Sankalp Arora, Jayastu Senapati, Victoria Chu, Naveen Pemmaraju, Prithviraj Bose, Elias Jabbour, Tapan Kadia, Jing Ning, Dyana Saenz, Linghsa Zhou, Habiba Halim, Yesid Alvarado, Maro Ohanian, Courtney DiNardo, Koichi Takahashi, Nitin Jain, Farhad Ravandi, Hagop Kantarjian, Guillermo Garcia-Manero, Lucia Masarova, Naval Daver
Monotherapy with JAK2 inhibitors reduces splenomegaly and symptom burden but has limited effect on natural progression of myelofibrosis. We conducted a phase 2 clinical trial evaluating the combination of azacitidine (AZA) and ruxolitinib (RUX) in myelofibrosis. The interim analysis with 46 patients (median follow-up 28 months) demonstrated a response rate of 72%. We now report the final results of this clinical trial. The study primary endpoint was objective response rate by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria. From 3/2013 to 10/2021, 61 patients were treated (median age 66 years; range, 46-87 years). By baseline blast count, 54 patients had chronic phase disease (.