Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A: post hoc analysis of phase III trials using patient-reported outcomes

Priyanka Raheja; Nana Kragh; Linda Bystrická; Daniel Eriksson; Khaoula Aroui; Marwa Mezghani; Sylvaine Barbier; Silvia Linari

doi:10.1177/20406207241257917

Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A: post hoc analysis of phase III trials using patient-reported outcomes

Ther Adv Hematol. 2024 Jul 30:15:20406207241257917. doi: 10.1177/20406207241257917. eCollection 2024.

Authors

Priyanka Raheja¹, Nana Kragh², Linda Bystrická², Daniel Eriksson², Khaoula Aroui³, Marwa Mezghani³, Sylvaine Barbier⁴, Silvia Linari⁵

Affiliations

¹ Haematology Department, Haemophilia Centre, The Royal London Hospital, Barts Health NHS Trust, Whitechapel Road, London E1 1FR, UK.
² Sobi, Stockholm, Sweden.
³ Putnam, Les Berges du lac, Tunisia.
⁴ Putnam, Lyon, France.
⁵ Department of Oncology, Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy.

Abstract

Background: Hemophilia-associated bleeding and resultant joint pain and mobility restrictions can predispose patients to poor health-related quality of life (HRQoL). Therefore, efficacy of a treatment needs to address more than just annualized bleed rates.

Objectives: Describe the evolution of HRQoL, pain, and activity in patients with hemophilia A, treated with efmoroctocog alfa prophylaxis.

Design: A post hoc analysis from Kids A-LONG (NCT01458106), A-LONG (NCT01181128), and long-term extension study ASPIRE (NCT01454739) assessed change in pain and activity-related patient-reported outcomes (PROs).

Methods: Physical health, pain, and HRQoL were assessed by PROs for a cumulative treatment duration of up to ~6 years. The primary endpoint was change from baseline in EuroQoL (EQ)-5D and Haemophilia Quality of Life Questionnaire (Haem-A-QoL).

Results: 118 adult/adolescents and 71 pediatric patients were included. The proportion of adults and adolescents reporting no problem in the EQ-5D analysis of 'pain/discomfort' significantly increased from A-LONG baseline (35.04%; 41/117) to ASPIRE month 30 (44.68%; 21/47; p = 0.024). Mean (standard deviation) Haem-A-QoL subdomain scores for 'feeling' and 'physical health' at A-LONG baseline improved by -3.24 (15.13; p = 0.018) and -3.85 (23.07; p = 0.047), respectively, at study end. Proportion of pediatric patients reporting no problem on the EQ-5D analysis of 'pain/discomfort', significantly increased from A-LONG baseline (75.0%; 42/56) to ASPIRE baseline (95.56%; 43/45; p = 0.046). Satisfaction levels for pediatric patients were high at A-LONG baseline and maintained until study end.

Conclusion: Long-term efmoroctocog alfa prophylaxis reduces pain and improves HRQoL in adult and adolescent patients with hemophilia A. In pediatric patients, it reduces perceived pain and maintains satisfaction levels.

Trial registration: NCT01458106, NCT01181128, NCT01454739.

Keywords: hemophilia A; pain; patient-reported outcomes measures; quality of life; rFVIIIFc protein.

Associated data

ClinicalTrials.gov/NCT01458106
ClinicalTrials.gov/NCT01454739
ClinicalTrials.gov/NCT01181128