Growth Hormone Treatment for Idiopathic Short Stature.
Authors: Cutfield WS, Albert BB
Abstract
ISS is the commonest cause of short stature and poor growth and is arbitrarily defined as a height < -2 SDS without an identified cause. ISS consists largely of normal children with the remainder unrecognised conditions, mainly syndromes and genetic (monogenic and polygenic) causes. Growth response to rhGH is widely variable reflecting the heterogeneity of ISS. Further identification of genetic causes of ISS will better characterise treatment response. rhGH during childhood has been shown in RCTs to improve adult height by approximately 4 cm which is less than se...
Source: Pediatric Endocrinology Reviews - November 1, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
Growth Hormone Treatment for Achondroplasia.
Authors: Yorifuji T, Higuchi S, Kawakita R
Abstract
Achondroplasia (ACH) is the most common form of skeletal dysplasia causing rhizomelic, short-limb short stature. Short- and long-term clinical trials have been conducted with rhGH, with similar results across these studies. At supraphysiological dose of GH, height gain of 1-1.5 SDS on the population curve was observed during the first 1-3 years, which was then followed by a smaller increase in growth rate persisting for 5-6 years. These studies led to the approval of rhGH for ACH in Japan where rhGH has been used for 20 years at 0.05 mg/kg/day. Although t...
Source: Pediatric Endocrinology Reviews - November 1, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
Psychosocial Aspects of Short Stature and rhGH Treatment: Implicit Trends over 60+ Years.
Authors: Gardner M, Scerbak T, Sandberg DE
Abstract
Between 1958 and today, advances in research and the clinical management of short stature with GH have occurred. Initially, limited supply of pituitary-derived hGH led to strict criteria for diagnosing GH deficiency and tightly controlled treatment protocols. With the advent of biosynthetic GH, the supply has increased, the number of indications for treatment has grown, and the focus of intervention changed from hormone replacement to treatment of short stature. Improved psychosocial adaptation is an underlying, albeit largely unspoken and inadequately re...
Source: Pediatric Endocrinology Reviews - November 1, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
rhGH Abuse for Sports Performance.
Authors: Rogol AD
Abstract
Doping is at least as old as the ancient Olympics. Substances taken to improve athletic performance ranged from stimulants to hallucinogenic plant substances, but more recently include anabolic agents. Recombinant human growth hormone, rhGH, is one agent with a relatively short history of use, but few data to unequivocally show that it actually improves performance. However, rhGH has therapeutic use for those GH deficient and the concept of a therapeutic use exemption for those with documented deficiency is outlined along with doping control methods. The athlete's biological pass...
Source: Pediatric Endocrinology Reviews - November 1, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
Monitoring rhGH Safety: rhGH Registries, SAGhE and Future Needs.
Authors: Miller BS, Rosenfeld RG
Abstract
The safety of growth hormone (GH) therapy in children has been studied extensively. The identification of Creutzfeldt-Jacob disease in individuals who received pituitary-derived GH led to heightened surveillance for safety issues related to recombinant human GH (rhGH). An excellent safety profile of rhGH has been demonstrated in large Phase IV registries comprising > 600,000 patient-years of rhGH exposure and long-term safety cohorts of adults treated with GH as children. Increased mortality risk has been reported but eliminated when corrected for small size at ...
Source: Pediatric Endocrinology Reviews - November 1, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
Long-Acting Growth Hormone Preparations in the Treatment of Children.
Authors: Lal RA, Hoffman AR
Abstract
Human growth hormone (hGH), which had been in use since 1958, was supplanted by recombinant human growth hormone (rhGH) in 1985 for those with growth hormone deficiency (GHD). Adherence to daily subcutaneous growth hormone is challenging for patients. Thus, several companies have pursued the creation of long acting rhGH. These agents can be divided broadly into depot formulations, PEGylated formulations, pro-drug formulations, non-covalent albumin binding GH and GH fusion proteins. Nutropin Depot is the only long acting rhGH ever approved by the U.S. Food and Drug Admin...
Source: Pediatric Endocrinology Reviews - November 1, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
For Debate: The Significance of Etiologic Diagnosis in Neonates with Overgrowth Syndromes. Lesson Learned from the Simpson-Golabi-Behmel Syndrome.
Authors: Plachý L, Elblová L, Neuman V, Fencl F, Bláhová K, Straňák Z, Lebl J, Průhová Š
Abstract
Overgrowth syndromes are rare genetic disorders characterized by excessive pre- and postnatal growth accompanied by dysmorphic features and developmental disorders. In addition to other health hazards, the life expectancy of affected children may be compromised due to an increased risk of developing tumors. To demonstrate the need for early recognition, correct diagnostic evaluation and adequate follow-up, we present a family with recurrent Simpson-Golabi-Behmel syndrome (SGBS). SGBS is a X-linked neo...
Source: Pediatric Endocrinology Reviews - October 31, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
Is There a Difference between Ultrasonographic (US) Uterine Changes of Oral Versus Transdermal (TD) 17 β Estradiol (17β E2) in Girls with Turner Syndrome (TS)? Own Experience and Literature Review.
CONCLUSIONS: According to our experience, in a group of TS patients randomized to oral vs TD 17β E2 and monitored with trans-abdominal US, both groups achieved similar increases in uterine size comparable to normal women. To confirm our observation a larger sample and a longer evaluation period is needed.
PMID: 30371036 [PubMed - in process] (Source: Pediatric Endocrinology Reviews)
Source: Pediatric Endocrinology Reviews - October 31, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
Challenges in Prenatal Treatment with Dexamethasone.
Authors: McCann-Crosby B, Placencia FX, Adeyemi-Fowode O, Dietrich J, Franciskovich R, Gunn S, Axelrad M, Tu D, Mann D, Karaviti L, Sutton VR
Abstract
Classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency causes elevated androgen levels, which can lead to virilization of female external genitalia. Prenatal dexamethasone treatment has been shown to be effective in preventing virilization of external genitalia when started prior to 7-9 weeks of gestation in females with classic CAH. However, CAH cannot be diagnosed prenatally until the end of the first trimester. Treating pregnant wom...
Source: Pediatric Endocrinology Reviews - October 31, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
The Management of Permanent Primary Hypoparathyroidism in Children and Adolescents: A Complex Task.
Authors: De Sanctis V, Soliman AT, Di Maio S, Kattamis C
Abstract
Management of hypoparathyroidism (hypoPT), depends on the etiology and the severity of hypocalcemia. Treatment goals include control of hypocalcemic symptoms preserving serum calcium in the low-normal range and phosphate in the high normal range. While correction of serum calcium to low-normal range does not fully correct mineral and bone metabolism it may be associated with increased risk of complications such as nephrolithiasis, nephrocalcinosis and soft tissue calcifications. Therefore, it is imperative to find out ways to individualize t...
Source: Pediatric Endocrinology Reviews - October 31, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
Clinical Perspectives of Mitochondrial Disorders.
Authors: Finsterer J
Abstract
Mitochondrial disorders are increasingly recognised world-wide and represent a diagnostic and therapeutic challenge. This is due to the peculiarities of mitochondrial genetics and the extreme genotypic and phenotypic heterogeneity of these disorders. Traditional time-consuming and expensive diagnostic steps are increasingly replaced by first-line genetic approaches. Despite recent advances in the treatment and prevention of mitochondrial disorders, therapeutic approaches are still limited mainly to non-invasive or invasive symptomatic measures.
PMID: 30371039 [PubMed -...
Source: Pediatric Endocrinology Reviews - October 31, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
Meeting Report: 2018 Annual Meeting of the Endocrine Society, Chicago IL (March 17-20, 2018), Selected Highlights.
Authors: Vidmar A, Ali SA, Chao L
Abstract
PMID: 30371040 [PubMed - in process] (Source: Pediatric Endocrinology Reviews)
Source: Pediatric Endocrinology Reviews - October 31, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
Meeting Report: The 51st Annual Meeting of the Japanese Society for Pediatric Endocrinology (JSPE), Osaka, Japan, September 28th-30th, 2017.
Authors: Urakami T
Abstract
PMID: 30371041 [PubMed - in process] (Source: Pediatric Endocrinology Reviews)
Source: Pediatric Endocrinology Reviews - October 31, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
For Debate: Personalized Health Care: As Exemplified by Home Sodium Measurements in a Child with Central Diabetes Insipidus and Impaired Thirst Perception.
CONCLUSION: This case is an example of personalized health care and has led to better self-reliance and quality of life.
PMID: 29806747 [PubMed - in process] (Source: Pediatric Endocrinology Reviews)
Source: Pediatric Endocrinology Reviews - May 30, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
Review of Current Care Models for Transgender Youth and Application to the Development of a Multidisciplinary Clinic - The Seattle Children's Hospital Experience.
We describe our experience at Seattle Children's Hospital in the development of a multidisciplinary Gender Clinic which incorporates the expertise of social work, mental health professionals, pediatric endocrinology, adolescent medicine, and bioethics. Other institutions may build from our experience, with the ultimate goal of further decreasing health disparities for young transgender patients.
PMID: 29806748 [PubMed - in process] (Source: Pediatric Endocrinology Reviews)
Source: Pediatric Endocrinology Reviews - May 30, 2018 Category: Endocrinology Tags: Pediatr Endocrinol Rev Source Type: research
