Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature (ISS)
Treatment: Nutropin AQ Inj 10 milligrams/2 milliliters
The insurer denied Nutropin AQ Inj 10 milligrams/2 milliliters. 
The denial was reversed.

This is a young female patient diagnosed with Idiopathic Short Stature (ISS). A request for Nutropin was made. The patient's growth hormone (GH) stimulation test in late 2019 with arginine and clonidine peaked at 13.6 and 13, respectively. The patient's height velocity was reported to be 3.9 cm/year. The patient's bone age was appropriate for her chronological age. The patient's breasts and pubic hair were at tanner 1 stage, her IGF1 was 219 and her height was 125.1 cm (-2.23 standard deviations (SDS)) and in early 2020 it was 126.4 cm (-2.26 SDS) with height velocity over 4 months of 3.9 cm/year. The patient's mother's height was 175.26 cm and father's height was 167.64 cm; thus, the mid parental height was 164.94 cm (0.25 SDS). The patient's predicted adult height based on the most recent bone age was 145.1 cm, 57.1 inches (-2.8 SD). 

Classic growth hormone (GH) deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of GH deficiency published by the GH research society1, 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 auxological, biochemical and sometimes radiological findings.  The auxological 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 SDS below the mean; 2) height more than 1.5 SDS below the mid parental height; 3) height more than 2 SDS below the mean and a height velocity over 1 year more than 1 SDS below the mean for chronological age, or a decrease in height SDS of more than 0.5 over 1 year in children over 2 years of age; 4) in the absence of short stature, a height velocity more than 2 SDS below the mean over 1 year or more than 1.5 SDS sustained over 2 years. The biochemical criteria include low IGF1 or IGFBP3 or GH stimulation testing with a peak GH less than 10ng/ml. In this case, the patient did not meet biochemical criteria for GH deficiency.  Growth hormone stimulation testing peaked at greater than 10. 

Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines.4,5,6,7,8,9 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 had a pretreatment height less than or equal to -2.25 SD.  The patient's height velocity and bone age would not allow the patient to reach a normal adult height.  The request therefore meets idiopathic short stature criteria.  The request is consistent with generally accepted standards of medical practice and is therefore medically necessary.

The health plan did not act reasonably with sound medical judgment, and in the best interest of the patient.

The carrier's denial of coverage for Nutropin AQ Inj 10 milligrams/2 milliliters should be reversed.  The medical necessity is substantiated.