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首页> 外文期刊>Clinical kidney journal. >Erdheim–Chester disease: from palliative care to targeted treatment
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Erdheim–Chester disease: from palliative care to targeted treatment

机译：埃德海姆–切斯特病：从姑息治疗到靶向治疗

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Erdheim–Chester disease (ECD) is a life-threatening multi-systemic non-Langerhans histiocytosis with cardiovascular complications as the leading cause of death. ECD affects the kidneys in up to 30% of cases, with fibrotic tissue deposition in the perirenal fat and renal hilum. Diagnosis is usually based on histological analysis of the pathologic tissue, which typically shows xanthogranulomatous infiltrates of foamy CD68+/CD1a- histiocytes surrounded by fibrosis. A consistent percentage of patients affected by ECD develop renal failure and hypertension as a consequence of renal artery stenosis and hydronephrosis. These conditions have been generally treated with the placement of stents and nephrostomies that frequently led to disappointing outcomes. Before the introduction of interferon-alpha (IFNα) treatment, the mortality rate was as high as 57% in the long term. Recent studies have granted new insights into the pathogenesis of ECD, which seems to bear a dual component of clonal and inflammatory disease. These advances led to use specific therapies targeting either the oncogenes (BRAFV600E) or the effectors of the immune response implicated in ECD (IL-1, TNFα). Drugs such as anakinra (recombinant human IL-1 receptor antagonist), infliximab (monoclonal antibody against TNFα) and vemurafenib (inhibitor of mutant BRAF) showed promising results in small single-centre series. Although larger trials will be needed to address the impact of these drugs on ECD prognosis and to select the most effective treatment, targeted therapies hold the premises to drastically change the outcome of this condition.
机译：Erdheim–Chester病（ECD）是威胁生命的多系统非Langerhans组织细胞增生症，其心血管并发症是主要的死亡原因。 ECD最多可影响30％的肾脏，其纤维化组织沉积在肾周脂肪和肾门中。诊断通常基于病理组织的组织学分析，其通常显示被纤维化包围的泡沫CD68 + / CD1a-组织细胞的黄原细胞瘤浸润。由于肾动脉狭窄和肾积水，始终有一定百分比的受ECD影响的患者发展为肾功能衰竭和高血压。这些情况通常通过放置支架和肾切除术治疗，这常常导致令人失望的结果。从长期来看，在引入干扰素-α（IFNα）治疗之前，死亡率高达57％。最近的研究为ECD的发病机理提供了新的见解，而ECD似乎具有克隆和炎症性疾病的双重成分。这些进展导致使用针对癌基因（BRAF V600E ）或涉及ECD的免疫应答效应因子（IL-1，TNFα）的特异性疗法。 anakinra（重组人IL-1受体拮抗剂），infliximab（抗TNFα单克隆抗体）和vemurafenib（突变型BRAF抑制剂）等药物在小型单中心系列试验中显示出了令人鼓舞的结果。尽管将需要进行较大规模的试验来解决这些药物对ECD预后的影响并选择最有效的治疗方法，但靶向治疗为彻底改变这种疾病的结果提供了前提。

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