用西斯普拉丁重复治疗可能为患有复发性/逆转性肝母细胞瘤的儿童提供生存优势:一个机构经验
Katherine M Somers1, Rachel Bernstein Tabbouche1, Alexander Bondoc2
1Division of Pediatric Hematology/Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Cancers
|August 12, 2023
概括
在复发期间使用西斯普拉丁治疗可能有利于患有肝细胞瘤 (HB) 的儿童. 儿科肝癌中复发性疾病很常见,但不一定与更糟糕的预后有关,尽管毒性需要谨慎管理.
科学领域:
- 儿科瘤学 儿科瘤学
- 肝胆道恶性瘤 肝胆道恶性瘤
- 儿科肝癌研究儿童肝癌研究
背景情况:
- 肝母细胞瘤 (HB) 是最常见的儿科肝癌.
- 对于治疗复发性/耐药性HB (rrHB) 的数据有限.
研究的目的:
- 为了表征患有 rrHB 的儿科患者.
- 描述疾病的特征,生物学,治疗和结果.
主要方法:
- 对 rrHB 患者 (2000 - 2019) 的机构回顾性审查.
- 收集的临床,放射和组织学数据.
- 分析结果,包括生存和毒性.
主要成果:
- 确定了30名 rrHB 患者;22人复发,8人耐火.
- 复发时的平均年龄为32.5个月.
- 总生存率为50%,与复发时使用西斯相关 (p=0.012).
- 常见的突变:CTNNB1 (13/17),NRF2 (4/17). 这两种突变是最常见的.
- 最常见的复发地点:肺部 (12),肝脏 (11).
- 多重复发性疾病 (≥3次复发) 发生在47%的患者中,预后没有恶化.
- 耳毒性的高发病率 (80%轻度听力损失,54%需要助听器).
结论:
- 在复发的HB时,西斯普拉丁重复治疗可能会提供生存优势.
- 多重复发性疾病是常见的,并不是预后更糟糕.
- 将治愈与累积毒性的平衡对于长期结果至关重要.
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