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

Orly Klein, Jessica Buddenbaum, Noah Tucker, Allen R Chen, Christopher Gamper, David Loeb, Elias Zambidis, Nicolas Llosa, Jeffrey Huo, Nancy Robey, Mary Jo Holuba, Yvette L. Kasamon, Shannon R. McCurdy, Richard F Ambinder, F Javier Bolanos Meade, Leo Luznik, Ephraim J Fuchs, Richard J Jones, Kenneth R Cooke, Heather Symons

Research output: Contribution to journalArticle


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.

Original languageEnglish (US)
Pages (from-to)325-332
Number of pages8
JournalBiology of Blood and Marrow Transplantation
Issue number2
StatePublished - Feb 1 2017



  • Acute leukemia
  • Cyclophosphamide
  • HLA-haploidentical transplantation
  • Lymphoma
  • Myelodysplastic syndrome
  • Nonmyeloablative bone marrow transplantation

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this