Core Outcome Measures in Effectiveness Trials

Defining core outcome set in adults with eosinophilic esophagitis

General Information

Summary:
Eosinophilic esophagitis (EoE) is a chronic, immune-mediated condition with a current prevalence of about 1 / 2,000 inhabitants in industrialized countries. Untreated eosinophil-predominant inflammation leads to esophageal remodeling with stricture formation and consecutive food bolus impactions with the inherent risks of esophageal perforation. The optimal long-term therapeutic strategy still needs to be defined. To date, no specific therapies for EoE management have been approved by regulatory authorities in United States and Europe. Furthermore, different stakeholders do not come to an agreement on the therapeutic endpoints to be assessed in clinical trials and observational studies.
“Defining clinically meaningful therapeutic endpoints in EoE” study is a feasibility study that includes the development of a core outcome set for use in future trials of various therapies for management of EoE in adult patients.

Contributors:
Alain M. Schoepfer, MD1* (principal investigator), Alex Straumann, MD, Ekaterina Safroneeva, PhD, Yvonne Romero, MD, Jeffrey A. Alexander, MD, David A. Katzka, MD, Ikuo Hirano, MD, Nirmala Gonsalves, MD, Glenn T. Furuta, MD, Evan S. Dellon, MD, John Leung, MD, Margaret H. Collins, MD, Christian Bussmann, MD, Sandeep K. Gupta, MD, Seema S. Aceves, MD, PhD, Mirna Chehade, MD, Fouad J. Moawad, MD, Tiffany H. Taft, PsyD, Robert O. Newbury, MD, Thomas C. Smyrk, MD, John T. Woosley, MD, Guang-Yu Yang, MD, PhD.

1 Division of Gastroenterology and Hepatology, Centre Hospitalier Universitaire Vaudois / CHUV, Lausanne, Switzerland

Further Study Information

Current Stage:
Ongoing
Date:
November 2015 - January 2018
Funding source(s):
Swiss National Science Foundation

Health Area

Disease Category
Gastroenterology

Disease Name
Eosinophilic esophagitis

Target Population

Age Range
18 - 100

Sex
Either


Nature / type of Intervention
Diet and nutrition
Drug
Procedure

Method(s)

Consensus meeting
Delphi process
Focus group(s)
Interview
Literature review
Survey

This study will include a systematic review of the literature to identify a list of outcomes that have previously been reported in various clinical trials and prospective observational studies. This list of outcomes will be used in a Delphi study with clinicians, including gastroenterologists, allergists and pathologist. The Delphi study is anticipated to include three rounds. In the first round, authors of the study will ask clinicians to score the outcome list and to add any outcomes they consider to be relevant. In the second round, scores will be presented to a given stakeholder group, and the participants will have the opportunity to rescore outcomes. To define patient needs, surveys, focus groups, and semi-structured interviews will be conducted in parallel. In the final round of the Delphi process, participants will be shown the distribution of scores for each outcome as well as a summary of the results of the qualitative analysis of data obtained by the means of survey/focus groups/ interviews with patients. A final consensus meeting will conducted with all stakeholders, including patients, reviewing outcomes to be recommended as “core outcome set”.


Stakeholders Involved

Clinical experts
Consumers (patients)
Regulatory agency representatives

Study Type

COS for clinical trials or clinical research
COS for practice

The site uses cookies, some may have been set already. Please refer to our privacy policy & cookie usage statement.
If you continue to use the site we'll assume you're happy to accept the cookies.