A validated pediatric disease risk index for allogeneic hematopoietic cell transplantation

Muna Qayed, Kwang Woo Ahn, Carrie L. Kitko, Mariam H. Johnson, Nirali N. Shah, Christopher Dvorak, Karin Mellgren, Brian D. Friend, Michael R. Verneris, Wing Leung, Jacek Toporski, John Levine, Joseph Chewning, Alan Wayne, Urvi Kapoor, Brandon Triplett, Kirk R. Schultz, Gregory A. Yanik, Mary Eapen

Research output: Contribution to journalArticlepeer-review

22 Scopus citations

Abstract

A disease risk index (DRI) that was developed for adults with hematologic malignancy who were undergoing hematopoietic cell transplantation is also being used to stratify children and adolescents by disease risk. Therefore, to develop and validate a DRI that can be used to stratify those with AML and ALL by their disease risk, we analyzed 2569 patients aged <18 years with acute myeloid (AML; n = 1224) or lymphoblastic (ALL; n = 1345) leukemia who underwent hematopoietic cell transplantation. Training and validation subsets for each disease were generated randomly with 1:1 assignment to the subsets, and separate prognostic models were derived for each disease. For AML, 4 risk groups were identified based on age, cytogenetic risk, and disease status, including minimal residual disease status at transplantation. The 5-year leukemia-free survival for low (0 points), intermediate (2, 3, 5), high (7, 8), and very high (>8) risk groups was 78%, 53%, 40%, and 25%, respectively (P < .0001). For ALL, 3 risk groups were identified based on age and disease status, including minimal residual disease status at transplantation. The 5-year leukemia-free survival for low (0 points), intermediate (2-4), and high (≥5) risk groups was 68%, 51%, and 33%, respectively (P < .0001). We confirmed that the risk groups could be applied to overall survival, with 5-year survival ranging from 80% to 33% and 73% to 42% for AML and ALL, respectively (P < .0001). This validated pediatric DRI, which includes age and residual disease status, can be used to facilitate prognostication and stratification of children with AML and ALL for allogeneic transplantation. Key Points: • The pediatric DRI stratified children with AML and ALL into clinically distinct risk groups based on pretransplantation information. • Risk stratification was based on age at transplant, cytogenetics, and disease status including minimal residual disease at transplant.

Original languageEnglish
Pages (from-to)983-993
Number of pages11
JournalBlood
Volume137
Issue number7
DOIs
StatePublished - 18 Feb 2021

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