【Conference Summary】2022 International Conference on Thrombosis and Hemostasis (ISTH) Cutting-edge Information Sharing

The International Congress on Thrombosis and Hemostasis (ISTH) is a top academic event in the field of international coagulation, and will be held in London, England in 2022 in the form of a combination of offline and
online.
In order to convey the academic progress in the first time, Novo Nordisk Medical Department organized the 2022 ISTH Frontier Information Sharing Meeting from August 22 to 25, 2022
.
This conference invited many experts in the field of hemophilia to come together to interpret hot topics and pay attention to Chinese voices
.

The first session is to see the victory of tomorrow over the present - the progress of the diagnosis and treatment of ISTH hemophilia

Professor Sun Jing of Southern Hospital of Southern Medical University reviewed the progress
related to hemophilia at the ISTH Conference from both the aspects of diagnosis and treatment.
In terms of diagnosis, "joint ultrasound + artificial intelligence" has carried out a number of preliminary explorations to help determine joint bleeding
.
In terms of treatment, prolonged half-life coagulation factors and bispecific antibodies will give patients more treatment options
.
Finally, Professor Sun also shared "Vigilance Gap: A Journey to the Treatment of Hemophilia", which comprehensively depicts the beautiful vision
of hemophilia management in the future from the dimensions of individualized treatment, sports and quality of life.

Professor Wang Xuefeng of Ruijin Hospital, affiliated to Shanghai Jiao Tong University School of Medicine, concluded that although he was unable to participate in the ISTH conference offline, the form of the domestic post-conference meeting helped domestic doctors to learn about international progress
in the first time.
The standardized application of ultrasound contributes to the joint health management of hemophilia patients, and advances in equipment and technology make telemedicine possible; The accelerated development of new drugs in China will also open a new chapter
in the management of hemophilia.

The second session is to keep the clouds open to see the moon - progress in the field of pediatrics of hemophilia

Professor Wu Runhui of Beijing Children's Hospital affiliated to Capital Medical University shared a number of academic achievements around "Exploration of Treatment Strategies for Children with Hemophilia in China", including 9 abstracts, 1 oral communication and RPTH Editing Award
.
Dr.
Huang Kun represented the team in an oral exchange of personalized preventive treatment
under the guidance of a 2.
5-year PK at ISTH.
With the optimization of preventive treatment programs, the annualized bleeding rate gradually decreased in each follow-up period, and the proportion of patients with zero bleeding was as high as 69%.

The RPTH Editor's Award of the Year was awarded to Dr.
Zekun Li of Beijing Children's Hospital in recognition of the team's work
on the immunotolerance induction program.

Professor Li Xiaojing of Chengdu New Century Women and Children's Hospital concluded that Professor Wu Runhui's team has done a number of pioneering research in the individualized preventive treatment and immunotolerance induction therapy for children with hemophilia in China, which has been recognized
by the international academic community.
Pediatricians in the field of hemophilia in China can carry out more collaborative research in the future to benefit children
.

Session 3 Dialing the Clouds to See the End of the Day Sometimes – Advances in Gene Therapy for Hemophilia

Professor Xue Feng of the Blood Disease Hospital of the Chinese Academy of Medical Sciences talked about the principle of gene therapy and the characteristics of AAV carriers, and reviewed the history of
hemophilia gene therapy in detail.
In 1999, the first case of intramuscular AAV injection for hemophilia patients did not achieve the desired therapeutic effect, but it began the exploration process
of this treatment.
By 2022, the first Phase 3 clinical trial of hemophilia B gene therapy was announced at EAHAD conferences with an average FIX: C of 36.
9±21.
4% (follow-up of 1.
5 years); the first Phase 3 clinical trial of hemophilia A gene therapy was published in the New England Journal of Medicine (NEJM) with an average FVIII:C of 24.
4± 29.
2% (2 years of follow-up).

The exploration of gene therapy for hemophilia in China is also in the ascendant
.
Professor Xue shared the exploration of hemophilia B gene therapy recently published in The Lancet Hematology, and after a median follow-up of 58 weeks, the patient's carrier-derived FIX: C was 36.
93± 20.
49 IU/dL
.

Professor Wu Jingsheng of the First Affiliated Hospital of the University of Science and Technology of China concluded that the results of the exploration of hemophilia B gene therapy in China are exciting, and both the level of coagulation factors and the number of bleeding have been greatly improved
.
China's hemophilia gene therapy started early, and now it is catching up and is at the forefront of
the world.
It is hoped that the research and development of various new therapies for hemophilia in China will be accelerated to benefit patients
.

In the fourth scene, the man was in the dark light—concerned about the woman's bleeding disease

Professor Huang Meijuan of Union Hospital Affiliated to Fujian Medical University elaborated on the common symptoms and effects of bleeding diseases in congenital women, and reported the results of international research: the vast majority of women gain knowledge about menstruation through their mothers, lack of understanding of PBAC scales, etc.
, and find it difficult to identify menorrhagia, which delays diagnosis and treatment
.
Professor Huang focused on 3 patient groups:

➤ Female hemophilia: ISTH recommends a new nomenclature, "female carrier" does not mean "asymptomatic"

➤ Pregnancy-related acquired hemophilia: preferred rFVIIa hemostasis, hormone monotherapy clearance inhibitors

➤ Hereditary FVII deficiency in women: menorrhagia and postpartum hemorrhage are common, and delivery management should be individualized

Professor Yang Renchi of the Blood Disease Hospital of the Chinese Academy of Medical Sciences concluded that patients with congenital female bleeding diseases also need the attention
of the academic community and society.
In the case of female hemophilia carriers, diagnosis, registration, and management
of such patients should be strengthened based on baseline FVIII/FIX and personal bleeding history.

summary

Through this conference, domestic experts timely conveyed the international academic progress in the field of hemophilia and interpreted hot academic topics
.
It is believed that with the joint efforts of all walks of life, the management of hemophilia will surely "make a new leap forward"
.