Towards personalized medicine in pediatric inflammatory bowel disease

    Research output: ThesisThesis fully internal (DIV)

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    Abstract

    In this thesis we aimed to contribute to the further development of personalized medicine in children and adolescents with inflammatory bowel disease (IBD). We focused on two different aspects: (1) the potential role of periodically measuring stool calprotectin levels, to see whether calprotectin can also be used to monitor response to treatment; and (2) profiling patients with pediatric-onset primary sclerosing cholangitis (PSC), a rare but severe hepatobiliairy disease that is strongly associated with IBD.
    Using periodically measured stool calprotectin levels to monitor response-to-treatment is a relatively new application of this diagnostic test. In patients receiving remission-induction therapy, a shift of calprotectin concentrations into the target range appears to be a good surrogate marker for mucosal healing. Reaching the target calprotectin range within 3 months after starting the treatment predicts a favourable disease course in children with Crohn’s disease. Vice versa, patients who fail to reach target calprotectin levels within 3 months after conventional induction therapy may be entitled to step-up to a more aggressive treatment.
    Until recently, childhood-onset PSC was believed to present with milder disease and a more favourable outcome than adult-onset PSC. By evaluating time-to-complication in children with PSC up until adulthood, we showed that paediatric- and adult-onset PSC run a similar progressive disease course. Furthermore, examination of the genetic material (DNA) of children with PSC and their biological parents revealed multiple rare candidate disease-causing variants.
    Original languageEnglish
    QualificationDoctor of Philosophy
    Awarding Institution
    • University of Groningen
    Supervisors/Advisors
    • Verkade, Henkjan, Supervisor
    • van Rheenen, Patrick, Co-supervisor
    Award date9-Oct-2019
    Place of Publication[Groningen]
    Publisher
    Print ISBNs978-94-6375-552-8
    Electronic ISBNs978-94-6375-553-5
    DOIs
    Publication statusPublished - 2019

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