Evidence regarding the efficacy and safety of desmopressin for treating nocturia in young children remains limited. Currently, it is only recommended for use in children over seven years old. Our aim was to evaluate the efficacy and safety of desmopressin in treating nocturia in young children.

This study focused on children under 8 years old who were experiencing nocturnal enuresis and receiving desmopressin treatment at our hospital from January 2017 to September 2023. Initially, all children received a 60 µg melt form of desmopressin at bedtime. Close follow-up was conducted to monitor adverse effects and evaluate outcomes. Patients with poor results had their dose gradually increased, up to 240 µg. An urodynamic study was performed before and after therapy. Outcome evaluation was conducted using the Global Response Assessment (GRA). The rate of adverse events was also recorded.

A total of 40 children participated in this study, with a mean age of 6.5±0.8 years and a mean follow-up period of 89±40.7 weeks. Twenty-seven children were classified as full responder (GRA=3), and 13 as partial responder (GRA≤2). Baseline patient characteristics were similar between the two groups (Table 1). At follow-up, significant improvements in all endpoints were observed from the early stage onward after desmopressin treatment compared to before treatment (Table 2). In subgroup analysis according to different GRA scores, desmopressin significantly reduced nocturia episodes in both groups. There were no differences in measured parameters between the groups (Table 3). No adverse events were noted during the study.

Regarding drug treatment, the first and most important thing must be safety and effectiveness. This study proves that desmopressin is effective and safe in young children. The age range of indications should be broadened according to this report. Furthermore, the differences between patients before treatment do not affect their satisfaction after treatment. The objective satisfaction may be different, but it does not affect the actual objective effect of the drug on improving nocturia episode. In other words, children of any age who suffer from nocturia may consider this medication. Furthermore, some children have no significant effect after first desmopressin use, but as time goes by, the proportion of patients who are effective also increases. This means that continued use can increase the efficacy of desmopressin.

Our results highlight the safety and efficacy of desmopressin in reducing nocturia episodes in young children without adverse effects. However, given their young age, children may not accurately report the efficacy and adverse events. Further well-designed randomized controlled trials are needed to confirm these results.