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. 2019 Dec 1;155(12):1404-1409.
doi: 10.1001/jamadermatol.2019.3236.

Factors Associated With Complete Remission After Rituximab Therapy for Pemphigus

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Factors Associated With Complete Remission After Rituximab Therapy for Pemphigus

Carolyn J Kushner et al. JAMA Dermatol. .

Erratum in

  • Error in the Figure.
    [No authors listed] [No authors listed] JAMA Dermatol. 2025 May 1;161(5):562. doi: 10.1001/jamadermatol.2025.0857. JAMA Dermatol. 2025. PMID: 40202737 Free PMC article. No abstract available.

Abstract

Importance: Rituximab has emerged as a front-line therapy for pemphigus, but prognostic factors for achieving complete remission off therapy (CROT) with oral systemic agents remain unknown.

Objectives: To describe rates of CROT and relapse and identify prognostic factors for achieving CROT after rituximab therapy for pemphigus.

Design, setting, and participants: A single-center, retrospective, cohort study was conducted at the University of Pennsylvania including 112 patients with pemphigus treated with rituximab with at least 12 months' clinical follow-up after the start of rituximab therapy. Multivariate regression analysis of factors predictive of CROT and Kaplan-Meier analysis of disease relapse were conducted. The study included patients treated with rituximab from March 15, 2005, until December 19, 2016. Data analysis was performed from December 2017 to June 2018.

Main outcomes and measures: The primary study outcome was CROT after 1 cycle. Secondary study outcomes included rate of CROT or the composite end point of CROT or complete remission on minimal therapy after 1 or more cycle, and median time to relapse. Multivariate regression analysis for prognostic variables for CROT, including age, sex, pemphigus subtype, body mass index (BMI) (calculated as weight in kilograms divided by height in meters squared), disease duration, and dosing regimen, was performed.

Results: A total of 112 patients with pemphigus with median 37.8 months (range, 12.1-130.7) follow-up after rituximab therapy were identified. Of these, 65 were women (58.0%). At the time of first rituximab infusion, median age was 52.3 years (range, 20.0-89.3). Including patients who received multiple cycles of rituximab, 79 patients (70.5%) achieved CROT after a median time of 10.5 months (range, 2.0-49.8), and 36 of 72 patients (50.0%) subsequently experienced relapse after a median of 23.3 months (interquartile range, 10.8-50.4 months). Considering only the first cycle of rituximab, 54 patients (48.2%) achieved CROT. Controlling for age, sex, pemphigus subtype, BMI, and disease duration, patients who received lymphoma vs rheumatoid arthritis dosing were 2.70-fold more likely to achieve CROT (odds ratio [OR], 2.70; 95% CI, 1.03-7.12; P = .04). Increasing age was associated with significant increases in achieving CROT (Wald test for trend, P = .01), whereas BMI greater than or equal to 35 was associated with a 0.14 OR (95% CI, 0.03-0.63; P = .01) for achieving CROT, regardless of the dosing regimen. In multivariate analysis, there was no significant difference in CROT rates with sex (OR, 1.01; 95% CI, 0.42-2.50; P = .97), pemphigus subtype (OR, 0.37; 95% CI, 0.09-1.51; P = .17), or disease duration (OR, 0.99; 95% CI, 0.98-1.00; P = .09).

Conclusions and relevance: Lymphoma dosing and older age may be associated with CROT and BMI greater than or equal to 35 may be a negative prognostic factor for CROT after rituximab therapy for pemphigus. These findings help inform clinical expectations and merit evaluation in future prospective clinical trials.

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Conflict of interest statement

Conflict of Interest Disclosures: Dr Payne is a cofounder and equity holder in Cabaletta Bio, Inc, focused on targeted immunotherapy of pemphigus. She is an inventor on patents licensed by Novartis and Cabaletta Bio for cellular immunotherapy of autoimmune diseases, has previously served as a consultant for Syntimmune, Inc, and has received grant funding from Sanofi. Dr Werth has received grants from Roche/Genentech and Syntimmune. She is a consultant for Roche/Genentech, Syntimmune, Janssen, and Principia. Dr Tsai reported receiving employment income and stock options from Loxo Oncology. Dr Werth reported receiving grants and personal fees from Genentech outside the submitted work. Dr Payne reported receiving grants, personal fees, and equity from Cabaletta Bio outside the submitted work; in addition, Dr Payne had a patent to US20170051035A1 issued and licensed. No other disclosures were reported.

Figures

Figure.
Figure.. Time to Relapse After Achieving Complete Remission Off Therapy (CROT)
Time to relapse after achieving first CROT (n = 72; median, 23.3 months). Vertical marks indicate censored patients.

Comment in

References

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