OMERACT working group develops mandatory set of domains for all Behçet syndrome trials
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An OMERACT working group has developed and approved a core set of five domains — disease activity, new organ involvement, quality of life, adverse events and death — that are mandatory for study in all Behçet syndrome trials.
“Although a number of trials have been conducted in [Behçet syndrome (BS)] with different agents, disease assessment has not been optimal,” Gülen Hatemi, MD, of Istanbul University, in Turkey, and colleagues wrote in Arthritis Care & Research. “There is a lack of standardized outcome measures that are widely accepted and standardly used in BS trials.”
“It has been problematic in the study of BS to compare results of randomized controlled trials with different agents or results of studies from different centers, combine results of studies with the same agent in meta-analyses, or combine datasets for additional analyses,” they added. “In summary, there are multiple challenges due to the heterogeneity of the disease that impede successful drug development.”
To develop a core set of reliable, validated and widely accepted outcome measures for all Behçet syndrome clinical trials, the Outcome Measures in Rheumatology Clinical Trials (OMERACT) Behçet syndrome Working Group launched an iterative, multi-stage, multi-year project. The majority of the working group were either members of the International Society for Behçet’s disease or patients with Behçet syndrome.
Overall, this effort included a systematic review, a survey among experts, an outcome measures focus group meeting involving all stakeholders, qualitative patient interviews, a Delphi exercise with physician experts from varying specialties and countries as well as patients and final endorsement via voting at the OMERACT 2018 meeting. A total of 111 participants who attended the OMERACT 2018 meeting voted, with 90.1% endorsing the final core set. According to the working group, Behçet syndrome trials often focus on specific manifestations and not the disease in its entirety. As such, their final core set includes five domains mandatory for study in all trials for the disease. These domains are disease activity, new organ involvement, quality of life, adverse events, and death.
There are also additional sub‐domains mandatory for study of specific organ‐systems. These include visual acuity, attack frequency, ocular severity and vascular leakage for the eye; tender and swollen joints for the musculoskeletal system; lesion amount and pain for the mucocutaneous system; vascular lesions, thrombophlebitis and post-thrombotic syndrome for the vascular system; clinical and endoscopic gastrointestinal activity for the gastrointestinal system; and central nervous system lesions, and cognitive and neurologic function for the central nervous system.
“This Core Set of domains includes an important difference from core sets previously developed for most other diseases such as rheumatoid arthritis or ANCA-associated vasculitis,” Hatemi and colleagues wrote. “Instead of a single domain set for use in all trials, there is a mandatory set of domains to be used in all trials of BS and separate sets of subdomains specific for each type of organ or system involvement for use in trials seeking to specifically assess that type of involvement.”
“This novel approach to domain selection addresses two key issues: i) a need to generate outcomes data comparable across all trials of BS; and ii) recognition that BS affects many different organ systems that are often studied separately and for which responses to treatments and the treatments themselves may differ,” they added. “Thus, the proposed Core Set provides a practical framework for harmonizing clinical trial designs in this multisystem disease.”
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