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

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

Research output: Contribution to journalArticle

24 Scopus citations

Abstract

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
Volume23
Issue number2
DOIs
StatePublished - Feb 1 2017
Externally publishedYes

Keywords

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

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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    Klein, O. R., Buddenbaum, J., Tucker, N., Chen, A. R., Gamper, C. J., Loeb, D., Zambidis, E., Llosa, N. J., Huo, J. S., Robey, N., Holuba, M. J., Kasamon, Y. L., McCurdy, S. R., Ambinder, R., Bolaños-Meade, J., Luznik, L., Fuchs, E. J., Jones, R. J., Cooke, K. R., & Symons, H. J. (2017). Nonmyeloablative Haploidentical Bone Marrow Transplantation with Post-Transplantation Cyclophosphamide for Pediatric and Young Adult Patients with High-Risk Hematologic Malignancies. Biology of Blood and Marrow Transplantation, 23(2), 325-332. https://doi.org/10.1016/j.bbmt.2016.11.016