Comparison of clinical efficacy and cost-effectiveness of rituximab infusion and intravenous dexamethasone pulse therapy in pemphigus vulgaris: an open prospective randomized controlled pilot study.

BACKGROUND

Rituximab (Rtx) and dexamethasone pulse (DP) are the two most commonly used therapeutic regimens in pemphigus vulgaris (PV).

OBJECTIVES

To compare the clinical efficacy, side-effect profile, cost-effectiveness and changes in desmoglein (Dsg) levels in patients with PV treated with an Rtx biosimilar or DP.

METHODS

This open-label prospective randomized controlled study (trial registration number CTRI/2020/04/032978) was conducted at the All India Institute for Medical Sciences in New Delhi, India, from November 2018 to September 2023. Fifty patients with active PV were randomized into two groups: an Rtx group and a DP group. Patients in both groups also received oral prednisolone in a tapering regimen with azathioprine or mycophenolate mofetil. Follow-up was conducted monthly until remission, then quarterly for at least a year or until relapse. Primary outcomes were remission rates and time to remission; secondary outcomes included relapse rates, adverse events and analysis of cost-effectiveness. Serum anti-Dsg titres were measured at baseline, remission and relapse.

RESULTS

Disease control was achieved within a median of 1 month in 96% of patients in both groups. Remission rates were 92% in the Rtx group and 84% in the DP group, with a similar median time to remission of 3 months. Relapse after attaining remission occurred twice as frequently in the DP group (76% vs. 39%) after a median of 10.5 months. Serum anti-Dsg1 and anti-Dsg3 declined significantly at remission and rose again at relapse. Adverse events, including gastrointestinal and general disorders, were more common in the DP group. Cost analysis revealed Rtx was 20% more cost-effective than DP.

CONCLUSIONS

While both regimens were equally effective in inducing remission in patients with PV, Rtx offered superior long-term disease control, fewer relapses and adverse events, and greater cost-effectiveness.