A Validated Risk Stratification That Incorporates MAGIC Biomarkers Predicts Long-Term Outcomes in Pediatric Patients with Acute GVHD
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A Validated Risk Stratification That Incorporates MAGIC Biomarkers Predicts Long-Term Outcomes in Pediatric Patients with Acute GVHD. / Qayed, Muna; Kapoor, Urvi; Gillespie, Scott; Westbrook, Adrianna; Aguayo-Hiraldo, Paibel; Ayuk, Francis A; Aziz, Mina; Baez, Janna; Choe, Hannah; DeFilipp, Zachariah; Etra, Aaron; Grupp, Stephan A; Hexner, Elizabeth; Holler, Ernst; Hogan, William J; Kowalyk, Steven; Merli, Pietro; Morales, George; Nakamura, Ryotaro; Pulsipher, Michael A; Schechter, Tal; Shah, Jay; Spyrou, Nikolaos; Srinagesh, Hrishikesh K; Wölfl, Matthias; Yanik, Gregory; Young, Rachel; Kitko, Carrie L; Ferrara, James L M; Levine, John E.
in: TRANSPL CELL THER, Jahrgang 30, Nr. 6, 06.2024, S. 603.e1-603.e11.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - A Validated Risk Stratification That Incorporates MAGIC Biomarkers Predicts Long-Term Outcomes in Pediatric Patients with Acute GVHD
AU - Qayed, Muna
AU - Kapoor, Urvi
AU - Gillespie, Scott
AU - Westbrook, Adrianna
AU - Aguayo-Hiraldo, Paibel
AU - Ayuk, Francis A
AU - Aziz, Mina
AU - Baez, Janna
AU - Choe, Hannah
AU - DeFilipp, Zachariah
AU - Etra, Aaron
AU - Grupp, Stephan A
AU - Hexner, Elizabeth
AU - Holler, Ernst
AU - Hogan, William J
AU - Kowalyk, Steven
AU - Merli, Pietro
AU - Morales, George
AU - Nakamura, Ryotaro
AU - Pulsipher, Michael A
AU - Schechter, Tal
AU - Shah, Jay
AU - Spyrou, Nikolaos
AU - Srinagesh, Hrishikesh K
AU - Wölfl, Matthias
AU - Yanik, Gregory
AU - Young, Rachel
AU - Kitko, Carrie L
AU - Ferrara, James L M
AU - Levine, John E
N1 - Copyright © 2024 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.
PY - 2024/6
Y1 - 2024/6
N2 - Acute graft versus host disease (GVHD) is a common and serious complication of allogeneic hematopoietic cell transplantation (HCT) in children but overall clinical grade at onset only modestly predicts response to treatment and survival outcomes. Two tools to assess risk at initiation of treatment were recently developed. The Minnesota risk system stratifies children for risk of nonrelapse mortality (NRM) according to the pattern of GVHD target organ severity. The Mount Sinai Acute GVHD International Consortium (MAGIC) algorithm of 2 serum biomarkers (ST2 and REG3α) predicts NRM in adult patients but has not been validated in a pediatric population. We aimed to develop and validate a system that stratifies children at the onset of GVHD for risk of 6-month NRM. We determined the MAGIC algorithm probabilities (MAPs) and Minnesota risk for a multicenter cohort of 315 pediatric patients who developed GVHD requiring treatment with systemic corticosteroids. MAPs created 3 risk groups with distinct outcomes at the start of treatment and were more accurate than Minnesota risk stratification for prediction of NRM (area under the receiver operating curve (AUC), .79 versus .62, P = .001). A novel model that combined Minnesota risk and biomarker scores created from a training cohort was more accurate than either biomarkers or clinical systems in a validation cohort (AUC .87) and stratified patients into 2 groups with highly different 6-month NRM (5% versus 38%, P < .001). In summary, we validated the MAP as a prognostic biomarker in pediatric patients with GVHD, and a novel risk stratification that combines Minnesota risk and biomarker risk performed best. Biomarker-based risk stratification can be used in clinical trials to develop more tailored approaches for children who require treatment for GVHD.
AB - Acute graft versus host disease (GVHD) is a common and serious complication of allogeneic hematopoietic cell transplantation (HCT) in children but overall clinical grade at onset only modestly predicts response to treatment and survival outcomes. Two tools to assess risk at initiation of treatment were recently developed. The Minnesota risk system stratifies children for risk of nonrelapse mortality (NRM) according to the pattern of GVHD target organ severity. The Mount Sinai Acute GVHD International Consortium (MAGIC) algorithm of 2 serum biomarkers (ST2 and REG3α) predicts NRM in adult patients but has not been validated in a pediatric population. We aimed to develop and validate a system that stratifies children at the onset of GVHD for risk of 6-month NRM. We determined the MAGIC algorithm probabilities (MAPs) and Minnesota risk for a multicenter cohort of 315 pediatric patients who developed GVHD requiring treatment with systemic corticosteroids. MAPs created 3 risk groups with distinct outcomes at the start of treatment and were more accurate than Minnesota risk stratification for prediction of NRM (area under the receiver operating curve (AUC), .79 versus .62, P = .001). A novel model that combined Minnesota risk and biomarker scores created from a training cohort was more accurate than either biomarkers or clinical systems in a validation cohort (AUC .87) and stratified patients into 2 groups with highly different 6-month NRM (5% versus 38%, P < .001). In summary, we validated the MAP as a prognostic biomarker in pediatric patients with GVHD, and a novel risk stratification that combines Minnesota risk and biomarker risk performed best. Biomarker-based risk stratification can be used in clinical trials to develop more tailored approaches for children who require treatment for GVHD.
KW - Humans
KW - Graft vs Host Disease/blood
KW - Child
KW - Biomarkers/blood
KW - Female
KW - Male
KW - Hematopoietic Stem Cell Transplantation/adverse effects
KW - Child, Preschool
KW - Adolescent
KW - Pancreatitis-Associated Proteins/blood
KW - Acute Disease
KW - Risk Assessment
KW - Infant
KW - Interleukin-1 Receptor-Like 1 Protein/blood
KW - Algorithms
KW - Transplantation, Homologous/adverse effects
KW - Treatment Outcome
U2 - 10.1016/j.jtct.2024.03.022
DO - 10.1016/j.jtct.2024.03.022
M3 - SCORING: Journal article
C2 - 38548227
VL - 30
SP - 603.e1-603.e11
JO - TRANSPL CELL THER
JF - TRANSPL CELL THER
SN - 2666-6375
IS - 6
ER -