High-dose cyclophosphamide as single-agent, short-course prophylaxis of graft-versus-host disease

Leo Luznik, Javier Bolaños-Meade, Marianna Zahurak, Allen R. Chen, B. Douglas Smith, Robert Brodsky, Carol Ann Huff, Ivan Borrello, William Matsui, Jonathan D. Powell, Yvette Kasamon, Steven N. Goodman, Allan Hess, Hyam I. Levitsky, Richard F. Ambinder, Richard J. Jones, Ephraim J. Fuchs

Research output: Contribution to journalArticlepeer-review


Because of its potent immunosuppressive yet stem cell-sparing activity, high-dose cyclophosphamide was tested as sole prophylaxis of graft-versus-host disease (GVHD) after myeloablative allogeneic bone marrow transplantation (alloBMT). We treated 117 patients (median age, 50 years; range, 21-66 years) with advanced hematologic malignancies; 78 had human leukocyte antigen (HLA)-matched related donors and 39 had HLA-matched unrelated donors. All patients received conventional myeloablation with busulfan/cyclophosphamide (BuCy) and T cell-replete bone marrow followed by 50 mg/kg/d of cyclophosphamide on days 3 and 4 after transplantation. The incidences of acute grades II through IV and grades III through IV GVHD for all patients were 43% and 10%, respectively. The non-relapse mortality at day 100 and 2 years after transplantation were 9% and 17%, respectively. The actuarial overall survival and event-free survivals at 2 years after transplantation were 55% and 39%, respectively, for all patients and 63% and 54%, respectively, for patients who underwent transplantation while in remission. With a median follow-up of 26.3 months among surviving patients, the cumulative incidence of chronic GVHD is 10%. These results suggest that high-dose posttransplantation cyclophosphamide is an effective single-agent prophylaxis of acute and chronic GVHD after BuCy conditioning and HLA-matched BMT (clinicaltrials.gov no. NCT00134017).

Original languageEnglish (US)
Pages (from-to)3224-3230
Number of pages7
Issue number16
StatePublished - Apr 22 2010

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology


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