Estimating risk of rapid disease progression in pediatric patients with autosomal dominant polycystic kidney disease: a randomized trial of tolvaptan.
Background: Tolvaptan preserves kidney function in adults with autosomal dominant polycystic kidney disease (ADPKD) at elevated risk of rapid progression. A trial (NCT02964273) evaluated tolvaptan safety and pharmacodynamics in children (5–17 years). However, progression risk was not part of study e...
| Publicado en: | Pediatric Nephrology Vol. 39; no. 5; pp. 1481 - 1491 |
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| Autores principales: | , , , , , , , |
| Formato: | research tables/charts Journal Article |
| Publicado: |
Springer Nature
May2024
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| Acceso en línea: | Ver este registro en EBSCOhost |
| fields | @attributes: recordID: 1 pdfLink: plink: https://search.ebscohost.com/login.aspx?direct=true&db=ccm&AN=176081529&site=ehost-live header: @attributes: shortDbName: ccm uiTerm: 176081529 longDbName: CINAHL Complete uiTag: AN controlInfo: bkinfo: dissinfo: jinfo: jid: 0931041X EF1 jtl: Pediatric Nephrology issn: 0931041X maglogo: N pubinfo: dt: May2024 vid: 39 iid: 5 pid: 237 pub: Springer Nature place: New York, New York artinfo: ui: 176081529 174195575 176081529 176081529 10.1007/s00467-023-06239-8 176081529 ppf: 1481 ppct: 10 formats: fmt: – @attributes: type: T – @attributes: type: P tig: atl: Estimating risk of rapid disease progression in pediatric patients with autosomal dominant polycystic kidney disease: a randomized trial of tolvaptan. aug: au: Mekahli, Djalila Guay-Woodford, Lisa M. Cadnapaphornchai, Melissa A. Goldstein, Stuart L. Dandurand, Ann Jiang, Huan Jadhav, Pravin Debuque, Laurie affil: https://ror.org/05f950310 PKD Research Group, Laboratory of Ion Channel Research, Department of Cellular and Molecular Medicine, KU Leuven, Louvain, Belgium sug: subj: Risk Assessment Disease Progression Risk Factors Polycystic Kidney, Autosomal Dominant Drug Therapy Polycystic Kidney, Autosomal Dominant Drug Therapy Vasopressins Antagonists and Inhibitors Receptors, Cell Surface Antagonists and Inhibitors Funding Source Human Child, Preschool Child Adolescence Retrospective Design Vasopressins Pharmacodynamics Vasopressins Adverse Effects Scales Male Female Confidence Intervals Descriptive Statistics Glomerular Filtration Rate Comparative Studies Child, Preschool: 2-5 years Child: 6-12 years Adolescent: 13-18 years Male Female ab: Background: Tolvaptan preserves kidney function in adults with autosomal dominant polycystic kidney disease (ADPKD) at elevated risk of rapid progression. A trial (NCT02964273) evaluated tolvaptan safety and pharmacodynamics in children (5–17 years). 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. Methods: 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 (≥ 2-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. Results: Of 90 evaluable subjects, primary reviews of 69 (77%) were concordant. The proportion considered at risk of rapid progression (final mean rating ≥ 3.5) by age group was: 15–17 years, 27/34 (79%); 12– < 15, 9/32 (28%); 4– < 12, 8/24 (33%). The panelists agreed on characteristics important for risk determination: age, kidney imaging, kidney function, blood pressure, urine protein, and genetics. Conclusions: High ratings concordance and agreement among reviewers on relevant clinical characteristics support the feasibility of pediatric risk assessment. A higher resolution version of the Graphical abstract is available as Supplementary information pubtype: Academic Journal doctype: research tables/charts Journal Article ougenre: Article language: English refInfo: holdings: @attributes: islocal: N |
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