Nonmyeloablative Haploidentical Bone Marrow Transplantation with Post-Transplantation Cyclophosphamide for Pediatric and Young Adult Patients with High-Risk Hematologic Malignancies

Biol Blood Marrow Transplant. 2017 Feb;23(2):325-332. doi: 10.1016/j.bbmt.2016.11.016. Epub 2016 Nov 22.


Lower-intensity conditioning regimens for haploidentical blood or marrow transplantation (BMT) are safe and efficacious for adult patients with hematologic malignancies. We report data for pediatric/young adult patients with high-risk hematologic malignancies (n = 40) treated with nonmyeloablative haploidentical BMT with post-transplantation cyclophosphamide from 2003 to 2015. Patients received a preparative regimen of fludarabine, cyclophosphamide, and total body irradiation. Post-transplantation immunosuppression consisted of cyclophosphamide, mycophenolate mofetil, and tacrolimus. Donor engraftment occurred in 29 of 32 (91%), with median time to engraftment of neutrophils >500/µL of 16 days (range, 13 to 22) and for platelets >20,000/µL without transfusion of 18 days (range, 12 to 62). Cumulative incidences of acute graft-versus-host disease (GVHD) grades II to IV and grades III and IV at day 100 were 33% and 5%, respectively. The cumulative incidence of chronic GVHD was 23%, with 7% moderate-to-severe chronic GVHD, according to National Institutes of Health consensus criteria. Transplantation-related mortality (TRM) at 1 year was 13%. The cumulative incidence of relapse at 2 years was 52%. With a median follow-up of 20 months (range, 3 to 148), 1-year actuarial overall and event-free survival were 56% and 43%, respectively. Thus, we demonstrate excellent rates of engraftment, GVHD, and TRM in pediatric/young adult patients treated with this regimen. This approach is a widely available, safe, and feasible option for pediatric and young adult patients with high-risk hematologic malignancies, including those with a prior history of myeloablative BMT and/or those with comorbidities or organ dysfunction that preclude eligibility for myeloablative BMT.

Keywords: Acute leukemia; Cyclophosphamide; HLA-haploidentical transplantation; Lymphoma; Myelodysplastic syndrome; Nonmyeloablative bone marrow transplantation.

Publication types

  • Evaluation Study

MeSH terms

  • Adolescent
  • Allografts
  • Bone Marrow Transplantation*
  • Child
  • Child, Preschool
  • Cyclophosphamide / therapeutic use*
  • Disease-Free Survival
  • Female
  • Graft vs Host Disease / etiology
  • Hematologic Neoplasms / therapy*
  • Histocompatibility
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Infant, Newborn
  • Male
  • Myelodysplastic Syndromes / therapy
  • Retrospective Studies
  • Risk
  • Transplantation Conditioning / methods*
  • Treatment Outcome
  • Young Adult


  • Immunosuppressive Agents
  • Cyclophosphamide