Safety profile of desmopressin tablet for enuresis in a prospective study
Charlotte Van Herzeele1, Pauline De Bruyne, Jonathan Evans
1Department of Pediatric Nephrology, University Hospital Ghent, De Pintelaan 185, 9000, Ghent, Belgium, charlotte.vanherzeele@ugent.be.
Insights
Oral desmopressin tablets are safe for treating childhood primary nocturnal enuresis. The study found treatment-emergent adverse events were infrequent and not clinically significant, indicating good tolerability in children.
Area of Science:
- Pediatric Nephrology
- Clinical Pharmacology
- Drug Safety
Background:
- Primary nocturnal enuresis affects many children, impacting quality of life.
- Desmopressin is a common treatment, but its safety in oral tablet form requires evaluation.
- The desmopressin response in primary nocturnal enuresis (DRIP) study assessed desmopressin's efficacy and safety.
Purpose of the Study:
- To evaluate the safety profile of oral desmopressin tablets in children with primary nocturnal enuresis.
- To assess adverse events and changes in body mass index (BMI) as safety endpoints.
Main Methods:
- An open-label, intention-to-treat, phase IV, multi-national study.
- 744 children aged 5-15 years with primary nocturnal enuresis received desmopressin orally once daily.
- Adverse events were systematically monitored at each study visit.
Main Results:
- 30% of patients experienced treatment-emergent adverse events (404 total).
- Adverse events were most common in gastrointestinal, infection, and respiratory categories, and generally unrelated to desmopressin.
- A slight, clinically insignificant increase in BMI was observed.
Conclusions:
- Oral desmopressin tablet treatment is well-tolerated in pediatric patients with primary nocturnal enuresis.
- Safety profile is consistent across different genders and age groups within the studied pediatric population.
Introduction:
This pre-specified sub-study of the desmopressin response in primary nocturnal enuresis study (DRIP study) evaluates the safety profile of the oral desmopressin tablet in children with primary nocturnal enuresis. Endpoints are adverse events and change in body mass index.
Methods:
The DRIP study was an open-label, intention-to-treat, phase IV, multi-national study. Overall, 936 patients were screened and 744 children aged 5-15 years with previously untreated primary nocturnal enuresis were eligible to receive the study medication desmopressin once daily as an oral tablet formulation. At each visit, adverse events were questioned and observed signs or symptoms were recorded.
Results:
Overall, 222 (30%) patients experienced 404 treatment-emergent adverse events. The proportion of patients experiencing treatment-emergent adverse events was similar regardless of patient gender or age. Most treatment-emergent adverse events were experienced in three system organ classes: gastrointestinal disorders; infections and infestations; and respiratory, thoracic and mediastinal disorders and were considered unrelated to the study drug. There was a slight increase in body mass index from screening levels during the study, however, clinically not significant.
Conclusion:
Desmopressin tablet treatment is well tolerated in children with primary nocturnal enuresis, regardless of patient gender or age.
Funding:
The desmopressin response in primary nocturnal enuresis study (DRIP- study) was funded by Ferring.
