Individualized therapy of hemophilia A with efmoroctocog alfa considering the clinical phenotype and prothrombotic risks caused by FV Leiden mutation: 616.151.514-085

Abstract

Summary. Factor-replacement therapy, modern standard-of-care approach in hemophilia, has significantly reduced the risk of life-threatening bleedings and increased the patients’ life expectancy almost to that of the average population. However, hemophilic arthropathy is still an unresolved problem. Prophylaxis with concentrates of coagulation factors has reduced the number of joint bleeds, but the patients’ compliance remains a serious challenge. To improve the compliance we can use both non-factor therapy and extended half-life factors (such as Efmoroctocog alfa), which reduce the number of intravenous injec- tions. At the same time, intensifying hemophilia care brings up the following question: is it safe to use new drugs in patients with concomitant thrombophilia markers? Nowadays, there is a growing number of publications concerning thromboses in patients with hemophilia, which may be the consequence of extended life expectancy, or intensive perioperative prophylaxis. Assessing individual thrombotic risks, including genetic and non-genetic markers, seems promising. Some markers, besides the expected prothrombotic effect, produce protective effect on the clinical course of hemophilia, which is evidenced by the experience of our department: hemophilic patients with FV Leiden mutation had milder arthropathy in terms of severity and the number of affected joints. Here we present 2 clinical cases of Efmoroctocog alfa use in patients with severe hemophilia A with similar anthropo- metric and laboratory characteristics but with different FV Leiden mutation status and disease phenotype. A dose of 50 IU/kg allowed for achieving normal coagulation in both patients, which was additionally confirmed by thrombin generation test, and performing minor surgery with no hemorrhagic complications. At the same time, the use of Efmoroctocog alfa did not induce hypercoagulation in both cases. Our data illustrate that Efmoroctocog alfa is preferable for both patients, considering different clinical phenotype and prothrombotic risk.

For citation: SoldatenkovaO.V., SoldatenkovV.E., BurakovV.V., KomissarovK.A., SilinaN.N., SmirnovaO.A., MatvienkoO.Yu., GertT.N. Individualized therapy of hemophilia A with efmoroctocog alfa considering the clinical phenotype and prothrombotic risks caused by FV Leiden mutation. Tromboz, gemostaz i reologiya. 2023;(3):71–82. (In Russ.).

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