摘要
目的评价芦可替尼在中国骨髓纤维化患者中的疗效和安全性。方法63例中国骨髓纤维化患者纳入研究，男32例，女31例，中位年龄55（25-79）岁。芦可替尼起始剂量：基线PLT（100- 200）×10^9/L者（25例）30 mg/d，基线PLT〉200×10^9/L者（38例）40 mg/d。使用MRI/CT、欧洲癌症研究与治疗组织生活质量调查问卷核心30（EORTC QLQ-C30）和骨髓纤维化症状评估表（MFSAF）v2.0对患者进行脾脏体积、生活质量和症状评估。结果截至12个月随访结束，47例（74.6%）患者仍在继续治疗，25例（39.7%）患者脾脏体积较基线缩小超过35%，首次达到脾脏体积缩小≥35％的中位时间为12.71（95%CI 12.14-35.00）周。治疗期间，85.7%（54/63）的患者有不同程度的脾脏缩小，中位最佳脾脏体积缩小百分比为35.5%，48周时中位脾脏缩小体积为34.7%。治疗48周时53.1 %（26/49）的患者MFSAF症状评分降低超过50%，生活质量得到改善。最常见的血液学不良事件包括贫血和血小板计数降低，但极少造成停药。非血液学不良事件以1/2级为主。结论芦可替尼使中国骨髓纤维化患者的脾脏体积获得持续缩小、症状改善，不良反应可耐受。临床试验注册诺华制药有限公司（NCT01392443）
ObjectiveTo evaluate the efficacy and safety of ruxolitinib in Chinese myelofibrosis patients.MethodsThis study enrolled 63 Chinese patients（32 males and 31 females）in total, whose median age was 55（25-79）years. The initial dose of ruxolitinib was 30mg/d（25 patients）with a baseline of PLT（100-200）×10^9/L and 40 mg/d（38 patients）with a baseline of PLT〉200×10^9/L. Spleen volume, quality of life（QOL）and symptoms were evaluated by MRI/CT, European Organization for Research and Treatment of Cancer QOL Questionnaire Core 30（EORTC QLQ-C30）and MF Symptom Assessment Form（MFSAF）v2.0 questionnaire. ResultsAt the time of this analysis（follow-up of 12 months）, 47 patients（74.6%）were still receiving treatment, 25 patients（39.7%）achieved ≥35% reduction in spleen volume from baseline. First time to achieve≥35% reduction was 12.71（95% CI 12.14-35.00）weeks. During the treatment, 85.7%（54 /63）of patients achieved reduction in spleen volume in different extent, the median optimal spleen volume reduction was 35.5% and the median spleen volume reduction was 34.7% at week 48. 53.1%（26/49）of patients achieved ≥50% reduction in spleen volume from baseline in total symptom score and QOL was improved at week 48. The most common hematologic adverse events were anemia and thrombocytopenia, which merely resulted in discontinuation of treatment. Non-hematologic adverse events were almost grade 1/2.ConclusionsThese data indicated that ruxolitinib treatment provided durable reductions in spleen volume and improvement in symptoms in Chinese myelofibrosis patients, and the adverse events were tolerated.Clinical trial registrationNovartis pharma（NCT01392443）
出处
《中华血液学杂志》
CAS
CSCD
北大核心
2016年第10期858-863,共6页
Chinese Journal of Hematology
基金
国家自然科学基金（81370643-H0812）
浙江省重点创新团队（2011R50015）
