Improved JAK inhibition in myelofibrosis—the long road ahead
Mené sur 311 patients atteints d'une myélofibrose et souffrant d'une thrombocytopénie (âge moyen : 63,7 ans), cet essai de phase III compare l'efficacité, du point de vue du taux de réduction du volume de la rate, de la réduction de plus de 50 % des symptômes en semaine 24, et la toxicité du pacritinib, un inhibiteur de JAK2, et la meilleure thérapie disponible, notamment le ruxolitinib, un inihibiteur de JAK1 et JAK2
Myelofibrosis (MF) is characterized by bone marrow fibrosis, extramedullary hematopoiesis and abnormal cytokine expression, which often manifest with severe splenomegaly, crippling constitutional symptoms and cytopenias.2 The identification of the Janus kinase 2 (JAK2) V617F mutation and the subsequent approval of ruxolitinib for the treatment of intermediate-risk and high-risk MF by the US Food and Drug Administration (FDA) in 2011 changed the therapeutic landscape in MF.2 Ruxolitinib effectively reduced splenomegaly and improved constitutional symptoms and quality of life in patients with intermediate-2 or high International Prognostic Scoring System risk compared with placebo or best available therapy (BAT) in the randomized phase 3 COMFORT I and COMFORT II trials, respectively.2
JAMA Oncology , commentaire, 2017