POEMS syndrome: 2011 update on diagnosis, risk-stratification, and management
POEMS syndrome: 2011 update on diagnosis, risk-stratification, and management
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DOI：
10.1002/ajh.22050
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发表时间：
2011-07-01
影响因子：
12.8
通讯作者：
Dispenzieri, Angela
中科院分区：
文献类型：
--
作者：
Dispenzieri, Angela
疾病概述：POEMS综合征是一种由基础浆细胞肿瘤引起的副肿瘤综合征。该综合征的主要标准是多发性神经根神经病、克隆性浆细胞病（PCD）、骨质病变、血管内皮生长因子升高和Castleman病的存在。次要特征包括器官肿大、内分泌病、特征性皮肤变化、视神经乳头水肿、血管外容量超负荷和血小板增多。诊断常常被延误，因为该综合征是罕见的，并可能被误认为其他神经系统疾病，最常见的慢性炎症性脱髓鞘性多神经根神经病。POEMS综合征应区别于POEMS综合征的Castleman病变体，后者没有克隆PCD，通常很少或没有周围神经病变，但具有POEMS综合征的几个次要诊断标准。诊断：POEMS综合征的诊断有三个主要标准，其中两个必须包括多神经根神经病和克隆性浆细胞疾病，和至少一个次要标准。危险分层：由于综合征的发病机制尚未完全了解，危险分层仅限于临床表型，而不是特定的分子标志物。临床标准的数量不是预后，但浆细胞疾病的程度是。髂嵴骨髓活检未发现浆细胞克隆的患者可进行局部放疗;克隆较广泛或播散的患者可进行全身治疗。风险适应性治疗：对于显性硬皮病浆细胞瘤患者，一线治疗是放疗。弥漫性硬化病变或弥散性骨髓受累的患者以及完成放射治疗后3 - 6个月疾病进展的患者应接受全身治疗。皮质类固醇是暂时性的，但烷化剂是治疗的主要手段，无论是低剂量的常规治疗还是高剂量的干细胞移植。抗VEGF抗体的益处是矛盾的。来那度胺显示出可控制的毒性。沙利度胺和硼替佐米也有活性，但它们的益处需要与其加重周围神经病变的风险进行权衡。及时识别和机构的支持性护理措施和治疗直接针对浆细胞的结果最好的结果。Am. J. Hematol. 86：592-601，2011. (C)2011 Wiley-Liss，Inc.
Disease overview: POEMS syndrome is a paraneoplastic syndrome due to an underlying plasma cell neoplasm. The major criteria for the syndrome are polyradiculoneuropathy, clonal plasma cell disorder (PCD), sclerotic bone lesions, elevated vascular endothelial growth factor, and the presence of Castleman disease. Minor features include organomegaly, endocrinopathy, characteristic skin changes, papilledema, extravascular volume overload, and thrombocytosis. Diagnoses are often delayed because the syndrome is rare and can be mistaken for other neurologic disorders, most commonly chronic inflammatory demyelinating polyradiculoneuropathy. POEMS syndrome should be distinguished from the Castleman disease variant of POEMS syndrome, which has no clonal PCD and typically little to no peripheral neuropathy but has several of the minor diagnostic criteria for POEMS syndrome.Diagnosis: The diagnosis of POEMS syndrome is made with three of the major criteria, two of which must include polyradiculoneuropathy and clonal plasma cell disorder, and at least one of the minor criteria.Risk stratification: Because the pathogenesis of the syndrome is not well understood, risk stratification is limited to clinical phenotype rather than specific molecular markers. The number of clinical criteria is not prognostic, but the extent of the plasma cell disorder is. Those patients with an iliac crest bone marrow biopsy that does not reveal a plasma cell clone are candidates for local radiation therapy; those with a more extensive or disseminated clone will be candidates for systemic therapy.Risk-adapted therapy: For those patients with a dominant sclerotic plasmacytoma, first line therapy is irradiation. Patients with diffuse sclerotic lesions or disseminated bone marrow involvement and for those who have progression of their disease 3 to 6 months after completing radiation therapy should receive systemic therapy. Corticosteroids are temporizing, but alkylators are the mainstay of treatment, either in the form of low dose conventional therapy or high dose with stem cell transplantation. The benefit of anti-VEGF antibodies is conflicting. Lenalidomide shows promise with manageable toxicity. Thalidomide and bortezomib also have activity, but their benefit needs to be weighed against their risk of exacerbating the peripheral neuropathy. Prompt recognition and institution of both supportive care measures and therapy directed against the plasma cell result in the best outcomes. Am. J. Hematol. 86:592-601, 2011. (C) 2011 Wiley-Liss, Inc.