Faria, Raquel

Introduction Cognitive symptoms such as ‘brain fog’ are frequent and disabling in patients with SLE. We aimed to develop and validate the Lupus Brain Fog Severity Scale (LBFSS), the first patient-reported outcome measure (PROM) for cognitive symptoms in SLE. Methods The LBFSS was developed using a multi-step international approach involving healthcare professionals and patients with SLE. Patients provided free-text descriptions of brain fog, generating candidate domains through thematic analysis (step 1). Domains were rated for relevance (step 2) and those retained converted into self-assessable items (step 3). The pilot LBFSS was iteratively refined (step 4). The finalised LBFSS was validated (step 5) in a large international SLE cohort. Psychometric evaluation included internal consistency, exploratory factor analysis and assessment of construct, convergent, known-groups and criterion-related validity. Results In step 1, 147 SLE participants across 39 countries generated 21 domains for cognitive symptoms, of which 13 were retained in consensus (step 2), converted into self-assessable items (step 3) and iteratively refined using patient and physician feedback. In step 5, validation of the LBFSS instrument in 378 patients from 36 countries demonstrated high internal consistency (Cronbach’s α=0.95) and a unidimensional structure explaining 62.2% of variance. LBFSS scores were significantly higher in participants reporting brain fog (p<0.0001), confirming known-group validity. Construct validity was supported by significant correlations with functional impact and symptom severity (ρ=0.46–0.65, p<0.0001), strong convergent validity with the Perceived Deficits Questionnaire-20 (ρ=0.84, p<0.0001) while the discriminative performance for presence versus absence of self-reported brain fog was high (area under the curve=0.85). Conclusion The LBFSS is a new, valid and feasible PROM to assess brain fog and cognitive symptoms in SLE.

Abstract Objectives Patient education is increasingly acknowledged as an important aspect of the management of systemic lupus erythematosus (SLE). The aim of the study was to develop the SLE Knowledge Assessment score (SLAKE), a digital multilingual self-assessment tool designed to quantify essential SLE knowledge. Methods International healthcare professionals (HCPs) and patient representatives engaged in a multi-step process to: identify essential SLE knowledge domains, select key domains via rating, and generate an item bank of 394 questions across 11 domains, which was then adapted into 19 languages. For validation, participants completed 44 questions (including 33 randomly selected), with scores calculated for total knowledge and the 11 specific domains. Statistical analyses examined associations between scores and demographic, clinical, and educational variables. Results SLAKE was used by 1182 SLE participants (1120 [94.8%] women, median age: 45 years [IQR: 35–54 years]), with a median SLE duration of 10 years (IQR: 4–20 years). The median SLAKE score was 37 (IQR: 34–40) of a maximum of 44 points while the median score across the 11 SLAKE domains ranged between 3 and 4 over a maximum of 4 points. There was a significant positive association between SLAKE score and SLE duration (p= 0.006), previous participation to a patient education course or a patient training for lupus (p&lt; 0.0001) and the education level (p&lt; 0.0001) but not with age (p= 0.48) or gender (p= 0.39). Conclusion SLAKE is a valid, multilingual, digital self-assessment tool that effectively measures essential SLE knowledge. Its randomized question bank and domain-specific scoring enable targeted education, ultimately supporting better disease management.

Objective Vacuoles E1 enzyme X‐linked autoinflammatory somatic syndrome (VEXAS) is a recently identified rare genetic disorder associated with somatic mutations in the UBA1 gene. VEXAS presents with a combination of inflammatory and hematologic manifestations, leading to increased morbidity and mortality. Methods Given the variability in disease presentation and the limited number of studies to date, no clinical documents currently exist to provide guidance to health care providers about the management of VEXAS. To address this gap, we formed an international multidisciplinary panel of VEXAS experts. Results Through formalized meetings and a voting process, the group developed consensus clinical guidance considerations for the management of VEXAS. These considerations offer practical advice on several key topics: (1) clinical features of VEXAS, (2) UBA1 screening methods, (3) the diagnosis of myelodysplastic syndromes (MDSs) in patients with VEXAS, and (4) prognosis and management. The aim is to provide expert guidance on which patients to test, how to test for VEXAS, how to approach MDS in the context of VEXAS, and considerations for management. Conclusion This work marks the first formal international consensus guidance for VEXAS and is intended to be used as a resource for clinicians seeking to understand the disease and its management.