2022CSH Prof. Yu Hu: Review and future prospects of hemophilia in the past 40 years

Hemohilia is an X-linked, recessive bleeding disorder caused by a deficiency of factor VIII or factor IX, which is related
to the corresponding coagulation factor gene mutation.
In recent years, new drugs such as recombinant human coagulation factor VIII and new therapies such as gene therapy have continued to emerge, which has greatly improved
the quality of life and survival of hemophilia patients.
On September 23-25, 2022, the 17th National Hematology Academic Conference of the Chinese Medical Association was grandly opened
at the National Exhibition and Convention Center in Shanghai.
The conference was hosted by the Chinese Medical Association and the Hematology Branch of the Chinese Medical Association, hosted by the Shanghai Medical Association, co-organized by Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, the First Affiliated Hospital of Soochow University, and the Union Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology, with the theme of "respect, inheritance, collaboration and innovation", specially invited famous experts at home and abroad to talk about the latest progress
in the field of blood diseases.
On this occasion, Yimai Tong sincerely invites Professor Hu Yu of Union Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology to review the development process of hemophilia in the past 40 years, share the current diagnosis and treatment status, and make future prospects for the readers!

Hemophilia is a hereditary hemorrhagic rare disease, patients are often referred to as "glass people", "porcelain dolls", etc.
, and the progress in this field has attracted everyone's attention
.
Could you please talk about the progress made in this regard in light of the development of hemophilia?

In recent decades, China has made great progress
in the prevention and treatment of hemophilia.
As early as 1985, the Tianjin Institute of Hematology of the Chinese Academy of Medical Sciences led the establishment of the National Hemofriendly Disease Collaborative Group, and successfully joined the World Hemohilia Alliance in 1990; Nowadays, hemophilia centers have spread all over the country, and Professor Yang Renchi has led the qualification certification of hemophilia centers, and since then, the diagnosis and treatment of hemophilia has been standardized and standardized
.

At the same time, the treatment concept of hemophilia is constantly being updated
.
In the past, blood products in China were relatively scarce, and only plasma coagulation factor VIII substitutes could be used, with poor efficacy, and patients would only use coagulation factor transfusion therapy when bleeding was severe; With the recombinant human coagulation factor VIII product, the clinical efficacy of hemophilia patients has been greatly improved, and by carrying out one of the current standard therapies - preventive treatment, that is, quantitative infusion of coagulation factor VIII before the patient's bleeding, and maintaining a stable blood concentration, can avoid severe bleeding and secondary joint muscle damage in patients, therefore, patients can grow healthily and carry out normal life work, which brings an overall improvement
to the treatment of hemophilia patients 。 Nowadays, China's medical insurance policy is getting better and better, coagulation factor VIII preparations have been strongly supported by the medical insurance bureau, and the economic burden of patients has been significantly reduced
.

All in all, in recent years, China's hemophilia prevention and treatment has made great progress, and has made rapid
progress in the standardization of the center, the establishment of the system and medical insurance.

Medical Pulse Pass: After years of development, the field of hemophilia treatment in China has undergone tremendous changes, as a blood person who has been deeply engaged in the blood field for many years, can you share your feelings about this?

At present, the concept of treatment of hemophilia has undergone a fundamental change
.
In the past, it was treated on demand, and now recombinant human coagulation factor VIII can be used for preventive treatment, and the recombinant human coagulation factor VIII preparation derived from human cell lines in China has also been put on the market, providing more treatment options
for hemophilia patients.
In addition, the treatment mode of hemophilia patients has also undergone significant changes, and the emergence of a new generation of recombinant human coagulation factor VIII has gradually made hemophilia gradually tailor-made and individualized treatment
.

Medical Pulse Communication: This national academic conference on hematology can be successfully held in a special period to provide an opportunity for the further development of China's blood field, can you please look forward to the development prospects of hemophilia in China?

In the past few decades, hemophilia in our country has made great progress; In the future, we may have new development opportunities in terms of medical insurance policies for hemophilia and the advanced nature of factor preparations, but a more important development trend is gene therapy
for hemophilia.
Nowadays, the gene therapy of hemophilia has been included in the scope of clinical research, in the near future, there will be a new type of treatment based on gene therapy, which is significantly different from the transfusion of coagulation factors, through the implantation of normal genes, can fundamentally solve the problem of insufficient coagulation factors in the body, so that the patient's coagulation factor VIII and so on to achieve normal levels, so as to achieve disease cure
.
These very promising and promising treatments will bring greater blessings
to hemophilia patients.

Medical Pulse Pass: A number of academicians and guests attended the opening ceremony of the Hematology Branch of the Chinese Medical Association, and also set up an honorary tribute moment for the older generation of experts, which reflects the respect and inheritance
of our blood scholars.
What are your feelings about this conference to share with us?

Under the requirements of the current epidemic prevention and control, this meeting can be held in spite of many difficulties, and it reflects a very high standard in terms of conference scale and organizational form, which can be described as a very successful conference
.
First of all, this conference has a distinct theme - respect, inheritance, collaboration, innovation, which is also reflected in the organization of the conference, including yesterday's exhibition on the history of the development of hematology, the documentary broadcast in a loop, the honor tribute ceremony at the conference site, etc.
, all of which express our respect for the achievements of previous generations of hematology scientists and their admiration
。 Secondly, this conference aims at the forefront of the field, the big coffee gathered, only the morning opening ceremony gathered a number of academicians, from the characteristics of elderly tumors to leukemia big data collection, from immune and cell therapy to the listing of new drugs, the expositions of these big coffee have opened up our clinical and scientific research ideas, but also promoted the integration between multiple disciplines, is a good trend
。 In addition, this conference set up a number of special sessions, such as innovation forum, slow grain special session, coagulation special session, severe infection special session, etc.
, which not only strengthens the integration between basic and clinical disciplines, but also promotes the answer to the hot and difficult questions in the field, I hope that everyone can gain something in this conference and grow!

Professor Hu Yu

• Member of the 13th National Committee of the Chinese People's Political Consultative Conference

• President of Union Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology

• Director of the Institute of Hematology, Huazhong University of Science and Technology

• Winner of the National Science Foundation for Outstanding Youth

• Distinguished Professor of "Yangtze River Scholars"

• National Model Winner of Teaching and Education

• Winner of the second prize of the National Science and Technology Progress Award

• Winner of the National Innovation Scramble Medal

• Chairman-designate of the Hematology Branch of the Chinese Medical Association (Leader of the Thrombosis and Hemostasis Group)

• Member of the Standing Committee of the Internal Medicine Branch of the Chinese Medical Association

• Vice President of the Hematology Branch of the Chinese Medical Doctor Association

• Vice Chairman of the Experimental Hematology Society of the Chinese Society of Pathophysiology

• Member of the Education Committee of the International Society for Thrombosis and Hemostasis

• Chairman of the Hematology Branch of Hubei Medical Association

• Associate Editor, Chinese Journal of Hematology/Journal of Clinical Hematology

• Associate Editor, Thrombosis Research/Thrombosis and Haemostasis

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