Reducing bleeding, protecting joints, and medication safety, the evidence is here!

do you know? There is such a group of children who cannot have a happy childhood due to the high risk of bleeding in daily activities
.

They suffer from a rare disease - hemophilia, hemophilia A which went mainly
.

In order to protect this special group, coagulation factor Ⅷ replacement therapy is currently used clinically
.

This issue, we discuss with recombinant human coagulation factor Ⅷ (rFⅧ) clinical research evidence rAHF-PFM in children with hemophilia A patients
.

ESPRIT study: Patients treated with rAHF-PFM prophylaxis have less bleeding, lower joint damage, and higher quality of life.
1 An independent, multicenter, parallel, open-label, randomized controlled study involving 40 children with severe hemophilia A.
The patients were randomly divided into the rAHF-PFM preventive treatment group (n=21) and the on-demand treatment group (n=19), and evaluated the number of bleeding events, the severity of joint damage, and the quality of life of the patients under the two treatment options
.

The results show: the number of bleeding events: Compared with on-demand treatment, rAHF-PFM prophylaxis can significantly reduce the number of bleeding (37.
9 vs 82.
4, P＜0.
01) (Figure 1) Figure 1 Number of bleeding events in patients treated with rAHF-PFM Joint damage Degree: The proportion of patients with joint damage in the preventive treatment group was significantly lower than that in the on-demand treatment group (29% vs 74%, P<0.
05).
The rAHF-PFM preventive treatment can effectively delay the occurrence of joint damage (Figure 2)
.

Figure 2 Quality of life of joint damage under rAHF-PFM treatment: rAHF-PFM preventive treatment can significantly improve the quality of life of children and reduce the time of parental care (Figure 3)
.

Figure 3 The quality of life of patients treated with rAHF-PFM RODIN study: the risk of inhibitors is lower under rAHF-PFM treatment 2 A multi-center, retrospective study involving 574 patients born between January 1, 2000 and 20101 Untreated pediatric patients with severe hemophilia A on the 1st, all children received FVIII products (blood-derived and recombinant) treatment
.

Compare the relationship between children receiving different FVIII products and the occurrence of inhibitors
.

The results show that: the risk of inhibitors: Compared with the third-generation rFVIII (rAHF-PFM, etc.
), the second-generation rFVIII has a 60% higher risk of inhibitors (HR 1.
60, 95% CI 1.
08-2.
37; Figure 4)
.

Figure 4 Post-marketing surveillance study on the occurrence of inhibitors of different FVIII products in Japan: rAHF-PFM preventive treatment with less bleeding and better hemostatic effect 3 An open-label, multi-center, prospective, non-controlled, non-intervention, observational study Include 114 patients with hemophilia A who have not received treatment (PUP) and receive rAHF-PFM preventive treatment or on-demand treatment, and evaluate the annual bleeding rate (ABR), hemostatic effect and safety of the patients under the two treatment options The results showed: bleeding: The ABR of patients with severe hemophilia A showed that the average ABR of rAHF-PFM prophylaxis was lower than that of on-demand treatment (7.
4 vs 15.
7, P=0.
0164) (Figure 5)
.

Figure 5 The average ABR hemostatic effect of patients with severe hemophilia A: The proportion of "excellent" hemostatic effects of preventive treatment programs is higher than that of on-demand treatment (Figure 6)
.

Figure 6 The safety of hemostatic effect in patients treated with rAHF-PFM: The incidence of inhibitors under rAHF-PFM treatment is similar to previous reports, and there is no new safety signal
.

AHEAD study: long-term preventive treatment of rAHF-PFM is effective and safe.
A global multi-center, non-interventional large-scale real-world study that included more than 1,000 patients with moderate to severe hemophilia A receiving rAHF-PFM treatment worldwide
.

The observation period of each patient is ≥ 4 years
.

The study endpoints include long-term joint health outcomes, bleeding and safety, etc.
4.
As of now, the 6-year interim analysis results show: bleeding: Compared with on-demand therapy, patients receiving rAHF-PFM prophylaxis have lower ABR (Figure 7) 5
.

Figure 7 Proportion of patients with ABR zero bleeding under rAHF-PFM treatment: Compared with on-demand treatment, the proportion of patients with zero bleeding in preventive treatment is higher (Figure 8)5
.

Figure 8 Joint health outcomes with zero bleeding in patients treated with rAHF-PFM: During the observation period, the average Gilbert score of children receiving preventive treatment was always low, and the average Gilbert score of adult patients receiving preventive treatment was also lower than that of on-demand treatment Patient (Figure 9) 5
.

Figure 9: Safety of joint health of patients in various age groups under rAHF-PFM treatment (higher scores indicate deterioration): The 6-year mid-term safety analysis is consistent with the safety results reported in previous related studies, confirming the long-term treatment of rAHF-PFM Security
.

To summarize a number of clinical studies and real-world studies, patients with childhood hemophilia A receiving rAHF-PFM preventive treatment can reduce bleeding, delay the occurrence of joint damage, improve joint health outcomes, and have better safety
.

References: 1.
Gringeri, A,et al.
J Thromb Haemost.
2011;9(4):700-710.
2.
Gouw SC,et al.
N Engl J Med.
2013 Jan 17;368(3):231-9.
3 .
Taki M, et al.
Int J Hematol.
2019 Jan;109(1):70-78.
4.
Khair K, et al.
Haemophilia.
2018 Jan;24(1):85-96.
5.
MC Ozelo,et al.
Presented at the 28th International Society on Thrombosis and Haemostasis (ISTH), 2020; Abstracts PB0900.
Approval number: VV-MEDMAT-52201 Approval time: 9/6/2021 Expiration time: 9/6/2023 Disclaimer This information is intended to help healthcare Professionals have a better understanding of the latest developments in the field of related diseases
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