Blood friends new life, not to live up to Shaohua I Chu sail · Starting again, the tour of the frontier progress of hemophilia (Tianjin Station) was successfully held

In recent years, the advent of innovative drugs or therapies has increasingly met the diverse clinical treatment needs

On September 15, 2022, the new progress series of clinical diagnosis and treatment of hemophilia (Tianjin Station) was successfully held, and Professor Yang Renchi of the Hematology Hospital of the Chinese Academy of Medical Sciences was invited to serve as the chairman of the conference, and invited Professor Xue Feng of the Hematology Hospital of the Chinese Academy of Medical Sciences, Professor Sun Lirong of the Affiliated Hospital of Qingdao University and Professor Zhou Zeping of the Second Affiliated Hospital of Kunming Medical University to share the wonderful content

Address to the Conference

At the beginning of the conference, Professor Yang Renchi, chairman of the conference, said that the conference had the honor of inviting three hemophilia experts to share relevant topics, and then announced the start

Figure 1 Professor Yang Renchi made a wonderful speech

Professor Xue Feng: There is a long way to go, and the disease burden of hemophilia patients in China is relatively heavy, and the clinical needs need to be met urgently

The World Hemophilia Alliance (WFH) Global Report 2020 shows that the incidence of hemophilia A is 24.

According to HTCCNC's data analysis of 166 hospitals in China from 2007 to 2019, the proportion of patients with severe hemophilia A in China is as high as 49.

Optimizing bleeding control is the key to the treatment of hemophilia, but patients and their families have a weak understanding of the disease, which requires patients such as hemophilia centers to actively educate patients to help patients identify bleeding types and conduct family therapy training

Figure 2 Professor Xue Feng introduced the overall situation of hemophilia

Bleeding is the most common symptom
of hemophilia.

Early treatment through whole blood or plasma solves the problem of survival to a certain extent, but long-term joint damage and even joint deformities caused by repeated bleeding will seriously cause patients to live
normally.

In addition, the pain caused by bleeding seriously affects the quality of life of patients, and studies have shown that more than 50% of patients feel pain every day or often, and recreational activities, walking, work, sleep, etc.
are affected
to varying degrees.

The bleeding burden of hemophilia patients in China is relatively heavy, especially the incidence of joint bleeding is still high
.

HTCCNC data showed that 61% of patients with hemophilia had joint bleeding and 36% had joint lesions; Another study shows that 71.
1% of patients in China only receive on-demand treatment, and 77.
9% of patients experience joint bleeding
.

Figure 3 Professor Xue Feng introduced the disease burden of hemophilia patients in China

Professor Xue Feng said that at present, coagulation factor is the standard alternative treatment drug for hemophilia, but there are problems such as poor bleeding control effect, poor quality of life of patients, poor venous burden and compliance, and inhibitor production, and there are still large clinical needs of hemophilia patients to be met
.

Professor Sun Lirong: Multi-dimensional verification, clinical trials combined with real-world research to prove the good efficacy of non-factorial drugs

The emergence of new therapies for hemophilia A has entered a period of rapid development, including non-factorial drugs, gene therapy, long-acting coagulation factors and other new drugs, which have greatly enriched the treatment options for
hemophilia.

Professor Sun Lirong mainly combined relevant core research and real-world data to elaborate on the frontier progress
of the non-factor drug Emmy Saizumab.

The results of long-term follow-up in the HAVEN 1-4 study showed a median effective follow-up of 120.
4 weeks, with an annualized bleeding rate (ABR) of only 1.
4 for treatment throughout the study period, and a bleeding rate of 82.
4%
at 121-144 weeks.

The HAVEN 5 study of Asian hemophilia patients showed a significant reduction in ABR after prophylaxis with Emmerizumab, a 96%
reduction compared to the no-prophylaxis group.

The results of the latest HAVEN 6 study show that the prophylaxis of Emilymab is consistently effective and safe in patients with mild to moderate inhibitor hemophilia A, with a consistently low bleeding rate during the study period, with a treated ABR of only 0.
9
.

Figure 4 Professor Sun Lirong shares the clinical research results of Emmy Cerivumab

Real-world data is a powerful complement to the conclusions of the RCT and can provide better treatment guidance for clinical practice and help optimize clinical decision-making
.

At present, a number of real-world studies of Emmerizumab are being conducted or reported, and data from France, the United Kingdom, Israel, Germany and other countries show that the use of Emilymab is good
with adult or pediatric hemophilia A patients with inhibitors.

Figure 5 Professor Sun Lirong shares the significance of real-world research

Professor Sun Lirong stressed that real-world data show that Emmy Saizumab can improve the coagulation function of children with hemophilia, and can achieve good and continuous bleeding control, which has brought good news
to many pediatric patients.

Professor Zhou Zeping: Acting according to the situation, Emmy Saizumab helps to enhance confidence in hemophilia surgery and comorbidity treatment

With the improvement of medical level, the clinical management of surgery and comorbidities in hemophilia A patients has gradually received clinical attention
.

Professor Zhou Zeping made a wonderful sharing
on this topic.

The UK National Hemohilia Database shows that with the increase in life expectancy, the number of patients with severe hemophilia A aged 60-79 years is increasing dramatically, there is an aging population, and the management of surgery and related comorbidities will increase accordingly; In terms of treatment, in 2018/2019, Emily salizumab was introduced for the preventive treatment of patients with and without inhibitor hemophilia A, and the number of patients using the drug gradually increased, while the number of patients receiving standard half-life coagulation factor therapy showed an overall downward trend
.

The results reflect the overall changes in patients with hemophilia A in the
UK.

Figure 6 Professor Zhou Zeping shares the latest database update of hemophilia

In terms of surgery, a number of clinical and real-world studies have shown that in patients receiving prophylactic treatment with Emmy Saizumab, minor surgery can be safely performed without additional coagulation factor supplementation; Major surgery
is safe when used in combination with other hemostatic agents.

In pooled analyses of HAVEN 1, 3, 4, and STASEY studies, in subgroups of people who received Emmerizumab prophylaxis > 50 years of age and had multiple CV risk factors, bleeding control and adverse events were similar to those receiving Emmerizumab prophylaxis, suggesting good
safety and efficacy in people over 50 years of age with cardiovascular risk.

The efficacy and safety of prophylaxis in the prophylaxis of Emily Selizumab are not affected by the common comorbidities in patients with hemophilia A in age or in the elderly, which is of great significance
for improving the confidence of hemophilia A comorbidities.

Figure 7 Professor Zhou Zeping shares the efficacy of Emmy celelimumab in patients with concomitant comorbidities

Professor Zhou Zeping believes that with the continuous improvement of China's hemophilia management system, the future will also face similar hemophilia comorbidities management problems in Europe and the United States, and solutions
to such problems should be actively explored in the context of the era of preventive treatment of the new drug Aimerizumab.

Meeting discussions

The participating experts had a heated discussion on hot topics such as sharing content and the needs of hemophilia patients, the main points of which are as follows:

• ABR and annualized joint bleeding rate (AJBR) are commonly used in clinical practice to evaluate the treatment of hemophilia, joint function score and imaging results are also very important, quality of life scoring indicators need to be developed according to the actual situation of each center
  .
  
  Indicators differ between pediatric and adult patients, and care needs to be taken to avoid traumatic bleeding and physiological bleeding at the same time; The evaluation of children's quality of life often involves more complex factors, and good doctor-patient communication and standardized medication are important
  .
  

• At present, the gap in the management level of hemophilia in different regions of China is still large, and the proportion of prevention and treatment in grass-roots areas is low, so it is necessary to face up to the gap and explore a solution model
  .
  
  

• If possible, Emmerizumab is ideal
  for preventive treatment in patients with hemophilia A with inhibitors.
  
  In clinical practice, it was found that the prophylaxis treatment of Emily citizumab with high-titer inhibitors had a good effect, and it is worth noting that the children benefited significantly, and the quality of life of children who had been using the drug for more than 2 years had significantly improved
  .
  

• Patients with previous untreated (PUP) hemophilia are mainly pediatric patients with greater difficulty using intravenous access, and if conditions permit, treatment with better compliance and good safety of Emily Saizumab is recommended
  .
  
  Clinical experience shows that children with PUP can achieve good results
  by receiving prophylactic treatment with emmy salizumab.
  

Summary of the meeting