Background/Purpose: Childhood Sjogren Disease (cSjD) is a rare disease that presents with a wide variety of symptoms. Compared to adult SjD, cSjD more frequently manifests with parotitis and glandular swelling. Despite causing marked morbidity and mortality, no treatments are currently approved for cSjD. In this work, we develop a personalized N-of-1 design to compare cSjD therapies. Our innovative design generates patient-level randomized evidence to inform individualized treatment decision and allows for evaluation of comparative effectiveness between drugs when aggregating data across participants. Moreover, to capture the full spectrum of the treatment effects on the multiple cSjD manifestations, we incorporate patient-prioritized outcomes in the design. The purpose of this abstract is to perform item development and reduction as the next step to development of patient-focused outcome measures. Here, we present the tools we developed to elicit from patients how they rank outcome measures by importance.

Methods: To design surveys for patients, caregivers, and providers about cSjD symptoms, we systematically reviewed the literature to delineate common signs and symptoms of cSjD and to ensure we were not missing any major symptoms (item generation). We then compared this list to the ESSDAI, a validated outcome measure for adult SjD, which includes signs and a wide variety of symptoms, but does not emphasize those glandular features that are more prominent in cSjD. The resulting cSjD symptom survey was reviewed by research professionals and a patient/parent dyad engaged as research partners.

Results: The survey uses the Q-methodology that asks participants to sort 10 symptom domains in relation to each other according to a predetermined distribution. Participants can adjust their ranking until they are satisfied with the ordering of symptoms by importance. The Q-sort will be distributed to an online cSjD group that is validated by the parent of a cSjD patient and contains > 250 parents of children with cSjD and will also be disseminated via the Childhood Arthritis and Rheumatology Research Alliance (CARRA) which champions collaborative research to prevent, treat, and cure pediatric rheumatic disease. After two survey rounds, responses will be adjudicated at an in-person meeting to reach consensus on endpoint prioritization. Administration of the surveys and consensus meetings will be compliant with the Declaration of Helsinki.

Conclusion: An N-of-1 trial of two therapeutic agents will use these prioritized outcome measures to determine the efficacy of both agents with each patient serving as their own control. Data will be aggregated to determine if one or both agents meet the primary outcome, using either each personalized prioritized endpoint or the consensus-derived hierarchical endpoint. The trial will serve as a platform to evaluate additional agents in cSjD and other rare childhood rheumatic diseases.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/use-of-lived-experiences-in-childhood-sjogren-disease-to-develop-outcome-measures-for-an-n-of-1-treatment-trial/