Successful Allogeneic Hematopoietic Stem Cell Transplantation in XIAP Deficiency Using Reduced-Intensity Conditioning

Pediatr Blood Cancer. 2016 Feb;63(2):355-7. doi: 10.1002/pbc.25756. Epub 2015 Sep 23.

Abstract

Hematopoietic stem cell transplantation (HSCT) is currently the only available curative therapy for X-linked inhibitor of apoptosis (XIAP) deficiency. Myeloablative conditioning regimens are associated with high mortality rates. Reduced-intensity conditioning (RIC) is recommended in order to decrease treatment-related toxicities, but RIC regimens increase the risk for mixed donor-recipient chimerism that may progress to graft loss. We report our experience with a patient with XIAP deficiency who was successfully treated with allogeneic HSCT using a RIC protocol. Post-transplant chimerism was vigilantly monitored and maintained with donor lymphocyte infusions and a stem cell boost to a level that prevented hemophagocytic lymphohistiocytosis recurrence.

Keywords: XIAP; donor-recipient chimerism; hematopoietic stem cell transplantation; reduced intensity conditioning.

Publication types

  • Case Reports

MeSH terms

  • Alemtuzumab
  • Antibodies, Monoclonal, Humanized / administration & dosage
  • Child, Preschool
  • Genetic Diseases, X-Linked / surgery*
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Lymphoproliferative Disorders / surgery*
  • Male
  • Melphalan / administration & dosage
  • Myeloablative Agonists / therapeutic use*
  • Transplantation Conditioning / methods*
  • Transplantation, Homologous / methods
  • Vidarabine / administration & dosage
  • Vidarabine / analogs & derivatives

Substances

  • Antibodies, Monoclonal, Humanized
  • Myeloablative Agonists
  • Alemtuzumab
  • Vidarabine
  • fludarabine
  • Melphalan

Supplementary concepts

  • Lymphoproliferative Syndrome, X-Linked, 2