Results of a 2-arm, phase 2 clinical trial using post-transplantation cyclophosphamide for the prevention of graft-versus-host disease in haploidentical donor and mismatched unrelated donor hematopoietic stem cell transplantation

Cancer. 2016 Nov 15;122(21):3316-3326. doi: 10.1002/cncr.30180. Epub 2016 Jul 12.


Background: High-dose, post-transplantation cyclophosphamide (PTCy) to prevent graft-versus-host disease (GVHD) has improved outcomes in haploidentical (HAPLO) stem cell transplantation (SCT). However, it remains unclear whether this strategy is effective in SCT from 1-antigen human leukocyte antigen (HLA)-mismatched unrelated donors (9/10 MUD) and how the outcomes of these patients compare with those of haploidentical transplantation recipients.

Methods: A parallel, 2-arm, nonrandomized phase 2 clinical trial was conducted of melphalan-based reduced-intensity conditioning with PTCy, tacrolimus, and mycophenolate mofetil to prevent GVHD in patients with high-risk hematologic malignancies who underwent HAPLO (n = 60) or 9/10 MUD (n = 46) SCT.

Results: The 1-year overall and progression-free survival rates were 70% and 60%, respectively, in the HAPLO arm and 60% and 47%, respectively, in the 9/10 MUD arm. The day +100 cumulative incidence of grade II to IV acute GVHD and grade III to IV acute GVHD was 28% and 3%, respectively, in the HAPLO arm and 33% and 13%, respectively, in the 9/10 MUD arm. The 2-year cumulative incidence of chronic GVHD was 24% in the HAPLO arm and 19% in the 9/10 MUD arm. The 1-year cumulative incidence of nonrelapse mortality was 21% in the HAPLO arm and 31% in the 9/10 MUD arm, and the 1-year relapse rate was 19% in the HAPLO arm and 25% in the 9/10 MUD arm.

Conclusions: Although this was a nonrandomized study and could not serve as a direct comparison between the 2 groups, the authors conclude that PTCy-based GVHD prophylaxis is effective for both HAPLO and 9/10 MUD SCTs. Prospective randomized trials will be required to compare the efficacies of alternative donor options for patients lacking HLA-matched donors. Cancer 2016;122:3316-3326. © 2016 American Cancer Society.

Keywords: 9/10 matched unrelated donors; graft-versus-host disease; haploidentical transplantation; hematologic malignancies; post-transplantation cyclophosphamide.

Publication types

  • Clinical Trial, Phase II
  • Comparative Study

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Cyclophosphamide / therapeutic use*
  • Drug Therapy, Combination
  • Feasibility Studies
  • Female
  • Follow-Up Studies
  • Graft vs Host Disease / etiology
  • Graft vs Host Disease / prevention & control*
  • Hematologic Neoplasms / therapy*
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Histocompatibility Testing
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Middle Aged
  • Mycophenolic Acid / therapeutic use
  • Prognosis
  • Prospective Studies
  • Tacrolimus / therapeutic use
  • Transplantation Conditioning
  • Transplantation, Homologous
  • Unrelated Donors*
  • Young Adult


  • Immunosuppressive Agents
  • Cyclophosphamide
  • Mycophenolic Acid
  • Tacrolimus