von Mackensen S, Eldar-Lissai A, Auguste P, Krishnan S, von Maltzahn R, Yu R, Wyrwich K W
Institute of Medical Psychology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany.
Biogen, Cambridge, MA, USA.
Haemophilia. 2017 May;23(3):383-391. doi: 10.1111/hae.13140. Epub 2016 Dec 27.
Patients with haemophilia on long-acting prophylactic treatment may experience an improvement in health-related quality of life (HRQoL) through reductions in breakthrough bleeds and associated complications, including long-term joint damage, compared with episodic treatment.
This analysis examined clinical trial data to understand the psychometric characteristics (reliability, validity and sensitivity to change over time) of the Haem-A-QoL Questionnaire in adult males with haemophilia.
Two recent, multinational, Phase 3 clinical trials of new, long-acting factor concentrates (A-LONG: rFVIIIFc; B-LONG: rFIXFc) assessed HRQoL in adolescent and adult males with severe haemophilia A or B respectively. The adults' baseline assessments, via the 46-item Haem-A-QoL Questionnaire, and change over time at the 6-month assessment were used in the psychometric analyses.
Internal consistency reliability was adequate (Cronbach's alpha > 0.70) for nine of the 10 Haem-A-QoL domains and for 'Total Score' in both trials at baseline (A-LONG, n = 133; B-LONG, n = 73). At baseline, several Haem-A-QoL domains and 'Total Score' demonstrated known-groups and convergent validity when compared with other trial measures, including the EQ-5D (items and total scores) and joint impairment. Change score correlations (baseline to 28 weeks) between the EQ-5D and the Haem-A-QoL 'Total Score', and 'Physical Health' and 'Feelings' domains were moderate in magnitude (│r│ ≥ 0.33; P < 0.03), demonstrating sensitivity to change for these outcome measures in A-LONG.
These psychometric analyses provide evidence of the reliability, validity and ability to detect change of the Haem-A-QoL to assess the HRQoL of adult males with severe haemophilia A and B in longitudinal clinical trials.
与按需治疗相比，接受长效预防性治疗的血友病患者可能会因突破性出血及相关并发症（包括长期关节损伤）减少而使健康相关生活质量（HRQoL）得到改善。
本分析通过检查临床试验数据，了解血友病A-QoL问卷在成年男性血友病患者中的心理测量特征（可靠性、有效性和随时间变化的敏感性）。
两项近期的、多国的、新的长效因子浓缩物3期临床试验（A-LONG：重组人凝血因子VIII融合蛋白；B-LONG：重组人凝血因子IX融合蛋白）分别评估了重度血友病A或B的青少年和成年男性的HRQoL。心理测量分析采用成年人通过46项血友病A-QoL问卷进行的基线评估以及6个月评估时随时间的变化情况。
在两项试验的基线期（A-LONG，n = 133；B-LONG，n = 73），血友病A-QoL的10个领域中有9个以及“总分”的内部一致性可靠性良好（Cronbach's α>0.70）。在基线期，与其他试验指标（包括EQ-5D（项目和总分）和关节损伤）相比，血友病A-QoL的几个领域和“总分”显示出已知组效度和收敛效度。EQ-5D与血友病A-QoL“总分”以及“身体健康”和“情感”领域之间的变化分数相关性（基线至28周）中等（│r│≥0.33；P<0.03），表明A-LONG中这些结局指标对变化具有敏感性。
这些心理测量分析为血友病A-QoL在纵向临床试验中评估重度血友病A和B成年男性的HRQoL的可靠性、有效性和检测变化的能力提供了证据。
