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A trial (NCT02964273) evaluated tolvaptan safety and pharmacodynamics in children (5\u201317\u00a0years). However, progression risk was not part of study eligibility criteria due to lack of validated criteria for risk assessment in children. As risk estimation is important to guide clinical management, baseline characteristics of the study participants were retrospectively evaluated to determine whether risk of rapid disease progression in pediatric ADPKD can be assessed and to identify parameters relevant for risk estimation.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>Four academic pediatric nephrologists reviewed baseline data and rated participant risk from 1 (lowest) to 5 (highest) based on clinical judgement and the literature. Three primary reviewers independently scored all cases, with each case reviewed by two primary reviewers. For cases with discordant ratings (\u2265\u20092-point difference), the fourth reviewer provided a secondary rating blinded to the primary evaluations. Study participants with discordant ratings and\/or for whom data were lacking were later discussed to clarify parameters relevant to risk estimation.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>Of 90 evaluable subjects, primary reviews of 69 (77%) were concordant. The proportion considered at risk of rapid progression (final mean rating\u2009\u2265\u20093.5) by age group was: 15\u201317\u00a0years, 27\/34 (79%); 12\u2013\u2009&lt;\u200915, 9\/32 (28%); 4\u2013\u2009&lt;\u200912, 8\/24 (33%). The panelists agreed on characteristics important for risk determination: age, kidney imaging, kidney function, blood pressure, urine protein, and genetics.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusions<\/jats:title>\n                <jats:p>High ratings concordance and agreement among reviewers on relevant clinical characteristics support the feasibility of pediatric risk assessment.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Graphical abstract<\/jats:title>\n                <jats:p>A higher resolution version of the Graphical abstract is available as Supplementary information<\/jats:p>\n                \n              <\/jats:sec>","DOI":"10.1007\/s00467-023-06239-8","type":"journal-article","created":{"date-parts":[[2023,12,13]],"date-time":"2023-12-13T16:02:30Z","timestamp":1702483350000},"page":"1481-1490","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Estimating risk of rapid disease progression in pediatric patients with autosomal dominant polycystic kidney disease: a randomized trial of tolvaptan"],"prefix":"10.1007","volume":"39","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0954-6088","authenticated-orcid":false,"given":"Djalila","family":"Mekahli","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6187-3927","authenticated-orcid":false,"given":"Lisa M.","family":"Guay-Woodford","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0460-2477","authenticated-orcid":false,"given":"Melissa A.","family":"Cadnapaphornchai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3711-8785","authenticated-orcid":false,"given":"Stuart L.","family":"Goldstein","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ann","family":"Dandurand","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Huan","family":"Jiang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5498-2124","authenticated-orcid":false,"given":"Pravin","family":"Jadhav","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurie","family":"Debuque","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2023,12,13]]},"reference":[{"key":"6239_CR1","doi-asserted-by":"publisher","first-page":"272","DOI":"10.3389\/fped.2017.00272","volume":"5","author":"S De Rechter","year":"2017","unstructured":"De Rechter S, Breysem L, Mekahli D (2017) Is autosomal dominant polycystic kidney disease becoming a pediatric disorder? 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Pediatr Nephrol 38:3043\u20133053. https:\/\/doi.org\/10.1007\/s00467-023-05926-w","journal-title":"Pediatr Nephrol"}],"container-title":["Pediatric Nephrology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00467-023-06239-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s00467-023-06239-8\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00467-023-06239-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,3,15]],"date-time":"2024-03-15T10:09:29Z","timestamp":1710497369000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s00467-023-06239-8"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,12,13]]},"references-count":39,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2024,5]]}},"alternative-id":["6239"],"URL":"https:\/\/doi.org\/10.1007\/s00467-023-06239-8","relation":{},"ISSN":["0931-041X","1432-198X"],"issn-type":[{"value":"0931-041X","type":"print"},{"value":"1432-198X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,12,13]]},"assertion":[{"value":"11 July 2023","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"6 November 2023","order":2,"name":"revised","label":"Revised","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"8 November 2023","order":3,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"13 December 2023","order":4,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"As reported previously (Schaefer F et al., <i>Eur J Pediatr<\/i> 2019;178:1013\u20131021), the clinical trial from which the data analyzed here were obtained was conducted in conformity with FDA regulations, International Conference on Harmonization Good Clinical Practice Guideline (E6), international ethical principles derived from the Declaration of Helsinki, Council for International Organizations of Medical Science guidelines, and applicable local laws and regulations.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"As reported previously for the original clinical trial from which the data analyzed here were obtained (Schaefer F et al., <i>Eur J Pediatr<\/i> 2019;178:1013\u20131021), written informed consent was obtained from a parent\/guardian or legally acceptable representative, as applicable for local laws, at screening, prior to the initiation of any protocol-required procedures. In addition, the subject must provide age-appropriate informed assent at screening and must have been able to understand that he or she could withdraw from the trial at any time.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Informed consent"}},{"value":"Dr. Mekahli, as a representative of UZ Leuven and KU Leuven, reports research grants from Otsuka Pharmaceuticals, and serves on advisory boards for Otsuka Pharmaceuticals, Sanofi Genzyme, and Reata, all paid to her institutions UZ Leuven and KU Leuven. She is supported by the Research Foundation Flanders (FWO) (G0C8920N and G060623N) and clinical senior research grant for D. Mekahli (1804123N).Dr. Guay-Woodford serves a consultant for and receives honoraria from Otsuka and Natera, Inc.Dr. Cadnapaphornchai serves on a pediatric advisory board of Otsuka Pharmaceuticals Inc.Dr. Goldstein has received grant funding from and served as a consultant for Otsuka.Dr. Dandurand was formerly employed by Otsuka.Dr. Jiang is an employee of Otsuka.Dr. Jadhav was formerly employed by Otsuka and serves as a consultant for Otsuka.Ms. Debuque is an employee of Otsuka.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}]}}