Outcome of transplantation for myelofibrosis.
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.
Ballen
Karen K
KK
Division of Hematology/Oncology, Massachusetts General Hospital, Boston, MA 02114, USA. kballen@partners.org
Shrestha
Smriti
S
Sobocinski
Kathleen A
KA
Zhang
Mei-Jie
MJ
Bashey
Asad
A
Bolwell
Brian J
BJ
Cervantes
Francisco
F
Devine
Steven M
SM
Gale
Robert Peter
RP
Gupta
Vikas
V
Hahn
Theresa E
TE
Hogan
William J
WJ
Kröger
Nicolaus
N
Litzow
Mark R
MR
Marks
David I
DI
Maziarz
Richard T
RT
McCarthy
Philip L
PL
Schiller
Gary
G
Schouten
Harry C
HC
Roy
Vivek
V
Wiernik
Peter H
PH
Horowitz
Mary M
MM
Giralt
Sergio A
SA
Arora
Mukta
M
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