回顾性研究难治性或复发性肝母细胞瘤患者的临床特征和结局：单中心经验。

A retrospective study of clinical features and outcome in patients with refractory or recurrent hepatoblastoma: A single institution experience.

机构信息

MacKay Medical College, New Taipei City, Taiwan; Division of Pediatric Hematology-Oncology, MacKay Memorial Hospital, Taipei, Taiwan; Division of Pediatric Hematology-Oncology, MacKay Children's Hospital, Taipei, Taiwan.

Division of Pediatric Surgery, MacKay Memorial Hospital, Taipei, Taiwan.

出版信息

Pediatr Neonatol. 2021 Jul;62(4):400-405. doi: 10.1016/j.pedneo.2021.03.018. Epub 2021 Apr 27.

DOI:10.1016/j.pedneo.2021.03.018

PMID:33967009

Abstract

BACKGROUND

Hepatoblastoma (HB) is the most common childhood primary hepatic malignancy. The overall survival rate in patients with HB has reached more than 80% over the past decades. The poor prognostic and high-risk HB have been defined, but the treatment and cure of refractory or relapsed HB is still an arduous task.

METHODS

The complete records of HB in patients under the age of 18 at the MacKay Memorial Hospital between 1990 and 2019 were examined.

RESULTS

The treatment results for 11 patients with refractory or relapsed HB are presented. The multi-modality treatment records were reviewed and the clinical characteristics associated with poor outcome included multifocal lesions, low α-fetoprotein, great vessel invasion and metastases. Delayed liver tumor surgery was carried out in eight cases. The median duration of follow-up for the 11 patients was 48.6 months (range 1.9 to 316.8 months). The 5-year and 10-year overall survival rate were 62.3% ± 15% (SE) and 49.9% ± 16.4% (SE), respectively. Most treatment-related toxicities were tolerable. The major concern during long term follow-up was irreversible high-frequency hearing loss.

CONCLUSION

Patients with refractory/relapsed HB are still a thorny issue and more research is needed to improve the outcome.

摘要

背景

肝母细胞瘤（HB）是儿童最常见的原发性肝恶性肿瘤。在过去几十年中，HB 患者的总体生存率已超过 80%。目前已经定义了预后不良和高危 HB，但难治性或复发性 HB 的治疗和治愈仍然是一项艰巨的任务。

方法

检查了 1990 年至 2019 年期间在马偕纪念医院年龄在 18 岁以下的 HB 患者的完整记录。

结果

报告了 11 例难治性或复发性 HB 患者的治疗结果。回顾了多模式治疗记录，与不良预后相关的临床特征包括多发病灶、低甲胎蛋白、大血管侵犯和转移。8 例患者延迟进行肝肿瘤手术。11 例患者的中位随访时间为 48.6 个月（范围 1.9 至 316.8 个月）。5 年和 10 年总生存率分别为 62.3%±15%（SE）和 49.9%±16.4%（SE）。大多数与治疗相关的毒性是可以耐受的。长期随访的主要关注点是不可逆的高频听力损失。