Lau Loretta M S, Stuurman Kyra, Weitzman Sheila
Division of Haematology/Oncology, Hospital for Sick Children, University of Toronto, Ontario, Canada.
Pediatr Blood Cancer. 2008 Mar;50(3):607-12. doi: 10.1002/pbc.21322.
The skeleton is the most frequently involved organ in Langerhans cell histiocytosis (LCH) in children, and bone LCH generally has an excellent long-term survival. Although permanent consequences (PC) are described in half of the survivors thus raising concerns for quality of life (QOL), the impact on quality of life has not been formally studied.
A retrospective review was performed on 124 LCH patients with skeletal involvement, followed by prospective evaluation of PC and health-related QOL using age-appropriate validated study tools (PedsQL or SF-36).
Of the 116 survivors, PC were found in 34 (29%) patients, of whom 8 had more than 1 PC. PC were significantly more frequent in multi-system (M-S) than in single system (S-S) LCH (73% vs. 17%; P < 0.0001). While diabetes insipidus (65%) was the most common PC in M-S LCH, cosmetic and orthopedic problems made up the majority of PC in S-S LCH. The median follow-up time was 8.5 years. 27 patients completed the PedsQL survey and 22 the SF-36 survey. The QOL scores for LCH patients were not significantly different from healthy children in the PedsQL survey and from young adults in the SF-36 survey. In addition, the QOL scores were very similar between patients with and without PC and between patients with M-S and S-S LCH.
Children with M-S LCH had a significantly higher risk of developing PC. Skeletal LCH did not adversely affect the quality of life of survivors, including those with PC who appeared to adapt to their disabilities and medical problems.
骨骼是儿童朗格汉斯细胞组织细胞增多症（LCH）中最常受累的器官，骨LCH通常具有良好的长期生存率。尽管有一半的幸存者出现了永久性后果（PC），这引发了对生活质量（QOL）的担忧，但对生活质量的影响尚未进行正式研究。
对124例有骨骼受累的LCH患者进行回顾性分析，随后使用适合年龄的经过验证的研究工具（儿童生活质量量表或SF-36）对PC和健康相关生活质量进行前瞻性评估。
在116名幸存者中，34例（29%）患者出现PC，其中8例有不止1种PC。多系统（M-S）LCH中出现PC明显比单系统（S-S）LCH更频繁（73%对17%；P<0.0001）。虽然尿崩症（65%）是M-S LCH中最常见的PC，但美容和骨科问题在S-S LCH中占PC的大多数。中位随访时间为8.5年。27例患者完成了儿童生活质量量表调查，22例完成了SF-36调查。在儿童生活质量量表调查中，LCH患者的生活质量得分与健康儿童无显著差异，在SF-36调查中与年轻成年人也无显著差异。此外，有PC和无PC的患者之间以及M-S和S-S LCH患者之间的生活质量得分非常相似。
M-S LCH患儿发生PC的风险显著更高。骨骼LCH对幸存者的生活质量没有不利影响，包括那些有PC的患者，他们似乎能适应自身残疾和医疗问题。
