Ho P Joy, Bajel Ashish, Burbury Kate, Dunlop Lindsay, Durrant Simon, Forsyth Cecily, Perkins Andrew C, Ross David M
Royal Prince Alfred Hospital, and, Sydney, NSW, Australia.
University of Sydney, NSW, Sydney, NSW, Australia.
Intern Med J. 2017 Mar;47(3):262-268. doi: 10.1111/imj.13341.
Ruxolitinib is a dual janus kinase 1 (JAK1)/JAK2 inhibitor used to treat splenomegaly and symptoms associated with myelofibrosis (MF). Current therapeutic options for symptomatic MF include supportive care, myelosuppressive therapy (such as hydroxycarbamide) and janus kinase (JAK) inhibitors (in particular ruxolitinib). Allogeneic stem cell transplantation remains the only potentially curative treatment for MF, and younger transplant-eligible patients should still be considered for allogeneic stem cell transplantation; however, this is applicable only to a small proportion of patients. There is now increasing and extensive experience of the efficacy and safety of ruxolitinib in MF, both in clinical trials and in 'real-world' practice. The drug has been shown to be of benefit in intermediate-1 risk patients with symptomatic splenomegaly or other MF-related symptoms, and higher risk disease. Optimal use of the drug is required to maximise clinical benefit, requiring an understanding of the balance between dose-dependent responses and dose-limiting toxicities. There is also increasing experience in the use of ruxolitinib in the pre-transplantation setting. This paper aims to utilise several 'real-life' cases to illustrate several strategies that may help to optimise clinical practice.
芦可替尼是一种双重 Janus 激酶 1（JAK1）/JAK2 抑制剂，用于治疗脾肿大及与骨髓纤维化（MF）相关的症状。有症状 MF 的当前治疗选择包括支持性治疗、骨髓抑制治疗（如羟基脲）和 Janus 激酶（JAK）抑制剂（特别是芦可替尼）。异基因干细胞移植仍然是 MF 唯一可能治愈的治疗方法，年龄较轻且符合移植条件的患者仍应考虑进行异基因干细胞移植；然而，这仅适用于一小部分患者。目前，无论是在临床试验还是“真实世界”实践中，关于芦可替尼治疗 MF 的疗效和安全性都有越来越多且广泛的经验。该药物已被证明对有症状脾肿大或其他 MF 相关症状的中危 -1 风险患者以及高危疾病有益。需要优化使用该药物以最大化临床获益，这需要了解剂量依赖性反应和剂量限制性毒性之间的平衡。在移植前使用芦可替尼的经验也在增加。本文旨在利用几个“真实病例”来说明几种可能有助于优化临床实践的策略。
