Pilot clinical trial of dehydroepiandrosterone (DHEA) versus placebo for Sjögren's syndrome.
OBJECTIVE
To screen for potential efficacy and assess feasibility and safety of dehydroepiandrosterone (DHEA) as a treatment for Sjögren's syndrome (SS).


METHODS
A 24-week randomized, double-blinded, pilot trial of oral DHEA (200 mg/day) versus placebo was conducted. The primary comparison was to a hypothesized 20% placebo response rate. If 14 consecutive subjects on DHEA did not respond, a Phase III trial would be considered futile. A placebo group of 14 subjects was planned to verify placebo response rate and estimate sample size required for a definitive trial. Response criteria required 20% improvement in at least 2 of 3 domains. Analysis of covariance was used to adjust for baseline differences and for stratified randomization. Outcome measures included visual analog scale questionnaires for dry eye and dry mouth symptoms, lissamine green ocular dye staining and Schirmer I tests, stimulated salivary flow, IgG, and erythrocyte sedimentation rate (ESR).


RESULTS
Randomization resulted in 14 DHEA and 14 placebo group subjects. At baseline, mean +/- SD for DHEA versus placebo groups were Schirmer I tests 4.5 +/- 4.5 versus 5.4 +/- 6.1 mm/5 minutes; Van Bijsterveld score 5.3 +/- 2.1 versus 5.5 +/- 2.2; unstimulated saliva 0.03 +/- 0.05 versus 0.04 +/- 0.10 ml/minute; IgG 1,699 +/- 749 versus 1,712 +/- 621 g/dl; and ESR 40 +/- 31 versus 44 +/- 28 mm/hour. Apart from changes over the trial in dry mouth symptoms, no significant differences were noted between the DHEA and placebo groups for dry eye symptoms, objective measures of ocular dryness, stimulated salivary flow; IgG, or ESR. Four DHEA and one placebo group patient dropped out because of adverse effects. Although 7 subjects met response criteria in the DHEA group, 5 met the criteria in the placebo group, and there was no significant difference between groups.


CONCLUSIONS
DHEA showed no evidence of efficacy in SS. Without evidence for efficacy, patients with SS should avoid using unregulated DHEA supplements, since long-term adverse consequences of exposure to this hormone are unknown.
Pillemer
Stanley R
SR
National Institute of Dental and Craniofacial Research, Bethesda, Maryland 28092, USA. PillemerS@medimmune.com
Brennan
Michael T
MT
Sankar
Vidya
V
Leakan
Rose Anne
RA
Smith
Janine A
JA
Grisius
Margaret
M
Ligier
Sophie
S
Radfar
Lida
L
Kok
Marc R
MR
Kingman
Albert
A
Fox
Philip C
PC
D000276:Adjuvants, Immunologic / Q000008:administration & dosage* / Q000009:adverse effects; D003687:Dehydroepiandrosterone / Q000008:administration & dosage* / Q000009:adverse effects; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008875:Middle Aged; D010865:Pilot Projects; D010919:Placebos; D012859:Sjogren's Syndrome / Q000188:drug therapy*; D016896:Treatment Outcome
D000276:Adjuvants, Immunologic; D010919:Placebos; D003687:Dehydroepiandrosterone