Validation and refinement of the Disease Risk Index for allogeneic stem cell transplantation

Philippe Armand, Haesook T. Kim, Brent R. Logan, Zhiwei Wang, Edwin P. Alyea, Matt E. Kalaycio, Richard T. Maziarz, Joseph H. Antin, Robert J. Soiffer, Daniel J. Weisdorf, J. Douglas Rizzo, Mary M. Horowitz, Wael Saber

Research output: Contribution to journalArticlepeer-review

690 Scopus citations

Abstract

Because the outcome of allogeneic hematopoietic cell transplantation (HCT) is predominantly influenced by disease type and status, it is essential to be able to stratify patients undergoing HCT by disease risk. The Disease Risk Index (DRI) was developed for this purpose. In this study, we analyzed 13 131 patients reported to the Center for International Blood and Marrow Transplant Research who underwent HCT between 2008 and 2010. The DRI stratified patients into 4 groups with 2-year overall survival (OS) ranging from 64% to 24% and was the strongest prognostic factor, regardless of age, conditioning intensity, graft source, or donor type. A randomly selected training subgroup of 9849 patients was used to refine the DRI, using a multivariable regression model for OS. This refined DRI had improved prediction ability for the remaining 3282 patients compared with the original DRI or other existing schemes. This validated and refined DRI can be used as a 4- or 3-group index, depending on the size of the cohort under study, for prognostication; to facilitate the interpretation of single-center, multicenter, or registry studies; to adjust center outcome data; and to stratify patients entering clinical trials that enroll patients across disease categories.

Original languageEnglish (US)
Pages (from-to)3664-3671
Number of pages8
JournalBlood
Volume123
Issue number23
DOIs
StatePublished - 2014

Bibliographical note

Funding Information:
This work was supported by an American Society of Clinical Oncology Career Development Award (P.A.), by grant U19 AI 29530 from the National Institute of Allergy and Infectious Diseases (NIAID), and grant PO1 HL 070149 from the National Heart, Lung and Blood Institute (NHLBI). The Center for International Blood and Marrow Transplant Research is supported by Public Health Service grant/cooperative Agreement U24-CA76518 from the National Cancer Institute (NCI), the NHLBI, and the NIAID, by grant/ cooperative agreement 5U01HL069294 from NHLBI and NCI, by contract HHSH234200637015C with Health Resources and Services Administration/Department of Health and Human Services), by grants N00014-06-1-0704 and N00014-08-1-0058 from the Office of Naval Research, and by grants from Allos, Inc.; Amgen, Inc.; Angioblast; an anonymous donation to the Medical College of Wisconsin; Ariad; Be the Match Foundation; Blue Cross and Blue Shield Association; Buchanan Family Foundation; CaridianBCT; Celgene Corporation; CellGenix, GmbH; Children’s Leukemia Research Association; Fresenius-Biotech North America, Inc.; Gamida Cell Teva Joint Venture Ltd.; Genentech, Inc.; Genzyme Corporation; GlaxoSmithKline; Kiadis Pharma; The Leukemia &

Funding Information:
This work was supported by an American Society of Clinical Oncology Career Development Award (P.A.), by grant U19 AI 29530 from the National Institute of Allergy and Infectious Diseases (NIAID), and grant PO1 HL 070149 from the National Heart, Lung and Blood Institute (NHLBI). The Center for International Blood and Marrow Transplant Research is supported by Public Health Service grant/cooperative Agreement U24-CA76518 from the National Cancer Institute (NCI), the NHLBI, and the NIAID, by grant/ cooperative agreement 5U01HL069294 from NHLBI and NCI, by contract HHSH234200637015C with Health Resources and Services Administration/Department of Health and Human Services), by grants N00014-06-1-0704 and N00014-08-1-0058 from the Office of Naval Research, and by grants from Allos, Inc.; Amgen, Inc.; Angioblast; an anonymous donation to the Medical College of Wisconsin; Ariad; Be the Match Foundation; Blue Cross and Blue Shield Association; Buchanan Family Foundation; CaridianBCT; Celgene Corporation; CellGenix, GmbH; Children's Leukemia Research Association; Fresenius-Biotech North America, Inc.; Gamida Cell Teva Joint Venture Ltd.; Genentech, Inc.; Genzyme Corporation; GlaxoSmithKline; Kiadis Pharma; The Leukemia and Lymphoma Society; The Medical College of Wisconsin; Millennium Pharmaceuticals, Inc.; Milliman USA, Inc.; Miltenyi Biotec, Inc.; National Marrow Donor Program; Optum Healthcare Solutions, Inc.; Otsuka America Pharmaceutical, Inc.; Seattle Genetics; Sigma-Tau Pharmaceuticals; Soligenix, Inc.; Swedish Orphan Biovitrum; Therakos, Inc.; and Wellpoint, Inc.

Publisher Copyright:
© 2014 by The American Society of Hematology.

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