Appeal type: Medical necessity
Decision: Overturned

Diagnosis: 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 with the diagnosis of short stature, idiopathic short stature,
poor growth. He has normal IGF-1 (insulin-like growth factor 1) level. His current
height at -2.27 SDS (standard deviation score). He has a poor growth velocity of 3.8
cm (centimeters) per year. He had normal GH (growth hormone) stimulation test
peak of 12 ng/dL (nanograms per deciliter). His height prediction is 5 feet and 1/2
inches.

Yes, the requested Norditropin is medically necessary.
This patient has the diagnosis of idiopathic short stature. In the USA, for children
who meet FDA (United States Food and Drug Administration) criteria are
considered candidates for GH (growth hormone) treatment based on that studies
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 hormonedeficient short stature, defined by height SDS (standard deviation score) less than
or equal to -2.25, and associated with growth rates unlikely to permit attainment of
adult height in the normal range < (less 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 -2.25 SD (standard
deviations) meeting the diagnosis of idiopathic short stature criteria, his initial height
was at -2.27 SDS (standard deviation score) with poor growth velocity of 3.8 cm
(centimeters) per year, and a poor height prediction of 5 feet and 1/2 inch.

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 -2.2 showed that GH (growth hormone) treatment had a height gain after
1 year of growth hormone treatment.

Similarly, in Ross et al, ISS (idiopathic short stature) with baseline height standard
deviation score (HSDS)-2 were assessed over 5 years of GH (growth hormone)
treatment, had a HSDS (height standard deviation score) at year 5 of 1.6, showing
that Long-term GH (growth hormone) therapy resulted in a significant increase in
mean HSDS (height standard deviation score) and corrected HSDS (height
standard deviation score) from baseline values in ISS (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 (HSDS) of -3 to -2.25, a height velocity < (less
than) 25th percentile for their bone age, and peak stimulated GH (growth hormone)
> (greater than) 10 ng/ml (nanograms per milliliter). At 4 years, ISS (idiopathic short
stature) subjects had an average height gains of +1.3 SDS (standard deviation
score).

Another study by Sotos et al, showed that in 123 children with idiopathic short
stature height SDS (standard deviation score) of -2 treated with rhGH (recombinant
human growth hormone ) eighty eight of children (68 males and 20 females)
attained an adult height or near adult height of -0.71 SDS (standard deviation
score) (0.74 SD [standard deviations]) (95% CI [confidence interval], -0.87 to -0.55)
with a benefit over untreated controls of 9.5 cm (centimeters) (7.4 to 11.6 cm
[centimeters]) for males.

Another study by Kristrom B. et al, that included non-GHD (growth hormone
deficiency) children with height below 2 SDS (standard deviation score)
chronological age 10-15 years for boys, showed GH (growth hormone) treatment
resulted in a greater height SDS (standard deviation score) change than in
the control group.

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 (> [less than] 10 micrograms/Liter), 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 centimeters/year.

Similarly, in uncontrolled trials, growth velocity increased after 1 year, and height
SD (standard deviation) score increased from -2.72 at baseline to -2.19. In
controlled studies, the adult height of the GH (growth hormone)-treated group
significantly exceeded controls by 0.84 SD (standard deviations), and in uncontrolled trials the adult height attained after GH (growth hormone) treatment (-1.62 SD [standard deviations]) exceeded that predicted at baseline (-2.18 SDs [standard deviations]).

Thus, the literature shows ample evidence for the use of growth hormone therapy to
promote growth in children with idiopathic short stature.