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Venetoclax monotherapy induced rapid and sustained response in a frail patient with refractory AL amyloidosis: Less is more?
International Journal of Hematology ( IF 1.7 ) Pub Date : 2020-03-23 , DOI: 10.1007/s12185-020-02857-2
Pui-Lun Yip 1 , June S M Lau 1 , Ching-Pong Lam 1
Affiliation  

Immunoglobulin light chain amyloidosis (AL) is a plasma cell disorder characterized by accumulation of misfolded proteins, which can induce organ damage. Venetoclax is active in multiple myeloma patients, in particular those with t(11;14) translocation. t(11;14) translocation is the most common cytogenetic abnormality in AL patients; venetoclax may thus be a useful additional treatment option for this disease. However, a recent trial in multiple myeloma patients (BELLINI) reported increased mortality associated with venetoclax versus placebo in combination with bortezomib and dexamethasone. In this report, we describe an AL patient who had suffered from recurrent infection during previous treatment, but who responded to and tolerated well single-agent venetoclax for more than 1 year. The present report indicates that venetoclax monotherapy may be active and safe for refractory AL amyloidosis.



中文翻译:

Venetoclax单药治疗在难治性AL淀粉样变性的体弱患者中引起快速而持续的反应:少即是多?

免疫球蛋白轻链淀粉样变性病(AL)是一种浆细胞疾病,其特征在于错误折叠的蛋白质积累,会导致器官损伤。Venetoclax在多发性骨髓瘤患者中活跃,尤其是那些t(11; 14)易位的患者。t(11; 14)易位是AL患者最常见的细胞遗传学异常;因此,venetoclax可能是对该疾病有用的其他治疗选择。但是,最近在多发性骨髓瘤患者中进行的一项试验(BELLINI)报告说,与硼替佐米和地塞米松联合使用Venetoclax与安慰剂相比,死亡率增加。在本报告中,我们描述了一位AL患者,该患者在先前的治疗中曾反复感染,但对单药性静脉曲张反应良好并耐受1年以上。

更新日期:2020-03-23
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