[«KCE Trials¼ : innovation based on practice oriented clinical trials]. / «KCE Trials¼ : des essais cliniques plus proches de la pratique.

Many questions in healthcare can only be answered after the conduct of clinical trials. For medicinal products and medical devices the industry finances most studies to bring their products to market. However, there is a need for further research in certain areas e.g. children, older people and comparative research of different treatment options (comparative effectiveness). In addition, interventions that are less industry-driven such as surgery, radiotherapy, psychotherapy, diet, physical medicine, needappropriately funded large scale clinical trials. Such clinical trials can be a good investment for the government and the healthcare payer. At the end of 2015 the Belgian Healthcare Knowledge Centre (KCE) received the mission and budget to run a programme of practice-oriented comparative clinical trials. Two years later the recruitment of patients in the first trials is ongoing. In addition to its yearly national calls for trial proposals, early in 2018 KCE launched its first international common call for comparative clinical trials with its Dutch counterpart ZonMw (BeNeFIT).

Les études cliniques financées par l'industrie, essentiellement effectuées dans le but d'obtenir la mise sur le marché de médicaments et de dispositifs médicaux, laissent de nombreuses questions cliniques sans réponse satisfaisante. Ainsi, par exemple, les produits y sont généralement comparés à un placebo, alors que la question réellement pertinente pour le clinicien serait une comparaison avec d'autres options thérapeutiques. De même, les domaines qui présentent un intérêt moindre pour l'industrie sont rarement explorés par des études à large échelle; c'est le cas, notamment, de la chirurgie, de la radiothérapie, des psychothérapies, de l'alimentation et de la médecine physique. Un programme d'études cliniques non commerciales, financé par les pouvoirs publics, permet de remédier à ces problèmes tout en constituant un excellent investissement pour les autorités de santé et le contribuable. Fin 2015, le Centre fédéral d'Expertise des Soins de Santé (KCE) a été chargé de mettre sur pied un tel programme d'études cliniques comparatives et axées sur la pratique. Deux ans plus tard, le recrutement des premiers essais bat son plein. Un appel annuel à sujets d'études a été mis en place et, début 2018, un premier appel international conjoint du KCE et de son homologue néerlandais le ZonMw a également été lancé (BeNeFIT).

Clustering of increased small intestinal permeability in families with Crohn's disease.

BACKGROUND & AIMS: Small intestinal permeability is increased in a proportion of patients with Crohn's disease (CD) and a subset of their healthy relatives. A primary permeability defect was postulated in the pathogenesis of the disease. The aim of this study was to identify a possible genetic pattern in the distribution of CD and/or abnormal permeability. METHODS: Differential urinary excretion of lactulose and mannitol (L/ M) in complete CD families was determined. Controls included healthy families and families with ulcerative colitis. Pedigrees were used to compare the distribution of CD and/or increased permeability. RESULTS: The L/M was significantly increased in patients with CD. Seventeen of 67 first-degree relatives (25%) had a ratio greater than the upper limit (P95 = 0.0170). Permeability results of CD families showed a highly significant familial aggregation. The lack of a genetic pattern in relation with CD and occurrence of disturbed permeability especially within generation, points toward a shared environmental factor. Five of 14 healthy spouses (36%) of patients with CD had also an increased permeability, and prevalence of increased permeability was not higher in families with known familial occurrence (P = 0.85). CONCLUSIONS: This large family study confirms an increased permeability in a subset of healthy relatives of patients with CD. However, the absence of a typical family pattern and the high prevalence in spouses is in favor of a common nongenetic factor or a subclinical disease manifestation.

Familial aggregation in Crohn's disease: increased age-adjusted risk and concordance in clinical characteristics.

BACKGROUND & AIMS: Because the mode of Crohn's disease inheritance is unknown, age-adjusted risk estimates and knowledge of disease characteristics will aid genetic counseling and modeling. The aim of this study is to determine the prevalence of familial occurrence of inflammatory bowel disease in first-degree relatives of patients with Crohn's disease and estimate their age-adjusted risks. It also evaluates agreement in disease characteristics between generations within families with a history of Crohn's disease. METHODS: Six hundred forty patients with Crohn's disease and 800 control subjects were questioned about the occurrence of inflammatory bowel disease in their first-degree relatives. Agreement for age at diagnosis, initial disease location, disease behavior, and number of bowel resections was determined in 68 families with two or more members affected and compared with data in 100 unrelated patients with Crohn's disease. RESULTS: Probands with Crohn's disease had a more frequent positive family history than controls. The age at diagnosis between probands with and without a positive family history was insignificant. Crude and age-adjusted risk elements were higher in relatives of patients, especially daughters, compared with those of controls. The age at diagnosis was older for parents than offspring but similar between siblings. Initial disease location was especially striking between siblings. CONCLUSIONS: This study confirms familial aggregation and a high degree of disease concordance in Crohn's disease. The age at diagnosis and initial disease location was especially strong within generations.