Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy.
Haematologica. 2013 Mar;98(3):433-6. doi: 10.3324/haematol.2012.073593. Epub 2012 Sep 14.
Immune-modulatory drugs are active in immunoglobulin light-chain amyloidosis and the addition of alkylating agents can potentiate their action. In this phase II prospective trial we used cyclophosphamide, lenalidomide and dexamethasone in the treatment of 21 patients who were refractory (n=13, 62%) or relapsed (n=8, 38%) after prior treatment including melphalan in all cases, bortezomib in 4 and thalidomide in 6. Median number of cycles administered was 4 (range 2-9 cycles). Severe adverse events were observed in 57% of patients, most common being neutropenia (29%). The hematologic response rate was 62%, with one complete response and 5 very good partial responses. Overall median survival was three years. The achievement of CR/VGPR was associated with a significant survival advantage. The combination of cyclophosphamide, lenalidomide and dexamethasone is an effective treatment for relapsed/refractory AL amyloidosis, and good quality hematologic response should be the aim of treatment in this setting. (clinicaltrials.gov identifier: NCT00607581).
免疫调节药物在免疫球蛋白轻链淀粉样变性中具有活性，并且烷化剂的添加可以增强其作用。在这项 II 期前瞻性试验中，我们在 21 例先前接受过治疗（所有病例均接受美法仑治疗，4 例接受硼替佐米治疗，6 例接受沙利度胺治疗）且难治（n=13，62%）或复发（n=8，38%）的患者中使用环磷酰胺、来那度胺和地塞米松进行治疗。中位治疗周期数为 4 个（范围 2-9 个周期）。57%的患者出现严重不良事件，最常见的是中性粒细胞减少症（29%）。血液学缓解率为 62%，包括 1 例完全缓解和 5 例非常好的部分缓解。总体中位生存时间为 3 年。获得 CR/VGPR 与生存优势显著相关。环磷酰胺、来那度胺和地塞米松的联合治疗是复发性/难治性 AL 淀粉样变性的有效治疗方法，在这种情况下，良好的血液学缓解质量应是治疗的目标。（临床试验标识符：NCT00607581）。
