Raheja Priyanka, Kragh Nana, Bystrická Linda, Eriksson Daniel, Aroui Khaoula, Mezghani Marwa, Barbier Sylvaine, Linari Silvia
Haematology Department, Haemophilia Centre, The Royal London Hospital, Barts Health NHS Trust, Whitechapel Road, London E1 1FR, UK.
Sobi, Stockholm, Sweden.
Ther Adv Hematol. 2024 Jul 30;15:20406207241257917. doi: 10.1177/20406207241257917. eCollection 2024.
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.
Describe the evolution of HRQoL, pain, and activity in patients with hemophilia A, treated with efmoroctocog alfa prophylaxis.
A 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).
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).
118 adult/adolescents and 71 pediatric patients were included. The proportion of adults and adolescents reporting no problem in the EQ-5D analysis of '' significantly increased from A-LONG baseline (35.04%; 41/117) to ASPIRE month 30 (44.68%; 21/47; = 0.024). Mean (standard deviation) Haem-A-QoL subdomain scores for '' and '' at A-LONG baseline improved by -3.24 (15.13; = 0.018) and -3.85 (23.07; = 0.047), respectively, at study end. Proportion of pediatric patients reporting no problem on the EQ-5D analysis of '', significantly increased from A-LONG baseline (75.0%; 42/56) to ASPIRE baseline (95.56%; 43/45; = 0.046). Satisfaction levels for pediatric patients were high at A-LONG baseline and maintained until study end.
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.
NCT01458106, NCT01181128, NCT01454739.
血友病相关的出血以及由此导致的关节疼痛和活动受限会使患者的健康相关生活质量（HRQoL）较差。因此，一种治疗方法的疗效需要解决的不仅仅是年化出血率问题。
描述接受阿法依库凝血因子预防治疗的甲型血友病患者的健康相关生活质量、疼痛和活动情况的演变。
对儿童A-LONG（NCT01458106）、A-LONG（NCT01181128）以及长期扩展研究ASPIRE（NCT01454739）进行的一项分析评估了与疼痛和活动相关的患者报告结局（PROs）的变化。
通过PROs评估身体健康、疼痛和健康相关生活质量，累计治疗时长最长可达约6年。主要终点是欧洲五维健康量表（EQ）-5D和血友病生活质量问卷（Haem-A-QoL）相对于基线的变化。
纳入了118名成人/青少年和71名儿科患者。在EQ-5D分析中报告“无问题”的成人和青少年比例从A-LONG基线时的35.04%（41/117）显著增至ASPIRE第30个月时的44.68%（21/47；P = 0.024）。在研究结束时，A-LONG基线时“身体功能”和“日常活动”的Haem-A-QoL子域平均（标准差）得分分别改善了-3.24（15.13；P = 0.018）和-3.85（23.07；P = 0.047）。在EQ-5D分析中报告“无问题”的儿科患者比例从A-LONG基线时的75.0%（42/56）显著增至ASPIRE基线时的95.56%（43/45；P = 0.046）。儿科患者在A-LONG基线时的满意度较高，并一直维持到研究结束。
长期阿法依库凝血因子预防治疗可减轻成年和青少年甲型血友病患者的疼痛并改善其健康相关生活质量。对于儿科患者，它可减轻感知到的疼痛并维持满意度水平。
NCT01458106、NCT01181128、NCT01454739。
