METHODS: We conducted a retrospective analysis of all adults with LCH (≥ 18 years) seen at Peking Union Medical College Hospital (Beijing, China) between January 2001 and December 2022.
RESULTS: Among the 445 newly diagnosed adults with LCH, 90 patients had liver involvement at diagnosis and 22 patients at relapse. The median age was 32 years (range, 18-66 years). Of 112 evaluable patients, 108 had full liver function testing, including alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase (ALP), γ-glutamyl transpeptidase (GGT), and total bilirubin and albumin. Elevated ALP was seen in 63.0% and GGT in 86.1%; 14.8% had elevated bilirubin. Next-generation sequencing of 54 patients revealed frequent BRAFN486_P490 (29.6%), BRAFV600E (18.5%), and MAP2K1 (14.8%).
RESULTS: After a median 40 months\' follow-up (range 1-168 months), 3-year progression-free survival (PFS) and overall survival were 49.7% and 86.6% respectively. In multivariable analyses, ≥3 abnormal liver function tests (HR 3.384, 95% CI 1.550-7.388, P = .002) associated with inferior PFS; immunomodulatory drug therapy (HR 0.073, 95% CI, 0.010-0.541, P = .010) correlated with superior PFS versus chemotherapy.
CONCLUSIONS: In summary, elevated GGT and ALP were common in adults with LCH liver involvement. Greater than equal to 3 abnormal liver function tests predicted poor outcomes. Immunomodulatory drug therapy was associated with favorable progression-free survival compared to chemotherapy.
方法:我们对北京协和医院(北京,中国)2001年1月至2022年12月。
结果:在445名新诊断为LCH的成年人中,90例患者在诊断时肝脏受累,22例患者复发。中位年龄为32岁(范围,18-66岁)。在112名可评估的患者中,108人进行了完整的肝功能测试,包括丙氨酸转氨酶,天冬氨酸转氨酶,碱性磷酸酶(ALP),γ-谷氨酰转肽酶(GGT),总胆红素和白蛋白.63.0%的ALP升高,86.1%的GGT升高;14.8%的胆红素升高。54例患者的下一代测序显示频繁的BRAFN486_P490(29.6%),BRAFV600E(18.5%),和MAP2K1(14.8%)。
结果:经过中位40个月的随访(范围1-168个月),3年无进展生存率(PFS)和总生存率分别为49.7%和86.6%。在多变量分析中,≥3次异常肝功能检查(HR3.384,95%CI1.550-7.388,P=.002)与PFS较差相关;免疫调节药物治疗(HR0.073,95%CI,0.010-0.541,P=.010)与PFS优于化疗相关。
结论:总之,GGT和ALP升高在LCH肝脏受累的成人中很常见.大于等于3个异常肝功能测试预测不良结果。与化疗相比,免疫调节药物治疗与良好的无进展生存期相关。