Clinical characteristics and the long-term outcome of patients with atypical POEMS syndrome variant with undetectable monoclonal gammopathy
Clinical characteristics and the long-term outcome of patients with atypical POEMS syndrome variant with undetectable monoclonal gammopathy
复制标题
不可检测的单克隆丙种球蛋白病的非典型 POEMS 综合征变异患者的临床特征和长期结果
DOI：
10.1007/s00277-018-03589-4
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发表时间：
2019-03-01
影响因子：
3.5
通讯作者：
Li，Jian
中科院分区：
文献类型：
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作者：
He，Tianhua;Zhao，Ailin;Li，Jian
多发性神经病、器官肿大、内分泌病、M蛋白和皮肤变化（POEMS）综合征的诊断需要多发性神经病和单克隆浆细胞增殖作为两个强制性标准。我们的目的是总结POEMS变异的临床表现和治疗反应，没有单克隆丙种球蛋白病的证据。我们查询了2012年8月至2017年7月期间转诊至北京协和医院的所有患者的医疗文件，并回顾了13例患有未检出单克隆丙种球蛋白病的非典型POEMS综合征患者的临床和实验室特征，并与已发表的原型进行了比较。多发性神经病、器官肿大、皮肤变化和血管外液体超负荷的患病率分别为100%、100%、92%和100%。其他临床表现，如内分泌病，肺动脉高压，视神经乳头水肿，血小板增多症，红细胞增多症影响相似的百分比的患者中看到的原型。入组的POEMS变体的中位血清血管内皮生长因子（VEGF）水平为4998 pg/ml（范围为2155- 11，029 pg/ml）。长期随访发现，所有12例接受自体干细胞移植、美法仑治疗或来那度胺/沙利度胺治疗的患者均获得临床改善，其中8例VEGF水平下降50%或恢复正常。中位无进展生存期为101.5个月。我们的研究结果提出了一种POEMS综合征变体，具有特征性临床表现，VEGF水平升高，对靶向浆细胞治疗反应良好。
The diagnosis of polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes (POEMS) syndrome requires polyneuropathy and monoclonal plasma cell proliferation as two mandatory criteria. Our aim was to summarize clinical manifestations and treatment responses of POEMS variants with no evidence of monoclonal gammopathy. We queried all medical documentation of patients referred to Peking Union Medical College Hospital from August 2012 to July 2017, and reviewed the clinical and laboratory features of 13 patients with atypical POEMS syndrome with undetectable monoclonal gammopathy, and compared to prototypes published. The prevalence of polyneuropathy, organomegaly, skin changes, and extravascular fluid overload were 100%, 100%, 92%, and 100%, respectively. Other clinical manifestations, such as endocrinopathy, pulmonary hypertension, papilledema, thrombocytosis, and polycythemia affected similar percentages of patients as seen in prototypes. POEMS variants enrolled had a median serum vascular endothelial growth factor (VEGF) level of 4998 pg/ml (range 2155–11,029 pg/ml). Long-term follow-up found that all 12 patients received autologous stem cell transplant, melphalan-based therapy or lenalidomide/thalidomide-based therapy obtained clinical improvement, of which eight experienced decreased levels of VEGF by 50% or back to normal. The median progression-free survival was 101.5 months. Our findings raised a variant of POEMS syndrome variants with featured clinical manifestations, elevated VEGF levels, and good response to therapies targeting plasma cell.