Bander Evan D, Garton Andrew L A, Pasquini Luca, Reiner Anne S, Yildirim Onur, Ilica Ahmet T, Donzelli Maria, Haque Sofia, Souweidane Mark M
Department of Neurological Surgery, Memorial Sloan Kettering Cancer Center, New York City, New York, USA.
Radiology, Memorial Sloan Kettering Cancer Center, New York City, New York, USA.
Neurooncol Adv. 2025 Jul 19;7(1):vdaf128. doi: 10.1093/noajnl/vdaf128. eCollection 2025 Jan-Dec.
Diffuse intrinsic pontine glioma (DIPG) carries a high mortality rate and lacks effective treatment options with a median overall survival (OS) of 8-12 months. Convection-enhanced delivery (CED) has demonstrated safety in phase I trials, but efficacy is indeterminate. Evaluating anatomic patterns of relapse may aid in determining therapeutic efficacy of local CED drug delivery strategies.
Sixty-three children with DIPG were retrospectively reviewed for first radiographic progression. All patients were treated using conventional external beam radiation (EBRT) and 31 were treated with CED of radiolabeled 124-iodine-omburtamab (NCT01502917). Anatomic patterns of initial progression were coded by independent neuroradiologists. OS and cumulative incidence of progression at each anatomic site were assessed in a competing risk analysis with death as a competing variable and were stratified based on CED treatment.
Median OS was 14.67 months for the cohort. Patients receiving CED demonstrated higher rates of progression in general, when considering progression at all anatomical sites (HR 1.79, = .047); no significant difference was found in OS when stratified by CED treatment ( = .22). However, CED treatment was associated with significantly lower cumulative incidence of local pontine and medullary progression (HR: 0.42, = .03; HR 0.14, = .01, respectively) relative to non-CED-treated patients.
Anatomically defined patterns of relapse provide evidence for locoregional control in children with DIPG treated with radioimmunotherapy administered by CED. Future CED or local surgical therapy trials can benefit from including detailed patterns of relapse as a prospective outcome.
弥漫性脑桥内在型胶质瘤(DIPG)死亡率高,缺乏有效的治疗方案,总体中位生存期(OS)为8 - 12个月。对流增强递送(CED)在I期试验中已证明其安全性,但疗效尚不确定。评估复发的解剖学模式可能有助于确定局部CED药物递送策略的治疗效果。
对63例DIPG患儿进行回顾性研究,以观察首次影像学进展情况。所有患者均接受了传统外照射放疗(EBRT),其中31例接受了放射性标记的124碘 - 奥布妥单抗的CED治疗(NCT01502917)。独立神经放射科医生对初始进展的解剖学模式进行编码。在以死亡为竞争变量的竞争风险分析中评估每个解剖部位的OS和进展累积发生率,并根据CED治疗进行分层。
该队列的中位OS为14.67个月。考虑到所有解剖部位的进展情况,接受CED治疗的患者总体进展率较高(风险比1.79,P = 0.047);按CED治疗分层时,OS未发现显著差异(P = 0.22)。然而,与未接受CED治疗的患者相比,CED治疗与脑桥和延髓局部进展的累积发生率显著降低相关(风险比分别为0.42,P = 0.03;0.14,P = 0.01)。
解剖学定义的复发模式为接受CED放射性免疫治疗的DIPG患儿的局部区域控制提供了证据。未来的CED或局部手术治疗试验可以将详细的复发模式作为前瞻性结果纳入,从而从中受益。