Effective systemic therapies suppress toxic light chain production leading to an increased proportion of patients with light chain (AL) amyloidosis who survive longer albeit with end-stage renal disease. There is a critical need to identify patients in this population who benefit from renal transplantation. This multicenter, observational study from five countries includes 237 patients with AL amyloidosis who underwent renal transplantation between 1987 and 2020. With a median follow-up of 8.5 years, the median overall survival from renal transplantation was 8.6 years and was significantly longer in patients with complete and very good partial hematologic responses (CR + VGPR) compared to less than VGPR (9 versus 6.8 years; HR: 1.5, P = 0.04 [95% CI: 1–2.1]) at renal transplantation. Median graft survival was 7.8 years and was better in the CR + VGPR group (8.3 vs 5.7 years, HR: 1.4, P = 0.05 [95% CI: 1–2]). The frequency and time to amyloid recurrence in the graft was also lower (16% vs 37%, p = 0.01) and longer (median time not achieved vs 10 years, p = 0.001) in the CR + VGPR group. Comparing CR vs. VGPR there was no difference in overall or graft survival. Although 69 patients (29%) experienced hematologic relapse, treatment effectively prevented graft loss in the majority (87%). Renal transplantation in selected AL amyloidosis patients is associated with extended overall and renal graft survival. Patients with hematologic CR or VGPR have the most favorable outcomes, and these patients should be considered for renal transplantation.
有效的系统性治疗能够抑制毒性轻链的产生，使得更多轻链（AL）淀粉样变性患者得以长期生存，尽管仍伴有终末期肾病。当前亟需明确此类患者中哪些可从肾移植中获益。这项多中心观察性研究涵盖了来自五个国家、在1987年至2020年间接受肾移植的237例AL淀粉样变性患者。中位随访8.5年结果显示，肾移植后中位总生存期为8.6年；其中在移植时达到完全及非常好的部分血液学缓解（CR + VGPR）的患者，其生存期显著长于未达到VGPR的患者（9年 vs 6.8年；HR: 1.5, P=0.04 [95% CI: 1–2.1]）。移植肾中位存活期为7.8年，且在CR + VGPR组更优（8.3年 vs 5.7年，HR: 1.4, P=0.05 [95% CI: 1–2]）。该组移植肾淀粉样变性复发的发生率更低（16% vs 37%，p=0.01）、复发时间更晚（中位时间未达到 vs 10年，p=0.001）。对比CR与VGPR患者，其总体生存率与移植肾存活率无显著差异。尽管有69例患者（29%）出现血液学复发，但多数（87%）通过治疗有效避免了移植肾丧失。选择性对AL淀粉样变性患者进行肾移植可延长总体生存期及移植肾存活时间。达到血液学完全缓解或非常好的部分缓解的患者预后最佳，应被纳入肾移植的考虑范围。
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