Gestione terapeutica dell’emofilia A

Translated title of the contribution: [Autom. eng. transl.] Therapeutic management of hemophilia A

Chiara Cadeddu, Gualtiero Ricciardi, Chiara De Waure, Maria Rosaria Gualano, Nicola Nicolotti, Francesco Di Nardo

Research output: Contribution to journalArticle

Abstract

[Autom. eng. transl.] Treatment of the patient with haemophilia A is carried out with the infusion of the deficient coagulation factor, therefore of factor VIII. The therapy can be conducted "on demand", in the course of a current haemorrhagic episode, to limit its severity and induce resolution, or for a prophylactic purpose, to reduce the risk of joint damage. In particular, primary prophylaxis constitutes the gold standard for the prevention of joint degeneration, as established by the World Federation of Hemophilia (WFH) itself. The substitution treatment, as also declined in the guidelines of the Italian Association of Hemophilia Centers, should preferably be implemented with products from combining DNA, if available. In fact, plasma-derived FVIII presents potential safety problems that need to be properly kept in mind, not so much for "historical" infections (HIV, HCV, HBV), widely spread in the 1980s, as for the risks infectious from pathogens not known or still difficult to control. The recombinant factors are instead obtained with the recombinant DNA technique and are distinguished in first, second and third generation products according to the presence of human and plasma-derived animal proteins used as an additive in the culture medium and / or as a stabilizer; in Italy the ones presented in Table 5 are currently available. Although from the point of view of efficacy all the products show a good profile, clearly with those of newer generation it was possible to achieve a greater level of safety with reference to the infectious potential. Furthermore, there is evidence to suggest that the elimination of the B domain from the FVIII molecule, carried out in some of the available recombinant products, may influence the clinical effects of the product itself. However, the problem of inhibitor development remains open, the most feared clinical complication of substitution treatment due to its dramatic impact both in terms of management and the resources to be dedicated. Finally, given the complexity of the pathology, the individual producing companies should evaluate the possibility of enriching the therapeutic options with initiatives designed to add value to the individual product in the integral management of the patient.
Translated title of the contribution[Autom. eng. transl.] Therapeutic management of hemophilia A
Original languageItalian
Pages (from-to)17-30
Number of pages14
JournalItalian Journal of Public Health
Publication statusPublished - 2011

Keywords

  • Emofilia
  • HTA
  • Trattamento

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