Article

Development of a validated patient-reported symptom metric for pediatric Eosinophilic Esophagitis: qualitative methods

Division of Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
BMC Gastroenterology (Impact Factor: 2.11). 11/2011; 11:126. DOI: 10.1186/1471-230X-11-126
Source: PubMed

ABSTRACT Previous attempts to measure symptoms in pediatric Eosinophilic Esophagitis (EoE) have not fully included patients and parents in the item development process. We sought to identify and validate key patient self-reported and parent proxy-reported outcomes (PROs) specific to EoE.
We developed methodology for focus and cognitive interviews based on the Food and Drug Administration (FDA) guidelines for PROs, the validated generic PedsQL™ guidelines, and the consolidated criteria for reporting qualitative research (COREQ). Both child (ages 8-12 and 13-18) and parent-proxy (ages 2-4, 5-7, 8-12, and 13-18) interviews were conducted.
We conducted 75 interviews to construct the new instrument. Items were identified and developed from individual focus interviews, followed by cognitive interviews for face and content validation. Initial domains of symptom frequency and severity were developed, and open-ended questions were used to generate specific items during the focus interviews. Once developed, the instrument construct, instructions, timeframe, scoring, and specific items were systematically reviewed with a separate group of patients and their parents during the cognitive interviews.
To capture the full impact of pediatric EoE, both histologic findings and PROs need to be included as equally important outcome measures. We have developed the face and content validated Pediatric Eosinophilic Esophagitis Symptom Score (PEESS™ v2.0). The PEESS™ v2.0 metric is now undergoing multisite national field testing as the next iterative instrument development phase.

Download full-text

Full-text

Available from: Kevin Hommel, Aug 08, 2015
0 Followers
 · 
316 Views
  • [Show abstract] [Hide abstract]
    ABSTRACT: To present the clinical, endoscopic, and histologic features of eosinophilic esophagitis (EoE), review the current diagnostic guidelines for EoE, and present an approach for diagnosis of EoE. It will also highlight selected techniques that are under development that may be useful in the future for diagnosis of EoE. Recently updated guidelines emphasize that EoE is a clinicopathologic condition. Specifically, three criteria must be met to diagnose EoE: clinical symptoms of esophageal dysfunction; an esophageal biopsy with a maximum eosinophil count of at least 15 eosinophils per high-power microscopy field, with few exceptions; and exclusion of other possible causes of esophageal eosinophilia, including proton-pump inhibitor responsive esophageal eosinophilia (PPI-REE). A PPI trial is typically required both to assess for PPI-REE and to evaluate for the presence of concomitant gastroesophageal reflux disease. EoE is a chronic, immune-mediated disorder. Because no single symptom, endoscopic finding, or histopathologic feature is pathognomonic, diagnosis can be challenging. In the future, symptom scores, tissue or serum biomarkers, and genetic testing may play a role in diagnosis, but these methods have yet to be validated and are not yet recommended for routine clinical use.
    Current opinion in gastroenterology 03/2012; 28(4):382-8. DOI:10.1097/MOG.0b013e328352b5ef · 3.66 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Like many rare diseases, eosinophilic esophagitis (EoE) is a poorly understood disorder, and assessment tools to accurately determine disease activity, remission, and natural history have long been inadequate. Clinical outcome end points able to assess the effectiveness of candidate therapeutic agents in clinical trials have been a particular deficiency and are urgently needed. With no approved therapy available to patients and with the prevalence of EoE on the increase, collaborative approaches to drug development are becoming ever more important. We describe a collaborative effort mobilized across institutions, including both the public and private sectors, that was initiated within the past 18 months expressly to address the need for further clinical research into the cause and treatment of EoE. Collaborators include the North American Society of Pediatric Gastroenterology, Hepatology and Nutrition; the International Gastrointestinal Eosinophilic Researchers; and the US Food and Drug Administration. This effort has resulted in the elucidation of several parameters essential for effective EoE registration trials, including the need for clinically meaningful end points that measure changes in clinical symptoms in addition to the assessment of intraepithelial mucosal eosinophilia. The development and use of biomarkers, particularly in early-phase drug development, have become an important focus for investigations that might reduce clinical reliance on serial invasive monitoring. The concerted efforts described here to develop rational therapeutics and drug development paradigms in EoE also appear to provide a model for effective collaboration in the context of drug development for rare diseases and perhaps more generally for public health initiatives.
    The Journal of allergy and clinical immunology 07/2012; 130(3):613-6. DOI:10.1016/j.jaci.2012.07.011 · 11.25 Impact Factor
  • Source
    [Show abstract] [Hide abstract]
    ABSTRACT: Eosinophilic esophagitis (EoE) is recognized as a common, allergy-associated cause of chronic esophageal symptoms affecting both children and adults. Research has begun to shed light on its epidemiology with consistent results from various geographical areas. Differences in clinical presentation, endoscopic aspects and response to treatment have all been reported for patients of different ages, and the question as to whether adult and pediatric EoE are manifestations of a single entity or in fact two distinct disorders has been posed. The most relevant differences between pediatric and adult EoE come from evolutionary changes in the consequences of the disease, including fibrous remodeling, and the ability to express symptoms. However, most studies support a common pathogenesis and similar histopathological features for adult and pediatric patients, being the same diagnostic criteria applied to them. This article comprehensively reviews the most recently published information and addresses important questions about the natural history of EoE.
    Expert Review of Clinical Immunology 11/2012; 8(8):733-45. DOI:10.1586/eci.12.68 · 3.34 Impact Factor
Show more