Hepatic Response Dynamics in Newly Diagnosed Patients with Light-chain Amyloidosis: A Retrospective Cohort Study

Hematologic response is well established in systemic light chain (AL) amyloidosis treatment, but hepatic response patterns remain unclear. This study explored hepatic response dynamics in hepatic-involved patients with AL amyloidosis who achieved hematologic partial response within 24 months post-treatment.

Using two- and four-level hepatic response criteria (two-level: hepatic organ response [hepatic OR], hepatic no response [hepatic NR]; four-level: hepatic complete response [hepaCR], hepatic very good partial response [hepaVGPR], hepatic partial response [hepaPR], hepatic no response [hepaNR]), responses were assessed at 3, 6, 12, and 24 months.

Among 137 patients (median follow-up: 55.0 months), hepatic OR and ≥ hepaPR plateaued at 24 months, with hepatic OR achieved in 75.6% (65/86), hepaCR in 10.5% (9/86), hepaVGPR in 16.3% (14/86), and hepaPR in 38.4% (33/86). The two-level hepatic response better predicted overall survival than the four-level criteria. Patients achieving hepatic OR within 24 months had significantly better prognosis than non-responders (log-rank p = 0.008; HR, 2.782; 95% CI, 1.260-6.141), with no impact from response speed. Hematologic complete response (CR_H) at 3 months predicted higher likelihood of 24-month hepatic OR (OR 2.571, 95% CI, 1.387-4.767, p = 0.003).

The study highlights the importance of monitoring hepatic response dynamics, identifying 3-month CR_H and 24-month hepatic OR as key treatment milestones. A graphical abstract is also available with this article.