Epidemiology and clinical management of adult patients with severe hemophilia A without a history of FVIII inhibitors in Spain: results from a Delphi study
- 2024, Volume 43 - Number 2
- Published: October 25, 2024
Epidemiology and clinical management of adult patients with severe hemophilia A without a history of FVIII inhibitors in Spain: results from a Delphi study
Víctor Jiménez-Yuste 1, Nieves Alonso-Escobar 2, Olga Benítez-Hidalgo 3, Ángel Bernardo-Gutiérrez 4, Santiago Bonanad-Boix 5, M.ª Fernanda López-Fernández 6, Francisco J. López-Jaime 7, José Mateo-Arranz 8, Ramiro J. Núñez-Vázquez 9, Manuel Rodríguez-López 10, Ariadna Giró-Perafita 11, Erika Lorenzo-Vivas 12
1 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; 2 Hospital Universitario de Badajoz, Badajoz, España; 3 Hospital Universitari Vall d’Hebron, Barcelona, España; 4 Hospital Universitario Central de Asturias, Oviedo, España; 5 Hospital Universitario y Politécnico La Fe, Valencia, España; 6 Complejo Hospitalario Universitario A Coruña, A Coruña, España; 7 Hospital Regional Universitario de Málaga, Málaga, Spain; 8 Hospital de la Santa Creu i Sant Pau; Barcelona, España; 9 Hospital Universitario Virgen del Rocío, Sevilla, España; 10 Servicio de Hematología y Hemoterapia, Hospital Universitario Álvaro Cunqueiro, Instituto de Investigación Sanitaria Galicia-Sur (IISGS), EOXI, Vigo, España; 11 Omakase Consulting S.L, Barcelona, España; 12 BioMarin Pharmaceuticals España S.L. España
Víctor Jiménez-Yuste, Nieves Alonso-Escobar, Olga Benítez-Hidalgo, Ángel Bernardo-Gutiérrez, Santiago Bonanad-Boix, M.ª Fernanda López-Fernández, Francisco J. López-Jaime, José Mateo-Arranz, Ramiro J. Núñez-Vázquez, Manuel Rodríguez-López, Ariadna Giró-Perafita, Erika Lorenzo-Vivas
La información completa de afiliaciones y autor de correspondencia está disponible en la versión original en PDF.
*Correspondence: Ángel Bernardo-Gutiérrez, Email not available
Summary
Introduction: Hemophilia A (HA) is a rare X-linked bleeding disorder causing defective coagulation. Severe HA (sHA) results in spontaneous bleeds and degenerative joint disease. Objective: The objective of the study is to understand the epidemiology, disease burden, management, and unmet needs of adult patients with sHA without a history of factor VIII (FVIII) inhibitors. Methods: Ten leading hematologists participated in the Delphi study. A literature review was conducted to develop the first Delphi questionnaire. Responses were analyzed descriptively. Prevalence and incidence of sHA were estimated based on expert reported numbers and hospital population covered, extrapolated to Spain’s overall population. Results: Prevalence of adults with HA was estimated at 3081. Out of these, 1263 (41%, interquartile range [IQR] [28-46%]) were diagnosed with sHA and 998 (79%, IQR [71-88%]) would not have a history of FVIII inhibitors. Incidence of sHA was estimated in 43 patients per year. Most patients with sHA are treated with continuous prophylaxis (88%, IQR [75-97%]). About 20% IQR [7-41%] of patients with sHA treated with FVIII replacement therapy still experience spontaneous bleeds in many cases despite continuous prophylaxis. Conclusion: Prevalence of sHA in adult population in Spain was estimated at 0.265/10,000 inhabitants, which means 1263 adult patients with sHA in Spain, of which 998 (79%) would not have a history of FVIII inhibitors.
