Posttransplant Cyclophosphamide for HLA-haploidentical Transplantation in Patients With Mucopolysaccharidosis

J Pediatr Hematol Oncol. 2018 Aug;40(6):e350-e354. doi: 10.1097/MPH.0000000000001157.

Abstract

We successfully used a haploidentical transplantation protocol with posttransplant cyclophosphamide (CY) (50 mg/kg/d on days +3 and +4) for in vivo T-cell depletion in patients with mucopolysaccharidosis using reduced-intensive conditioning regimens, followed by a busulfan-based conditioning regimen, which included busulfan (12 to 16 mg/kg) and fludarabine(150 to 200 mg/m)+rabbit antihuman thymocyte globulin (7.5 to 10 mg/kg) as a conditioning regimen. Cyclosporine or tacrolimus, methotrexate, mycophenolate mofetil, and methylprednisolone were administered to prevent graft-versus-host disease (GVHD). After follow-up for a median period of 1.5 years, all 8 patients without preexisting severe comorbidities and early transplant referrals are alive, with 100% donor chimerism and excellent performance status. Only 1 patient developed chronic GVHD(II). We conclude that posttransplant CY is effective in vivo for T-cell depletion to promote full donor engraftment in patients with mucopolysaccharidosis. In addition, with posttransplant CY, the procedure reduced the rate of GVHD and the cost of transplant and improved the patients' quality of life.

MeSH terms

  • Allografts
  • Antilymphocyte Serum / administration & dosage
  • Busulfan / administration & dosage
  • Child, Preschool
  • Cyclophosphamide / administration & dosage*
  • Disease-Free Survival
  • Female
  • Follow-Up Studies
  • Graft vs Host Disease / mortality
  • Graft vs Host Disease / prevention & control*
  • HLA Antigens*
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Infant
  • Male
  • Mucopolysaccharidoses / mortality
  • Mucopolysaccharidoses / pathology
  • Mucopolysaccharidoses / therapy*
  • Survival Rate
  • Transplantation Conditioning*
  • Vidarabine / administration & dosage
  • Vidarabine / analogs & derivatives

Substances

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