文章：

自体造血干细胞移植后接受二线治疗的轻链淀粉样变性患者的治疗及结局

Treatment and outcomes of patients with light chain amyloidosis who received a second line of therapy post autologous stem cell transplantation

原文发布日期：2022-04-11

DOI: 10.1038/s41408-022-00655-z

类型: Article

开放获取: 是

英文摘要：

We retrospectively reviewed 292 patients who received a second line of therapy post ASCT for their light chain amyloidosis. Most patients (40%) were treated with an alkylator + PI ± dex or PI ± dex followed by an alkylator + 2nd-gen IMiD ± dex or 2nd-gen IMiD ± dex (26%), an alkylator ± steroid or steroid monotherapy (19%), a 2nd-gen IMiD + PI ± dex (6%), an alkylator + thalidomide ± dex (5%), or daratumumab-based therapy (4%). The rate of CR or VGPR was 70% among the daratumumab-based group, 62% in the alkylator + PI ± dex or PI ± dex group, 55% in the alkylator + 2nd-gen IMiD ± dex or 2nd-gen IMiD ± dex group, 47% in the 2nd-gen IMiD + PI ± dex group, 24% in the alkylator ± steroid or steroid monotherapy group, and 18% in the alkylator + thalidomide ± dex group. The median OS was NR for the 2nd-gen IMiD + PI ± dex group and the daratumumab group, 130.4 months in the alkylator + 2nd-gen IMiD ± dex or 2nd-gen IMiD ± dex group, 100 months for the alkylator + PI ± dex or PI ± dex group, 36 months for the alkylator ± steroid or steroid monotherapy group, and 21 months for the alkylator + thalidomide ± dex group (P < 0.0001). The median OS was 100 months in patients who received melphalan 200 mg/m2 compared to 41 months in the 140 mg/m2 group (P < 0.0001). In conclusion, patients receiving novel therapy post ASCT and melphalan conditioning dosing at 200 mg/m2 at diagnosis had better outcomes.
 

摘要翻译： 

我们回顾性分析了292名在自体干细胞移植后接受二线治疗的轻链淀粉样变性患者。多数患者（40%）接受了烷化剂联合蛋白酶体抑制剂±地塞米松、或蛋白酶体抑制剂±地塞米松治疗；其次为烷化剂联合二代免疫调节剂±地塞米松、或二代免疫调节剂±地塞米松（26%）；烷化剂±类固醇或类固醇单药治疗（19%）；二代免疫调节剂联合蛋白酶体抑制剂±地塞米松（6%）；烷化剂联合沙利度胺±地塞米松（5%）；以及基于达雷妥尤单抗的治疗方案（4%）。完全缓解或非常好的部分缓解率在达雷妥尤单抗组为70%，烷化剂联合蛋白酶体抑制剂±地塞米松或蛋白酶体抑制剂±地塞米松组为62%，烷化剂联合二代免疫调节剂±地塞米松或二代免疫调节剂±地塞米松组为55%，二代免疫调节剂联合蛋白酶体抑制剂±地塞米松组为47%，烷化剂±类固醇或类固醇单药治疗组为24%，烷化剂联合沙利度胺±地塞米松组为18%。二代免疫调节剂联合蛋白酶体抑制剂±地塞米松组和达雷妥尤单抗组的中位总生存期未达到；烷化剂联合二代免疫调节剂±地塞米松或二代免疫调节剂±地塞米松组为130.4个月；烷化剂联合蛋白酶体抑制剂±地塞米松或蛋白酶体抑制剂±地塞米松组为100个月；烷化剂±类固醇或类固醇单药治疗组为36个月；烷化剂联合沙利度胺±地塞米松组为21个月（P < 0.0001）。接受200毫克/平方米马法兰治疗的患者中位总生存期为100个月，而140毫克/平方米组为41个月（P < 0.0001）。总之，在自体干细胞移植后接受新型治疗、且诊断时采用200毫克/平方米马法兰预处理剂量的患者具有更好的预后。

原文链接：

Treatment and outcomes of patients with light chain amyloidosis who received a second line of therapy post autologous stem cell transplantation