Reduced-intensity hematopoietic cell transplantation for patients with primary myelofibrosis: a cohort analysis from the center for international blood and marrow transplant research

Biol Blood Marrow Transplant. 2014 Jan;20(1):89-97. doi: 10.1016/j.bbmt.2013.10.018. Epub 2013 Oct 23.

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.

Keywords: Allogeneic transplantation; Myelofibrosis; Prognosis; Reduced intensity.

Publication types

  • Multicenter Study
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adult
  • Aged
  • Cohort Studies
  • Female
  • HLA Antigens / immunology
  • Hematopoietic Stem Cell Transplantation*
  • Histocompatibility Testing
  • Humans
  • Male
  • Middle Aged
  • Multivariate Analysis
  • Myeloablative Agonists / therapeutic use*
  • Primary Myelofibrosis / immunology
  • Primary Myelofibrosis / mortality
  • Primary Myelofibrosis / pathology
  • Primary Myelofibrosis / therapy*
  • Prognosis
  • Recurrence
  • Risk
  • Siblings
  • Survival Analysis
  • Transplantation Conditioning / methods*
  • Transplantation, Homologous
  • Unrelated Donors / classification

Substances

  • HLA Antigens
  • Myeloablative Agonists