文章：

沙利度胺联合泼尼松加或不加达那唑治疗骨髓纤维化：贫血反应发生率及持久性的回顾性分析

Thalidomide plus prednisone with or without danazol therapy in myelofibrosis: a retrospective analysis of incidence and durability of anemia response

原文发布日期：2018-01-15

DOI: 10.1038/s41408-017-0029-4

类型: Article

开放获取: 是

英文摘要：

Low-dose thalidomide and prednisone alone or combined are effective therapies in some persons with primary myelofibrosis (PMF) and anemia with or with RBC transfusion dependence. Danazol is also effective in some persons with PMF and anemia. Responses to these drugs are typically incomplete and not sustained. It is unclear whether adding danazol to thalidomide and prednisone would improve efficacy. We retrospectively compared the outcomes of 88 subjects with PMF and anemia receiving thalidomide and prednisone without (n = 46) or with danazol (n = 42). The primary end point was anemia response, which was 71% (95% confidence interval (CI), 57, 85%) in subjects receiving thalidomide/prednisone/danazol compared with 46% (32, 60%; P = 0.014) in those receiving thalidomide/prednisone. Response rates in subjects who were RBC transfusion dependent was also higher in the danazol cohort (61% (38, 84%)) vs. 25% (6, 44%); P = 0.024). Time to response was rapid (median, 2 months (range, 1–11 months)) and similar between the cohorts. Response duration was longer in the thalidomide/prednisone/danazol cohort (HR 2.18 (1.18–5.42); P = 0.019). Adverse effects were mild and similar between the cohorts. In conclusion, thalidomide/prednisone/danazol seems superior to thalidomide/prednisone in persons with PMF and anemia. Our conclusion requires confirmation in a randomized trial.

摘要翻译： 

低剂量沙利度胺与泼尼松单用或联用，对部分原发性骨髓纤维化（PMF）伴贫血（无论是否依赖红细胞输注）患者具有疗效。达那唑对部分PMF伴贫血患者亦有效。这些药物的疗效通常不完全且不持续。目前尚不清楚在沙利度胺和泼尼松基础上加用达那唑是否能提升疗效。我们回顾性比较了88例接受沙利度胺联合泼尼松治疗（46例）或联合达那唑治疗（42例）的PMF伴贫血患者的结局。主要终点为贫血反应率：沙利度胺/泼尼松/达那唑组达71%（95%置信区间[CI]为57%-85%），而沙利度胺/泼尼松组为46%（95%CI为32%-60%；P=0.014）。在红细胞输注依赖患者中，达那唑组缓解率更高（61%[95%CI为38%-84%]对比25%[95%CI为6%-44%]；P=0.024）。两组起效时间均较快（中位时间2个月，范围1-11个月）且无显著差异。沙利度胺/泼尼松/达那唑组缓解持续时间更长（风险比[HR]为2.18[95%CI为1.18-5.42]；P=0.019）。两组不良反应均较轻微且发生率相近。结论：对于PMF伴贫血患者，沙利度胺/泼尼松/达那唑联合方案优于沙利度胺/泼尼松方案。该结论需通过随机试验进一步验证。

原文链接：

Thalidomide plus prednisone with or without danazol therapy in myelofibrosis: a retrospective analysis of incidence and durability of anemia response