In my introductory comments to the practice management section last year, I wrote about cultivating competencies for value-based care. One of the key competencies was patient centeredness. Patient-reported outcomes (PROs) and patient experience measures specifically were highlighted as examples of meaningful tools for achieving patient centeredness. Starting with this month’s contribution by Drs Reed and Dellon on PROs in esophageal disease, we begin a series of articles focused on this important construct. We will follow this article with reports focused on PRO for patients with irritable bowel syndrome, inflammatory bowel disease, and chronic liver disease. These reports will not only review the importance of PROs, but also highlight the most practical approaches to measuring disease-specific PROs in clinical practice all with the goal of improving the care of our patients.
Ziad Gellad, MD, MPH, AGAF, Special Section Editor
Patients seek medical care for symptoms affecting their quality of life,1 and this is particularly true of digestive diseases, in which many common conditions are symptom predominant. However, clinician and patient perception of symptoms often conflict,2 and formalized measurement tools may have a role for optimizing symptom assessment. Patient-reported outcomes (PROs) directly capture patients’ health status from their own perspectives and can bridge the divide between patient and provider interpretation. The US Food and Drug Administration (FDA) defines PROs as “any report of the status of a patient’s health condition that comes directly from the patient without interpretation of the patient’s response by a clinician or anyone else.”3
For the clinical assessment of esophageal diseases, existing physiologic and structural testing modalities cannot ascertain patient disease perception or measure the impact of symptoms on health care–associated quality of life. In contrast, by capturing patient-centric data, PROs can provide insight into the psychosocial aspects of patient disease perceptions; capture health-related quality of life (HRQL); improve provider understanding; highlight discordance between physiologic, symptom, and HRQL measures; and formalize follow-up evaluation of treatment response.1,4 Following up symptoms such as dysphagia or heartburn over time in a structured way allows clinically obtained data to be used in pragmatic or comparative effectiveness studies. PROs are now an integral part of the FDA’s drug approval process.
In this article, we review the available PROs capturing esophageal symptoms with a focus on dysphagia and heartburn measures that were developed with rigorous methodology; it is beyond the scope of this article to perform a thorough review of all upper gastrointestinal (GI) PROs or quality-of-life PROs. We then discuss how esophageal PROs may be incorporated into clinical practice now, as well as opportunities for PRO use in the future.
Esophageal symptom-specific patient-reported outcomes
The literature pertinent to upper GI and esophageal-specific PROs is heterogeneous, and the development of PROs has been variable in rigor. Two recent systematic reviews identified PROs pertinent to dysphagia and heartburn (Table 1) and both emphasized rigorous measures developed in accordance with FDA guidance.3
Patel et al5 identified 34 dysphagia-specific PRO measures, of which 10 were rigorously developed (Table 1). These measures encompassed multiple conditions including esophageal cancer (Functional Assessment of Cancer Therapy Esophageal Cancer Subscale, European Organization for Research and Treatment of Cancer Quality-of-Life with esophageal Cancer 25 items, European Organization for Research and Treatment of Cancer Quality-of-Life with esophageal cancer 18 items, upper aerodigestive neoplasm-attributable oropharyngeal dysphagia [M.D. Anderson dysphagia inventory], mechanical and neuromyogenic oropharyngeal dysphagia [swallow quality-of-life questionnaire], Sydney Swallow Questionnaire, [swallowing quality of care], achalasia [Measure of Achalasia Disease Severity], eosinophilic esophagitis [Dysphagia Symptom Questionnaire], and general dysphagia symptoms and gastroesophageal reflux [Patient-Reported Outcomes Measurement Information System Gastrointestinal Symptom Scales (PROMIS-GI)]. PROMIS-GI, produced as part of the National Institutes of Health PROMIS program, includes rigorous measures for general dysphagia symptoms and gastroesophageal reflux in addition to lower gastrointestinal symptom measures.
The systematic review by Vakil et al6 found 15 PRO measures for gastroesophageal reflux disease (GERD) symptoms that underwent psychometric evaluation (Table 1). Of these, 5 measures were devised according to the developmental steps stipulated by the US FDA and the European Medicines Agency, and each measure has been used as an end point for a clinical trial. The 5 measures include the GERD Symptom Assessment Scale, the Nocturnal Gastro-oesophageal Reflux Disease Symptom Severity and Impact Questionnaire, the Reflux Questionnaire, the Reflux Disease Questionnaire, and the Proton Pump Inhibitor Acid Suppression Symptom Test (Table 1). Additional PROs capturing esophageal symptoms include the eosinophilic esophagitis symptom activity index, Eckardt score (used for achalasia), Mayo dysphagia questionnaire, and GERD-Q (Table 1).