In silico clinical trials for pediatric orphan diseases

A. Carlier, Aliaksei S. Vasilevich, M. Marechal, Jan de Boer, L. Geris (Corresponding author)

Research output: Contribution to journalArticleAcademicpeer-review

14 Citations (Scopus)
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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.

Original languageEnglish
Article number2465
Number of pages9
JournalScientific Reports
Issue number1
Publication statusPublished - 1 Dec 2018
Externally publishedYes


  • Adolescent
  • Biomarkers, Pharmacological/metabolism
  • Bone Morphogenetic Proteins/therapeutic use
  • Bone Regeneration/drug effects
  • Case-Control Studies
  • Child
  • Clinical Trials as Topic
  • Female
  • Humans
  • Male
  • Pseudarthrosis/congenital
  • Rare Diseases/drug therapy
  • Tibia/drug effects
  • Virtual Reality


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