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Efficacy of Lifitegrast Ophthalmic Solution, 5.0%, in Patients with Moderate to Severe Dry Eye Disease: A Post Hoc Analysis of 2 Randomized Clinical Trials

Lookup NU author(s): Professor Francisco FigueiredoORCiD



This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).


© 2021 American Medical Association. All rights reserved.Importance: An investigation of the treatment effect of lifitegrast ophthalmic solution, 5.0%, in different subgroups by severity of dry eye disease (DED) seems warranted. Objective: To explore the heterogeneity across different subgroups of DED and identify which participants were most likely to achieve clinically meaningful benefit with lifitegrast treatment. Design, Setting, and Participants: This post hoc responder analysis was performed using the data from the phase 3 OPUS-2 and OPUS-3 studies, which were 12-week, prospective, double-masked, multicenter, placebo-controlled, randomized, parallel-arm clinical trials that previously demonstrated the efficacy of lifitegrast in DED. Pooled data were stratified into 4 subgroups based on severity of inferior corneal staining score (ICSS; ≤1.5 vs >1.5) and eye dryness score (EDS; <60 or ≥60) at baseline. Data were collected from December 7, 2012, to October 5, 2015, and post hoc analysis was performed from April 14, 2020, to July 30, 2021. Interventions: Lifitegrast or placebo twice daily for 84 days. Main Outcomes and Measures: Proportion of participants with (1) a clinically meaningful improvement in signs (ICSS or total corneal staining score [TCSS]) and symptoms (EDS or global visual analog scale [VAS]) and (2) a composite response for a given sign and symptom end point pair at day 84 were measured. Clinically meaningful improvement was defined as at least 30% improvement in symptoms (EDS or global VAS) and either at least a 1-point improvement in ICSS or at least a 3-point improvement in TCSS. For the composite responder analysis, the end point pairs were defined as at least a 30% reduction in EDS and at least a 1-point improvement in ICSS; at least a 30% reduction in EDS and at least a 3-point improvement in TCSS; at least a 30% improvement in global VAS and at least a 1-point improvement in ICSS; and at least a 30% improvement in global VAS and at least a 3-point improvement in TCSS. Results: In total, 1429 participants (716 in the placebo group and 713 in the lifitegrast group) were analyzed (1087 women [76.1%]; mean [SD] age, 58.7 [14.3] years). For the overall pooled population, responder and composite responder rates favored lifitegrast vs placebo (odds ratio range, 1.29 [95% CI, 1.05-1.59] to 2.10 [95% CI, 1.68-2.61]; P ≤.02). In the composite analysis, the subgroup with ICSS of greater than 1.5 and EDS of at least 60 at baseline (ie, moderate to severe DED) demonstrated a 1.70- to 2.11-fold higher odds of achieving clinically meaningful improvement with lifitegrast across all sign and symptom end point pairs (P ≤.001). Conclusions and Relevance: These post hoc findings suggest that lifitegrast ophthalmic solution, 5.0%, treatment may be associated with a response in participants with moderate to severe signs and symptoms of DED. Trial Registration: Identifier: NCT02284516.

Publication metadata

Author(s): Holland EJ, Jackson MA, Donnenfeld E, Piccolo R, Cohen A, Barabino S, Rolando M, Figueiredo FC

Publication type: Article

Publication status: Published

Journal: JAMA Ophthalmology

Year: 2021

Volume: 139

Issue: 11

Pages: 1200-1208

Online publication date: 07/10/2021

Acceptance date: 15/08/2021

Date deposited: 01/11/2021

ISSN (print): 2168-6165

ISSN (electronic): 2168-6173

Publisher: American Medical Association


DOI: 10.1001/jamaophthalmol.2021.3943


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Funder referenceFunder name
Novartis Pharma AG.