背景:睡眠障碍,疼痛,和疲劳是轴性脊柱关节炎(axSpA)的主要症状/影响。三个定制的患者报告结果测量信息系统(PROMIS®)简短表格(睡眠障碍,疼痛干扰,和疲劳)已被提议用于axSpA以评估这些关键疾病概念。本研究旨在进一步了解axSpA的患者体验,并评估三种定制的PROMIS®ShortForms的内容有效性,以支持其在axSpA临床试验中的使用。
方法:非介入,横截面,定性(概念启发[CE]和认知汇报[CD])研究。参与者参加了90分钟的电话采访。CE部分使用开放式问题来引出有关axSpA症状和影响的信息。CD部分涉及“大声思考”练习,参与者宣读每个指令,item,和定制的PROMIS®简短表单的响应选项,并分享了他们的反馈。与会者还讨论了项目的相关性,响应选项和召回期。逐字访谈笔录要经过主题和内容分析。
结果:总计,有28名参与者(非放射学axSpA,n=12;强直性脊柱炎,n=16),来自美国(n=20)和德国(n=8)。平均年龄52.8岁,57%为男性;自诊断以来的平均时间为9.5年。CE部分确定了以axSpA为特征的12种不同症状:疼痛,睡眠问题,疲劳/疲倦,刚度,肿胀,视力/眼睛问题,限制身体运动,头痛/偏头痛,痉挛,姿势/身材的变化,平衡/协调问题,和麻木。疼痛,睡眠问题,≥90%的参与者经历了疲劳/疲劳,同时发生并使彼此恶化。参与者报告axSpA影响了他们的生活,涉及健康相关生活质量(HRQoL)的六个领域:身体功能(100%),情绪健康(89%),工作/志愿服务(79%),社会功能(75%),日常生活活动(61%)和认知功能(54%)。影响最常见的是疼痛,刚度,和疲劳。CD显示PROMIS®仪器在概念上是全面的,并且很好理解,所有项目与≥50%的参与者相关。
结论:疼痛,睡眠问题和疲劳是axSpA的主要症状,并与HRQoL影响相关.这些结果用于更新axSpA的概念模型,该模型最初是基于有针对性的文献综述而开发的。确认了定制的PROMIS®简短表单的可解释性和内容有效性,每个人都被认为充分评估了与axSpA相关的关键影响,使它们适用于axSpA临床试验。
Sleep disturbance, pain, and fatigue are key symptoms/impacts of axial spondyloarthritis (axSpA). Three customized Patient-Reported Outcomes Measurement Information System (PROMIS®) Short Forms (Sleep Disturbance, Pain Interference, and Fatigue) have been proposed for use in axSpA to assess these key disease concepts. This
study was designed to further understand the patient experience of axSpA and evaluate the content validity of the three customized PROMIS® Short Forms to support their use in axSpA clinical trials.
Non-interventional, cross-sectional, qualitative (concept elicitation [CE] and cognitive debriefing [CD])
study. Participants took part in 90-min telephone interviews. The CE section used open-ended questions to elicit information about axSpA symptoms and impacts. The CD section involved a \'think-aloud\' exercise where participants read out each instruction, item, and response option for the customized PROMIS® Short Forms and shared their feedback. Participants also discussed the relevance of the items, response options and recall period. Verbatim interview transcripts were subject to thematic and content analysis.
In total, there were 28 participants (non-radiographic axSpA, n = 12; ankylosing spondylitis, n = 16), from the US (n = 20) and Germany (n = 8). Mean age was 52.8 years, and 57% were male; mean time since diagnosis was 9.5 years. The CE section identified 12 distinct symptoms that characterized axSpA: pain, sleep problems, fatigue/tiredness, stiffness, swelling, vision/eye issues, restricted body movements, headache/migraine, spasms, change in posture/stature, balance/coordination problems, and numbness. Pain, sleep problems, and fatigue/tiredness were experienced by ≥ 90% of participants, occurring simultaneously and exacerbating one another. Participants reported axSpA impacted their lives across six domains of health-related quality of life (HRQoL): physical functioning (100%), emotional wellbeing (89%), work/volunteering (79%), social functioning (75%), activities of daily living (61%) and cognitive functioning (54%). Impacts were most frequently associated with pain, stiffness, and fatigue. CD showed the PROMIS® instruments were conceptually comprehensive and well understood, with all items relevant to ≥ 50% of participants.
Pain, sleep problems and fatigue are pivotal symptoms of axSpA and associated with HRQoL impacts. These results were used to update a conceptual model of axSpA which was originally developed based on a targeted literature review. Interpretability and content validity of the customized PROMIS® Short Forms were confirmed, with each deemed to adequately assess key impacts associated with axSpA, making them suitable for use in axSpA clinical trials.