Murray Lindsey T, McCormack Julie, Grobeiu Ioana, Wiklund Ingela, Kimel Miriam, Van Nooten Floortje
Evidera, 7101 Wisconsin Avenue, Suite 1400, Bethesda, MD, 20814, USA.
Dompé farmaceutici S.p.A, Via Santa Lucia, 6, 20122, Milan, MI, Italy.
J Patient Rep Outcomes. 2020 May 4;4(1):30. doi: 10.1186/s41687-020-00192-y.
Neurotrophic keratopathy/keratitis (NK) is a rare disease of the cornea that can lead to anatomical loss of the eye. Little is known about the NK experience from the patients' perspective. The objectives of this study were to examine the symptomatic experience and impacts of NK on patients and assess the overall comprehension, relevance, and content validity of a new questionnaire.
This was a cross-sectional, qualitative study conducted with NK patients with varying levels of disease severity, recruited from one clinical site. One-on-one interviews using concept elicitation and cognitive interviewing techniques were conducted.
Fourteen NK patients participated; 64.3% were female (n = 9), mean age was 65.7 ± 13.3, and 14.3% (n = 2), 21.4% (n = 3), and 64.3% (n = 9) were classified as Mackie stage I, stage II, or stage III, respectively. Participants reported 24 concepts, including: redness (n = 12, 86%), sensitivity to light (n = 11, 79%), general discomfort (n = 9, 64%), dry eye (n = 9, 64%), reduced visual acuity (n = 9, 64%), blurred vision (n = 8, 57%), and eye fatigue (n = 8, 57%). No new concepts were reported after the 13th interview. The most frequently reported impacts included frustration (n = 10, 71%), driving impairment (n = 8, 57%), reading impairment (n = 7, 50%), difficulty watching television (n = 7, 50%), and concern with potentially losing their eyesight due to NK (n = 6, 43%). Participants provided positive feedback on the draft NK Questionnaire (NKQ) and felt that it was comprehensive and relevant to their experience with NK. Additionally, the recall period, instructions, item concepts, and response options were well-understood by participants. Minor revisions were made to the tool for consistency (i.e., the timeframe "in the past 7 days" was added to items 12-14); item 14 was modified to include "how often"; examples were added to item 9.
The results of the concept elicitation portion of the qualitative study support the content validity of the draft NKQ. The clinically significant concepts identified in the literature and raised during concept elicitation are included as items in the questionnaire. Further assessment of the psychometric properties should be conducted in support of this new tool to measure the effect of new treatments on symptoms and impacts associated with NK.
神经营养性角膜病变/角膜炎（NK）是一种罕见的角膜疾病，可导致眼球的解剖结构丧失。从患者角度对NK的了解甚少。本研究的目的是调查NK患者的症状体验及其对患者的影响，并评估一份新问卷的整体理解度、相关性和内容效度。
这是一项横断面定性研究，从一个临床地点招募了疾病严重程度不同的NK患者。采用概念激发和认知访谈技术进行一对一访谈。
14名NK患者参与研究；64.3%为女性（n = 9），平均年龄为65.7±13.3岁，14.3%（n = 2）、21.4%（n = 3）和64.3%（n = 9）的患者分别被归类为麦基I期、II期或III期。参与者报告了24个概念，包括：眼红（n = 12，86%）、畏光（n = 11，79%）、全身不适（n = 9，64%）、干眼（n = 9，64%）、视力下降（n = 9，64%）、视物模糊（n = 8，57%）和眼疲劳（n = 8，57%）。第13次访谈后未报告新的概念。最常报告的影响包括沮丧（n = 10，71%）、驾驶障碍（n = 8，57%）、阅读障碍（n = 7，50%）、看电视困难（n = 7，50%）以及担心因NK而可能失明（n = 6，43%）。参与者对NK问卷初稿（NKQ）给予了积极反馈，并认为它全面且与他们的NK经历相关。此外，参与者对回忆期、说明、项目概念和回答选项理解良好。对该工具进行了一些小的修订以保持一致性（例如，在第12 - 14项中添加了“在过去7天内”的时间范围）；第14项进行了修改以包括“频率如何”；第9项添加了示例。
定性研究的概念激发部分结果支持了NKQ初稿的内容效度。文献中确定并在概念激发过程中提出的具有临床意义的概念被纳入问卷项目。应进一步评估该工具的心理测量特性，以支持这一用于测量新治疗对与NK相关的症状和影响的新工具。
