Schündeln Michael M, Fritzemeier Sebastian, Goretzki Sarah C, Hauffa Pia K, Munteanu Martin, Kiewert Cordula, Hauffa Berthold P, Fleischhack Gudrun, Tippelt Stephan, Grasemann Corinna
Pediatric Hematology and Oncology, Department of Pediatrics III, University Hospital Essen, University of Duisburg-Essen, Essen, Germany.
Pediatric Endocrinology and Diabetology, Department of Pediatrics II, University Hospital Essen, University Duisburg-Essen, Essen, Germany.
Front Pediatr. 2022 Jul 18;10:913343. doi: 10.3389/fped.2022.913343. eCollection 2022.
Childhood primary brain tumors (CPBT) are the second largest group of childhood malignancies and associated with a high risk for endocrine late effects.
To assess endocrine late effects and their relevance for the development of osteopathologies in survivors.
This single center cross sectional study investigated data from 102 CPBT survivors with a mean age of 13.0 years and a mean age at diagnosis of 8.7 years. Clinical, biochemical, radiographic, and anamnestic data regarding endocrine and bone health were obtained at study visits. In addition, data regarding tumor stage and therapy was obtained by chart review. An expert opinion was applied to define presence of osteopathologies.
Impaired bone health, defined by at least one pathological screening parameter, was present in 65% of patients. 27.5% were found to have overt osteopathologies per expert opinion. 37.8% displayed a severe vitamin D deficiency (25-OH vitamin D < 10 ng/ml) and 11% a secondary hyperparathyroidism. Patients with osteopathologies had lower 25-OH vitamin D levels compared to patients without osteopathologies. Multiple endocrine late effects were present: diabetes insipidus in 10.8%, aberrant pubertal development in 13.7%, central hypocortisolism in 14.9%, thyroid dysfunction in 23.8% and growth hormone deficiency in 21.8%. A total of 31.3% of survivors displayed any endocrinopathy. Tumors located near hypothalamic structures and patients who received irradiation had a higher likelihood of endocrine morbidity.
This study indicates that endocrine deficiencies are common in pediatric survivors of CPBTs. Osteopathologies are present in this cohort. A prominent effect of hormonal deficiencies on bone health was not detected, possibly because patients were sufficiently treate for their endocrine conditions or indicating resilience of the childhood bone remodeling process. Vitamin D deficiency is frequent and should be treated as recommended.
儿童原发性脑肿瘤（CPBT）是儿童恶性肿瘤的第二大类型，且与内分泌晚期效应的高风险相关。
评估内分泌晚期效应及其对幸存者骨病发展的相关性。
这项单中心横断面研究调查了102例CPBT幸存者的数据，这些幸存者的平均年龄为13.0岁，诊断时的平均年龄为8.7岁。在研究访视时获取了有关内分泌和骨骼健康的临床、生化、影像学和既往史数据。此外，通过病历审查获取了有关肿瘤分期和治疗的数据。采用专家意见来定义骨病的存在情况。
65%的患者存在骨健康受损，根据至少一项病理筛查参数定义。根据专家意见，27.5%的患者被发现有明显的骨病。37.8%的患者表现出严重的维生素D缺乏（25-羟基维生素D<10 ng/ml），11%的患者有继发性甲状旁腺功能亢进。与无骨病的患者相比，有骨病的患者25-羟基维生素D水平较低。存在多种内分泌晚期效应：尿崩症占10.8%，青春期发育异常占13.7%，中枢性皮质醇缺乏占14.9%，甲状腺功能障碍占23.8%，生长激素缺乏占21.8%。共有31.3%的幸存者表现出任何内分泌病。位于下丘脑结构附近的肿瘤患者和接受过放疗的患者发生内分泌疾病的可能性更高。
本研究表明，内分泌缺乏在CPBT的儿科幸存者中很常见。该队列中存在骨病。未检测到激素缺乏对骨骼健康有显著影响，可能是因为患者的内分泌状况得到了充分治疗，或者表明儿童骨骼重塑过程具有恢复力。维生素D缺乏很常见，应按推荐进行治疗。
