Background: Paediatric hepatocellular carcinoma (HCC), including its fibrolamellar variant (FLC), is a rare malignancy with distinct biological behaviour and limited therapeutic options. While complete surgical resection is a key determinant of survival, many patients present with unresectable tumours at diagnosis. The role of neoadjuvant chemotherapy in improving resectability, particularly in histologically distinct subtypes, remains inconclusive.Methods: We retrospectively analysed 43 patients (<18 years) with histologically confirmed conventional HCC (cHCC,n= 27) or FLC (n= 16) enrolled in the German Pediatric Liver Tumour Registry. We assessed clinical characteristics, treatment response, surgical outcomes, and survival. Special focus was placed on the impact of neoadjuvant chemotherapy in initially unresectable tumours.Results: FLC and cHCC exhibited significant differences in clinical presentation, such as age of presentation, AFP elevation, or presence of underlying liver disease. Although overall survival did not significantly differ between groups, cHCC tumours showed a markedly higher response to chemotherapy (62.5% partial remission vs. 0% in FLC). Complete resection (R0) was achieved in 77% of all patients and was the strongest predictor of survival. Importantly, a subset of cHCC patients who initially had unresectable tumours became eligible for curative surgery following neoadjuvant chemotherapy. Notably, delayed resection after chemotherapy led to outcomes comparable to those with upfront surgery, whereas progression during chemotherapy was associated with a universally poor prognosis.Conclusions: This study supports upfront resection as the preferred strategy in paediatric HCC and FLC whenever feasible. In cHCC, neoadjuvant chemotherapy demonstrated a favourable response profile and contributed to secondary resectability in a subset of initially unresectable cases, supporting a potential role within a multimodal treatment approach. In contrast, FLC showed limited responsiveness to current systemic therapies. These findings emphasise the importance of histology-specific strategies and highlight the ongoing need for more effective systemic options, particularly for unresectable FLC.
背景：儿童肝细胞癌（HCC）及其纤维板层亚型（FLC）是一种罕见的恶性肿瘤，具有独特的生物学行为且治疗选择有限。虽然完全手术切除是生存的关键决定因素，但许多患者在确诊时已存在不可切除的肿瘤。新辅助化疗在提高可切除性方面的作用，尤其是在组织学不同的亚型中，目前尚无定论。 方法：我们回顾性分析了德国儿童肝脏肿瘤登记处收录的43例经组织学确诊为经典型肝细胞癌（cHCC，n=27）或纤维板层亚型（FLC，n=16）的儿童患者（年龄<18岁）。我们评估了其临床特征、治疗反应、手术结果和生存情况，特别关注新辅助化疗对初始不可切除肿瘤的影响。 结果：FLC与cHCC在临床表现上存在显著差异，例如发病年龄、甲胎蛋白升高或是否存在基础肝病。尽管两组间的总生存率无显著差异，但cHCC对化疗的反应率明显更高（部分缓解率为62.5%，而FLC为0%）。所有患者中77%实现了完全切除（R0），这是生存的最强预测因素。重要的是，一部分初始不可切除的cHCC患者在接受新辅助化疗后获得了根治性手术的机会。值得注意的是，化疗后延迟切除的患者，其结局与直接手术的患者相当，而化疗期间出现进展则普遍预后不良。 结论：本研究支持，在可行的情况下，直接手术切除是儿童HCC和FLC的首选策略。对于cHCC，新辅助化疗显示出良好的反应特征，并使一部分初始不可切除的病例获得了二次切除机会，这支持了其在多模式治疗方案中的潜在作用。相比之下，FLC对当前全身性疗法的反应有限。这些发现强调了制定基于组织学亚型的个体化策略的重要性，并凸显了对更有效全身治疗方案（尤其是针对不可切除FLC）的持续需求。
肿瘤(癌症)患者之家