Photodynamic therapy for malignant brain tumors in children and young adolescents.
Photodynamic therapy for malignant brain tumors in children and young adolescents.
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DOI：
10.3389/fonc.2022.957267
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发表时间：
2022
影响因子：
4.7
通讯作者：
Kawamata, Takakazu
中科院分区：
文献类型：
--
作者：
Chiba, Kentaro;Aihara, Yasuo;Oda, Yuichi;Fukui, Atsushi;Tsuduki, Shunsuke;Saito, Taiichi;Nitta, Masayuki;Muragaki, Yoshihiro;Kawamata, Takakazu
光动力疗法（PDT）靶向切除后的肿瘤细胞残余物。在这里，我们评估了PDT治疗儿童和青少年恶性脑肿瘤的可行性。这是一项单中心、非随机、I/II期临床研究。主要终点是他拉泊芬钠（TS）治疗的安全性（I期）和PDT后的总生存期（OS）（II期）。次要终点是PDT后的无进展生存期（PFS）。从初始入组病例开始，每3例病例通过10 - 20 - 40 mg/m2剂量递增确定TS剂量。8例患者在PDT时平均年龄为170.2个月（129-214个月），接受了9次手术，PDT后平均随访时间为16.8个月（1-42个月）。组织病理学诊断包括幕上间变性室管膜瘤（n = 2）、间变性星形细胞瘤（n = 1）、伴有H3 K27 M突变的弥漫性中线胶质瘤（n = 1）、胶质母细胞瘤（n = 3）和儿科高级别胶质瘤（n = 1）。结果是5名患者存活，3名患者死亡。8例患者中有6例复发，其余2例在PDT后无复发。因此，OS和PFS分别计算为21和6个月。通常观察到癫痫发作和发热，这可能是手术相关症状。未观察到成人常见不良反应光敏性皮疹或肝功能障碍。我们的研究结果表明，TS可以安全地用于儿童，剂量与成人相当，因为TS给药没有严重并发症。然而，由于参与者数量较少，我们无法对PDT的疗效做出明确的结论。由于小儿脑肿瘤的罕见性和术前鉴别诊断的困难，考虑到广泛的组织病理学发现，积累病例是困难的。此外，儿科患者与遮光管理相关的心理压力比最初预期的更严重。总之，与成人剂量相当的TS用于6至20岁的儿童和青少年可能是安全的。然而，需要进一步的研究来阐明其有效性。
Photodynamic therapy (PDT) targets tumor cell remnants after resection. Here, we evaluated the feasibility of PDT for malignant brain tumors in children and young adolescents. This was a single-center, non-randomized, phase I/II clinical study. The primary endpoints were the safety of treatment with talaporfin sodium (TS) (phase I) and overall survival (OS) after PDT (phase II). The secondary endpoint was progression-free survival (PFS) after PDT. The TS dose was determined by dose escalation from 10 to 20 to 40 mg/m2 for every three cases starting from the initial enrolled case. Eight patients with a mean age of 170.2 months (129–214 months) at the time of PDT received nine procedures with a mean follow-up duration of 16.8 months (1–42 months) after PDT. Histopathological diagnoses included supratentorial anaplastic ependymoma (n = 2), anaplastic astrocytoma (n = 1), diffuse midline glioma with H3K27M mutation (n = 1), glioblastoma (n = 3), and pediatric high-grade glioma (n = 1). The outcome was survival in five patients and death in three patients. Recurrence occurred in six of the eight patients; the remaining two were recurrence-free after PDT. Therefore, OS and PFS were calculated as 21 and 6 months, respectively. Seizures and fevers, which were likely surgery-related symptoms, were commonly observed. Photosensitive skin rashes or liver dysfunction, which are common adverse effects in adults, were not observed. Our results showed that TS can be used safely in children at doses comparable to those used in adults, as there was no major complication associated with TS administration. However, we cannot make a definitive conclusion about the efficacy of PDT because of the small number of participants. Accumulating cases was difficult because of the rarity of pediatric brain tumors and the difficulty in making a preoperative differential diagnosis, considering the wide range of histopathological findings. Moreover, the psychological stress associated with light-shielding management in pediatric patients was more severe than initially expected. In conclusion, TS at doses comparable to those used in adults may be safe for use in children and young adolescents between the ages of 6 and 20 years. However, further studies are needed to clarify its efficacy.
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影响因子：
158.5
作者：
Stupp, R;Mason, WP;Ryan, G
通讯作者：
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