Journal of the American Academy of Dermatology · 2025
Characterization and treatment outcomes of rituximab therapy in super-responders and rituximab-refractory pemphigus patients: A single-center retrospective study.
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Wu Zhili, Jiang Jiefeng, Duan Keyun, Li Guomin, Guo Feng, Zhang Jiao, Xue Ruzeng
Аннотация
BACKGROUND: Rituximab (RTX) is a first-line treatment for pemphigus, yet responses vary. Standardized definitions for super-responder (SR) and rituximab-refractory patient (RRP) are lacking, and their predictive factors remain unclear.
OBJECTIVE: To establish definitions for SR and RRP, compare their baseline characteristics, identify predictive factors, and evaluate the safety profile of RTX treatment over 48 weeks.
METHODS: This retrospective study analyzed baseline data from patients with pemphigus receiving initial RTX. Univariate and multivariate logistic regression identified independent predictors for SR and RRP.
RESULTS: Of 205 patients, 61 (29.8%) were SRs, 55 (26.8%) RRPs, and 89 (43.4%) moderate responders. Independent predictors for SRs were lower baseline Pemphigus Disease Area Index, higher baseline B-cell percentage, and negative direct immunofluorescence for C3. For RRPs, independent predictors were higher initial dose of corticosteroids and history of prior immunosuppressants use. The overall incidence of Treatment-emergent adverse events (TEAEs) within 48 weeks was 29.3%, primarily cutaneous/mucosal infections, with no significant difference among groups.
LIMITATIONS: Single center design and limited sample size.
CONCLUSION: This study proposes practical definitions for SR and RRP in RTX-treated pemphigus and identifies distinct, readily obtainable baseline predictors, which may facilitate early identification of patients with differing treatment responses, providing personalized treatment strategies.
OBJECTIVE: To establish definitions for SR and RRP, compare their baseline characteristics, identify predictive factors, and evaluate the safety profile of RTX treatment over 48 weeks.
METHODS: This retrospective study analyzed baseline data from patients with pemphigus receiving initial RTX. Univariate and multivariate logistic regression identified independent predictors for SR and RRP.
RESULTS: Of 205 patients, 61 (29.8%) were SRs, 55 (26.8%) RRPs, and 89 (43.4%) moderate responders. Independent predictors for SRs were lower baseline Pemphigus Disease Area Index, higher baseline B-cell percentage, and negative direct immunofluorescence for C3. For RRPs, independent predictors were higher initial dose of corticosteroids and history of prior immunosuppressants use. The overall incidence of Treatment-emergent adverse events (TEAEs) within 48 weeks was 29.3%, primarily cutaneous/mucosal infections, with no significant difference among groups.
LIMITATIONS: Single center design and limited sample size.
CONCLUSION: This study proposes practical definitions for SR and RRP in RTX-treated pemphigus and identifies distinct, readily obtainable baseline predictors, which may facilitate early identification of patients with differing treatment responses, providing personalized treatment strategies.