Background/Purpose: Sjögren’s Syndrome (SS) is a systemic autoimmune disease associated with a wide range of symptoms and long-term complications. While clinical indices are useful in diagnosis and assessment of disease activity, patient-reported outcome (PRO) measures are needed to measure the severity of symptoms from a patient perspective. The objective of this study was to develop a patient-reported diary for use in clinical trials to assess the severity of key symptoms of SS in alignment with Food and Drug Administration (FDA) PRO guidance documents.

Methods: Development began with targeted reviews of the literature and relevant PRO measures to identify symptoms of SS which are both highly prevalent in the patient population and have the potential to improve within the context and timeframe of a clinical trial. Following the development of an item pool addressing the initially identified symptoms, semi-structured interviews were conducted with clinical experts (n=2) to gather feedback on this preliminary item pool and to identify any additional concepts of importance. Following minor revisions to the draft items based on clinician feedback, three iterative sets of hybrid concept elicitation (CE) and cognitive debriefing (CD) interviews were conducted with a total of 17 patients with primary SS (64.7% female; median age 58.0 years [range 28-70 years]) to refine the diary and further assess its content validity.

Results: Based on the targeted literature review, the most prevalent symptoms of importance to patients with SS included dryness of the eyes, mouth, skin, and female genitalia along with fatigue and muscle or joint pain. While additional symptoms that tend to occur at later stages of the disease process were identified (e.g., brain fog, neuropathic pain), these symptoms would not be relevant across the full disease spectrum and thus were not included in the preliminary item pool. While the clinical experts generally endorsed the initial content of the diary, they recommended the exclusion of skin dryness, noting that this was not a common complaint among their patients.

The symptoms most commonly reported by patients participating in the qualitative interviews were consistent with those addressed in the preliminary item pool and final diary, including dry eyes (100%), dry mouth (100%), dry skin (47%), genital dryness (46% of female participants), fatigue (77%), and muscle or joint pain (53%). Of note, while the concept of skin dryness was not fully endorsed by the clinical experts, patients supported its inclusion in the final measure. With minor modifications, patients also indicated that the diary items were easy to understand and answer. Finally, no concept of importance was identified as missing from the diary by more than 3 of the 17 interview participants, and many of these were impacts rather than symptoms of SS (e.g., dental decay, depression).

Conclusion: The Sjögren’s Syndrome Symptom Diary is a content valid measure designed for use in clinical trials to assess the severity of the symptoms most commonly experienced by patients with SS. Following psychometric evaluation, this measure will have the potential to support product labeling claims.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/development-of-a-symptom-diary-for-use-in-patients-with-sjogrens-syndrome/