Abstract General Information

Title

DEVELOPMENT OF A NOVEL PREDICTION TOOL FOR RESPONSE TO FIRST-LINE TREATMENTS OF MONOSYMPTOMATIC NOCTURNAL ENURESIS - A RANDOMIZED, CONTROLLED INTERNATIONAL MULTICENTER STUDY (DRYCHILD)

Introduction and objective

Nocturnal urine volume and bladder reservoir function are key pathogenic factors behind monosymptomatic nocturnal enuresis (MNE). Whether information about these factors is important for prediction of response to first-line treatments is currently unclear. We investigated the predictive value of clinical characterization for response to first-line treatments.

Method

This randomized, controlled, international multicenter study was conducted in 324 treatment-naïve children (6–14 years) with primary MNE. Oral and written consent was obtained. 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 treatment and children with reduced MVV and no nocturnal polyuria received an enuresis alarm. In the other group, treatment with desmopressin or alarm was randomly allocated.

Results

In total, 281 children (72% males) were included in the analysis. Among children where treatment was individualized, 74% experienced a response to treatment compared to 61% where treatment was randomly selected (P = 0.032). In children with reduced MVV and no nocturnal polyuria, individualized treatment was associated with a 46% improvement in response. The study identified nocturnal bladder capacity as a novel and significant predictive factor for treatment response. Furthermore, we developed a clinically relevant prediction model for response to dDAVP with a positive predictive value of 80% and a negative predictive value of 75% (ROC 0.85).

Conclusion

Tailoring first-line treatments of primary MNE based on patient characterization improves the likelihood of response. The present study provides the first strong evidence that voiding diaries should be performed before selecting first-line treatment modality. Information from voiding diaries together with information on age, sex and BMI provides the basis for predicting response to first-line treatments.