In silico clinical trials for pediatric orphan diseases.

[en] 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.

Disciplines :

Engineering, computing & technology: Multidisciplinary, general & others

Author, co-author :

Carlier, Aurélie

Vasilevich, A.

de Boer, J.

Geris, Liesbet ; Université de Liège - ULiège > Département d'aérospatiale et mécanique > Génie biomécanique

Other collaborator :

Maréchal, Marina; KU Leuven

Language :

English

Title :

In silico clinical trials for pediatric orphan diseases.

Publication date :

2018

Journal title :

Scientific Reports

eISSN :

2045-2322

Publisher :

Nature Publishing Group, London, United Kingdom

Volume :

Issue :

Pages :

2465

Peer reviewed :

Peer Reviewed verified by ORBi

European Projects :

FP7 - 279100 - BRIDGE - Biomimetic process design for tissue regeneration: from bench to bedside via in silico modelling

Funders :

CE - Commission Européenne [BE]

Available on ORBi :

since 18 June 2018

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