Evidence map›Paper›PMID 29439650›Full record

Trial reportBMC nephrology2018

A model to predict disease progression in patients with autosomal dominant polycystic kidney disease (ADPKD): the ADPKD Outcomes Model.

Phil McEwan, Hayley Bennett Wilton, Albert C M Ong, Bjarne Ørskov, Richard Sandford, Francesco Scolari, Maria-Cristina V Cabrera, Gerd Walz, Karl O'Reilly, Paul Robinson

Registry-linked trialOpen access · goldAbstract readClinical Trial, Phase IIIControlled Clinical Trial
In one paragraph

Trial report in BMC nephrology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00428948 (A Phase 3, Multi-center, Double-blind, Placebo-controlled, Parallel-arm Trial to Determine Long-term Safety and Efficacy of Oral Tolvaptan Tablets Regimens in Adult Subjects With Autosomal Dominant Polycystic Kidney Disease), which is not on this map. Cited by 27 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed, 1 pooled it
3.8field-weighted citation impact, top 6% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT00428948 phase3completednot on this map

A Phase 3, Multi-center, Double-blind, Placebo-controlled, Parallel-arm Trial to Determine Long-term Safety and Efficacy of Oral Tolvaptan Tablets Regimens in Adult Subjects With Autosomal Dominant Polycystic Kidney Disease

TypeinterventionalSponsorOtsuka Pharmaceutical Development & Commercialization, Inc.Ran2007 to 2012Enrolled1,445ConditionsPolycystic Kidney Disease, Autosomal DominantArmsTolvaptan, Placebo
3 · Its place in the literature

Who cites it

27 citing papers in PubMed, 1 synthesis or guideline pooled it, 52 citations in OpenAlex.

  1. Pooled it
  2. The long-term effects of dapagliflozin in chronic kidney disease: a time-to-event analysis.Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association · 2024
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  20. Oral delivery of metformin by chitosan nanoparticles for polycystic kidney disease.Journal of controlled release : official journal of the Controlled Release Society · 2021
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors at 8 institutions in 5 countries.

Phil McEwanSwansea Centre for Health Economics, Swansea University, Swansea, UK.
Hayley Bennett WiltonHealth Economics and Outcomes Research Ltd, Cardiff, UK.
Albert C M OngAcademic Nephrology Unit, University of Sheffield Medical School, Sheffield, UK.
Bjarne ØrskovDepartment of Internal Medicine, Section of Nephrology, Zealand University Hospital, Roskilde, Denmark.
Richard SandfordAcademic Laboratory of Medical Genetics, Addenbrooke's Treatment Centre, Cambridge, UK.
Francesco ScolariDepartment of Nephrology, University of Brescia, Brescia, Italy.
Maria-Cristina V CabreraHospital Universitario La Paz, Madrid, Spain.
Gerd WalzDepartment of Nephrology, University Medical Centre Freiburg, Freiburg, Germany.
Karl O'ReillyOtsuka Pharmaceutical Europe Ltd, Gallions Wexham Springs, Framewood Road, Wexham, SL3 6PJ, UK. KO'Reilly@otsuka-europe.com.
Paul RobinsonOtsuka Pharmaceutical Europe Ltd, Gallions Wexham Springs, Framewood Road, Wexham, SL3 6PJ, UK.
Health Economics and Outcomes Research (United Kingdom) · GBOtsuka (United Kingdom) · GBAddenbrooke's Hospital · GBHospital Universitario La Paz · ESSheffield Kidney Institute · GBUniversity Medical Center Freiburg · DEUniversity of Brescia · ITZealand University Hospital · DK

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundAutosomal dominant polycystic kidney disease (ADPKD) is the leading inheritable cause of end-stage renal disease (ESRD); however, the natural course of disease progression is heterogeneous between patients. This study aimed to develop a natural history model of ADPKD that predicted progression rates and long-term outcomes in patients with differing baseline characteristics.

methodsThe ADPKD Outcomes Model (ADPKD-OM) was developed using available patient-level data from the placebo arm of the Tolvaptan Efficacy and Safety in Management of ADPKD and its Outcomes Study (TEMPO 3:4; ClinicalTrials.gov identifier NCT00428948). Multivariable regression equations estimating annual rates of ADPKD progression, in terms of total kidney volume (TKV) and estimated glomerular filtration rate, formed the basis of the lifetime patient-level simulation model. Outputs of the ADPKD-OM were compared against external data sources to validate model accuracy and generalisability to other ADPKD patient populations, then used to predict long-term outcomes in a cohort matched to the overall TEMPO 3:4 study population.

resultsA cohort with baseline patient characteristics consistent with TEMPO 3:4 was predicted to reach ESRD at a mean age of 52 years. Most patients (85%) were predicted to reach ESRD by the age of 65 years, with many progressing to ESRD earlier in life (18, 36 and 56% by the age of 45, 50 and 55 years, respectively). Consistent with previous research and clinical opinion, analyses supported the selection of baseline TKV as a prognostic factor for ADPKD progression, and demonstrated its value as a strong predictor of future ESRD risk. Validation exercises and illustrative analyses confirmed the ability of the ADPKD-OM to accurately predict disease progression towards ESRD across a range of clinically-relevant patient profiles.

conclusionsThe ADPKD-OM represents a robust tool to predict natural disease progression and long-term outcomes in ADPKD patients, based on readily available and/or measurable clinical characteristics. In conjunction with clinical judgement, it has the potential to support decision-making in research and clinical practice.

Indexed as

Disease ProgressionModels, TheoreticalAdultAgedAntidiuretic Hormone Receptor AntagonistsDouble-Blind MethodFemaleHumansMaleMiddle AgedPolycystic Kidney, Autosomal DominantPredictive Value of TestsTolvaptanTreatment OutcomeAntidiuretic Hormone Receptor AntagonistsTolvaptanDisease modellingEnd-stage renal diseaseESRDKidney volumeRenal function declineRenal progression

Identifiers

PMID29439650
PMCPMC5810027
OpenAlexW2789740077

What OpenQuestion holds

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LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.