Global Real-World Evidence of Tolvaptan in Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Bibliographic record
Abstract
Background: ADPKD is a rare, hereditary, systemic kidney disease characterized by progressive renal damage. Patients frequently develop end stage kidney disease, requiring renal replacement therapy. Tolvaptan is the first and only treatment shown to slow kidney function decline in adults who are at risk of rapidly progressing ADPKD. The goal of this literature review was to understand real-world effectiveness and safety data currently available on tolvaptan treatment. Methods: A review of the literature was conducted in January 2020 in Embase (including Medline) with no language, timeframe, or geography restrictions. Observational studies of ADPKD patients receiving tolvaptan were identified; outcomes of interest included clinical effectiveness and safety, healthcare resource utilization and costs, and quality of life (QoL). Results: A total of 43 relevant publications were identified. Studies were conducted in Canada, Japan, and across Europe with sample sizes ranging from a single case report to registry analyses of more than 1,000 patients. Clinical results from 6 studies reported a slowing of total kidney volume (TKV) growth and no significant changes in annual decline of estimated glomerular filtration rate (eGFR) over a range of 3 months to 2 years following tolvaptan initiation. Commonly reported adverse events included polyuria (˜10%) and liver function-related events (˜9%). Reported in 6 studies, 15.6% of patients discontinued tolvaptan treatment, primarily for aquaretic symptoms. Two studies reported that tolvaptan treatment did not appear, over a 1-year period, to negatively impact QoL, with more than 75% of patients reporting little impact on daily activities. No eligible economic studies were identified. Conclusions: Patients with ADPKD receiving tolvaptan in the real-world experienced improved clinical outcomes without negative impact on QoL. Additional studies assessing real-world evidence supporting tolvaptan treatment in this population are needed. Funding: Commercial Support - Otsuka Pharmaceuticals Development & Commercialization, Inc.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.026 | 0.078 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.004 | 0.004 |
| Bibliometrics | 0.006 | 0.008 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.004 | 0.002 |
| Open science | 0.001 | 0.002 |
| Research integrity | 0.002 | 0.002 |
| Insufficient payload (model declined to judge) | 0.005 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".