Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature. 
Treatment: Norditropin FlexPro 15MG/1.5. 

The insurer denied the Norditropin FlexPro 15MG/1.5. 
The denial is overturned. 

The patient is a male diagnosed with short stature. This request is for Norditropin. He started GH (growth hormone) therapy in the spring (Norditropin). His GH (growth hormone) stimulation test with arginine and L-dopa (levodopa) several years ago peaked at that time. 

His weight when starting GH (growth hormone) therapy was 122 lbs (pounds). His IGF-I (insulin-like growth factor 1) at the beginning of the year was 210 (148 - 551). IGFBP-3 (insulin-like growth factor binding protein 3) was normal for age. 

The patient's height on therapy in the summer was 152.10 cm (-1.90 SDS [standard deviation score]). His pretreatment height approximately two years ago 143.51 cm (centimeters) (-1.50 SDS [standard deviation score]) and toward the beginning of the year was 148.894 cm (centimeters) (-2.03 SDS [standard deviation score]), with height velocity over one year five months of 3.8 cm (centimeters)/year.

The patient's mother's height is 157.48 cm (centimeters), and his father's height is 185.42 cm (centimeters). His mid-parental height is 177.95 cm (centimeters) (+0.15 SDS [standard deviation score]). 

Yes, the treatment with Norditropin FlexPro is medically necessary. 

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

Classic GH (growth hormone) deficiency- 
Classic GH (growth hormone) 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 GH (growth hormone) 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 SD (standard deviations) below the mean; 
2) Height more than 1.5 SD (standard deviations) below the mid parental height SD (standard deviations); 
3) Height more than 2 SD (standard deviations) below the mean and a height velocity over one year more than 1 SD (standard deviations) below the mean for chronological age, or a decrease in height SD (standard deviation) 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 SD (standard deviations) below the mean over one year or more than 1.5 SD (standard deviations) sustained over two years. The biochemical criteria include low IGF1 (insulin-like growth factor 1) or IGFBP3 (insulin-like growth factor binding protein 3) or GH (growth hormone) stimulation testing with a peak GH (growth hormone) less than 10 ng (nanogram)/ml (milliliter). 
		

The patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at greater than 10. 

Alternate GH (growth hormone) deficiency criteria- 
In addition, GH (growth hormone) is indicated for the treatment of GH (growth hormone) deficiency in certain patients who do not fail GH (growth hormone) stimulation tests based on the Lawson Wilkins Pediatric Endocrine Society (LWPES) alternate criteria [2] which state, "No gold standard exists for the diagnosis of GHD [growth hormone deficiency]. Although children severely affected by GHD [growth hormone deficiency] fail GH [growth hormone] stimulation tests, there is no doubt that some children with GHD [growth hormone deficiency] achieve stimulated GH [growth hormone] concentrations above the arbitrary cutoffs that have been applied. A trial of GH [growth hormone] therapy should be approved for children with otherwise unexplained short stature who pass GH [growth hormone] stimulation tests, but who meet most of the following criteria: (1) height greater than 2.25 SD [standard deviations] below the mean for age or greater than 2 SD [standard deviations] below the mid parental height percentile; (2) growth velocity less than 25th percentile for bone age;(3) bone age greater than 2 SD [standard deviations] below the mean for age; (4) low serum insulin-like growth factor 1 (IGF-I) and/or insulin-like growth factor binding protein 3 (IGFBP3); and/or (5) other clinical features suggestive of GHD [growth hormone deficiency]." 

This patient met criterion 1 as pretreatment height was more than 2 SD (standard deviations) below midparental target height SD (standard deviation). Height velocity is less than the 25th percentile. The patient had an excellent response to growth hormone. This is a clinical feature suggestive of growth hormone deficiency. The patient therefore met three of the above criteria. The patient is expected to benefit from growth hormone and has benefited from growth hormone so far. The request is therefore medically necessary.