title: Clinical use of ruxolitinib in an academic medical center in unselected patients with myeloproliferative neoplasms not on clinical study.
abstract: Ruxolitinib is the only approved therapy for myelofibrosis (MF). However, its use in patients with myeloproliferative neoplasms (MPN) not participating in clinical studies has been poorly described. We reviewed the medical records of 45 patients (35 MF, 10 others) treated with ruxolitinib at our center, off clinical study, during the year after its approval. Patients had advanced features and were refractory to multiple therapies. Ruxolitinib was effective in reducing splenomegaly (51% response rate) and constitutional symptoms (42% response rate). It controlled blood counts in patients with polycythemia and thrombocythemia but was not effective in patients with non-classic MPNs. Ruxolitinib was an active therapy in patients previously treated with a JAK inhibitor and was safely combined with hypomethylating agents in patients with elevated blasts. Median overall survival was 24 months; 10 patients transformed to acute leukemia. Its use in combination with other active agents should be further explored in clinical studies.
authors: a Department of Leukemia , The University of Texas MD Anderson Cancer Center , Houston , TX , USA.
authors: Kiran
authors: K
authors: Naqvi
authors: a Department of Leukemia , The University of Texas MD Anderson Cancer Center , Houston , TX , USA.
authors: Naval
authors: N
authors: Daver
authors: a Department of Leukemia , The University of Texas MD Anderson Cancer Center , Houston , TX , USA.
authors: Naveen
authors: N
authors: Pemmaraju
authors: a Department of Leukemia , The University of Texas MD Anderson Cancer Center , Houston , TX , USA.
authors: Prithviraj
authors: P
authors: Bose
authors: a Department of Leukemia , The University of Texas MD Anderson Cancer Center , Houston , TX , USA.
authors: Guillermo
authors: G
authors: Garcia-Manero
authors: a Department of Leukemia , The University of Texas MD Anderson Cancer Center , Houston , TX , USA.
authors: Jorge
authors: J
authors: Cortes
authors: a Department of Leukemia , The University of Texas MD Anderson Cancer Center , Houston , TX , USA.
authors: Hagop M
authors: HM
authors: Kantarjian
authors: a Department of Leukemia , The University of Texas MD Anderson Cancer Center , Houston , TX , USA.
authors: Srdan
authors: S
authors: Verstovsek
mesh_terms: D000046:Academic Medical Centers; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D053614:Janus Kinase 2 / Q000037:antagonists & inhibitors / Q000235:genetics; D008297:Male; D008875:Middle Aged; D009154:Mutation; D009196:Myeloproliferative Disorders / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics; D009570:Nitriles; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*; D011720:Pyrazoles / Q000627:therapeutic use*; D011743:Pyrimidines; D011788:Quality of Life; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D009570:Nitriles; D047428:Protein Kinase Inhibitors; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib; D053614:Janus Kinase 2
keywords: Myelofibrosis; myeloproliferative neoplasms; ruxolitinib; splenomegaly