Comparability of European League Against Rheumatology-Recommended Pharmacological Treatments of Oral Ulcers Associated with Behçet's Disease: A Systematic Literature Review of Randomized Controlled Trials.

Oral ulcers are the cardinal manifestation in Behçet's disease (BD). The 2018 European League Against Rheumatism (EULAR) recommendations describe treatments for BD-associated oral ulcers with mucocutaneous involvement; however, little comparative effectiveness information for these agents is available. In the absence of head-to-head trials, an indirect treatment comparison (ITC) could provide useful evidence regarding comparative effectiveness of BD treatments. The purpose of this study was to conduct a comparative systematic literature review (SLR) and similarity assessment of randomized controlled trials (RCTs) investigating the oral ulcer-related efficacy outcomes of EULAR-recommended treatments for BD-associated oral ulcers to determine the feasibility of an ITC. An SLR was performed to identify relevant RCTs indexed in MEDLINE/Embase before May 29, 2019. RCT similarities for the ITC were assessed based on a step-wise process recommended by the International Society for Pharmacoeconomics and Outcomes Research. In total, 317 articles were identified, of which 14 RCTs, reflecting 11 EULAR-recommended treatments, were evaluated in a similarity assessment. Number of oral ulcers, resolution of oral ulcers, and healing time for oral ulcers were identified as the possible oral ulcer-related outcomes. After completing the similarity assessment of these outcomes, it was determined that a robust ITC was infeasible for the three oral ulcer-related outcomes due to heterogeneity in outcomes reporting, study design, and/or patient characteristics. More broadly, the results underscore the need for and consistent use of standardized measures for oral ulcer outcomes to facilitate comparative research. In the absence of head-to-head RCTs and infeasibility of quantitative ITC, comparative assessments for BD-associated oral ulcers are limited, including comparative effectiveness and cost-effectiveness evaluations. Healthcare decision-makers must continue to base treatment decisions on the extent and strength of available evidence (eg, robust RCTs), clinical guidelines, real-world experience, and patient considerations.

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© 2020 Nazareth et al.

Déclaration de conflit d'intérêts

Ms Nazareth was employed by Celgene Corporation at the time of the study. Ms Hart, Dr Ronnebaum, and Dr Patel are employees of Pharmerit - an OPEN Health Company, which was the recipient of consulting fees from Celgene and Amgen Inc. Dr Mehta was employed by Celgene Corporation at the time of the study. Prof Dr med Kötter has served as a speaker for AbbVie, Boehringer Ingelheim, Chugai, Eli Lilly, Medac, Merck Sharp & Dohme, Novartis, Pfizer, Roche, and Sanofi and reports participation in a clinical trial of apremilast vs placebo (RELIEF) for Celgene. The authors report no other potential conflicts of interest for this work.