Patient-reported outcomes in esophageal diseases

Utilization of esophageal patient-reported outcomes in practice

Before incorporating a PRO into clinical practice, providers must appreciate the construct(s), intent, developmental measurement properties, validation strategies, and responsiveness characteristics associated with the measure.⁴ PROs can be symptom- and/or condition-specific. For example, this could include dysphagia associated with achalasia or eosinophilic esophagitis, postoperative dysphagia from spine surgery, or general dysphagia symptoms regardless of the etiology (Table 1). Intent refers to the context in which a PRO should be used and generally is stratified into 3 areas: population surveillance, individual patient-clinician interactions, and research studies.⁴ A thorough analysis of PRO developmental properties exceeds the scope of this article. However, several key considerations are worth discussing. Each measure should clearly delineate the construct, or outcome, in addition to the population used to create the measure (eg, patients with achalasia). PROs should be assessed for reliability, construct validity, and content validity. Reliability pertains to the degree in which scores are free from measurement error, the extent to which items (ie, questions) correlate, and test–retest reliability. Construct validity includes dimensionality (evidence of whether a single or multiple subscales exist in the measure), responsiveness to change (longitudinal validity), and convergent validity (correlation with additional construct-specific measures). Central to the PRO development process is the involvement of patients and content experts (content validity). PRO measures should be readily interpretable, and the handling of missing items should be stipulated. The burden, or time required for administering and scoring the instrument, and the reading level of the PRO need to be considered.⁸ In short, a PRO should measure something important to patients, in a way that patients can understand, and in a way that accurately reflects the underlying symptom and disease.

Although PROs traditionally represent a method for gathering data for research, they also should be viewed as a means of improving clinical care. The monitoring of change in a particular construct represents a common application of PROs in clinical practice. This helps quantify the efficacy of an intervention and can provide insight into the comparative effectiveness of alternative therapies. For example, in a patient with an esophageal stricture, a dysphagia-specific measure could be used at baseline before an endoscopy and dilation, in follow-up evaluation after dilation, and then as a monitoring tool to determine when repeat dilation may be needed. Similarly, the Eckardt score has been used commonly to monitor response to achalasia treatments. Clinicians also may use PROs in real time to optimize patient management. The data gathered from PROs may help triage patients into treatment pathways, trigger follow-up appointments, supply patient education prompts, and produce patient and provider alerts.⁸ For providers engaging in clinical research, PROs administered at the point of patient intake, whether electronically through a patient portal or in the clinic, provide a means of gathering baseline data.⁹ A key question, however, is whether it is practical to use a PRO routinely in the clinic, esophageal function laboratory, or endoscopy suite.

These practical issues include cultivating a conducive environment for PRO utilization, considering the burden of the measure on the patient, and utilization of the results in an expedient manner.⁹ To promote seamless use of a PRO in clinical work-flows, a multimodal means of collecting PRO data should be arranged. Electronic PROs available through a patient portal, designed with a user-friendly and intuitive interface, facilitate patient completion of PROs at their convenience, and ideally before a clinical or procedure visit. For patients without access to the internet, tablets and/or computer terminals within the office are convenient options. Nurses or clinic staff also could help patients complete a PRO during check-in for clinic, esophageal testing, or endoscopy. The burden a PRO imposes on patients also limits the utility of a measure. For instance, PROs with a small number of questions are more likely to be completed, while scales consisting of 30 of more items are infrequently finished. Clinicians also should consider how they plan to use the results of a PRO before implementing one; if the data will not be used, then the effort to implement and collect it will be wasted. Moreover, patients will anticipate that the time required to complete a PRO will translate to an impact on their management plan and will more readily complete additional PROs if previous measures expediently affected their care.⁹