A systematic review evaluating the efficacy and factor consumption of long-acting recombinant factor VIII products for the prophylactic treatment of hemophilia A

SummaryIn this open multicenter study the safety and efficacy of recombinant factor VIII (rFVIII) was assessed in 39 previously treated patients with hemophilia A (factor VIII basal activity ≤15%).Recombinant FVIII was administered for prophylaxis and treatment of bleeding episodes and for surgical procedures. A total of 3679 infusions of rFVIII were given. Efficacy of rFVIII as assessed by subjective evaluation of response to infusion and mean annual consumption of rFVIII was comparable to that of plasma derived FVIII concentrates. The incremental recovery of FVIII (2.4 ± 0,83%/IU/kg, 2.12 ± 0.61%/IU/kg, resp.) was within the expected range. No clinical significant FVIII inhibitor was detected in this trial. Five of 16 susceptible patients showed a seroconversion for parvovirus B19. However, the results are ambiguous in two cases and might be explained otherwise in one further case. Thus, in two patients a reliable seroconversion for parvovirus B19 was observed.

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Long-term efficacy and safety of prophylaxis with recombinant factor VIII in Chinese pediatric patients with hemophilia A: a multi-center, retrospective, non-interventional, phase IV (ReCARE) study

Current Medical Research and Opinion ◽

10.1080/03007995.2017.1310720 ◽

2017 ◽

Vol 33 (7) ◽

pp. 1223-1230 ◽

Cited By ~ 3

Author(s):

Changgang Li ◽

Xinsheng Zhang ◽

Yongqiang Zhao ◽

Runhui Wu ◽

Qun Hu ◽

...

Keyword(s):

Factor Viii ◽

Pediatric Patients ◽

Hemophilia A ◽

Recombinant Factor Viii ◽

Efficacy And Safety ◽

Term Efficacy ◽

Phase Iv

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MODERN POSSIBILITIES OF SUBSTITUTION THERAPY AND HEMOPHILIA A PREVENTION IN CHILDREN

PEDIATRIA Journal named after G N SPERANSKY ◽

10.24110/0031-403x-2021-100-2-182-187 ◽

2021 ◽

Vol 100 (2) ◽

pp. 182-187

Author(s):

P.A. Zharkov ◽

Keyword(s):

Factor Viii ◽

Half Life ◽

Hemophilia A ◽

Safety Data ◽

Residual Activity ◽

Recombinant Factor Viii ◽

Substitution Therapy ◽

Life Effectiveness ◽

Intravenous Injections ◽

Prophylactic Use

Currently, the prophylactic use of factor VIII concentrate is the «gold standard» for treatment of an uncomplicated severe hemophilia A without inhibitors. However, there are a number of difficulties associated with frequent intravenous injections to maintain the activity of factor VIII above 1% in children and adolescents, which cannot but affect the adherence of patients to this type of treatment. The article discusses modern approaches to extend the half-life of recombinant factor VIII allowing to reduce the frequency of infusions and increase the residual activity of the deficient factor. On the example of efmoroctocog alpha, the first recombinant factor VIII concentrate registered in our country with a prolonged half-life, effectiveness and safety data of this class of drugs approved for use in children is presented.

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