In silico clinical trials for pediatric orphan diseases

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

Onderzoeksoutput: Bijdrage aan tijdschriftTijdschriftartikelAcademicpeer review

14 Citaten (Scopus)
16 Downloads (Pure)


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.

Originele taal-2Engels
Aantal pagina's9
TijdschriftScientific Reports
Nummer van het tijdschrift1
StatusGepubliceerd - 1 dec 2018
Extern gepubliceerdJa


Duik in de onderzoeksthema's van 'In silico clinical trials for pediatric orphan diseases'. Samen vormen ze een unieke vingerafdruk.

Citeer dit