Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic short stature

Treatment: Omnitrope

The insurer denied coverage for Omnitrope

The denial is upheld.

This is a teenager diagnosed with idiopathic short stature (ISS). This request is for Omnitrope. As per the letter, the patient was started on Anastrozole to delay bone maturation and growth hormone (GH) therapy almost 3 years ago. The patient's height velocity is reported to have improved from 5 centimeters (cm)/year (y) to 13.7 cm/y. The patient's GH stimulation test with arginine and glucagon peaked at 16. The patient has continued to take GH, currently Nutropin 1.4 milligrams (mg) daily. The patient's testes were 15-20 milliliters (ml). The patient's insulin-like growth factor-1 (IGF1) was 172; insulin-like growth factor-binding protein 3 (IGFBP3) was 4.54. The patient's magnetic resonance imaging (MRI) was normal. 

The patient's bone age was 13y8 months (m) at calculated age (CA) 14y9m. The patient's weight was 53.3 kilograms (kg). The patient's pretreatment height was 142.8 cm (-1.66 standard deviations [SDS]). The patient's height on therapy at 14.78 years old (y/o) was 164.8 cm (-0.56 SDS). The patient's mid parental height is 172.9 cm (-0.55 SDS).

Classic GH deficiency

Classic GH deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of GH deficiency published by the GH research society, the Lawson Wilkins Pediatric Endocrinology Society, and the medical guidelines for GH use provided by the American Association of Clinical Endocrinologists. The above referenced guidelines are the latest available guidelines from these respective societies. The diagnosis is based on height, height velocity, biochemical and sometimes radiological findings. The height and height velocity criteria based on consensus guidelines upon which a biochemical evaluation is to be initiated are 1) severe short stature, defined as a height more than 3 standard deviations (SD) below the mean; 2) height more than 1.5 SD below the mid parental height SD; 3) height more than 2 SD below the mean and a height velocity over 1 y more than 1 SD below the mean for chronological age, or a decrease in height SD of more than 0.5 over 1 y in children over 2 y of age; 4) in the absence of short stature, a height velocity more than 2 SD below the mean over 1 y or more than 1.5 SD sustained over 2 y. The biochemical criteria include low IGF1 or IGFBP3 or GH stimulation testing with a peak GH less than 10.

The patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at greater than 10. The patient's GH stimulation test with arginine and glucagon peaked at 16. Growth factors are normal.

The patient does not meet growth hormone deficiency criteria.

Idiopathic Short Stature

Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines. Food and Drug Administration (FDA) approval for this indication is for "non-growth hormone-deficient short stature, defined by height 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 excludes other causes associated with short stature that should be observed or treated by other means."

The patient does not have a pretreatment height less than or equal to -2.25 SD. The patient's pretreatment height was 142.8 cm (-1.66 SDS).

The patient therefore does not meet idiopathic short stature criteria. 

The request does not meet any nationally accepted criteria for use of growth hormone. The request is therefore not medically necessary.

Based on the above, the insurer's denial must be upheld. The health care plan did act reasonably and with sound medical judgment and in the best interest of the patient. 

The medical necessity for Omnitrope is not substantiated.