Cross-sectional epidemiological study to understand the approach to treatment in the routine care of patients with hemophilia B in Spain and Portugal
- 2023, Volume 42 - Number 2
- Published: December 15, 2023
Cross-sectional epidemiological study to understand the approach to treatment in the routine care of patients with hemophilia B in Spain and Portugal
M.ª Teresa Álvarez-Román 1, Manuela Carvalho 2, Olga Benítez-Hidalgo 3, Cristina Catarino 4, Luis J. García-Frade 5, Manuel Rodríguez-López 6, Margarida Antunes 7, Nuria Fernández-Mosteirín 8, Paula Kjöllerström 9, Sara Morais 10, Bernat Galmes 11, María F. López-Fernández 12, Lluis Riera-Sans 13, Joana F. Rodrigues 14, Víctor Jiménez-Yuste 15, Ramón Salvado 16
1 Servicio de Hematología y Hemoterapia, Hospital Universitario La Paz, Madrid, España; 2 Congenital Coagulopathies Reference Centre, Centro Hospitalar Universitário São João, Porto, Portugal; 3 Hospital Universitari Vall d’Hebron, Barcelona, España; 4 Serviço de Imuno-hemoterapia, Centro Hospitalar e Universitário Lisboa Norte, Lisboa, Portugal; 5 Servicio de Hematología y Hemoterapia Hospital Universitario Río Hortega, Valladolid, España; 6 Servicio de Hematología y Hemoterapia, Hospital Universitario Álvaro Cunqueiro, Instituto de Investigación Sanitaria Galicia-Sur (IISGS), EOXI, Vigo, España; 7 Serviço de Imuno-hemoterapia, Centro Hospitalar Universitário Lisboa Central, Lisboa, Portugal; 8 Servicio de Hematología y Hemoterapia, Hospital Universitario Miguel Servet, Zaragoza, España; 9 Pediatric Hematology Unit, Centro Hospitalar Universitário de Lisboa Central, Lisboa, Portugal; 10 Thrombosis and Hemostasis Unit of Clinical Hematology Service, Centro Hospitalar Universitário do Porto, Porto, Portugal; 11 Hematología y Hemoterapia, Hospital Universitario Son Espases, Palma de Mallorca, España; 12 Servicio de Hematología, Hospital Universitario A Coruña, A Coruña, España; 13 CSL Behring, Barcelona, España; 14 CSL Behring Iberia, Lisboa, Portugal; 15 Servicio de Hematología y Hemoterapia, Hospital Universitario La Paz, Madrid, España Caso presentado en el simposio del Grupo Español de Citología Hematológica (GECH) en el LXVI Congreso Nacional SEHH-XL Congreso Nacional SETH; 16 Serviço de Imuno-Hemoterapia, Centro de Referência de Coagulopatias Congénitas, Centro Hospitalar e Universitário de Coimbra, Coimbra, Portugal
M.ª Teresa Álvarez-Román, Manuela Carvalho, Olga Benítez-Hidalgo, Cristina Catarino, Luis J. García-Frade, Manuel Rodríguez-López, Margarida Antunes, Nuria Fernández-Mosteirín, Paula Kjöllerström, Sara Morais, Bernat Galmes, María F. López-Fernández, Lluis Riera-Sans, Joana F. Rodrigues, Víctor Jiménez-Yuste, Ramón Salvado
La información completa de afiliaciones y autor de correspondencia está disponible en la versión original en PDF.
*Correspondence: M.ª Teresa Álvarez-Román. Email: talvarezroman@gmail.com
Summary
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.
Keywords: Hemophilia B. Spain. Portugal. Epidemiologic studies. Treatment outcome. Quality of life.
