Autor(es):
Álvarez-Román, MT ; Carvalho, M ; Benítez-Hidalgo, O ; Catarino, C ; García-Frade, LJ ; Rodríguez-López, M ; Antunes, M ; Fernández-Mosteirín, N ; Kjöllerström, P ; Morais, S ; Galmes, B ; López-Fernández, MF ; Riera-Sans, L ; Rodrigues, JF ; Jiménez-Yuste, V ; Salvado, R
Data: 2023
Identificador Persistente: http://hdl.handle.net/10400.17/4978
Origem: Repositório do Centro Hospitalar de Lisboa Central, EPE
Assunto(s): Hemophilia B; Epidemiologic studies; Treatment outcome; Quality of life; Spain; Portugal; HDE HEM PED
Descrição
Introduction: Treatment options for patients with hemophilia B (PWHB) have improved in the last decade, but epidemiological studies are required to optimize clinical management and define unmet needs. Objective: The objective of the study is to investigate current disease management and quality of life (QoL) in PWHB in Spain and Portugal. Methods: Epidemiological, cross-sectional, multicenter study with moderately severe and severe PWHB. Sociodemographic, epidemiological, clinical, treatment, and QoL data were collected retrospectively over a 24-month period. Results: Of the 75 patients included in the study, 78.7% received prophylactic treatment and 21.3% on demand; 65.3% were treated with SHL-FIX and 10.7% with rIX-FP. Bleeding occurred in 81.3%, mainly in the form of spontaneous bleeding. Prophylaxis resulted in a lower annualized bleeding rate, better joint health, and better QoL, compared to on-demand treatments. Patients treated with rIX-FP throughout the study had better disease control and QoL than those treated with SHL-FIX. Conclusion: With the largest sample to date of PWHB from Spain and Portugal, our understanding of the management and impact of hemophilia B is broadened and, in accordance with previous evidence, it shows the benefits of prophylaxis and the advantages that the FIX extended half life products life FIX can offer.
