Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic short stature.
Treatment: Nutropin AQ.
The insurer denied coverage for Nutropin AQ.

The denial is overturned. 

This patient is a male child diagnosed with idiopathic short stature (ISS). This request is for Nutropin. He was started on growth hormone (GH) therapy and his dose was increased from 1.9 mg [milligrams] to 2.1 mg, and was then further increased to 2.5 mg, 0.41mg/kg[kilograms]/wk[week]. His GH stimulation test peaked at 31.3. His height velocity on therapy is reported to be 7 cm[centimeters]/yr[year]. The physician reports that the patient is having progressive puberty. The patient's most recent weight is 42.2 kg. His mother's height is 156.21 cm and father's height is 165.10 cm and mid prenatal height is 167.155 cm (-1.35 SDS[standard deviation score]). His most recent height on therapy was 146 cm (-2.47 SDS). His pretreatment height was 130 cm (-2.69 SDS) which increased to 135 cm (-2.76 SDS) with height velocity over 1 year of 5 cm/yr. 

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 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 SD [standard deviations] 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 a certain 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 years. The biochemical criteria include low IGF1 [Insulin Like Growth Factor 1] or IGFBP3 [Insulin Like Growth Factor Binding Protein 3] or GH stimulation testing with a peak GH less than 10ng/ml [nanograms per milliliter]. The patient did not meet biochemical criteria. GH stimulation testing peaked at greater than 10. 

GH in the treatment of ISS is supported by randomized controlled trials and consensus guidelines. The 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 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 ISS criteria. The patient is benefiting from GH. Continuation of GH is consistent with generally accepted standards of medical practice and is therefore medically necessary.

The standard of care regarding dosing is to increase the dose to allow for a height velocity that is near the upper limit of the normal range for pubertal status. The patient's height velocity of 7 cm per year is not at goal given progressive puberty. The dose increase is consistent with generally accepted standards of medical practice and is therefore medically necessary.

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 is substantiated. The insurer's denial is overturned.