Safety and Efficacy of Pediatric Growth Hormone Therapy: Results From the Full KIGS Cohort

Mohamad Maghnie, Michael B. Ranke, Mitchell E. Geffner, Elpis Vlachopapadopoulou, Lourdes Ibanez, Martin Carlsson, Wayne Cutfield, Raoul Rooman, Roy Gomez, Michael P. Wajnrajch, Agnes Linglart, Renata Stawerska, Peter E. Clayton, Feyza Darendeliler, Anita C. S. Hokken-Koelega, Reiko Horikawa, Toshiaki Tanaka, Helmuth Gunther Dorr, Kerstin Albertsson-Wikland, Michel PolakAdda Grimberg*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

45 Citations (Scopus)
125 Downloads (Pure)

Abstract

Context The Kabi/Pfizer International Growth Database (KIGS) is a large, international database (1987-2012) of children treated with recombinant human growth hormone (rhGH) in real-world settings. Objective This work aimed to evaluate the safety and efficacy of rhGH from the full KIGS cohort. Methods Data were collected by investigators from children with growth disorders treated with rhGH (Genotropin [somatropin]; Pfizer). Safety was evaluated in all treated patients, and efficacy in those treated for 1 year or more. A subgroup included patients treated for 5 years or more (>= 2 years prepubertal) who had reached near-adult height (NAH). Main outcomes included adverse events (AEs), serious AEs (SAEs), and height growth. Results The full KIGS cohort (N = 83 803 [58% male]) was treated for idiopathic GH deficiency (IGHD; 46.9%), organic GHD (10.0%), small for gestational age (SGA; 9.5%), Turner syndrome (TS; 9.2%), idiopathic short stature (ISS; 8.2%), and others (16.2%). Median rhGH treatment duration was 2.7 years and observation 3.1 years. SAEs occurred in 3.7% of patients and death in 0.4%. The most common SAEs were recurrence of craniopharyngioma (n = 151), neoplasm (n = 99), and cancer (n = 91); and scoliosis (n = 91). Median first-year delta height-SD score (SDS) (Prader) in prepubertal patients was 0.66 (IGHD), 0.55 (ISS), 0.58 (TS), and 0.71 (SGA). Median gains in NAH-SDS were 1.79 (IGHD), 1.37 (ISS), and 1.34 (SGA) for boys, and 2.07 (IGHD), 1.62 (ISS), 1.07 (TS), and 1.57 (SGA) for girls. Conclusion Data from KIGS, the largest and longest running international database of rhGH-treated children, show that rhGH is safe and increases short-term height gain and adult height across GHD and non-GHD conditions.

Original languageEnglish
Pages (from-to)3287-3301
Number of pages15
JournalJournal of Clinical Endocrinology and Metabolism
Volume107
Issue number12
Early online date14 Sept 2022
DOIs
Publication statusPublished - 1 Dec 2022

Bibliographical note

Publisher Copyright:
© 2022 The Author(s). Published by Oxford University Press on behalf of the Endocrine Society.

Fingerprint

Dive into the research topics of 'Safety and Efficacy of Pediatric Growth Hormone Therapy: Results From the Full KIGS Cohort'. Together they form a unique fingerprint.

Cite this