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卡非佐米是伴有周围和自主神经病的淀粉样变性患者的有效一线治疗药物。

Carfilzomib is an effective upfront treatment in AL amyloidosis patients with peripheral and autonomic neuropathy.

机构信息

National Amyloidosis Centre, University College London (Royal Free Campus), London, UK.

Department of Haematology, University College London Hospitals, London, UK.

出版信息

Br J Haematol. 2019 Dec;187(5):638-641. doi: 10.1111/bjh.16122. Epub 2019 Aug 6.

Abstract

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).

摘要

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。

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