Eight-year Program of Clinical Medicine, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing, China.
Department of Ophthalmology and Key Laboratory of Ocular Fundus Diseases|, Peking Union Medical College Hospital , Chinese Academy of Medical Sciences, No.1 Shuaifuyuan Wangfujing Dongcheng District, Beijing, 100730, China.
Orphanet J Rare Dis. 2020 May 19;15(1):116. doi: 10.1186/s13023-020-01392-x.
Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes (POEMS) syndrome is a rare paraneoplastic syndrome involving multisystem. Optic disc edema (ODE) is the most common ocular manifestation in patients with POEMS syndrome and serves as an independent prognostic factor. However, parameters previously used to estimate its severity were inconvenient and costly. This study was designed to bring forward a novel and practical parameter, optic disc edema area, to evaluate ODE in patients with this disease and applied it to assess effectiveness of lenalidomide combined with dexamethasone in respect of ODE.
Forty-one treatment-naive patients with POEMS syndrome were enrolled in this single-center prospective study and treated with lenalidomide combined with dexamethasone. They received ocular examination to determine optic disc edema (ODE) area and other optic manifestations. Meanwhile, serum VEGF was measured before and after treatment. Among 41 enrolled patients, 38 received complete ocular examinations, and 25 of which had ODE at initial visit. Binocular mean ODE area of patients with ODE was significantly related to ODE grade (r = 0.620, p = 0.003) and peripapillary retinal thickness (r = 0.760, p < 0.001) before treatment. Serum VEGF was significantly higher in patients with ODE than their counterparts (p = 0.025) and positively correlated with binocular mean ODE area (r = 0.460, p = 0.036). After treatment, ODE area, along with serum VEGF, decreased markedly (p < 0.001).
ODE area was a reliable index to evaluate ODE severity and could precisely reflect ODE improvement through systemic treatment. Additionally, it was related to serum VEGF, a key factor in disease pathogenesis, suggesting its potential as an indicator of the overall severity of this disease.
Clinicaltrials, NCT01816620. Registered March 222,013.
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改变（POEMS）综合征是一种罕见的副肿瘤综合征，涉及多系统。视盘水肿（ODE）是 POEMS 综合征患者最常见的眼部表现，也是独立的预后因素。然而，以前用于评估其严重程度的参数既不方便也不经济。本研究旨在提出一种新的实用参数——视盘水肿面积，以评估该疾病患者的 ODE，并应用于评估来那度胺联合地塞米松治疗 ODE 的疗效。
这项单中心前瞻性研究纳入了 41 例初治的 POEMS 综合征患者，接受来那度胺联合地塞米松治疗。他们接受了眼部检查，以确定视盘水肿（ODE）的面积和其他眼部表现。同时，在治疗前后测量了血清 VEGF。在纳入的 41 例患者中，有 38 例完成了完整的眼部检查，其中 25 例初诊时存在 ODE。有 ODE 的患者双眼平均 ODE 面积与 ODE 分级（r=0.620，p=0.003）和治疗前视盘周围视网膜厚度（r=0.760，p<0.001）显著相关。有 ODE 的患者血清 VEGF 显著高于无 ODE 的患者（p=0.025），与双眼平均 ODE 面积呈正相关（r=0.460，p=0.036）。治疗后，ODE 面积和血清 VEGF 均显著降低（p<0.001）。
ODE 面积是评估 ODE 严重程度的可靠指标，通过全身治疗能精确反映 ODE 的改善情况。此外，它与血清 VEGF 相关，血清 VEGF 是疾病发病机制中的关键因素，这表明它可能成为反映该疾病整体严重程度的指标。
Clinicaltrials.gov，NCT01816620。注册于 2013 年 3 月 22 日。
