title: Allogeneic hematopoietic stem cell transplantation for myelofibrosis.
abstract: Fifty-six patients, 10 to 66 years of age, with idiopathic myelofibrosis (IMF) or end-stage polycythemia vera or essential thrombocythemia received allogeneic hematopoietic 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 3 patients achieved engraftment. While 50 patients showed complete donor chimerism, 3 patients were found to be mixed chimeras at 26, 48, and 86 months after transplantation, respectively. Two patients died from relapse/progressive disease, and 18 died from other causes. There are 36 patients surviving at 0.5 to 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 posttransplantation mortality. Patients conditioned with a regimen of busulfan targeted to plasma levels of 800 to 900 ng/mL plus cyclophosphamide had a higher probability of survival (76% [CI, 62%-91%]) than other patients. Results with unrelated donors were comparable with those with HLA-identical sibling transplants. Thus, allogeneic hematopoietic cell transplantation offers long-term relapse-free survival for patients with myelofibrosis.
authors.affiliation: Fred Hutchinson Cancer Research Center, 1100 Fairview Ave N, D1-100, PO Box 19024, Seattle, WA 98109-1024, USA. jdeeg@fhcrc.org
authors.forename: H Joachim
authors.initials: HJ
authors.lastname: Deeg
authors.forename: Theodore A
authors.initials: TA
authors.lastname: Gooley
authors.forename: Mary E D
authors.initials: ME
authors.lastname: Flowers
authors.forename: George E
authors.initials: GE
authors.lastname: Sale
authors.forename: John T
authors.initials: JT
authors.lastname: Slattery
authors.forename: Claudio
authors.initials: C
authors.lastname: Anasetti
authors.forename: Thomas R
authors.initials: TR
authors.lastname: Chauncey
authors.forename: Kristine
authors.initials: K
authors.lastname: Doney
authors.forename: George E
authors.initials: GE
authors.lastname: Georges
authors.forename: Hans-Peter
authors.initials: HP
authors.lastname: Kiem
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Martin
authors.forename: Effie W
authors.initials: EW
authors.lastname: Petersdorf
authors.forename: Jerald
authors.initials: J
authors.lastname: Radich
authors.forename: Jean E
authors.initials: JE
authors.lastname: Sanders
authors.forename: Brenda M
authors.initials: BM
authors.lastname: Sandmaier
authors.forename: E Houston
authors.initials: EH
authors.lastname: Warren
authors.forename: Robert P
authors.initials: RP
authors.lastname: Witherspoon
authors.forename: Rainer
authors.initials: R
authors.lastname: Storb
authors.forename: Frederick R
authors.initials: FR
authors.lastname: Appelbaum
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use* / Q000633:toxicity; D002066:Busulfan / Q000627:therapeutic use; D002423:Cause of Death; D002648:Child; D003520:Cyclophosphamide / Q000627:therapeutic use; D005260:Female; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D006801:Humans; D008297:Male; D008875:Middle Aged; D011087:Polycythemia Vera / Q000150:complications / Q000401:mortality / Q000628:therapy; D055728:Primary Myelofibrosis / Q000150:complications / Q000401:mortality / Q000628:therapy*; D012307:Risk Factors; D016019:Survival Analysis; D013920:Thrombocythemia, Essential / Q000150:complications / Q000401:mortality / Q000628:therapy; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods; D014184:Transplantation, Homologous
chemical_list: D003520:Cyclophosphamide; D002066:Busulfan