RESULTS: Sixty-seven patients (67/350-19.1%) had PPM LCH at presentation. Severe lung lesions were present in 24 of them. The median follow-up period was 61 months (IQR: 3.4-8.3). The 5-year overall survival (OS) and event free survival (EFS) was 89% and 56.6% respectively. The EFS, for severe radiological lesions triad was 38% ± 20.7 versus 66% ± 16.2 for non-severe lesions triad p 0.002, while for presence of chest X-ray changes 27% ± 22.344 versus absence of chest X ray changes 66% ± 14.7 p 0.001, for clinical respiratory manifestations 13% ± 13.9 versus none 62% ± 22.9 p < 0.001, for RO- with severe lung lesions 47% ± 30.4 versus RO- without severe lung lesions 69% ± 5.9 p 0.04. There was a tendency for the independent prognostic impact of severe lung involvement; aHR = 1.7 (95% CI 0.92-3.13, p = 0.09).
CONCLUSIONS: Although the lung is a low -risk organ per se in LCH, our study demonstrates a non negligeable prognostic impact of severe lung involvement in the risk stratification of pediatric LCH. This warrants further study and external validation.
结果:67例患者(67/350-19.1%)出现PPMLCH。其中24例存在严重的肺部病变。中位随访期为61个月(IQR:3.4-8.3)。5年总生存率(OS)和无事件生存率(EFS)分别为89%和56.6%。EFS,对于严重的放射学病变三联征,非严重病变三联征分别为38%±20.7和66%±16.2p0.002,而对于胸部X线变化的存在,为27%±22.344与不存在胸部X线变化的66%±14.7p0.001,对于临床呼吸道表现,为13%±13.9与无62%±22.9p<0.001,对于有严重肺部病变的RO-有47%±30.4严重肺部受累对预后有独立影响的趋势;aHR=1.7(95%CI0.92-3.13,p=0.09)。
结论:尽管肺本身是LCH的低风险器官,我们的研究表明,在小儿LCH的危险分层中,严重肺部受累对预后的影响不可忽视.这需要进一步研究和外部验证。