Patient-reported outcomes (PROs) in rheumatology are not just personal lists of physical complaints or so-called “organ recitals.” In fact, PROs can both guide treatment decisions in daily practice and serve as key endpoints for clinical trials.
That’s the informed opinion of Clifton O. Bingham III, MD, director of the Johns Arthritis Center in Baltimore, Maryland, who discussed clinical and research applications of PROs at the 2024 Rheumatoid Arthritis Research Summit presented by the Arthritis Foundation and the Hospital for Special Surgery in New York City.
“Integrating PROs into practice settings can enhance the clinician’s ability to understand their patients and monitor disease impact, and they are increasingly available for clinical care and are being qualified for outcome measures for clinical trials,” Bingham said.
“I posit to you that some of this ability to better characterize things like anxiety and depression levels of patients more precisely may help us to identify those patients who are less likely to respond to therapy and may require different interventions than disease-modifying therapies for their disease,” he told the audience.
PRO Examples
The term PRO encompasses a broad range of measures that may include health-related quality of life measures, symptoms and their affects, patient satisfaction, and the patient’s experience with care.
PROs are important for rheumatology care and research because “we now have the capacity to make what we used to think were the subjective experiences of disease more objective. We now have ways that we can put numbers and measurements to the experiences that patients have about their illness and use that information as a way to understand more about the patients who are in front of us and also how their disease changes over time,” Bingham said.
Patients are the best — or in some cases, the only — judges of many aspects of their health, and they are best suited to report on certain events and outcomes, he said.
PROs that are currently included in core outcome measures used to guide care and in clinical trials in pain scores as reported by visual analog scales; functioning, as measured by the Health Assessment Questionnaire-Disability Index (HAQ-DI); and patient global assessment.
In international qualitative studies in which patients with rheumatoid arthritis (RA) were asked what was most important to them, the usual suspects of pain, function, and fatigue were routinely cited across the studies. But patients in studies from these groups (RAPP-PI, RAID, and OMERACT) also said that other factors important to their well-being included good sleep, enjoyment of life, independence, ability to participate in valued activities, and freedom from emotional distress, Bingham noted.
The Promise of PROMIS
The science of clinical measurement has advanced dramatically during his career, as Bingham said.
“There have been significant changes in the science behind how you develop and validate outcomes measures. The fields of clinimetrics and psychometrics have evolved substantially. These are now grounded in what we call ‘modern measurement’ approaches, which focus on item-response theory, constructing interval scales of measurement in things that are very precise in their ability to detect change over time,” he said.
One such measurement instrument is the Patient-Reported Outcome Measurement Information System (PROMIS®), developed at the National Institutes of Health using advanced measurement science.
The system, administered through either computer or paper questionnaires, is designed to improve precision of health-related quality of life assessments in multiple domains, including most domains identified by patients with RA. It uses a T-score metric standardized to the US population.
“You can use this in a disease like rheumatoid arthritis, and you can find out how patients are doing in reference to the normative United States populations,” he said.
Bingham noted that his team has “very good data” to show that PROMIS system significantly outperforms existing instruments such as the HAQ.
How It Works
The system uses item banks, each with multiple items. For example, there are approximately 150 items for the physical function assessment portion. All the items are scored along a continuum, “from people who are completely disabled to those who can run marathons,” Bingham said.
Each item on the scale has a question and response component, ranging from “are you able to get in and out of bed?” to “are you able to walk from one room to another?” to “are you able to run 5 miles?”
To evaluate the PROMIS scale, Bingham and colleagues looked at the distribution of PROMIS T-scores for 1029 patients with RA at their center. The scales showed that patients with RA have higher levels of pain, fatigue, and sleep disturbances, as well as worse physical function, than population norms.
Bingham and colleagues also evaluated the performance of the system in patients with active RA who were starting on or switching to a different disease-modifying antirheumatic drug (DMARD). As they reported in 2019, among 106 participants who completed the 12-week study, all PROMIS scores improved after DMARD initiation (P ≤ .05). In addition, except for the depression domain, changes in all assessed PROMIS measures correlated with changes in Clinical Disease Activity Index scores.
To see whether integrating PROs into routine clinics could have an effect on care, Bingham and colleagues conducted a prospective cohort study, which showed that with the additional patient-reported data, clinicians changed or adjusted RA treatment in 16%-19% of visits, identified new symptoms in 27%-38%, and suggested nonpharmacologic interventions in 4%-11%.
“This is information that’s being used, and it’s going into changing medical decision making,” he said.
Summarizing his work, Bingham told the audience “I hope that I have convinced you that patients with RA prioritize domains that are impacted by their disease. PROMIS measures are really state-of-the-science methods to evaluate multiple aspects of health-related quality of life, and what I’ll note to you is that these have been translated into multiple languages internationally. There are Spanish-language versions, there are Chinese language versions, there are versions for every country in the [European Union] that have been validated and can be used.”
It’s a Start
In the Q & A following the presentation, Daniel H. Solomon, MD, MPH, from Brigham and Women’s Hospital in Boston, commented that “the measurement issues and automating measurements seems like it’s a fundamental practice issue — how to manage the system and how to manage patients better, and I feel like we’re kind of scratching the surface.”
He said that artificial intelligence and PROs in clinic offer some promise for improving care but added that “we can do better than this. We can figure out better systems for measuring PROs: Having patients measure PROs, having patients tell us about their PROs so they don’t have to come in, or coming in only when they need to come in, when they’re really flaring. There are lots of innovative ways of thinking about these tools, and it feels like we’re kind of on the cusp of really taking advantage.”
Bingham’s work is supported by the Patient-Centered Outcomes Research Institute, National Institutes of Health, Ira T. Fine Discovery Fund, Johns Hopkins Arthritis Center Discovery Fund, Camille J. Morgan Arthritis Research and Education Fund, and Scheer Family Foundation and Joanne and John Rogers. He disclosed consulting for AbbVie, Janssen, Lilly, and Sanofi and serving as a board member of the PROMIS health organization, co-chair of the Omeract Technical Advisory Group, and member of the C-PATH RA PRO working group. Solomon had no relevant disclosures.
