Successful allogeneic hemopoietic stem cell transplantation in a case of Wiskott-Aldrich syndrome and non-Hodgkin lymphoma

Pediatr Transplant. 2013 Sep;17(6):E146-8. doi: 10.1111/petr.12114. Epub 2013 Jun 17.

Abstract

WAS is a severe X-linked recessive disorder characterized by microthrombocytopenia, eczema, and immunodeficiency. A six-yr-old boy with WAS diagnosed as B-cell NHL (Stage III) localized in the liver who underwent successful HSCT from HLA-one antigen mismatch sibling donor has been presented here. His conditioning regimen included ATG, busulfan, and fludarabine. He received 2.3 × 10(6) /kg CD 34+ stem cells and 11 × 10(8) /kg nucleated cells at day 0. Neutrophil engraftment was achieved at day +14 and platelet engraftment at day +20. He has been in CR for more than two yr after transplantation. Thus, HSCT is an effective treatment for children with WAS even after development of lymphoma.

Keywords: Wiskott-Aldrich syndrome; allogenic stem cell transplantation; non-Hodgkin lymphoma; pediatrics.

Publication types

  • Case Reports

MeSH terms

  • Antilymphocyte Serum / administration & dosage
  • Blood Platelets / metabolism
  • Busulfan / administration & dosage
  • Child
  • HLA Antigens / chemistry
  • Hematopoietic Stem Cell Transplantation / methods*
  • Histocompatibility Testing
  • Humans
  • Liver Neoplasms / complications
  • Liver Neoplasms / therapy
  • Lymphoma, Non-Hodgkin / complications
  • Lymphoma, Non-Hodgkin / therapy*
  • Male
  • Neutrophils / metabolism
  • Remission Induction
  • Siblings
  • Transplantation Conditioning / methods
  • Treatment Outcome
  • Vidarabine / administration & dosage
  • Vidarabine / analogs & derivatives
  • Wiskott-Aldrich Syndrome / complications
  • Wiskott-Aldrich Syndrome / therapy*

Substances

  • Antilymphocyte Serum
  • HLA Antigens
  • Vidarabine
  • Busulfan
  • fludarabine