Rapporto ISTISAN 21/15 - National Registry of congenital bleeding disorders. Report 2019. Francesca Abbonizio, Romano Arcieri, Associazione Italiana Centri Emofilia (AICE) and Adele Giampaolo - ISS (EN)

Istituto Superiore di Sanità
National Registry of congenital bleeding disorders. Report 2019.
Francesca Abbonizio, Romano Arcieri, Associazione Italiana Centri Emofilia (AICE) and Adele Giampaolo
2021, iii, 50 p. Rapporti ISTISAN 21/15 (in Italian)

The National Registry of congenital bleeding disorders collects data relative to patients with congenital coagulopathies, with particular attention to the complications of therapy and the need of drugs necessary for the treatment. In the 2019 survey, there was no transmission of data relative to patient records, diagnosis and adverse events related to therapy; instead, the therapeutic plans on drugs prescribed for home therapies were received, collected through the usual direct flow from the Hemophilia Centers to the ISS. The therapeutic plans concerned about 45% of patients with severe Hemophilia A and B: prophylaxis was the most adopted regimen in patients with severe Hemophilia A (92.1%) and B (89.8%). Analyzing current inhibitor patients, 65.5% was <18 years of age; 29.1% of the inhibitor patients was treated with the combination of two different therapies. The FVIII prescribed in 2019 was about 360,000,000 International Units (IU), the FIX was about 28,500,000 IU. The amount of emicizumab (Hemlibra®) was approximately 49,000 mg. Extended half-life factors Elocta® and Alprolix® increased by 12.8 and 6.9%, respectively.
Key words: Hemophilia centers; Bleeding disorders; Hemophilia; Factor VIII; Plasma-derived products; Recombinant clotting factors