Digital tool better at capturing day-to-day variability in Sjögren’s symptoms
PEPSS may represent more sensitive way to assess changes, treatment effects
A web-based tool to assess daily symptoms of Sjögren’s disease is easy to implement, was well accepted, and effectively captures the high day-to-day symptom variability among patients, a three-month pilot study shows.
Data collected with the web app PEPSS over a pre-specified period differed from that captured with standard, paper-based EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI) questionnaires at the final visit, and may represent a more sensitive way to assess symptom changes and treatment effects.
“Our results demonstrate the usability and the relevance of our web-based… tool for capturing data that closely reflects daily experiences of patients with [Sjögren’s disease],” the researchers wrote about the tool, which will be further validated in an ongoing European clinical trial called NECESSITY (NCT05113004) that seeks to develop a more specific and meaningful assessment of treatment effectiveness in people with Sjögren’s.
The study, “Development of a web-based ecological momentary assessment tool to measure day-to-day variability of the symptoms in patients with Sjögren’s disease,” was published in Rheumatic & Musculoskeletal Diseases.
Sjögren’s disease is an autoimmune disorder wherein self-reactive antibodies primarily attack the glands that produce tears and saliva, leading to chronic dryness in the eyes and mouth, along with symptoms such as joint pain, rashes, dry cough, and prolonged fatigue.
“Symptoms of dryness, fatigue and pain are those which have the greatest daily impact on patients’ quality of life,” and their assessment “primarily involves the use of patient-reported outcomes (PROs), especially [ESSPRI],” the researchers wrote. “However, symptoms reported by patients are likely to be highly variable over time, which can introduce imprecision in assessing symptoms based on a few data points [cross-sectional assessments] … This may potentially be one explanation as to why several clinical trials have not been able to demonstrate treatment efficacy on the ESSPRI score.”
Assessing Sjögren’s symptoms in ‘real-time’
ESSPRI asks patients to rate the maximum intensity of dryness, fatigue, and pain in the two previous weeks on a 0-10 scale. Researchers in France surmised that “a more frequent and real-time assessment over a defined period of time would be useful to address the issues associated with usual cross-sectional assessments in a hospital setting.”
They developed PEPSS, a digital tool that’s designed to allow Sjögren’s core symptoms to be assessed on a daily basis and in a real-life setting. The app includes individual ESSPRI scales of dryness, pain, and fatigue, along with separate assessments of eye and mouth dryness, using a 0-10 scale.
The goal was to validate the tool’s use as a potential measure in future appropriately controlled trials. The pilot study involved 45 Sjögren’s patients (91.1% women) at three care reference centers. All completed the ESSPRI questionnaires at the study’s entry and final visit, and daily assessments on the PEPSS app over three months.
While symptom intensity remained stable for most individual symptoms, and for the total ESSPRI score between the first and final assessment, median daily symptom variability ranged between 0.5 to 0.8 points across the different symptoms measured.
“This magnitude corresponds to more than half of the minimal clinically important difference for the ESSPRI score,” wrote the researchers, who said the results confirmed that the web-based assessment tool could “detect symptom fluctuations of a magnitude that may be clinically relevant.”
When they compared the predicted value of symptom intensity at three months, based on the new digital tool, to the actual value of the ESSPRI questionnaires given at the last hospital visit, they found clinically meaningful differences. Particularly, the predicted value for all evaluated scales of the digital tool was generally smaller, indicating less symptom severity, than that obtained with the standard questionnaire.
A more sensitive measure
This indicates the web-based assessment tool favored periods of lower symptom intensity in the final result, potentially making it better for determining if symptoms have diminished.
“Having a tool that is more sensitive to periods of symptom improvement can be potentially valuable in the specific context of placebo-controlled therapeutic trials comparing PROs, by mitigating the impact of extreme values likely to be influenced by [potentially influencing] factors,” the researchers wrote.
There was a good patient adherence to the app, with 83.3% completing all symptom assessments, and its usability was rated as excellent. Most participants (77.8%) said they’d be willing to continue with the app, which researchers felt was noteworthy for its potential use in future trials.
“By capturing daily variations in symptom intensity, this tool has the potential to provide a more sensitive assessment of symptom changes and may contribute to the demonstration of effectiveness of therapeutic interventions in the specific context of [Sjögren’s disease],” the researchers wrote. “This constitutes only a first step towards the validation of PROs based on longitudinal data, whose properties will need to be demonstrated as superior to those of validated cross-sectional assessments.”
