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Rituximab May Be More Effective as First-Line Therapy for Pemphigus

– Study results follow previous findings that cast doubts over long-term efficacy


Patients with pemphigus who were treated with rituximab as a first-line therapy were more likely to achieve long-term remission without corticosteroid therapy or maintenance infusions of rituximab.

This finding emerged from a 7-year follow-up study of the trial, which included 90 patients from 25 dermatology departments in France and was conducted from 2010-2015.

A total of 43 patient participants (93%) who received rituximab plus prednisone and 17 patients (39%) who received prednisone alone achieved complete remission without corticosteroids at any time during follow-up.

The French research team published its findings in . The excerpts that follow were edited for length and clarity.

What motivated this investigation?

According to researchers, the original Ritux 3 trial helped pave the way for the approval of rituximab for the treatment of moderate-to-severe pemphigus vulgaris by both the FDA and the European Medicines Agency.

Subsequent research, however, has raised questions about the long-term efficacy of rituximab as a first-line treatment for pemphigus. In fact, no data from the long-term follow-up of patients who received rituximab as a first-line treatment were available prior to this study.

In this investigation, the research team hypothesized that first-line treatment of pemphigus using rituximab would be associated with a reduced risk of relapses and thus may increase the rate of sustained complete remission without corticosteroids compared with second- or third-line treatment using rituximab.

What were the study's key findings?

The 5- and 7-year disease-free survival rates for those treated with rituximab as first-line treatment were 76.7% and 72.1%, respectively.

Further, the 72.1% 7-year disease-free survival rate without corticosteroid therapy observed in the study was higher than previously reported in patients who received rituximab as a second- or third-line treatment.

Were there any notable safety findings?

No serious adverse events associated with immunosuppression were observed in patients who sustained complete remission until the end of the study. A total of 4 serious adverse events (1 case of stroke, 2 pneumonia, and 1 case of neutropenia) were observed in patients who relapsed and were re-treated with rituximab.

What are the study's primary take-home messages for dermatologists?

First-line rituximab treatment of patients with pemphigus was associated with long-term complete remission without corticosteroid therapy or any additional maintenance infusion of rituximab.

Patients with pemphigus need to be monitored to detect those who are at high risk of relapse. This could be done using the ELISA assay to measure anti-Dsg antibodies.

Clinical implications

  • First-line rituximab treatment of pemphigus was associated with long-term remission.
  • Patients with pemphigus need to be monitored to identify those at high risk of relapse.
  • Those at high risk of relapse could be identified by using the ELISA assay to measure anti-Dsg antibodies.

Study author Frederic Caux reported being an investigator from Argenx, Principia Biopharma, and Regeneron, and personal fees from Sanofi. Denis Jullien reported personal fees from AbbVie, Almirall, Amgen, BMS, Boehringer Ingelheim, Celgene, Janssen-Cilag, LEO, Lilly, MSD, MEDAC, Novartis, Pfizer, Sanofi, and UCB. Pascal Joly reported personal fees from Argenx and Janssen. Vivien Hébert reported personal fees from AbbVie, Lilly, Pfizer, and UCB.

Primary Source

JAMA Dermatology

Source Reference:

AAD Publications Corner

AAD Publications Corner