title: Ruxolitinib treatment for myelofibrosis: Efficacy and tolerability in routine practice.
abstract: Ruxolitinib has been shown in two randomized clinical trials to be effective in alleviating systemic symptoms and reducing spleen size in patients with myelofibrosis (MF). We retrospectively evaluated efficacy and tolerability of ruxolitinib in a cohort of unselected MF patients treated in routine clinical practice. One hundred and two patients who began ruxolitinib therapy were identified in 13 participating centers. Ninety three of the patients receiving ruxolitinib for at least 3 months were evaluated for treatment efficacy and toxicity. Median age at ruxolitinib initiation was 67 years. Indications for treatment were constitutional symptoms (15%), symptomatic splenomegaly (6%) or both (76%). Two patients received ruxolitinib for other indications. The median initial ruxolitinib dose was 30mg/day. Median duration of therapy was 11 months. Eighty two patients (88.2%) responded to therapy, 76 (84.4%) patients had improvement in constitutional symptoms and 60 patients (70.6%) had reduction in spleen length. While on ruxolitinib, 30% of patients had grade 3-4 anemia and 12.9% of patients had grade 3-4 thrombocytopenia. Thirteen patients (14%) discontinued therapy. This analysis of a cohort of MF patients treated with ruxolitinib in routine clinical practice demonstrates the efficacy and tolerability of this drug outside of a highly monitored clinical trial setting.
authors.affiliation: Department of Hematology, Meir Medical Center, Kfar Saba, Israel; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
authors.forename: Martin H
authors.initials: MH
authors.lastname: Ellis
authors.affiliation: Bruce Rappaport Faculty of Medicine, Technion, Israel Institute of Technology, Haifa, Israel; Department of Hematology and Bone Marrow Transplantation, Rambam Health Care Campus, Haifa, Israel. Electronic address: lavi.noa@gmail.com.
authors.forename: Noa
authors.initials: N
authors.lastname: Lavi
authors.affiliation: Hematology Unit, Bnai-Zion Medical Center, Haifa, Israel.
authors.forename: Elena
authors.initials: E
authors.lastname: Mishchenko
authors.affiliation: Department of Hematology, Ziv Medical Center Associated with the Bar Ilan University, Faculty of Medicine, Safed, Israel.
authors.forename: Najib
authors.initials: N
authors.lastname: Dally
authors.affiliation: Department of Hematology and Bone Marrow Transplantation, Hebrew University-Hadassah Medical Organization, Jerusalem, Israel.
authors.forename: David
authors.initials: D
authors.lastname: Lavie
authors.affiliation: Department of Hematology and Bone Marrow Transplantation, Soroka Medical Center, Beer-Sheva, Israel.
authors.forename: Anna
authors.initials: A
authors.lastname: Courevitch
authors.affiliation: Department of Hematology, Assaf Harofeh Medical Center, Ramla, Israel.
authors.forename: Odit
authors.initials: O
authors.lastname: Gutwein
authors.affiliation: Department of Hematology, Laniado Hospital-Sanz Medical Center, Netanya, Israel.
authors.forename: Shlomo
authors.initials: S
authors.lastname: Bulvik
authors.affiliation: Department of Hematology, Western Galilee Hospital, Nahariya, Israel.
authors.forename: Andrei
authors.initials: A
authors.lastname: Braester
authors.affiliation: Department of Hematology, The Emek Medical Center, Afula, Israel.
authors.forename: Evgeni
authors.initials: E
authors.lastname: Chubar
authors.affiliation: Maccabi Health Organization, Tel Aviv, Israel.
authors.forename: Sigal
authors.initials: S
authors.lastname: Tavor
authors.affiliation: Division of Hematology and Bone Marrow Transplantation, Sheba Medical Center, Tel Hashomer, Israel.
authors.forename: Adrian
authors.initials: A
authors.lastname: Duek
authors.affiliation: Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel; Department of Hematology, Tel Aviv Sourasky Medical Center, Tel Aviv, Israel.
authors.forename: Ilya
authors.initials: I
authors.lastname: Kirgner
authors.affiliation: Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel; Division of Hematology and Bone Marrow Transplantation, Sheba Medical Center, Tel Hashomer, Israel.
authors.forename: Maya
authors.initials: M
authors.lastname: Koren-Michowitz
keywords: Myelofibrosis; Population study; Ruxolitinib