Case Report: Haploidentical Bone Marrow Transplantation in Two Brothers With Wiskott–Aldrich Syndrome Using Their Father as the Donor
Affiliation
Department of Pediatrics, University of ArizonaBanner University Medical Center, University of Arizona
Banner Children's at Desert, University of Arizona
Department of Immunobiology, University of Arizona
Department of Medicine, University of Arizona
Department of Pathology, University of Arizona
The University of Arizona Cancer Center
Issue Date
2021Keywords
cyclophosphamidehaploidentical hematopoietic stem cell transplantation
immunodeficiency
myeloablative allogeneic hematopoietic cell transplantation
Wiskott Aldrich syndrome (WAS)
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Frontiers Media S.A.Citation
Smith, J., Alfonso, J. H., Reddivalla, N., Angulo, P., & Katsanis, E. (2021). Case Report: Haploidentical Bone Marrow Transplantation in Two Brothers With Wiskott–Aldrich Syndrome Using Their Father as the Donor. Frontiers in Pediatrics.Journal
Frontiers in PediatricsRights
Copyright © 2021 Smith, Alfonso, Reddivalla, Angulo and Katsanis. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY).Collection Information
This item from the UA Faculty Publications collection is made available by the University of Arizona with support from the University of Arizona Libraries. If you have questions, please contact us at repository@u.library.arizona.edu.Abstract
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. Copyright © 2021 Smith, Alfonso, Reddivalla, Angulo and Katsanis.Note
Open access journalISSN
2296-2360Version
Final published versionae974a485f413a2113503eed53cd6c53
10.3389/fped.2021.647505
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Except where otherwise noted, this item's license is described as Copyright © 2021 Smith, Alfonso, Reddivalla, Angulo and Katsanis. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY).