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Letter to the Editors-in-Chief| Volume 224, P60-64, April 2023

The efficacy of sequential MMF-rescue-regimen to eradicate inhibitors for refractory severe hemophilia A inhibitor children in China

  • Zhengping Li
    Affiliations
    Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China

    Hematologic Disease Laboratory, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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  • Zekun Li
    Affiliations
    Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China

    Hematologic Disease Laboratory, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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  • Jie Sun
    Affiliations
    Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China

    Hematologic Disease Laboratory, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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  • Xiaoling Cheng
    Affiliations
    Department of Pharmacy, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China
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  • Guoqing Liu
    Affiliations
    Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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  • Wanru Yao
    Affiliations
    Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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  • Kun Huang
    Affiliations
    Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China

    Hematologic Disease Laboratory, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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  • Di Ai
    Affiliations
    Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China

    Hematologic Disease Laboratory, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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  • Zhenping Chen
    Affiliations
    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Hematologic Disease Laboratory, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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  • Gang Li
    Affiliations
    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Hematologic Disease Laboratory, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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  • Yingzi Zhen
    Affiliations
    Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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  • Man-Chiu Poon
    Correspondence
    Correspondence to: M-C. Poon, University of Calgary, Foothills Medical Centre, 1403-29th Street NW, Calgary, Alberta T2N 2T9, Canada.
    Affiliations
    Department of Medicine, University of Calgary Cumming School of Medicine, Calgary, Alberta, Canada

    Department of Pediatrics, University of Calgary Cumming School of Medicine, Calgary, Alberta, Canada

    Department of Oncology, University of Calgary Cumming School of Medicine, Calgary, Alberta, Canada
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  • Runhui Wu
    Correspondence
    Correspondence to: R. Wu, Hemophilia Comprehensive Care Center, Hematology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, No.56 Nanlishi Road, West District, Beijing 100045, China.
    Affiliations
    Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, 100045, China

    National Key Discipline of Pediatrics (Capital Medical University), 100045, China

    Key Laboratory of Major Diseases in Children, Ministry of Education, 100045, China

    Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, 100045, China
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Published:February 23, 2023DOI:https://doi.org/10.1016/j.thromres.2023.02.011

      Highlights

      • Refractoriness was inability to achieve negative inhibitor titer over at least 12 months period taking LD-ITI+Rituximab-IS.
      • There is therefore a need to explore rescue-regimen(s) for refractory patients.
      • MMF might exert T-cell immunosuppression not covered by Rituximab which focuses on B-cell suppression.
      • The sequential MMF-rescue-regimen had efficacy in the patients refractory to our initial LD-ITI+Rituximab-IS regimen.

      Keywords

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