Evidence for the Use of Patient-Reported Outcome Measures in the Treatment of Patients With Noncommunicable Diseases: Systematic Review.

Abstract

Background: The use of patient-reported outcome measures (PROMs) as a clinical tool for screening and decision-making has gained widespread interest, with numerous implementation activities across specialties, even though the evidence has not been clear until now.

Objective: The aim of this study was to assess the evidence for using PROMs in clinical practice for patients with diabetes, chronic obstructive pulmonary disease (COPD), heart disease, rheumatoid arthritis (RA), and inflammatory bowel disease (IBD). Additionally, we sought to determine the characteristics of the most effective PROM interventions.

Methods: We conducted a systematic review of published randomized controlled trials (RCTs) on the use of PROMs for clinical purposes, such as systematic PROM assessment alone or with a predefined PROM-based decision-making method. Eligible studies included adult patients (>18 years) with diabetes, COPD, heart disease, RA, or IBD. We excluded studies using PROMs as an outcome measure or otherwise not meeting the inclusion criteria. We searched the PubMed/MEDLINE, CINAHL, EMBASE, and Web of Science databases until February 2023. Two investigators independently screened titles, abstracts, and relevant full texts. Three investigators completed data extraction and risk-of-bias assessment using version 2 of the Cochrane risk-of-bias tool for randomized trials (RoB 2). The data were presented in a narrative synthesis and in summarized form.

Results: The search yielded 21,203 papers, 686 (3.2%) full-text papers were screened, and 56 (8.2%) original studies were included in the review. The studies included patients with heart disease (n=17, 30.4%), COPD (n=13, 23.2%), diabetes (n=10, 17.9%), IBD (n=9, 16.1%), and RA (n=6, 10.7%), as well as patients with mixed diagnoses (n=1, 1.8%). All interventions incorporated systematic PROM assessments. Some interventions additionally used a predefined method for PROM-based decision-making (n=19, 33.9%) or PROM-based dialogue (n=9, 16.1%), while 5 (8.9%) interventions aimed to substitute face-to-face consultations. The predominant mode of PROM administration was over the phone, followed by electronic devices and apps. Endpoints included disease activity, health care use, mortality, mental well-being, quality of life, self-efficacy, self-care, daily functioning, and other outcomes. Six studies with a low risk of bias demonstrated a positive effect or noninferiority of the PROM intervention.

Conclusions: The evidence base for clinical use of PROMs is sparse, with few studies evaluated to have a low or a medium risk of bias. The clinical use of PROMs does not appear superior to usual care in the five included chronic diseases on any endpoint. To guide further research, we highlighted 6 (10.7%) studies with a low risk of bias and PROM interventions with a positive effect. These were characterized by symptom assessment with predefined cutoffs used for decision and dialogue support.

Trial registration: PROSPERO CRD42021226896; https://www.crd.york.ac.uk/PROSPERO/view/CRD42021226896.