Cheri Deal1, Susan Kirsch2, Jean-Pierre Chanoine2, Sarah Lawrence2, Elizabeth Cummings2, Elizabeth T Rosolowsky2, Seth D Marks2, Nan Jia2, Christopher J Child2. 1. University of Montreal and Centre hospitalier universitaire Sainte-Justine (Deal), Montréal, Que.; Lilly Research Laboratories (Kirsch), Toronto, Ont.; Endocrinology and Diabetes Unit (Chanoine), British Columbia Children's Hospital, Vancouver, BC; Division of Endocrinology and Metabolism (Lawrence), Children's Hospital of Eastern Ontario, Ottawa, Ont.; Division of Endocrinology (Cummings), IWK Health Centre, Dalhousie University, Halifax, NS; Division of Endocrinology (Rosolowsky), Department of Pediatrics, University of Alberta, Edmonton Clinic Health Academy, Edmonton, Alta.; Department of Pediatrics and Child Health (Marks), Children's Hospital Health Sciences Centre Winnipeg, University of Manitoba, Winnipeg, Man.; Lilly Research Laboratories (Jia), Indianapolis, Ind.; Eli Lilly and Company (Child), Windlesham, UK Cheri.L.Deal@umontreal.ca. 2. University of Montreal and Centre hospitalier universitaire Sainte-Justine (Deal), Montréal, Que.; Lilly Research Laboratories (Kirsch), Toronto, Ont.; Endocrinology and Diabetes Unit (Chanoine), British Columbia Children's Hospital, Vancouver, BC; Division of Endocrinology and Metabolism (Lawrence), Children's Hospital of Eastern Ontario, Ottawa, Ont.; Division of Endocrinology (Cummings), IWK Health Centre, Dalhousie University, Halifax, NS; Division of Endocrinology (Rosolowsky), Department of Pediatrics, University of Alberta, Edmonton Clinic Health Academy, Edmonton, Alta.; Department of Pediatrics and Child Health (Marks), Children's Hospital Health Sciences Centre Winnipeg, University of Manitoba, Winnipeg, Man.; Lilly Research Laboratories (Jia), Indianapolis, Ind.; Eli Lilly and Company (Child), Windlesham, UK.
Abstract
BACKGROUND: Country-specific data on outcomes of treatment with recombinant human growth hormone are lacking. We present such data for children treated with growth hormone in Canada. METHODS: We describe characteristics and outcomes of 850 children (mean age at baseline 8.5 yr) treated with growth hormone constituting the Canadian cohort of the multinational phase IV prospective observational Genetics and Neuroendocrinology of Short-stature International Study (GeNeSIS). The diagnosis associated with short stature was as determined by the investigator. Auxological data were evaluated yearly until near-adult height. Adverse events were assessed in all growth-hormone-treated patients. RESULTS: The diagnosis ascribed as the cause of short stature was growth hormone deficiency in 526 children (61.9%), predominantly organic rather than idiopathic, particularly congenital pituitary abnormalities and intracranial tumours. All diagnostic groups with sufficient patients for analysis had increased height velocity standard deviation score (SDS) and height SDS during growth hormone treatment. For patients who reached near-adult height (n = 293), the mean height SDS was within the normal range for about 80% of patients with organic growth hormone deficiency (n = 131) or idiopathic growth hormone deficiency (n = 50), 50% of patients with idiopathic short stature (n = 10) and 46% of patients with Turner syndrome (n = 79). Eleven deaths were reported, 7 in patients with organic growth hormone deficiency. Serious adverse events considered related to growth hormone treatment (n = 19) were isolated except for medulloblastoma recurrence (n = 2) and adenoidal hypertrophy (n = 2). INTERPRETATION: Growth hormone treatment was effective and had a good safety profile in Canadian children. Growth hormone dosages were lower than in the US and global GeNeSIS cohorts, and a greater proportion of treated Canadian children had organic growth hormone deficiency. STUDY REGISTRATION: ClinicalTrials.gov, no. NCT01088412. Copyright 2018, Joule Inc. or its licensors.
BACKGROUND: Country-specific data on outcomes of treatment with recombinant humangrowth hormone are lacking. We present such data for children treated with growth hormone in Canada. METHODS: We describe characteristics and outcomes of 850 children (mean age at baseline 8.5 yr) treated with growth hormone constituting the Canadian cohort of the multinational phase IV prospective observational Genetics and Neuroendocrinology of Short-stature International Study (GeNeSIS). The diagnosis associated with short stature was as determined by the investigator. Auxological data were evaluated yearly until near-adult height. Adverse events were assessed in all growth-hormone-treated patients. RESULTS: The diagnosis ascribed as the cause of short stature was growth hormone deficiency in 526 children (61.9%), predominantly organic rather than idiopathic, particularly congenital pituitary abnormalities and intracranial tumours. All diagnostic groups with sufficient patients for analysis had increased height velocity standard deviation score (SDS) and height SDS during growth hormone treatment. For patients who reached near-adult height (n = 293), the mean height SDS was within the normal range for about 80% of patients with organic growth hormone deficiency (n = 131) or idiopathic growth hormone deficiency (n = 50), 50% of patients with idiopathic short stature (n = 10) and 46% of patients with Turner syndrome (n = 79). Eleven deaths were reported, 7 in patients with organic growth hormone deficiency. Serious adverse events considered related to growth hormone treatment (n = 19) were isolated except for medulloblastoma recurrence (n = 2) and adenoidal hypertrophy (n = 2). INTERPRETATION:Growth hormone treatment was effective and had a good safety profile in Canadian children. Growth hormone dosages were lower than in the US and global GeNeSIS cohorts, and a greater proportion of treated Canadian children had organic growth hormone deficiency. STUDY REGISTRATION: ClinicalTrials.gov, no. NCT01088412. Copyright 2018, Joule Inc. or its licensors.
Authors: D B Allen; P Backeljauw; M Bidlingmaier; B M K Biller; M Boguszewski; P Burman; G Butler; K Chihara; J Christiansen; S Cianfarani; P Clayton; D Clemmons; P Cohen; F Darendeliler; C Deal; D Dunger; E M Erfurth; J S Fuqua; A Grimberg; M Haymond; C Higham; K Ho; A R Hoffman; A Hokken-Koelega; G Johannsson; A Juul; J Kopchick; P Lee; M Pollak; S Radovick; L Robison; R Rosenfeld; R J Ross; L Savendahl; P Saenger; H T Sorensen; K Stochholm; C Strasburger; A Swerdlow; M Thorner Journal: Eur J Endocrinol Date: 2015-11-12 Impact factor: 6.664
Authors: Philippe Backeljauw; Shankar Kanumakala; Sandro Loche; Karl Otfried Schwab; Roland Werner Pfäffle; Charlotte Höybye; Elena Lundberg; Tadej Battelino; Berit Kriström; Tomasz Giemza; Hichem Zouater Journal: Horm Res Paediatr Date: 2021-07-07 Impact factor: 2.852