Successful Reduced Intensity Allogeneic Transplant With Full Donor Chimerism and Good Quality of Life in Adolescent Patient With Wiskott-Aldrich Syndrome

J Pediatr Hematol Oncol. 2017 Jul;39(5):e267-e269. doi: 10.1097/MPH.0000000000000851.

Abstract

Wiskott-Aldrich syndrome (WAS) is an X-linked disease characterized by microthrombocytopenia, eczema, immune deficiency, and autoimmune phenomena. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment. Myeloablative conditioning is the most common regimen used for HSCT in patients with WAS to avoid the risk of mixed donor chimerism and autoimmunity post-HSCT. There is limited data on the use of reduced intensity conditioning for HSCT in patients with WAS. Here, we report a case with severe phenotype of WAS transplanted successfully with reduced intensity conditioning, which is an acceptable conditioning regimen and can be considered in patients with WAS with significantly impaired organ functions.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Quality of Life
  • Transplantation Chimera
  • Transplantation Conditioning / methods*
  • Transplantation, Homologous
  • Wiskott-Aldrich Syndrome / therapy*