Factor product utilization and health outcomes in patients with haemophilia A and B on extended half-life concentrates: A Canadian observational study of real-world outcomes

Haemophilia. 2021 Sep;27(5):751-759. doi: 10.1111/hae.14369. Epub 2021 Jun 23.

Abstract

Introduction: Recombinant factors VIII and IX Fc (rFVIIIFc/rFIXFc) became available in Canada in 2016 and were the only extended half-life (EHL) factor concentrates available in Canada until 2018.

Objectives: We aim to describe the change in product utilization in Canadians who switched to rFVIIIFc/rFIXFc.

Methods: This prospective and retrospective cohort study enrolled males aged ≥6 years with moderate or severe haemophilia who switched to rFVIIIFc/rFIXFc and those who remained on standard half-life (SHL) between 2016 and 2018. Factor utilization and annualized bleeding rates (ABR) were collected at baseline, 1-year and 2-years. Due to low prospective enrolment (n = 25 switchers), prospective and retrospective data were pooled.

Results: 125 switchers (93 rFVIIIFc, 32 rFIXFc) and 33 non-switchers were included. The median age was 17 (rFVIIIFc) and 38 years (rFIXFc). Prior to switch, over 80% were on prophylaxis. There was a statistically significant reduction in the prescribed weekly prophylactic dose after the switch to rFVIIIFc/rFIXFc for all age groups, with a corresponding reduction (15-16%) in actual annualized FIX utilization in switchers (combined adults and children) to rFIXFc, and a smaller non-significant reduction in actual annualized FVIIII utilization (7%) in children who switched to rFVIIIFc. A significant reduction in the median ABR was only observed in children who switched to rFVIIIFc, but not in adults who switched to rFVIIIFc or rFIXFc.

Conclusion: Switching from SHL to EHL products led to a small reduction in factor utilization, while preserving a low ABR in children and adults with haemophilia. Further patient-reported outcomes data will further elucidate the role of EHL in the haemophilia landscape.

Keywords: extended half-life; haemophilia A; haemophilia B; prophylaxis; utilization.

Publication types

  • Observational Study

MeSH terms

  • Adolescent
  • Adult
  • Canada
  • Child
  • Factor VIII / therapeutic use
  • Half-Life
  • Hemophilia A* / drug therapy
  • Humans
  • Male
  • Outcome Assessment, Health Care
  • Prospective Studies
  • Recombinant Fusion Proteins
  • Retrospective Studies

Substances

  • Recombinant Fusion Proteins
  • Factor VIII