Oral Presentation ESA-SRB-ANZOS 2025 in conjunction with ENSA

Evolving morbidities and transition challenges for survivors of childhood posterior fossa tumours (128689)

Quynh-Nhu Nguyen 1 2 3 4 , Angelina Lim 5 , Jack Cullen 5 , David Metz 6 7 , Greg Wheeler 1 3 , Margaret Zacharin 5 6
  1. Victorian Paediatric Integrated Cancer Service, Parkville, Victoria, 3052
  2. Stem Cells and Development, Monash Biomedicine Discovery Institute, and Department of Anatomy and Developmental Biology, Monash University, Clayton, Victoria, Australia
  3. Peter MacCallum Cancer Centre, Parkville, Victoria, 3052
  4. Department of Paediatric Endocrinology & Diabetes, Monash Children's Hospital, Clayton, Victoria, 3168
  5. Department of Endocrinology & Diabetes, Royal Children's Hospital Melbourne, Parkville, Victoria, 3052
  6. Murdoch Children's Research Institute, Parkville, Victoria, 3052
  7. Department of Paediatrics, Monash University, Clayton, Vic, 3168

Aims 

Medulloblastoma and ependymoma comprise the commonest childhood infratentorial malignancies. Most patients receive cranial/craniospinal irradiation, surgery, plus combination chemotherapy. Sequelae include multiple pituitary hormone deficiencies, disinhibition of hypothalamic restraint on puberty, risks of thyroid malignancy, reduced fertility, neurocognitive impairment, and later second malignant neoplasms (SMN).  Care is usually undertaken in tertiary survivorship clinics, but awareness of complications and their management is increasingly important for clinicians outside this setting. We aimed to characterise long-term outcomes and identify unmet needs in patients treated for posterior fossa malignancies in childhood or adolescence. 

Methods  

Audit of long-term health outcomes for 136 patients with medulloblastoma (N=106) or ependymoma (N=30) treated at the Royal Children’s Hospital and Peter MacCallum Cancer Centre, referred to endocrinology 1980-2025. 

Results  

Amongst 118 5-year survivors, most developed ≥1 pituitary hormone deficiencies. Growth hormone (GH) deficiency occurred in 55; significant functional benefits were reported by 25/27 treated in adulthood. TSH and ACTH deficiency were frequently reported.  In females, primary ovarian insufficiency (32) manifested as pubertal failure/arrest, or post-pubertal failure.  In males, biochemical evidence of impaired fertility was frequent and oligoazoospermia observed on semen analysis; 10 required treatment for androgen deficiency. Malignancy was found in 17/39 patients who developed thyroid nodules. Other SMN occurred in 16, most commonly sarcomas, high-grade gliomas, haematologic malignancies and skin cancers (4); 10 developed meningiomas and 9, bowel polyps. 10 patients had strokes. Educational outcomes were poorer than the general population; many patients derived self-reported benefits from low-dose stimulant therapy (34/37). 

Conclusions  

We report a high burden of complex and evolving morbidity requiring multidisciplinary care and life-long surveillance, underlining the need to educate and support adult practitioners caring for these patients. Additionally, our data suggests that adult GH replacement and the use of stimulants may significantly improve neurocognitive function and quality of life.