Hepatic involvement in light chain amyloidosis: analysis of 130 patients and predictors of hepatic response and survival.

IF 7.4 2区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY

Amyloid-Journal of Protein Folding Disorders Pub Date : 2025-06-12 DOI:10.1080/13506129.2025.2516790

Matthew J Rees, Nadia Toumeh, Angela Dispenzieri, Morie Gertz, Binoy Yohannan, Suheil Albert Atallah-Yunes, Prashant Kapoor, Taxiarchis Kourelis, Nelson Leung, Suzanne Hayman, Francis Buadi, David Dingli, Joselle Cook, Rahma Warsame, Moritz Binder, Wilson Gonsalves, S Vincent Rajkumar, Shaji Kumar, Eli Muchtar

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引用次数: 0

Abstract

Organ response is key to improving outcomes in light chain (AL) amyloidosis. We investigated factors associated hepatic response (HepR) in a large cohort of patients with hepatic AL amyloidosis.

Methods: Retrospective study of newly-diagnosed AL amyloidosis patients (n = 130) with liver involvement evaluated at the Mayo Clinic between 2000-2021. Patients were eligible if they had documented liver involvement and baseline alkaline phosphatase (ALP)≥1.5x upper limit of normal (ULN). HepR was defined as >50% reduction in ALP from baseline or ALP normalization. HepRs were assessed at 6, 12, 24 month after treatment initiation and the best HepR at any time point.

Results: The median baseline ALP was 2.88-fold the ULN (ALP:ULN, IQR: 2.15-4.41), and the median bilirubin was 0.7 mg/dL. HepR rates increased with time from 28% at 6 months, 36% at 12 months and 48% at 24 months. The median time to HepR was 21.5 months (95%CI = 15.4-29.5). Baseline ALP ≥ 4xULN consistently predicted HepR across all time points. Hematological response (HemR) also independently predicted HepR at 12, 24 months and best response. At best hepatic response, kappa isotype, and front-line ASCT were further independent predictors of HepR.

Conclusions: The degree of baseline ALP elevation and HemR are reliable predictors of HepR.

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轻链淀粉样变累及肝脏：130例患者的分析及肝脏反应和生存的预测因素。

器官反应是改善轻链（AL）淀粉样变预后的关键。我们研究了与肝AL淀粉样变性患者肝反应相关的因素（HepR）。方法：回顾性研究2000-2021年间在梅奥诊所评估的新诊断的AL淀粉样变患者（n = 130）。如果患者有肝脏受累和基线碱性磷酸酶(ALP)≥1.5倍正常上限（ULN），则符合条件。HepR定义为ALP较基线或ALP正常化降低50%。在治疗开始后6、12、24个月评估HepR，并在任何时间点评估最佳HepR。结果：中位基线ALP为ULN的2.88倍（ALP:ULN, IQR: 2.15-4.41），中位胆红素为0.7 mg/dL。HepR率随时间增加，6个月时为28%，12个月时为36%，24个月时为48%。到HepR的中位时间为21.5个月（95%CI = 15.4-29.5）。基线ALP≥4xULN在所有时间点上一致预测HepR。血液学反应（HemR）也能独立预测12个月、24个月和最佳反应时的HepR。在最佳的肝脏反应中，kappa同型和一线ASCT进一步成为HepR的独立预测因子。结论：基线ALP升高程度和HemR是预测HepR的可靠指标。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Amyloid-Journal of Protein Folding Disorders 生物-生化与分子生物学

CiteScore

10.60

自引率

10.90%

发文量

审稿时长

6-12 weeks

期刊介绍： Amyloid: the Journal of Protein Folding Disorders is dedicated to the study of all aspects of the protein groups and associated disorders that are classified as the amyloidoses as well as other disorders associated with abnormal protein folding. The journals major focus points are: etiology, pathogenesis, histopathology, chemical structure, nature of fibrillogenesis; whilst also publishing papers on the basic and chemical genetic aspects of many of these disorders. Amyloid is recognised as one of the leading publications on amyloid protein classifications and the associated disorders, as well as clinical studies on all aspects of amyloid related neurodegenerative diseases and major clinical studies on inherited amyloidosis, especially those related to transthyretin. The Journal also publishes book reviews, meeting reports, editorials, thesis abstracts, review articles and symposia in the various areas listed above.