Clinical features and outcomes of adult Langerhans cell histiocytosis: a single-center experience
Clinical features and outcomes of adult Langerhans cell histiocytosis: a single-center experience
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DOI：
10.1007/s12185-020-02892-z
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发表时间：
2020-06-08
影响因子：
2.1
通讯作者：
Tojo, Arinobu
中科院分区：
文献类型：
--
作者：
Kobayashi, Masayuki;Ando, Shohei;Tojo, Arinobu
朗格汉斯细胞组织细胞增生症（LCH）是一种以CD 1a + CD 207+骨髓树突状细胞积聚为特征的克隆性扩张肿瘤。由于LCH是一种罕见的疾病，并且被认为主要影响儿童，成人LCH的临床特征和治疗结果的记录很少。我们回顾性分析了2005年至2018年期间转诊至东京大学医学科学研究所的53例LCH成人患者。诊断时的中位年龄为42岁，女性略占优势（57%）。发病和诊断之间的时间因患者而异（中位数，8个月;范围，0-144个月）。总的来说，40%的患者有单个器官受累，60%的患者有多个器官受累。总体而言，最常受影响的器官是骨骼（62%），其次是中枢神经系统（34%）和肺（28%）。26例患者需要全身治疗，25例患者接受了特殊C方案。20例（80%）接受Special C方案治疗的患者显示部分缓解或更好，但毒性较好。除了一个病人外，其他人都还活着。尽管中位随访时间为35.5个月，但中位无进展生存期尚未达到。免疫组织化学显示，39%的患者BRAF-V600 E阳性，这一比例低于北美和欧洲的既往报告。
Langerhans cell histiocytosis (LCH) is a clonally expanding neoplasm characterized by the accumulation of CD1a + CD207 + myeloid dendritic cells. As LCH is a rare disease and is presumed to mainly affect children, the clinical features and treatment outcomes of adult LCH have been poorly documented. We retrospectively reviewed 53 adult patients with LCH who were referred to the Institute of Medical Science, the University of Tokyo from 2005 to 2018. The median age at diagnosis was 42 years with a slight female predominance (57%). The time between onset and diagnosis varied among patients (median, 8 months; range, 0-144 months). In total, 40% of the patients had single organ involvement and 60% had multiple organ involvement. Overall, the most frequently affected organ was bone (62%), followed by the central nervous system (34%), and the lung (28%). Twenty-six patients required systemic treatment, and 25 patients underwent the Special C regimen. Twenty patients (80%) who underwent Special C regimen showed a partial response or better with favorable toxicity. All but one patient is still alive. Median progression-free survival has not been reached despite a median follow-up of 35.5 months. Immunohistochemistry revealed that 39% of patients were positive for BRAF-V600E, which was a lower proportion than in previous reports from North America and Europe.