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CC BY 4.0 · TH Open 2024; 08(01): e155-e163
DOI: 10.1055/s-0044-1781466
Original Article

Safety and Efficacy of Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) in Previously Untreated Patients with Hemophilia B

Richard Lemons
1   Department of Pediatrics and Primary Children's Hospital, University of Utah, Salt Lake City, Utah, United States
,
Michael Wang
2   Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Colorado, United States
,
Julie Curtin
3   The Children's Hospital at Westmead, New South Wales, Australia
,
Lynda Mae Lepatan
4   Department of Health Research and Pediatrics, Cebu Normal University—Vicente Sotto Memorial Medical Center College of Medicine, Cebu, Philippines
,
Christoph Male
5   Department of Paediatrics, Medical University of Vienna, Vienna, Austria
,
Flora Peyvandi
6   Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
7   Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
,
Mario von Depka Prondzinski
8   Werlhof Institute, Hannover, Germany
,
Rongrong Wang
9   CSL Behring, King of Prussia, Pennsylvania, United States
,
William McKeand
9   CSL Behring, King of Prussia, Pennsylvania, United States
,
Wilfried Seifert
10   CSL Behring, Marburg, Germany
,
Johannes Oldenburg
11   Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany
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Abstract

Introduction Recombinant fusion protein linking coagulation factor IX (FIX) with albumin (rIX-FP) has been shown to be an effective, well-tolerated treatment for patients with severe hemophilia B who had previously received factor replacement therapy. This study investigated the safety and efficacy of rIX-FP in previously untreated patients (PUPs).

Methods Patients with moderately severe/severe hemophilia B (≤2% FIX) previously untreated with FIX replacement products received rIX-FP (25–75 IU/kg) prophylaxis weekly or on-demand treatment over ≥50 exposure days (EDs). Primary outcomes were the number of patients who developed FIX inhibitors and mean incremental recovery (IR) following a 50 IU/kg dose of rIX-FP. Secondary outcomes included incidence of adverse events (AEs) and annualized bleeding rates (ABRs).

Results In total, 12 PUPs with a median age of 0 years (range, 0–11 years) were treated with rIX-FP for a median of 50 EDs (6/12 prophylaxis; 6/12 on-demand then prophylaxis). Overall, 11/12 patients did not develop FIX inhibitors; one 11-year-old patient developed an inhibitor against FIX after 8 EDs and was ultimately withdrawn. Mean (standard deviation) IR was 1.2 (0.4, n = 8) (IU/dL)/(IU/kg). Of the 137 treatment-emergent AEs recorded, five were attributed to rIX-FP. On the prophylaxis regimen, median ABR was 1.0 (range, 0–3.9, n = 12). No thromboembolic events or deaths occurred during the study.

Conclusion This study provides data to support the safety and efficacy of rIX-FP in PUPs requiring on-demand or prophylactic treatment for moderately severe/severe hemophilia B, consistent with results in previously treated patients. Overall, 1/12 patients developed an inhibitor against FIX.

Keywords

factor IX - hemophilia B - pediatric - previously untreated patients - prophylaxis

Supplementary Material



Publication History

Received: 14 August 2023

Accepted: 31 January 2024

Article published online:
26 March 2024

© 2024. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution License, permitting unrestricted use, distribution, and reproduction so long as the original work is properly cited. (https://creativecommons.org/licenses/by/4.0/)

Georg Thieme Verlag KG
Rüdigerstraße 14, 70469 Stuttgart, Germany

 

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