Treatment Patterns and Clinical Outcomes of Patients With Systemic Light Chain Amyloidosis in the United States:...

This study described real-world treatment patterns and outcomes in patients with systemic light chain (AL) amyloidosis in the United States (US).

De-identified chart review data were obtained from 117 adult patients diagnosed with systemic AL amyloidosis (01/01/2014-12/31/2021) who initiated 1 L treatment, outside of a clinical trial setting, on or after January 1, 2014, and received ≥ 1 treatment at a US medical center.

Among patients, (median age: 63.2 years; female: 50.4%; White: 73.5%; Black/African American: 14.5%), most patients received 2 or more lines of treatment (62.4%) with 30.8% receiving 2 lines of treatment, predominantly cyclophosphamide, bortezomib, and dexamethasone (CyBorD)-, daratumumab-, and dexamethasone-based regimens. In first-line, one third of patients had hematologic response (complete response [CR]: 31.6%; very good partial response (29.1%); for later lines, CR rates tended to decrease. By 57.5 months, ≥60% of patients were still alive (Kaplan-Meier rates - 12 months: 92.0%; 24 months: 84.4%). Median progression-free survival (41.4 months), event-free survival (24.4 months), and time to next treatment (43.2 months) were unexpectedly longest in second line.

The study demonstrated that there was heterogeneity in the treatment of AL amyloidosis, and patients who receive treatment can survive for several years after diagnosis without progression.