Case Report: Haploidentical Bone Marrow Transplantation in Two Brothers With Wiskott–Aldrich Syndrome Using Their Father as the Donor

Jasmine Smith, Jessica Hass Alfonso, Naresh Reddivalla, Pablo Angulo, Emmanuel Katsanis

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish (US)
Article number647505
JournalFrontiers in Pediatrics
Volume9
DOIs
StatePublished - Oct 27 2021

Keywords

  • cyclophosphamide
  • haploidentical hematopoietic stem cell transplantation
  • immunodeficiency
  • myeloablative allogeneic hematopoietic cell transplantation
  • Wiskott Aldrich syndrome (WAS)

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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