Westermann Carly R, Davidson Tom B, Waters Kaaren, Margol Ashley S, Cheung Clement C
Frank H. Netter MD School of Medicine at Quinnipiac University, North Haven, CT, USA.
Cancer and Blood Disease Institute and Division of Hematology and Oncology, Children's Hospital Los Angeles, Los Angeles, CA, USA.
J Pediatr Endocrinol Metab. 2024 Dec 16;38(1):58-64. doi: 10.1515/jpem-2024-0243. Print 2025 Jan 29.
Immune checkpoint inhibitors (ICIs) are emerging treatment options for children with brain tumors, who are already at risk for developing endocrinopathies due to tumor location and treatment. Endocrine ICI-related adverse effects (irAEs) are common in adults but poorly characterized in the pediatric population. The aims of this study were to determine in pediatric brain tumor patients in a single institution (1) if endocrine surveillance took place before and after ICIs were initiated, and (2) the occurrence of endocrine irAEs.
This is a retrospective chart review of 22 pediatric brain tumor patients treated with ICIs at Children's Hospital Los Angeles between 2010 and 2022. We analyzed endocrine laboratory results, patient demographics, and treatment course.
Most patients (82 %) received surveillance in at least one endocrine system before ICI treatment - all had thyroid function tested (100 %) whereas non-thyroid endocrine functions were seldomly assessed (6-22 %). Only those patients with surveillance prior to treatment had ongoing surveillance after ICI initiation - 100 % for thyroid function and 17-39 % for other endocrine systems. Hypothyroidism was the only endocrine problem diagnosed after ICI initiation, in two patients (9 %). Of note, most patients (68 %) expired during or shortly after ICI treatment.
This is one of the first institutional surveys of pediatric ICIs in a high-volume pediatric brain tumor center. Thyroid surveillance commonly occurred in pediatric patients, revealing diagnoses of hypothyroidism, which is consistent with adult data. However, little information is available for non-thyroid endocrine conditions, reflecting the need for comprehensive and systematic endocrine surveillance.
免疫检查点抑制剂（ICI）正在成为治疗脑肿瘤儿童的新选择，而这些儿童由于肿瘤位置和治疗本身就有发生内分泌疾病的风险。内分泌ICI相关不良反应（irAE）在成人中很常见，但在儿科人群中特征描述较少。本研究的目的是在单一机构中确定儿科脑肿瘤患者：（1）在开始ICI治疗前后是否进行了内分泌监测，以及（2）内分泌irAE的发生情况。
这是一项对2010年至2022年期间在洛杉矶儿童医院接受ICI治疗的22例儿科脑肿瘤患者的回顾性病历审查。我们分析了内分泌实验室结果、患者人口统计学资料和治疗过程。
大多数患者（82%）在ICI治疗前至少接受了一个内分泌系统的监测——所有患者都进行了甲状腺功能检测（100%），而非甲状腺内分泌功能很少被评估（6%-22%）。只有那些在治疗前接受监测的患者在ICI开始后继续接受监测——甲状腺功能监测率为100%，其他内分泌系统监测率为17%-39%。甲状腺功能减退是ICI开始后诊断出的唯一内分泌问题，有两名患者（9%）出现该情况。值得注意的是，大多数患者（68%）在ICI治疗期间或治疗后不久死亡。
这是在一家大型儿科脑肿瘤中心对儿科ICI进行的首批机构调查之一。甲状腺监测在儿科患者中普遍进行，发现了甲状腺功能减退的诊断结果，这与成人数据一致。然而，关于非甲状腺内分泌疾病的信息很少，这反映了全面和系统的内分泌监测的必要性。
