ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: Pediatr Blood Cancer. 2019 Oct;66(10):e27927. yeh@mmh.org.tw

This monocentric observational study analysed the efficacy of denosumab in addition to calcium and vitamin D supplementation on bone mineral density (BMD) in 113 young childhood cancer survivors (CCS) with initial BMD Z -score < –1.5.

Low BMD is common in CCS, due to cancer-related factors affecting bone health both directly (cancer-induced osteolysis) and indirectly (nutrition, physical activity, corticosteroid and/or irradiation therapy). The role of denosumab (an inhibitor of receptor activator of nuclear factor kappa-B ligand, RANKL used as antiresorptive medication) is well documented in adults, while it is still rarely used in children. To date, this is the first study to show a significant improvement of BMD Z -score in 20 CCS treated with denosumab, after the completion of chemotherapy.

Overall, denosumab treatment was safe, even if 8 patients experienced hypocalcaemia, which was promptly recognized and corrected. Mean height adjusted BMD Z-score at baseline was −2.68 but increased to −2.0, −1.96, and −1.33 after 0.5, 1, and 1.5 years after denosumab treatment, respectively. These results appear encouraging, but they need to be confirmed in larger cohorts with a longer follow-up. In particular, in this cohort, 5/20 patients were >18 years old at the start of denosumab treatment and so information was limited on the effects of denosumab on linear growth and pubertal growth spurt, since this drug acts on bone remodelling. In addition, neither the fracture risk nor the dose-response effect of therapy was analysed and the observation period reached 1.5-year duration only in 8 treated patients. A controlled trial would be desirable to better evaluate the benefits of denosumab on BMD and the risk of adverse effects, in particular in young growing patients.