Low-dose ruxolitinib shows effective in treating myelofibrosis
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ORIGINAL ARTICLE
Low-dose ruxolitinib shows effective in treating myelofibrosis Yunfan Yang 1 & Hongmei Luo 1 & Yuhuan Zheng 1 & Zhongqing Zou 1 & Ting Niu 1 & Yongqian Jia 1 & Huanling Zhu 1 & Ting Liu 1 & Yu Wu 1 & Hong Chang 1 & Jie Ji 1 & Jian Li 1 & Ling Pan 1 Received: 16 August 2020 / Accepted: 15 October 2020 # Springer-Verlag GmbH Germany, part of Springer Nature 2020
Abstract The aim of this study was to investigate the effect of low-dose ruxolitinib (daily dose ≤ 10 mg) for the treatment of myelofibrosis (MF). A retrospective analysis was performed on a total of 88 patients with myeloproliferative neoplasm-associated MF (MPNMF) who were diagnosed and treated in West China Hospital, Sichuan University, China. A total of 44 MPN-MF patients received a low dose of ruxolitinib (daily dose ≤ 10 mg), while another 44 patients received 10–25 mg twice daily. Low-dose ruxolitinib treatment resulted in slow, but gradual spleen response. Compared with baseline, the mean changes in palpable spleen length in the low- and high-dose groups were −26.9 and −49.0% after 12 weeks of treatment, respectively, and −46.7 and −64.1% after 48 weeks of treatment, respectively. In the low dose group, the median myeloproliferative neoplasm symptom assessment form (MPN-SAF) total symptom score (TSS) decreased by 37.8 and 35.9% at the 12 weeks and 48 weeks after treatment, respectively. No statistical difference was observed in MPN-SAF TSS among different dose groups. After 48 weeks of treatment, bone marrow (BM) fibrosis improved in 43.3% (13/30) of evaluated patients and was stable in 56.7% (17/30) patients. In the lowdose treated group, BM fibrosis improved in 50% patients and was stable in remaining 50%. Low-dose ruxolitinib is effective in treating MF. Keywords Myelofibrosis . Ruxolitinib . Splenomegaly . Bone marrow fibrosis . Dose
Introduction Myelofibrosis (MF) is a Philadelphia chromosome-negative myeloproliferative neoplasm (MPN) that can be divided into primary MF (PMF), post-essential thrombocythemia MF (Post-ET MF), and post-polycythemia vera MF (Post-PV MF) [1, 2]. It usually manifests with the fibrosis of the bone marrow, splenomegaly, anemia, and thrombocytopenia and is characterized by symptoms, such as fever, weight loss, night sweats, fatigue, itchy skin, early satiety, and abdominal pain [3, 4]. The survival of MF patients varies greatly, and the median survival is approx. 5 years [5]. JAK/STAT cell signaling dysregulation is considered as a core feature of MF [6, 7]. Ruxolitinib is an oral JAK1/JAK2 inhibitor commonly used in the treatment of MF worldwide. A phase III Controlled Myelofibrosis Study with Oral JAK Inhibitor * Ling Pan [email protected] 1
Department of Hematology, Institute of Hematology, West China Hospital, Sichuan University, #37 GuoXue Xiang Street, 610041 Chengdu, China
Treatment (COMFORT)-I and COMFORT-II study revealed that ruxolitinib can improve splenomegaly and MF symptoms [8, 9]. Moreover, the long-term follow-up of two COMFORT studies showed that ruxolitinib could improve su
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