ESPE Abstracts

Background: The treatment of high-risk neuroblastoma still represents a clinical challenge in pediatric oncology. About 50% of children survive after a multimodal treatment based on conventional and high dose chemotherapy, surgery, radiotherapy, differentiating treatment and immunotherapy.

Aim: To evaluate endocrine long-term sequels in a pediatric cohort with high-risk neuroblastoma.

Patients and Methods: The medical records of long-term neuroblastoma survivors with at least 4 years follow-up from diagnosis were reviewed. All patients were treated during a 22 years period.

Results: Forty-seven patients, median age 12.5 years (range 7-27) are alive with 9.7 years median follow-up (range 4-23). We observed long-term complications in 46/47 patients (98%), with a median number of 4 second health events per patient (range 0-10). The most common sequelae are endocrine alterations observed in 66% of long-term survivors followed by cardiac toxicities 64%, hepatic focal nodular hyperplasia in 44%, chronic respiratory diseases in 45%, hearing deficiency in 36% and chronic kidney disease 32%. In particular thyroid toxicities in 83% of patients were observed. Among them, 44% had thyroid toxicities revealed by ultrasound screening: non-cancerous thyroid nodules (32%), papillary thyroid carcinoma (4%), Plummer's adenoma (4%) and autoimmune thyroiditis (4%) and 56% manifested other thyroid toxicities: hypothyroidism (28%) and isolated hyper-TSH (40%). All patients with hypothyroidism were treated through L-thyroxine therapy. Seven out of 30 patients (24%) developed gonadal toxicities, of which 6 females and 1 male. Among them we observed 6 patients with hypergonadotropic hypogonadism, only one patient with hypogonadotropic hypogonadims. A small percentage of patients (20%) showed other endocrinological manifestations including primary hyposurrenalism, central hyposurrenalism and GHD. Moreover, Six patients developed a second neoplasm: 1 ganglioneuroma, 1 osteosarcoma, 1 papillary thyroid carcinoma and 3 myelodysplasia syndrome.

Conclusion: A high proportion of long-term survivors is affected by treatment-associated chronic health conditions, most commonly endocrinological conditions. Further studies with large series are needed to clarify the impact of treatments and to underscore the importance of medical intervention and long-term monitoring of these at-risk subjects to increase overall quality-of-life. We recommend that during follow-up of neuroblastoma children, special attention should be paid to their endocrine function.