HLA-Haploidentical Peripheral Blood Stem Cell Transplantation with Post-Transplant Cyclophosphamide after Busulfan-Containing Reduced-Intensity Conditioning

Biol Blood Marrow Transplant. 2015 Sep;21(9):1646-52. doi: 10.1016/j.bbmt.2015.06.008. Epub 2015 Jun 18.

Abstract

Allogeneic hematopoietic stem cell transplantation (allo-SCT) using post-transplant cyclophosphamide (PTCy) is increasingly performed. We conducted a multicenter phase II study to evaluate the safety and efficacy of PTCy-based HLA-haploidentical peripheral blood stem cell transplantation (PTCy-haploPBSCT) after busulfan-containing reduced-intensity conditioning. Thirty-one patients were enrolled; 61% patients were not in remission and 42% patients had a history of prior allo-SCT. Neutrophil engraftment was achieved in 87% patients with a median of 19 days. The cumulative incidence of grades II to IV and III to IV acute graft-versus-host disease (GVHD) and chronic GVHD at 1 year were 23%, 3%, and 15%, respectively. No patients developed severe chronic GVHD. Day 100 nonrelapse mortality (NRM) rate was 19.4%. Overall survival, relapse, and disease-free survival rates were 45%, 45%, and 34%, respectively, at 1 year. Subgroup analysis showed that patients who had a history of prior allo-SCT had lower engraftment, higher NRM, and lower overall survival than those not receiving a prior allo-SCT. Our results suggest that PTCy-haploPBSCT after busulfan-containing reduced-intensity conditioning achieved low incidences of acute and chronic GVHD and NRM and stable donor engraftment and low NRM, particularly in patients without a history of prior allo-SCT.

Keywords: Busulfan; HLA-haploidentical transplantation; Post-transplant cyclophosphamide; Reduced-intensity conditioning.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Busulfan / administration & dosage*
  • Cyclophosphamide / administration & dosage*
  • Disease-Free Survival
  • Female
  • HLA Antigens*
  • Hematologic Neoplasms* / mortality
  • Hematologic Neoplasms* / therapy
  • Humans
  • Male
  • Middle Aged
  • Peripheral Blood Stem Cell Transplantation*
  • Prospective Studies
  • Survival Rate
  • Transplantation Conditioning*

Substances

  • HLA Antigens
  • Cyclophosphamide
  • Busulfan