ESPE Yearbook of Paediatric Endocrinology

Introduction: As in previous years, most of the included papers concern the medium and long-term complications of cancer therapy. Issues related to long-term surveillance strategies represent an emerging topic of discussion. These highlighted papers have confirmed and consolidated knowledge in the following areas:

– The hormone deficiencies resulting from cancer treatment are predominantly secondary to radiotherapy.

– Chemotherapy tends to cause a minor long-term endocrine damage, although some agents (particularly alkylating agents) have significant gonadotoxic effects.

– Reduced-intensity conditioning regimens have been proposed and are increasingly used in medically fragile patients or in patients with non-malignant diseases to limit the side-effects of hematopoietic stem cell transplantation. Preliminary data suggest that these new regimens reduce the risk of primary ovarian insufficiency.

– Gonadotoxicity of cancer therapy is widely known, but emerging data also suggest a primary effect of cancer on the gonads. Especially neoplasms of the central nervous system and hematological cancers can have a detrimental effect on the gonads. Germ cell abnormalities can be observed even before the start of cancer treatment.

– Immunotherapy and molecular targeted therapies are evolving and represent standard treatment options for some cancers. Checkpoint inhibitors may cause hypophysitis within weeks or months after treatment onset, and may lead to potentially irreversible hypothalamic–pituitary dysfunction. Both tyrosine kinase inhibitors and immune-checkpoint inhibitors have been reported to cause acute thyroid toxicity. The adverse effects of these new drugs need to be extensively studied to inform surveillance recommendations.