scholarly journals Growth Hormone Improves Short-Term Growth in Patients with Temple Syndrome

2018 ◽  
Vol 90 (6) ◽  
pp. 407-413 ◽  
Author(s):  
Diana S. Brightman ◽  
Oluwakemi Lokulo-Sodipe ◽  
Beverly A. Searle ◽  
Deborah J.G. Mackay ◽  
Justin H. Davies ◽  
...  

Background/Aims: Temple syndrome is an imprinting disorder caused by maternal uniparental disomy of chromosome 14 (mat UPD14), paternal deletion of 14q32 or paternal hypomethylation of the intergenic differentially methylated region (MEG3/DLK1 IG-DMR). Patients with Temple syndrome have pre- and postnatal growth restriction, short stature, hypotonia, small hands and feet and precocious puberty. We sought to determine whether treatment with growth hormone improves growth outcomes in patients with Temple syndrome. Methods: This was a retrospective observational study reviewing the medical records of 14 patients with Temple syndrome, 7 of whom were treated with growth hormone. Results: After 1 year of growth hormone treatment, the height standard deviation score (SDS) increased a median of 1.31 SDS with a median increased height velocity of 5.30 cm/year. Conclusions: These results suggest short-term improvement in height SDS with growth hormone treatment similar to the response in patients treated under the small for gestational age indication. We recommend considering growth hormone therapy in all patients with Temple syndrome who have short stature.

2018 ◽  
Vol 90 (3) ◽  
pp. 169-180 ◽  
Author(s):  
Roland Pfäffle ◽  
Christof Land ◽  
Eckhard Schönau ◽  
Paul-Martin Holterhus ◽  
Judith L. Ross ◽  
...  

Background/Aims: To describe characteristics, auxological outcomes and safety in paediatric patients with growth disorders treated with growth hormone (GH), for cohorts from the USA, Germany and France enrolled in GeNeSIS, a post-authorisation surveillance programme. Methods: Diagnosis and biochemical measurement data were based on reporting from, and GH treatment was initiated at the discretion of, treating physicians. Auxological outcomes during the first 4 years of GH treatment and at near-adult height (NAH) were analysed. Serious and treatment-emergent adverse events were described. Results: Children in the USA (n = 9,810), Germany (n = 2,682) and France (n = 1,667) received GH (dose varied between countries), most commonly for GH deficiency. Across diagnostic groups and countries, mean height velocity standard deviation score (SDS) was > 0 and height SDS increased from baseline during the first 4 years of treatment, with greatest improvements during year 1. Most children achieved NAH within the normal range (height SDS >−2). No new or unexpected safety concerns were noted. Conclusion: GH treatment improved growth indices to a similar extent for patients in all three countries despite variations in GH doses. Data from these three countries, which together contributed > 60% of patients to GeNeSIS, indicated no new safety signals and the benefit-risk profile of GH remains unchanged.


2014 ◽  
Vol 55 (1) ◽  
pp. 53 ◽  
Author(s):  
Ho-Seong Kim ◽  
Sei Won Yang ◽  
Han-Wook Yoo ◽  
Byung Kyu Suh ◽  
Cheol Woo Ko ◽  
...  

2017 ◽  
Vol 187 (3) ◽  
pp. 553-569 ◽  
Author(s):  
Peggy Stock ◽  
Maximilian Bielohuby ◽  
Martin S. Staege ◽  
Mei-Ju Hsu ◽  
Martin Bidlingmaier ◽  
...  

2011 ◽  
Vol 74 (3) ◽  
pp. 346-353 ◽  
Author(s):  
Sebastián Susperreguy ◽  
Liliana Muñoz ◽  
Natalia Y. Tkalenko ◽  
Ivan D. Mascanfroni ◽  
Vanina A. Alamino ◽  
...  

1998 ◽  
Vol 8 (4) ◽  
pp. 320
Author(s):  
P Vaguin ◽  
A Shanske ◽  
J Ellison ◽  
P Dowling ◽  
J Heinrich ◽  
...  

2017 ◽  
Vol 102 (4) ◽  
pp. 1291-1298 ◽  
Author(s):  
Amélie Poidvin ◽  
Alain Weill ◽  
Emmanuel Ecosse ◽  
Joel Coste ◽  
Jean-Claude Carel

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