Selumetinib in paediatric low-grade glioma: a new era?
Mené sur 50 patients pédiatriques (âge : de 3 à 21 ans) atteints d'un gliome de bas grade, récidivant, réfractaire ou en progression, présentant une aberration du gène BRAF (fusion KIAA1549 ou mutation V600E) ou associé à une neurofibomatose de type 1, cet essai de phase II évalue l'efficacité, du point de vue de la proportion de patients obtenant une réponse objective pendant au moins 8 semaines, et la toxicité du sélumétinib, un inhibiteur de MEK1/2
Since the first publication on the activity of the carboplatin–vincristine combination in paediatric low-grade glioma in 1993, chemotherapy has become the mainstay of treatment for this condition, particularly in young children (aged 0–10 years). However, 5-year event-free survival in the non-neurofibromatosis type 1 (NF1) population is in the range of 40% and most patients require several lines of treatment. Patients with NF1 have a longer event-free survival than those without NF1, ranging between 65% and 85%. No chemotherapy protocol has shown superior activity, and although the chemotherapeutic management of paediatric low-grade glioma varies between cooperative groups, countries, and institutions, overall, it follows the same principles.
The Lancet Oncology , commentaire, 2018