Xu Junqing, Xu Zefeng, Qin Tiejun, Li Bing, Fang Liwei, Zhang Hongli, Hu Naibo, Pan Lijuan, Qu Shiqiang, Zhang Yue, Xiao Zhijian
Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, The State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
Zhonghua Xue Ye Xue Za Zhi. 2014 Aug;35(8):698-702. doi: 10.3760/cma.j.issn.0253-2727.2014.08.006.
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.
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.
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.
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.
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。
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。
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。
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。
