E. Zanon, S. Pasca, P. Radossi, E. Bonetti, A. C. Giuffrida, A. Tosetto
Abstract
The replacement therapy with clotting factor concentrates is considered the treatment of choice for patients with hemophilia, both concentrates of plasma origin and those recombinants are in fact considered safe and effective in preventing or treating bleeding. Prophylaxis is the gold standard of therapies, especially in patients with severe or severe-moderate hemophilia, but in some cases the compliance of patients is reduced due to the need of frequent infusions. In recent years, several new standard half-life or extended half-life concentrates have been marketed.
These drugs have improved the compliance of patients to therapy, and, in many cases, reduced the annual bleeding rate and improved their quality of life. Thanks to these innovative products, even some severe adult patients, always reluctant to long-term therapies, have accepted to start a prophylactic treatment. Lonoctocog-alfa (Afstyla®), the first and only developed recombinant FVIII (rFVIII) single chain is one of these new drugs. Here we report our experience with rFVIII single-chain in a population of hemophilia A patients, followed for one year and compared with their previous treatment with octocog-alfa.
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Overall comparison of total ABR (AtBR), joint ABR (AjBR) and spontaneous ABR (AsBR) between the two different one-year treatments for each enrolled patient. PRO: prophylaxis; OD: on-demand