Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature
Treatment: Humatrope (somatropin)
The insurer denied the Humatrope (somatropin).
The denial is overturned. 

This patient is a female diagnosed with idiopathic short stature (ISS). This request is for Humatrope. The patient has been on growth hormone (GH) therapy (Omnitrope) for almost two years. Her height velocity improved from 4.3 centimeters per year (cm/yr) to 7.5 centimeters per year (cm/yr). Her growth hormone (GH) stimulation test with arginine and clonidine peaked at 10.3. Earlier this year, her breasts and pubic hair were at tanner 3. One month prior, her breasts and pubic hair were at tanner 1. Towards the beginning of the year, her insulin-like growth factor one (IGF1) was 131 (131-495); Z-score was -1.04 standard deviations (SD); insulin-like growth factor binding protein three (IGFBP3) was 4.35 (2.27-5.91). Three months later, her insulin-like growth factor one (IGF1) was 150 (normal).

The patient's height on therapy was 137.6 centimeters (cm) (-3.02 standard deviations [SDS]).  

The patient's pretreatment height was 122.4 centimeters (cm) (-2.80 standard deviations [SDS]) and the year prior it was 124.7 centimeters (cm) (-2.91 standard deviations [SDS]) with height velocity over 8 months of 3.4 centimeters per year (cm/yr). 

The patient's mother's height is 157.48 centimeters (cm) and father's height is 175.26 centimeters (cm) and so mid parental height is 159.87 centimeters (cm) (-0.54 standard deviations [SDS]). 

Yes, the proposed Humatrope is medically necessary.

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

Classic growth hormone (GH) deficiency:
Classic growth hormone (GH) deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of GH (growth hormone) deficiency published by the GH (growth hormone) Research Society [1], the Lawson Wilkins Pediatric Endocrinology Society [2, 3], and the medical guidelines for growth hormone (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 3.0 standard deviations (SD) below the mean; 2) height more than 1.5 standard deviations (SD) below the mid parental height standard deviation (SD); 3) height more than 2.0 standard deviations (SD) below the mean and a height velocity over one year more than 1.0 standard deviations (SD) below the mean for chronological age, or a decrease in height standard deviation (SD) of more than 0.5 over one year in children over two years of age; 4) in the absence of short stature, a height velocity more than 2.0 standard deviations (SD) below the mean over one year or more than 1.5 standard deviations (SD) sustained over two years. The biochemical criteria include low insulin-like growth factor one (IGF1) or insulin-like growth factor binding protein three (IGFBP3) or growth hormone (GH) stimulation testing with a peak growth hormone (GH) less than ten nanograms per milliliter (ng/mL).

This patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at greater than ten.

Idiopathic short stature:
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]. Food and Drug Administration (FDA) approval for this indication is for "non-growth hormone-deficient short stature, defined by height standard deviation score (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."

This patient had a pretreatment height less than or equal to -2.25 standard deviations (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 reported to have an out velocity of nine centimeters per year on treatment. The patient has benefited from treatment. The request is consistent with generally accepted standards of medical practice and is therefore medically necessary.