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POEMS综合征:诊断延迟及采用来那度胺、环磷酰胺和泼尼松治疗成功,随后进行自体外周干细胞移植

POEMS syndrome: diagnostic delay and successful treatment with lenalidomide, cyclophosphamide and prednisone followed by autologous peripheral stem cell transplantation.

作者信息

Ordway Sarah, Gilbert Laura, Wanko Sam

机构信息

Internal Medicine, Walter Reed National Military Medical Center, Bethesda, Maryland, USA.

出版信息

BMJ Case Rep. 2018 Jul 26;2018:bcr-2017-223168. doi: 10.1136/bcr-2017-223168.

Abstract

Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes (POEMS) syndrome is a rare systemic disease, often unrecognised in the primary care setting. POEMS syndrome is associated with plasma cell dyscrasias and upregulation of vascular endothelial growth factor leading to systemic oedema, papilloedema and pulmonary hypertension. A wide constellation of presenting symptoms often leads to late diagnosis. Unrecognised and untreated disease rapidly leads to death from neuropathic exhaustion or cardiopulmonary failure. Treatment is extrapolated from other plasma cell dyscrasias such as multiple myeloma. Autologous peripheral blood stem cell transplantation (PBSCT) is often an important component of treatment. There is no established standard of care for POEMS syndrome. Therapies include lenalidomide, bortezomib and targeted monoclonal antibodies. We present a patient with POEMS syndrome who achieved rapid complete response to triple therapy consisting of lenalidomide, cyclophosphamide and prednisone, followed by high-dose chemotherapy and PBSCT.

摘要

多神经病、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全身性疾病,在初级医疗环境中常未被识别。POEMS综合征与浆细胞异常增殖及血管内皮生长因子上调有关,可导致全身性水肿、视乳头水肿和肺动脉高压。一系列广泛的症状表现常常导致诊断延迟。未被识别和未治疗的疾病会迅速导致因神经衰竭或心肺功能衰竭而死亡。治疗方法是从其他浆细胞异常增殖性疾病如多发性骨髓瘤推断而来。自体外周血干细胞移植(PBSCT)通常是治疗的重要组成部分。POEMS综合征尚无既定的护理标准。治疗方法包括来那度胺、硼替佐米和靶向单克隆抗体。我们报告了一名POEMS综合征患者,该患者对由来那度胺、环磷酰胺和泼尼松组成的三联疗法迅速产生完全反应,随后接受了大剂量化疗和PBSCT。

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