Schmid-Mohler Gabriela, Caress Ann-Louise, Spirig Rebecca, Benden Christian, Yorke Janelle
Division of Nursing, Midwifery and Social Work, School of Health Sciences, University of Manchester, Manchester, England.
Centre of Clinical Nursing Science, University Hospital Zurich, Zurich, Switzerland.
Respir Care. 2018 Mar;63(3):353-366. doi: 10.4187/respcare.05638. Epub 2018 Jan 9.
Symptom burden increases during pulmonary exacerbations of cystic fibrosis (CF), and patient-reported outcome measures (PROMs) are often used to evaluate symptoms as either primary or secondary outcomes. However, there is currently no guidance on the use of PROMs to assess symptom burden during pulmonary exacerbations.
A systematic literature search was conducted to identify PROMs measuring symptom experience, management, or influencing factors, which were developed for CF patients and had been used at least once during pulmonary exacerbations. The PROMs included were assessed for relevance and psychometrics, according to the criteria of the United States FDA guideline and the Consensus-Based Standards for the Selection of Health Measurement Instruments (COSMIN) checklist.
Five PROMs were identified, all measuring symptom perception. The CF Respiratory Symptom Diary (CFRSD) and the Symptom Scoring System were developed to assess symptom severity during pulmonary exacerbations. Of the other 3, which also included symptom scores of 2 quality of life measures, one assessed symptom severity exclusively, and 2 measured symptom severity in addition to other dimensions (such as symptom distress). All 5 instruments measured respiratory symptoms. Other relevant symptoms, such as energy and emotions, were covered by 4 instruments; pain and gastrointestinal symptoms were covered by 2 measures. All of the instruments demonstrated good internal consistency and sensitivity to change over a period up to 4 weeks. The symptom scores of the 2 quality of life measures with longer recall periods are not suitable for measuring assessed changes in a period of < 2 weeks. Criterion validity for gastrointestinal subscores has not been established. Discriminant validity was established in all of the instruments reviewed except for the Symptom Score System.
Of the current PROMs used during CF pulmonary exacerbations, only 2 have been developed for this purpose, and only the CFRSD fulfilled all FDA guideline criteria. To date, there is no instrument that assesses exacerbation-specific symptom distress.
在囊性纤维化（CF）肺部加重期，症状负担会增加，患者报告结局测量指标（PROMs）常被用作评估症状的主要或次要结局。然而，目前尚无关于使用PROMs评估肺部加重期症状负担的指南。
进行系统的文献检索，以确定用于测量CF患者症状体验、管理或影响因素的PROMs，这些指标在肺部加重期至少使用过一次。根据美国食品药品监督管理局（FDA）指南标准和基于共识的健康测量工具选择标准（COSMIN）清单，对纳入的PROMs进行相关性和心理测量学评估。
共识别出5个PROMs，均用于测量症状感知。CF呼吸症状日记（CFRSD）和症状评分系统旨在评估肺部加重期的症状严重程度。其他3个指标（其中还包括2个生活质量测量指标的症状评分）中，一个仅评估症状严重程度，另外2个除其他维度（如症状困扰）外还测量症状严重程度。所有5种工具均测量呼吸症状。4种工具涵盖了其他相关症状，如精力和情绪；2种测量方法涵盖了疼痛和胃肠道症状。所有工具在长达4周的时间内均显示出良好的内部一致性和对变化的敏感性。回顾期较长的2个生活质量测量指标的症状评分不适用于测量2周内评估的变化。尚未确定胃肠道子评分的标准效度。除症状评分系统外，所有纳入审查的工具均建立了判别效度。
在CF肺部加重期目前使用的PROMs中，只有2个是为此目的而开发的，只有CFRSD符合所有FDA指南标准。迄今为止，尚无评估加重期特异性症状困扰的工具。
