Clinical practice guideline for the treatment of Sjögren's disease through off-label drug use in China (English edition)

中国非适应症用药治疗干燥综合征的临床实践指南(英文版)

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Abstract

OBJECTIVE: To develop evidence-based clinical practice guideline for the treatment of primary Sjögren's disease (SJD) through off-label drug use in China, addressing the current lack of approved therapies and suboptimal management options for this autoimmune disease. METHODS: The clinical questions outlined in the guideline were formulated using the PICO (Population, Intervention, Comparison, Outcome) framework, with efficacy criteria based on the STAR (Sjögren's Tool for Assessing Response) indices. Evidence retrieval encompassed databases such as PubMed, Embase, CNKI, among others up to December 31, 2022; this was supplemented by snowball searching. A multidisciplinary expert panel systematically evaluated the randomized controlled trial (RCT) evidence using the Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) methodology and formulated recommendations. Recommendations were categorized as strong (1), weak (2), or consensus-based recommendations, based on thresholds of expert agreement. RESULTS: The guideline presents a total of 21 recommendations: including 2 strong recommendations, 14 weak recommendations, and 5 consensus-based recommendations. These encompass systemic treatments for SJD-related symptoms such as xerostomia and xerophthalmia; systemic disease activity; hypergammaglobulinemia; and considerations for special populations like pregnant patients. Key therapeutic agents include hydroxychloroquine, leflunomide, iguratimod, tripterygium glycosides, and low-dose recombinant human IL-2-employing stratified approaches according to disease severity and organ involvement. For refractory cases that do not respond adequately to initial treatments, rituximab, cyclophosphamide, and mycophenolate mofetil are recommended. The guideline emphasizes individualized treatment plans along with safety monitoring and multidisciplinary care-particularly important for high-risk groups such as pregnant women who test positive for anti-SSA antibodies. CONCLUSIONS: This guideline represents a significant advancement in the management of SJD by introducing structured approaches tailored to patient needs while highlighting areas requiring further research. Key Points • By disaggregating the STAR composite index into its constituent outcome measures, the guideline formulates precise clinical inquiries. • GRADE methodology was used to provide standardized, evidence-based recommendations. • 21 final recommendations for managing glandular, systemic, and organ-specific manifestations in SJD, considering real-world variability. • Prioritizes domestic therapies approaches tailored for Chinese practice.

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