title: Outcome of transplantation for myelofibrosis.
abstract: Myelofibrosis is a myeloproliferative disorder incurable with conventional strategies. Several small series have reported long-term disease-free survival (DSF) after allogeneic hematopoietic cell transplantation (HCT). In this study, we analyze the outcomes of 289 patients receiving allogeneic transplantation for primary myelofibrosis between 1989 and 2002, from the database of the Center for International Bone Marrow Transplant Research (CIBMTR). The median age was 47 years (range: 18-73 years). Donors were HLA identical siblings in 162 patients, unrelated individuals in 101 patients, and HLA nonidentical family members in 26 patients. Patients were treated with a variety of conditioning regimens and graft-versus-host disease (GVHD) prophylaxis regimens. Splenectomy was performed in 65 patients prior to transplantation. The 100-day treatment-related mortality was 18% for HLA identical sibling transplants, 35% for unrelated transplants, and 19% for transplants from alternative related donors. Corresponding 5-year overall survival (OS) rates were 37%, 30%, and 40%, respectively. DFS rates were 33%, 27%, and 22%, respectively. DFS for patients receiving reduced-intensity transplants was comparable: 39% for HLA identical sibling donors and 17% for unrelated donors at 3 years. In this large retrospective series, allogeneic transplantation for myelofibrosis resulted in long-term relapse-free survival (RFS) in about one-third of patients.
authors.affiliation: Division of Hematology/Oncology, Massachusetts General Hospital, Boston, MA 02114, USA. kballen@partners.org
authors.forename: Karen K
authors.initials: KK
authors.lastname: Ballen
authors.forename: Smriti
authors.initials: S
authors.lastname: Shrestha
authors.forename: Kathleen A
authors.initials: KA
authors.lastname: Sobocinski
authors.forename: Mei-Jie
authors.initials: MJ
authors.lastname: Zhang
authors.forename: Asad
authors.initials: A
authors.lastname: Bashey
authors.forename: Brian J
authors.initials: BJ
authors.lastname: Bolwell
authors.forename: Francisco
authors.initials: F
authors.lastname: Cervantes
authors.forename: Steven M
authors.initials: SM
authors.lastname: Devine
authors.forename: Robert Peter
authors.initials: RP
authors.lastname: Gale
authors.forename: Vikas
authors.initials: V
authors.lastname: Gupta
authors.forename: Theresa E
authors.initials: TE
authors.lastname: Hahn
authors.forename: William J
authors.initials: WJ
authors.lastname: Hogan
authors.forename: Nicolaus
authors.initials: N
authors.lastname: Kröger
authors.forename: Mark R
authors.initials: MR
authors.lastname: Litzow
authors.forename: David I
authors.initials: DI
authors.lastname: Marks
authors.forename: Richard T
authors.initials: RT
authors.lastname: Maziarz
authors.forename: Philip L
authors.initials: PL
authors.lastname: McCarthy
authors.forename: Gary
authors.initials: G
authors.lastname: Schiller
authors.forename: Harry C
authors.initials: HC
authors.lastname: Schouten
authors.forename: Vivek
authors.initials: V
authors.lastname: Roy
authors.forename: Peter H
authors.initials: PH
authors.lastname: Wiernik
authors.forename: Mary M
authors.initials: MM
authors.lastname: Horowitz
authors.forename: Sergio A
authors.initials: SA
authors.lastname: Giralt
authors.forename: Mukta
authors.initials: M
authors.lastname: Arora
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D018572:Disease-Free Survival; D005260:Female; D006084:Graft Rejection / Q000453:epidemiology; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation / Q000401:mortality*; D006648:Histocompatibility / Q000235:genetics; D006801:Humans; D053208:Kaplan-Meier Estimate; D017567:Karnofsky Performance Status; D008297:Male; D008875:Middle Aged; D011183:Postoperative Complications / Q000401:mortality; D055728:Primary Myelofibrosis / Q000401:mortality* / Q000628:therapy*; D012008:Recurrence; D012189:Retrospective Studies; D012307:Risk Factors; D035781:Siblings; D013163:Splenomegaly; D019172:Transplantation Conditioning / Q000379:methods / Q000706:statistics & numerical data; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult