Blood: Recombinant FVIII drug Rurioctocog alfa pegol preventive treatment of haemophilia type A

Standard VIII factor (FVIII) preventive therapy for type A haemophilia (PwHA) is based on the patient's weight, FVIII defects and bleeding, and the dosage and frequency of medication are adjusted according to the patient's clinical response.
evidence that targeting 1% FVIII levels does not prevent bleeding episodes in all patients with haemophiliac type A.
Rurioctocog alfa pegol is a long-acting FVIII drug that has been approved by the FDA and EMA to be marketed in the United States and the European Union, respectively, for the treatment of (1) on-demand treatment and control of bleeding events;
Rurioctocog alfa pegol minimum ≥1% effective and well-to-do in PwHA patients, but there is evidence that this level does not prevent bleeding events in all PwHA patients, and the minimum FVIII level according to Pharmacodynamics (PK) should be kept above 10%.
PROPEL was a randomized Phase 3 trial that compared the safety and effectiveness of the two FVIII minimum levels in patients aged 12-65 years, with an annualized bleeding rate of ≥2, and PwHA severe patients who had previously received FVIII treatment.
patients were randomly divided into two groups, with control group FVIII with a minimum of 1-3% and an increased group of 8-12%, with Rurioctocog alfa pegol treated for 12 months.
treatment adjustment period is the first 6 months.
main endpoints: percentage of PwHA patients with zero total bleeding in the last 6 months (overall analysis of the disease, n, 115; programme analysis set, PPAS, n, 95).
the study process, 115 male PwHA patients were randomly grouped.
in the control and improvement groups, the estimates for PwHA patients with zero bleeding were 42% (95% CI 29-55%) and 62% (49-7) respectively in FAS. 5%) (P=0.055), 40% (27-55%) and 67% (52-81%) (P=0.015) in PPA, respectively.
the frequency and consumption of each group varies greatly.
rate of adverse reactions occurred in 60.9% (70/115).
6% (7/115) of the severe adverse reactions in the group, including 1 case of severe adverse reactions related to treatment in the improved group ARM (temporary anti-FVIII inhibitors).
no deaths or severe thrombosis events, and there was no interruption in treatment associated with adverse reactions.
, raising the minimum threshold for FVIII does not raise new security issues compared to previous studies.
results show that increasing the minimum level of FVIII can increase the proportion of patients without bleeding PwHA, while emphasizing the importance of personalized treatment.