Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Short stature

Treatment: Nutropin AQ

The insurer denied coverage for Nutropin AQ

The denial is overturned
 

This is a child who has been diagnosed with idiopathic short stature (ISS). This request is for Nutropin AQ. As reported; the child has been doing well on growth hormone (GH) therapy. The GH stimulation test with arginine and glucagon peaked at 11. The height velocity is reported to be improved from 3.6 to 7.57 centimeters per year (cm/yr. The Insulin-like growth factor binding protein 3 (IGFBP3) was 3.58. The insulin-like growth factor-1 (IGF1) was 40 (39-198) 

The pretreatment height was 93.7 cm (-3.65 SDS).
The height on therapy was 116.2 cm (-2.21 SDS).
The mid parental height is 144.6 cm (-2.88 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 yr more than 1 SD below the mean for chronological age, or a decrease in height SD of more than 0.5 over 1 yr in children over 2 yr of age; 4) in the absence of short stature, a height velocity more than 2 SD below the mean over 1 yr 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 per milliliter (ng/ml).
		
The patient did not meet biochemical criteria. 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 [4-9]. 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 standard deviation (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 patient is a continuing therapy patient who has benefited from growth hormone. The height standard deviation has improved on growth hormone. Continuation is consistent with accepted standards of medical practice and is therefore medically necessary.

The carrier's denial of a request for a Formulary Exception for Nutropin AQ to treat idiopathic short stature (ISS) is reversed.