Definition of Organ Involvement and Treatment Response in Primary Systemic Amyloidosis (AL): A Consensus Opinion from the 10th International Symposium on Amyloid and Amyloidosis.
Definition of Organ Involvement and Treatment Response in Primary Systemic Amyloidosis (AL): A Consensus Opinion from the 10th International Symposium on Amyloid and Amyloidosis.
复制标题
原发性系统性淀粉样变性 (AL) 器官受累和治疗反应的定义：第十届淀粉样蛋白和淀粉样变性国际研讨会的共识意见。
DOI：
10.1182/blood.v104.11.754.754
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发表时间：
2004
期刊：
影响因子：
20.3
通讯作者：
G. Grateau
中科院分区：
文献类型：
--
作者：
M. Gertz;R. Comenzo;R. Falk;J. Fermand;B. Hazenberg;P. Hawkins;G. Merlini;P. Moreau;P. Ronco;Sanchorawala;O. Sezer;A. Solomon;G. Grateau
简介 以前，识别器官受累和定义淀粉样蛋白 (AL) 反应的具体标准几乎没有什么用处。然而，针对浆细胞的新疗法，包括常规剂量化疗、清髓性化疗和针对破坏原纤维结构的药物，经常用于治疗患者。目前，不同机构的反应和器官参与标准不同，因此很难直接比较结果。结果与淀粉样蛋白涉及的器官数量直接相关，并且准确定义什么构成器官受累不仅仅是学术活动。该领域的 13 位领导者受邀提交了制定当前指南的机构标准。结果 专家组针对以下问题制定了共识指南：淀粉样变性的诊断需要什么？、区分系统性淀粉样变性和局限性淀粉样变性、淀粉样变性如何被定性为 AL 型？、器官受累的定义、器官反应标准（心脏、肾脏、肝脏、神经和软组织）和器官进展、血液学反应标准（包括利用新开发的免疫球蛋白比浊法游离轻链测定）以及血液学和血液学进展性疾病的定义。以器官为基础。结论 定义淀粉样变性 AL 的器官受累和反应标准（包括血液学和器官）一直具有挑战性。共识小组的 13 名成员制定了建议供全世界治疗该疾病患者的医生使用的标准，并允许对治疗相关结果采用统一的报告标准。未来，新成像技术以及血清肌钙蛋白和脑天然肽等血清生物标志物的整合将进一步定义器官受累和反应的定义。
Introduction Previously, specific criteria for recognizing organ involvement and defining response in amyloid (AL) had minimal utility. However, new therapies directed at the plasma cell, including conventional dose chemotherapy, myeloablative chemotherapy, and agents directed at disruption of fibril structure are regularly being used to treat patients. Currently criteria for response and organ involvement differ from institution to institution, making it difficult to directly compare outcomes. Outcomes are directly related to the number of organs involved with amyloid and accurate definition as to what constitutes organ involvement are more than an academic exercise. Thirteen leaders in the field were invited to submit institutional criteria from which the current guidelines were developed. Results The panel drew up consensus guidelines for the following questions: What is required for a diagnosis of amyloidosis?, differentiating systemic from localized amyloidosis, How is amyloidosis characterized as AL type?, Definitions of organ involvement, Criteria for organ response (heart, kidney, liver, nerve and soft tissue) and organ progression, hematologic response criteria (including utilization of the newly developed immunoglobulin nephelometric free light chain assay) and Definitions for progressive disease both hematologic and organ based. Conclusion Defining organ involvement and response criteria, both hematologic and organ-based for amyloidosis AL has always been challenging. The thirteen members of the consensus panel have defined criteria proposed to be used worldwide by physicians who treat patients with this disease and to permit uniform reporting criteria of treatment-related outcomes. In the future integration of new imaging techniques, as well as serum biomarkers such as serum troponin and brain naturetic peptide will further define the definitions of organ involvement and response.