ArticleCPT: pharmacometrics & systems pharmacology2020
A Quantitative Systems Pharmacology Model of Gaucher Disease Type 1 Provides Mechanistic Insight Into the Response to Substrate Reduction Therapy With Eliglustat.
Article in CPT: pharmacometrics & systems pharmacology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis that pooled it.
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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.
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Who cites it
14 citing papers in PubMed, 1 synthesis or guideline pooled it, 24 citations in OpenAlex.
- Recent applications of quantitative systems pharmacology and machine learning models across diseases.Journal of pharmacokinetics and pharmacodynamics · 2022Pooled it
- From Small Data to Big Decisions: How Clinical Pharmacology Shapes Rare Disease Development.Journal of clinical pharmacology · 2026Review
- What Is a Digital Twin in QSP, and Are We Doing It Right?CPT: pharmacometrics & systems pharmacology · 2026Article
- QSP-Copilot: An AI-Augmented Platform for Accelerating Quantitative Systems Pharmacology Model Development.CPT: pharmacometrics & systems pharmacology · 2025Article
- Transforming Pediatric Rare Disease Drug Development: Enhancing Clinical Trials and Regulatory Evidence With Virtual Patients.CPT: pharmacometrics & systems pharmacology · 2025Article
- Unlocking the Mysteries of Rare Disease Drug Development: A Beginner's Guide for Clinical Pharmacologists.Clinical and translational science · 2025Review
- Landscape of regulatory quantitative systems pharmacology submissions to the U.S. Food and Drug Administration: An update report.CPT: pharmacometrics & systems pharmacology · 2024Article
- Advancing precision medicine therapeutics for Parkinson's utilizing a shared quantitative systems pharmacology model and framework.Frontiers in systems biology · 2024Article
- Systems biology platform for efficient development and translation of multitargeted therapeutics.Frontiers in systems biology · 2023Article
- Quantitative system pharmacology as a legitimate approach to examine extrapolation strategies used to support pediatric drug development.CPT: pharmacometrics & systems pharmacology · 2022Review
- Long-term eliglustat treatment of Gaucher patients over up to 10 years in Vienna.Wiener klinische Wochenschrift · 2022Article
- QSPcc reduces bottlenecks in computational model simulations.Communications biology · 2021Article
- History and Future Perspectives on the Discipline of Quantitative Systems Pharmacology Modeling and Its Applications.Frontiers in physiology · 2021Review
- Reconstruction of the Cytokine Signaling in Lysosomal Storage Diseases by Literature Mining and Network Analysis.Frontiers in cell and developmental biology · 2021Article
Corrections and comments
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Authors and funding
16 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gaucher's disease type 1 (GD1) leads to significant morbidity and mortality through clinical manifestations, such as splenomegaly, hematological complications, and bone disease. Two types of therapies are currently approved for GD1: enzyme replacement therapy (ERT), and substrate reduction therapy (SRT). In this study, we have developed a quantitative systems pharmacology (QSP) model, which recapitulates the effects of eliglustat, the only first-line SRT approved for GD1, on treatment-naïve or patients with ERT-stabilized adult GD1. This multiscale model represents the mechanism of action of eliglustat that leads toward reduction of spleen volume. Model capabilities were illustrated through the application of the model to predict ERT and eliglustat responses in virtual populations of adult patients with GD1, representing patients across a spectrum of disease severity as defined by genotype-phenotype relationships. In summary, the QSP model provides a mechanistic computational platform for predicting treatment response via different modalities within the heterogeneous GD1 patient population.
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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 Socratic graph.