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With the gradual improvement of the diagnosis and treatment system and the continuous emergence of coagulation factor drugs, the living conditions of hemophilia patients, who were once dubbed "glass man" and "porcelain doll" by the public, have improved significantly.
The cloudy sky gathers the color of summer, and the leaves move with the sound of autumn.
Chairman's speech
At the beginning of the meeting, Professor Wu Jingsheng, the chairman of the conference, said that this meeting was honored to invite Professor Cai Lisheng and Professor Chen Shu to share with you about the pharmacokinetics (PK) of hemophilia and the new recombinant human coagulation factor Turoctocog alfa.
Figure 1 Prof.
Based on individual differences, scientific and standardized pharmacokinetic testing guides individualized treatment of hemophilia
Although the traditional method of calculating the dosage of coagulation factors in patients with hemophilia based on body weight can meet the needs of some patients, with the continuous improvement of patients' health needs, the treatment of hemophilia urgently needs a new dosing strategy to guide medication
The principle of individualized treatment to guide the diagnosis and treatment of hemophilia has become a common recommendation in domestic and foreign guidelines.
"Chae's formula" is a one-compartment model mathematical formula improved on the basis of classical PK, which can calculate the half-life, and estimate the peak concentration and recovery rate
During the perioperative period, it should be ensured that the activity of FⅧ is within the range of activity specified in the guidelines.
The PK tool can be used to calculate the FⅧ usage plan.
It should be noted that intraoperative bleeding or hemostasis will consume FⅧ.
If the operation time exceeds 4 hours or the bleeding is heavy, FVIII can be temporarily supplemented
.
The individualized application of Turoctocog alfa can help patients stop bleeding quickly, and this product, together with public welfare actions, has helped more patients greatly reduce the financial burden
.
Figure 2 Guidelines recommend that the diagnosis and treatment of hemophilia follow the principle of individualized treatment
Figure 3 Chua's formula content and application
Figure 4 Turoctocog alfa individualized application and its advantages
Verified from multiple perspectives, Turoctocog alfa is safe, portable, effective and beneficial to the people, benefiting many patients with hemophiliaFVIII replacement therapy remains the standard treatment for hemophilia A, and the advent of recombinant human FVIII has had a profound impact on this treatment regimen
.
Professor Chen Shu shared new thoughts on how the genetically recombinant human FⅧ preparation Turoctocog alfa can help more patients with hemophilia A realize their dreams of normal life.
The main points are as follows:
Plasma-derived FVIII has many potential clinical limitations, such as limited supply, inconvenient infusion, and risk of viral infection, especially the screening/inactivation of plasma-derived FVIII viruses such as parvovirus, prion, immunodeficiency virus, etc.
There are limitations and cannot guarantee patient safety
.
Turoctocog alfa's unique B domain deletion, good portability and usability, and 5-step purification process greatly make up for the shortcomings of traditional plasma-derived FVIII
.
A 30-month study confirmed that all batches of Turoctocog alfa showed titer changes within limits, and excellent stability underpins its good portability
.
The Guardian 1/3/2 study confirmed that long-term treatment of Turoctocog alfa can effectively prevent and treat bleeding, and all the included patients who received previous treatment (PTP) had no inhibitor formation; the main trial period and the overall trial period of the Guardian 7 study showed that Turoctocog alfa had hemostasis.
The efficiency is more than 95%, and the annualized bleeding rate (ABR) of patients receiving preventive treatment is only 1.
18, and there is no inhibitor formation; the results of the post-marketing study Guardian 5 show that Turoctocog alfa has a hemostatic effective rate of 87.
3%, and the median ABR of patients receiving preventive treatment is 87.
3%.
1.
97, the inhibitor incidence rate was 0, which once again confirmed the good efficacy and safety of Turoctocog alfa from the real world dimension
.
Turoctocog alfa has been adjusted in price.
While greatly reducing the economic burden of patients, it is expected that hemophilia patients treated with plasma-derived FVIII can be converted to Turoctocog alfa for standard-dose preventive treatment, helping patients achieve their dreams of normal life
.
In addition, the implementation of projects such as "Easy Health Guardian" can further improve the availability of medication and benefit patients
.
Figure 5 Turoctocog alfa has excellent stability
Figure 6 Turoctocog alfa has good hemostatic efficiency
Figure 7 Turoctocog alfa Huimin price helps hemophilia patients realize their dream of a normal life
meeting discussionThe three professors had a full and lively discussion on the above content
.
The main points are as follows:
In order to help more doctors in primary hospitals understand individualized treatment, the "Cai's formula" can be considered as a guide or manual in the future
.
At present, the formula is still in the exploratory stage, and it still needs to be verified by clinical application data from more regional hospitals in China
.
It is very important to ensure patient education and timely assessment at the hospital during the epidemic
.
Through the previous data collection, experience summarization and review by the comprehensive management center, the current management of hemophilia patients has been significantly improved, and patients' awareness of hemophilia, the acceptance of preventive treatment, and social participation have been improved.
, but also very cooperative with hospital management
.
Price cuts and policy support will further promote the improvement of the above situation and form a virtuous circle
.
It is hoped that doctors, patients, related companies, and the media in the field of hemophilia will jointly speak out to promote the implementation of medical insurance policies, and give more care and policy preference to hemophilia patients, so that patients can actually feel the benefits
.
Figure 8 The experts at the conference had a heated discussion
Summary of the meetingAfter wonderful topic sharing and fully heated discussions, the meeting was a complete success
.
Professor Wu Jingsheng expressed his gratitude for the sharing of the speakers and the strong support of related companies, and expressed his hope that Professor Cai Lisheng's "Cai's formula" can be promoted nationwide in the future, filling the gap in the pharmacokinetics of hemophilia in China and going global; It is hoped that Professor Chen Shu can lead the Chongqing Hemophilia Comprehensive Management Center to more standardized diagnosis and treatment of hemophilia patients, so as to achieve the center construction goals and benefit more patients
.
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