Assessment of joint bleeding and target joints in patients with severe or moderately severe hemophilia B (factor IX ?2%) receiving prophylaxis with rIX-FP in the PROLONG-9FP clinical trial program
Objective: To assess the impact of prophylaxis with rIX-FP, a fusion protein linking recombinant factor IX (FIX) with human albumin, on joint outcomes. Methods: Joint outcomes were assessed in pediatric (<12 years) and adult/adolescent (?12 years) patients receiving rIX-FP prophylaxis every 7, 10...
| Autores: | , , , , , |
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| Tipo de recurso: | artículo |
| Fecha de publicación: | 2023 |
| País: | España |
| Institución: | Servizo Galego de Saúde (SERGAS) |
| Repositorio: | RUNA. Repositorio da Consellería de Sanidade e Sergas |
| OAI Identifier: | oai:runa.sergas.gal:20.500.11940/21106 |
| Acceso en línea: | https://portalcientifico.sergas.gal//documentos/64995bb571c692789f1e075f http://hdl.handle.net/20.500.11940/21106 |
| Access Level: | acceso abierto |
| Palabra clave: | Adult Adolescent Humans Child Factor IX Hemophilia B Recombinant Fusion Proteins Hemostasis Hemarthrosis Hemophilia A AS A Coruña CHUAC |
| Sumario: | Objective: To assess the impact of prophylaxis with rIX-FP, a fusion protein linking recombinant factor IX (FIX) with human albumin, on joint outcomes. Methods: Joint outcomes were assessed in pediatric (<12 years) and adult/adolescent (?12 years) patients receiving rIX-FP prophylaxis every 7, 10, or 14 days; patients (>18 years) well-controlled on a 14-day regimen could switch to a 21-day regimen. Target joints were defined as ?3 spontaneous bleeds into a single joint within a 6-month period. Results: For adult/adolescent (n = 63) and pediatric (n = 27) patients, median (Q1, Q3) annualized joint bleeding rate was 0.39 (0.00, 2.31), 0.80 (0.00, 2.85), 0.20 (0.00, 2.58), and 0.00 (0.00, 1.78) when treated with 7-, 10-, 14-, or 21-day prophylaxis. 50.0%, 38.9%, 45.5%, and 63.6% of adult/adolescent patients had no joint bleeds when treated with 7-, 10-, 14-, or 21-day prophylaxis, respectively, and 40.7%, 37.5%, and 37.5% of pediatric patients had no joint bleeds when treated with 7-, 10-, or 14-day prophylaxis. Ten adult and two pediatric patients developed target joints; all resolved by the end of the study. Conclusion: Prophylaxis with rIX-FP produced low joint bleeding rates and provided excellent hemostatic efficacy in the treatment of joint bleeds. All target joints reported resolved with rIX-FP prophylaxis. |
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