Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature.
Treatment: Nutropin AQ Injection NuSpin 5.
The insurer denied: Nutropin AQ Injection NuSpin 5.
The denial is overturned.

The patient is a pre-adolescent girl followed by her pediatric endocrinologist for short stature. Her height used to track close to the 25-50th percentile several years ago. However after that, she had growth deceleration such that her height is now close to the second percentile. Her recent growth velocity is 3.5 centimeters/year, which is - (minus) 2.5 SDS (standard deviation score).

The patient's mid-parental height is close to the 25th percentile. The patient's screening laboratory testing came back normal, including IGF-1 (insulin-like growth factor 1) of 118 (normal 49-451) and IGFBP-3 (insulin-like growth factor binding protein 3) of 4880 (normal 2072-5504). She also passed her growth hormone (GH) stimulation testing with peak GH (growth hormone) of 19.7. Of note, her maternal uncle has history of neurosecretory growth hormone dysfunction, treated with GH (growth hormone). A distant cousin received GH (growth hormone) treatment as well.

The patient's pre-treatment height is 124 centimeters (Z=- [minus] 2.13), while growth velocity Z-score is - [minus] 1.61.

The patient's endocrinologist would like to initiate GH (growth hormone) treatment. Coverage was denied coverage by the health plan for not meeting criteria. Based on her Plan's coverage guidelines for GH (growth hormone) treatment, patient must have one of the following conditions to quality:
1) GH (growth hormone) deficiency
2) Prader-Willi syndrome
3) Growth failure in children small for gestational age
4) Turner syndrome
5) Noonan syndrome
6) SHOX (short stature homeobox-containing gene) gene deficiency
7) Growth failure associated with chronic renal insufficiency

An appeal letter from the patient's endocrinologist notes that the patient meets criteria for GH (growth hormone) therapy under the idiopathic short stature (ISS) indication as approved by the Food and Drug Administration (FDA).

The requested Nutropin is medically necessary.
Although patient did not meet her Plan's specific coverage guidelines for GH (growth hormone) treatment, it is medically necessary in this instance. The patient's height is currently tracking significantly less than her mid-parental height percentile. Further, she does have poor growth velocity, which will not provide catch-up growth to help her reach her mid-parental height.

The use of growth hormone therapy is considered medically necessary if the criteria for idiopathic short stature is met. The Food and Drug Administration (FDA) approved the use of growth hormone for the treatment of idiopathic short stature as defined by height standard deviation score (SDS) less than or equal to - (minus) 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 diagnostic evaluation have excluded other causes associated with short stature, and whose expected adult height is less than 63 inches (160 centimeters) for boys and 59 inches (150 centimeters) for girls.

The patient's clinical characteristics did meet the criteria for idiopathic short stature. Although her current height is not more than 2.25 SD (standard deviations) below the mean for age/sex, her height is close to this, and with her current poor growth velocity, it will be a matter of time before she will reach this cutoff. Further, with her current growth velocity, it is likely that her final height will fall below 59 inches without treatment.

This patient does meet criteria for growth hormone therapy based on current medical literature and guidelines. Therefore, the request for Nutropin is medically necessary for this patient.

The recommended duration of treatment is 12 months.