Background Histotripsy is a nonthermal, nonionizing, noninvasive, focused US technique that relies on cavitation for mechanical tissue breakdown at the focal point. Preclinical data have shown its safety and technical success in the ablation of liver tumors. Purpose To evaluate the safety and technical success of histotripsy in destroying primary or metastatic liver tumors. Materials and Methods The parallel United States and European Union and England #HOPE4LIVER trials were prospective, multicenter, single-arm studies. Eligible patients were recruited at 14 sites in Europe and the United States from January 2021 to July 2022. Up to three tumors smaller than 3 cm in size could be treated. CT or MRI and clinic visits were performed at 1 week or less preprocedure, at index-procedure, 36 hours or less postprocedure, and 30 days postprocedure. There were co-primary end points of technical success of tumor treatment and absence of procedure-related major complications within 30 days, with performance goals of greater than 70% and less than 25%, respectively. A two-sided 95% Wilson score CI was derived for each end point. Results Forty-four participants (21 from the United States, 23 from the European Union or England; 22 female participants, 22 male participants; mean age, 64 years ± 12 [SD]) with 49 tumors were enrolled and treated. Eighteen participants (41%) had hepatocellular carcinoma and 26 (59%) had non-hepatocellular carcinoma liver metastases. The maximum pretreatment tumor diameter was 1.5 cm ± 0.6 and the maximum post-histotripsy treatment zone diameter was 3.6 cm ± 1.4. Technical success was observed in 42 of 44 treated tumors (95%; 95% CI: 84, 100) and procedure-related major complications were reported in three of 44 participants (7%; 95% CI: 2, 18), both meeting the performance goal. Conclusion The #HOPE4LIVER trials met the co-primary end-point performance goals for technical success and the absence of procedure-related major complications, supporting early clinical adoption. Clinical trial registration nos. NCT04572633, NCT04573881 Published under a CC BY 4.0 license. Supplemental material is available for this article. See also the editorial by Nezami and Georgiades in this issue.

中文翻译：

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#HOPE4LIVER 单臂关键试验用于原发性和转移性肝脏肿瘤的组织解剖学。


背景 组织解剖学是一种非热、非电离、非侵入性、聚焦的美国技术，依靠空化作用在焦点处进行机械组织破坏。临床前数据表明其在肝脏肿瘤消融方面的安全性和技术成功。目的 评估组织解剖学破坏原发性或转移性肝脏肿瘤的安全性和技术成功率。材料和方法 美国、欧盟和英国的#HOPE4LIVER 平行试验是前瞻性、多中心、单组研究。 2021年1月至2022年7月，在欧洲和美国的14个地点招募了符合条件的患者。最多可以治疗三个小于3厘米的肿瘤。 CT 或 MRI 以及临床就诊在术前 1 周或更短时间、初次手术时、术后 36 小时或更短时间以及术后 30 天进行。共同主要终点是肿瘤治疗的技术成功和 30 天内不存在与手术相关的主要并发症，绩效目标分别高于 70% 和低于 25%。每个终点均得出双边 95% Wilson 评分 CI。结果 44 名患有 49 个肿瘤的参与者（21 名来自美国，23 名来自欧盟或英国；22 名女性参与者，22 名男性参与者；平均年龄，64 岁±12 [SD]）入组并接受治疗。 18 名参与者 (41%) 患有肝细胞癌，26 名参与者 (59%) 患有非肝细胞癌肝转移。治疗前最大肿瘤直径为1.5 cm±0.6，组织解剖后最大治疗区直径为3.6 cm±1.4。 44 个治疗肿瘤中的 42 个观察到技术成功 (95%; 95% CI: 84, 100)，44 名参与者中的 3 名报告了与手术相关的主要并发症 (7%; 95% CI: 2, 18)，两者均满足绩效目标。结论 #HOPE4LIVER 试验达到了技术成功的共同主要终点性能目标，并且不存在与手术相关的主要并发症，支持早期临床采用。临床试验注册号NCT04572633、NCT04573881 根据 CC BY 4.0 许可证发布。本文提供了补充材料。另请参阅本期 Nezami 和 Georgiades 的社论。