POEMS综合征10例临床特征及诊治分析

Analysis of clinical features, diagnosis and treatment of 10 patients with POEMS syndrome

目的 分析POEMS综合征患者的临床特征及诊治情况。方法 回顾性分析2012年3月至2021年4月在大连医科大学附属第一医院诊治的10例POEMS综合征患者的临床资料,其中男性8例,女性2例,中位发病年龄57.5岁(39~77岁),发病至确诊中位时间17个月(4~48个月)。总结其临床特征、诊疗经过及转归。结果 10例患者中,慢性起病9例,周围神经病变10例,器官肿大8例,皮肤改变8例,内分泌改变8例,血小板增多2例。9例患者初诊未明确诊断,初诊至确诊中位时间6个月(1~35个月)。9例接受了糖皮质激素单药或联合来那度胺、马法兰或硼替佐米等抗浆细胞治疗。因呼吸衰竭或疾病进展死亡3例,其余7例患者中,临床疗效评价7例达RC;神经病变疗效评价6例达R_(N),1例达SD_(N);血液学疗效评价1例达CR_(H),6例达SD_(H);血VEGF疗效评价2例达PRV, 5例达SDV。10例患者中位随访时间48个月(7~123个月),5年OS率70%。结论 POEMS综合征起病隐匿,临床表现复杂多样且呈高度异质性,对于不明原因的慢性进展性周围神经病伴皮肤色素沉着应警惕该病,及时进行M蛋白的筛查有助于POEMS综合征的早期诊断。

Objective To analyze the clinical features, diagnosis and treatment of patients with POEMS syndrome.Methods We analyzed the clinical data of 10 patients with POEMS syndrome in the First Affiliated Hospital of Dalian Medical University from March 2012 to April 2021. There were 8 males and 2 females, the median age of onset was 57.5 years old (39-77 years old) and the median time from onset to definite diagnosis was 17 months (4-48 months). We summarized the clinical features, diagnosis, treatment and outcomes.Results Among the 10 patients, 9 patients had chronic onset, 10 patients had peripheral neuropathy, 8 patients had organ enlargement, 8 patients had skin changes, 8 patients had endocrine changes and 2 patients had thrombocytosis. Nine patients could not have a definite diagnosis initially, the median time from the primary to the definite diagnosis was 6 months (1-35 months). Nine patients received antiplasma cell therapy with glucocorticoid alone or in combination with lenalidomide, melphalan or bortezomib. Three patients died of respiratory failure or disease progression. Among the other 7 patients, 7 got RC in clinical response, 6 got R_(N) and 1 got SD_(N) in neuropathy response, 1 got CR_(H) and 6 got SDH in hematological response, 2 got PRV and 5 got SDV in VEGF response, median follow-up time was 48 months (7-123 months). The overall 5-year survival rate was 70%.Conclusion POEMS syndrome has insidious onset associated with clinical complexity and high heterogeneity. We should be alert to the unexplained chronic peripheral neuropathy with skin pigmentation, and screening of M protein timely is helpful for the early diagnosis of POEMS syndrome.