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Paediatric Inflammatory Bowel Diseases Network for Safety, Efficacy, Treatment and Quality improvement of care

Periodic Reporting for period 3 - PIBD-SETQuality (Paediatric Inflammatory Bowel Diseases Network for Safety, Efficacy, Treatment and Quality improvement of care)

Reporting period: 2019-01-01 to 2019-12-31

The incidence, particularly of paediatric onset Inflammatory Bowel Diseases (PIBD, Crohn’s disease (CD) and ulcerative colitis (UC)) has risen dramatically in recent decades. A significant research gap at present involves identification of strategies to maximise therapeutic effectiveness without increasing risks from the treatment. The major goal of this proposal is the
1) Development of an accessible and feasible risk-stratified treatment algorithm for new onset paediatric IBD (based on an existing inception cohort) and validation of this algorithm in a new independent inception cohort
2) Generation of a homogenized high-powered and secured data base to collect and analyse longterm real world data of children with IBD (inception cohort) in a prospective registry designed to analyze effectiveness and safety signals and correlate them to individual risk factors (genetic, immunological and microbiological)
3) Design and performance of a risk algorithm-based prospective large-scale multicenter randomized clinical trial (RCT). For the first time a RCT will be based on a stratification of patients into high versus low risk groups for early complicated/relapsing disease based on predictors at presentation in order to provide optimal therapy (further optimized by drug monitoring), and using different treatment strategies in each arm.
To address the specific aims of this project three approaches devided into 11 work packages (plus 2 WP on project management and publication/communication/dissemination) were designed :
The search for risk factors for severe disease evolution in existing or historical patient cohorts of CD (WP1) and UC (WP2) is completed and validated allowing to create disease specific risk algorithms.
Hugh amount of preparationary work was invested in the organization of the randomized controlled clinical trial (RCT, WP3) with integrated pharmacological analyses (WP4), health economic analyses (WP8) and microbiological analyses (WP10). Efforts were concentrated on obtaining ethical and legal clearance in different countries: these were obtained in 11 countries and 58 sites have been activated.
In Q4 2018 a total of 62 patients have been included in the trial, with 46 patients in High Risk group and 16 patients in Low Risk Group.
The logistics for sampling and performing the pharmacological analyses within the RCT (WP4) is running as well as the Health economic and quality of life questionnaires (WP8).
A prerequisite for the generation of a longitudinal inception cohort (WP7) is a highly secured and stable IT system (WP6). The planned PIBD Cloud database is fully functional and under use since Q1 2017.Similarly to the RCT, the authorization process and ethical clearance for the Inception Cohort (WP7) took longer as initially expected. By December 31th 2018, the number of participating centers has increased to 15 centers. Next to these active centers 15 other centers are currently in the process of obtaining approval from their local ethical committees. The number of included patients has increased to 205. In 41 of those patients biomaterial was collected for immunological analysis of WP9. Similarly, for the genetic analyses (WP11) of patients developping rare complications, the methods (exome sequencing) are fully established and functional in the lab (partner 1, UDP), however, as per initial timing, the analyses did not yet start, since a sufficient number of patients needs to be obtained. First patients are recruited and samples stored for analysis.
WP 12 and 13 are dedicated to the coordination/management and communication/dissemination/ publication of the project’s achievement : the creation of the procets website is completed for external and also restricted internal use.
All preliminary steps are settled for this project and patient recruitement started to the RCT and to the inception cohort, however, results of the studies will be only available at the end of the inclusion/ follow-up period of the studies.
The following important potential impacts form PIBDSetQuality are expected :

Significantly decreasing treatment related risks (infections, new immune-mediated diseases and tumours/cancers) in the paediatric population

Establishing novel and/or more effective treatment schemes for healthcare interventions in the paediatric population
In summary, focussing PIBD-SETQuality on PIBD patients targets the most severe patients early in their lives and disease progression and can lead to major benefits in the short, medium and long term.
1. Individual benefits: Identification and improved treatment of patients at most risk of severe disease progression will have major impacts on improved health, well being, and quality of life of the individual. These will be demonstrated within both the RCT and the registry data.
2. The families: Improved health for an individual also benefits their family both in terms of psycosocial and economic outcomes, by the ability to lead ‘normal’ lives, retaining full employment and reducing stresses.
3. There is an positive impact to the local health economy by identifying effective and safe treatments directed at the patients who will most benefit from them with improved utilisation of scarce resources. This is a direct benefit translatable across all of Europe and beyond.
4. Educational achievement and job prospects are improved. By improving health and reducing side effects of treatment leads to improved school attendance, educational achievement and job prospects. This will benefit the broader economy in the long term by improving the productivity of the patients and their carers.
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