Dispenzieri Angela
Division of Hematology, Mayo Clinic, Rochester, Minnesota, USA.
Am J Hematol. 2023 Dec;98(12):1934-1950. doi: 10.1002/ajh.27081. Epub 2023 Sep 21.
POEMS syndrome is a life-threatening condition due to an underlying plasma cell neoplasm. The major criteria for the syndrome are polyradiculoneuropathy, clonal plasma cell disorder, 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.
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.
Because the pathogenesis of the syndrome is not well understood, risk stratification is limited to clinical phenotype rather than specific molecular markers. Risk factors include low serum albumin, age, pleural effusion, pulmonary hypertension, and reduced estimated glomerular filtration rate.
RISK-ADAPTED THERAPY: For those patients with a dominant plasmacytoma, first-line therapy is irradiation. Patients with diffuse sclerotic lesions or disseminated bone marrow involvement should receive systemic therapy. Corticosteroids are temporizing, but alkylators and lenalidomide are the mainstays of treatment, the former either in the form of low-dose conventional therapy or as high-dose conditioning for stem cell transplantation. Thalidomide and bortezomib also have activity, but their benefit needs to be weighed against their risk of exacerbating the peripheral neuropathy. Daratumumab combinations also appear promising based on case series. Prompt recognition and institution of both supportive care measures and therapy directed against the plasma cell result in the best outcomes.
POEMS综合征是一种由潜在浆细胞肿瘤引起的危及生命的疾病。该综合征的主要标准包括多神经根神经病、克隆性浆细胞疾病、硬化性骨病变、血管内皮生长因子升高以及存在卡斯特曼病。次要特征包括器官肿大、内分泌病、特征性皮肤改变、视乳头水肿、血管外容量超负荷和血小板增多症。
POEMS综合征的诊断需具备三项主要标准，其中两项必须包括多神经根神经病和克隆性浆细胞疾病，以及至少一项次要标准。
由于该综合征的发病机制尚不清楚，风险分层仅限于临床表型而非特定分子标志物。风险因素包括低血清白蛋白、年龄、胸腔积液、肺动脉高压和估计肾小球滤过率降低。
对于那些以浆细胞瘤为主的患者，一线治疗是放疗。有弥漫性硬化性病变或骨髓播散性受累的患者应接受全身治疗。皮质类固醇只是临时用药，而烷化剂和来那度胺是主要治疗药物，前者以低剂量传统疗法形式或作为干细胞移植的高剂量预处理。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相权衡。基于病例系列，达雷妥尤单抗联合用药似乎也很有前景。及时识别并采取支持性护理措施以及针对浆细胞的治疗可取得最佳疗效。
