Machine learning to forecast rituximab responses for paediatric immune thrombocytopenia: Forging a path towards personalized medical care.

Journal: British journal of haematology
Published Date: Jul 15, 2025

Abstract

Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterized by decreased platelet counts and increased bleeding risk. Although paediatric ITP often resolves spontaneously, some children do not respond to first-line treatments, thus requiring rituximab as a second-line therapy to reduce bleeding risks and corticosteroid exposure. Currently, there is no reliable method to predict the efficacy of rituximab. Our study aimed to develop a machine learning (ML) model to predict the initial response to rituximab in these patients. We analysed data from 156 paediatric ITP patients treated at Beijing Children's Hospital between 2020 and 2023 and identified 25 key predictive features. Among the four evaluated ML models, the multilayer perceptron model exhibited the highest predictive accuracy. SHapley Additive exPlanations analysis revealed that antinuclear antibody titre, thyroglobulin antibody, corticosteroid response and bleeding severity were significant positive predictors, while thyroid peroxidase antibody, CD3 CD4 IL-17 T cells and the duration of disease before rituximab treatment were negatively associated with treatment responses. This ML model could be used to predict rituximab responses in paediatric ITP, which is expected to optimize treatment strategies and improve patient outcomes.

Authors

  • Jingyao Ma
    Hematology Department, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.
  • Chang Cui
    Internal Medicine-Cardiovascular Department, The First Affiliated Hospital of Nanjing Medical University, Nanjing, China.
  • Juntao Ouyang
    Hematology Department, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.
  • Zheyan Lin
    Hematology Department, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.
  • Xi Lin
    Department of Ultrasound, Sun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, 651 Dongfeng East Road, Guangzhou 510060, China. Electronic address: linxi@sysucc.org.cn.
  • Yu Hu
    Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
  • Zhifa Wang
    Hematology Department, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.
  • Shuyue Dong
    Hematology Department, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.
  • Jinxi Meng
    Hematology Department, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.
  • Wensheng Zhang
    Department of Anesthesiology, West China Hospital, Sichuan University, Chengdu, China.
  • Xiaoling Cheng
    Department of Pharmacy, Beijing Children's Hospital, Capital Medical University, Beijing, China.
  • Zhenping Chen
    Department of Clinical Laboratory Center; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
  • Yongqiang Tang
  • Runhui Wu
    Hematology Department, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.

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