Douglas Genevieve, Harrison Claire, Forsyth Cecily, Bennett Michael, Stevenson William, Hounsell John, Ratnasingam Sumita, Ritchie David, Ross David M, Grigg Andrew
a Department of Clinical Haematology , Austin Hospital, University of Melbourne , Heidelberg , VIC , Australia.
b Department of Haematology , Guys and St Thomas' NHS Foundation Trust , London , UK.
Leuk Lymphoma. 2017 Jan;58(1):89-95. doi: 10.1080/10428194.2016.1187269. Epub 2016 Jul 25.
Hydroxyurea (Hu) is widely used as first-line cytoreductive therapy for patients with high-risk Philadelphia-negative myeloproliferative neoplasms (Ph-neg MPN), but a small proportion of patients have refractory disease or experience adverse effects. Studies have demonstrated busulfan (Bu) to be an active first-line agent, but data on its role as second-line or later therapy are minimal. To evaluate its efficacy and safety in this context, we undertook a multicenter audit of Ph-neg MPN patients who had received Bu as therapy for Hu intolerance or failure. Of 51 patients identified, 38 (75%) achieved either complete or partial hematological response following at least one Bu cycle. Bu was generally well tolerated, with only 21/135 (15%) cycles complicated by adverse effects, predominantly cytopenia; only 6% of cycles were ceased due to treatment complications. Bu is an effective and well-tolerated agent in patients with Ph-neg MPN in the setting of Hu intolerance or unresponsiveness.
羟基脲（Hu）被广泛用作高危费城阴性骨髓增殖性肿瘤（Ph阴性MPN）患者的一线细胞减灭疗法，但一小部分患者患有难治性疾病或出现不良反应。研究表明白消安（Bu）是一种有效的一线药物，但关于其作为二线或后续治疗作用的数据极少。为了评估其在这种情况下的疗效和安全性，我们对因不耐受Hu或Hu治疗失败而接受Bu治疗的Ph阴性MPN患者进行了一项多中心审核。在确定的51例患者中，38例（75%）在至少一个Bu疗程后达到完全或部分血液学缓解。Bu总体耐受性良好，仅21/135（15%）个疗程出现不良反应，主要是血细胞减少；仅6%的疗程因治疗并发症而停止。在Hu不耐受或无反应的情况下，Bu是治疗Ph阴性MPN患者的一种有效且耐受性良好的药物。
