Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler)

Bone Marrow Transplant. 2005 Feb;35(3):265-9. doi: 10.1038/sj.bmt.1704786.

Abstract

Hurler syndrome (MPS1H) is a progressive inborn error of mucopolysaccharide metabolism leading to premature death. Allogeneic hematopoietic cell transplantation (HCT) can achieve stabilization and improve long-term survival. However, large studies have shown that preparative regimen-related toxicity (RRT) and graft failure rates have been relatively high. We transplanted five Hurler children with a fludarabine-based conditioning regimen, consisting of fludarabine/busulphan/ATG for matched family donor (MFD), with the addition of melphalan for mismatched family donor and matched unrelated donor (MUD) transplantations. Median age at HCT was 27 months (range 10-36). The source of stem cells was bone marrow in one MFD and CD34-selected PBSC in four patients. Median CD34+ cell dose was 25 x 10(6)/kg (range 11.5-54). No RRT > grade II was observed. All patients are surviving at a median of 32 months (range 14-41) and show sustained donor engraftment with 3/5 having full donor chimerism, and 2/5 mixed chimerism (> 85%). We conclude that this regimen is feasible and has low toxicity in Hurler children. In combination with high doses of CD34+ selected cells (> 10 x 10(6)/kg) and donor lymphocyte infusions, stable engraftment could be achieved in unrelated and mismatched related transplantations.

Publication types

  • Clinical Trial

MeSH terms

  • Antigens, CD34*
  • Antilymphocyte Serum / administration & dosage
  • Busulfan / administration & dosage
  • Child, Preschool
  • Graft Survival
  • Hematopoiesis
  • Hematopoietic Stem Cell Transplantation / methods*
  • Histocompatibility
  • Histocompatibility Testing*
  • Humans
  • Infant
  • Lymphocyte Transfusion
  • Mucopolysaccharidosis I / therapy*
  • Transplantation Chimera
  • Transplantation Conditioning / methods*
  • Transplantation, Homologous
  • Treatment Outcome
  • Vidarabine / administration & dosage*
  • Vidarabine / analogs & derivatives*

Substances

  • Antigens, CD34
  • Antilymphocyte Serum
  • Vidarabine
  • Busulfan
  • fludarabine