ESPE Abstracts (2015) 84 P-1-82

Assessment of Primary Cancers in Growth Hormone-Treated Paediatric Patients Compared with General Population Registries: An Epidemiological Analysis of a Large, Multinational, Prospective Observational Study

Christopher Childa, Alan Zimmermannb, Nan Jiab, Leslie Robisonc, Jürgen Brämswigd & Werner Blume


aEli Lilly and Company, Windlesham, UK; bEli Lilly and Company, Indianapolis, IN, USA; cSt Jude Children’s Research Hospital, Memphis, TN, USA; dUniversitäts-Kinderklinik, University of Münster, Münster, Germany; eUniversity of Giessen, University Children’s Hospital, Giessen, Germany


Background: Concern remains regarding the potential influence of growth hormone (GH) treatment on neoplasia because of the general growth-inducing effect of GH and associations between high serum IGF1 concentrations and certain cancers in adulthood. Many studies that evaluated risk for primary cancer in GH-treated patients without previous malignancy found no increased rates of primary neoplasia. A higher risk for colorectal cancer was observed in a single-country cohort treated with cadaveric GH.

Objective and hypotheses: To assess primary cancer occurrence in the prospective, multinational GeNeSIS observational study of paediatric GH use and compare observed cases with rates from general population cancer registries (USA: SEER; other countries: GLOBOCAN).

Method: Study data and serious adverse event reports for patients with ≧1 follow-up visit were examined to identify and exclude those without previous history of malignancy and to ascertain incident cases of primary cancer. 19054 GH-treated patients without previous cancer were identified (40% female; 63% with GH deficiency, 13% idiopathic short stature, 9% Turner syndrome, 6% born small for gestational age, and 9% other diagnoses; mean±SD baseline age 9.5±4.0 years; mean follow-up time 3.4±2.5 years).

Results: Thirteen incident primary cancers were ascertained, with mean±SD age at cancer onset of 13.5±2.7 years. The standardized incidence ratio (SIR) (95% CI) for primary cancers was 1.02 (0.54–1.75) for all countries combined; no individual country had a significantly elevated SIR. Ten of the affected patients (four lymphoma cases, three germ cell tumours, Ewing sarcoma, osteosarcoma, and skin cancer) had no neoplastic history. Three patients had neoplastic history/predisposition (rectal adenocarcinoma in a patient with neurofibromatosis and Gardner syndrome, pancreatic neuroendocrine tumour in a patient with neurofibromatosis, and malignant schwannoma in a patient with previous pilocytic astrocytoma).

Conclusion: Risk for all sites primary cancers in GH-treated patients without previous cancer was no higher in GeNeSIS than in general population cancer registries.

Conflict of interest: The author is an employee and stockholder of Eli Lilly and Company.

Funding: Sponsored by Eli Lilly and Company.