长期乙丙睾酮α预防治疗可改善血友病 A 患者的疼痛感、精神和身体健康:利用患者报告结果对 III 期试验进行事后分析

IF 3.4 3区 医学 Q2 HEMATOLOGY
Priyanka Raheja, Nana Kragh, Linda Bystrická, Daniel Eriksson, Khaoula Aroui, Marwa Mezghani, Sylvaine Barbier, Silvia Linari
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引用次数: 0

摘要

背景:血友病相关性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)低下。目的:描述接受易氟沙星α预防性治疗的 A 型血友病患者的 HRQoL、疼痛和活动能力的变化情况。设计:对儿童A-LONG(NCT01458106)、A-LONG(NCT01181128)和长期扩展研究ASPIRE(NCT01454739)进行事后分析,评估患者报告的疼痛和活动相关结果(PROs)的变化。方法:在长达约6年的累积治疗期间,通过PROs评估患者的身体健康、疼痛和HRQoL。结果:共纳入 118 名成人/青少年和 71 名儿童患者。从 A-LONG 基线(35.04%;41/117)到 ASPIRE 第 30 个月(44.68%;21/47;p = 0.024),在 EQ-5D 分析中报告 "疼痛/不适 "无问题的成人和青少年比例显著增加。研究结束时,A-LONG基线的 "感觉 "和 "身体健康 "的Haem-A-QoL子域平均分(标准差)分别提高了-3.24 (15.13; p = 0.018)和-3.85 (23.07; p = 0.047)。在 EQ-5D 分析的 "疼痛/不适 "项目中,报告没有问题的儿科患者比例从 A-LONG 基线(75.0%;42/56)到 ASPIRE 基线(95.56%;43/45;p = 0.046)显著增加。儿科患者的满意度在A-LONG基线时很高,并一直维持到研究结束。结论:长期易复方血塞通α预防治疗可减轻成人和青少年A型血友病患者的疼痛并改善其HRQoL,在儿科患者中可减轻疼痛感并维持满意度。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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
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.
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来源期刊
CiteScore
4.30
自引率
0.00%
发文量
54
审稿时长
7 weeks
期刊介绍: Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
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