Multicenter randomized, double-blind, placebo-controlled, clinical trial of dapsone as a glucocorticoid-sparing agent in maintenance-phase pemphigus vulgaris.
OBJECTIVE
To determine the efficacy of dapsone as a glucocorticoid-sparing agent in maintenance-phase pemphigus vulgaris (PV).


METHODS
A randomized, double-blind, placebo-controlled study with a crossover arm for those who failed treatment.


METHODS
A US multicenter outpatient study. Patients A total of 19 subjects enrolled among 5 centers, 9 randomized to receive dapsone and 10 to receive placebo. Inclusion criteria were biopsy and direct immunofluorescence-proven PV controlled with glucocorticoids and/or cytotoxic agents, disease in maintenance phase, and aged 18 to 80 years. Physicians had tried at least 2 tapers of glucocorticoids unsuccessfully and had 30 days of stable steroid dosage. Treatment for any patient unable to taper glucocorticoids by more than 25% within 4 months was declared a failure, and the patient was allowed to switch to the opposite medication while maintaining the double-blind. Main Outcome Measure The ability of patients to taper to 7.5 mg/d or less within 1 year of reaching the maximum dosage of the study drug.


RESULTS
Of the 9 patients receiving dapsone, 5 were successfully treated, 3 failed treatment, and 1 dropped out of the study. Of the 10 patients receiving placebo, 3 were successfully treated, and 7 failed treatment. This primary end point favored the dapsone-treated group but was not statistically significant (P = .37). Four patients who failed treatment while receiving placebo were switched to treatment with dapsone. Of these, 3 were successfully treated after switching to dapsone treatment, and 1 failed treatment. We found that, overall, 8 of 11 patients (73%) receiving dapsone vs 3 of 10 (30%) receiving placebo reached the primary outcome of a prednisone dosage of 7.5 mg/d or less.


CONCLUSIONS
This trial demonstrates a trend to efficacy of dapsone as a steroid-sparing drug in maintenance-phase PV.
Werth
Victoria P
VP
Department of Dermatology, University of Pennsylvania, 2 Rhodes Pavilion, 3600 Spruce St, Philadelphia, PA 19119, USA. werth@mail.med.upenn.edu
Fivenson
David
D
Pandya
Amit G
AG
Chen
Diana
D
Rico
M Joyce
MJ
Albrecht
Joerg
J
Jacobus
David
D
D000284:Administration, Oral; D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000890:Anti-Infective Agents / Q000008:administration & dosage*; D018592:Cross-Over Studies; D003622:Dapsone / Q000008:administration & dosage*; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D004334:Drug Administration Schedule; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D010392:Pemphigus / Q000175:diagnosis* / Q000188:drug therapy*; D011241:Prednisone / Q000008:administration & dosage*; D011336:Probability; D012016:Reference Values; D018570:Risk Assessment; D012720:Severity of Illness Index; D016896:Treatment Outcome
D000890:Anti-Infective Agents; D003622:Dapsone; D011241:Prednisone