Context: Since the identification of GH deficiency due to resistance to GHRH in patients with pseudohypoparathyroidism type Ia (PHP-Ia), no study investigated the effects of recombinant human GH (rhGH) therapy on height velocity (HV) in these patients. Objectives, Patients and Methods: To address this question, eight prepubertal PHP-Ia children with GH deficiency (seven girls and one boy, aged 5.8-12 yr) underwent a 3- to 8-yr treatment with rhGH. Height and HV were measured before and at 6-month intervals during therapy. Nine sex- and age-matched children with idiopathic GH deficiency were monitored during rhGH therapy for comparison. Results: In PHP-Ia children, height SD scores increased from -2.4 ± 0.58 to -1.8 ± 0.47 (P = 0.04) after 12 months, this increase being maintained after the second (-1.6 ± 0.6) and third (-1.15 ± 0.6) year of therapy, similarly to what recorded in children with idiopathic GH deficiency. The HV and HV SD scores after 3 yr maintained a significant increase from 3.5 ± 0.6 to 7.0 ± 0.9 cm/yr (P < 0.0001) and from -2.8 ± 0.8 to +2.2 ± 1.0 (P < 0.0001), respectively. Six patients treated for 4-8 yr had a reduced pubertal spurt and did not improve their near-adult height, with the only exception of one patient in whom estrogen production was blocked by GnRH analogs. Conclusions: We report the first study on the efficacy of rhGH replacement therapy in prepubertal children with PHP-Ia and provide indication that treatment of GH deficiency should be started soon due to the rather limited time window for a potentially effective therapy. Copyright
Recombinant human GH replacement therapy in children with pseudohypoparathyroidism type Ia : first study on the effect on growth / G. Mantovani, E. Ferrante, C. Giavoli, A. Linglart, M. Cappa, M. Cisternino, M. Maghnie, L. Ghizzoni, L. De Sanctis, A.G. Lania, P. Beck Peccoz, A. Spada. - In: THE JOURNAL OF CLINICAL ENDOCRINOLOGY AND METABOLISM. - ISSN 0021-972X. - 95:11(2010 Nov), pp. 5011-5017.
|Titolo:||Recombinant human GH replacement therapy in children with pseudohypoparathyroidism type Ia : first study on the effect on growth|
|Settore Scientifico Disciplinare:||Settore MED/13 - Endocrinologia|
|Data di pubblicazione:||nov-2010|
|Digital Object Identifier (DOI):||http://dx.doi.org/10.1210/jc.2010-1649|
|Appare nelle tipologie:||01 - Articolo su periodico|