Venetoclax monotherapy induced rapid and sustained response in a frail patient with refractory AL amyloidosis: Less is more?

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.