儿童朗格罕斯细胞组织细胞增生症34例临床分析

Clinical features and prognosis of Langerhans cell histiocytosis in children: an analysis of 34 cases

摘要:

目的 探讨儿童朗格罕斯细胞组织细胞增生症（LCH）的临床特点和预后，以期提高LCH诊疗水平。方法 对34例初发LCH儿童患者进行回顾性分析。结果 34例患者中位年龄14.5个月（22 d至60个月），其中0~2岁的23例、> 2岁的11例；高危组17例，低危组17例。30例患者接受化疗，6周化疗总有效率67%（20/30），12个月总有效率87%（26/30），3年总生存（OS）率为86%±6%，3年无事件生存（EFS）率为64%±9%。高危组患者6周化疗有效率46.7%，3年OS为72%±12%，3年EFS为46%±13%，均低于低危组（86.7%、100%、82%±9%），差异均有统计学意义（P < 0.05）。高危组12个月化疗有效率（80%）与低危组（93%）的差异无统计学意义（P > 0.05）；复发率和死亡率均为27%，而低危组无复发和死亡。结论 LCH总体生存率较高，但高危组6周化疗有效率低，远期预后较差。

Abstract:

Objective To investigate the clinical features and prognosis of children with Langerhans cell histiocytosis (LCH). Methods A retrospective analysis was performed for the clinical data of 34 children with newly diagnosed LCH. Results The 34 children had a median age of 14.5 months (range:22 d to 60 months). Of all 34 children, 23 were aged 0-2 years and 11 were aged > 2 years. There were 17 children in the high-risk group and 17 in the low-risk group. Thirty children received chemotherapy, and the 6-week and 12-month overall response rates were 67% (20/30) and 87% (26/30), respectively. The 3-year overall survival (OS) rate was 86%±6% and the 3-year event-free survival (EFS) rate was 64%±9%. Compared with the low-risk group, the high-risk group had significantly lower 6-week chemotherapy response rate (47% vs 87%; P < 0.05), 3-year OS rate (72%±12% vs 100%; P < 0.05), and 3-year EFS rate (46%±13% vs 82%±9%; P < 0.05). There was no significant difference in the 12-month chemotherapy response rate between the high-risk and low-risk groups (80% vs 93; P > 0.05). The high-risk group had a recurrence rate of 27% and a mortality rate of 27%. There were no recurrence or deaths in the low-risk group. Conclusions Children with LCH have a high overall survival rate, but the high-risk patient has a low 6-week response rate of induction chemotherapy and poor long-term prognosis.