Faculty Collaboration Database

A validated pediatric disease risk index for allogeneic hematopoietic cell transplantation. Blood 2021 Feb 18;137(7):983-993

Date

11/19/2020

Pubmed ID

33206937

Pubmed Central ID

PMC7918183

DOI

10.1182/blood.2020009342

Scopus ID

2-s2.0-85101021587 (requires institutional sign-in at Scopus site)   21 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.

Author List

Qayed M, Ahn KW, Kitko CL, Johnson MH, Shah NN, Dvorak C, Mellgren K, Friend BD, Verneris MR, Leung W, Toporski J, Levine J, Chewning J, Wayne A, Kapoor U, Triplett B, Schultz KR, Yanik GA, Eapen M

Authors

Kwang Woo Ahn PhD Professor in the Institute for Health and Equity department at Medical College of Wisconsin
Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adolescent
Age Factors
Allografts
Antineoplastic Combined Chemotherapy Protocols
Child
Child, Preschool
Cohort Studies
Combined Modality Therapy
Disease-Free Survival
Female
Hematopoietic Stem Cell Transplantation
Humans
Infant
Kaplan-Meier Estimate
Leukemia, Myeloid, Acute
Male
Neoplasm, Residual
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Prognosis
Random Allocation
Risk Assessment
Risk Factors
Severity of Illness Index