-
Categories
-
Pharmaceutical Intermediates
-
Active Pharmaceutical Ingredients
-
Food Additives
- Industrial Coatings
- Agrochemicals
- Dyes and Pigments
- Surfactant
- Flavors and Fragrances
- Chemical Reagents
- Catalyst and Auxiliary
- Natural Products
- Inorganic Chemistry
-
Organic Chemistry
-
Biochemical Engineering
- Analytical Chemistry
-
Cosmetic Ingredient
- Water Treatment Chemical
-
Pharmaceutical Intermediates
Promotion
ECHEMI Mall
Wholesale
Weekly Price
Exhibition
News
-
Trade Service
In recent years, the continuous advent of innovative drugs or therapies has increasingly met the diversified clinical treatment needs
of patients with hemophilia A.
However, individual patient variability, complexity in the management of comorbidities, and diversity of treatment strategies also pose many challenges
for clinicians.
Based on this, Beijing Hengji Health Management Development Foundation launched a tour on the frontier progress of hemophilia, in order to share the progress of hemophilia diagnosis and treatment from multiple angles and provide guidance
for the clinical practice of hemophilia in China.
The lake is bright and autumn, the wind is turbulent, and the journey of hemophilia treatment is still continuing
.
On September 19, 2022, the series of new advances in clinical diagnosis and treatment of hematological diseases was successfully held in Hemophilia Frontier Progress Tour (Beijing Station), and this conference specially invited Professor Zhao Yongqiang of Peking Union Medical College Hospital as the chairman of the conference, and invited Professor Wang Shujie of Peking Union Medical College Hospital and Professor Wu Runhui of Beijing Children's Hospital affiliated to Capital Medical University Share the wonderful content
related to hemophilia with Professor Chen Shu of the Second Affiliated Hospital of Chongqing Medical University.
Yimaitong has specially compiled the essence of the content for readers
.
Address to the conference
At the beginning of the meeting, Professor Zhao Yongqiang said that this conference was honored to invite famous experts in the field of hemophilia in China to sort out
the progress of hemophilia diagnosis and treatment.
The meeting was then declared open.
Figure 1 Professor Zhao Yongqiang delivered a wonderful speech
Professor Wang Shujie: Under the background of non-factor drug preventive treatment, the individualized plan helps hemophilia patients enjoy the improvement of life medical level and the
update of treatment concepts have a far-reaching impact on hemophilia treatment decisions.
Point 1: The primary goal of hemophilia management is bleeding control, preventive treatment is recognized as the standard treatmentThe relevant guidelines of the World Hemophilia Federation (WFH) have made it clear that preventive treatment is the standard treatment for hemophilia patients, and the primary goal is to control bleeding and eventually achieve no spontaneous bleeding
。 Patients who received early prophylaxis tended to show better long-term survival outcomes; For patients with severe hemophilia A or B, prophylaxis with coagulation factor concentrate (standard or extended half-life FVIII/FIX) or other hemostatic agents is recommended to prevent spontaneous and breakthrough bleeding, including haemarthrosis, which may lead to joint disease, as soon as possible before the onset of joint disease and, ideally, before the age of 3 years; In patients with pre-existing joint injuries who are not receiving prophylaxis, tertiary prevention is recommended to slow disease progression
.
Figure 2 Professor Wang Shujie shared the standard treatment model of hemophilia
Key point two: Even if the majority of Chinese hemophilia patients are treated prophylactically, bleeding AHEAD study is a prospective, non-interventional, multicenter clinical trial conducted in 22 countries, and a total of 141 patients
were included in the Chinese subgroup 。 The results showed that even after receiving preventive treatment, the overall proportion of patients with bleeding events was as high as 75.
8%.
Subgroup analysis in China showed that patients with hemophilia receiving prophylaxis had lower annualized bleeding rate (ABR) and annualized joint bleeding rate (AJBR) than those who received on-demand treatment alone (8.
2 vs 29.
4, 4.
1 vs 8.
6).
However, most patients have bleeding
despite prophylactic therapy.
Figure 3 Professor Wang Shujie shared the limitations of traditional coagulation factor therapy in hemophilia patients
Key point 3: More than 80% of patients treated with emelicilizumab can maintain zero bleeding, and target joint elimination of more than 98% coagulation factor replacement therapy benefits many hemophilia patients, but also has problems such as
poor bleeding control.
The novel non-factorial drug emizizumab has shown good efficacy
in this regard.
The results of the HAVEN 1-4 series of studies showed that since the 24th week of treatment, more than 80% of patients required treatment with 0 bleeding times and more than 97% of patients with ≤ 3 bleeding times required treatment at each treatment interval; Compared to the 530 target joints observed at baseline, the target joint elimination rate was as high as 98.
9%.
Figure 4 Professor Wang Shujie shared the good clinical efficacy of non-factor drugs
Professor Wang Shujie believes that although traditional coagulation factor drugs have improved the survival outcomes of hemophilia patients as a whole, it is of great significance
to explore innovative drugs such as emizelizumab to allow patients to achieve zero bleeding or even live like normal people in the context of increasing clinical needs of patients.
Professor Wu Runhui: Focusing on children, real-world research on hemophilia highlights the value of non-factor drug treatment Hemophilia as a hereditary rare disease, its impact on children has always been a topic of
clinical concern.
Professor Wu Runhui mainly shared the application
of emelizumab in children or adolescents with hemophilia based on the frontiers of real-world research.
Key point 1: Emmecilizumab significantly reduces bleeding frequency and improves musculoskeletal scores in children and adolescents A single-center, prospective study of Cairo University Pediatric Hospital in Egypt included 14 children with hemophilia A who received emelizumab prophylaxis, and the results showed that the bleeding rate of 11 (78.
6%) children decreased to 0 after 6 months of treatment (baseline bleeding frequency 6-8 times/year), and only 3 patients had at least one breakthrough bleeding, but none of them led to target joint development; The average musculoskeletal score decreased from 17 to 4
after treatment.
Fig.
5 Professor Wu Runhui shared the real-world research progress of emelizumab in children and adolescents with inhibitors
Key Point 2: Emelizumab can optimize the way hemophilia is administered and avoid the use of infusion portsA real-world study of Saarland University Children's Hospital in Germany included 14 children and adolescents with severe hemophilia A (including 3 PUPs), which aimed to explore the adherence of emelizumab in children with severe hemophilia A, the difficulty of dosing vial doses, and the need to
use coagulation factors in non-severe bleeding 。 The results showed that emelizumab used less additional coagulation factors during treatment, and almost no PUPs required additional use of coagulation factors.
Only a few patients
with additional clotting factors due to port removal, head trauma, and traumatic muscle bleeding have been reported.
Figure 6 Professor Wu Runhui shared a real-world study on reducing venous burden on children with hemophilia with emelizumab
Key point 3: Emelizuzumab in children with hemophilia can effectively reduce bleeding and has a good safety profileA real-world study of the Hannover Medical School in Germany included 5 infants and young children aged 3-26 months at the beginning of treatment, and the results showed that no spontaneous bleeding occurred during the prophylactic treatment of emelizumab, and all children were negative
for inhibitors.
Figure 7 Professor Wu Runhui shared the real-world study of emelizumab in the treatment of infants and young children with moderate to severe hemophilia
to hemophilia families.
Professor Chen Shu: Focus on the difficulties, the experience of surgery for hemophilia patients with inhibitors in the era of non-factor drug preventive treatment Sharing
that surgery for patients with hemophilia with inhibitors has long been a clinical problem, and many workers in the field of hemophilia have made unremitting explorations
on this.
Professor Chen Shu mainly shared
the experience of surgery in hemophilia patients under the background of non-factor drug prevention.
Key point 1: From the guideline point of view, in the context of non-factor preventive therapy, patients with hemophilia with inhibitors are recommended for surgery For hemophilia A with inhibitors who are being treated with emizelizumab and require major surgery or invasive surgery, WFH recommends additional supplementation with products containing FVIII for those with low inhibitor response; For inhibitor hyperresponsiveness, given the risk of thrombotic microangiopathy, WFH recommends rFVIIa as the first choice; Patients with hemophilia A with inhibitors who require minor or invasive surgery are recommended for low-dose or no clotting factor replacement
.
The 2021 version of the Chinese hemophilia management guidelines also made similar recommendations, for hemophilia A patients receiving emelizumab prophylaxis, if it is major surgery, patients with high titer are preferred rFVIIa, try not to use or use PCC with caution, and patients with low titer inhibitors can also be infusion of high-dose FVIII first; For minor surgery, low-dose FVIII replacement therapy or no replacement therapy
is recommended.
For the safety of emelizumab in combination with bypass preparations, which is of clinical concern, in the HAVEN 1, HAVEN 2 and HAVEN 4 studies, the simultaneous use of rFVIIa in hemophilia A with inhibitors was analyzed, and the results of the study found that There were no serious adverse events (SAEs), thrombotic microangiopathy, or thrombotic events
associated with rFVIIa plus emelizumab therapy.
Fig.
8 Professor Chen Shu shared guidelines for perioperative recommendations for patients with hemophilia with inhibitors
Key Point 2: The STASEY trial is a multicenter, single-arm, phase IIIb study that included 195 patients aged ≥12 years of age with hemophilia A with inhibitors from 85 centers around the world and enrolled 195 patients aged 12 years aged 12 years with hemophilia A with inhibitors, to evaluate the efficacy and safety of emelizumab prophylaxis, and to analyze the patient's undergone major and minor surgery
。 The results showed that 37 patients with hemophilia A inhibitors underwent 56 minor surgeries, and 57.
1% of them were performed without additional prophylactic drugs.
Most (18/22) major surgeries were performed with additional prophylactic medications, and although 66.
7% (12/18) of them experienced postoperative bleeding, only half were bleeding
requiring treatment.
This suggests that most patients with hemophilia A with inhibitors who are receiving emelizumab prophylaxis can safely undergo minor surgery without the need for additional prophylaxis; Major surgery
can be performed safely with additional preventive drugs.
HAVEN 1-4 SERIES DATA ANALYSIS ALSO FITS THIS CONCLUSION
.
Figure 9 Professor Chen Shu shares the results of STASEY research
Professor Chen Shu believes that the introduction of new drugs provides more choices for patients with hemophilia with inhibitors, and many authoritative research results have also accumulated valuable experience for the surgical management of patients with inhibitors.
Meeting discussion
The participating experts had a lively discussion on the above topics in combination with clinical practice, and the main points are as follows:
- The adequacy of preventive treatment directly affects the patient's bleeding situation, which in turn affects the patient's quality of life; Joint lesions are an indicator
of long-term treatment outcomes in patients with hemophilia. - As far as inhibitors are concerned, the principle should be "prevention is greater than cure"
.
When treated as needed, bypassing agents are effective hemostatic agents in patients with hemophilia with inhibitors; In preventive treatment, especially for pediatric patients, if conditions permit, the non-factor drug emmezizumab
should be preferred from the perspective of efficacy and adherence. The treatment of hemophilia should emphasize individualization, allow patients to fully participate in treatment decisions, and formulate the best treatment strategy
for patients based on factors such as patients' physical condition, family conditions, and tolerance level.
Since the approval of emizizumab, the indications have been increasing
.
In clinical practice, it has been found that the drug also has a certain effect on other types of hemophilia, and it is expected to be further explored in the future to benefit more patients
.
After wonderful sharing and heated discussions, the conference came to an end
.
Professor Zhao Yongqiang, chairman of the conference, said that the three speakers gave in-depth explanations on the clinical application, efficacy and safety of emelizumab, a non-factor drug in the new era, from different perspectives, providing valuable opinions for the treatment of hemophilia, and looking forward to meeting again in the future to discuss hemophilia-related topics
.
Editor: Valamor Review: Janet Typesetting: moly Execution: moly
Poke "Read Original" to see more conference topics, not to be missed
