A Multicenter National Study of AL Amyloidosis in China: Insights Into Real‐World Diagnosis, Treatment, and Prognosis

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This national, multicenter, retrospective study analyzed 1070 patients with newly diagnosed systemic light-chain (AL) amyloidosis in China (2008-2025). Findings reveal increasing annual diagnoses and a marked shift in first-line therapy from proteasome inhibitor (PI)-based regimens to predominantly daratumumab-based regimens since 2024. Monthly kinetic assessments demonstrated that daratumumab-based induction produced deeper and faster hematologic responses than PI-based therapy [≥ very good hematologic partial response (HemVGPR): 80.3% vs. 70.8%, p = 0.012; median time to ≥ HemVGPR: 1.2 vs. 1.8 months, p = 0.001], with significantly superior cardiac overall response (63.1% vs. 53.3%, p = 0.030). Early mortality rates at 1, 3, and 6 months were 5.5%, 13.2%, and 16.2%, respectively, with daratumumab-based therapy identified as an independent protective factor for 6-month mortality. Median event-free survival (EFS) was 44.4 months, while median overall survival (OS) was not reached. Achievement of hematologic complete response (HemCR) conferred superior EFS and OS over HemVGPR (both p < 0.001), with minimal residual disease negativity further improving EFS in HemCR patients (p = 0.043). Concurrent hepatic and cardiac involvement defined a high-risk subgroup with poor outcomes (median EFS 12.8 months, OS 51.0 months). Multivariable analysis confirmed Eastern Cooperative Oncology Group (ECOG) performance status (PS) > 2, liver involvement, and gain1q as adverse prognostic factors for EFS, whereas ≥ HemVGPR was protective. For OS, liver involvement, concomitant multiple myeloma, and ECOG PS > 2 were independent risk factors, while ≥ HemVGPR and cardiac response predicted improved survival. This study establishes a contemporary benchmark for AL amyloidosis management in China, confirming the superior real-world efficacy of daratumumab-based frontline therapy.

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