Appeal type: Medical necessity
Decision: Upheld

Diagnosis:  Short Stature.
Treatment:  Genotropin.

The insurer denied coverage for Genotropin.  The denial is upheld.

This is a teenage patient diagnosed with idiopathic short stature (ISS). This request is for Genotropin. As per the letter, the patient also has a history of attention deficit disorder (ADD) for which they have been treated.
 
The patient's growth hormone (GH) stimulation test with arginine and glucagon peaked at 12.

The patient's bone age was 10 years (y) at calculated age (CA) 11y1 month (m) and 12y6m at CA 12y2m. The patient's height velocity is reported to be 4 centimeters (cm)/year. The patient's insulin-like growth factor 1 (IGF1) was 171 (82-299 range, mean 170). Subsequently, the patient's insulin-like growth factor-binding protein 3 (IGFBP3) was 3.45; IGF1 was 168. The patient's testes and pubic hair was at tanner 1. 

The patient's height at 11.67 y was 137.8 centimeters (cm) (-1.32 standard deviations [SDS]) and at 12.2 y was 140 cm (-1.41 SDS) with height velocity over 0.67 y of 4.2 cm/y. The patient's predicted adult height 162.7 cm (-1.97 SDS).  The patient's mid parental height is 172.9 cm (-0.55 SDS).

The peer reviewed literature including consensus clinical guidelines, randomized controlled trials are summarized below:

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(1), the Lawson Wilkins Pediatric Endocrinology Society(2,3), and the medical guidelines for GH use provided by the American Association of Clinical Endocrinologists (4). 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 yr. The biochemical criteria include low IGF1 or IGFBP3 or GH stimulation testing with a peak GH less than 10 nanograms (ng)/milliliters (ml).

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 12. 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 (4-9). 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" (10-13).

The patient does not have a pretreatment height less than or equal to -2.25 SD. 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 Genotropin is not substantiated.