Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature.		

Treatment: Nutropin AQ NuSpin 20 20 milligrams (MG)/2 milliliters (ML) subcutaneous (SC) solution pen-injector (SOPN).

The insurer denied coverage for Nutropin AQ NuSpin 20 20 milligrams (MG)/2 milliliters (ML) subcutaneous (SC) solution pen-injector (SOPN).

The denial is overturned.

The patient is diagnosed with idiopathic short stature (ISS). This request is for Nutropin. As reported, the patient started growth hormones (GH). As per the visit note, the patient is on Nutropin 1.4 milligrams (mg) nightly. The patient's GH stimulation test with arginine and clonidine peaked at 10.1.  The patient's insulin-like growth factor 1 (IGF1) was 117 (80-398); insulin-like growth factor-binding protein 3 (IGFBP3) was 5670 (1932-5858).  The patient's bone age was 8 years (y) at a calculated age (CA) of 9y3 months (m).

The patient's weight was 28.1 kilograms (kg).  The patient's pretreatment height at 9.67 years old was 121.5 centimeters (cm) (-2.50 standard deviations [SDS]).  The patient's height on therapy at 10.198 years old was 124.5 cm (-2.33 SDS). The patient's predicted adult height is 157.48 cm (-2.69 SDS).  The patient's mid-parental height is 165.1 cm (-1.64 SDS).

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 year more than 1 SD below the mean for chronological age, or a decrease in height SD 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 SD below the mean over 1 year 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 of 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 clonidine peaked at 10.1. Growth factors are normal.

The patient does not meet growth hormone deficiency criteria.  

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 had a pretreatment height less than or equal to -2.25 SD. The patient will not reach a normal adult height. The patient's adult predicted height is 157.48 cm (-2.69 SDS).

The request therefore meets idiopathic short stature criteria.

Based on the above, the insurer's denial must be reversed. The health care plan did not act reasonably and with sound medical judgment and in the best interest of the patient. 

The medical necessity for Nutropin AQ NuSpin 20 20 milligrams (MG)/2 milliliters (ML) subcutaneous (SC) solution pen-injector (SOPN) is substantiated.