Abstract
BackgroundComprehensive assessment of systemic lupus erythematosus (SLE) and its treatment requires patient-reported outcome (PRO) measures to capture impacts and fluctuating symptoms. The objective of this study was to develop PROs, in accordance with the Food and Drug Administration (FDA) PRO Guidance, to assess fluctuations in SLE symptoms and its impact.MethodsFollowing independent review board approval, six US rheumatology practices recruited patients with SLE to participate in concept elicitation (CE) interviews, in order to identify important SLE symptoms and their impacts. The SLE Symptom Severity Diary (SSD) and SLE Impact Questionnaire (SIQ) were drafted based on CE interview results and clinician input. The PROs were revised based on patient feedback from cognitive debriefing (CD) interviews, clinician feedback, and a translatability assessment.ResultsForty-one patients completed CE interviews. Commonly-reported symptoms included fatigue (98%), joint pain (93%), and rash (88%). The most frequently reported impact was difficulty with chores/housework (61%). Eighteen patients completed CD interviews. The PROs were considered comprehensive, clear, and relevant.The SSD contains 17 items assessing energy/vitality, joint and muscle pain/stiffness/swelling, flu-like symptoms, cognition, numbness/tingling, skin symptoms and hair loss using an 11-point numeric response scale and a 24-h recall period (with the exception of hair loss). It also evaluates steroid status and dose. The SIQ contains 50 items, uses a 5-point Likert scale and a 7-day recall period, to assess disease impacts including patients’ ability to make plans, work, and physical/social/emotional functioning.ConclusionThe SSD and SIQ are comprehensive SLE-specific PROs developed in accordance with the FDA PRO Guidance. Following assessment of their measurement properties, they may be useful in clinical studies and clinical practice to measure fluctuations in, and the impact of, symptoms in patients with SLE.
Highlights
Comprehensive assessment of systemic lupus erythematosus (SLE) and its treatment requires patientreported outcome (PRO) measures to capture impacts and fluctuating symptoms
Literature review The literature review identified 68 PROs that had previously been used for evaluating patients with SLE; Table 1 summarizes the characteristics of PROs that matched some of the desired criteria for this study
The most commonly-cited PRO was the Medical Outcomes Short Form-36 (SF-36), which is a generic measure of healthrelated quality of life (HRQoL), provides a broad measure of functioning and well-being, but was not developed in an SLE population and does not assess all concepts that are important to patients with SLE [11]
Summary
Comprehensive assessment of systemic lupus erythematosus (SLE) and its treatment requires patientreported outcome (PRO) measures to capture impacts and fluctuating symptoms. The objective of this study was to develop PROs, in accordance with the Food and Drug Administration (FDA) PRO Guidance, to assess fluctuations in SLE symptoms and its impact. As recognized by the Food and Drug Administration (FDA) [2] and European Medicines Agency (EMA) [3], clinical and laboratory measures of SLE disease severity do not assess the impact of symptom- and treatment-related effects of SLE on patients’ daily lives. Reliable patient-reported outcome (PRO) measures are important tools in both SLE clinical studies and clinical practice to enable comprehensive assessment of the disease and its treatments. In combination with results from CD interviews, clinician assessment of the PRO should be used to revise and refine the PRO
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