多发性神经病,器官肿大,内分泌病,单克隆丙种球蛋白病,和皮肤变化(POEMS)综合征是一种罕见的副肿瘤疾病,皮肤症状的患病率很高。很少有研究关注皮肤对这种疾病的全身治疗的反应。
评估低剂量来那度胺加地塞米松治疗后的皮肤反应,并确定其与血管内皮生长因子(VEGF)和血液学反应的关系。
共纳入41例至少18岁的新诊断POEMS综合征的中国患者。其中36人完成了12个周期的治疗。皮肤,血清VEGF,在治疗期间的1、2、3、6、9和12个月记录了血液和神经反应。
皮肤表现与基线时的血清VEGF水平无关。在色素沉着过度的患者中,血管瘤,多毛症,白色指甲,或者是单独的肢端发色,26/34(76.5%),11/30(36.7%),14/15(93.3%),21/21(100%)4/4(100%)在12个月时达到完全应答。12个月后色素沉着过度的减少反映了良好的VEGF反应(P=0.017)。
低剂量来那度胺加地塞米松治疗可有效逆转POEMS综合征患者的皮肤变化。
Clinicaltrials.gov,标识符NCT01816620。
Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome is a rare paraneoplastic disease with a high prevalence of skin symptoms. Few studies have focused on skin responses to systemic treatment of this disease.
To evaluate skin responses after treatment with low-dose lenalidomide plus dexamethasone and determine their relationship with vascular endothelial growth factor (VEGF) and hematological responses.
A total of 41 consecutive Chinese patients who were at least 18 years of age with newly diagnosed POEMS syndrome were enrolled. 36 of them completed 12 cycles of treatment. Skin, serum VEGF, hematological and neurological responses were documented at 1, 2, 3, 6, 9, and 12 months during treatment.
Skin manifestations were not associated with serum VEGF levels at baseline. Of the patients with hyperpigmentation, hemangiomas, hypertrichosis, white nails, or acrocyanosis separately, 26/34 (76.5%), 11/30 (36.7%), 14/15 (93.3%), 21/21 (100%), and 4/4 (100%) achieved complete responses at 12 months. Reduction in hyperpigmentation after 12 months reflected a good VEGF response (P = 0.017).
Low-dose lenalidomide plus dexamethasone therapy is effective in reversing skin changes in patients with POEMS syndrome.
Clinicaltrials.gov, identifier NCT01816620.