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[低剂量沙利度胺与泼尼松联合或不联合达那唑治疗原发性骨髓纤维化相关性贫血的比较]

[Comparison of low-dose thalidomide and prednisone combined with or without danazol for the treatment of primary myelofibrosis-associated anemia].

作者信息

Xu Junqing, Xu Zefeng, Qin Tiejun, Li Bing, Fang Liwei, Zhang Hongli, Hu Naibo, Pan Lijuan, Qu Shiqiang, Zhang Yue, Xiao Zhijian

机构信息

Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, The State Key Laboratory of Experimental Hematology, Tianjin 300020, China.

出版信息

Zhonghua Xue Ye Xue Za Zhi. 2014 Aug;35(8):698-702. doi: 10.3760/cma.j.issn.0253-2727.2014.08.006.

Abstract

OBJECTIVE

To observe the clinical effects of low-dose thalidomide (THAL) and prednisone (PRED) with or without danazol (DANA) in patients with primary myelofibrosis (PMF) associated anemia.

METHODS

A cohort of 58 PMF patients with anemia (Hb<100 g/L) were retrospectively studied. Of them, 28 patients were treated with THAL and PRED (THAL-PRED group), and the rest with THAL, PRED and DANA (THAL-PRED-DANA group). The hematological response was assessed according to the modified criteria of the International Working Group in 2006, and the myelofibrosis degree was evaluated at 3 and 12 month after treatment.

RESULTS

The total response rate was 56.9%(33/58) including 1.7% (1/58) partial remission (PR) and 55.2% (32/58) clinical improvement (CI). There was no statistical difference in the response rate between THAL-PRED and THAL-PREDDANA groups (50.0% vs 63.3%, P=0.306). However, the median response duration of clinical improvement, erythroid response (CI-E) and total response prolonged in THAL-PRED-DANA than THALPRED group (61.5w vs 22w, P=0.015; 75w vs 30w, P=0.007, respectively). Myelofibrosis degree at 3 and 12 months after treatment decreased significantly than before treatment (P=0.000 and 0.005, respectively). Side-effects in both groups were only grade 1-2.

CONCLUSION

Low-dose THAL together with PRED appeared to be effective in the treatment of PMF-associated anemia, and the response duration would prolong significantly if combined with DANA.

摘要

目的

观察低剂量沙利度胺(THAL)联合泼尼松(PRED),以及联合或不联合达那唑(DANA)治疗原发性骨髓纤维化(PMF)相关贫血患者的临床疗效。

方法

回顾性研究58例贫血(血红蛋白<100 g/L)的PMF患者。其中,28例患者接受THAL和PRED治疗(THAL-PRED组),其余患者接受THAL、PRED和DANA治疗(THAL-PRED-DANA组)。根据2006年国际工作组的修订标准评估血液学反应,并在治疗后3个月和12个月评估骨髓纤维化程度。

结果

总缓解率为56.9%(33/58),包括1.7%(1/58)的部分缓解(PR)和55.2%(32/58)的临床改善(CI)。THAL-PRED组和THAL-PRED-DANA组的缓解率无统计学差异(50.0%对63.3%,P = 0.306)。然而,THAL-PRED-DANA组临床改善、红系反应(CI-E)和总反应的中位缓解持续时间比THAL-PRED组延长(分别为61.5周对22周,P = 0.015;75周对30周,P = 0.007)。治疗后3个月和12个月的骨髓纤维化程度比治疗前显著降低(分别为P = 0.000和0.005)。两组的副作用均仅为1-2级。

结论

低剂量THAL联合PRED似乎对PMF相关贫血有效,若联合DANA,缓解持续时间将显著延长。

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