Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
Cancer Res Treat. 2023 Jul;55(3):1053-1057. doi: 10.4143/crt.2022.1535. Epub 2023 Jan 19.
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.
厄尔-当-切斯特病（ECD），亦称非朗格汉斯细胞组织细胞增生症，是一种发病机制尚不明确的多系统疾病。基于少量病例研究，聚乙二醇化干扰素-α（PEG-IFN-α）已被用作一线治疗选择。然而，针对 ECD 患者给予聚乙二醇化干扰素 α-2b（ROPEG-IFN-α 2b）的相关应用数据十分有限。在此，我们报告两例使用两种 PEG-IFN-α 治疗的严重 ECD 病例。1 例存在心脏和骨骼受累且存在 BRAF V600E 突变的患者接受每周 PEG-IFN-α 2a 治疗。另 1 例存在骨骼受累且无 BRAF V600E 突变的患者每月接受 ROPEG-IFN-α 2b 治疗。两种 PEG-IFN-α 均在 ECD 患者中表现出良好的疾病控制、良好的生存结局和可管理的毒性。这些结果提示 ROPEG-IFN-α 2b 可与 PEG-IFN-α 2a 等效用于晚期 ECD 的治疗。
