Hishiki Tomoro, Ida Kohmei, Watanabe Kenichiro, Mori Makiko, Fujimura Junya, Nogami Yuki, Iehara Tomoko, Hoshino Ken, Yokoi Akiko, Honda Shohei, Kazama Takuro, Sekiguchi Masahiro, Kitagawa Norihiko, Matsumura Risa, Nomura Motonari, Yamada Yohei, Hanaki Ryo, Kaneda Hide, Takama Yuichi, Inoue Takeshi, Tanaka Yukichi, Miyazaki Osamu, Nagase Hiroki, Takimoto Tetsuya, Yoshimura Kenichi, Hiyama Eiso
Department of Pediatric Surgery, Chiba University Graduate School of Medicine, Chiba, Japan.
Department of Pediatrics, Teikyo University Mizonokuchi Hospital, Kawanagawa, Japan.
Pediatr Blood Cancer. 2025 Aug;72(8):e31770. doi: 10.1002/pbc.31770. Epub 2025 May 9.
Complete resection of the primary tumor is critical for the survival of children with hepatoblastomas (HBs). This prospective clinical study aimed to clarify the outcome of a chemotherapy regimen comprising cisplatin and doxorubicin (PLADO) followed by definitive surgery conducted at the appropriate time in patients with intermediate-risk HB.
Intermediate-risk HB was defined as patients without evidence of metastatic disease who met any of the following criteria: age ≥3 years; PRETEXT IV disease; presence of more than one PRETEXT annotation factor. The study protocol consisted of four preoperative and two postoperative courses of PLADO. The appropriate surgeries were conducted at optimized timings via real-time central surgical reviews.
The 3-year progression-free and overall survivals of the 33 intermediate-risk patients included were 78.7% and 87.9%, respectively. Preoperative PLADO resulted in a partial response in 83.9% of the patients. Microscopic complete resection was ultimately obtained in 31 (94%) patients. No patient required more than six preoperative courses before surgery or liver transplantation (LTx). Two patients never had surgery due to tumor progression.
The outcome for patients with intermediate-risk HB was satisfactory. PLADO, combined with surgery (including LTx) conducted at the optimal time, appeared to cure most patients in this study.
对于肝母细胞瘤(HB)患儿的生存而言,原发肿瘤的完整切除至关重要。这项前瞻性临床研究旨在阐明由顺铂和阿霉素(PLADO)组成的化疗方案,随后在中度风险HB患者的适当时间进行确定性手术的结果。
中度风险HB定义为无转移疾病证据且符合以下任何标准的患者:年龄≥3岁;PRETEXT IV期疾病;存在多个PRETEXT注释因素。研究方案包括四个术前和两个术后PLADO疗程。通过实时中央手术评估在优化时机进行适当的手术。
纳入的33例中度风险患者的3年无进展生存率和总生存率分别为78.7%和87.9%。术前PLADO使83.9%的患者产生部分缓解。最终31例(94%)患者实现了显微镜下完整切除。没有患者在手术或肝移植(LTx)前需要超过六个术前疗程。两名患者因肿瘤进展从未接受手术。
中度风险HB患者的结果令人满意。在本研究中,PLADO与在最佳时间进行的手术(包括LTx)相结合似乎治愈了大多数患者。