Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature
Treatment: Nutropin AQ
The insurer denied the Nutropin AQ
The denial is overturned

The patient is a male with the diagnosis of non growth hormone deficiency short stature based on a height at -2.45 normal bone age, growth velocity of 2.64 cm/yr or - (minus)5.4 SDS (standard deviation score), normal growth hormone stimulation test of  11.3 ng/dL with an IGF-1 (insulin-like growth factor 1) of - (minus) 1.1 SDS (standard deviation score), patient has a height prediction of 159.70 or 62.8 inches.

Yes, the proposed treatment is medically necessary.
This patient has the diagnosis of idiopathic short stature. In the United States of America, for children who meet FDA (Food and Drug Administration) criteria are considered candidates for GH (growth hormone) treatment based on that studies that have shown GH (growth hormone) treatment increases the mean height of treated cohorts. Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines. Food and Drug Administration (FDA) approval for this indication is for "non-growth hormone-deficient short stature, defined by height SDS (standard deviation score) less than or equal to - (minus) 2.25, and associated with growth rates unlikely to permit attainment of adult height in the normal range < (greater than) 63 inches in boys, 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 did have a pretreatment height less than or equal to - (minus) 2.25 SD (standard deviation) meeting the diagnosis of idiopathic short stature criteria, his initial height was at - (minus) 2.64 SDS (standard deviation score) with an extreme growth velocity and a height prediction that is less than 63 inches. These values were also suggested in a consensus statement from the Growth Hormone Research Society, PES, and the European Society for Pediatric Endocrinology (ESPE).  Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines. 

There is enough data that suggest that patient with this characteristics are good candidates for treatment. A recent study by Seul Ah Kim et al. 12 patients were in the ISS (idiopathic short stature group) who had a height SDS (standard deviation score) of  - (minus) 2.2 showed that GH (growth hormone) treatment had a height gain  after 1 year of growth hormone treatment of  7.68 (plus or minus) 1.44 cm. Similarly, in Ross et al, idiopathic short stature (n=733) with baseline height standard deviation score (HSDS)-2 were assessed over 5 years of growth hormone treatment, had a  HSDS (height standard deviation score) at year 5 of 1.6, showing that Long-term growth hormone therapy resulted in a significant increase in mean height standard deviation score and corrected height standard deviation score from baseline values in idiopathic short stature.
Another study by Counts et al, showed that in a  4-year, open-label, multicenter, randomized trial in prepubertal, had a bone age of 3-10 years for males and a  height standard deviation score (Ht SDS) of - (minus) 3 to - (minus) 2.25, a height velocity < (greater than) 25th percentile for their bone age, and peak stimulated GH (growth hormone) > (greater than) 10 ng/ml. At 4 years, idiopathic short stature subjects had an average height gains of + (plus) 1.3 SDS (standard deviation score). 
Another study by Sotos et al, showed that in 123 children with idiopathic short stature height standard deviation score of - (minus) 2 treated with rhGH (human growth hormone) eighty eight of children (68 males and 20 females) attained an adult height or near adult height of - (minus) 0.71 standard deviation score (0.74 standard deviation) with a benefit over untreated controls of 9.5 cm (7.4 to 11.6 cm) for males. 
Another by study Kristrom B. et al,  that included non-growth hormone deficiency children with height below 2 standard deviation score chronological age 10-15 years for boys, showed growth hormone treatment resulted in greater height standard deviation score)change than in the control group (1.1  [plus or minus] 0.84 vs 0.4  [plus or minus] 0.85).  
In a meta-analysis by Finkelstein et al, which included patient with idiopathic short stature: initial height below the 10th percentile, normal stimulated GH (growth hormone) levels (> [greater than] 10 microg/L), absence of comorbid conditions showed that 1-year growth velocity of the GH (growth hormone)-treated group significantly exceeded that of controls by 2.86 cm/y. Similarly, in uncontrolled trials, growth velocity increased after 1 year, and height SD (standard deviation) score increased from - (minus) 2.72 at baseline to - (minus) 2.19. In controlled studies, the adult height of the GH (growth hormone) -treated group significantly exceeded controls by 0.84 SD (standard deviation), and in uncontrolled trials the adult height attained after GH (growth hormone) treatment (- [minus] 1.62 standard deviations) exceeded that predicted at baseline (- [minus] 2.18 (standard deviations). Thus, the literature shows that the use of growth hormone therapy to promote growth in children with idiopathic short stature.

No the health plan did not acted reasonably, with sound medical judgment and in the best interest of the patient.