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 M Borrello, William Matsui, Jonathan 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

251 Scopus citations


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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