Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic Short Stature. 
Treatment: Norditropin Flexpro 15mg/1.5mL Pen, 2mg Injected Subcutaneously Once Daily for One Year, Pre-service. 

The insurer denied the Norditropin Flexpro 15mg/1.5mL Pen, 2mg Injected Subcutaneously Once Daily for One Year, Pre-service. 
The denial is upheld. 

The patient is a male diagnosed with idiopathic short stature (ISS). This request is for Norditropin. His height velocity is reported to be 6.4 centimeters per year. 

Approximately two years ago, the patient's insulin-like growth factor one (IGF1) was 374 (29-424); his Z-score was +1.5 standard deviations (SD); his insulin-like growth factor binding protein three (IGFBP3) was 5890 (1828-6592). 

His height was 129 centimeters (-1.73 standard deviations [SDS]) approximately 2 years later his height was 140.1 centimeters (-1.32 standard deviations [SDS]) with height velocity over one year nine months of 6.2 centimeters per year. 

His mother's height is 161.29 centimeters and father's height is 182.88 centimeters and mid parental height is 178.59 centimeters (+0.24 standard deviations [SDS]). 

The additional information shows GH (growth hormone) stimulation testing with arginine and clonidine with a peak GH (growth hormone) above 10 at 10.5 and 11. 

Earlier in the year, his IGF1 (Insulin-like growth factor-1) was 631; Z-score was +2.2 SD; IGFBP3 (Insulin-like growth factor binding protein 3) was 7850. 

No, the proposed treatment is not medically necessary. 

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

Classic Growth Hormone (GH) Deficiency: 

Classic growth hormone 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 3.0 standard deviations below the mean; 2) height more than 1.5 standard deviations below the mid parental height standard deviation; 3) height more than 2.0 standard deviations below the mean and a height velocity over one year more than 1.0 standard deviations below the mean for chronological age, or a decrease in height standard deviations 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 below the mean over one year or more than 1.5 standard deviations 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 factor (GH) stimulation testing with a peak growth hormone less than ten nanograms per milliliter (ng/mL). 

This patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at >10 and growth factors are normal. The patient did not meet auxological criteria as the patient's height and height velocity are normal. 

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 approval for this indication is for "non-growth hormone-deficient short stature, defined by height standard deviation (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 does not have a pretreatment height less than or equal to -2.25 standard deviations. The patient therefore does not meet idiopathic short stature criteria. 

The request does not meet any nationally accepted criteria for use of growth hormone. The request is therefore not medically necessary.