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儿童期结节病:一种罕见但引人入胜的疾病。

Childhood sarcoidosis: A rare but fascinating disorder.

机构信息

Division of Pediatric Rheumatology, Louisiana State University Medical Center and Children's Hospital of New Orleans, LA, USA.

出版信息

Pediatr Rheumatol Online J. 2008 Sep 23;6:16. doi: 10.1186/1546-0096-6-16.

Abstract

Childhood sarcoidosis is a rare multisystemic granulomatous disorder of unknown etiology. In the pediatric series reported from the southeastern United States, sarcoidosis had a higher incidence among African Americans. Most reported childhood cases have occurred in patients aged 13-15 years. Macrophages bearing an increased expression of major histocompatibility class (MHC) II molecules most likely initiate the inflammatory response of sarcoidosis by presenting an unidentified antigen to CD4+ Th (helper-inducer) lymphocytes. A persistent, poorly degradable antigen driven cell-mediated immune response leads to a cytokine cascade, to granuloma formation, and eventually to fibrosis. Frequently observed immunologic features include depression of cutaneous delayed-type hypersensitivity and a heightened helper T cell type 1 (Th1) immune response at sites of disease. Circulating immune complexes, along with signs of B cell hyperactivity, may also be found. The clinical presentation can vary greatly depending upon the organs involved and age of the patient. Two distinct forms of sarcoidosis exist in children. Older children usually present with a multisystem disease similar to the adult manifestations, with frequent hilar lymphadenopathy and pulmonary infiltrations. Early-onset sarcoidosis is a unique form of the disease characterized by the triad of rash, uveitis, and arthritis in children presenting before four years of age. The diagnosis of sarcoidosis is confirmed by demonstrating a typical noncaseating granuloma on a biopsy specimen. Other granulmatous diseases should be reasonably excluded. The current therapy of choice for sarcoidosis in children with multisystem involvement is oral corticosteroids. Methotrexate given orally in low doses has been effective, safe and steroid sparing in some patients. Alternative immunosuppressive agents, such as azathioprine, cyclophosphamide, chlorambucil, and cyclosporine, have been tried in adult cases of sarcoidosis with questionable efficacy. The high toxicity profile of these agents, including an increased risk of lymphoproliferative disorders and carcinomas, has limited their use to patients with severe disease refractory to other agents. Successful steroid sparing treatment with mycophenolate mofetil was described in an adolescent with renal-limited sarcoidosis complicated by renal failure. Novel treatment strategies for sarcoidosis have been developed including the use of TNF-alpha inhibitors, such as infliximab. The long-term course and prognosis is not well established in childhood sarcoidosis, but it appears to be poorer in early-onset disease.

摘要

儿童结节病是一种罕见的多系统肉芽肿性疾病,病因不明。在美国东南部的儿科系列报道中,结节病在非裔美国人中的发病率较高。大多数报道的儿童病例发生在 13-15 岁的患者中。巨噬细胞表达增加的主要组织相容性复合体 (MHC) II 分子,通过向 CD4+ Th(辅助诱导)淋巴细胞呈现未知抗原,最有可能引发结节病的炎症反应。持续存在、难以降解的抗原驱动的细胞免疫反应导致细胞因子级联反应、肉芽肿形成,最终导致纤维化。经常观察到的免疫特征包括皮肤迟发型超敏反应减弱和疾病部位辅助 T 细胞 1(Th1)免疫反应增强。循环免疫复合物以及 B 细胞活性增加的迹象也可能存在。临床表现差异很大,取决于受累器官和患者年龄。儿童中存在两种不同形式的结节病。年龄较大的儿童通常表现为多系统疾病,类似于成人表现,常伴有肺门淋巴结肿大和肺部浸润。早发性结节病是一种独特的疾病形式,其特征是在四岁之前出现皮疹、葡萄膜炎和关节炎三联征的儿童。通过在活检标本上显示典型的非坏死性肉芽肿来确诊结节病。应合理排除其他肉芽肿性疾病。对于多系统受累的儿童结节病,目前的治疗选择是口服皮质类固醇。低剂量口服甲氨蝶呤在一些患者中有效、安全且可减少皮质类固醇用量。其他免疫抑制剂,如硫唑嘌呤、环磷酰胺、苯丁酸氮芥和环孢素,已在结节病的成人病例中尝试使用,但疗效存在争议。这些药物的高毒性谱,包括淋巴增生性疾病和癌症的风险增加,限制了它们在对其他药物有抗性的严重疾病患者中的使用。在伴有肾功能衰竭的肾局限性结节病并发肾衰竭的青少年中,描述了霉酚酸酯成功的皮质类固醇节约治疗。已经开发了针对结节病的新的治疗策略,包括使用 TNF-α 抑制剂,如英夫利昔单抗。儿童结节病的长期病程和预后尚不清楚,但在早发性疾病中似乎更差。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/88a3/2559831/f2a7fc502556/1546-0096-6-16-1.jpg

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