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Development of a Novel Prediction Tool for Response to First-Line Treatments of Monosymptomatic Nocturnal Enuresis: A
Cecilie Siggaard Jørgensen1,2, Lien Dossche3,4, Rongqun Zhai5
1Department of Paediatrics and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark.
Insights
Individualized treatment for monosymptomatic nocturnal enuresis (MNE) using voiding diaries significantly improves treatment response in children. This approach is particularly effective for specific MNE subtypes, enhancing outcomes compared to random treatment allocation.
Area of Science:
- Pediatric Urology
- Sleep Medicine
- Clinical Research
Background:
- Monosymptomatic nocturnal enuresis (MNE) is influenced by nocturnal urine volume and bladder capacity.
- Effective first-line treatments are crucial for managing MNE in children.
Purpose of the Study:
- To evaluate the predictive value of nocturnal urine volume and bladder function for treatment response in children with MNE.
- To compare individualized treatment selection versus random allocation for first-line MNE therapies.
Main Methods:
- A randomized, controlled, international, multicenter study involved 324 children (6-14 years) with primary MNE.
- Treatment was either individualized based on voiding diaries (desmopressin or alarm) or randomly allocated.
- 281 children were included in the final statistical analysis.
Main Results:
- Individualized treatment led to a 21% higher response rate compared to random selection (RR 1.21, P=.032).
- Children with reduced maximum voided volume (MVV) and no nocturnal polyuria showed a 46% improved response with individualized treatment (RR 1.46, P=.003).
- A prediction model for desmopressin (dDAVP) response demonstrated high accuracy (AUC 0.85).
Conclusions:
- Treatment selection guided by voiding diaries enhances first-line therapy response in MNE, especially for specific patient subtypes.
- Integrating voiding diary data with clinical and demographic information aids in predicting treatment outcomes for MNE.
Purpose:
Nocturnal urine volume and bladder reservoir function are key pathogenic factors behind monosymptomatic nocturnal enuresis (MNE). We investigated the predictive value of these together with other demographic and clinical variables for response to first-line treatments in children with MNE.
Materials And Methods:
A randomized, controlled, international, multicenter study was conducted in 324 treatment-naïve children (6-14 years old) with primary MNE. The children were randomized to treatment with or without prior consideration of voiding diaries. In the group where treatment choice was based on voiding diaries, children with nocturnal polyuria and normal maximum voided volume (MVV) received desmopressin (dDAVP) treatment, and children with reduced MVV and no nocturnal polyuria received an enuresis alarm. In the other group, treatment with dDAVP or alarm was randomly allocated.
Results:
A total of 281 children (72% males) were qualified for statistical analysis. The change of responding to treatment was 21% higher in children where treatment was individualized compared to children where treatment was randomly selected (risk ratio = 1.21 [1.02-1.45], P = .032). In children with reduced MVV and no nocturnal polyuria (35% of all children), individualized treatment was associated with a 46% improvement in response compared to random treatment selection (risk ratio = 1.46 [1.14-1.87], P = .003). Furthermore, we developed a clinically relevant prediction model for response to dDAVP treatment (receiver operating characteristic curve 0.85).
Conclusions:
The present study demonstrates that treatment selection based on voiding diaries improves response to first-line treatment, particularly in specific subtypes. Information from voiding diaries together with clinical and demographic information provides the basis for predicting response.
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