Research Questions and Outcomes Prioritized by Patients With Dry Eye

Abstract

Importance: Dry eye is a common ocular surface condition with significant influence on patient quality of life and societal economic burden. There is an urgent need to prioritize new research for dry eye.

Objective: To identify and rank research questions and outcomes important to patients with dry eye.

Design, setting, and participants: This study was conducted using the following 6 steps: (1) identifying research questions from a previous survey of clinicians who treat patients with dry eye; (2) identifying outcomes from existing research (systematic reviews and their cited clinical trials in the Cochrane Eyes and Vision US Satellite database of eyes and vision reviews, and National Eye Institute-funded clinical trials registered on ClinicalTrials.gov) as of June 13, 2017; (3) identifying a sample of patients with dry eye from the email subscribers to the online newsletter KeratoScoop; (4) and (5) conducting a 2-round Delphi survey of those patients online in November and December 2017, respectively; and (6) designating and ranking questions and outcomes as important.

Main outcomes and measures: Importance assigned to research questions and outcomes for dry eye. A research question or outcome ranked by at least 75% of patients as 6 or higher on a scale of 0 to 10 was considered important.

Results: Among the 420 patients from 15 countries who completed both rounds of the Delphi survey, most were 60 years of age or older (233 [56%]), female (348 [83%]), white (393 [94%]), and of non-Hispanic ethnicity (398 [95%]). Among the 12 questions that clinicians had previously prioritized, patients rated 8 as important. The top 3 questions pertained to effectiveness of patient education, environmental modifications, and topical anti-inflammatory eye drops for dry eye. Among the 109 outcomes identified in existing research on dry eye, patients rated 26 as important. Ten of these 26 were unpopular in existing research, with fewer than 10% of 158 studies reporting these outcomes. Of the 10 most important outcomes, 9 were associated with symptoms or quality of life. The 3 outcomes rated most important by patients were ocular burning or stinging, ocular discomfort, and ocular pain.

Conclusions and relevance: This study identified research questions and outcomes important to patients with dry eye. A considerable gap was noted between outcomes in existing research on dry eye and outcomes patients consider important. Future research on dry eye should consider addressing the important research questions and outcomes identified herein, taking into account the patient perspective.

Figure 1.. Steps and Flow of Survey Participants, Research Questions, and Outcomes in This Study

NEI indicates National Eye Institute.

Figure 2.. Research Questions, Ratings of Importance, and Ranking in Round 1 of the Delphi Survey

IQR indicates interquartile range.

Figure 3.. Outcomes, Ratings of Importance, and Ranking in Round 2 of the Delphi Survey

IQR indicates interquartile range. ^a The 18 popular outcomes in existing research are ocular burning or stinging; ocular discomfort; ocular dryness; visual acuity; patient’s overall assessment of ocular surface symptoms; ocular foreign body sensation; tear film stability; ocular adverse events; artificial tear use; tear production or volume; corneal staining; nonocular adverse events; conjunctival hyperemia; ocular surface staining; conjunctival staining; conjunctival impression cytology; dryness of the mouth; and salivary flow. ^bThe 10 unpopular outcomes in existing research are ocular pain; influence of dry eye disease on patient’s daily life; vision-related quality of life; patient’s acceptability or satisfaction with treatment; ocular gritty or sandy sensation; overall assessment of treatment effectiveness assessed by patients; ocular tiredness or fatigue; photosensitivity or photophobia; intolerance to air drafts; and treatment cost.

Figure 4.. Preferred Outcome Measurement Periods for All 28 Outcomes Rated in Round 2 of the Delphi Survey

^aFor the 18 popular outcomes in existing research (ocular burning or stinging; ocular discomfort; ocular dryness; visual acuity; patient’s overall assessment of ocular surface symptoms; ocular foreign body sensation; tear film stability; ocular adverse events; artificial tear use; tear production or volume; corneal staining; nonocular adverse events; conjunctival hyperemia; ocular surface staining; conjunctival staining; conjunctival impression cytology; dryness of the mouth; and salivary flow), we obtained preferred measurement periods during Delphi round 1 (n = 622 patients). For each outcome, we allowed patients to indicate multiple measurement periods. ^bFor the 10 unpopular outcomes in existing research (ocular pain; influence of dry eye disease on patient’s daily life; vision-related quality of life; patient’s acceptability or satisfaction with treatment; ocular gritty or sandy sensation; overall assessment of treatment effectiveness assessed by patients; ocular tiredness or fatigue; photosensitivity or photophobia; intolerance to air drafts; and treatment cost), we obtained preferred measurement periods during Delphi round 2 (n = 420 patients). For each outcome, we allowed patients to indicate multiple measurement periods.