Prospective phase II trial to evaluate the complications and kinetics of chimerism induction following allogeneic hematopoietic stem cell transplantation with fludarabine and busulfan

Am J Hematol. 2007 Oct;82(10):873-80. doi: 10.1002/ajh.20977.

Abstract

This prospective trial assessed the safety and efficacy of allogeneic hematopoietic stem cell transplantation from a HLA-matched donor with a reduced-intensity regimen (RIST) consisting of iv fludarabine 30 mg/m(2) for 6 days and oral busulfan 4 mg/kg/day for 2 days in patients older than 50 years with hematological malignancies. Cyclosporine alone or cyclosporine with short-term methotrexate was randomized for graft-versus-host disease prophylaxis. After 30 patients had been enrolled, an interim analysis was performed, and this report focuses on a precise evaluation of the toxicity profile and chimerism kinetics. Sustained engraftment in all patients, no severe regimen-related toxicity (RRT) within 20 days, and no transplant-related mortality through Day 100 were observed. T-cell (CD3+) full-donor (over 90%) chimerism was observed in 22 of the 30 patients, while the remaining eight had mixed-donor chimerism over 77% on Day 90. Thereafter, five subsequently converted to full-donor chimerism without donor lymphocyte infusion by day 120 (n = 4) or Day 180 (n = 1). Two showed persistent mixed chimerism without relapse through Day 180. Grade III-IV acute graft-versus-host disease and extensive chronic graft-versus-host disease occurred in 10% and 73%, respectively. With a median follow-up of 1.5 years, overall survival and disease-free survival at 1 year was 83% and 62%, respectively. Seven patients hematologically relapsed overall, and five of them had myelodysplastic syndrome with poor prognostic factors. In older patients, RIST with fludarabine and busulfan was associated with acceptable toxicities and a satisfactory antileukemia effect, regardless of the early chimerism status.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Busulfan / administration & dosage
  • Busulfan / pharmacokinetics
  • Busulfan / therapeutic use*
  • Cyclosporine / administration & dosage
  • Cyclosporine / therapeutic use
  • Drug Therapy, Combination
  • Female
  • Graft Survival*
  • Graft vs Host Disease / mortality
  • Graft vs Host Disease / prevention & control
  • Hematologic Neoplasms / blood
  • Hematologic Neoplasms / mortality
  • Hematologic Neoplasms / surgery
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cell Transplantation* / mortality
  • Hematopoietic Stem Cell Transplantation* / statistics & numerical data
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Immunosuppressive Agents / therapeutic use
  • Lymphocyte Count
  • Lymphocyte Transfusion
  • Male
  • Methotrexate / administration & dosage
  • Methotrexate / therapeutic use
  • Middle Aged
  • Myelodysplastic Syndromes / blood
  • Myelodysplastic Syndromes / mortality
  • Myelodysplastic Syndromes / surgery
  • Postoperative Complications / mortality
  • Prospective Studies
  • Transplantation Conditioning* / adverse effects
  • Transplantation, Homologous / mortality
  • Transplantation, Homologous / statistics & numerical data
  • Vidarabine / administration & dosage
  • Vidarabine / analogs & derivatives*
  • Vidarabine / pharmacokinetics
  • Vidarabine / therapeutic use

Substances

  • Immunosuppressive Agents
  • Cyclosporine
  • Vidarabine
  • Busulfan
  • fludarabine
  • Methotrexate