ESPE2023 Poster Category 1 GH and IGFs (48 abstracts)
1Pediatric Endocrinology and diabetes unit, The Edmond and Lilly Safra Children's Hospital, Sheba Medical Center, Ramat Gan, Israel. 2The Sackler School of Medicine, Tel Aviv university, Tel Aviv, Israel. 3Division of Pediatric Hemato-Oncology, The Edmond and Lilly Safra Children's Hospital, Sheba Medical Center, Ramat Gan, Israel
Background and aim: Medulloblastoma is a highly malignant childhood brain tumor, requiring treatment with high dose cranio-spinal irradiation and aggressive chemotherapy. Survivors of are at high risk for multiple endocrine deficiencies, including growth retardation and growth hormone (GH) deficiency. Previous studies reported decreased final adult height in survivors of childhood medulloblastoma, with height standard deviation score (SDS) ranging from -1.9 to -1.54±1.06 in patients treated with GH, and from -5 to −3.2±1.3 in those not treated with GH. We aimed to describe growth outcomes, response to GH treatment, and final height in survivors of childhood medulloblastoma.
Methods: Retrospective analysis of 85 children, adolescents and young adults (males=54) treated for medulloblastoma in a tertiary care center, with a follow-up of at least one year from diagnosis.
Results: Fourteen patients (16.4%) have already completed linear growth at the time of diagnosis. The remaining 71 patients were included in the current analysis. Sixty-two patients (87.3%) exhibited growth retardation over the follow-up period, of whom 36 (58%) were formally diagnosed with GH deficiency, and 23 have been treated with GH. Twenty-eight patients achieved final height at the time of analysis, with a height SDS of -1.46±1.4. Thirteen of these patients were treated with GH, with a mean final height SDS of -1.29±1.5. One GH treated patient (4.3%) had recurrence of medulloblastoma, and three patients had secondary carcinoma of thyroid. In the group not treated with GH, six patients (12.5%) had recurrence of their disease and two had secondary neoplasms (meningioma and uterine leiomyosarcoma).
Conclusion: Growth retardation and GH deficiency are observed in the vast majority of medulloblastoma patients. Final height SDS in our cohort was higher compared to previously published series. We suggest that with careful follow-up and early intervention, optimization of height outcome can be achieved. Long term safety is still a concern, and should be discussed with the family prior to initiating treatment.