title: Reduced-intensity hematopoietic cell transplantation for patients with primary myelofibrosis: a cohort analysis from the center for international blood and marrow transplant research.
abstract: We evaluated outcomes and associated prognostic factors in 233 patients undergoing allogeneic hematopoietic cell transplantation (HCT) for primary myelofibrosis (MF) using reduced-intensity conditioning (RIC). The median age at RIC HCT was 55 yr. Donors were a matched sibling donor (MSD) in 34% of RIC HCTs, an HLA well-matched unrelated donor (URD) in 45%, and a partially matched/mismatched URD in 21%. Risk stratification according to the Dynamic International Prognostic Scoring System (DIPSS) was 12% low, 49% intermediate-1, 37% intermediate-2, and 1% high. The probability of survival at 5 yr was 47% (95% confidence interval [CI], 40% to 53%). In a multivariate analysis, donor type was the sole independent factor associated with survival. Adjusted probabilities of survival at 5-yr were 56% (95% CI, 44% to 67%) for MSD, 48% (95% CI, 37% to 58%) for well-matched URD, and 34% (95% CI, 21% to 47%) for partially matched/mismatched URD (P = .002). The relative risk (RR) for NRM was 3.92 (P = .006) for well-matched URD and 9.37 (P < .0001) for partially matched/mismatched URD. Trends toward increased NRM (RR, 1.7; P = .07) and inferior survival (RR, 1.37; P = .10) were observed in DIPSS intermediate-2/high-risk patients compared with DIPSS low/intermediate-1 risk patients. Our data indicate that RIC HCT is a potentially curative option for patients with MF, and that donor type is the most important factor influencing survival in these patients.
authors.affiliation: Princess Margaret Cancer Center, University of Toronto, Toronto, Canada. Electronic address: vikas.gupta@uhn.on.ca.
authors.forename: Vikas
authors.initials: V
authors.lastname: Gupta
authors.affiliation: Mount Sinai Medical Center, New York, New York.
authors.forename: Adriana K
authors.initials: AK
authors.lastname: Malone
authors.affiliation: Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Parameswaran N
authors.initials: PN
authors.lastname: Hari
authors.affiliation: Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Kwang Woo
authors.initials: KW
authors.lastname: Ahn
authors.affiliation: Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Zhen-Huan
authors.initials: ZH
authors.lastname: Hu
authors.affiliation: Section of Hematology, Division of Experimental Medicine, Department of Medicine, Imperial College, London, UK.
authors.forename: Robert Peter
authors.initials: RP
authors.lastname: Gale
authors.affiliation: Massachusetts General Hospital, Boston, Massachusetts.
authors.forename: Karen K
authors.initials: KK
authors.lastname: Ballen
authors.affiliation: West Virginia University Hospitals, Morgantown, West Virginia.
authors.forename: Mehdi
authors.initials: M
authors.lastname: Hamadani
authors.affiliation: Hematology Department and BMT Unit, Complejo Hospitalario de Navarra, Pamplona, Spain.
authors.forename: Eduardo
authors.initials: E
authors.lastname: Olavarria
authors.affiliation: Taussig Cancer Institute, Cleveland Clinic, Cleveland, Ohio.
authors.forename: Aaron T
authors.initials: AT
authors.lastname: Gerds
authors.affiliation: Bone Marrow and Stem Cell Transplant Center, Emory University Hospital, Atlanta, Georgia.
authors.forename: Edmund K
authors.initials: EK
authors.lastname: Waller
authors.affiliation: Blood and Marrow Transplant Program, Medical University of South Carolina, Charleston, South Carolina.
authors.forename: Luciano J
authors.initials: LJ
authors.lastname: Costa
authors.affiliation: Dana Farber Cancer Institute, Boston, Massachusetts.
authors.forename: Joseph H
authors.initials: JH
authors.lastname: Antin
authors.affiliation: Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, Texas.
authors.forename: Rammurti T
authors.initials: RT
authors.lastname: Kamble
authors.affiliation: Stem Cell Transplant Program, Weill Cornell Medical College, New York, New York.
authors.forename: Koen M
authors.initials: KM
authors.lastname: van Besien
authors.affiliation: Vanderbilt University Medical Center, Nashville, Tennessee.
authors.forename: Bipin N
authors.initials: BN
authors.lastname: Savani
authors.affiliation: Academische Ziekenhuis Maastricht, Maastricht, The Netherlands.
authors.forename: Harry C
authors.initials: HC
authors.lastname: Schouten
authors.affiliation: Clinical Haematology and BMT Service, Royal Melbourne Hospital, Victoria, Australia.
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Szer
authors.affiliation: Department of Hematology, University Hospital, Grenoble, France.
authors.forename: Jean-Yves
authors.initials: JY
authors.lastname: Cahn
authors.affiliation: Seidman Cancer Center, University Hospitals Case Medical Center, Cleveland, Ohio.
authors.forename: Marcos J
authors.initials: MJ
authors.lastname: de Lima
authors.affiliation: Department of Hematology/Oncology, Shands HealthCare and University of Florida, Gainesville, Florida.
authors.forename: Baldeep
authors.initials: B
authors.lastname: Wirk
authors.affiliation: King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia.
authors.forename: Mahmoud D
authors.initials: MD
authors.lastname: Aljurf
authors.affiliation: Stem Cell Transplantation and Cellular Therapy, M.D. Anderson Cancer Center, Houston, Texas.
authors.forename: Uday
authors.initials: U
authors.lastname: Popat
authors.affiliation: Division of Hematology, Oncology, and Transplantation, University of Minnesota Medical Center, Minneapolis, Minnesota.
authors.forename: Nelli
authors.initials: N
authors.lastname: Bejanyan
authors.affiliation: Mayo Clinic Rochester, Rochester, Minnesota.
authors.forename: Mark R
authors.initials: MR
authors.lastname: Litzow
authors.affiliation: Department of Hematology/Oncology, Shands HealthCare and University of Florida, Gainesville, Florida.
authors.forename: Maxim
authors.initials: M
authors.lastname: Norkin
authors.affiliation: Haematology and Bone Marrow Transplant Unit, Royal Adelaide Hospital/SA Pathology, Adelaide, Australia.
authors.forename: Ian D
authors.initials: ID
authors.lastname: Lewis
authors.affiliation: All Children's Hospital, St Petersburg, Florida.
authors.forename: Gregory A
authors.initials: GA
authors.lastname: Hale
authors.affiliation: Taussig Cancer Institute, Cleveland Clinic, Cleveland, Ohio.
authors.forename: Ann E
authors.initials: AE
authors.lastname: Woolfrey
authors.affiliation: Baylor University Medical Center, Dallas, Texas.
authors.forename: Alan M
authors.initials: AM
authors.lastname: Miller
authors.affiliation: Division of Hematology, Oncology, and Transplantation, University of Minnesota Medical Center, Minneapolis, Minnesota.
authors.forename: Celalettin
authors.initials: C
authors.lastname: Ustun
authors.affiliation: Vanderbilt University Medical Center, Nashville, Tennessee.
authors.forename: Madan H
authors.initials: MH
authors.lastname: Jagasia
authors.affiliation: Division of Hematology/Oncology, Cedars-Sinai Medical Center, Los Angeles, California.
authors.forename: Michael
authors.initials: M
authors.lastname: Lill
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University, Portland, Oregon.
authors.forename: Richard T
authors.initials: RT
authors.lastname: Maziarz
authors.affiliation: Stem Cell Transplantation and Cellular Therapy, M.D. Anderson Cancer Center, Houston, Texas.
authors.forename: Jorge
authors.initials: J
authors.lastname: Cortes
authors.affiliation: Taussig Cancer Institute, Cleveland Clinic, Cleveland, Ohio.
authors.forename: Matt E
authors.initials: ME
authors.lastname: Kalaycio
authors.affiliation: Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Wael
authors.initials: W
authors.lastname: Saber
mesh_terms: D000328:Adult; D000368:Aged; D015331:Cohort Studies; D005260:Female; D006680:HLA Antigens / Q000276:immunology; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D008297:Male; D008875:Middle Aged; D015999:Multivariate Analysis; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D055728:Primary Myelofibrosis / Q000276:immunology / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D011379:Prognosis; D012008:Recurrence; D012306:Risk; D035781:Siblings; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D061349:Unrelated Donors / Q000145:classification
chemical_list: D006680:HLA Antigens; D019653:Myeloablative Agonists
keywords: Allogeneic transplantation; Myelofibrosis; Prognosis; Reduced intensity