Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Growth Hormone Deficiency
Treatment: Genotropin Injection 0.2MG (milligrams) #3/30 days
The insurer denied: Genotropin Injection 0.2MG (milligrams) 3/30 days
The denial is overturned.

The patient is an adolescent male. He is followed by his pediatric endocrinologist for short stature. His height tended to track close to the fifth to tenth percentile. However, after 4 years, this drifted to below the fifth percentile. The patient's height last year was tracing the third percentile. His bone age was read by his endocrinologist as closest to the standard, normal. The patient's workup showed low-normal insulin-like growth factor (IGF-1) levels at 90nanograms per milliliter (ng/mL) (normal range 82-299). Insulin-like growth factor-binding protein-3 levels were normal at 4.13 milligrams per liter (mg/L). Insulin-like growth factor (IGF)-1 was normal at 149 (normal 37-459). The patient showed low growth velocity at 2.9 centimeters per year (cm/year) on his succeeding follow-up; height drifted further to the second percentile. The patient underwent growth hormone (GH) stimulation testing, which he passed with peak growth hormone of 20.9 nanograms per milliliter (ng/mL). His height has drifted further to the first percentile. Predicted adult height is 63 inches, while midparental height is 67.5 inches. Growth velocity remains low at 3.1 centimeters per year. The patient is in Tanner 1 puberty. The patient's pediatric endocrinologist is recommending growth hormone therapy using Genotropin for idiopathic short stature (ISS). Coverage was denied for not meeting criteria (no 
documented central nervous system [CNS] disorder and insulin-like growth factor-1 not greater than two standard deviations below the mean). Further, his Plan-approved indications for 
treatment consist of the following: A) United States Food and Drug Administration (FDA)-approved Indications: growth hormone deficiency, Turner syndrome, Noonan syndrome, small for gestational age, Prader-Willi syndrome, chronic kidney disease, short stature homeobox-containing gene deficiency and B) compendial uses: Human immunodeficiency virus-associated wasting/cachexia; short bowel syndrome; growth failure associated with cerebral palsy, congenital adrenal hyperplasia, cystic fibrosis, and Russell-silver syndrome. An appeal letter from the patient's treating physician mentions concern that the patient may have mild growth hormone resistance or primary insulin-like growth factor deficiency, and is, therefore, recommending growth hormone therapy.

The requested health service is medically necessary.

The use of growth hormone therapy is considered medically necessary if the criteria for growth hormone therapy in idiopathic short stature are met. The United States Food and Drug 
Administration (FDA) approved the use of growth hormone in 2003 for the treatment of idiopathic short stature as defined by height standard deviation score (SDs) less than or equal to -
2.25 and associated with growth rates unlikely to permit attainment of adult height in the normal range, in pediatric patients whose epiphyses are not closed and for whom diagnostic evaluation have excluded other causes associated with short stature. 
Based on the clinical information provided, the patient's clinical characteristics did meet the criteria for treatment under the United States Food and Drug Administration (FDA)-approved indication of idiopathic short stature, as the patient's pre-treatment height is more than 2.25 standard deviations below the mean for age/sex and adult predicted height is below 63 inches. Other causes of growth failure have been excluded including growth hormone deficiency given normal growth hormone markers, insulin-like growth-1 and insulin-like growth factor-binding protein-3, as well as normal responses to growth hormone stimulation testing. Regarding the consensus guidelines for growth hormone (1), use of growth hormone is not recommended as a routine practice. Those idiopathic short stature patients with an extremely short adult height prediction could be considered physically disabled in that it may be difficult for them to navigate a world built to accommodate much taller adults. For these children, even a small increase in adult height may be a considerable benefit. While this was denied because the patient does not have any of the Plan-approved medical conditions for growth hormone treatment, this patient does meet criteria for growth hormone therapy for idiopathic short stature based on current medical literature and guidelines. Therefore, the request for Genotropin is medically necessary for this patient.