Daratumumab-based therapy in systemic AL amyloidosis including advanced cardiac involvement: a single center study

To evaluate the real-world effectiveness, overall survival (OS), durability of response, and tolerability of daratumumab-based therapy for systemic amyloid light-chain (AL) amyloidosis in Japan, where bortezomib and daratumumab were only recently introduced, using 3-year follow-up data.

We retrospectively reviewed 43 patients diagnosed with AL amyloidosis. Fifteen patients received daratumumab-containing regimens (Dara group) and 28 received non-daratumumab therapy (non-Dara group). Clinical characteristics, hematologic response (HR), organ response (OR), OS, and treatment tolerability were compared. Four patients with Mayo 2012 stage IV disease underwent stepwise dose modification of daratumumab-based therapy.

The Dara group showed significantly higher HR (93% vs. 35.7%) and OR (71.4% vs. 19.2%) and improved OS compared with the non-Dara group. At a median follow-up of 34 months, 11 of the 12 surviving patients in the Dara group maintained a complete HR. In Mayo stage IV cases, reduced-intensity induction with daratumumab and dexamethasone, followed by gradual intensification, achieved an OR in most patients evaluated.

Daratumumab-based therapy resulted in high response rates, durable remission, and improved OS in patients with systemic AL amyloidosis. Daratumumab-based strategies may benefit patients across risk groups, including those with advanced cardiac involvement.