Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia

Bone Marrow Transplant. 2017 Apr;52(4):570-573. doi: 10.1038/bmt.2016.301. Epub 2017 Jan 9.

Abstract

We developed a haploidentical transplantation protocol with post-transplant cyclophosphamide (CY) for in vivo T-cell depletion (TCD) using a novel adapted-dosing schedule (25 mg/kg on days +3 and +4) for Fanconi anemia (FA). With median follow-up of 3 years (range, 37 days to 6.2 years), all six patients engrafted. Two patients with multiple pre-transplant comorbidities died, one from sepsis and one from sepsis with associated chronic GVHD. Four patients without preexisting comorbidities and early transplant referrals are alive with 100% donor chimerism and excellent performance status. We conclude that adjusted-dosing post-transplant CY is effective in in vivo TCD to promote full donor engraftment in patients with FA.

MeSH terms

  • Child
  • Child, Preschool
  • Cyclophosphamide / administration & dosage*
  • Drug Administration Schedule
  • Fanconi Anemia / mortality
  • Fanconi Anemia / therapy*
  • Female
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Lymphocyte Depletion / methods*
  • Male
  • T-Lymphocytes
  • Transplantation, Haploidentical / methods*

Substances

  • Immunosuppressive Agents
  • Cyclophosphamide