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硼替佐米、多柔比星和地塞米松诱导治疗联合自体干细胞移植治疗 AL 淀粉样变性:与一线干细胞移植的回顾性比较。

Vincristine, Doxorubicin, and Dexamethasone Induction before Autologous Stem Cell Transplantation in Patients with AL Amyloidosis: A Retrospective Comparison with Frontline Stem Cell Transplantation.

机构信息

Nephrology Center, Toranomon Hospital Kajigaya, Japan.

Okinaka Memorial Institute for Medical Research, Toranomon Hospital, Japan.

出版信息

Intern Med. 2022 Oct 1;61(19):2853-2860. doi: 10.2169/internalmedicine.9039-21. Epub 2022 Feb 26.

Abstract

Objective High-dose melphalan and autologous stem cell transplantation (ASCT) therapy for AL amyloidosis are now associated with reduced mortality based on the application of strict criteria. However, there is no long-term evidence concerning the performance of induction therapy with newer agents, such as bortezomib or daratumumab. Concerns regarding long-term relapse despite treatment with ASCT exist, and missing the opportunity to perform ASCT might occur if induction proves to not be efficacious and cardiac amyloidosis progression deprives the patients of a chance to receive ASCT. We herein report good amyloid control by vincristine, doxorubicin, and dexamethasone (VAD) induction therapy and argue the importance of induction therapy before ASCT. Methods We compared patients who underwent VAD induction and ASCT (VAD+ASCT) with patients who underwent frontline ASCT in our hospital. Patients A total of 26 patients with histologically proven AL amyloidosis were included (18 in the VAD+ASCT group and 8 in the frontline ASCT). Results In the VAD+ASCT group, the 10-year overall survival and renal response rates were 82% and 43%, respectively. The renal response rate at two years in the VAD+ASCT group was significantly better than that in the frontline ASCT group. Although there was no significant difference in the survival rates between the two groups, the time to next treatment or death was significantly better in the VAD+ASCT group than in the the frontline ASCT group. Acute kidney injury was the most frequent reason for failure to receive two courses of VAD, and early mortality was mainly due to gastrointestinal complications. Conclusion Considering that only those who underwent 2 courses of VAD experienced a 10-year renal response, induction therapy was deemed to be directly related to the long-term control of AL amyloidosis.

摘要

目的 基于严格标准的应用,高剂量马法兰和自体干细胞移植(ASCT)疗法现在与降低死亡率相关。然而,对于新型药物如硼替佐米或达雷妥尤单抗的诱导治疗的长期疗效尚无证据。尽管进行了 ASCT 治疗,但仍存在长期复发的担忧,如果诱导治疗无效且心脏淀粉样变性进展使患者失去接受 ASCT 的机会,可能会错过进行 ASCT 的机会。我们在此报告长春新碱、多柔比星和地塞米松(VAD)诱导治疗对淀粉样蛋白的良好控制,并强调在 ASCT 之前进行诱导治疗的重要性。

方法 我们比较了在我院接受 VAD 诱导和 ASCT(VAD+ASCT)的患者与接受一线 ASCT 的患者。

结果 在 VAD+ASCT 组中,10 年总生存率和肾脏反应率分别为 82%和 43%。VAD+ASCT 组的两年肾脏反应率明显优于一线 ASCT 组。尽管两组的生存率无显著差异,但 VAD+ASCT 组的无进展生存期或死亡率明显优于一线 ASCT 组。未能接受两疗程 VAD 的最常见原因是急性肾损伤,早期死亡主要是由于胃肠道并发症。

结论 考虑到只有接受了两疗程 VAD 的患者才有 10 年的肾脏反应,因此诱导治疗被认为与 AL 淀粉样变性的长期控制直接相关。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1208/9593158/ef61df13083b/1349-7235-61-2853-g001.jpg

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