Development and validation of a prognostic scoring model to risk stratify childhood acute myeloid leukaemia.

Content Provider	Europe PMC
Author	Li, Jun Liu, Lipeng Zhang, Ranran Wan, Yang Gong, Xiaowen Zhang, Li Yang, Wenyu Chen, Xiaojuan Zou, Yao Chen, Yumei Guo, Ye Ruan, Min Zhu, Xiaofan
Abstract	SummaryTo create a personal prognostic model and modify the risk stratification of paediatric acute myeloid leukaemia, we downloaded the clinical data of 597 patients from the Therapeutically Applicable Research to Generate Effective Treatments (TARGET) database as a training set and included 189 patients from our centre as a validation set. In the training set, age at diagnosis, ‐7/del(7q) or ‐5/del(5q), core binding factor fusion genes, FMS‐like tyrosine kinase 3‐internal tandem duplication (FLT3‐ITD)/nucleophosmin 1 (NPM1) status, Wilms tumour 1 (WT1) mutation, biallelic CCAAT enhancer binding protein alpha (CEBPA) mutation were strongly correlated with overall survival and included to construct the model. The prognostic model demonstrated excellent discriminative ability with the Harrell's concordance index of 0.68, 3‐ and 5‐year area under the receiver operating characteristic curve of 0.71 and 0.72 respectively. The model was validated in the validation set and outperformed existing prognostic systems. Additionally, patients were stratified into three risk groups (low, intermediate and high risk) with significantly distinct prognosis, and the model successfully identified candidates for haematopoietic stem cell transplantation. The newly developed prognostic model showed robust ability and utility in survival prediction and risk stratification, which could be helpful in modifying treatment selection in clinical routine.
Related Links	https://europepmc.org/backend/ptpmcrender.fcgi?accid=PMC9543487&blobtype=pdf
Page Count	10
ISSN	00071048
Journal	British Journal of Haematology [Br J Haematol]
Volume Number	198
DOI	10.1111/bjh.18354
PubMed Central reference number	PMC9543487
Issue Number	6
PubMed reference number	35880261
e-ISSN	13652141
Language	English
Publisher	John Wiley and Sons Inc.
Publisher Date	2022-07-25
Publisher Place	Hoboken
Access Restriction	Open
Rights License	This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes. © 2022 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.
Subject Keyword	acute leukaemia myeloid leukaemia paediatric haematology prognostic factors
Content Type	Text
Resource Type	Article
Subject	Hematology