The Canadian Enuresis Study and Evaluation Short- and Long-term Safety and Efficacy of an Oral Desmopressin Preparation

Abstract

OBJECTIVE: To evaluate the long-term (12 months) efficacy and safety of oral desmopressin (DDAVP). MATERIAL AND METHODS: A total of 256 healthy children (6-18 years old) with nocturnal enuresis with a frequency of > or = 10 wet nights during a 4-week observation period were eligible for inclusion in the study. Initially 0.2 mg of DDAVP was given for 14 nights. Those achieving a > 90% reduction in the number of wet nights over the observation period (full responders) began a 12-week continuous treatment period at this dose. The remaining children received 0.4 mg for an additional 14 nights. Those achieving a > or = 50% reduction in the number of wet nights (responders) commenced a 12-week continuous treatment period at this dose. Children with a < 50% reduction in the number of wet nights at this point were withdrawn from the study. Each 12-week treatment period was followed by a treatment-free period of 7-28 days. Children who remained dry during that period were assigned a full response and terminated the trial. Children with > or = 2 wet nights during that period immediately began a new 12-week treatment period at the previous dose. This was repeated for 12 months and thereafter the medication dose was tapered by halving over a 4-week period. RESULTS: A total of 117/236 children who completed the titration period (49.6%; 95% confidence interval 40-57%) responded (> 50% reduction over baseline). Throughout the study their response rate remained constant at approximately 74%. Continuous treatment reduced the median number of wet nights during the observation period from 5.75 to 1.00 per week. A total of 12.4% of children received the 0.2 mg dose and 87.6% the 0.4 mg dose. The proportion of full responses increased over the course of the study from 5.8% to 37.5%. DDAVP was well tolerated: the majority of reported adverse events were mild, although two adverse events leading to withdrawal were reported. CONCLUSIONS: Oral DDAVP provides an effective and well-tolerated means of providing long-term control in children with nocturnal enuresis. Long-term treatment increases the response rate.