Increment of prednisone dosage improves the response rate of myelofibrosis-associated anemia
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摘要: 目的 观察提高泼尼松剂量单药治疗骨髓纤维化贫血的疗效。方法 回顾性分析接受较高剂量泼尼松单药治疗20例伴有贫血的骨髓纤维化患者的临床资料，主要内容包括性别、年龄、诊断、外周血、骨髓形态学、免疫学、细胞遗传学、基因突变、骨髓活检、肝脾肿大、体质性症状及既往治疗情况。结果 20例患者中男16例、女4例，中位年龄57(45，68)岁，治疗前中位血红蛋白水平60(55，80)g/L，口服泼尼松0.7~1.0 mg/(kg·d)[中位剂量0.8 mg/(kg·d)]至少3个月，75.0%(15/20)患者经过中位起效时间3(2，8)周的治疗获得临床反应，中位疗效持续时间为19(6，45)个月。非输血依赖10例患者中，8例患者血红蛋白上升大于20 g/L; 10例输血依赖患者中，2例患者治疗有效但因不良反应无法耐受早期停药，其余8例输血依赖患者中7例摆脱输血依赖。根据IWG-MRT关于贫血的疗效评价标准，2例患者获得完全缓解，6例患者获得部分缓解，7例患者获得临床改善。20例患者均未见明显的血液学不良反应; 非血液学不良反应主要为继发感染和神经毒性。结论 增加泼尼松剂量[中位剂量0.8 mg/(kg·d)]持续服用至少3个月，能够提高骨髓纤维化患者贫血治疗反应率，改善患者的生活质量。Abstract: Objective To evaluate the efficacy of corticosteroid monotherapy through increment of prednisone dosage on myelofibrosis-associated anemia.Methods The outcome and clinical data of 20 myelofibrosis patients treated with medium-dose prednisone were retrospectively analyzed, including gender, age, diagnosis, whole blood count, bone marrow examination, immunophenotyping, cytogenetics changes, somatic mutations, bone marrow biopsy, hepatomegaly and/or splenomegaly, constitutional symptoms and prior treatment.Results Among the 20 patients, there were 16 males and 4 females, with a median age of 57(45, 68) years. Before treatment, the median hemoglobin level was 60(55, 80) g/L. Oral prednisone dose of 0.7-1.0 mg/(kg·d), median dose of 0.8 mg/(kg·d) was taken for at least 3 months. Fifteen patients(75.0%) obtained clinical response after treatment with a median onset time of 3(2, 8) weeks. The median duration of efficacy was 19(6, 45) months. Among the 10 transfusion-independent patients, the hemoglobin increased by more than 20 g/L in 8 patients. Among the 10 transfusion-dependent patients, 2 cases were effective but could not tolerate early withdrawal due to adverse events; among the other 8 transfusion-dependent patients, 7 cases got rid of blood transfusion dependence. According to the revised International Working Group for Myelofibrosis Research and Treatment(IWG-MRT) criteria, 2 cases achieved complete remission, 6 cases achieved partial remission and 7 cases achieved clinical improvement. There were no obvious hematological adverse events in 20 patients. Non hematological adverse events were mainly secondary infection and neurotoxicity.Conclusion Increment of prednisone dosage, with a median dose of 0.8 mg/(kg·d) continuously at last 3 months results in higher response rate of myelofibrosis-associated anemia, together with improved quality of life.
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Key words:
- myelofibrosis /
- increment of prednisone dosage /
- anemia
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表 1 增加泼尼松剂量后治疗有反应的15例患者治疗前后临床数据比较
患者编号 年龄/岁 性别 骨髓纤维化类型 治疗前后血红蛋白(最好的血红蛋白反应)/(g·L-1) 起效时间/周 持续时间/月 1 37 男 血小板增多症后继发 90 vs 115 2 45 2 50 男 原发 输血依赖vs 90 2 5 3 27 男 原发 74 vs 135 4 35+ 4 42 男 血小板增多症后继发 输血依赖vs 83 3 6 5 62 男 原发 输血依赖vs 91 9 27 6 47 男 原发 69 vs 91 8 55+ 7 78 男 原发 66 vs 89 3 4 8 65 男 血小板增多症后继发 输血依赖vs 86 3 6 9 60 男 原发 输血依赖vs 91 8 4 10 57 男 原发 70 vs 115 4 19+ 11 49 男 原发 78 vs 118 4 24+ 12 45 女 原发 65 vs 115 2 80 13 61 男 原发 71 vs 105 2 59+ 14 50 女 原发 输血依赖vs 90 3 10+ 15 66 男 血小板增多症后继发 输血依赖vs 72 2 8+ 
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