Randomized controlled trial of adjuvant oral dexamethasone pulse therapy in pemphigus vulgaris: PEMPULS trial.
ABSTRACT To determine the therapeutic effect of adjuvant dexamethasone pulse therapy when given in addition to conventional treatment of pemphigus vulgaris.
A randomized, placebo-controlled trial.
International European, multicenter outpatient and inpatient study.
Of the 20 enrolled patients, 11 were randomized to the dexamethasone pulse (DP) group and 9 to the placebo pulse (PP) group.
Oral dexamethasone in 300-mg pulses or PPs 3 days per month. During the intervention, the DP and PP groups received conventional treatment with prednisolone, 80 mg/d, which was tapered across 19 weeks, and azathioprine sodium, 3 mg/kg per day, until the end of the study. Monthly pulses were continued until prednisolone treatment was tapered to 0 mg.
Number of patients in remission, time to and duration of remission, cumulative prednisolone dose, and occurrence of adverse events during 1 year of follow-up.
Eight of the 11 DP-treated patients and all 9 PP-treated patients achieved remission. Mean time to remission was 173 days with DP and 176 days with PP. The mean duration of remission within the first year was 151 days for DP and 141 days for PP. Mean cumulative prednisolone dose was 5300 mg for DP and 4882 mg for PP. Weight gain (>5% of baseline) occurred in 8 DP-treated patients compared with 1 PP-treated patient (P<.01). We found no statistically significant difference (P>.05) of an adjuvant effect of DP on remission of pemphigus vulgaris.
In patients with new pemphigus vulgaris disease activity, there was no benefit of oral DP therapy given in addition to conventional treatment.
clinicaltrials.gov Identifier: NCT00127764.
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ABSTRACT: Validated outcome measures are essential in monitoring disease severity. Specifically in dermatology, which relies heavily on the clinical evaluation of the patient and not on laboratory values and radiographic tests, outcome measures help standardize patient care. Validated cutaneous scoring systems, much like standardized laboratory values, facilitate disease management and follow therapeutic response. Several cutaneous autoimmune dermatoses, specifically cutaneous lupus erythematosus (CLE), dermatomyositis (DM), and pemphigus vulgaris (PV), lack such outcome measures. As a result, evaluation of disease severity and patients' response to therapy over time is less reliable. Ultimately, patient care is compromised. These diseases, which are often chronic and relapsing and remitting, are also often refractory to treatment. Without outcome measures, new therapies cannot be systematically assessed in these diseases. Clinical trials that are completed without standardized outcome measures produce less reliable results. Therefore, the development of validated outcome measures in these autoimmune dermatoses is critical. However, the process of developing these tools is as important, if not more so, than their availability. This review examines the steps that should be considered when developing outcome measures, while further examining their importance in clinical practice and trials. Finally, this review more closely looks at CLE, DM, and PV and addresses the recent and ongoing progress that has been made in the development of their outcome measures.Archives for Dermatological Research 02/2008; 300(1):3-9. · 2.28 Impact Factor