Proposal of new clinical diagnostic criteria for POEMS syndrome
Proposal of new clinical diagnostic criteria for POEMS syndrome
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DOI：
10.1136/jnnp-2018-318514
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发表时间：
2019-02-01
影响因子：
11
通讯作者：
Kuwabara, Satoshi
中科院分区：
文献类型：
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作者：
Suichi, Tomoki;Misawa, Sonoko;Kuwabara, Satoshi
目的采用合适的统计学方法和疾病对照，提出多发性神经病、脏器肿大、内分泌疾病、M蛋白和皮肤变化(POEMS)综合征的最佳诊断标准。方法采用回顾性队列研究方法，对104例疑似POEMS综合征患者进行为期至少12个月的金标准追踪研究，以严格排除其他类似POEMS综合征的疾病，并确认对POEMS综合征特异性治疗的反应。30例慢性炎症性脱髓鞘多神经根神经病患者(对照组)和30例多发性骨髓瘤或免疫球蛋白轻链淀粉样变性患者(单克隆性浆细胞增殖对照组)。应用Logistic回归分析确定POEMS综合征的临床和实验室异常的最佳组合。结果根据受试者操作特征曲线下的最佳表现，诊断标准被定义为存在三个主要标准(多发性神经病(典型的脱髓鞘)、单克隆性浆细胞增生性障碍和血管内皮生长因子升高)和至少四个次要标准中的两个(水肿/渗出、皮肤变化、器官肿大和硬化性骨损害)。其敏感性和特异度分别为100%和100%，其诊断准确率与以往较为复杂的标准相当。结论统计学上明确、简单易行的POEMS综合征诊断标准，可加速POEMS综合征的早期诊断和治疗，从而改善患者的预后。需要进行更大规模的前瞻性研究来证实其有效性。
Objective To propose the optimal diagnostic criteria for polyneuropathy, organomegaly, endocrinopathy, M-protein and skin changes (POEMS) syndrome using appropriate statistical methods and disease controls.Methods This retrospective cohort study included 104 consecutive patients with suspected POEMS syndrome, among whom a gold standard group of 60 patients with definitive POEMS syndrome diagnosis were followed for at least 12 months to strictly exclude other disorders mimicking POEMS syndrome and to confirm response to POEMS syndrome-specific treatment. Thirty patients with chronic inflammatory demyelinating polyradiculoneuropathy (demyelinating polyradiculoneuropathy controls) and 30 with multiple myeloma or immunoglobulin light chain amyloidosis (monoclonal plasma cell proliferation controls) were also included. Logistic regression analyses were performed to determine optimal combination of clinical and laboratory abnormalities, characteristic of POEMS syndrome.Results The diagnostic criteria were statistically defined as the presence of the three major criteria (polyneuropathy (typically demyelinating), monoclonal plasma cell proliferative disorder and elevated vascular endothelial growth factor) and at least two of the four minor criteria (oedema/effusion, skin changes, organomegaly and sclerotic bone lesions), based on best performance by area under the receiver operating characteristic curve analyses. The sensitivity and specificity were 100% and 100%, respectively; the diagnostic accuracy of the proposed criteria was equivalent to somewhat complicated previous criteria.Conclusions The statistically defined, simple diagnostic criteria for POEMS syndrome could accelerate early diagnosis and treatment, thereby contribute to better outcome in patients with this serious disease. Prospective larger studies are required to confirm the validity.