Assessment of Real-World Patient-Reported Outcomes in Patients Initiating Biologic Agents for the Treatment of Autoimmune Diseases: An Observational Study in Four Patient-Powered Research Networks

Abstract

Timothy Beukelman,1 Millie D Long,2 Rennie L Rhee,3 Michael D Kappelman,4 Peter A Merkel,3,5 William Benjamin Nowell,6 Cassie Clinton,7 Sarah Ringold,8 Vincent Del Gaizo,9 Brian Price,10 Dianne G Shaw,11 Shilpa Venkatachalam,6 David Cuthbertson,12 Fenglong Xie,7 Xian Zhang,4 Jeffrey R Curtis7 1Department of Pediatrics, Division of Rheumatology, University of Alabama at Birmingham, Birmingham, AL, USA; 2Department of Medicine, Division of Gastroenterology and Hepatology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; 3Department of Medicine, Division of Rheumatology, University of Pennsylvania, Philadelphia, PA, USA; 4Department of Pediatrics, Division of Pediatric Gastroenterology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; 5Department of Biostatistics, Epidemiology, and Informatics; Division of Epidemiology, University of Pennsylvania, Philadelphia, PA, USA; 6Global Healthy Living Foundation, Upper Nyack, NY, USA; 7Department of Medicine, Division of Clinical Immunology and Rheumatology, University of Alabama at Birmingham, Birmingham, AL, USA; 8Department of Pediatrics, Division of Rheumatology, Seattle Children’s Hospital, Seattle, WA, USA; 9Childhood Arthritis and Rheumatology Research Alliance (CARRA) and PARTNERS PPRN, Milwaukee, WI, USA; 10Crohn’s and Colitis Foundation and IBD-Partners, New York, NY, USA; 11Vasculitis Patient Powered Research Network, Kansas City, MO, USA; 12Health Informatics Institute, University of South Florida, Tampa, FL, USACorrespondence: Timothy Beukelman, Email [email protected]: The most reliable and meaningful approach for inclusion of patient-reported outcomes (PROs) in the evaluation of real-world clinical effectiveness of biologics in the treatment of autoimmune diseases is u ncertain. This study aimed to assess and compare the proportions of patients who had abnormalities in PROs measuring important general health domains at the initiation of treatment with biologics, as well as the effects of baseline abnormalities on subsequent improvement.Methods: PROs were collected for patient participants with inflammatory arthritis, inflammatory bowel disease, and vasculitis using Patient-Reported Outcomes Measurement Information System instruments. Scores were reported as T-scores normalized to the general population in the United States. Baseline PROs scores were collected near the time of biologic initiation, and follow-up scores were collected 3 to 8 months later. In addition to summary statistics, the proportion of patients with PROs abnormalities (scores ≥ 5 units worse than the population norm) was determined. Baseline and follow-up scores were compared, and an improvement of ≥ 5 units was considered significant.Results: There was wide variation across autoimmune diseases in baseline PROs scores for all domains. For example, the proportion of participants with abnormal baseline pain interference scores ranged from 52% to 93%. When restricted to participants with baseline PROs abnormalities, the proportion of participants experiencing an improvement of ≥ 5 units was substantially higher.Conclusion: As expected, many patients experienced improvement in PROs following initiation of treatment with biologics for autoimmune diseases. Nevertheless, a substantial proportion of participants did not exhibit abnormalities in all PROs domains at baseline, and these participants appear less likely to experience improvement. For PROs to be reliably and meaningfully included in the evaluation of real-world medication effectiveness, more knowledge and careful consideration are needed to select the most appropriate patient populations and subgroups for inclusion and evaluation in studies measuring change in PROs.Keywords: patient-reported outcome measures, patient outcome assessment, biological therapy, autoimmune diseases

Keywords

• patient reported outcome measures
• patient outcome assessment
• biological therapy
• autoimmune diseases