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复发性或难治性小儿肝母细胞瘤和肝细胞癌的RELIVE联盟:对问题的范围审查及提出的解决方案

The RELIVE consortium for relapsed or refractory pediatric hepatoblastoma and hepatocellular carcinoma: a scoping review of the problem and a proposed solution.

作者信息

O'Neill Allison F, Trobaugh-Lotrario Angela, Geller James I, Hiyama Eiso, Watanabe Kenichiro, Aerts Isabelle, Fresneau Brice, Toutain Fabienne, Sullivan Michael J, Katzenstein Howard M, Morland Bruce, Branchereau Sophie, Zsiros József, Maibach Rudolf, Ansari Marc

机构信息

Department of Pediatric Oncology, Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA, USA.

Providence Sacred Heart Children's Hospital, Spokane, WA, USA.

出版信息

EClinicalMedicine. 2024 Feb 15;69:102446. doi: 10.1016/j.eclinm.2024.102446. eCollection 2024 Mar.

Abstract

Liver tumors account for approximately 2% of all pediatric malignancies. Children with advanced stages of hepatoblastoma (HB) are cured only 50-70% of the time while children with advanced hepatocellular carcinoma (HCC) have a <20% 5-year overall survival. This scoping review was performed to highlight the paucity of rigorous, reliable data guiding the management of relapsed pediatric HB or HCC. When these patients are enrolled on prospective trials, the trials are often histology-agnostic, exclude patients less than a year of age, lack a liquid formulary of the drug under study, exclude recipients of a solid organ transplant, and enroll only 1-2 patients limiting the ability to deduce efficacious regimens for current use or future study. We highlight the creation of a global pediatric consortium intended to source retrospective relapse data from over 100 institutions spanning 4 continents. The data collected from this effort will inform future relapse trials.

摘要

肝肿瘤约占所有儿童恶性肿瘤的2%。患有晚期肝母细胞瘤(HB)的儿童只有50%-70%的治愈率,而患有晚期肝细胞癌(HCC)的儿童5年总生存率低于20%。本综述旨在强调在指导复发性儿童HB或HCC治疗方面,缺乏严格、可靠的数据。当这些患者参加前瞻性试验时,试验往往不区分组织学类型,排除年龄小于1岁的患者,缺乏所研究药物的液体制剂,排除实体器官移植受者,且仅招募1-2名患者,限制了推导当前使用或未来研究有效方案的能力。我们强调创建一个全球儿科联盟,旨在从四大洲100多个机构获取回顾性复发数据。这项工作收集的数据将为未来的复发试验提供参考。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2ef1/10879668/37d3d38bb0f5/gr1.jpg

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