This SR highlights the lack of long-term RCTs reporting final height data and other important qualitative outcomes , such as quality of life .
ABSTRACT Genotropin ( somatropin ) is licensed for the treatment of children with growth hormone deficiency , Prader-Willi syndrome , Turner syndrome , chronic renal insufficiency and in children born small for gestational age .
This systematic review ( SR ) evaluated the clinical efficacy and effectiveness of Genotropin in these conditions to inform a NICE Technology Appraisal of growth hormone for the treatment of growth failure in children .
The d3/fl-growth hormone (GH) receptor polymorphism does not influence the effect of GH treatment (66 microg/kg per day) or the spontaneous growth in short non-GH-deficient small-for-gestational-age children: results from a two-year controlled prospective study in 170 Spanish patients.
CONTEXT The d3/fl-GH receptor ( d3/fl-GHR , exon 3-deleted/full-length GHR ) has recently been associated with responsiveness to GH therapy . OBJECTIVE The objective of the study was to evaluate whether the d3/fl-GHR genotypes influence the intensity of spontaneous and /or GH therapy-stimulated growth in small-for-gestational-age ( SGA ) patients . DESIGN This was a 2-yr prospect i ve , controlled , r and omized trial . SETTING Thirty Spanish hospitals participated .  Auxologic and GH secretion evaluation was hospital based , whereas molecular analyses and auxologic data computation were central ized . PATIENTS Patients included 170 short SGA children : 140 remained prepubertal and 30 entered puberty during the second follow-up year . INTERVENTION Eighty-six were treated with GH ( 66 microg/kg.d ) for 2 yr and 84 were not treated . MAIN OUTCOME MEASURES Previous and 2-yr follow-up auxologic data were recorded at each hospital , d3/fl-GHR genotypes determined , and data analyzed for patients who remained prepubertal ( group 1 , 68 GH treated and 72 non-GH treated ) and for all the patients ( group 2 ) . RESULTS In group 1 GH-treated patients , growth velocity , and height-sd score during the first and second years , total 2-yr height gain ( 18.5 + /- 2.4 cm in d3/d3 ; 18.4 + /- 2.6 in d3/fl ; 19.5 + /- 2.3 in fl/fl ) , Delta 2-yr height increase ( 9.1 + /- 2.4 cm in d3/d3 ; 9.4 + /- 3.0 in d3/fl ; 10.4 + /- 2.1 in fl/fl ) , first-year growth prediction and studentized residual values ( 0.08 + /- 1.26 in d3/d3 ; 0.28 + /- 1.21 in d3/fl ; 0.67 + /- 0.95 in fl/fl ) did not differ among the d3/fl-GHR genotypes . In group 1 non-GH-treated patients , neither growth velocity nor height-sd score changed significantly , and values were similar in each d3/fl-GHR genotype . Results in all patients ( group 2 ) were similar to those in group 1 . CONCLUSIONS In short non-GH-deficient SGA children , both spontaneous growth rate and responsiveness to 66 microg/k.d GH therapy were similar for each d3/fl-GHR genotype carried
This SR highlights the lack of long-term RCTs reporting final height data and other important qualitative outcomes , such as quality of life .
ABSTRACT Genotropin ( somatropin ) is licensed for the treatment of children with growth hormone deficiency , Prader-Willi syndrome , Turner syndrome , chronic renal insufficiency and in children born small for gestational age .
This systematic review ( SR ) evaluated the clinical efficacy and effectiveness of Genotropin in these conditions to inform a NICE Technology Appraisal of growth hormone for the treatment of growth failure in children .