St. Jude Children's Research Hospital, Department of Surgery, Memphis, TN, USA; The University of Tennessee Health Science Center, Department of Surgery, Memphis, TN, USA.
St. Jude Children's Research Hospital, Department of Surgery, Memphis, TN, USA; The University of Tennessee Health Science Center, Department of Surgery, Memphis, TN, USA; Le Bonheur Children's Hospital, Department of Surgery, Memphis, TN, USA.
J Pediatr Surg. 2023 Jun;58(6):1081-1087. doi: 10.1016/j.jpedsurg.2023.02.022. Epub 2023 Feb 18.
The utility of repeated surgical interventions in hepatoblastoma to achieve no evidence of disease (NED) is not well-defined. We examined the effect of aggressive pursuit of NED status on event-free (EFS) and overall survival (OS) in hepatoblastoma with subgroup analysis of high-risk patients.
Hospital records were queried for patients with hepatoblastoma from 2005 to 2021. Primary outcomes were OS and EFS stratified by risk and NED status. Group comparisons were performed using univariate analysis and simple logistic regression. Survival differences were compared with log-rank tests.
Fifty consecutive patients with hepatoblastoma were treated. Forty-one (82%) were rendered NED. NED was inversely correlated with 5-year mortality (OR 0.006; CI 0.001-0.056; P < .01). Ten-year OS (P < .01) and EFS (P < .01) were improved by achieving NED. Ten-year OS was similar between 24 high-risk and 26 not high-risk patients when NED was attained (P = .83). Fourteen high-risk patients underwent a median of 2.5 pulmonary metastasectomies, 7 for unilateral disease, and 7 for bilateral, with a median of 4.5 nodules resected. Five high-risk patients relapsed, and three were salvaged.
NED status is necessary for survival in hepatoblastoma. Repeated pulmonary metastasectomy and/or complex local control strategies to obtain NED can achieve long-term survival in high-risk patients.
Level III - Treatment Study - Retrospective Comparative Study.
在肝母细胞瘤中多次手术干预以达到无疾病证据（NED）的效果尚未明确。我们研究了积极追求 NED 状态对肝母细胞瘤无事件生存（EFS）和总生存（OS）的影响，并对高危患者进行了亚组分析。
我们对 2005 年至 2021 年期间患有肝母细胞瘤的患者的医院记录进行了查询。主要结果是根据风险和 NED 状态分层的 OS 和 EFS。使用单因素分析和简单逻辑回归进行组间比较。使用对数秩检验比较生存差异。
连续治疗了 50 例肝母细胞瘤患者。41 例（82%）达到 NED。NED 与 5 年死亡率呈负相关（OR 0.006；CI 0.001-0.056；P<0.01）。达到 NED 可改善 10 年 OS（P<0.01）和 EFS（P<0.01）。24 例高危患者和 26 例非高危患者达到 NED 时，10 年 OS 相似（P=0.83）。14 例高危患者接受了中位数为 2.5 次的肺转移切除术，7 例单侧疾病，7 例双侧疾病，中位数切除了 4.5 个结节。5 例高危患者复发，3 例患者得到挽救。
NED 状态是肝母细胞瘤生存的必要条件。反复进行肺转移切除术和/或复杂的局部控制策略以获得 NED 可以使高危患者获得长期生存。
III 级-治疗研究-回顾性比较研究。
