Development of a Novel Prediction Tool for Response to First-Line Treatments of Monosymptomatic Nocturnal Enuresis-A Randomized, Controlled International Multicenter Study (DRYCHILD)

Cecilie Siggaard Jørgensen; Lien Dossche; Rongqun Zhai; Michal Maternik; Konstantinos Kamperis; Anders S Breinbjerg; Sevasti Karamaria; Kristina Thorsteinsson; Britt Borg; Yihe Wang; Shuai Li; Ann Raes; Lu Wei; Aleksandra Żurowska; Søren Hagstrøm; Johan Vande Walle; Wen Jian Guo; Søren Rittig

doi:10.1097/JU.0000000000004129

Development of a Novel Prediction Tool for Response to First-Line Treatments of Monosymptomatic Nocturnal Enuresis-A Randomized, Controlled International Multicenter Study (DRYCHILD)

Cecilie Siggaard Jørgensen^*, Lien Dossche, Rongqun Zhai, Michal Maternik, Konstantinos Kamperis, Anders S Breinbjerg, Sevasti Karamaria, Kristina Thorsteinsson, Britt Borg, Yihe Wang, Shuai Li, Ann Raes, Lu Wei, Aleksandra Żurowska, Søren Hagstrøm, Johan Vande Walle, Wen Jian Guo, Søren Rittig

^*Corresponding author for this work

Department of Clinical Medicine - Department of Paediatrics

Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Journal article › Research › peer-review

5 Citations (Scopus)

Abstract

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-na€ıve 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. Clinical Trial Registration No.: NCT03389412.

Original language	English
Journal	The Journal of Urology
Volume	212
Issue	4
Pages (from-to)	539-549
Number of pages	11
ISSN	0022-5347
DOIs	https://doi.org/10.1097/JU.0000000000004129
Publication status	Published - Oct 2024

Keywords

alarm
desmopressin
monosymptomatic nocturnal enuresis
predictive factors
voiding diaries

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10.1097/JU.0000000000004129

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Jørgensen, C. S., Dossche, L., Zhai, R., Maternik, M., Kamperis, K., Breinbjerg, A. S., Karamaria, S., Thorsteinsson, K., Borg, B., Wang, Y., Li, S., Raes, A., Wei, L., Żurowska, A., Hagstrøm, S., Walle, J. V., Guo, W. J., & Rittig, S. (2024). Development of a Novel Prediction Tool for Response to First-Line Treatments of Monosymptomatic Nocturnal Enuresis-A Randomized, Controlled International Multicenter Study (DRYCHILD). The Journal of Urology, 212(4), 539-549. https://doi.org/10.1097/JU.0000000000004129

@article{75c82c34860c42e6a4575bb812ea8c99,

title = "Development of a Novel Prediction Tool for Response to First-Line Treatments of Monosymptomatic Nocturnal Enuresis-A Randomized, Controlled International Multicenter Study (DRYCHILD)",

abstract = "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-na€ıve 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. Clinical Trial Registration No.: NCT03389412.",

keywords = "alarm, desmopressin, monosymptomatic nocturnal enuresis, predictive factors, voiding diaries",

author = "J{\o}rgensen, {Cecilie Siggaard} and Lien Dossche and Rongqun Zhai and Michal Maternik and Konstantinos Kamperis and Breinbjerg, {Anders S} and Sevasti Karamaria and Kristina Thorsteinsson and Britt Borg and Yihe Wang and Shuai Li and Ann Raes and Lu Wei and Aleksandra {\.Z}urowska and S{\o}ren Hagstr{\o}m and Walle, {Johan Vande} and Guo, {Wen Jian} and S{\o}ren Rittig",

year = "2024",

month = oct,

doi = "10.1097/JU.0000000000004129",

language = "English",

volume = "212",

pages = "539--549",

journal = "The Journal of Urology",

issn = "0022-5347",

publisher = "Wolters Kluwer Health",

number = "4",

}

Jørgensen, CS, Dossche, L, Zhai, R, Maternik, M, Kamperis, K , Breinbjerg, AS, Karamaria, S, Thorsteinsson, K, Borg, B, Wang, Y, Li, S, Raes, A, Wei, L, Żurowska, A, Hagstrøm, S, Walle, JV, Guo, WJ & Rittig, S 2024, 'Development of a Novel Prediction Tool for Response to First-Line Treatments of Monosymptomatic Nocturnal Enuresis-A Randomized, Controlled International Multicenter Study (DRYCHILD)', The Journal of Urology, vol. 212, no. 4, pp. 539-549. https://doi.org/10.1097/JU.0000000000004129

Development of a Novel Prediction Tool for Response to First-Line Treatments of Monosymptomatic Nocturnal Enuresis-A Randomized, Controlled International Multicenter Study (DRYCHILD). / Jørgensen, Cecilie Siggaard; Dossche, Lien; Zhai, Rongqun et al.
In: The Journal of Urology, Vol. 212, No. 4, 10.2024, p. 539-549.

Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Journal article › Research › peer-review

TY - JOUR

T1 - Development of a Novel Prediction Tool for Response to First-Line Treatments of Monosymptomatic Nocturnal Enuresis-A Randomized, Controlled International Multicenter Study (DRYCHILD)

AU - Jørgensen, Cecilie Siggaard

AU - Dossche, Lien

AU - Zhai, Rongqun

AU - Maternik, Michal

AU - Kamperis, Konstantinos

AU - Breinbjerg, Anders S

AU - Karamaria, Sevasti

AU - Thorsteinsson, Kristina

AU - Borg, Britt

AU - Wang, Yihe

AU - Li, Shuai

AU - Raes, Ann

AU - Wei, Lu

AU - Żurowska, Aleksandra

AU - Hagstrøm, Søren

AU - Walle, Johan Vande

AU - Guo, Wen Jian

AU - Rittig, Søren

PY - 2024/10

Y1 - 2024/10

N2 - 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-na€ıve 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. Clinical Trial Registration No.: NCT03389412.

AB - 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-na€ıve 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. Clinical Trial Registration No.: NCT03389412.

KW - alarm

KW - desmopressin

KW - monosymptomatic nocturnal enuresis

KW - predictive factors

KW - voiding diaries

UR - http://www.scopus.com/inward/record.url?scp=85200409852&partnerID=8YFLogxK

U2 - 10.1097/JU.0000000000004129

DO - 10.1097/JU.0000000000004129

M3 - Journal article

C2 - 38950376

SN - 0022-5347

VL - 212

SP - 539

EP - 549

JO - The Journal of Urology

JF - The Journal of Urology

IS - 4

ER -

Development of a Novel Prediction Tool for Response to First-Line Treatments of Monosymptomatic Nocturnal Enuresis-A Randomized, Controlled International Multicenter Study (DRYCHILD)

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