Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Disease in Children, Ministry of Education; Department of Hematology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Nanlishi Road No. 56, Xicheng District, Beijing, 100045, P.R. China.
Hematologic Disease Laboratory, Beijing Pediatric Research Institute; Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Disease in Children, Ministry of Education; Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, 100045, P. R. China.
BMC Pediatr. 2024 May 7;24(1):316. doi: 10.1186/s12887-024-04764-5.
Langerhans cell histiocytosis (LCH) is the most common histiocytic disorder in children, and liver involvement in LCH is rare. This retrospective study reported the clinical features and prognosis of patients with hepatic LCH. Liver involvement was defined by histopathological findings, liver dysfunction or abnormalities, or ultrasound imaging. A total of 130 patients (14.5%) with hepatic LCH out of 899 in the LCH population were enrolled. Patients with liver involvement had greater frequencies of skin, lung, hearing system, and haematologic system involvement, and hemophagocytic lymphohistiocytosis (P<0.001, 0.001, 0.002, 0.009, and <0.001, respectively). Overall survival and progression-free survival were lower in LCH patients with liver involvement than in those without liver involvement (P<0.001 and <0.001). In patients with liver involvement, the overall survival (OS) and progression-free survival (PFS) rates were lower in patients with cholangitis than in those without cholangitis (P<0.020 and 0.030). For the treatment response, the response rate of hepatic LCH patients to initial first-line therapy (n=89) was 22.5%. However, there was no significant difference in the response rate or recurrence rate between patients who shifted from first-line treatment to second-line treatment (n=29) or to targeted therapy (n=13) (P=0.453 and 1.000). The response rate of hepatic LCH patients who received initial second-line therapy (n=13) was 38.5%. Two of these patients subsequently experienced bone recurrence. The response rate of hepatic LCH patients who received initial targeted therapy (n=16) was 75.0%. Three patients subsequently experienced recurrence, including 2 in the bone and 1 in the liver and skin. A total of 39.3% of patients who received second-line treatment had severe myelosuppression (grade III-IV), and 50.8% had varying degrees of gastrointestinal events, whereas there was no severe toxicity in patients who received first-line treatment and targeted therapy. Four patients underwent liver transplantation because of liver cirrhosis. The patients' liver disease improved within a follow-up period of 18-79 months. This study demonstrated that LCH with liver involvement, especially cholangitis, indicates a poor prognosis. Targeted therapy provides a good treatment response and less toxicity. However, it may relapse after withdrawal. Liver transplantation is still a reliable salvage option for patients with end-stage liver disease.
朗格汉斯细胞组织细胞增生症（LCH）是儿童中最常见的组织细胞疾病，肝脏受累在 LCH 中较为少见。本回顾性研究报告了肝脏 LCH 患者的临床特征和预后。肝脏受累通过组织病理学发现、肝功能或异常、或超声成像来定义。在 899 例 LCH 患者中，共有 130 例（14.5%）存在肝脏 LCH。有肝脏受累的患者更频繁地出现皮肤、肺、听觉系统和血液系统受累，以及噬血细胞性淋巴组织细胞增生症（P<0.001、0.001、0.002、0.009 和 <0.001，分别）。有肝脏受累的 LCH 患者的总生存率和无进展生存率均低于无肝脏受累的患者（P<0.001 和 <0.001）。在有肝脏受累的患者中，有胆管炎的患者的总生存率（OS）和无进展生存率（PFS）均低于无胆管炎的患者（P<0.020 和 0.030）。对于治疗反应，接受初始一线治疗（n=89）的肝脏 LCH 患者的反应率为 22.5%。然而，从一线治疗转为二线治疗（n=29）或靶向治疗（n=13）的患者之间的反应率或复发率无显著差异（P=0.453 和 1.000）。接受初始二线治疗（n=13）的肝脏 LCH 患者的反应率为 38.5%。其中 2 例患者随后出现骨复发。接受初始靶向治疗（n=16）的肝脏 LCH 患者的反应率为 75.0%。3 例患者随后出现复发，其中 2 例在骨，1 例在肝和皮肤。接受二线治疗的患者中，39.3%出现严重骨髓抑制（III-IV 级），50.8%出现不同程度的胃肠道事件，而接受一线治疗和靶向治疗的患者没有严重毒性。4 例患者因肝硬化而行肝移植。患者的肝病在 18-79 个月的随访期间得到改善。本研究表明，伴有肝脏受累的 LCH，特别是胆管炎，预示着预后不良。靶向治疗提供了良好的治疗反应和较少的毒性，但停药后可能会复发。肝移植仍然是终末期肝病患者的可靠挽救选择。
