Actual Status of Inhibitor Hemophilia Management in Russia

Background: The number of inhibitor hemophilia patients in Russia is presently about 200 patients (4% of all hemophilia patients). Current medical community is moving towards increasing the efficacy and safety of treatment, as well as the standardization of services and improvement of standards of care in rare diseases area. Patient registries and databases are key tools for the improvement of patient care, healthcare resources planning as well as social, economical and quality of life outcomes. Due to the lack of data on clinical course of disease, treatment approaches and outcomes, National Hematology Society initiated continuously running prospective, multi-center, open-label, non-interventional, observational Registry of INhibitor Hemophilia patients (RING) in July 2014.

Aims: to document the natural history of hemophilia A or B disease in subjects with any type of inhibitors and to describe long-term treatment related outcomes in routine clinical practice.

Methods: Data of all inhibitor patients are collected by physicians from patients medical records documenting the routine patient's examination and reflecting the medical practice at the particular treatment center. All data are entered into e-database using on-line access secured by the Operator.

Results: In total 183 patients (101 adult and 82 children) from 47 centers were included in the Registry by June 2015. The majority of patients (90%) have severe hemophilia. The age of inhibitors diagnostics was: ≤10 years - 83%, 11-20 years - 3%, 21- 50 years - 5%, ≥51 years - 3% of patients. Chronic viral infections are diagnosed in 55 (30%) patients: 1 - HIV, 5- HBV and 49 - HCV. In 28% of patients annual bleeding rate (ABR) are ≥ 16, in 42% - 6-15, in 21% - ≤ 5. Approximately 25% of patients have zero joint ABR. Life threatening bleedings were registered in 25 patients (14%). Only 15 (8%) patients undergo immune tolerance induction (ITI); treatment with bypassing agents is administered in 151 (82%) patients. Due to the fact that activated prothrombin complex concentrate (aPCC) is not supplied by the State guarantees program 83% of patients receive rFVIIa (on-demand treatment - 50% and prophylaxis - 33%) and only 17% of patients have an access to aPCC treatment. The current data show that 34 (27%) patients with rFVIIa treatment need ≥3 injections to stop a bleeding episode thus they are identified as non-responders.

Conclusion: The clear view of current treatment situation based on available Registry data demonstrates the necessity of administering alternative treatment approaches for non-responding to rFVIIa inhibitor patients, which would optimize resources of the State guarantees program.