文章：

加用环磷酰胺和更高剂量的地塞米松并不能改善接受硼替佐米治疗的AL型淀粉样变性患者的预后

Addition of cyclophosphamide and higher doses of dexamethasone do not improve outcomes of patients with AL amyloidosis treated with bortezomib

原文发布日期：2017-06-16

DOI: 10.1038/bcj.2017.47

类型: Original Article

开放获取: 是

英文摘要：

Bortezomib, in combination with dexamethasone (VD) or with the addition of cyclophosphamide (VCD), is highly effective in patients with amyloid light-chain (AL) amyloidosis. Currently, VCD is considered as a primary regimen for patients with AL, but it is not clear whether the addition of cyclophosphamide to VD further and significantly improves efficacy, given the substantial activity of bortezomib itself. We retrospectively compared the outcomes of 101 patients with AL amyloidosis who received VD (n=59) or VCD (n=42) in two consecutive periods. Early mortality after adjustment for Mayo stage was similar. On intent to treat, a hematologic response rate was 68% for patients treated with VD and 78% for VCD (P=0.26), while complete response+very good partial response (CR+VGPR) rate was 47.5% and 35%, respectively. Higher doses of dexamethasone or twice-weekly bortezomib were not associated with significantly higher CR+VGPR rates. Organ responses occurred in similar rates between the two groups. Median survival was similar (33 vs 36 months, P=0.45) even after adjustment for Mayo stage and dose and schedule of bortezomib and dexamethasone. In conclusion, bortezomib even with low doses of dexamethasone is effective for the treatment of AL amyloidosis; higher doses of dexamethasone and addition of cyclophosphamide do not seem to have a profound effect on efficacy and survival.

摘要翻译： 

硼替佐米联合地塞米松（VD方案）或在此基础上加用环磷酰胺（VCD方案）对轻链（AL）型淀粉样变性患者具有显著疗效。目前VCD方案被视为AL患者的一线治疗方案，但鉴于硼替佐米本身已具有强效作用，加用环磷酰胺是否能进一步显著提升疗效尚不明确。我们回顾性比较了连续两个时期接受VD方案（59例）与VCD方案（42例）治疗的101例AL淀粉样变性患者的预后结果。经梅奥分期校正后，两组早期死亡率相近。意向治疗分析显示，VD组血液学缓解率为68%，VCD组为78%（P=0.26），而完全缓解+非常好的部分缓解（CR+VGPR）率分别为47.5%和35%。增加地塞米松剂量或采用每周两次硼替佐米给药并未显著提高CR+VGPR率。两组器官缓解率相当。即使校正梅奥分期及硼替佐米、地塞米松的剂量与给药方案后，中位生存期仍无显著差异（33个月vs36个月，P=0.45）。结论表明，硼替佐米即使联合低剂量地塞米松对AL淀粉样变性治疗亦具疗效，而增加地塞米松剂量或联用环磷酰胺似乎对疗效和生存期无显著提升作用。

原文链接：

Addition of cyclophosphamide and higher doses of dexamethasone do not improve outcomes of patients with AL amyloidosis treated with bortezomib