Objective: To analyze the efficacy and side effects of the combination regimen containing bortezomib, cyclophosphamide 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-banding and FISH. RQ-PCR-MAGE C1/CT7/ABL had high positive incidence in amyloidosis and can be observed 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 C1/CT7 might be helpful in the early detection of the clonal plasma cell.
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140 mg/L。PCR法检测黑色素瘤相关基因（MAGE C1/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10例，无Ⅲ~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。MAGE C1/CT7可早期显示克隆性浆细胞的存在。