Kastritis E, Gavriatopoulou M, Roussou M, Fotiou D, Ziogas D C, Migkou M, Eleutherakis-Papaiakovou E, Panagiotidis I, Kanellias N, Psimenou E, Papadopoulou E, Pamboucas C, Manios E, Gakiopoulou H, Ntalianis A, Tasidou A, Giannouli S, Terpos E, Dimopoulos M A
Department of Clinical Therapeutics, National and Kapodistrian University of Athens, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece.
Blood Cancer J. 2017 Jun 16;7(6):e570. doi: 10.1038/bcj.2017.47.
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.
硼替佐米联合地塞米松（VD）或加用环磷酰胺（VCD），对轻链型（AL）淀粉样变性患者疗效显著。目前，VCD被视为AL患者的一线治疗方案，但鉴于硼替佐米自身的显著活性，VD加用环磷酰胺是否能进一步显著提高疗效尚不清楚。我们回顾性比较了101例连续两个阶段接受VD（n=59）或VCD（n=42）治疗的AL淀粉样变性患者的治疗结果。调整梅奥分期后的早期死亡率相似。意向性分析显示，接受VD治疗的患者血液学缓解率为68%，接受VCD治疗的患者为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%。更高剂量的地塞米松或每周两次的硼替佐米与显著更高的CR+VGPR率无关。两组器官反应发生率相似。即使调整了梅奥分期、硼替佐米和地塞米松的剂量及给药方案，中位生存期也相似（33个月对36个月，P=0.45）。总之，硼替佐米即使联合低剂量地塞米松对AL淀粉样变性也有效；更高剂量的地塞米松和加用环磷酰胺似乎对疗效和生存期没有显著影响。
