Abstract：
Erdheim-Chester disease (ECD), also known as non-Langerhans cell histiocytosis, is a multi-systemic disease with unclear pathogenesis. Based on a small number of case studies, pegylated interferon-α (PEG-IFN-α) has been used as the front-line treatment option. However, there are limited data regarding administration of ropegylated-interferon α-2b (ROPEG-IFN-α 2b) for ECD patients. Herein, we report two cases of severe ECD treated with two types of PEG-IFN-α. One patient with heart and skeleton involvement and BRAF V600E mutation was treated with weekly PEG-IFN-α 2a. Another patient with bone involvement and no BRAF V600E mutation was administered monthly ROPEG-IFN-α 2b. The two types of PEG-IFN-α showed excellent disease control, excellent survival outcomes, and manageable toxicities in ECD patients. These results suggest that ROPEG-IFN-α 2b could be used equivalently to PEG-IFN-α 2a for management of advanced ECD.
摘要：
埃尔德海姆-切斯特病（ECD），也被称为非朗格汉斯细胞组织细胞增生症，是一种多系统的疾病，发病机制尚不清楚。基于少量的案例研究，聚乙二醇化干扰素-α（PEG-IFN-α）已被用作一线治疗选择。然而,关于对ECD患者给予Repegyl干扰素α-2b(ROPEG-IFN-α2b)的数据有限.在这里，我们报告了2例使用两种类型的PEG-IFN-α治疗的严重ECD。每周使用PEG-IFN-α2a治疗一名心脏和骨骼受累且BRAFV600E突变的患者。另一名骨受累且无BRAFV600E突变的患者每月给予ROPEG-IFN-α2b。两种类型的PEG-IFN-α显示出良好的疾病控制，优秀的生存结果，ECD患者的毒性和可控的毒性。这些结果表明，ROPEG-IFN-α2b可以与PEG-IFN-α2a等效地用于晚期ECD的管理。
