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Blood. 2016 Apr 7;127(14):1761-9. doi: 10.1182/blood-2015-09-669234. Epub 2016 Jan 11.

Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial.

Author information

1
Istituto di Ricovero e Cura a Carattere Scientifico Ca' Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy;
2
The Israeli National Haemophilia Center, Chaim Sheba Medical Center, Tel Hashomer, Israel;
3
Department of Coagulation Disorders and Anemia, Specialized Hospital for Active Treatment Joan Pavel, Sofia, Bulgaria;
4
Centre Hospitalier Régional Universitaire de Brest, Hôpital A. Morvan, Brest, France;
5
Department of Hematology, Ogikubo Hospital, Tokyo, Japan;
6
Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany;
7
Rush Hemophilia and Thrombophilia Center, Rush University, Chicago, IL;
8
Centre Régional de Traitement de l'Hémophilie, Hôpital Edouard Herriot, University Claude Bernard, Lyon, France;
9
Division of Haematology and Haemostaseology, Medical University of Vienna, Vienna, Austria;
10
Werlhof Institut, Hannover, Germany;
11
Unitat Hemofilia, Hospital Vall d'Hebron, Barcelona, Spain;
12
Haemophilia and Thrombosis Centre, Department of Haematology, San Bortolo Hospital, Vicenza, Italy; Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy;
13
Nagoya University Hospital, Nagoya, Japan;
14
Hospital la Paz, Madrid, Spain; and.
15
Clinical Research and Development, CSL Behring, King of Prussia, PA.

Abstract

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.

PMID:
26755710
PMCID:
PMC4825413
DOI:
10.1182/blood-2015-09-669234
[Indexed for MEDLINE]
Free PMC Article

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