Abstract：
Patients (pts) with polycythemia vera (PV) suffer from pruritus, night sweats, and other symptoms, as well as from thromboembolic complications and progression to post-PV myelofibrosis. Ruxolitinib (RUX) is approved for second-line therapy in high-risk PV pts with hydroxyurea intolerance or resistance. The RuxoBEAT trial (NCT02577926, registered on October 1, 2015, at clinicaltrials.gov) is a multicenter, open-label, two-arm phase-IIb trial with a target population of 380 pts with PV or ET, randomized to receive RUX or best available therapy. This pre-specified futility analysis assesses the early clinical benefit and tolerability of RUX in previously untreated PV pts (6-week cytoreduction was allowed). Twenty-eight patients were randomly assigned to receive RUX. Compared to baseline, after 6 months of treatment, there was a significant reduction of median hematocrit (46 to 41%), the median number of phlebotomies per year (4.0 to 0), and median patient-reported pruritus scores (2 to 1), and a trend for reduced night sweat scores (1.5 to 0). JAK2V617F allele burden, as part of the scientific research program, also significantly decreased. One hundred nine adverse events (AEs) occurred in 24/28 patients (all grade 1 to 3), and no pt permanently discontinued treatment because of AEs. Thus, treatment with ruxolitinib in untreated PV pts is feasible, well-tolerated, and efficient regarding the above-mentioned endpoints.
摘要：
真性红细胞增多症（PV）患者（pts）患有瘙痒，盗汗,和其他症状，以及从血栓栓塞并发症和进展到肺静脉后骨髓纤维化。Ruxolitinib（RUX）被批准用于对羟基脲不耐受或耐药的高危PV患者的二线治疗。RuxoBEAT试验（NCT02577926，于2015年10月1日在clinicaltrials.gov注册）是一个多中心，开放标签，两臂IIb期试验，目标人群为380名PV或ET患者，随机接受RUX或最佳可用疗法。这种预先指定的无效性分析评估了RUX在先前未治疗的PVpts中的早期临床益处和耐受性（允许进行6周的细胞减少）。28名患者被随机分配接受RUX。与基线相比，经过6个月的治疗，中位血细胞比容显着降低（46％至41％），每年静脉切除术的中位数（4.0至0），和患者报告的瘙痒评分中位数（2比1），和减少夜间出汗分数的趋势（1.5至0）。JAK2V617F等位基因负荷，作为科学研究计划的一部分，也明显下降。24/28名患者（均为1至3级）发生了109起不良事件（AE），并且没有pt因为AE而永久停止治疗。因此，鲁索利替尼治疗未经治疗的PVpts是可行的，耐受性良好,并且对于上述端点是有效的。
