Extended half-life recombinant factor VIII treatment of hemophilia A in Brazil: an expert consensus statement.

Autor:	Ozelo MC; Universidade Estadual de Campinas (Unicamp), Campinas, SP, Brazil., Antunes SV; Universidade Federal de Sao Paulo (USP), São Paulo, SP, Brazil., Villaca PR; Hospital das Clínicas da Universidade de São Paulo (HC USP), São Paulo, SP, Brazil., Oliveira LC; Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo (HCFMRP/USP), Ribeirão Preto, SP, Brazil., Pinto IS; Fundação Centro de Hematologia e Hemoterapia do Pará (Hemopa), Belém, PA, Brazil., Lorenzato CS; Centro de Hematologia e Hemoterapia do Paraná (Hemopar), Londrina, PR, Brazil., Prezotti ANL; Centro de Hematologia e Hemoterapia do Espírito Santo (Hemoes), Vitória, ES, Brazil., Picoli RM; Cerner Enviza, São Paulo, SP, Brazil. Electronic address: renato.picoli@cernerenviza.com.
Jazyk:	angličtina
Zdroj:	Hematology, transfusion and cell therapy [Hematol Transfus Cell Ther] 2024 Jan-Mar; Vol. 46 (1), pp. 36-41. Date of Electronic Publication: 2022 Dec 23.
DOI:	10.1016/j.htct.2022.11.008
Abstrakt:	Introduction: Treatment of hemophilia A in Brazil is offered to all patients at no cost. However, several unmet medical needs exist. Method: In this study, we applied the Delphi method to discuss with seven hemophilia A specialists the challenges that patients and the health system face regarding hemophilia A treatment and opportunities for improvement. Results: A consensus was obtained regarding the number of weekly infusions and patient adherence to treatment. The bleeding profile, unfavourable pharmacokinetics (PKs), low adherence and high daily activity were patient profiles that would benefit from using the extended half-life (EHL) recombinant factor VIII (rFVIII). The advantages of treatment with the EHL rFVIII were the lower number of infusions per week, which could increase patient adherence and decrease the risk of bleeds, due to a more constant plasma level, a lower value. Additionally, the EHL rFVIII could improve quality of life, especially in patients with high daily activity, such as adolescents and young adults. The panelists mentioned that EHL rFVIII, if available, could be offered first to the priority group (adolescents between 12 and 19 years old), followed by adults (20 to 64 years old) and elderly people (over 65 years old). Conclusion: In summary, the EHL rFVIII offers the optimal prophylaxis by decreasing the dose frequency, increasing the treatment adherence and improving the QoL, without compromising safety and efficacy. Competing Interests: Conflicts of interest Hemophilia experts received consulting fees from Bayer to participate in this study. RP is an employee of Cerner Enviza, which received consulting fees from Bayer to conduct this study. LCO was granted research support from Novo Nordisk, Pfizer, Shire (a Takeda company), provides consultant advice to Novo Nordisk, Roche, Sanofi, Shire (a Takeda company), Bayer, and works in the Speaker Bureau to Novo Nordisk, Roche, Shire (a Takeda company). MCO received research support from BioMarin, Novo Nordisk, Pfizer, Sanofi, Roche, and Takeda and has participated as a speaker and/or in advisory boards sponsored by Bayer, BioMarin, Novo Nordisk, Pfizer, Roche and Takeda. PRV was granted research support for the institution from BioMarin, Takeda, Novartis; provides consultant advice to Bayer, CSL-Behring, Novo Nordisk, Roche, Sanofi, Takeda; participates in the Speaker Bureau to Bayer, BioMarin, Novo Nordisk, Roche, Sanofi and Takeda. CSL was granted research support from Roche, Biomarin and Takeda. ISP was granted research support from Roche, Biomarin, Bayer, Novo Nordisk and Takeda. SVA was granted research support from Bayer, Novo Nordisk and Takeda. (Copyright © 2022 Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular. Published by Elsevier España, S.L.U. All rights reserved.)
Databáze:	MEDLINE
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