Recombinant factor IX Fc prophylaxis reduces pain and increases levels of physical activity, with sustained, long-term improvements in patients with hemophilia B: analysis of phase III trials using patient-reported outcomes.

Astermark, Jan;Hermans, Cédric;Ezzalfani, Monia;Sidhom, Alaeddine;Eriksson, Daniel;et.al.
(2023) Therapeutic Advances in Hematology — Vol. 14, p. 1-13 (2023)

Files

2013_Astermark_Ther_Adv_Hematol.pdf
  • Open Access
  • Adobe PDF
  • 2.9 MB
Download

Details

Authors
  • Astermark, Jan
    Author
  • Hermans, CédricUCLouvain
    Author
  • Ezzalfani, Monia
    Author
  • Sidhom, Alaeddine
    Author
  • Eriksson, Daniel
    Author
Show more
Abstract
BACKGROUND: Pain is a common symptom of hemophilia that may adversely affect patients' quality of life (QoL). Previous post hoc analyses of prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) have been published for adults and adolescents, demonstrating improvements in health-related QoL (HRQoL) when assessed by the haemophilia-specific QoL (HaemAQoL) questionnaire. OBJECTIVE: To describe in depth the evolution of QoL, pain- and activity-related domains and questions for pediatric, adolescent, and adult patients with hemophilia B treated with rFIXFc prophylaxis. DESIGN: A post hoc analysis of data from a series of clinical trials. METHODS: This post hoc, long-term analysis assessed patient-reported outcomes (PROs) from the Kids B-LONG (NCT01440946: pediatric) and B-LONG (NCT01027364: adults and adolescents) parent studies and the B-YOND (NCT01425723: all age groups) extension study. RESULTS: Ninety-two adult and adolescent patients that started in the B-LONG study were assessed, with a median (range) duration of follow-up of 58.9 (0.0-78.4) months. The Haem-A-QoL total score was significantly reduced from baseline by 4.45 (p ⩽ 0.01), as were the subdomains 'physical health' (9.10; p = 0.001), 'sports and leisure' (11.25; p ⩽ 0.01), 'treatment' (2.69; p = 0.05), and 'view of self' (5.81; p = 0.002). Thirty pediatric patients that started in the Kids B-LONG study were assessed, with a median (min-max) duration of follow-up of 36.7 (9.0-59.9) months. The high level of satisfaction demonstrated by the PROs at baseline was maintained. CONCLUSION: rFIXFc prophylaxis reduced perceived pain and increased levels of physical activity with sustained, long-term improvements in QoL in adult and adolescent patients with hemophilia B and maintained high QoL scores in pediatric patients.
Affiliations

Citations

  • APA
  • Chicago
  • FWB

Astermark, J., Hermans, C., Ezzalfani, M., Sidhom, A., Barbier, S., Kragh, N., Falk, A., & Eriksson, D. (2023). Recombinant factor IX Fc prophylaxis reduces pain and increases levels of physical activity, with sustained, long-term improvements in patients with hemophilia B: analysis of phase III trials using patient-reported outcomes. Therapeutic Advances in Hematology, 14, 1-13. https://doi.org/10.1177/20406207231170701 (Original work published 2023)