POEMS syndrome: definitions and long-term outcome
POEMS syndrome: definitions and long-term outcome
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DOI：
10.1182/blood-2002-07-2299
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发表时间：
2003-04-01
期刊：
影响因子：
20.3
通讯作者：
Gertz, MA
中科院分区：
文献类型：
--
作者：
Dispenzieri, A;Kyle, RA;Gertz, MA
POEMS 综合征（指多发性神经病、器官肿大、内分泌病、M 蛋白和皮肤变化）仍然知之甚少。确定诊断、治疗效果和预后所需的特征存在模糊性。我们确定了 99 名 POEMS 综合征患者。最低标准是感觉运动周围神经病变和单克隆血浆增殖性疾病的证据。为了区分 POEMS 与意义未明的单克隆丙种球蛋白病相关的神经病，还包括其他标准：骨病变、Castleman 病、器官肿大（或淋巴结病）、内分泌病、水肿（外周水肿、腹水或积液）和皮肤变化。就诊时的中位年龄为 51 岁； 63%是男性。中位生存期为 165 个月。除了杵状指 (P = .03) 和血管外容量超负荷 (P = .04) 之外，没有任何表现特征（包括表现特征的数量）可以预测生存。对治疗的反应 (P < .001) 可预测生存。观察到肺动脉高压、肾功能衰竭、血栓事件和充血性心力衰竭，这些症状似乎是该综合征的一部分。 18 名患者 (118%) 随着时间的推移出现了新的疾病表现。超过 50% 的患者对放射有反应，22% 至 50% 的患者对泼尼松以及马法兰和泼尼松联合疗法有反应。我们得出的结论是，POEMS 综合征患者的中位生存期为 165 个月，与诊断时的综合征特征、骨病变或浆细胞数量无关。该综合征经常出现其他特征，但经典多发性骨髓瘤的并发症很少出现。
The POEMS syndrome (coined to refer to polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes) remains poorly understood. Ambiguity exists over the features necessary to establish the diagnosis, treatment efficacy, and prognosis. We identified 99 patients with POEMS syndrome. Minimal criteria were a sensorimotor peripheral neuropathy and evidence of a monoclonal plasma-proliferative disorder. To distinguish POEMS from neuropathy associated with monoclonal gammopathy of undetermined significance, additional criteria were included: a bone lesion, Castleman disease, organomegaly (or lymphadenopathy), endocrinopathy, edema (peripheral edema, ascites, or effusions), and skin changes. The median age at presentation was 51 years; 63% were men. Median survival was 165 months. With the exception of fingernail clubbing (P = .03) and extravascular volume overload (P = .04), no presenting feature, including the number of presenting features, was predictive of survival. Response to therapy (P < .001) was predictive of survival. Pulmonary hypertension, renal failure, thrombotic events, and congestive heart failure were observed and appear to be part of the syndrome. In 18 patients (118%), new disease manifestations developed over time. More than 50% of patients had a response to radiation, and 22% to 50% had responses to prednisone and a combination of melphalan and prednisone, respectively. We conclude that the median survival of patients with POEMS syndrome is 165 months, independent of the number of syndrome features, bone lesions, or plasma cells at diagnosis. Additional features of the syndrome often develop, but the complications of classic multiple myeloma rarely develop.