Evidence map›Paper›PMID 40242785›Full record

ArticleKidney medicine2025

Effectiveness of Tolvaptan for Autosomal Dominant Polycystic Kidney Disease in US Clinical Practice With Comparison to Historical Control Data.

Ronald D Perrone, Diana Garbinsky, Sasikiran Nunna, Hema K Gandhi, Ancilla W Fernandes, Gabriela Burgos, Abisola Olopoenia, Marc DeCongelio, Martine C Maculaitis, Xiaolei Zhou

Abstract read
In one paragraph

Article in Kidney medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
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.

Ronald D PerroneDivision of Nephrology, Tufts Medical Center, Boston, MA.
Diana GarbinskyRTI Health Solutions, Research Triangle Park, NC.
Sasikiran NunnaOtsuka Pharmaceutical Development & Commercialization Inc, Rockville, MD.
Hema K GandhiOtsuka Pharmaceutical Development & Commercialization Inc, Rockville, MD.
Ancilla W FernandesOtsuka Pharmaceutical Development & Commercialization Inc, Rockville, MD.
Gabriela BurgosOracle Life Sciences, Austin, TX.
Abisola OlopoeniaOracle Life Sciences, Austin, TX.
Marc DeCongelioOracle Life Sciences, Austin, TX.
Martine C MaculaitisOracle Life Sciences, Austin, TX.
Xiaolei ZhouRTI Health Solutions, Research Triangle Park, NC.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Rationale & Objective: Data from clinical practice are needed to characterize the effectiveness of pharmacotherapy outside the controlled setting of clinical trials but lack an untreated placebo group for comparison. To assess the effectiveness of tolvaptan for autosomal dominant polycystic kidney disease (ADPKD) in nephrology practice, we performed a chart review of US patients and compared it with a historical matched control cohort. Study Design: Patient data from charts were provided by US nephrologists who participated in an online survey. Historical control data for patients with ADPKD not treated with tolvaptan were extracted from a database of ADPKD clinical studies (Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease, HALT Progression of Polycystic Kidney Disease, and OVERTURE). Setting & Participants: Nephrologist respondents (n = 57) provided baseline data and up to 4 years of follow-up on estimated glomerular filtration rate (eGFR) for tolvaptan-treated adults (n = 149). Historical ADPKD-affected controls were adults in Mayo imaging risk classes 1C-1E (ie, at increased risk of rapid progression, consistent with the tolvaptan indication). Exposure: Cases had to receive tolvaptan continuously for ≥2 years. Historical controls received nontolvaptan standard of care, including various antihypertensive regimens. Outcome: Annual rate of eGFR change. Analytical Approach: Cases and controls were matched on baseline clinical characteristics (matched set A: age, sex, and chronic kidney disease stage [110 matched pairs]; matched set B: age, sex, and eGFR [98 matched pairs]) and compared using a mixed model. Results: The annual rate of eGFR decline was slower in tolvaptan-treated patients versus historical controls, by 1.40 mL/min/1.73 m Limitations: Risk of bias from convenience sampling and potential residual confounding after case/historical control matching. Conclusions: Tolvaptan was associated with slower eGFR decline in routine clinical practice, consistent with the results of controlled trials.

Indexed as

Autosomal dominant polycystic kidney diseaseclinical practiceestimated glomerular filtration ratehistorical control studytolvaptan

Identifiers

PMID40242785
PMCPMC12002913

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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.