title: Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation.
abstract: From 2002 to 2007, 103 patients with primary myelofibrosis or postessential thrombocythemia and polycythemia vera myelofibrosis and a median age of 55 years (range, 32-68 years) were included in a prospective multicenter phase 2 trial to determine efficacy of a busulfan (10 mg/kg)/fludarabine (180 mg/m(2))-based reduced-intensity conditioning regimen followed by allogeneic stem cell transplantation from related (n = 33) or unrelated donors (n = 70). All but 2 patients (2%) showed leukocyte and platelet engraftment after a median of 18 and 22 days, respectively. Acute graft-versus-host disease grade 2 to 4 occurred in 27% and chronic graft-versus-host disease in 43% of the patients. Cumulative incidence of nonrelapse mortality at 1 year was 16% (95% confidence interval, 9%-23%) and significantly lower for patients with a completely matched donor (12% vs 38%; P = .003). The cumulative incidence of relapse at 3 years was 22% (95% confidence interval, 13%-31%) and was influenced by Lille risk profile (low, 14%; intermediate, 22%; and high, 34%; P = .02). The estimated 5-year event-free and overall survival was 51% and 67%, respectively. In a multivariate analysis, age older than 55 years (hazard ratio = 2.70; P = .02) and human leukocyte antigen-mismatched donor (hazard ratio = 3.04; P = .006) remained significant factors for survival. The study was registered at www.clinicaltrials.gov as #NCT 00599547.
authors.affiliation: Center for Stem Cell Transplantation, University Hospital Hamburg-Eppendorf, Martinistrasse 52, D-20246 Hamburg, Germany. nkroeger@uke.uni-hamburg.de
authors.forename: Nicolaus
authors.initials: N
authors.lastname: Kröger
authors.forename: Ernst
authors.initials: E
authors.lastname: Holler
authors.forename: Guido
authors.initials: G
authors.lastname: Kobbe
authors.forename: Martin
authors.initials: M
authors.lastname: Bornhäuser
authors.forename: Rainer
authors.initials: R
authors.lastname: Schwerdtfeger
authors.forename: Herrad
authors.initials: H
authors.lastname: Baurmann
authors.forename: Arnon
authors.initials: A
authors.lastname: Nagler
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Bethge
authors.forename: Matthias
authors.initials: M
authors.lastname: Stelljes
authors.forename: Lutz
authors.initials: L
authors.lastname: Uharek
authors.forename: Hannes
authors.initials: H
authors.lastname: Wandt
authors.forename: Andreas
authors.initials: A
authors.lastname: Burchert
authors.forename: Paolo
authors.initials: P
authors.lastname: Corradini
authors.forename: Jörg
authors.initials: J
authors.lastname: Schubert
authors.forename: Martin
authors.initials: M
authors.lastname: Kaufmann
authors.forename: Peter
authors.initials: P
authors.lastname: Dreger
authors.forename: Gerald G
authors.initials: GG
authors.lastname: Wulf
authors.forename: Hermann
authors.initials: H
authors.lastname: Einsele
authors.forename: Tatjana
authors.initials: T
authors.lastname: Zabelina
authors.forename: Hans Michael
authors.initials: HM
authors.lastname: Kvasnicka
authors.forename: Jürgen
authors.initials: J
authors.lastname: Thiele
authors.forename: Ronald
authors.initials: R
authors.lastname: Brand
authors.forename: Axel R
authors.initials: AR
authors.lastname: Zander
authors.forename: Dietger
authors.initials: D
authors.lastname: Niederwieser
authors.forename: Theo M
authors.initials: TM
authors.lastname: de Witte
mesh_terms: D000328:Adult; D000368:Aged; D002066:Busulfan / Q000627:therapeutic use; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000453:epidemiology; D006801:Humans; D008297:Male; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000627:therapeutic use; D055728:Primary Myelofibrosis / Q000401:mortality / Q000601:surgery*; D012008:Recurrence; D033581:Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use
chemical_list: D019653:Myeloablative Agonists; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine