The implementation of the state program “7 highcost nosologies” and the active work of Russian hematologists have significantly improved the specialized care for children and adults with Hemophilia. Russian hemophilia patient registry as of 10.25.2018 contained information about 7433 patients, of whom with hemophilia A – 6525 people. About 400 people were diagnosed with hemophilia with inhibitors. The inhibitor predominantly appeared at child and young age (up to 20 years). There is a high supply of coagulation factors concentrates for the treatment of hemophilia in the Russian Federation – 8.1 IU of coagulation factor VIII per capita in 2018, which corresponds to the graduation “full integration into society” according to the scale proposed by the World Hemophilia Federation. Due to the sufficient availability of coagulation factors, it is possible to conduct elimination of inhibitors by immune tolerance induction. Treatment with antiinhibitor coagulant complex and eptacog alfa (activated) requires a good venous access and is not always effective. Treatment results remain unsatisfactory in 67 % of adult patients with severe hemophilia with low inhibitor titer due to the number of bleeding per year exceeds 4. Unsatisfactory treatment results are noted in more than 1/ 3 patients with a high inhibitor titer, despite the ongoing prophylaxis with bypassing agents. Currently, clinical studies of fundamentally new drugs for hemophilia treatment, including the inhibitory form, are ongoing. One such drug is emicizumab, which is a bispecific humanized monoclonal antibody that bridges activated factor IX and factor X to restore the function of missing activated factor VIII Emicizumab is not neutralized by inhibitors to FVIII, which allows it to be successfully used in the inhibitory form of hemophilia A. The results of HAVEN 1 and HAVEN 2 studies showed the advantages of using emicizumab in prophylactic regimen in children and adults with the inhibitory form of hemophilia A compared with bypassing agents.
Introduction. Cryosupernatant is blood component. Cryosupernatant is the supernatant plasma removed during the preparation of cryoprecipitate. Aim. To provide information on the composition and methods of production, storage, transportation and clinical use of Cryosupernatant. General fi ndings. In comparison with fresh frozen plasma (FFP) and cryoprecipitate, Cryosupernatant plasma is depleted in factor VIII, fi brinogen factor von Willebrand (VWF). Cryosupernatant is defi cient in high molecular weight multimers of VWF, but contains VWF metalloproteinase. The concentrations of factor V, antithrombin III, albumin and immunoglobulins are the same as in FFP and cryoprecipitate. The indications for Cryosupernatant transfusions are massive blood loss in patients with factor VIII inhibitor, plasma exchange in patients with thrombotic thrombocytopenic purpura. For children the doses of Cryosupernatant should be 10-15 mL/kg.
Patients with a severe and moderate form of hemophilia A have traditionally been prescribed standard prevention with a coagulation factor VIII (FVIII), the goal of which is to achieve zero bleedings per year and a remaining activity of FVIII no fewer than 1 %. The standard approach does not allow achieving these goals in many patients due to a variety of factors: age of the patient, lifestyle, level of physical activity, condition of joints, muscle tone, patient compliance, individual pharmacokinetic (PK) response to FVIII administration. The target remaining activity of FVIII may be 2, 3 or even 5 % depending on the level of physical activity. Nowadays an individualized approach to the treatment of patients with severe hemophilia A based on the patient's PK profile is actively being explored and implemented in clinical practice. Individualization of prevention in patients with severe hemophilia is a real need for the physician and patient. There is a high variability in the values of the half-life period of FVIII in different patients. It is necessary to monitor the duration of the time period when the remaining activity of FVIII is less than 1 %, i.e., the period which is directly linked to the risk of spontaneous hemorrhage. For the patients getting treatment of Octocog alfa there has been developed the software (SW) myPKFiT* on the basis of web application which allows to simulate a dosage regimen taking into account the patient's PC profile based on the determination of FVIII activity in 2 blood sample. The SW allows changing (increase) the target level of the remaining activity of FVIII considering the lifestyle and the level of physical activity of the patient. The ability of SW allows the patient to demonstrate the activity of FVIII at various doses and intervals of drugs, as well as identify the risks that arise when a drug is missed. Therefore, myPKFiT solves an important task of the individualized approach to selection and correction of therapy, improves the collaboration and mutual understanding between the physician and the patient, up regulates of the patient adherence to the therapy and achieves optimal results.
Heparin-induced thrombocytopenia (HIT) is a serious and potentially life-threatening side effect of heparinotherapy. It is an antibody-mediated process that causes platelet activation, increases the procoagulant characteristics of the blood and, as a result, endangering limbs and life-threatening thrombosis. Venous thrombosis is more common than arterial thrombosis, especially deep vein thrombosis of the lower limbs and pulmonary artery thrombosis. Mortality from complications of heparinotherapy occurs with a frequency of 20–30 % of cases. Diagnosis of HIT is difficult. Such basic symptoms as thrombocytopenia and thrombosis are extremely non-specific and may be present in cancer patients and patients with cardiosurgical pathologies without the impact of heparin. Women are twice as likely to have HIT as men. This review describes pathogenesis, clinical features, modern diagnostic methods, risk factors for the emergence of this formidable complication of heparinotherapy, gives an overview of the most frequent use of drugs for the treatment of HIT, and gives modern clinical recommendations for different groups of patients.
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