Growth hormone improves mobility and body composition in infants and toddlers with Prader-Willi syndrome
ABSTRACT To determine the effect of growth hormone (GH) on body composition and motor development in infants and toddlers with Prader-Willi syndrome (PWS).
Twenty-nine subjects with PWS (4-37 months of age) were randomized to GH treatment (1mg/m 2 /day) or observation for 12 months. Percent body fat, lean body mass, and bone mineral density were measured by dual x-ray absorptiometry; energy expenditure was measured by deuterium dilution; and motor constructs of mobility (M) and stability (S) were assessed using the Toddler Infant Motor Evaluation (TIME).
GH-treated subjects, compared with controls, demonstrated decreased percent body fat (mean, 22.6% +/- 8.9% vs 28.5% +/- 7.9%; P < .001), increased lean body mass (mean, 9.82 +/- 1.9 kg vs 6.3 +/- 1.9 kg; P < .001), and increased height velocity Z scores (mean, 5. 0 +/- 1.8 vs 1.4 +/- 1.0; P < .001). Patients who began GH before 18 months of age showed higher mobility skill acquisition compared with controls within the same age range (mean increase in raw score, 284 +/- 105 vs 206 +/- 63; P < .05).
GH treatment of infants and toddlers with PWS for 12 months significantly improves body composition and when begun before 18 months of age increases mobility skill acquisition. These results suggest that GH therapy instituted early in life may lessen deterioration of body composition in PWS while also accelerating motor development.
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- "More recently growth hormone has emerged as a primary treatment for PWS. Randomized controlled trials have demonstrated significant improvement in height, growth velocity, mobility and body-mass index . However, this success has been accompanied by increasing concern that this treatment regimen may result in development or worsening of OSA. "
ABSTRACT: To review the effectiveness and safety of surgical intervention for obstructive sleep apnea in Prader-Willi syndrome. The muscle hypotonia and obesity associated with Prader-Willi syndrome (PWS) result in a high rate of obstructive sleep apnea (OSA). The use of growth hormone therapy in these patients has been associated with sudden death, raising concerns that such treatment may exacerbate obstructive sleep apnea. As a result, it has been suggested that children with PWS be evaluated for OSA and indications for adenotonsillectomy prior to instituting growth hormone therapy. The true effectiveness of surgical intervention in these cases, however, remains in doubt. Retrospective review of patients with a diagnosis of PWS who underwent adenoidectomy or adenotonsillectomy from January 2001 to July 2009 at a regional, tertiary care children's hospital. Patients underwent pre-operative and post-operative polysomnography. Differences between pre-operative and post-operative body-mass index (BMI), apnea-hypopnea index (AHI), and median oxygen saturation and oxygen saturation nadir were analyzed. Five patients were identified during the study period. Three patients underwent adenotonsillectomy, 1 patient adenoidectomy alone, and another adenotonsillectomy with uvulopalatopharyngoplasty (UPPP). While median AHI was found to have decreased from 16.4 to 4.4, no statistically significant change could be demonstrated (p=0.274). Mean O(2) and nadir O(2) saturation also improved, but without reaching statistical significance. No intra-operative complications were noted. Our series, and other small case series, have demonstrated that complete resolution of sleep apnea in PWS patients is difficult to obtain with upper airway surgery alone. It is suggested that children with PWS being considered for growth hormone therapy undergo assessment for OSA by polysomnography. Patients identified with OSA should be referred for management by tonsillectomy and/or continuous positive airway pressure (CPAP) and then reassessed for residual airway obstruction prior to instituting hormonal therapy.International journal of pediatric otorhinolaryngology 09/2010; 74(11):1270-2. DOI:10.1016/j.ijporl.2010.08.001 · 1.32 Impact Factor
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- "Turner syndrome occurs in females when all or part of an X chromosome is lost, resulting in estrogen deficiency and short stature among a myriad of other markers (Landin-Wilhelmsen et al., 2001). Prader-Willi syndrome is characterized by obesity, short stature and mental retardation, and growth hormone therapy aids in improving body composition and lean body mass (Carrel et al., 2004). Chronic renal failure and muscular wasting in advanced AIDS patients (Gharib, 2003) are also treated by growth hormone therapy. "
ABSTRACT: The combination of materials design and advances in nanotechnology has led to the development of new therapeutic protein delivery systems. The pulmonary, nasal, buccal and other routes have been investigated as delivery options for protein therapy, but none result in improved patient compliances and patient quality of life as the oral route. For the oral administration of these new systems, an understanding of protein transport is essential because of the dynamic nature of the gastrointestinal tract and the barriers to transport that exist.Models have been developed to describe the transport between the gastrointestinal lumen and the bloodstream, and laboratory techniques like cell culture provide a means to investigate the absorption and transport of many therapeutic agents. Biomaterials, including stimuli-sensitive complexation hydrogels, have been investigated as promising carriers for oral delivery. However, the need to develop models that accurately predict protein blood concentration as a function of the material structure and properties still exists.Chemical Engineering Science 11/2009; 64(22):4553-4565. DOI:10.1016/j.ces.2009.04.050 · 2.61 Impact Factor
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- "GH treatment (Allen and Carrel 2004). Many clinicians believe that the benefi ts of GH treatment outweigh the risks in children with PWS; however, there is still debate. "
ABSTRACT: Somatotropin (growth hormone, GH) of recombinant DNA origin has provided a readily available and safe drug that has greatly improved management of children and adolescents with GH deficiency (GHD) and other disorders of growth. In the US and Europe, regulatory agencies have given approval for the use of GH in children and adults who meet specific criteria. However, clinical and ethical controversies remain regarding the diagnosis of GHD, dosing of GH, duration of therapy and expected outcomes. Areas which also require consensus include management of pubertal patients, transitioning pediatric patients to adulthood, management of children with idiopathic short stature and the role of recombinant IGF-1 in treatment. Additionally, studies have demonstrated anabolic benefits of GH in children who have inflammatory-based underlying disease and efficacy of GH in overcoming growth delays in people treated chronically with corticosteroids. These areas are open for possible new uses of this drug. This review summarizes current indications for GH use in children and discusses areas of clinical debate and potential anabolic uses in chronic illness.Targets & therapy 01/2009; 2(4):655-61. DOI:10.2147/BTT.S2252