Symptom-based patient-reported outcomes in adults with eosinophilic esophagitis: value for treatment monitoring and randomized controlled trial design.

Details

Serval ID
serval:BIB_FC69CBB375E7
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Institution
Title
Symptom-based patient-reported outcomes in adults with eosinophilic esophagitis: value for treatment monitoring and randomized controlled trial design.
Journal
Current opinion in allergy and clinical immunology
Author(s)
Safroneeva E., Schoepfer A.M.
ISSN
1473-6322 (Electronic)
ISSN-L
1473-6322
Publication state
Published
Issued date
04/2019
Peer-reviewed
Oui
Volume
19
Number
2
Pages
169-174
Language
english
Notes
Publication types: Journal Article ; Research Support, Non-U.S. Gov't ; Review
Publication Status: ppublish
Abstract
In adults with eosinophilic esophagitis (EoE), a chronic, inflammatory immune-mediated condition of the esophagus, both inflammation and fibrosis are likely associated with symptom generation. Therefore, assessing symptom-based patient-reported outcomes (PROs), defined by US Food and Drug Administration as 'any report of the status of a patient's health condition that comes directly from the patients, without interpretation of the patient's response by a clinician or anyone else', is important in the context of trials and observational studies of emerging therapies.
For purposes of treatment monitoring, lack of symptoms does not predict the absence of biologic inflammation; hence, endoscopy with esophageal biopsies should be performed to check for residual inflammation. Lack of inflammation does not predict lack of symptoms, and the presence of subepithelial fibrosis cannot be excluded. No published instrument currently measures the frequency of dysphagia described all possible ways, strategies of living with this symptom and various pain types. In randomized controlled trials, in which symptom response was detected using validated PRO measures, only modest decreases in symptom scores were observed.
Accessing full EoE symptom spectrum and optimizing PRO measures remains a challenge that should be tackled to reliably assess response to existing and emerging therapies.
Keywords
Adult, Deglutition Disorders, Eosinophilic Esophagitis/diagnosis, Eosinophilic Esophagitis/physiopathology, Humans, Monitoring, Physiologic/methods, Patient Reported Outcome Measures, Practice Guidelines as Topic, Randomized Controlled Trials as Topic, Surveys and Questionnaires, Treatment Outcome
Pubmed
Web of science
Create date
08/02/2019 13:57
Last modification date
05/04/2020 6:20
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