ArticleAmerican journal of hematology2026
A Multicenter National Study of AL Amyloidosis in China: Insights Into Real-World Diagnosis, Treatment, and Prognosis.
Article in American journal of hematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
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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