Age at Growth Hormone Therapy Start and First-Year Responsiveness to Growth Hormone Are Major Determinants of Height Outcome in Idiopathic Short Stature

Ranke, Michael B.; Lindberg, Anders; Price, David A.; Darendelıler, Feyza; Albertsson‐Wikland, Kerstin; Wilton, Patrick; Reiter, Edward O.

doi:10.1159/000098707

Cited by 89 publications

(108 citation statements)

References 85 publications

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“…Compared to our data in patients with SHOX deficiency, prediction of growth response in GH deficiency seems to be more accurate [21][22][23]. Based on the fact that the growth response in the first year is associated with the long-term outcome, it is reasonable to evaluate the growth response as early as possible to predict the long-term outcome [28][29][30]. Although the experience with GH therapy over 30 years suggests that GH treatment is safe [31,32], discussions about adverse effects especially in the long-term flared up again recently because of results from the French cohort of the Safety and Appropriateness of Growth Hormone Treatments in Europe (SAGhE) study [33,34].…”

Section: Discussionmentioning

confidence: 62%

Growth response to growth hormone treatment in patients with SHOX deficiency can be predicted by the Cologne prediction model

Hoyer‐Kuhn

Franklin

Jones

et al. 2017

Journal of Pediatric Endocrinology and Metabolism

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Background: Growth hormone (GH) treatment in children with short stature homeobox-containing gene (SHOX) deficiency is recognized to increase height velocity (HV) and adult height. Prediction of growth response continues to be a challenge. A comparatively accurate method is the Cologne prediction model developed in children with GH deficiency. The aim was to investigate whether this model also applies to patients with SHOX deficiency. Methods: Included were 48 patients with SHOX deficiency confirmed by DNA analysis and treated with 0.05 mg/kg/ day of somatropin. Prediction by the Cologne model uses the following variables: relative bone age (BA) retardation, baseline insulin-like growth factor-I (IGF-I), urinary deoxypyridinoline (DPD) cross-links at 4 weeks and HV at 3 months. Results: HV and height standard deviation scores (SDS) increased significantly during the first year of treatment. Predicted and observed HV (cm/year) showed a Pearson correlation coefficient of 0.50 (p < 0.001; root-meansquare error = 1.63) and for first-year change in height SDS a Pearson correlation coefficient of 0.751 (p < 0.001; root-mean-square error = 0.32). Poor response could be adequately predicted using SDS change, with sensitivity and specificity both above 70% for certain thresholds. Conclusions:The results demonstrate that the Cologne model can be used to predict growth response in patients

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Section: Discussionmentioning

confidence: 62%

Growth response to growth hormone treatment in patients with SHOX deficiency can be predicted by the Cologne prediction model

Hoyer‐Kuhn

Franklin

Jones

et al. 2017

Journal of Pediatric Endocrinology and Metabolism

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“…Several studies have demonstrated that growth response on GH treatment is positively influenced by a young age at the start of treatment (Ranke et al 2007). Moreover, optimal long-term outcome, such as stature, in inherited disorders depends on early diagnosis and good metabolic control (MaCdonald et al 2012).…”

Section: Discussionmentioning

confidence: 99%

Growth Hormone Deficiency and Lysinuric Protein Intolerance: Case Report and Review of the Literature

et al. 2014

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Background: Lysinuric protein intolerance (LPI; MIM# 222700) is a rare metabolic disorder caused by a defective cationic amino acids (CAA) membrane transport leading to decreased circulating plasma CAA levels and resulting in dysfunction of the urea cycle. Short stature is commonly observed in children with LPI and has been associated with protein malnutrition. A correlation between LPI and growth hormone deficiency (GHD) has also been postulated because of the known interaction between the AA arginine, ornithine, and lysine and growth hormone (GH) secretion. Our report describes a case of GHD in an LPI patient, who has not presented a significant increase in growth velocity with recombinant-human GH (rhGH) therapy, suggesting some possible pathogenic mechanisms of growth failure.Case Presentation: The proband was a 6-year-old boy, diagnosed as suffering from LPI, erythrophagocytosis (HP) in bone marrow, and short stature. Two GH provocative tests revealed GHD. The patient started rhGH therapy and a controlled-protein diet initially with supplementation of oral arginine and then of citrulline. At 3-year follow-up, no significant increase in growth velocity and in insulin-like growth factor-1 (IGF-1) levels was observed. Inadequate nutrition and low plasmatic levels of arginine, ornithine, lysine, and HP may have contributed to his poor growth. Conclusion:Our case suggests that growth failure in patients with GHD and LPI treated with rhGH could have a complex and multifactorial pathogenesis. Persistently low plasmatic levels of lysine, arginine, and ornithine, associated with dietary protein and caloric restriction and systemic inflammation, could determine a defect in coupling GH to IGF-1 production explaining why GH replacement therapy is not able to significantly improve growth impairment. We hypothesize that a better understanding of growth failure pathophysiology in these patients could lead to the development of more rational strategies to treat short stature in patients with LPI.

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“…Generally growth data after one year of treatment with recombinant GH are regarded significant for the response to therapy also during the following years and for the final height outcome [11][12][13][14][15][16][17][18][19]. For our whole group of NSD-patients we found a mean gain of H-SDS of 0,77 ± 0,48, HV almost doubled (8,24 ± 1,58 cm/year versus 4,32 ± 1,68 cm/year at baseline) and HV-SDS increased by 4,40 ± 3,5.…”

Section: Igf-i-and Igfbp3 After 3 Months and Growth Response After 12mentioning

confidence: 99%

Evaluation of Prepubertal Patients with Suspected Neurosecretory Dysfunction of Growth Hormone Secretion: Diagnostic Steps and Treatment Response

Sydlik

Weissenbacher

Roeb

et al. 2017

Advances in Endocrinology

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Background and Aims. Existence and diagnostic procedures of neurosecretory dysfunction of growth hormone (NSD) are still a matter of debate. The aim of our study was (a) to find out if prediagnostic auxological and laboratory data could serve as an indicator for pathologic and normal spontaneous GH-secretion and (b) to evaluate the response to GH-therapy in NSD-patients. Methods. Of 90 children (unicentric study) with normal response to GH-stimulation tests, in whom 12-hour night profiles for GHsecretion were performed, 49 were diagnosed with NSD (NSD group). Their auxologic data, IGF-I/IGFBP3-levels as well as the night profiles, were analysed and compared to those of the non-NSD group. Additionally, follow-up auxological data of the GH-treated NSD-patients were collected. Results. Prediagnostic auxologic and laboratory data did not differ between the two groups. Instead, for all analysed criteria of spontaneous GH-secretion (number of peaks, maximal and mean secretion) a significant difference was found. Children with NSD showed a good response to GH-treatment after 1 (ΔH-SDS +0,77 ± 0,48) as well as 4 years (+1,51 ± 0,75). Conclusion. According to our results, analysing spontaneous GH-secretion remains the only method to identify NSD. Yet, as response to GH-treatment is comparable to results in idiopathic GHD, it is worth to consider this diagnosis.

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Age at Growth Hormone Therapy Start and First-Year Responsiveness to Growth Hormone Are Major Determinants of Height Outcome in Idiopathic Short Stature

Cited by 89 publications

References 85 publications

Growth response to growth hormone treatment in patients with SHOX deficiency can be predicted by the Cologne prediction model

Growth response to growth hormone treatment in patients with SHOX deficiency can be predicted by the Cologne prediction model

Growth Hormone Deficiency and Lysinuric Protein Intolerance: Case Report and Review of the Literature

Evaluation of Prepubertal Patients with Suspected Neurosecretory Dysfunction of Growth Hormone Secretion: Diagnostic Steps and Treatment Response

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