The current and emerging landscape of pediatric growth hormone deficiency (GHD) pharmacotherapy

Activity Description / Statement of Need:

Growth hormone deficiency (GHD) is characterized by inadequate secretion of growth hormone by the pituitary gland. The condition may arise from a variety of causes, including tumors, radiation, medications, traumatic brain injury, or genetic defects. In children, GHD is characterized by pronounced short stature, defined as 2 or more standard deviations from the mean based on age and sex. Because short stature may be caused by a variety of other factors, including genetics, hypothyroidism, and Turner syndrome, estimating the prevalence of GYHD in the pediatric population is challenging. Studies suggest that GHD may occur in 1 out of every 4,000 to 10,000 children. There is no gold standard for diagnosis of GHD in children. Clinical practice guidelines from the Pediatric Endocrine Society currently caution against the use of growth hormone provocation testing in the diagnosis of GHD given the unreliability of results. Auxology, biochemical evaluation, imaging, and physical examination remain the cornerstones of GHD diagnosis in children. Clinicians should be aware of appropriate strategies of diagnosis of GHD in pediatric patients, as improper diagnosis may delay effective treatment and increase risk for secondary complications.

Target Audience:

The following healthcare professionals: Endocrinologists, pediatricians, and primary care physicians; physician assistants, nurse practitioners, and pharmacists who practice in endocrinology; and any other healthcare professionals with an interest in or who clinically encounter patients with GHD.

Cost: Free

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Details

Activity Description / Statement of Need:

Growth hormone deficiency (GHD) is characterized by inadequate secretion of growth hormone by the pituitary gland. The condition may arise from a variety of causes, including tumors, radiation, medications, traumatic brain injury, or genetic defects. In children, GHD is characterized by pronounced short stature, defined as 2 or more standard deviations from the mean based on age and sex. Because short stature may be caused by a variety of other factors, including genetics, hypothyroidism, and Turner syndrome, estimating the prevalence of GYHD in the pediatric population is challenging. Studies suggest that GHD may occur in 1 out of every 4,000 to 10,000 children. There is no gold standard for diagnosis of GHD in children. Clinical practice guidelines from the Pediatric Endocrine Society currently caution against the use of growth hormone provocation testing in the diagnosis of GHD given the unreliability of results. Auxology, biochemical evaluation, imaging, and physical examination remain the cornerstones of GHD diagnosis in children. Clinicians should be aware of appropriate strategies of diagnosis of GHD in pediatric patients, as improper diagnosis may delay effective treatment and increase risk for secondary complications.

Target Audience:

The following healthcare professionals: Endocrinologists, pediatricians, and primary care physicians; physician assistants, nurse practitioners, and pharmacists who practice in endocrinology; and any other healthcare professionals with an interest in or who clinically encounter patients with GHD.

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