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Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial.
Santagostino, Elena; Martinowitz, Uri; Lissitchkov, Toshko; Pan-Petesch, Brigitte; Hanabusa, Hideji; Oldenburg, Johannes; Boggio, Lisa; Negrier, Claude; Pabinger, Ingrid; von Depka Prondzinski, Mario; Altisent, Carmen; Castaman, Giancarlo; Yamamoto, Koji; Álvarez-Roman, Maria-Teresa; Voigt, Christine; Blackman, Nicole; Jacobs, Iris.
Afiliación
  • Santagostino E; Istituto di Ricovero e Cura a Carattere Scientifico Ca' Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy;
  • Martinowitz U; The Israeli National Haemophilia Center, Chaim Sheba Medical Center, Tel Hashomer, Israel;
  • Lissitchkov T; Department of Coagulation Disorders and Anemia, Specialized Hospital for Active Treatment Joan Pavel, Sofia, Bulgaria;
  • Pan-Petesch B; Centre Hospitalier Régional Universitaire de Brest, Hôpital A. Morvan, Brest, France;
  • Hanabusa H; Department of Hematology, Ogikubo Hospital, Tokyo, Japan;
  • Oldenburg J; Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany;
  • Boggio L; Rush Hemophilia and Thrombophilia Center, Rush University, Chicago, IL;
  • Negrier C; Centre Régional de Traitement de l'Hémophilie, Hôpital Edouard Herriot, University Claude Bernard, Lyon, France;
  • Pabinger I; Division of Haematology and Haemostaseology, Medical University of Vienna, Vienna, Austria;
  • von Depka Prondzinski M; Werlhof Institut, Hannover, Germany;
  • Altisent C; Unitat Hemofilia, Hospital Vall d'Hebron, Barcelona, Spain;
  • Castaman G; Haemophilia and Thrombosis Centre, Department of Haematology, San Bortolo Hospital, Vicenza, Italy; Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy;
  • Yamamoto K; Nagoya University Hospital, Nagoya, Japan;
  • Álvarez-Roman MT; Hospital la Paz, Madrid, Spain; and.
  • Voigt C; Clinical Research and Development, CSL Behring, King of Prussia, PA.
  • Blackman N; Clinical Research and Development, CSL Behring, King of Prussia, PA.
  • Jacobs I; Clinical Research and Development, CSL Behring, King of Prussia, PA.
Blood ; 127(14): 1761-9, 2016 04 07.
Article en En | MEDLINE | ID: mdl-26755710
A global phase 3 study evaluated the pharmacokinetics, efficacy, and safety of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in 63 previously treated male patients (12-61 years) with severe hemophilia B (factor IX [FIX] activity ≤2%). The study included 2 groups: group 1 patients received routine prophylaxis once every 7 days for 26 weeks, followed by either 7-, 10-, or 14-day prophylaxis regimen for a mean of 50, 38, or 51 weeks, respectively; group 2 patients received on-demand treatment of bleeding episodes for 26 weeks and then switched to a 7-day prophylaxis regimen for a mean of 45 weeks. The mean terminal half-life of rIX-FP was 102 hours, 4.3-fold longer than previous FIX treatment. Patients maintained a mean trough of 20 and 12 IU/dL FIX activity on prophylaxis with rIX-FP 40 IU/kg weekly and 75 IU/kg every 2 weeks, respectively. There was 100% reduction in median annualized spontaneous bleeding rate (AsBR) and 100% resolution of target joints when subjects switched from on-demand to prophylaxis treatment with rIX-FP (P< .0001). The median AsBR was 0.00 for all prophylaxis regimens. Overall, 98.6% of bleeding episodes were treated successfully, including 93.6% that were treated with a single injection. No patient developed an inhibitor, and no safety concerns were identified. These results indicate rIX-FP is safe and effective for preventing and treating bleeding episodes in patients with hemophilia B at dosing regimens of 40 IU/kg weekly and 75 IU/kg every 2 weeks. This trial was registered at www.clinicaltrials.gov as #NCT0101496274.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Factor IX / Hemofilia B / Albúminas Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies Límite: Adolescent / Adult / Child / Humans / Male / Middle aged Idioma: En Revista: Blood Año: 2016 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Factor IX / Hemofilia B / Albúminas Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies Límite: Adolescent / Adult / Child / Humans / Male / Middle aged Idioma: En Revista: Blood Año: 2016 Tipo del documento: Article Pais de publicación: Estados Unidos