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Prepublished online as a Blood First Edition Paper on August 14, 2003; DOI 10.1182/blood-2003-06-1856.

Submitted June 10, 2003
Accepted August 5, 2003
Allogeneic hemopoietic stem cell transplantation for myelofibrosis
H Joachim Deeg*, Theodore A Gooley, Mary E D Flowers, George E Sale, John T Slattery, Claudio Anasetti, Thomas R Chauncey, Kristine Doney, George E Georges, Hans-Peter Kiem, Paul J Martin, Effie W Petersdorf, Jerald Radich, Jean E Sanders, Brenda M Sandmaier, E Houston Warren, Robert P Witherspoon, Rainer Storb, and Frederick R Appelbaum
Fred Hutchinson Cancer Research Center, Seattle, WA, USA; University of Washington, Seattle, WA, USA
Seattle VA Medical Center, Seattle, WA, USA
* Corresponding author; email: jdeeg{at}fhcrc.org.
Fifty-six patients, 10-66 years of age, with idopathic myelofibrosis (IMF) or endstage polycythemia vera or essential thrombocythemia received allogeneic hemopoietic cell transplants from related (n=36) or unrelated (n=20) donors. Forty-four patients were prepared with busulfan plus cyclophosphamide, and 12 with total body irradiation plus chemotherapy. The source of stem cells was marrow in 33, and peripheral blood in 23 patients. All but three patients achieved engraftment. While 50 patients showed complete donor chimerism, three patients were found to be mixed chimeras 26, 48, and 86 months post-transplant, respectively. Two patients died from relapse/progressive disease, and 18 from other causes. Thirty-six patients are surviving at 0.5-11.6 (median 2.8) years for a 3-year Kaplan-Meier estimate of 58% (CI 43%, 73%). Dupriez score, cytogenetic abnormalities and degree of marrow fibrosis were the most significant risk factors for post-transplant mortality. Patients conditioned with a regimen of busulfan targeted to plasma levels of 800-900 ng/mL plus cyclophosphamide had a higher probability of survival (76% [CI 62%, 91%]) than other patients. Results with unrelated donors were comparable to those with HLA-identical sibling transplants. Thus, allogeneic hemopoietic cell transplantation offers long-term relapse-free survival for patients with myelofibrosis.

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