脑干胶质瘤（BSG）是一类临床难治性恶性肿瘤，目前尚无统一有效的治疗方案。超声波和辐射可以激活血卟啉并产生声动力和放射动力效应来杀死癌细胞。因此，我们开展了首个声动力疗法（SDT）联合放疗（RT）治疗BSG的I期临床试验，以验证其安全性和有效性。我们对 11 名 BSG 患者进行了一项 SDT 联合 RT 的研究，这些患者在血卟啉给药后接受了 SDT 和 RT。在此期间进行磁共振成像以评估肿瘤，并记录不良事件。记录的所有不良事件均为1-2级；未观察到 3 级或更严重的不良事件。治疗耐受性良好，未观察到剂量限制性毒性。治疗过程中没有出现与治疗相关的死亡。 11名患者中的8名（72.7%）保持疾病稳定，2名（18.2%）达到部分缓解，并且截至最后一次随访日期肿瘤仍在缩小。患者的中位无进展生存期 (PFS) 为 9.2 (95% 置信区间 [CI] 6.2-12.2) 个月，中位总生存期 (OS) 为 11.7 (95% CI 9.6-13.8) 个月。因此，SDT联合RT具有良好的安全性和可行性，初步显示出较高的治疗潜力。© 2024 UICC。

Brainstem gliomas (BSGs) are a class of clinically refractory malignant tumors for which there is no uniform and effective treatment protocol. Ultrasound and radiation can activate hematoporphyrin and produce sonodynamic and radiodynamic effects to kill cancer cells. Therefore, we conducted the first phase I clinical trial of sonodynamic therapy (SDT) combined with radiotherapy (RT) for the treatment of BSGs to verify its safety and efficacy. We conducted a study of SDT combined with RT in 11 patients with BSGs who received SDT and RT after hematoporphyrin administration. Magnetic resonance imaging was performed during this period to assess the tumor, and adverse events were recorded. All adverse events recorded were grade 1-2; no grade 3 or more serious adverse events were observed. Treatment was well tolerated, and no dose-limiting toxicities were observed. There were no treatment-related deaths during the course of treatment. 8 of 11 patients (72.7%) maintained stable disease, 2 (18.2%) achieved partial response, and the tumors were still shrinking as of the last follow-up date. The median progression-free survival (PFS) for patients was 9.2 (95% confidence interval [CI] 6.2-12.2) months, and the median overall survival (OS) was 11.7 (95% CI 9.6-13.8) months. Therefore, SDT combined with RT has a favorable safety and feasibility and shows a preliminary high therapeutic potential.© 2024 UICC.