantation (haplo-BMT) with post-transplant cyclophosphamide (PT-CY) has more recently been applied in WAS. Here, we report two brothers who underwent successful T-cell replete haplo-BMT with PT-CY at ages 9 months and 4 years using their father as the donor. Our myeloablative regimen was well-tolerated with minimal organ toxicity and no acute or chronic graft vs. host disease (GvHD). Haplo-BMT may be considered as a safe and effective option for patients with WAS who do not have available human leukocyte antigen (HLA) matched donors. |
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Bibliography: | This article was submitted to Pediatric Immunology, a section of the journal Frontiers in Pediatrics Edited by: M. Teresa De La Morena, Seattle Children's Hospital, United States Reviewed by: Satya Yadav, Medanta the Medicity Hospital, India; Jignesh D. Dalal, Case Western Reserve University, United States; Ramya Uppuluri, Apollo Speciality Hospitals, India; Jan Stary, University Hospital in Motol, Czechia; Herbert Pichler, St. Anna Kinderspital, Austria; Aleksandra Petrovic, Johns Hopkins All Children's Hospital, United States |
ISSN: | 2296-2360 2296-2360 |
DOI: | 10.3389/fped.2021.647505 |