ArticleScientific reports2018
In silico clinical trials for pediatric orphan diseases.
Article in Scientific reports, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers, 1 of them a synthesis that pooled 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.
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.
Who cites it
22 citing papers in PubMed, 1 synthesis or guideline pooled it, 45 citations in OpenAlex.
- In Silico Clinical Trials in Drug Development: A Systematic Review.Therapeutic innovation & regulatory science · 2026Pooled it
- A Step-by-Step Workflow for Performing In Silico Clinical Trials With Nonlinear Mixed Effects Models.CPT: pharmacometrics & systems pharmacology · 2025Article
- Advancing the frontier of rare disease modeling: a critical appraisal of in silico technologies.NPJ digital medicine · 2025Review
- Comparison of machine learning models for mucopolysaccharidosis early diagnosis using UAE medical records.Scientific reports · 2025Article
- Review
- Review
- Digital twins, synthetic patient data, and in-silico trials: can they empower paediatric clinical trials?The Lancet. Digital health · 2025Review
- Reform progress and achievements of China's incentive policies for pediatric medicine over the last decade.Frontiers in pharmacology · 2025Article
- Advancing rare disease therapeutics through digital twins: Opportunities in drug development and precision dosing.Computational and structural biotechnology journal · 2025Review
- Artificial Intelligence (AI): A Potential Game Changer in Regenerative Orthopedics-A Scoping Review.Indian journal of orthopaedics · 2024Article
- The Impact of Artificial Intelligence on Optimizing Diagnosis and Treatment Plans for Rare Genetic Disorders.Cureus · 2023Review
- In silico cancer immunotherapy trials uncover the consequences of therapy-specific response patterns for clinical trial design and outcome.Nature communications · 2023Article
- Mapping the use of computational modelling and simulation in clinics: A survey.Frontiers in medical technology · 2023Article
- De-risking clinical trial failure through mechanistic simulation.Immunotherapy advances · 2022Article
- Bayesian Augmented Clinical Trials in TB Therapeutic Vaccination.Frontiers in medical technology · 2021Article
- In silico trial to test COVID-19 candidate vaccines: a case study with UISS platform.BMC bioinformatics · 2020Article
- Tackling rare diseases: Clinical trials on chips.Experimental biology and medicine (Maywood, N.J.) · 2020Review
- Predicting the artificial immunity induced by RUTI® vaccine against tuberculosis using universal immune system simulator (UISS).BMC bioinformatics · 2019Article
- Singlet oxygen model evaluation of interstitial photodynamic therapy with 5-aminolevulinic acid for malignant brain tumor.Journal of biomedical optics · 2019Article
- Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 3 institutions in 2 countries.
Funding
Abstract
To date poor treatment options are available for patients with congenital pseudarthrosis of the tibia (CPT), a pediatric orphan disease. In this study we have performed an in silico clinical trial on 200 virtual subjects, generated from a previously established model of murine bone regeneration, to tackle the challenges associated with the small, pediatric patient population. Each virtual subject was simulated to receive no treatment and bone morphogenetic protein (BMP) treatment. We have shown that the degree of severity of CPT is significantly reduced with BMP treatment, although the effect is highly subject-specific. Using machine learning techniques we were also able to stratify the virtual subject population in adverse responders, non-responders, responders and asymptomatic. In summary, this study shows the potential of in silico medicine technologies as well as their implications for other orphan diseases.
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What OpenQuestion holds
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.