Reduced-Intensity Hematopoietic Cell Transplantation for Patients with Primary Myelofibrosis: A Cohort Analysis from the Center for International Blood and Marrow Transplant Research

Vikas Gupta, Adriana K. Malone, Parameswaran N. Hari, Kwang Woo Ahn, Zhen Huan Hu, Robert Peter Gale, Karen K. Ballen, Mehdi Hamadani, Eduardo Olavarria, Aaron T. Gerds, Edmund K. Waller, Luciano J. Costa, Joseph H. Antin, Rammurti T. Kamble, Koen M. van Besien, Bipin N. Savani, Harry C. Schouten, Jeffrey Szer, Jean Yves Cahn, Marcos J. de LimaBaldeep Wirk, Mahmoud D. Aljurf, Uday Popat, Nelli Bejanyan, Mark R. Litzow, Maxim Norkin, Ian D. Lewis, Gregory A. Hale, Ann E. Woolfrey, Alan M. Miller, Celalettin Ustun, Madan H. Jagasia, Michael Lill, Richard T. Maziarz, Jorge Cortes, Matt E. Kalaycio, Wael Saber

Research output: Contribution to journalArticlepeer-review

94 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)89-97
Number of pages9
JournalBiology of Blood and Marrow Transplantation
Volume20
Issue number1
DOIs
StatePublished - Jan 2014

Bibliographical note

Funding Information:
Financial disclosure: The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement U24 CA076518 from the National Cancer Institute (NCI) , the National Heart, Lung and Blood Institute (NHLBI) , and the National Institute of Allergy and Infectious Diseases (NIAID) ; Grant/Cooperative Agreement U10 HL069294 from the NHLBI and NCI; Contract HHSH250201200016C with Health Resources and Services Administration; Grants N00014-12-1-0142 and N00014-13-1-0039 from the Office of Naval Research ; and grants from Allos Therapeutics, Amgen , anonymous donation to the Medical College of Wisconsin, Ariad, Be the Match Foundation, Blue Cross and Blue Shield Association, Celgene, Fresenius-Biotech North America, Gamida Cell Teva Joint Venture, Genentech, Gentium, Genzyme, GlaxoSmithKline, HistoGenetics, Kiadis Pharma; Leukemia and Lymphoma Society, Medical College of Wisconsin; Merck & Co, Millennium: Takeda Oncology, Milliman USA, Miltenyi Biotec, National Marrow Donor Program, Onyx Pharmaceuticals, Optum Healthcare Solutions, Osiris Therapeutics, Otsuka America Pharmaceutical, Remedy Informatics, Sanofi US, Seattle Genetics, Sigma-Tau Pharmaceuticals, Soligenix, StemCyte, Stemsoft Software, Swedish Orphan Biovitrum, Tarix Pharmaceuticals, TerumoBCT, Teva Neuroscience, Therakos, and Wellpoint. The views expressed in this article do not reflect the official policy or position of the National Institutes of Health, Department of the Navy, Department of Defense, or any other agency of the US Government.

Keywords

  • Allogeneic transplantation
  • Myelofibrosis
  • Prognosis
  • Reduced intensity

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