Successful unrelated cord blood transplantation for chronic granulomatous disease: a case report and review of the literature

Pediatr Transplant. 2009 May;13(3):384-9. doi: 10.1111/j.1399-3046.2008.00996.x. Epub 2008 Jul 9.

Abstract

CGD is a rare inherited immunodeficiency disorder that is caused by disability of oxidative killing. We presented a two-yr-old boy with CGD who was suffering from multiple systemic abscesses. He received the first BMT from his HLA-haploidentical mother after conditioning with Flu, melphalan, and ATG. Although the maximum of 42% donor chimerism was achieved, it disappeared 73 days after the BMT. Then, we performed 5/6-matched unrelated cord blood re-transplantation after conditioning with Flu, Bu, and TBI (2 Gy). Engraftment and complete donor chimerism were achieved on days 18 and 19, respectively. The patient is now free from infection and maintains complete donor chimerism without GVHD 36 months after the cord blood re-transplantation. We postulate that the unrelated CBT has a potential to be an alternative strategy and might be beneficial for patients with CGD who do not have an HLA-identical donor.

Publication types

  • Case Reports
  • Review

MeSH terms

  • Anti-Bacterial Agents / therapeutic use
  • Child, Preschool
  • Cord Blood Stem Cell Transplantation*
  • Granulomatous Disease, Chronic / complications
  • Granulomatous Disease, Chronic / therapy*
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Infections / etiology
  • Infections / therapy
  • Male
  • Recurrence
  • Reoperation
  • Treatment Failure
  • Treatment Outcome

Substances

  • Anti-Bacterial Agents