National Amyloidosis Centre, University College London (Royal Free Campus), London, UK.
Department of Haematology, University College London Hospitals, London, UK.
Br J Haematol. 2019 Dec;187(5):638-641. doi: 10.1111/bjh.16122. Epub 2019 Aug 6.
Bortezomib is standard treatment in AL amyloidosis (AL), but is contraindicated in patients with significant neuropathy. Carfilzomib, a second-generation proteosomal inhibitor, results in a lower incidence of neuropathy than bortezomib, but data in AL is scant. We report a cohort of five AL patients treated with upfront carfilzomib. All had cardiac, peripheral and autonomic neuropathy at presentation. All achieved at least a very good partial haematological response. There was no worsening in cardiac function, peripheral or autonomic neuropathy. Carfilzomib is an effective upfront treatment option in AL patients with peripheral and/or autonomic neuropathy (without severe cardiac or renal involvement).
硼替佐米是淀粉样变性（AL）的标准治疗方法，但在有明显神经病变的患者中禁用。卡非佐米是第二代蛋白酶体抑制剂，其导致神经病变的发生率低于硼替佐米，但在 AL 中的数据很少。我们报告了一组接受卡非佐米一线治疗的 5 例 AL 患者。所有患者在就诊时均有心脏、周围和自主神经病。所有患者均至少获得非常好的部分血液学缓解。心脏功能、周围或自主神经病均无恶化。卡非佐米是伴有周围和/或自主神经病（无严重心脏或肾脏受累）的 AL 患者的有效一线治疗选择。
