Department of Hematology, Peking Union Medical Hospital, Dongcheng District, Chinese Academy of Medical Sciences and Peking Union Medical College, 1 Shuai Fu Yuan Hu Tong, Beijing, 100730, People's Republic of China.
State Key Laboratory of Complex Severe and Rare Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, People's Republic of China.
Orphanet J Rare Dis. 2022 Feb 7;17(1):39. doi: 10.1186/s13023-022-02193-0.
Non-Langerhans cell histiocytosis, including Erdheim-Chester disease (ECD), Rosai-Dorfman disease (RDD), indeterminate cell histiocytosis (ICH), and unclassified histiocytosis, is a rare disorder lacking a standard treatment strategy. We report our experience using intermediate-dose cytarabine as the first or subsequent therapy in non-Langerhans cell histiocytosis.
Nine ECD patients, 5 RDD patients, 1 ICH patient and 1 unclassified histiocytosis patient were enrolled. Intermediate-dose cytarabine therapy was administered as 0.5-1.0 g/m of intravenous cytarabine every 12 h for 3 days every 5 weeks. The median age at cytarabine initiation was 47.5 years (range 18-70 years). The median number of cycles of cytarabine administered was 5.5 (range 2-6). The overall response rate (ORR) was 87.5% in the overall cohort, including 12.5% with complete response and 75.0% with partial response. One patient experienced disease recurrence 19 months after cytarabine therapy. The median follow-up duration for the entire cohort was 15.5 months (range 6-68 months). The estimated 2-year progression-free survival and overall survival rates were 85.6% and 92.3%, respectively. The most common toxicity was haematological adverse events, including grade 4 neutropenia and grade 3-4 thrombocytopenia. No treatment-related deaths occurred.
Intermediate-dose cytarabine is an efficient treatment option for non-Langerhans cell histiocytosis patients, especially for those with CNS involvement.
非朗格汉斯细胞组织细胞增生症，包括 Erdheim-Chester 病（ECD）、Rosai-Dorfman 病（RDD）、未分类组织细胞增生症和不确定细胞组织细胞增生症，是一种罕见疾病，缺乏标准的治疗策略。我们报告了使用中剂量阿糖胞苷作为非朗格汉斯细胞组织细胞增生症的一线或后续治疗的经验。
纳入了 9 例 ECD 患者、5 例 RDD 患者、1 例 ICH 患者和 1 例未分类组织细胞增生症患者。中剂量阿糖胞苷治疗方案为每 12 小时静脉滴注 0.5-1.0 g/m2阿糖胞苷，每 5 周连用 3 天，共 5.5 个周期（范围 2-6 个）。阿糖胞苷起始时的中位年龄为 47.5 岁（18-70 岁）。总体反应率（ORR）在整个队列中为 87.5%，包括 12.5%的完全缓解和 75.0%的部分缓解。1 例患者在阿糖胞苷治疗后 19 个月复发。整个队列的中位随访时间为 15.5 个月（6-68 个月）。估计的 2 年无进展生存率和总生存率分别为 85.6%和 92.3%。最常见的毒性是血液学不良事件，包括 4 级中性粒细胞减少和 3-4 级血小板减少症。无治疗相关死亡。
中剂量阿糖胞苷是治疗非朗格汉斯细胞组织细胞增生症患者的有效治疗选择，特别是对于有中枢神经系统受累的患者。
