达拉图穆马
医学
淀粉样变性
淀粉样变性
重症监护医学
硼替佐米
临床试验
器官功能障碍
多发性骨髓瘤
内科学
免疫学
免疫球蛋白轻链
抗体
败血症
作者
Giovanni Palladini,Giampaolo Merlini
出处
期刊:Blood
[American Society of Hematology]
日期:2022-05-12
卷期号:139 (19): 2918-2930
被引量:28
标识
DOI:10.1182/blood.2020008737
摘要
The treatment of patients with systemic light chain (AL) amyloidosis is a challenge to hematologists. Despite its generally small size, the underlying clone causes a rapidly progressing, often devastating multiorgan dysfunction through the toxic light chains that form amyloid deposits. Clinical manifestations are deceitful and too often recognized at an irreversible stage. However, hematologists are in the unique position to diagnose AL amyloidosis at a presymptomatic stage, checking biomarkers of amyloid organ involvement in patients with monoclonal gammopathies at higher risk to develop the disease. Adequate technology and expertise are needed for a prompt and correct diagnosis, particularly for ruling out non-AL amyloidoses that are now also treatable. Therapy should be carefully tailored based on severity of organ involvement and clonal characteristics, and early and continual monitoring of response is critical. Three recent randomized clinical trials moved AL amyloidosis to evidence-based era. Above all, the daratumumab-bortezomib combination is a new standard-of-care for newly diagnosed patients, inducing rapid and deep responses that translate into high rates of organ response. The availability of new effective drugs allows to better personalize the therapy, reduce toxicity, and improve outcomes. Patients should be treated within clinical trials whenever possible.
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