Abstract

Objective: To study the outcome of patients with AL amyloidosis who were ineligible for high dose melphalan (HDM) and autologous stem cell transplantation (ASCT).

Methods: A real-life retrospective observational cohort study of Dutch patients with AL amyloidosis ineligible for HDM and ASCT was performed at the University Medical Center Groningen from January 2001 until April 2017. Primary outcome measure was overall survival (OS). Secondary outcome measures were hematological response (HR), organ responses, and treatment toxicity.

Results: Eighty-four patients were included. Ineligibility was due to NYHA class III/IV (n = 58), otherwise advanced disease (n = 11), advanced age (n = 14), or treatment refusal (n = 1). Early death (<3 months) rate was high (44%). Median OS improved from 4 months in period 2001–2009 (n = 36) to 8 months in period 2009–2017 (n = 48, p = .02). HR was seen in 29%, and 42% of the patients, respectively. Median OS was 36 months after induction treatment with bortezomib (n = 32) and 18 months with immunomodulatory imide drug (IMID) (n = 16), both higher than median OS (7 months) with other regimens (n = 27). Incidence of toxicity was high (51%).

Conclusion: OS improved in this high-risk group over the years, especially after introduction of new treatment modalities. However, early death rate remains high, illustrating the need for more effective treatment.

摘要

目的：研究不适合高剂量美法仑（HDM）和自体干细胞移植（ASCT）的AL淀粉样变性患者的结局。

方法：自2001年1月至2017年4月，在格罗宁根大学医学中心对不适合HDM和ASCT的荷兰AL淀粉样变性患者进行了一项现实生活的回顾性观察队列研究。主要终点指标是总体生存率（OS）。次要结果指标是血液学反应（HR），器官反应和治疗毒性。

结果：包括84例患者。不符合资格的 患者是由于NYHA III / IV级（n = 58），其他疾病（n  = 11），老年（n  = 14）或拒绝治疗（n  = 1）。早期死亡（<3个月）的比率很高（44％）。OS中位数从2001–2009年的4个月（n  = 36）改善到2009–2017年的8个月（n  = 48，p  = .02）。分别在29％和42％的患者中发现了HR。硼替佐米诱导治疗后中位OS为36个月（n  = 32），免疫调节酰亚胺药物（IMID）为18个月（n = 16），均高于其他方案的中位OS（7个月）（n  = 27）。毒性的发生率很高（51％）。

结论：多年来，该高危人群的OS改善，尤其是在采用新的治疗方式后。但是，早期死亡率仍然很高，这说明需要更有效的治疗。