Universität zu Köln

De Rechter, Stephanie, Bammens, Bert, Schaefer, Franz, Liebau, Max C. and Mekahli, Djalila ORCID: 0000-0003-0954-6088 (2018). Unmet needs and challenges for follow-up and treatment of autosomal dominant polycystic kidney disease: the paediatric perspective. Clin. Kidney J., 11. S. I14 - 13. OXFORD: OXFORD UNIV PRESS. ISSN 2048-8513

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Abstract

Awareness is growing that the clinical course of autosomal dominant polycystic kidney disease (ADPKD) already begins in childhood, with a broad range of both symptomatic and asymptomatic features. Knowing that parenchymal destruction with cyst formation and growth starts early in life, it seems reasonable to assume that early intervention may yield the best chances for preserving renal outcome. Interventions may involve lifestyle modifications, hypertension control and the use of disease-modifying treatments once these become available for the paediatric population with an acceptable risk and side-effect profile. Until then, screening of at-risk children is controversial and not generally recommended since this might cause psychosocial and financial harm. Also, the clinical and research communities are facing important questions as to the nature of potential interventions and their optimal indications and timing. Indeed, challenges include the identification and validation of indicators, both measuring and predicting disease progression from childhood, and the discrimination of slow from rapid progressors in the paediatric population. This discrimination will improve both the cost-effectiveness and benefit-to-risk ratio of therapies. Furthermore, we will need to define outcome measures, and to evaluate the possibility of a potential therapeutic window of opportunity in childhood. The recently established international register ADPedKD will help in elucidating these questions. In this review, we provide an overview of the current knowledge on paediatric ADPKD as a future therapeutic target population and its unmet challenges.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code De Rechter, Stephanie UNSPECIFIED UNSPECIFIED UNSPECIFIED Bammens, Bert UNSPECIFIED UNSPECIFIED UNSPECIFIED Schaefer, Franz UNSPECIFIED UNSPECIFIED UNSPECIFIED Liebau, Max C. UNSPECIFIED UNSPECIFIED UNSPECIFIED Mekahli, Djalila UNSPECIFIED orcid.org/0000-0003-0954-6088 UNSPECIFIED
URN:	urn:nbn:de:hbz:38-163038
DOI:	10.1093/ckj/sfy088
Journal or Publication Title:	Clin. Kidney J.
Volume:	11
Page Range:	S. I14 - 13
Date:	2018
Publisher:	OXFORD UNIV PRESS
Place of Publication:	OXFORD
ISSN:	2048-8513
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language LEFT-VENTRICULAR MASS; STAGE RENAL-DISEASE; BLOOD-PRESSURE; PROGRESSION; CHILDREN; VOLUME; ADPKD; PREDICTORS; TOLVAPTAN; OUTCOMES Multiple languages Urology & Nephrology Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/16303