The efficacy of and indications for hematopoietic stem cell transplantation (HSCT) in pediatric Langerhans cell histiocytosis (LCH) remain undetermined. This retrospective study analyzed 30 children with refractory LCH who underwent HSCT in Japan between 1996 and 2014. Eleven patients received a myeloablative conditioning (MAC) regimen, while 19 patients received a reduced-intensity conditioning (RIC) regimen. Among the 26 patients with complete data, 23 patients had risk organ (RO) involvement during clinical course. Disease status at HSCT was no active disease (NAD) (4), active disease-regression (AD-r) (2), active disease-stable (AD-s) (4), and active disease-progressive (AD-p) (16). Seventeen of the 30 patients (57%) were alive with a median follow-up of 433 days (range 9–5307) after HSCT. Death occurred within 3 months after HSCT in eight of 13 patients. RIC and MAC patients were similar in both overall survival (OS) (56.8% vs. 63.6%, respectively, p = 0.789) and failure-free survival (56.8% vs. 54.6%, respectively, p = 0.938). Regarding disease status at HSCT, the six patients with NAD/AD-r experienced better outcomes than the 20 with AD-s/AD-p (5-year OS, 100% vs. 54.5%, respectively, p = 0.040). Disease state at the time of HSCT was the most important prognostic factor.

中文翻译：

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全国难治性朗格汉斯细胞组织细胞增生症儿童造血干细胞移植的回顾性研究

尚无确定造血干细胞移植（HSCT）在小儿Langerhans细胞组织细胞增生症（LCH）中的功效和适应症。这项回顾性研究分析了1996年至2014年间在日本接受HSCT的30例难治性LCH儿童。其中11例接受了清髓调理（MAC）方案，而19例接受了低强度调理（RIC）方案。在26名具有完整数据的患者中，有23名患者在临床过程中受累了风险器官（RO）。HSCT的疾病状态为无活动性疾病（NAD）（4），活动性疾病消退（AD-r）（2），活动性疾病稳定（AD-s）（4）和活动性疾病进展（AD-p） ）（16）。30例患者中有17例（57％）还活着，HSCT后平均随访433天（范围9-5307）。13例患者中有8例在HSCT后3个月内死亡。RIC和MAC患者的总生存期（OS）相似（分别为56.8％和63.6％，p  = 0.789）和无故障生存率（分别为56.8％和54.6％，p  = 0.938）。关于HSCT的疾病状况，六例NAD / AD-r患者的预后要好于20例AD-s / AD-p患者（5年OS，100％对54.5％，p  = 0.040）。HSCT时的疾病状态是最重要的预后因素。