Everolimus for astrocytomas in tuberous sclerosis complex
Mené sur 117 patients atteints d'un astrocytome subépendymal à cellules géantes associé à une sclérose tubéreuse de Bourneville, cet essai international multicentrique de phase III évalue l'efficacité et la toxicité de l'évérolimus, un inhibiteur de la voie mTOR
Tuberous sclerosis complex is a variegated complex disease characterised by the occurrence of benign hamartomas in several organs, including skin, kidney, lung, and brain, and neuropsychological complications such as mental retardation, autism, and epilepsy. This autosomal-dominant inherited disease has a prevalence of one in 6000 individuals and a heterogeneous phenotype. Subependymal giant cell astrocytomas, slow-growing glioneuronal tumours, are a neurological manifestation affecting 5—15% of ...
The Lancet , commentaire, 2011