A randomized controlled trial of low-dose recombinant human growth hormone in the treatment of malnourished elderly medical patients. Recombinant human growth hormone treatment in children with thalassemia major. Growth hormone treatment in pediatric burns: a safe therapeutic approach. Changes in non-22-kilodalton (kDa) isoforms of growth hormone (GH) after administration of 22-kDa recombinant human GH in trained adult males. Long-acting PEGylated recombinant human growth hormone (Jintrolong) for children with growth hormone deficiency: phase II and phase III multicenter, randomized studies Pilot study of growth hormone administration during the refeeding of malnourished anorexia nervosa patients. Treatment of adults with growth hormone (GH) deficiency with recombinant human GH. A short course of recombinant human growth hormone treatment stimulates osteoblasts and activates bone remodeling in normal human volunteers. Recombinant human growth hormone in short children born small for gestational age. German Study Group. Baseline characteristics and effects of growth hormone therapy over two years in younger and elderly adults with adult onset GH deficiency. Limited effects of growth hormone replacement in patients with GH deficiency during long-term cure of acromegaly Recombinant human chorionic gonadotropin but not dihydrotestosterone alone stimulates osteoblastic collagen synthesis in older men with partial age-related androgen deficiency. Efficacy and safety of a new ready-to-use recombinant human growth hormone solution Long‐term cardiovascular effects of growth hormone treatment in GH‐deficient adults Preliminary data in a small group of patients Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease. Once-Weekly Administration of Sustained-Release Growth Hormone in Korean Prepubertal Children with Idiopathic Short Stature: A Randomized, Controlled Phase II Study Recombinant human growth hormone treatment in pediatric burn patients and its role during the hepatic acute phase response
High-dose recombinant human GH ( rhGH ) has been shown to improve the nutritional status of malnourished older adults . It is uncertain whether low-dose rhGH is effective and whether its effect on nutritional status will lead to any improvement in physical function . There is also no data on the outcome after a short course of rhGH treatment . The objectives of this study were to determine the efficacy of low-dose rhGH treatment for 4 weeks in malnourished elderly patients , its effect on physical functions , and the intermediate term outcome after a 4-week rhGH treatment . The study design was a r and omized , placebo-controlled , double-blind trial conducted in a university teaching hospital . The patients were 19 medically stable malnourished elderly subjects . Intervention in the rhGH group was as follows : rhGH ( Saizen , Serono , Switzerl and ) 0.09 IU/kg body weight ( BW ) 3 times weekly were given together with appropriate dietary intervention as prescribed by the dietitian . In the placebo group , equal volumes of normal saline per kilogram BW were given 3 times weekly together with the dietary intervention . The baseline demographic , anthropometric , nutritional , and hematological variables , measures of physical function , and insulin-like growth factor I levels in both groups were comparable . Compared with the placebo group , the GH-treated group showed a more rapid gain in BW ( after 3 weeks , + 1.27 + /- 0.36 vs. -0.28 + /- 0.37 kg ; P = 0.008 ) , total lean body mass ( change after 3 weeks by bio-impedance analysis , + 1.45 + /- 0.36 vs. -0.37 + /- 0.48 kg ; P = 0.009 ) and a faster improvement in 5-m walking time ( decrease after 4 weeks , 23.79 + /- 9.41 vs. 0.45 + /- 4.62 sec ; P = 0.047 ) . The hemoglobin level rose more in the rhGH than the placebo groups ( change at 8 weeks , + 0.84 + /- 0.34 vs. -0.42 + /- 0.29 g/dL ; P = 0.012 ) . Serum albumin level also showed a greater delayed increase in the rhGH group than in the placebo group ( change at 8 weeks , + 5.1 + /- 0.8 vs. 1.6 + /- 1.2 g/dL ; P = 0.023 ) . There was no statistically significant difference for other nutritional variables . There was a greater rise in the mean serum insulin-like growth factor I level at 4 weeks in the GH than in the placebo groups ( 197 + /- 58 vs. 54 + /- 26 U/L ; P = 0.034 ) . The improvement in the rhGH group gradually diminished on follow-up and became statistically insignificant 8 weeks after stopping rhGH treatment . There were no GH-related adverse effects . Low-dose rhGH was an effective and safe adjuvant to dietary augmentation for stable malnourished elderly subjects . It led to a faster gain in total lean body mass , which was associated with greater improvement in walking speed when compared with dietary intervention alone . There were no apparent side effects BACKGROUND To evaluate the growth hormone reserve and the growth hormone response to recombinant human growth hormone ( GH ) in prepubertal thalassemic children with growth retardation . METHODS Twenty thalassemic patients with short stature and delayed bone age were studied . Patients were r and omized into GH-treated ( n = 10 ) and non-GH treated ( control ; n = 10 ) groups . The GH-treated group received recombinant human (rh)-GH ( Genotropin ) at the dose of 0.7 IU/kg per week for 12 months . RESULTS There was a significant discordance between GH response to pharmacologic stimuli and physiological secretion of GH/GHRH testing . Following the administration of rhGH , growth velocity increased from 2.47 + /- 0.48 cm/year to 6.27 + /- 0.76 cm/year ( P = 0.005 ) , whereas there was not a similar change in the non-GH-treated group . The height velocities of the two groups during the 1 year follow-up period were significantly different ( 6.27 + /- 0.76 vs 3.99 + /- 0.34 cm/year ; P = 0.025 ) . There were significant differences between the height velocity improvements and height velocity st and ard deviation scores of the two groups as well . CONCLUSION The present study has demonstrated that rhGH is a safe and efficacious mode of treatment in thalassemic children OBJECTIVE To determine the safety and efficacy of recombinant human growth hormone ( rhGH ) in the treatment of children who are severely burned . SUMMARY BACKGROUND DATA During the last decade , we have used recombinant human growth hormone ( rhGH ; 0.2 mg/kg/day s.q . ) to successfully treat 130 children with more than 40 % total body surface area ( TBSA ) burns to enhance wound healing and decrease protein loss . A significant increase in the mortality of adult patients in the intensive care unit who were given rhGH  has recently been reported in two large European trials which questions the therapeutic safety of rhGH . METHODS The records of 263 children who were burned were review ed . Patients receiving either rhGH at 0.2 mg/kg/day subcutaneously as part of a r and omized clinical trial ( n = 48 ) or therapeutically ( n = 82 ) were compared with r and omized placebo-administered controls ( n = 54 ) , contiguous matched controls ( n = 48 ) , and matched patients admitted after August 1997 , after which no patients were treated with rhGH ( n = 31 ) . Morbidity and mortality , which might be altered by rhGH therapy , were considered with specific attention to organ function or failure , infection , hemodynamics , and calcium , phosphorous , and albumin balance . RESULTS A 2 % mortality was observed in both rhGH and saline placebo groups in the controlled studies , with no differences in septic complications , organ dysfunction , or heart rate pressure product identified . In addition , no difference in mortality could be shown for those given rhGH therapeutically versus their controls . No patient deaths were attributed to rhGH in autopsies review ed by observers blinded to treatment . Hyperglycemic episodes and exogenous insulin requirements were higher among rhGH recipients , whereas exogenous albumin requirements and the development of hypocalcemia was reduced . CONCLUSIONS Data indicate that rhGH used in the treatment of children who were severely burned is safe and efficacious GH is being used by elite athletes to enhance sporting performance . To examine the hypothesis that exogenous 22-kDa recombinant human GH ( rhGH ) administration could be detected through suppression of non-22-kDa isoforms of GH , we studied seventeen aerobically trained males ( age , 26.9 + /- 1.5 yr ) r and omized to rhGH or placebo treatment ( 0.15 IU/kg/day for 1 week ) . Subjects were studied at rest and in response to exercise ( cycle-ergometry at 65 % of maximal work capacity for 20 min ) . Serum was assayed for total GH ( Pharmacia IRMA and pituitary GH ) , 22-kDa GH ( 2 different 2-site monoclonal immunoassays ) , non-22-kDa GH ( 22-kDa GH-exclusion assay ) , 20-kDa GH , and immunofunctional GH . In the study , 3 h after the last dose of rhGH , total and 22-kDa GH concentrations were elevated , reflecting exogenous 22-kDa GH . Non-22-kDa and 20-kDa GH levels were suppressed . Regression of non-22-kDa or 20-kDa GH against total or 22-kDa GH produced clear separation of treatment groups . In identical exercise studies repeated between 24 and 96 h after cessation of treatment , the magnitude of the responses of all GH isoforms was suppressed ( P < 0.01 ) , but the relative proportions were similar to those before treatment . We conclude : 1 ) supraphysiological doses of rhGH in trained adult males suppressed exercise-stimulated endogenous circulating isoforms of GH for up to 4 days ; 2 ) the clearest separation of treatment groups required the simultaneous presence of high exogenous 22-kDa GH and suppressed 20-kDa or non-22-kDa GH concentrations ; and 3 ) these methods may prove useful in detecting rhGH abuse in athletes Objective We assessed the efficacy and safety of a weekly pegylated human growth hormone ( PEG-rhGH ) ( Jintrolong ) vs daily rhGH for children with growth hormone deficiency ( GHD ) . Design Phase II and III , multicenter , open-label , r and omized controlled trials . Methods 108 and 343 children with treatment-naive GHD from 6 hospitals in China were enrolled in the phase II and III studies respectively . Patients in the phase II study were r and omized 1:1:1 to weekly Jintrolong ( 0.1 mg/kg/week PEG-rhGH complex ) , weekly Jintrolong ( 0.2 mg/kg/week PEG-rhGH complex ) or daily rhGH ( 0.25 mg/kg/week ) for 25 weeks . Patients in the phase III study were r and omized in a 2:1 ratio to weekly Jintrolong ( 0.2 mg/kg/week ) or daily rhGH ( 0.25 mg/kg/week ) for 25 weeks . The primary endpoint for both studies was height velocity ( HV ) increase at the end of treatment . Other growth-related parameters , safety and compliance were also monitored . Results The phase II study established the preliminary efficacy , safety and recommended dose of Jintrolong PEG-rhGH . In the phase III study , we demonstrated significantly greater HV increases in patients receiving Jintrolong treatment ( from 2.26 ± 0.87 cm/year to 13.41 ± 3.72 cm/year ) vs daily rhGH ( from 2.25 ± 0.82 cm/year to 12.55 ± 2.99 cm/year ) at the end of treatment ( P < 0.05 ) . Additionally , significantly greater improvement in the height st and ard deviation scores was associated with Jintrolong throughout the treatment ( P < 0.05 ) . Adverse event rates and treatment compliance were comparable between the two groups . Conclusion Jintrolong PEG-rhGH at a dose of 0.2 mg/kg/week for 25 weeks is effective and safe for GHD treatment and is non-inferior to daily rhGH OBJECTIVE In anorexia nervosa ( AN ) , medical stabilization and nutritional repletion are pivotal steps toward physical and psychological recovery . Nutritional stabilization is often difficult in this patient group . Recombinant human growth hormone ( rhGH ) has been safely used as adjuvant therapy in other groups of malnourished patients . We hypothesize that rhGH treatment will hasten medical stabilization in AN patients . STUDY DESIGN Fifteen patients admitted for inpatient treatment for AN , ages 12 - 18 years , were enrolled in a 28-day r and omized , double-blind , placebo-controlled study . Patients received rhGH ( 0.05 mg/kg subcutaneously ) or an equivalent volume of placebo daily . Outcome measures included time to reach medical/cardiovascular stability , rate of weight gain , and duration of hospitalization . All patients received a st and ard refeeding protocol . RESULTS Mean admission body mass index was 14.5 kg/m2 . The rhGH and placebo groups did not differ significantly in admission weight , BMI or daily caloric intake . Patients treated with rhGH  reached medical/cardiovascular stability more rapidly than those treated with placebo ( median 17 vs. 37 days , p = 0.02 ) . Numerical but not statistically significant improvements were seen in weight gain and length of hospitalization in the rhGH group . CONCLUSION Patients treated with rhGH achieved medical/cardiovascular stability more rapidly than those treated with placebo , and this , in turn , decreased the length of stay In a double blind , cross-over placebo-controlled trial , we studied the effects of 26 weeks of replacement therapy with recombinant human GH on body composition , metabolic parameters , and well-being in 10 patients with adult-onset GH deficiency ( GHD ) . All patients received appropriate thyroid , adrenal , and gonadal replacement therapy . The dose of recombinant human GH was 0.25 - 0.5 U/kg.week ( 0.013 - 0.026 mg/kg.day ) and was administered sc daily at bedtime . One patient was withdrawn from the study because of edema and atrial fibrillation . Body composition was estimated with three independent methods : computed tomography , bioelectric impedance , and total body potassium combined with total body water assessment s. The Comprehensive Psychological Rating Scale and the Symptom Check List-90 were used to assess any change in psychopathology . After 26 weeks of treatment , adipose tissue ( AT ) mass decreased 4.7 kg ( P < 0.001 ) . Subcutaneous AT decreased by an average of 13 % , whereas visceral AT was reduced by 30 % . Muscle volume increased by 2.5 kg ( 5 % ; P < 0.05 ) . According to the four-compartment model derived from assessment s of total body potassium and total body water , body cell mass and extracellular fluid volume increased significantly by 1.6 and 3.0 kg , whereas body fat decreased by 6.1 kg . Results obtained by the bioelectric impedance technique were similar . The mean ( + /- SD ) concentrations of insulin-like growth factor-I increased from 0.26 ( 0.06 ) at baseline to 2.56 ( 1.55 ) and 2.09 ( 1.03 ) kU/L after 6 and 26 weeks of treatment . Calcium and serum phosphate , osteocalcin , and procollagen-III concentrations were significantly higher , and intact PTH concentrations were reduced after 6 and 26 weeks of treatment , respectively . Total and free T3 concentrations were significantly increased after 6 and 26 weeks of treatment , whereas free T4 concentrations were reduced at 6 weeks , but after 26 weeks , free T4 concentrations had returned to pretreatment values . Finally , after 26 weeks of treatment , there was a decrease in the Comprehensive Psychological Rating Scale score ( P < 0.05 ) . The results show that GH replacement in GHD adults results in marked alterations in body composition , fat distribution , and bone and mineral metabolism and reduces psychiatric symptoms . Finally , we conclude that the observed beneficial effects of replacement therapy with GH are of sufficient magnitude to consider treatment of GHD adults The effects of recombinant human growth hormone ( rhGH ) on biochemical markers of bone turnover and bone mineral content ( BMC ) were investigated in 20 normal male volunteers ( aged 22 - 31 years ) r and omized to treatment for 7 days with either rhGH  ( 0.1 IU/kg subcutaneously twice a day ) or placebo .  Serum somatomedin C rose during treatment ( p less than 0.001 ) but was not significantly different from baseline at day 14 . The fasting urinary hydroxyproline/creatinine ( p less than 0.001 ) and calcium/creatinine ratios ( p less than 0.01 ) increased during treatment and remained elevated for 4 and 2 weeks , respectively . Serum bone gamma-carboxyglutamic acid-containing protein ( BGP ) increased during treatment ( p less than 0.001 ) and remained elevated for 6 months ( p less than 0.02 ) . Serum bone alkaline phosphatase ( B-AP ) , after an initial fall in the treatment period ( p less than 0.001 ) , increased slightly in the following months ( p less than 0.01 ) . In the rhGH group BMC was significantly higher than the pre study value at day 14 ( p less than 0.05 ) but was unaltered at the end of study . The simultaneous increase in markers of bone resorption and formation during rhGH treatment followed by a decline in resorption parameters within a few weeks and the prolonged effect on BGP and B-AP demonstrate that rhGH treatment stimulates osteoblasts and activates bone remodeling The effects of recombinant human growth hormone ( rhGH ) treatment in 69 prepubertal , non-GH deficient children born small for gestational age ( SGA ) were evaluated over 2 years . At start of the study mean age was 5.1 years , mean bone age was 3.8 years and mean height SDS was -4.0 . The children were r and omly allocated to 3 groups receiving no treatment or daily subcutaneous injections of rhGH at a dose of 0.1 IU/kg body weight ( group 0.1 IU ) or 0.2 IU/kg body weight ( group 0.2 IU ) . At start of the study mean height velocity SDS was -1.4 in the control group , -0.7 in group 0.1 IU and -1.4 in group 0.2 IU . After 2 years there was a significant increase in height velocity SDS in children treated with rhGH as compared to untreated children . Mean height velocity SDS after the first year of treatment was -1.2 in the control group , 2.8 in group 0.1 IU and 5.5 in group 0.2 IU . Corresponding values during the second year were -0.9 , 1.6 and 2.9 . A statistically significant difference was observed between the groups receiving 0.1 IU/kg/day and 0.2 IU/kg/day during the first year of treatment , whereas no difference between the treatment groups was found during the second year . Catch-up growth , i.e. a height velocity 1 SD above the mean , was achieved for 86 % of group 0.1 IU and 95 % of group 0.2 IU during the first year of treatment and was maintained for 65 % and 79 % of the patients in group 0.1 IU and 0.2 IU respectively in the second year . GH treatment was associated with a distinct acceleration of bone age . Tolerance of treatment was good . No clear trends were seen in any of the laboratory variables . In conclusion , this study shows that daily rhGH given at a dose of 0.1 - 0.2 IU/kg/day for 24 months is an effective and safe therapy to increase linear growth and induce catch-up growth in short SGA children CONTEXT The effects of GH replacement in elderly GH-deficient ( GHD ) adults are not well known . OBJECTIVE / DESIGN / PATIENTS In this prospect i ve , single-center , open-label study , baseline characteristics and the effects of 2-yr GH replacement were determined in 24 GHD adults above 65 yr of age and in 24 younger GHD patients ( mean age , 37 yr ; range , 27 - 46 yr ) . All patients had adult onset disease , and both groups were comparable in terms of the number of pituitary hormonal deficiencies , gender , body mass index , and waist/hip ratio . Duration of hypopituitarism was , however , longer in the elderly patients . RESULTS The mean maintenance dose of GH was 0.31 ( sem , 0.03 ) mg/d in the elderly GHD patients and 0.44 ( 0.04 ) mg/d in the younger patients . The less marked response in IGF-I sd score , total body fat , and extracellular water in the elderly patients lost significance when the dose of GH was accounted for in the statistical analyses . Despite the lower dose in the elderly GHD group , these patients had a more marked reduction in waist/hip ratio and serum low-density lipoprotein-cholesterol level , and these differences remained also after correction for duration of hypopituitarism . There was no difference at baseline or in responsiveness in lean mass , bone mineral density , and glucose homeostasis . CONCLUSIONS This study identifies elderly GHD adults as a GH-sensitive group in whom a low dose of GH can improve body composition and serum lipid profile without any significant impairment of glucose metabolism .  GH replacement should therefore be considered in elderly GHD adults The aim of this study was to assess the effects of replacement with recombinant human growth hormone ( rhGH ) in patients with GH deficiency ( GHD ) after treatment of acromegaly . Intervention study . Sixteen patients ( 8 men , age 56 years ) , treated for acromegaly by surgery and radiotherapy , with an insufficient GH response to insulin-induced hypoglycaemia , were treated with 1 year of rhGH replacement . Study parameters were assessed at baseline and after 1 year of rhGH replacement . Study parameters were cardiac function , body composition , bone mineral density ( BMD ) , fasting lipids , glucose , bone turnover markers , and Quality of Life ( QoL ) . During rhGH replacement  IGF-I concentrations increased from −0.4 ± 0.7 to 1.0 ± 1.5 SD ( P = 0.001 ) , with a mean daily dose of 0.2 ± 0.1 mg in men and 0.3 ± 0.2 mg in women . Nonetheless , rhGH replacement  did not alter cardiac function , lipid and glucose concentrations , body composition or QoL. Bone turnover markers ( PINP and β crosslaps ) levels increased ( P = 0.005 and P = 0.021 , respectively ) , paralleled by a small , but significant decrease in BMD of the hip . The beneficial effects of rhGH replacement in patients with GHD during cure from acromegaly are limited in this study Several r and omized trials of and rogen supplementation in older men have been undertaken . However , the relative contributions of testosterone ( T ) and estrogens on bone metabolism in aging men are controversial . Within the setting of two double-blind , placebo-controlled studies , we evaluated the effect of dihydrotestosterone ( DHT ) and recombinant human chorionic gonadotropin ( rhCG ) on bone turnover in healthy , community-dwelling older men with partial and rogen deficiency ( total T < or = 15 nmol/liter ) . In the first study , 35 men ( age 68.3 + /- 6.8 yr ; baseline T , 13.9 + /- 3.3 nmol/liter ) were r and omized to receive either daily transdermal DHT ( n = 17 ) or placebo for 3 months . In the second study , 40 men ( age 67.4 + /- 5.4 yr ; baseline T , 11.4 + /- 2.2 nmol/liter ) were r and omized to receive either rhCG s.c . ( n = 20 ) , two injections weekly , or placebo for 3 months . The following parameters were measured before , monthly during , and 1 month after treatment : serum T , estradiol ( E2 ) , and LH ; markers of bone formation , serum amino-terminal propeptide of type I procollagen ( S-PINP ) and osteocalcin ; markers of bone resorption , serum carboxyterminal cross-linked telopeptide of type I collagen and urinary deoxypyridinoline . Compared with placebo , treatment with DHT significantly increased serum DHT and suppressed LH and T levels , whereas E2 concentrations and markers of bone turnover did not change . In contrast , rhCG therapy significantly increased both T and E2 , with the increases in E2 being supraphysiological . At the same time , rhCG significantly increased S-PINP concentrations with peak levels after 1 month ( Delta40 % ; P = 0.02 compared with placebo ) . In contrast , serum osteocalcin and carboxyterminal cross-linked telopeptide of type I collagen and urinary deoxypyridinoline levels did not change . The change in S-PINP levels correlated with the change in E2 levels ( r = 0.59 ; P = 0.02 ) but not with a change in T. We conclude that in older men with partial age-related and rogen deficiency , rhCG treatment stimulates osteoblastic collagen formation proportionally to increased E2 concentrations but does not alter markers of mature osteoblastic function or bone resorption . In contrast , treatment with a pure , nonaromatizable and rogen ( DHT ) has no effect on bone turnover despite a 20-fold increase in serum levels . Bone resorption was not accelerated during unchanged ( DHT ) or increased ( rhCG ) E2 levels , suggesting that minimal E2 levels are needed to maintain stable resorption , although direct and rogen receptor-mediated effects can not be excluded . If and rogen supplementation is required for aging men , aromatizable and rogens with sufficient endogenous estrogenic activity may have the most beneficial effects on bone We report 24-month interim results of two multicenter phase III studies in previously untreated children with growth failure secondary to GH deficiency ( GHD ) that were paramount to the development of a new recombinant human GH ( rhGH , somatropin ) , approved as the first ‘ biosimilar ’ in Europe . Study 1 consisted of 3 parts performed in 89 children . The objective was to compare efficacy and safety of the lyophilized formulation of the new somatropin [ Somatropin Powder ( S and oz ) ] with a licensed reference rhGH preparation and the liquid formulation of the new somatropin [ Somatropin Solution ( S and oz ) ] and to assess long-term efficacy and safety of this ready-to-use Somatropin Solution . Study 2 was performed in 51 children and design ed to demonstrate efficacy and safety of Somatropin Powder and to confirm its low immunogenic potential ; rhGH was given sc at a daily dose of 0.03 mg/kg . Primary [ body height , height SD score ( HSDS ) , height velocity , and height velocity ( HV ) SD score ( HVSDS ) ] and secondary [ IGF-I and IGF binding protein 3 ( IGFBP-3 ) ] efficacy end-points and safety parameters were assessed regularly . In study 1 , all treatments showed comparable increases in growth . The baseline-adjusted difference between Somatropin Powder and the reference rhGH product in mean HV was −0.20 cm/yr ( 95 % confidence interval ( CI ) [ −1.34;0.94 ] ) and in mean HVSDS was 0.76 ( 95 % CI [ −0.57;2.10 ] ) after 9 months . These very small differences demonstrate comparable therapeutic efficacy between the two treatments . The results of study 2 were consistent with those seen in study 1 . Equivalent therapeutic efficacy and clinical comparability in terms of safety and immunogenicity between Somatropin Powder and the reference rhGH product and between Somatropin Powder and Somatropin Solution was demonstrated . The safety and immunogenicity profiles were similar and as expected from experience with rhGH preparations OBJECTIVE The long‐term cardiovascular effects of GH administration in adults are of major clinical importance , given the increasing use of such treatment . We have evaluated long‐term cardiovascular effects of recombinant human GH ( rhGH ) substitution in GH deficient men OBJECTIVE To conduct an open-label , multinational , multicenter study examining the safety and efficacy of recombinant human acid alpha-glucosidase ( rhGAA ) in treatment of infantile-onset Pompe disease . STUDY DESIGN We enrolled 8 infant patients who had Pompe disease with GAA activity < 1 % of normal , cardiomyopathy , and hypotonia . In the 52-week initial phase , rhGAA was infused intravenously at 10 mg/kg weekly ; an extension phase continued survivors ' treatment with 10 to 20 mg/kg of rhGAA weekly or 20 mg/kg every 2 weeks for as long as 153 weeks . Safety measurements included adverse events , laboratory tests , and anti-rhGAA antibody titers . Efficacy evaluations included survival , ventilator use , echocardiograms , growth , and motor and cognitive function . RESULT After 52 weeks of treatment , 6 of 8 patients were alive , and 5 patients were free of invasive ventilator support . Clinical improvements included ameliorated cardiomyopathy and improved growth and cognition . Five patients acquired new motor milestones ; 3 patients walked independently . Four patients died after the initial study phase ; the median age at death or treatment withdrawal for all patients was 21.7 months , significantly later than expected for patients who were not treated . Treatment was safe and well tolerated ; no death was drug-related . CONCLUSION rhGAA improved ventilator-free survival , cardiomyopathy , growth , and motor function in patients with infantile-onset Pompe disease compared with outcomes expected for patients without treatment Background / Aims : To determine the optimal dose of LB03002 , a sustained-release , once-weekly formulation of recombinant human growth hormone ( rhGH ) , and to compare its efficacy and safety with daily rhGH in children with idiopathic short stature ( ISS ) . Methods : This multicenter , r and omized , open-label , phase II study included GH-naïve , prepubertal children with ISS , r and omized to receive daily rhGH 0.37 mg/kg/week ( control , n = 16 ) , LB03002 0.5 mg/kg/week ( n = 14 ) , or LB03002 0.7 mg/kg/week ( n = 16 ) . The primary endpoint was height velocity ( HV ) change at week 26 . Results : At week 26 , the least square ( LS ) means for HV change ( cm/year ) with control , LB03002 0.5 mg/kg/week , and LB03002 0.7 mg/kg/week were 5.08 , 3.65 , and 4.38 , and the LS means for the change in height st and ard deviation score were 0.65 , 0.49 , and 0.58 , respectively . The lower bound of the 90 % confidence interval for the difference between LB03002 0.7 mg/kg/week and the control in the LS mean for HV change ( –1.72 ) satisfied the noninferiority margin ( –1.75 ) . Adverse events were generally mild and short-lived . Conclusion : A once-weekly regimen of LB03002 0.7 mg/kg demonstrated noninferiority to the daily regimen of rhGH 0.37 mg/kg/week in terms of HV increments . LB03002 was well tolerated and its safety profile was comparable with that of daily rhGH Objective : Recombinant human growth hormone ( rHGH ) has been shown to increase mortality in adult trauma patients ; however , little has been reported on its side effects in children . The acute phase response has been suggested to be a contributing factor to trauma mortality . Therefore , the purpose of this study was to examine the effects of exogenous rHGH on the acute phase response in pediatric burn patients . Design : Prospect i ve , r and omized , double‐blind study . Setting : Shriners Hospital for Children . Patients : Thermally injured pediatric patients , ranging in age from 0.1 to 16 yrs . Interventions : Twenty‐eight thermally injured children received either 0.2 mg/kg/day of rHGH or saline ( placebo ) within 3 days of admission and for at least 25 days . Measurements and Main Results : Measurements were patient demographics , incidence of sepsis , inhalation injury , mortality , serum constitutive proteins , acute phase proteins , proinflammatory cytokines and insulin‐like growth factor‐I ( IGF‐I ) , insulin‐like growth factor binding protein (IGFBP)‐1 , and IGFBP‐3 . No differences could be demonstrated in age , gender , burn size , incidence in sepsis ( 20 % vs. 26 % ) , inhalation injury ( 46 % vs. 27 % ) , or mortality ( 8 % vs. 7 % ) between those receiving rHGH or placebo . Serum IGF‐I and IGFBP‐3 increased with rHGH treatment , whereas serum IGFBP‐1 decreased compared with placebo ( p < .05 ) . Burned children treated with rHGH required significantly less albumin substitution to maintain normal levels compared with placebo ( p < .05 ) . Those receiving rHGH demonstrated a decrease in serum C‐reactive protein and serum amyloid‐A and an increase in serum retinol‐binding protein compared with placebo ( p < .05 ) . rHGH  decreased serum tumor necrosis factor‐α and interleukin (IL)‐1β , whereas no changes were found for serum IL‐1α , IL‐6 , and IL‐10 compared with placebo ( p < .05 ) . Free fatty acids were elevated in burned children who received rHGH ( p < .05 ) . Conclusion : Data indicate that rHGH does not increase mortality . rHGH  decreased acute phase proteins , tumor necrosis factor‐α , and IL‐1β , which is associated with increases in constitutive hepatic proteins and
RhGh did not consistently affect blood pressure or bone mineral density .
RhGH consistently improved quality of life ( QoL ) parameters reflected in AGHDA-scores .
Conclusion RhGH replacement in elderly subjects with GHD  decreases LDL cholesterol levels and improves QoL , but the effects on other parameters are not unequivocal .
There were no data on the efficacy and safety  of rhGH treatment in octogenarians with GHD
Context Recombinant human GH ( rhGH ) is prescribed for the treatment of adults with GH deficiency ( GHD ) .
However , conflicting data are available on the efficacy of rhGH treatment in elderly GHD patients .
Objective To assess the efficacy of rhGH treatment in elderly GHD subjects .