MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis

作者：Rondelli Damiano^*; Goldberg Judith D; Isola Luis; Price Leah S; Shore Tsiporah B; Boyer Michael; Bacigalupo Andrea; Rambaldi Alessandro; Scarano Marco; Klisovic Rebecca B; Gupta Vikas; Andreasson Bjorn; Mascarenhas John; Wetzler Meir; Vannucchi Alessandro M; Prchal Josef T; Najfeld Vesna; Orazi Attilio; Weinberg Rona S; Miller Crystal; Barosi Giovanni; Silverman Lewis R; Prosperini Giuseppe; Marchioli Roberto; Hoffman Ronald

来源：Blood, 2014, 124(7): 1183-1191.

DOI：10.1182/blood-2014-04-572545

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.

• 出版日期2014-8-14