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    Home > Active Ingredient News > Blood System > Based on the present, looking forward to the future, the big coffee talks about the major progress in the field of hemophilia

    Based on the present, looking forward to the future, the big coffee talks about the major progress in the field of hemophilia

    • Last Update: 2022-02-23
    • Source: Internet
    • Author: User
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    Hemophilia is a group of rare X-linked recessive hemorrhagic diseases, divided into hemophilia A (FVIII deficiency) and hemophilia B (FIX deficiency).
    Completely cured, damage the patient's quality of life
    .

    In recent years, with the continuous improvement of technology and system, some clinical progress has been made in the prevention and treatment of hemophilia, but there are still unmet clinical needs
    .

    The 63rd American Hematology Annual Meeting (ASH) in 2021 has been successfully held online and offline from December 11 to 14, focusing on the frontier research of blood diseases and the improvement of the level of disease diagnosis and treatment
    .

    In order to further improve the level of diagnosis and treatment of hemophilia, the 2022 Blood Pioneer Forum - Post ASH was successfully held on January 15, 2022.
    The conference was jointly chaired by Professor Yang Renchi of Hematology Hospital of Chinese Academy of Medical Sciences and Professor Zhao Yongqiang of Peking Union Medical College Hospital.
    , many big coffees summed up their experience, looked forward to the future, and discussed issues such as hemophilia management, prevention, inhibitor therapy, and real-world research
    .

    The essence of this conference is as follows, for readers
    .

    At the beginning of the meeting, Prof.
    Yang Renchi and Prof.
    Zhao Yongqiang made opening speeches, emphasizing that the conference focused on the clinical management and problems of hemophilia, which is of great significance to the diagnosis and treatment of hemophilia in China, and announced the official start of the conference
    .

    Imagining the new thinking of hemophilia management in the new era in the future, Professor Wang Shujie from Peking Union Medical College Hospital first shared his thoughts on the management of hemophilia patients in the new era at this conference
    .

    Professor Wang evaluated the prevalence of hemophilia in the world from an epidemiological perspective, and showed the audience the physical and psychological distress caused by bleeding to the lives of hemophilia patients from the perspective of patients.

    .

    After that, Professor Wang systematically introduced the development history of hemophilia drugs in detail, from blood-derived products-recombinant products-extended half-life FⅧ-emerizumab, with the advancement of technology, the bleeding control of patients with hemophilia A has been improved.
    Great improvement
    .

    Bleeding control is the primary goal of hemophilia management, and prophylaxis is the established standard of care compared to on-demand therapy
    .

    However, many hemophilia A patients receiving FVIII prophylaxis still experience breakthrough bleeding, and the inhibitor patients have significant bleeding.
    Compared with the general population, the quality of life of hemophilia A patients is still significantly different
    .

    The emergence of emilacizumab has effectively improved this situation.
    The results of the HAVEN1-6 clinical study on emilacizumab confirmed that whether it is severe or mild to moderate, with or without inhibitors, emilacizumab Antibodies showed sustained and good bleeding control, and continued to improve joint health, quality of life, study, work, and mobility in patients with hemophilia A, and greatly reduced the number of injections
    .

    In the end, Professor Wang shared the story of the first hemophiliac who reached the top of Mount Everest in 2017, and imagined a free and bright future for hemophiliacs
    .

    It is very important to improve the hemophilia registration system.
    Professor Xue Feng from the Hospital of Hematology, Chinese Academy of Medical Sciences introduced the Chinese hemophilia registration system and emphasized the important role of the system in the overall prevention and management of hemophilia
    .

    Professor Xue first introduced the development history of the domestic hemophilia diagnosis and treatment system and the establishment of the China Hemophilia Collaborative Group (HTCCNC).
    necessary
    .

    Afterwards, Professor Xue introduced the system in detail from the aspects of definition, function and type
    .

    The Chinese hemophilia registration system has experienced paper version, web version and national policy support.
    In 2009, the Ministry of Health officially issued a notice on the establishment of a hemophilia case information management system, which further improved and clarified the system.

    .

    Prof.
    Xue systematically and in detail introduced the operation of the electronic registration system, including key operations such as entering personal information, clinical features, joint status, and virology, and emphasized that the system has statistical functions and is of great significance to the management of hemophilia in China
    .

    Keeping up with the hotspot sharing of the treatment progress of patients with cutting-edge inhibitors, Professor Wu Runhui from Beijing Children's Hospital Affiliated to Capital Medical University explained and shared the treatment needs and progress of patients with hemophilia A inhibitors
    .

    Professor Wu first systematically explained the formation and disease characteristics of inhibitors from the perspectives of mechanism, classification, and epidemiological characteristics, and then explained the clinical needs of patients with inhibitors.
    Identify hazards and systematically explain the challenges of current inhibitor therapy
    .

    Finally, Prof.
    Wu introduced in detail the progress made by emeclizumab in the treatment of patients with inhibitors from the aspects of efficacy, perioperative management, combined ITI therapy and combined rFVIIa administration: Bleeding of joints in patients with severe hemophilia A with inhibitors; most patients with hemophilia A and inhibitors who are receiving prophylaxis with emecizumab can safely undergo some minor surgery without additional prophylaxis; Mecilizumab-treated patients with FVIII inhibitors can safely perform major surgery with additional prophylactic drugs; emeclizumab may improve the success rate of ITI by effectively controlling bleeding; HAVEN1, 2 A comprehensive analysis of and 4 clinical studies showed that the concomitant use of emeclizumab and rFVIIa was safe and effective
    .

    No adverse events occurred after administration according to the bypass preparation management guidelines
    .

    Nursing "Flowers" strives to create a warm haven for children with hemophilia.
    Professor Xu Weiqun from the Children's Hospital Affiliated to Zhejiang University School of Medicine shared the progress of prevention and treatment of children with hemophilia and real-world research
    .

    Professor Xu stressed that hemophilia may bring great distress to children and seriously affect the quality of life in the future.
    Children with hemophilia should start preventive treatment as soon as possible
    .

    At the same time, the preventive treatment of children also has problems such as invasive treatment (venipuncture), high frequency of infusion, and generation of inhibitors
    .

    Then, Prof.
    Xu introduced in detail the research progress of non-factor drugs, including Emicizumab, Fitusiran, gene drugs, etc.
    , and explained the mechanism of action of various drugs
    .

    Prof.
    Xu focused on the core study data of HAVEN 2 with respect to emeclizumab, saying that compared with previous bypass drug treatment, prophylactic treatment of emeclizumab reduced by 99% (95% CI, 97.
    7–99.
    4) bleeding risk
    .

    Based on the excellent efficacy of the drug, some authoritative guidelines currently believe that emeclizumab can be used for the prophylactic treatment of hemophilia in newborns and infants, but more prospective studies are needed to support it
    .

    Finally, Prof.
    Xu systematically shared the application data of Emicizumab in the real world
    .

    Results of a multicenter, observational study showed a reduction in bleeding events in all patients receiving emilacizumab, good bleeding outcomes in children <12 years without inhibitors, and good surgical outcomes while receiving emilacizumab , and the security is good
    .

    Prof.
    Xu focused on the real-world study of emeclizumab in the prevention and treatment of patients with hemophilia A - the Milan single-center experience.
    The results of the study confirmed that emacizumab has good efficacy and is more effective in children.
    is obvious; monitoring of emilacizumab blood levels can help identify patients who develop ADA
    .

    At the end of the conference summary meeting, Prof.
    Yang Renchi and Prof.
    Zhao Yongqiang affirmed and re-emphasized the importance of this conference, and said that the emergence of non-factor innovative drugs will play an important role in the prevention and treatment of hemophilia
    .

    At the same time, he also pointed out that the current hemophilia treatment has problems such as high treatment cost and poor compliance.
    It is hoped that clinicians, pharmaceutical companies and government departments across the country will work together to incorporate these innovative drugs into the medical insurance system as soon as possible to benefit Chinese hemophiliacs
    .

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