Therapeutic Area Data Standards for Autosomal Dominant Polycystic Kidney Disease: A Report From the Polycystic Kidney Disease Outcomes Consortium (PKDOC)

Perrone, Ronald D.; Neville, Jon; Chapman, Arlene B.; Gitomer, Berenice; Miskulin, Dana C.; Torres, Vicente E.; Czerwiec, Frank S.; Dennis, Eslie; Kisler, Bron; Kopko, Steve; Krasa, Holly B.; Leroy, Elisabeth; Castedo, Juliana; Schrier, Robert W.; Broadbent, Steve

doi:10.1053/j.ajkd.2015.04.044

Cited by 23 publications

(23 citation statements)

References 17 publications

(17 reference statements)

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“…The predictive value of TKV, together with age and eGFR, was confirmed by the PKD Outcomes Consortium, a collaborative effort including the PKD Foundation, the FDA, the Critical Path Institute, academic centers, and pharma. 21,22 This work led to the FDA and European Medicines Agency qualification of TKV, together with age and eGFR, as a prognostic biomarker.…”

Section: Step 2 Confirm the Diagnosis Of Rapidly Progressive Diseasementioning

confidence: 99%

A Practical Guide for Treatment of Rapidly Progressive ADPKD with Tolvaptan

Chebib

Perrone

Chapman

et al. 2018

JASN

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185

184

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In the past, the treatment of autosomal dominant polycystic kidney disease (ADPKD) has been limited to the management of its symptoms and complications. Recently, the US Food and Drug Administration (FDA) approved tolvaptan as the first drug treatment to slow kidney function decline in adults at risk of rapidly progressing ADPKD. Full prescribing information approved by the FDA provides helpful guidelines but does not address practical questions that are being raised by nephrologists, internists, and general practitioners taking care of patients with ADPKD, and by the patients themselves. In this review, we provide practical guidance and discuss steps that require consideration before and after prescribing tolvaptan to patients with ADPKD to ensure that this treatment is implemented safely and effectively. These steps include confirmation of diagnosis; identification of rapidly progressive disease; implementation of basic renal protective measures; counseling of patients on potential benefits and harms; exclusions to use; education of patients on aquaresis and its expected consequences; initiation, titration, and optimization of tolvaptan treatment; prevention of aquaresis-related complications; evaluation and management of liver enzyme elevations; and monitoring of treatment efficacy. Our recommendations are made on the basis of published evidence and our collective experiences during the randomized, clinical trials and open-label extension studies of tolvaptan in ADPKD.

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Section: Step 2 Confirm the Diagnosis Of Rapidly Progressive Diseasementioning

confidence: 99%

A Practical Guide for Treatment of Rapidly Progressive ADPKD with Tolvaptan

Chebib

Perrone

Chapman

et al. 2018

JASN

Self Cite

185

184

View full text Add to dashboard Cite

show abstract

“…These data sources used a variety of different imaging modalities (magnetic resonance imaging, computed tomography, and ultrasound), different methods/definitions, and different data‐capture strategies. In order to pool data, first a Clinical Data Interchange Standards Consortium data standard was developed for ADPKD and then subject matter experts resolved methodologic differences between studies [data repository; data standards] . Analysis of the pooled data revealed that baseline TKV, in combination with patient age and baseline estimated glomerular filtration rate, can accurately predict the risk and cadence of disease progression in patients with ADPKD [prognostic and predictive biomarker] .…”

Section: Resultsmentioning

confidence: 99%

“…In order to pool data, first a Clinical Data Interchange Standards Consortium data standard was developed for ADPKD and then subject matter experts resolved methodologic differences between studies [data repository; data standards]. 7 Analysis of the pooled data revealed that baseline TKV, in combination with patient age and baseline estimated glomerular filtration rate, can accurately predict the risk and cadence of disease progression in patients with ADPKD [prognostic and predictive biomarker]. 8,9 The US Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) recently qualified TKV as a prognostic biomarker that can be used by drug developers in submissions of investigational new drug (IND) applications, new drug applications (NDAs), and biologics license applications without the relevant CDER review group reconsidering and reconfirming the suitability of the biomarker [biomarker qualification].…”

Section: Case Study 1: Designing Clinical Trials To Evaluate Therapiementioning

confidence: 99%

Application of a Dynamic Map for Learning, Communicating, Navigating, and Improving Therapeutic Development

Wagner

Dahlem

Hudson

et al. 2017

Clinical Translational Sci

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Drug discovery and development is commonly schematized as a “pipeline,” and, although appreciated by drug developers to be a useful oversimplification, this cartology may perpetuate inaccurate notions of straightforwardness and is of minimal utility for process engineering to improve efficiency. To create a more granular schema, a group of drug developers, researchers, patient advocates, and regulators developed a crowdsourced atlas of the steps involved in translating basic discoveries into health interventions, annotated with the steps that are particularly prone to difficulty or failure. This Drug Discovery, Development, and Deployment Map (4DM), provides a network view of the process, which will be useful for communication and education to those new to the field, orientation and navigation of individual projects, and prioritization of technology development and re‐engineering endeavors to improve efficiency and effectiveness. The 4DM is freely available for utilization, modification, and further development by stakeholders across the translational ecosystem.

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“…However, some studies have indicated a lack of correlation between TKV growth rate and decline in renal function [19,20], leading to discussions regarding the use of TKV as a secondary endpoint. The Polycystic Kidney Disease Outcomes Consortium recently established a therapeutic data standard for ADPKD [21]. This allowed for observational data from multiple sources, including the Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP) [21], to be integrated into a single data set that included ≤30 years of follow-up TKV data from 2,355 patients with ADPKD [22].…”

Section: Introductionmentioning

confidence: 99%

“…The Polycystic Kidney Disease Outcomes Consortium recently established a therapeutic data standard for ADPKD [21]. This allowed for observational data from multiple sources, including the Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP) [21], to be integrated into a single data set that included ≤30 years of follow-up TKV data from 2,355 patients with ADPKD [22]. Analysis of this dataset showed that baseline TKV had a significant impact on the risk of a 30% decline in estimated glomerular filtration rate (eGFR) and progression to ESRD, independent of baseline eGFR and age [22].…”

Section: Introductionmentioning

confidence: 99%

A Review of the Imaging Techniques for Measuring Kidney and Cyst Volume in Establishing Autosomal Dominant Polycystic Kidney Disease Progression

et al. 2018

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Background: Autosomal dominant polycystic kidney disease (ADPKD) is the commonest inherited renal disorder; it is defined by progressive renal cyst formation and subsequent renal enlargement that leads to end-stage renal disease. Until recently, only symptomatic treatments for ADPKD existed. However, therapies that address the underlying pathophysiology of ADPKD are now available and accurate identification of the rate of disease progression is essential. Summary: Published data on the different imaging modalities for measuring kidney and cyst volumes in ADPKD are reviewed. The advantages and drawbacks of the different techniques for calculating kidney volume from renal imaging are also examined, including the use of manual planimetry, stereology, and the ellipsoid equation, as well as the prospect of semi- and fully automatic techniques. The translation of these approaches into clinical practice and their role in informing treatment decisions is discussed. Key Messages: These new therapies require the accurate monitoring of disease progression, which along with diagnosis and prognosis, relies on the effective use of renal imaging techniques. There is growing support for the use of total kidney volume as a measure of cyst burden and as a prognostic predictor of renal function in ADPKD, showing promise as a marker of disease progression.

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Therapeutic Area Data Standards for Autosomal Dominant Polycystic Kidney Disease: A Report From the Polycystic Kidney Disease Outcomes Consortium (PKDOC)

Cited by 23 publications

References 17 publications

A Practical Guide for Treatment of Rapidly Progressive ADPKD with Tolvaptan

A Practical Guide for Treatment of Rapidly Progressive ADPKD with Tolvaptan

Application of a Dynamic Map for Learning, Communicating, Navigating, and Improving Therapeutic Development

A Review of the Imaging Techniques for Measuring Kidney and Cyst Volume in Establishing Autosomal Dominant Polycystic Kidney Disease Progression

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