title: MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis.
abstract: From 2007 to 2011, 66 patients with primary myelofibrosis or myelofibrosis (MF) preceded by essential thrombocythemia or polycythemia vera were enrolled into a prospective phase 2 clinical trial of reduced-intensity allogeneic hematopoietic stem cell transplantation (AHSCT), Myeloproliferative Disorder Research Consortium 101 trial. The study included patients with sibling donors (n = 32) receiving fludarabine/melphalan (FluMel) as a preparative regimen and patients with unrelated donors (n = 34) receiving conditioning with FluMel plus anti-thymocyte globulin (ATG). Patient characteristics in the 2 cohorts were similar. Engraftment occurred in 97% of siblings and 76% of unrelated transplants, whereas secondary graft failure occurred in 3% and 12%, respectively. With a median follow-up of 25 months for patients alive, the overall survival (OS) was 75% in the sibling group (median not reached) and 32% in the unrelated group (median OS: 6 months, 95% confidence interval [CI]: 3, 25) (hazard ratio 3.9, 95% CI: 1.8,8.9) (P < .001). Nonrelapse mortality was 22% in sibling and 59% in unrelated AHSCT. Survival correlated with type of donor, but not with the degree of histocompatibility match, age, or JAK2(V617F) status. In patients with MF with sibling donors, AHSCT is an effective therapy, whereas AHSCT from unrelated donors with FluMel/ATG conditioning led to a high rate of graft failure and limited survival. This trial was registered at www.clinicaltrials.gov as #NCT00572897.
authors: University of Illinois Hospital & Health Sciences System and University of Illinois Cancer Center, Chicago, IL; Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY;
authors: Damiano
authors: http://orcid.org/0000-0001-5243-401X
authors: D
authors: Rondelli
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; New York University School of Medicine, New York, NY;
authors: Judith D
authors: http://orcid.org/0000-0003-3758-6976
authors: JD
authors: Goldberg
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY;
authors: Luis
authors: L
authors: Isola
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; New York University School of Medicine, New York, NY;
authors: Leah S
authors: LS
authors: Price
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Weill Cornell Medical College, New York, NY;
authors: Tsiporah B
authors: TB
authors: Shore
authors: University of Utah School of Medicine, Salt Lake City, UT;
authors: Michael
authors: http://orcid.org/0000-0002-0452-2987
authors: M
authors: Boyer
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Ospedale San Martino, Genoa, Italy;
authors: Andrea
authors: A
authors: Bacigalupo
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy;
authors: Alessandro
authors: A
authors: Rambaldi
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Foundation Mario Negri Sud, S. Maria Imbaro, Italy;
authors: Marco
authors: M
authors: Scarano
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; The Ohio State University Comprehensive Cancer Center, Columbus, OH;
authors: Rebecca B
authors: RB
authors: Klisovic
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Princess Margaret Hospital Cancer Center, Toronto, ON, Canada;
authors: Vikas
authors: V
authors: Gupta
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Nephro-Urology Hospital Organization, Uddevalla, Sweden;
authors: Bjorn
authors: B
authors: Andreasson
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY;
authors: John
authors: http://orcid.org/0000-0002-8400-0483
authors: J
authors: Mascarenhas
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Roswell Park Cancer Institute, Buffalo, NY;
authors: Meir
authors: M
authors: Wetzler
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; University of Florence, Florence, Italy;
authors: Alessandro M
authors: AM
authors: Vannucchi
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; University of Utah School of Medicine, Salt Lake City, UT;
authors: Josef T
authors: JT
authors: Prchal
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY;
authors: Vesna
authors: V
authors: Najfeld
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Weill Cornell Medical College, New York, NY;
authors: Attilio
authors: A
authors: Orazi
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; New York Blood Center, New York, NY; and.
authors: Rona S
authors: RS
authors: Weinberg
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY;
authors: Crystal
authors: C
authors: Miller
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Istituto Di Ricovero e Cura a Carattere Scientifico Policlinico S. Matteo Foundation, Pavia, Italy.
authors: Giovanni
authors: G
authors: Barosi
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY;
authors: Lewis R
authors: LR
authors: Silverman
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Foundation Mario Negri Sud, S. Maria Imbaro, Italy;
authors: Giuseppe
authors: G
authors: Prosperini
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Foundation Mario Negri Sud, S. Maria Imbaro, Italy;
authors: Roberto
authors: R
authors: Marchioli
authors: Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY;
authors: Ronald
authors: R
authors: Hoffman
mesh_terms: D000328:Adult; D000368:Aged; D000704:Analysis of Variance; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D001782:Blood Donors; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000209:etiology / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006648:Histocompatibility; D006801:Humans; D053614:Janus Kinase 2 / Q000235:genetics; D053208:Kaplan-Meier Estimate; D008297:Male; D008558:Melphalan / Q000627:therapeutic use; D008875:Middle Aged; D009154:Mutation; D055728:Primary Myelofibrosis / Q000235:genetics / Q000628:therapy*; D011446:Prospective Studies; D035781:Siblings; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D061349:Unrelated Donors; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use
chemical_list: D000961:Antilymphocyte Serum; C507924:JAK2 protein, human; D053614:Janus Kinase 2; D014740:Vidarabine; C024352:fludarabine; D008558:Melphalan