Contributions
Abstract: PB1465
Type: Publication Only
Session title: Bleeding disorders (congenital and acquired)
Background
The emergence of modern concentrates of coagulation factor VIII (FVIII) has changed the management tactics and improved the quality of life of patients with hemophilia A, compared to the period before 2005, when such patients in Russia received therapy with cryoprecipitate and fresh frozen plasma (FFP).
Aims
The objective of the observational study was a retrospective analysis (2017 - 2019) of the efficacy of using a recombinant antihemophilic factor (coagulation factor VIII drug with a deleted B domain) - Moroctocog alpha for the prophylactic treatment of patients with severe and moderate forms of hemophilia A living in the Amur Region and in the Khabarovsk Territory of the Far Eastern Federal District of Russia.
Methods
Patients with hemophilia A over the age of 20 were divided into 2 groups. In group 1 - 21 patients received substitution therapy with Moroctocog alpha; age composition: 40-50 years old (n = 10), 30-39 years old (n = 6), 20-29 years old (n = 5). All patients were diagnosed with hemophilic arthropathy: deforming arthrosis of the knee (n = 21), ankle (n = 12), elbow (n = 16) joints. Group 2 included 21 patients who received plasma FVIII and octocog alpha at the age of 40-50 years (n = 8), 30-39 years (n = 8), 20-29 years (n = 5). All patients were diagnosed with hemophilic arthropathy: deforming arthrosis of the knee (n = 21), ankle (n = 13), elbow (n = 17) joints. In both groups, there were 13 patients with severe and 8 with moderate forms of hemophilia A. In both groups, FVIII concentrates at a dose of 20-40 IU / kg of body weight were injected every 2-3 days (2-3 times a week).
Results
Until 2005, when patients of both groups received therapy with cryoprecipitate or FFP 'on demand', all of them had frequent hemorrhagic complications from 52 to 6 episodes per year, bleeding and hemorrhage were of all severity, including life-threatening; patients with a severe form were hospitalized 8-10, and with a moderate form - 4 times a year. During the study period, against the background of prophylactic use of the Moroctocog alpha drug and other FVIII drugs no more than 2 episodes of bleeding (spontaneous and post-traumatic) of mild and moderate severity were noted in 7 patients of the first group and 6 patients of the second group. Spontaneous bleeding was diagnosed only in patients with low adherence to treatment, which was stopped by the administration of the drug in therapeutic doses, mainly on an outpatient basis, and rarely required hospitalization. The desired indicators of clinical control were achieved - no more than 2 cases of spontaneous hemarthrosis or other bleedings per year. In both groups, it was possible to achieve a 98% reduction in the number of bleedings compared to the period before 2005, when these patients received treatment with cryoprecipitate and FFP “on demand”. When using Moroctocog alpha there were no adverse reactions in patients.
Conclusion
It was concluded that Moroctocog alpha is not inferior to plasma concentrates FVIII and Octocog alpha in terms of efficacy and safety, and can be effectively used for the preventive treatment of bleeding in hemophilia A.
Keyword(s): Bleeding disorder, Factor VIII, Prevention, Treatment
Abstract: PB1465
Type: Publication Only
Session title: Bleeding disorders (congenital and acquired)
Background
The emergence of modern concentrates of coagulation factor VIII (FVIII) has changed the management tactics and improved the quality of life of patients with hemophilia A, compared to the period before 2005, when such patients in Russia received therapy with cryoprecipitate and fresh frozen plasma (FFP).
Aims
The objective of the observational study was a retrospective analysis (2017 - 2019) of the efficacy of using a recombinant antihemophilic factor (coagulation factor VIII drug with a deleted B domain) - Moroctocog alpha for the prophylactic treatment of patients with severe and moderate forms of hemophilia A living in the Amur Region and in the Khabarovsk Territory of the Far Eastern Federal District of Russia.
Methods
Patients with hemophilia A over the age of 20 were divided into 2 groups. In group 1 - 21 patients received substitution therapy with Moroctocog alpha; age composition: 40-50 years old (n = 10), 30-39 years old (n = 6), 20-29 years old (n = 5). All patients were diagnosed with hemophilic arthropathy: deforming arthrosis of the knee (n = 21), ankle (n = 12), elbow (n = 16) joints. Group 2 included 21 patients who received plasma FVIII and octocog alpha at the age of 40-50 years (n = 8), 30-39 years (n = 8), 20-29 years (n = 5). All patients were diagnosed with hemophilic arthropathy: deforming arthrosis of the knee (n = 21), ankle (n = 13), elbow (n = 17) joints. In both groups, there were 13 patients with severe and 8 with moderate forms of hemophilia A. In both groups, FVIII concentrates at a dose of 20-40 IU / kg of body weight were injected every 2-3 days (2-3 times a week).
Results
Until 2005, when patients of both groups received therapy with cryoprecipitate or FFP 'on demand', all of them had frequent hemorrhagic complications from 52 to 6 episodes per year, bleeding and hemorrhage were of all severity, including life-threatening; patients with a severe form were hospitalized 8-10, and with a moderate form - 4 times a year. During the study period, against the background of prophylactic use of the Moroctocog alpha drug and other FVIII drugs no more than 2 episodes of bleeding (spontaneous and post-traumatic) of mild and moderate severity were noted in 7 patients of the first group and 6 patients of the second group. Spontaneous bleeding was diagnosed only in patients with low adherence to treatment, which was stopped by the administration of the drug in therapeutic doses, mainly on an outpatient basis, and rarely required hospitalization. The desired indicators of clinical control were achieved - no more than 2 cases of spontaneous hemarthrosis or other bleedings per year. In both groups, it was possible to achieve a 98% reduction in the number of bleedings compared to the period before 2005, when these patients received treatment with cryoprecipitate and FFP “on demand”. When using Moroctocog alpha there were no adverse reactions in patients.
Conclusion
It was concluded that Moroctocog alpha is not inferior to plasma concentrates FVIII and Octocog alpha in terms of efficacy and safety, and can be effectively used for the preventive treatment of bleeding in hemophilia A.
Keyword(s): Bleeding disorder, Factor VIII, Prevention, Treatment