Immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A: interim results from a phase 3, prospective, multicenter, open-label study

Sidonio RF 1 , Thompson AA 2 , Peyvandi F 3 , Stasyshyn O 4 , Yeoh SL 5 , Sosothikul D 6 Show all authors , Antmen AB 7 , Maggiore C 8 , Engl W 9 , Ewenstein B 10 , Tangada S 10

Affiliations 

  • 1 Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, GA, USA
  • 2 Division of Hematology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA
  • 3 Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre and Fondazione Luigi Villa, Milan, Italy
  • 4 Academy of Medical Sciences of Ukraine, Lviv, Ukraine
  • 5 Department of Pediatrics, Hospital Pulau Pinang, George Town, Malaysia
  • 6 Department of Pediatrics, Faculty of Medicine, Chulalongkorn University, Bangkok, Thailand
  • 7 Department of Pediatric Hematology and Oncology, Acibadem Adana Hospital, Adana, Turkey
  • 8 IQVIA, Milan, Italy
  • 9 Pharmacometrics & Biostatistics, Baxalta Innovations GmbH, A Takeda Company, Vienna, Austria
  • 10 Takeda Development Center Americas, Inc., Cambridge, MA, USA
Expert Rev Hematol, 2023;16(10):793-801.
PMID: 37646148 DOI: 10.1080/17474086.2023.2247160

Abstract

AIM: To determine the immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients (PUPs) with severe hemophilia A (HA).

METHODS: This prospective, phase 3 study (NCT02615691) was conducted in PUPs, or patients with ≤2 exposure days (EDs) prior to screening, aged <6 years with severe HA. The primary endpoint was incidence of factor VIII (FVIII) inhibitor development. This protocol-specified interim analysis was conducted after 50 patients had completed ≥50 EDs without developing FVIII inhibitors or had developed a confirmed inhibitor at any time.

RESULTS: Of the enrolled patients, 59/80 (73.8%) received ≥1 dose of rurioctocog alfa pegol; 54 received prophylaxis, and 35 on-demand treatment. Incidence of inhibitor development was 0.19 (10/52). Total annualized bleeding rate (95% CIs) was 3.2 (2.0-5.0) for patients receiving prophylaxis and 3.2 (1.6-6.3) for on-demand treatment. Hemostatic efficacy of most bleedings was rated as 'excellent' or 'good' after 24 hours (122/131 [93.1%]) and at resolution (161/170 [94.7%]). Five patients received ≥1 dose of rurioctocog alfa pegol for immune tolerance induction (ITI) and 1 patient was defined as having ITI success. Thirteen patients experienced 14 treatment-related adverse events, including 10 cases of FVIII inhibitor development.

CONCLUSION: This is the first prospective study of rurioctocog alfa pegol for the treatment of PUPs with severe HA.

TRIAL REGISTRATION: This trial is registered at ClinicalTrials.gov (CT.gov identifier: NCT02615691).

* Title and MeSH Headings from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.

MeSH terms
Similar publications