For height outcome , articles were limited to studies reporting adult height and to systematic review s. FINDINGS Recombinant human growth hormone therapy of children with idiopathic short stature increases height in some children .
The cost-benefit ratio is controversial .
AND RELEVANCE Growth hormone treatment may be considered in some children with idiopathic short stature
IMPORTANCE Approximately 2 % of children are defined as having short stature .
Deciding when to pursue recombinant human growth hormone therapy to increase adult height is controversial .
OBJECTIVE To review the management of children with idiopathic short stature , including diagnostic evaluation and therapeutic options .
Patient, Physician, and Consumer Drivers: Referrals for Short Stature and Access to Specialty Drugs
Background : C and i date s for specialty drugs , the fastest growing and costliest pharmaceuticals , typically originate with primary care referrals . However , little is known about what drives such referrals — especially for large population s such as short , otherwise normal children ( idiopathic short stature ) . Recent exp and ed approval of growth hormone ( GH ) makes more than 585,000 US children eligible for such treatment , potentially costing over $ 11 billion/y . Methods : To quantify the relative impact of patient physiological indicators , physician characteristics , and consumer preferences on referrals to endocrinologists ( and potential access to GH ) for short children , a national study of 1268 r and omly selected US pediatricians was conducted , based on a full factorial experimental design in a structured survey . Results : While patient indicators ( height , growth pattern ) influenced referrals ( P < 0.001 ) , consumer drivers ( family concern ) and physician attitudes had almost as great an impact — especially for children with less severe growth impairment ( P < 0.001 ) . Physician belief that short stature impairs emotional well-being and physician characteristics ( female , older , shorter , beliefs about drug company information ) increased referrals ( P < 0.03–0.001)—independent of growth parameters . Conclusions : Referral recommendations that create the pool of c and i date s for the specialty drug GH are heavily swayed by physician characteristics and consumer preferences , particularly in the absence of compelling physiological evidence . This makes most of children with short stature strikingly susceptible to nonphysiological influences on referrals that render them c and i date s for this specialty drug . Only 1 additional referral per US pediatrician would likely increase GH costs by over $ 100 million/y
For height outcome , articles were limited to studies reporting adult height and to systematic review s. FINDINGS Recombinant human growth hormone therapy of children with idiopathic short stature increases height in some children .
The cost-benefit ratio is controversial .
AND RELEVANCE Growth hormone treatment may be considered in some children with idiopathic short stature
IMPORTANCE Approximately 2 % of children are defined as having short stature .
Deciding when to pursue recombinant human growth hormone therapy to increase adult height is controversial .
OBJECTIVE To review the management of children with idiopathic short stature , including diagnostic evaluation and therapeutic options .