Incidence of diabetes mellitus and neoplasia in japanese short-statured children treated with growth hormone in the Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS)

Susumu Yokoya, Tomonobu Hasegawa, Keiichi Ozono, Hiroyuki Tanaka, Susumu Kanzaki, Toshiaki Tanaka, Kazuo Chihara, Nan Jia, Christopher J. Child, Katsuichiro Ihara, Jumpei Funai, Noriyuki Iwamoto, Yoshiki Seino

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The primary goal of the Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS) was to assess the safety and effectiveness of Humatrope®, a GH preparation, in the treatment of pediatric patients with short stature. We report our findings in the GH-treated Japanese pediatric population focusing on the incidence of type 2 diabetes (T2D) and occurrence of neoplasms. A total of 2,345 Japanese patients were assessed for safety. During a mean observation period of 3.2 yr, T2D occurred in 3 patients (0.13%) and slowly progressive insulin-dependent diabetes mellitus (SPIDDM) related to underlying mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) in 1 patient (0.04%). Neoplasms were reported in 13 patients (0.56%), including 1 patient with brain tumor (germinoma) and 5 with craniopharyngiomas (4 recurrences); the remainder were benign, typically dermatological, neoplasms. The incidence of diabetes mellitus determined in the study did not differ from previous reports in GH-treated pediatric patients, and there was no apparent increase in the risk of new neoplastic lesions or malignant tumors.

Original languageEnglish
Pages (from-to)229-241
Number of pages13
JournalClinical Pediatric Endocrinology
Issue number4
Publication statusPublished - 2017



  • Diabetes mellitus
  • Neoplasia
  • Pediatric GH treatment
  • Safety
  • Short stature

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Endocrinology, Diabetes and Metabolism
  • Endocrinology

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