Predictors of Response to Growth Hormone Treatment and Final Height in Medulloblastoma Survivors

Background: Abnormal growth patterns and impaired final height (FH) are well- recognized long-term complications of childhood medulloblastoma, however data regarding response to growth hormone treatment (GHT) and determinants of FH are scarce. We aim to describe growth characteristics and response to GHT in survivors of childhood medulloblastoma, and to identify factors associated with FH outcome. Methods: A retrospective study comprising all pediatric patients treated for medulloblastoma in a tertiary care center between 1987-2023 and followed up for at least one year following diagnosis. Results: 65/67 patients included in the final analysis (97%) exhibited growth retardation, of whom 38 (58%) were diagnosed with GH deficiency, and 26 were treated with GH. Fifteen of the GH-treated patients achieved FH, with a mean FH-SDS of -1.42±1.47. Only eight (53%) achieved FH within the normal range. FH-SDS of survivors not treated with GH (−1.72 ± 1.25) was lower than that of GH-treated survivors, but this difference did not reach statistical significance. The most important predictor of FH outcome in GH-treated patients was height-SDS at initiation of treatment. Conclusion: Growth retardation and GH deficiency are observed in the vast majority of medulloblastoma patients. Our results suggest that early identification and treatment of GH deficiency are crucial for optimization of FH outcome. Long-term safety of GHT is still a concern and should be discussed with the family prior to initiating treatment.