Estimating risk of rapid disease progression in pediatric patients with autosomal dominant polycystic kidney disease : a randomized trial of tolvaptan
© 2023. The Author(s)..
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.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2024 |
|---|---|
| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:39 |
|---|---|
| Enthalten in: |
Pediatric nephrology (Berlin, Germany) - 39(2024), 5 vom: 15. März, Seite 1481-1490 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Mekahli, Djalila [VerfasserIn] |
|---|
| Links: |
|---|
| Anmerkungen: |
Date Completed 18.03.2024 Date Revised 26.03.2024 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1007/s00467-023-06239-8 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM365819700 |
|---|
| LEADER | 01000caa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM365819700 | ||
| 003 | DE-627 | ||
| 005 | 20240326235121.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2024 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1007/s00467-023-06239-8 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1348.xml |
| 035 | |a (DE-627)NLM365819700 | ||
| 035 | |a (NLM)38091246 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Mekahli, Djalila |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Estimating risk of rapid disease progression in pediatric patients with autosomal dominant polycystic kidney disease |b a randomized trial of tolvaptan |
| 264 | 1 | |c 2024 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 18.03.2024 | ||
| 500 | |a Date Revised 26.03.2024 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2023. The Author(s). | ||
| 520 | |a 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 | ||
| 520 | |a 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 | ||
| 520 | |a 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 | ||
| 520 | |a CONCLUSIONS: High ratings concordance and agreement among reviewers on relevant clinical characteristics support the feasibility of pediatric risk assessment | ||
| 650 | 4 | |a Randomized Controlled Trial | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a ADPKD | |
| 650 | 4 | |a Autosomal dominant polycystic kidney disease | |
| 650 | 4 | |a Chronic kidney disease | |
| 650 | 4 | |a Kidney volume | |
| 650 | 4 | |a Pediatric | |
| 650 | 4 | |a Risk assessment | |
| 650 | 4 | |a Tolvaptan | |
| 650 | 7 | |a Antidiuretic Hormone Receptor Antagonists |2 NLM | |
| 650 | 7 | |a Tolvaptan |2 NLM | |
| 650 | 7 | |a 21G72T1950 |2 NLM | |
| 700 | 1 | |a Guay-Woodford, Lisa M |e verfasserin |4 aut | |
| 700 | 1 | |a Cadnapaphornchai, Melissa A |e verfasserin |4 aut | |
| 700 | 1 | |a Goldstein, Stuart L |e verfasserin |4 aut | |
| 700 | 1 | |a Dandurand, Ann |e verfasserin |4 aut | |
| 700 | 1 | |a Jiang, Huan |e verfasserin |4 aut | |
| 700 | 1 | |a Jadhav, Pravin |e verfasserin |4 aut | |
| 700 | 1 | |a Debuque, Laurie |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Pediatric nephrology (Berlin, Germany) |d 1995 |g 39(2024), 5 vom: 15. März, Seite 1481-1490 |w (DE-627)NLM012612111 |x 1432-198X |7 nnns |
| 773 | 1 | 8 | |g volume:39 |g year:2024 |g number:5 |g day:15 |g month:03 |g pages:1481-1490 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1007/s00467-023-06239-8 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 39 |j 2024 |e 5 |b 15 |c 03 |h 1481-1490 | ||