Final Height in Children with Idiopathic Growth Hormone Deficiency Treated with Recombinant Human Growth Hormone: The Belgian Experience

Abstract

Background: The growth response to recombinant hGH (rhGH) treatment and final height of 61 Belgian children (32 boys) with idiopathic growth hormone deficiency (GHD) were studied. Patients/Methods: Two patient groups were compared: Group 1 with spontaneous puberty (n = 49), Group 2 with induced puberty (n = 12). The patients were treated with daily subcutaneous injections of rhGH in a dose of 0.5–0.7 IU/kg/week (0.17–0.23 mg/kg/week) from the mean ± SD age of 11.9 ± 3.1 years during 5.1 ± 2.1 years. Results: rhGH treatment induced a doubling of the height velocity during the first year and resulted in a normalisation of height in 53 (87%) patients. Final height was –0.7 ± 1.1 SDS, being 170.4 ± 7.2 cm in boys and 158.0 ± 6.4 cm in girls. Corrected for mid-parental height, final height was 0.0 ± 1.1 SDS. Ninety-two percent of the patients attained an adult height within the genetically determined target height range. Although height gain during puberty was smaller in the patients with induced puberty (boys: 17.1 ± 7.0 cm vs. 27.5 ± 6.6 cm (p < 0.005); girls: 9.6 ± 7.4 cm vs. 22.2 ± 6.1 cm (p < 0.005)), no differences in final height after adjustment for mid-parental height were found between patients with spontaneous or induced puberty. Conclusions: We conclude that patients with idiopathic GHD treated with rhGH administered as daily subcutaneous injections in a dose of 0.5–0.7 IU/kg/week reach their genetic growth potential, resulting in a normalisation of height in the majority of them, irrespective of spontaneous or induced puberty.

Final Height in Children with Idiopathic Growth Hormone Deficiency Treated with a Fixed Dose of Recombinant Growth Hormone

“…Previous reports of final height from various registry-based reports and clinic experience demonstrate variable FAH outcomes [6,7,8,9, 28] (table 3). The FAH in our study was higher than in the Kabi International Growth Study, the French and the Italian studies, and lower compared with the National Cooperative Growth Study, the Swedish and the Belgian group studies [6, 9, 28].…”

“…In fact, none of the studies using a range of rhGH dosing have demonstrated unequivocally that the GH dose significantly influences FAH outcome [6, 8, 18]. Cohen et al [17] suggested that the GH dose response curve in prepubertal children with GHD reaches a plateau at a dose of approximately 0.3 mg/kg/week.…”

“…These parameters have been previously described as independent variables for FAH outcome [3, 4,6,7,8,9, 29]. Previous studies have shown both a positive correlation and no correlation between birth weight and FAH [33, 34].…”

“…The introduction of DNA-derived recombinant human GH preparation (rhGH) in 1985 has facilitated optimization of GH therapy with significant improvements in final adult height (FAH) outcomes [2,6,7,8,9,10]. Ideally, with early diagnosis, appropriate dosing and uninterrupted therapy, FAH very close to the population norms should be achievable [6, 8, 10, 11]. Nonetheless, 20 years after the introduction of rhGH, optimal dosing guidelines continue to be debated [6, 7, 9].…”

Bone Age Progression during the First Year of Growth Hormone Therapy in Pre-Pubertal Children with Idiopathic Growth Hormone Deficiency, Turner Syndrome or Idiopathic Short Stature, and in Short Children Born Small for Gestational Age: Analysis of Data from KIGS (Pfizer International Growth Database)

“…In patients with GHD receiving GH therapy, no correlation was evident between pre-pubertal BA acceleration over the first years of GH therapy and final height [1, 13, 14,18,19,20]; neither was final height influenced by BA progression in short children born SGA [2]. Thus, although the BA/CA ratio may exceed 1.0 in patients receiving GH therapy, this does not seem to have an effect on the height attained.…”