Outcomes after Allogeneic Transplant in Patients with Wiskott-Aldrich Syndrome

Alexander Ngwube, I. Celine Hanson, Jordan Orange, Nicholas L. Rider, Filiz Seeborg, William Shearer, Lenora Noroski, Sarah Nicholas, Lisa Forbes, Kathryn Leung, Ghadir Sasa, Swati Naik, Meenakshi Hegde, Bilal Omer, Nabil Ahmed, Carl Allen, Stephen Gottschalk, Meng Fen Wu, Hao Liu, Malcolm BrennerHelen Heslop, Robert Krance, Caridad Martinez

Research output: Contribution to journalArticle

6 Scopus citations

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 × 10 9 /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.

Original languageEnglish (US)
Pages (from-to)537-541
Number of pages5
JournalBiology of Blood and Marrow Transplantation
Volume24
Issue number3
DOIs
StatePublished - Mar 2018

Keywords

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

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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    Ngwube, A., Hanson, I. C., Orange, J., Rider, N. L., Seeborg, F., Shearer, W., Noroski, L., Nicholas, S., Forbes, L., Leung, K., Sasa, G., Naik, S., Hegde, M., Omer, B., Ahmed, N., Allen, C., Gottschalk, S., Wu, M. F., Liu, H., ... Martinez, C. (2018). Outcomes after Allogeneic Transplant in Patients with Wiskott-Aldrich Syndrome. Biology of Blood and Marrow Transplantation, 24(3), 537-541. https://doi.org/10.1016/j.bbmt.2017.11.019