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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.
On October 12, 2022, the series of new advances in clinical diagnosis and treatment of blood diseases came to a successful end (Anhui Station), this conference specially invited Professor Wu Jingsheng of the First Affiliated Hospital of the University of Science and Technology of China as the chairman of the conference, and invited Professor Yu Ziqiang of the First Affiliated Hospital of Soochow University, Professor Xu Weiqun of the Children's Hospital Affiliated to Zhejiang University School of Medicine and Professor Su Rui of the First Affiliated Hospital of Xiamen University shared the wonderful content
related to hemophilia.
Yimaitong has specially compiled the essence of the content for readers
.
At the beginning of the conference, Professor Wu Jingsheng said that the conference invited three well-known domestic experts and scholars to share the cutting-edge progress of hemophilia, and I believe that the doctors and patients attending the conference can gain a lot.
The meeting was then declared open.
Figure 1 Professor Wu Jingsheng delivered a wonderful speech
Professor Yu Ziqiang: Escorting, non-factorial drug treatment to help hemophilia patients achieve continuous bleeding control can achieve long-term bleeding control
is of great significance
to reduce the disease burden of hemophilia patients and improve the quality of life.
Professor Yu Ziqiang gave a wonderful sharing
on the topic of non-factor drugs to achieve bleeding control.
Key point 1: Although traditional coagulation factor drug prophylaxis can improve bleeding status, there is still a high ABRAHEAD study, and a total of 141 patients (95 preventive treatment, 46 on-demand treatment) were included in the Chinese subgroup, of which 111 patients experienced bleeding events
.
Even with prophylaxis, the proportion of patients with bleeding events was as high as 75.
8%.
Compared with patients receiving on-demand treatment alone, patients with hemophilia who received prophylaxis had a lower annualized bleeding rate (ABR) and annualized joint bleeding rate (AJBR) (8.
2 vs.
29.
4, 4.
1 vs.
8.
6), but patients still had a greater risk of bleeding and the situation was not optimistic
.
Figure 2 Professor Yu Ziqiang shares the results of the AHEAD study
Key point 2: Long-term effectiveness analysis showed that emelizumab prevention therapy can be effective in controlling bleeding sustainably and effectivelyA pooled analysis of efficacy and safety including HAVEN1-4 long-term follow-up data (N=401) showed that the ABR required treatment was only 1.
4, (95% Cl, 1.
1-1.
7) during the entire study period, and the median ABR for bleeding to be treated was maintained at 0; In all four studies, the ABR at intervals of every 24 weeks decreased with duration of treatment; Since week 24 of treatment, more than 80% of patients require 0 bleeding times per treatment interval, and more than
97% of patients require treatment ≤ 3 bleeding times.
FIGURE 3 PROFESSOR YU ZIQIANG SHARED THE RESULTS OF THE SUMMARY ANALYSIS OF HAVEN1-4 LONG-TERM FOLLOW-UP DATA
to hemophilia patients in China.
Professor Xu Weiqun: Practice is true, real-world data highlights the good efficacy and safety
of non-factor drug treatmentKey point 1: A number of real-world studies have proved that non-factor drug preventive treatment can achieve good bleeding control EMHIRA's real-world data shows that the use of emelizuzumab prophylaxis in hemophilia A patients can effectively reduce bleeding events, and the proportion of patients with zero bleeding is as high as 76.
5%.
German eDiary smart drug record data show that after treatment with emelizumab, the AJBR decreased to 0.
45 and the ABR decreased to 0.
73; before switching to emelizumab, the average ABR was 5.
89 and the average ABR after conversion was 1.
09; and good bleeding control was
shown in patients with/without inhibitors.
And a number of real-world data have shown that during the treatment of emelizumab, the incidence of adverse drug events in hemophilia patients is lower and the safety is better
.
Fig.
4 Professor Xu Weiqun shared real-world efficacy and safety data of emelizumab
Key point 2: Non-factor drug prophylaxis can significantly improve the bleeding status of children with hemophilia, and does not induce inhibitor production The results of a single-center, prospective study showed that 78.
6% (11/14) of children with hemophilia received emizizumab prophylaxis for 6 months, the bleeding rate dropped to 0, only 3 cases had at least 1 breakthrough bleeding, but none of them caused target joints, and the average musculoskeletal score before and after treatment decreased from 17 to 4.
It was shown that the bleeding rate and musculoskeletal condition of the children were significantly improved
after the prophylactic treatment of emelizumab.
There are also real-world data showing no spontaneous bleeding during emelizumab treatment, and all children are negative
for inhibitors.
Figure 5 Professor Xu Weiqun shared real-world data on the preventive treatment of hemophilia with emelizumab with emizizumab
for hemophilia families.
Professor Su Rui: Endless searching, new thinking on
the clinical management of surgery and comorbidities of hemophilia patients in the non-factor era For a long time, surgery for hemophilia patients has been one of the clinical problems; With the continuous improvement of patients' survival status, how to manage hemophilia patients with comorbidities has gradually become a hot spot
in clinical attention.
Professor Su Rui systematically elaborated
on the above topics.
Key point 1: Clinical studies combined with real-world data prove that patients can safely undergo major and minor surgeries under the preventive treatment of emizizizumab The STASEY study showed that 37 patients with hemophilia A with inhibitors underwent 56 minor surgeries, 57.
1% of minor surgeries were performed without additional prophylactic therapeutic drugs, most major surgeries (81.
8%) were treated with additional clotting factor prophylaxis, and no thrombotic microangiopathy (TMA) or TE associated with emelizumab was found; The summary analysis of HAVEN 1-4 study data shows that under the preventive treatment of emelizumab, more than 60% of minor surgeries do not require the use of additional coagulation factors, more than 80% of major surgeries require the use of coagulation factors, and there are no deaths, thrombots, and TMA events in all operations, indicating that patients can safely undergo major and minor surgeries
under the preventive treatment of emelizumab through reasonable measures.
The Slovenian data presented at the recent EAHAD conference once again confirms this conclusion
from a real-world perspective.
Figure 6 Professor Su Rui shared the operation of hemophilia patients under the background of non-factor drug treatment
Key point 2: The safety and effectiveness of non-factor drug treatment in patients with hemophilia has confirmed that the risk of cardiovascular diseases such as hypertension in hemophilia patients is increased compared with control adults, and it is of great significance
to explore the management strategies of such patients with comorbidities in the context of the overall survival of hemophilia patients 。 The HAVEN 1, 3 and 4 and STASEY studies demonstrated that emelizumab prophylaxis provided stable bleeding control in patients with hemophilia A with comorbidities, and adverse events were similar in patients with and without comorbidities; Real-world data from the Israel National Center for Hemophilia showed that the ABR of emelizumab was significantly lower
than that before emelizumab treatment in hemophilia patients with hemophilia with cardiovascular disease risk factors such as dyslipidemia, morbid obesity, and ischemic heart disease.
Figure 7 Professor Su Rui shared the situation of patients with comorbidities hemophilia receiving non-factor drug treatment
Meeting discussion
The highest goal of hemophilia treatment is to enable patients to achieve normal clotting function and live
like normal people.
There is still much room to be explored along this path, but the advent of emelizumab has made an important contribution to achieving this goal, providing a good choice
for patients.
The preferred treatment for patients with hemophilia with inhibitors is immunoinduced tolerance (ITI).
Emelizumab prophylaxis at the same time as ITI reduces the dose and frequency of ITI and reduces bleeding events
.
At present, many centers in China are exploring the use of ITI combined with emelizumab for the prevention of hemophilia with inhibitors, and look forward to follow-up results
.
The results show that adolescent patients can safely play sports in school life without discomfort, and infants and young children have received emelizumab prophylaxis from 10 months, bleeding status is significantly improved, and family anxiety is alleviated
.
The clinical experience of the First Affiliated Hospital of Xiamen University shows that the patients currently receiving the prophylactic treatment of emelizumab have basically achieved zero bleeding
.
The clinical experience of the Children's Hospital Affiliated to Zhejiang University School of Medicine shows that the above patients have a strong call for emelizuzumab, and some patients receiving preventive treatment have significant
efficacy.
However, more exploration is needed to match the demand for the drug with its accessibility, and it is expected that the drug will be able to enter medical insurance
as soon as possible.
The conference summary
began with the end, the journey did not stop, and the series of new progress in clinical diagnosis and treatment of hematological diseases came to a
successful conclusion of the Hemophilia Frontier Progress Tour.
Professor Wu Jingsheng, chairman of the conference, said that the speakers shared the latest progress of hemophilia at home and abroad, thanked all experts, hemophilia patients and family members who participated online for their active participation, and looked forward to more progress
in hemophilia treatment in the future.
Editor: Valamor Review: Janet Typesetting: Quinta Executive: Quarterly
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