Carfilzomib is an effective upfront treatment in AL amyloidosis patients with peripheral and autonomic neuropathy.
Richa ManwaniShameem MahmoodSajitha SachchithananthamHelen J LachmannJulian D GillmoreKwee L YongNeil RabinRakesh PopatCharalampia KyriakouSarah WorthingtonFaye A SharpleyMark SmithRaakhee ShahSimon CheesmanPhilip N HawkinsAshutosh D WechalekarPublished in: British journal of haematology (2019)
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).