National Amyloidosis Centre, University College London, London, UK.
Biostatistics Unit, UCL Eastman Dental Institute, London, UK.
Br J Haematol. 2019 Dec;187(5):642-652. doi: 10.1111/bjh.16143. Epub 2019 Aug 13.
Autologous stem cell transplantation (ASCT) is considered to be the best method to achieve deep haematological/organ responses and improve survival in selected patients with AL amyloidosis. This field has been led by US centres and is less utilised in Europe. The introduction of effective chemotherapy agents for AL prompted us to re-evaluate UK outcomes of ASCT in affected patients. A total of 264 AL amyloidosis patients treated with an ASCT between 1994 and 2018 were identified. Patient baseline characteristics, transplant-related mortality (TRM) and overall survival (OS) were analysed. The median OS post-ASCT was 87 months [95% confidence interval (CI): 77-106 months]. The median time from ASCT to next treatment was 48 months (95% CI: 29-55 months). A haematological response was achieved in 94·8% of patients and was a strong predictor of time to next treatment [P < 0·0001, hazard ratio (HR) = 1·75, 95% CI = 1·35-2·28] and OS (P = 0·007, HR = 1·91, 95% CI = 1·19-3·07). Organ response was: cardiac (n = 28, 60·9%), renal (n = 101, 76%) and liver (n = 7, 13·5%). Overall TRM was 8·7%, with a significant reduction over time (1994-2000: 18·8%; 2001-2006: 13·6%; 2007-2012: 6·2%; 2013-2018: 1·1%). In conclusion, ASCT is significantly safer and remains a highly effective treatment with excellent long-term survival; it should be more widely considered as a treatment option for systemic AL amyloidosis.
自体干细胞移植（ASCT）被认为是在选定的 AL 淀粉样变性患者中实现深度血液学/器官反应和提高生存率的最佳方法。这一领域一直由美国中心主导，在欧洲的应用较少。有效的 AL 化疗药物的出现促使我们重新评估英国受影响患者 ASCT 的结果。共确定了 264 例 1994 年至 2018 年间接受 ASCT 治疗的 AL 淀粉样变性患者。分析了患者的基线特征、移植相关死亡率（TRM）和总生存率（OS）。ASCT 后中位 OS 为 87 个月[95%置信区间（CI）：77-106 个月]。ASCT 后至下一次治疗的中位时间为 48 个月（95%CI：29-55 个月）。94.8%的患者达到血液学反应，这是下一次治疗时间的强烈预测因素[P<0.0001，风险比（HR）=1.75，95%CI=1.35-2.28]和 OS（P=0.007，HR=1.91，95%CI=1.19-3.07）。器官反应为：心脏（n=28，60.9%）、肾脏（n=101，76%）和肝脏（n=7，13.5%）。总 TRM 为 8.7%，随时间显著降低（1994-2000 年：18.8%；2001-2006 年：13.6%；2007-2012 年：6.2%；2013-2018 年：1.1%）。总之，ASCT 更安全，仍然是一种非常有效的治疗方法，具有出色的长期生存率；它应该更广泛地被视为系统性 AL 淀粉样变性的治疗选择。
