Assessment of Endocrine and Metabolic Late Effects After Childhood Brain Tumor Treatment

Overview

This prospective, single-group interventional study evaluates endocrine and metabolic late effects in survivors of primary brain tumors treated during childhood. According to the study protocol, participants were prospectively assigned to undergo the Study-Specific Endocrine and Metabolic Late-Effects Assessment. The assessment included anthropometric measurements, repeated blood pressure measurements, clinical endocrine evaluation, fasting laboratory testing, and protocol-defined bone and vascular assessments. The purpose of the study was to identify the frequency and severity of endocrine and metabolic late effects and to evaluate their associations with tumor characteristics, previous cancer treatment, age at diagnosis, and physical functioning.

Survivors of primary brain tumors treated during childhood may develop endocrine, metabolic, neurological, psychological, and cardiovascular late effects several years after completion of cancer treatment. This prospective, single-group interventional study included individuals who had been treated for a primary brain tumor in Slovenia between 2008 and 2018 while aged 0 to 18 years and who had survived for at least 5 years after diagnosis. Following enrollment and informed consent, all participants were prospectively assigned, according to the study protocol, to undergo the same Study-Specific Endocrine and Metabolic Late-Effects Assessment. Participation in the study determined the standardized manner and intensity with which the protocol-defined procedures were performed; participants would not otherwise necessarily have received the same assessment package during routine clinical follow-up. The protocol-defined assessment included review of medical history and previous cancer treatment, measurement of body weight, height, waist and hip circumference, arm span and sitting height, repeated blood pressure measurements, clinical assessment of endocrine and pubertal status, and fasting laboratory testing of endocrine and metabolic markers. The study-specific assessment also included vascular evaluation by measurement of carotid intima-media thickness.When the relevant results were not already available, dual-energy X-ray absorptiometry for the assessment of bone mineral density and body composition, and pulse wave velocity assessment, were performed or arranged according to the study protocol. Medical record data on tumor type and location, age at diagnosis, treatment modalities, date of treatment completion, current therapy, and previously identified late effects were also collected. No investigational medicinal product was administered. The primary objective was to identify the occurrence and severity of endocrine and metabolic late effects following treatment of childhood brain tumors. Additional analyses evaluated associations between the identified late effects and tumor location, tumor type, treatment modality, age at diagnosis, and physical functioning.