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Biol Blood Marrow Transplant. 2018 Mar;24(3):537-541. doi: 10.1016/j.bbmt.2017.11.019. Epub 2017 Nov 28.

Outcomes after Allogeneic Transplant in Patients with Wiskott-Aldrich Syndrome.

Author information

1
Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas.
2
Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas. Electronic address: camartin@txch.org.

Abstract

Wiskott-Aldrich syndrome (WAS) is a rare X-linked disorder characterized by a triad of immunodeficiency, eczema, and thrombocytopenia. Currently, hematopoietic stem cell transplantation (HSCT) is the most reliable curative treatment with excellent results for patients with HLA-matched family or unrelated donors. However, even after fully myeloablative preparative regimens, mixed donor chimerism is a potential concern. We performed a retrospective chart review of 12 children who underwent allogeneic HSCT for WAS to report our experience. The median age at transplant was 10.5 months (range, 3 to 39). The median nucleated cell dose from the marrow was 4.55 × 109/kg (range, .3 to 7.9). The median times to neutrophil and platelet engraftment were 19 days (range, 13 to 27) and 18.5 days (range, 12 to 31), respectively. The rate of overall survival was 92% with median follow-up of 67 months (range, 3 to 146). Two patients developed grade IV acute graft-versus-host disease, and 1 died on day +99. Five of 12 patient's (42%) had mixed donor chimerism (range, 12% to 85%) at day +180. None of the pretransplant patient parameters was predictive of mixed chimerism. Nonetheless, of these 5 patients, 2 had normalization of the platelet count despite the mixed chimerism, 2 had full donor chimerism after receiving a second transplant with the same donor, and 1 remains transfusion dependent awaiting a second transplant. Hence, even with a significant rate of mixed chimerism, HSCT provides substantial benefit to WAS patients, with excellent overall survival.

KEYWORDS:

Hematopoietic cell transplant; Mixed chimerism; Primary immunodeficiency; Wiskott-Aldrich syndrome

PMID:
29196075
DOI:
10.1016/j.bbmt.2017.11.019

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