Successful treatment of chronic granulomatous disease with fludarabine-based reduced-intensity conditioning and unrelated bone marrow transplantation

Int J Hematol. 2008 Jan;87(1):88-90. doi: 10.1007/s12185-007-0017-8. Epub 2007 Dec 7.

Abstract

Allogeneic hematopoietic stem-cell transplantation (HSCT) for chronic granulomatous disease (CGD) with a reduced-intensity conditioning regimen can be expected to lead to less therapy-related mortality and late-onset impairment, whereas it has also been reported to increase the risk of unsustained mixed donor chimerism and late rejection after transplantation. Herein, we report a 4-year-old boy with CGD who was successfully treated with unrelated bone marrow transplantation with a reduced-intensity conditioning regimen (RIC). Fludarabine-based RIC, 4 Gy of total body irradiation, 120 mg/kg of cyclophosphamide, and 125 mg/m(2) of fludarabine, was adopted for transplantation, followed with 8.9 x 10(8)/kg mononucleated donor cells infused without T-cell depletion. Although hematopoietic engraftment was rapidly obtained by day +17, he developed unstable donor chimerism. After tacrolimus withdrawal, the patient showed grade III acute graft-versus-host disease (GVHD), and subsequently reached full donor chimerism by day +61. Twelve months post-transplant, the patient has remained well with stable and durable engraftment, 100% donor chimerism, and normal superoxide production, without the requirement of donor lymphocyte infusions (DLI).

Publication types

  • Case Reports

MeSH terms

  • Bone Marrow Transplantation*
  • Child, Preschool
  • Cyclophosphamide / therapeutic use
  • Granulomatous Disease, Chronic / therapy*
  • Humans
  • Male
  • Myeloablative Agonists / therapeutic use
  • Transplantation Conditioning / methods*
  • Transplantation, Homologous
  • Vidarabine / analogs & derivatives
  • Vidarabine / therapeutic use
  • Whole-Body Irradiation

Substances

  • Myeloablative Agonists
  • Cyclophosphamide
  • Vidarabine
  • fludarabine