Journal Scans
Safety and Efficacy of Everolimus, a mTOR Inhibitor, as Single Agent in a Phase 1/2 Study in Patients With Myelofibrosis
Phase 1/2 Results Promising for Everolimus in Myelofibrosis
P Guglielmelli, G Barosi, A Rambaldi, R Marchioli, A Masciulli, L Tozzi, F Biamonte, N Bartalucci, E Gattoni, ML Lupo, G Finazzi, A Pancrazzi, E Antonioli, MC Susini, L Pieri, E Malevolti, E Usala, U Occhini, A Grossi, S Caglio, S Paratore, A Bosi, T Barbui, AM Vannucchi
20110601
2011 Jul 1
Blood
Safety and Efficacy of Everolimus, a mTOR Inhibitor, as Single Agent in a Phase 1/2 Study in Patients With Myelofibrosis
Blood. 2011 Jul 1;[Epub Ahead of Print], P Guglielmelli, G Barosi, A Rambaldi, R Marchioli, A Masciulli, L Tozzi, F Biamonte, N Bartalucci, E Gattoni, ML Lupo, G Finazzi, A Pancrazzi, E Antonioli, MC Susini, L Pieri, E Malevolti, E Usala, U Occhini, A Grossi, S Caglio, S Paratore, A Bosi, T Barbui, AM Vannucchi
TAKE-HOME MESSAGE
Abstract
Abstract
In addition to dysregulated JAK/STAT signaling, activation of AKT/mTOR pathway occurs in myelofibrosis, a myeloproliferative neoplasm with no approved therapies. We conducted a phase 1/2 study with everolimus, a mTOR inhibitor, in 39 high- or intermediate-risk primary or post-polycythemia vera/post-essential thrombocythemia myelofibrosis subjects. Responses were evaluated in 30 patients of phase 2. No dose limiting toxicity was observed in phase 1 up to 10 mg/day. Using this dose in phase 2, grade...
