硼替佐米联合地塞米松、环磷酰胺治疗原发系统性淀粉样变性疗效观察  

Curative effect observation of patients with primary systemic amyloidosis treated by the combination of bortezomib with dexmethasone and cyclophosphamide

作　　者：路瑾[1] 王辉[2] 黄晓军[1] 

机构地区：[1]北京大学人民医院、北京大学血液病研究所,100044 [2]北京大学人民医院检验科

出　　处：《中华血液学杂志》2013年第4期345-348,共4页Chinese Journal of Hematology

基　　金：北京市科技计划项目（Z111107067311070）;中国医师协会多发性骨髓瘤基金（20090101）

摘　　要：目的探讨硼替佐米联合地塞米松、环磷酰胺治疗原发系统性淀粉样变性的疗效以及不良反应。方法回顾性分析22例原发系统性淀粉样变性患者临床表现、使用硼替佐米联合地塞米松、环磷酰胺治疗的疗效以及不良反应。结果22例患者中初治9例，复发13例，中位血清游离轻链140mg／L。PCR法检测黑色素瘤相关基因（MAGEC1／CT7）阳性率88．9％，可先于骨髓细胞流式细胞术以及形态学检测方法检出阳性克隆性浆细胞的存在。中位治疗2个疗程。初治患者总有效率88．9％；脏器缓解率55．6％，2例猝死。复发患者总有效率46．2％，脏器缓解率30．7％。22例患者中出现周围神经病变lO例，无Ⅲ一Ⅳ级神经病变出现。1例出现肝功能衰竭。1例因置入气管支架反复发生感染。结论硼替佐米联合地塞米松、环磷酰胺治疗原发系统性淀粉样变性患者疗效显著，尤其对于初治患者更为明显。MAGEC1／CT7可早期显示克隆性浆细胞的存在。Objective To analyze the efficacy and side effects of the combination regimen containing bortezomib, eyclophosphamide and dexamethasone （ BCD ） in the treatment of primary systemic amyloidosis （PSA）. Methods The clinical manifestation, efficacy and side effects of 22 PSA patients after treatment were retrospectively analyzed. Results Of the 22 PSA patients, 9 were newly diagnosed cases and 13 relapsed cases, 20（90.9% ） had symptomatic cardiac involvement and 20 （90.9%） had 2 or more organs involved. The median free light chain were 140 mg/L. There were no specific abnormality was found by G-ban- ding and FISH. RQ-PCR-MAGE C1/CTT/ABL had high positive incidence in amyloidosis and can be ob- served ahead of the flow cytometry and morphology examination. Hematological response occurred in 88.9% in newly diagnosed patients, with 55.6% organ response, 2 patients occured sudden death during BCD interval. For relapsed patients, the hematological response was 46.2% , with 30.7% organ response. Ten of the 22 PSA patients developed peripheral neurophathy, 1 liver dysfunction, 1 severe infection after intra-tracheal stent. Conclusions BCD produces rapid and high hematological responses in the PSA patients especially in newly diagnosed patients. It is well tolerated with few side effects. MAGE CI/CT7 might be helpful in the early detection of the clonal plasma cell.

关 键 词：淀粉样变性 硼替佐米 环磷酰胺 地塞米松 

分 类 号：R74[医药卫生—神经病学与精神病学]