Ruxolitinib is approved for the treatment of patients with polycythemia vera (PV) who are intolerant or resistant to hydroxyurea. While ruxolitinib discontinuation in myelofibrosis is associated with dismal outcomes, the analogous experience in PV has not been reported. Using a large, multi-institutional database of PV patients, we identified 93 patients with PV who were treated with ruxolitinib, of whom 22 discontinued therapy. Adverse events were the primary reason for discontinuation. After a median follow-up of 18.2 months following ruxolitinib discontinuation, no patients experienced a thrombotic event. One patient died 20.8 months after discontinuation. As compared with the 71 patients who were still receiving treatment with ruxolitinib at last follow up, patients who discontinued ruxolitinib were older at time of treatment initiation (67.5 versus 64.8 years, p = 0.0058), but had similar patient and disease characteristics. After discontinuation, only 4 patients (18 %) received subsequent cytoreductive therapy, including hydroxyurea in one patient and pegylated interferon α-2a in three patients. In stark contrast to the experience in myelofibrosis, discontinuation of ruxolitinib in PV was associated with generally favorable outcomes. However, there is a lack of available salvage therapies, highlighting the need for further therapeutic development in PV.

Ruxolitinib 被批准用于治疗对羟基脲不耐受或耐药的真性红细胞增多症 (PV) 患者。虽然在骨髓纤维化中停用鲁索替尼与令人沮丧的结果相关，但尚未报告在 PV 中的类似经历。使用大型、多机构的 PV 患者数据库，我们确定了 93 名接受鲁索替尼治疗的 PV 患者，其中 22 名停止治疗。不良事件是停药的主要原因。在停用鲁索替尼后中位随访 18.2 个月后，没有患者发生血栓事件。一名患者在停药后 20.8 个月死亡。与在最后一次随访时仍在接受鲁索替尼治疗的 71 名患者相比，停用鲁索替尼的患者在治疗开始时年龄较大（67. 5 岁与 64.8 岁，p = 0.0058），但具有相似的患者和疾病特征。停药后，只有 4 名患者 (18 %) 接受了后续的细胞减灭治疗，包括 1 名患者使用羟基脲和 3 名患者使用聚乙二醇干扰素 α-2a。与骨髓纤维化的经验形成鲜明对比的是，在 PV 中停用鲁索替尼与总体有利的结果相关。然而，缺乏可用的补救疗法，突出了 PV 进一步治疗开发的必要性。在 PV 中停用鲁索替尼与总体良好的结果相关。然而，缺乏可用的补救疗法，突出了 PV 进一步治疗开发的必要性。在 PV 中停用鲁索替尼与总体良好的结果相关。然而，缺乏可用的补救疗法，突出了 PV 进一步治疗开发的必要性。