Hematopoietic Cell Transplantation as Curative Therapy for Idiopathic Myelofibrosis, Advanced Polycythemia Vera, and Essential Thrombocythemia

Daniella M.B. Kerbauy, Theodore A. Gooley, George E. Sale, Mary E.D. Flowers, Kristine C. Doney, George E. Georges, Joanne E. Greene, Michael Linenberger, Effie Petersdorf, Brenda M. Sandmaier, Bart L. Scott, Mohamed Sorror, Derek L. Stirewalt, F. Marc Stewart, Robert P. Witherspoon, Rainer Storb, Frederick R. Appelbaum, H. Joachim Deeg*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

152 Scopus citations


A total of 104 patients, aged 18 to 70 years, with a diagnosis of chronic idiopathic myelofibrosis (CIMF), polycythemia vera (PV), or essential thrombocythemia (ET) with marrow fibrosis were transplanted from allogeneic (56 related and 45 unrelated) or syngeneic (n = 3) donors. Busulfan (BU) or total body irradiation (TBI)-based myeloablative conditioning regimens were used in 95 patients, and a nonmyeloablative regimen of fludarabine plus TBI was used in 9 patients. The source of stem cells was bone marrow in 43 patients and peripheral blood in 61 patients. A total of 63 patients were alive at a follow-up of 1.3-15.2 years (median, 5.3 years), for an estimated 7-year actuarial survival rate of 61%. Eleven patients had recurrent/persistent disease, of whom 8 died. Nonrelapse mortality was 34% at 5 years. Patients conditioned with targeted BU (plasma levels 800-900 ng/mL) plus cyclophosphamide (tBUCY) had a higher probability of survival (68%) than other patients. Dupriez score, platelet count, patient age, and comorbidity score were statistically significantly associated with mortality in univariate models. In a multivariable regression model, use of tBUCY (P = .03), high platelet count at transplantation (P = .01 for PV/ET; P = .39 for other diagnoses), younger patient age (P = .04), and decreased comorbidity score (P = .03) remained statistically significant for improved survival. Our findings show that hematopoietic cell transplantation offers potentially curative treatment for patients with ICMF, PV, or ET.

Original languageEnglish (US)
Pages (from-to)355-365
Number of pages11
JournalBiology of Blood and Marrow Transplantation
Issue number3
StatePublished - Mar 2007


  • Essential thrombocythemia
  • Hematopoietic cell transplantation
  • Myelofibrosis
  • Polycythemia vera

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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