Systemic light-chain (AL) amyloidosis is a rare and debilitating disease. Advances have been made in new treatments in recent years, yet real-world data on the management of the disease are scarce. EMN23 is a retrospective, observational study of patients who initiated first-line treatment in 2004–2018 in Europe, presenting the demographics, clinical characteristics, treatment patterns, and outcomes, from 4480 patients. Regimens based on bortezomib were the most frequently used as first-line therapy; only 6.2% of the patients received autologous stem cell transplant. Hematologic responses improved post-2010 (67.1% vs 55.6% pre-2010). The median overall survival (OS) was 48.8 (45.2–51.7) months; 51.4 (47.3–57.7) months pre-2010 and 46.7 (41.3–52.2) months post-2010. Early mortality was 13.4% and did not improve (11.4% vs 14.4% pre- and post-2010); furthermore, it remained high in patients with advanced cardiac disease (over 39% for stage IIIb). There was a significant improvement for stage IIIa (14.2 vs 30.7 months, p = 0.0170) but no improvement for stage IIIb patients (5.0 vs 4.5 months). This European real-world study of AL-amyloidosis emphasizes the unmet needs of early diagnosis, and the lack of improvement in survival outcomes of the frail stage IIIb population, despite the introduction of new therapies in recent years.
系统性轻链（AL）淀粉样变是一种罕见且严重消耗性的疾病。近年来，新疗法已取得进展，但关于该疾病管理的真实世界数据仍然匮乏。EMN23是一项回顾性观察研究，纳入了2004年至2018年间在欧洲开始一线治疗的4480名患者，呈现了其人口统计学特征、临床特点、治疗方案及结局。基于硼替佐米的方案是最常用的一线治疗；仅6.2%的患者接受了自体干细胞移植。2010年后血液学缓解率有所提高（67.1%对比2010年前的55.6%）。中位总生存期（OS）为48.8个月（45.2–51.7）；2010年前为51.4个月（47.3–57.7），2010年后为46.7个月（41.3–52.2）。早期死亡率为13.4%且未见改善（2010年前后分别为11.4%和14.4%）；此外，晚期心脏病患者的早期死亡率仍然很高（IIIb期患者超过39%）。IIIa期患者生存期有显著改善（14.2个月对比30.7个月，p=0.0170），但IIIb期患者未见改善（5.0个月对比4.5个月）。这项欧洲真实世界AL淀粉样变研究强调，尽管近年来引入了新疗法，但早期诊断的需求仍未得到满足，且脆弱IIIb期患者的生存结局缺乏改善。
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