Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature
Treatment Nutropin AQ
The insurer denied Nutropin AQ
The determination is overturned.

This is a patient with the diagnosis of idiopathic short stature (ISS) based on a height at the 1%ile [percentile] for age, growth deceleration dropping percentiles from the 3 to 5%ile, low insulin like growth factor-1 (IGF-1), normal growth hormone stimulation test with a peak above 10 nanograms per deciliter (ng/dl), small pituitary on magnetic resonance imaging (MRI) scan and delayed bone age, giving him a height prediction 64 to 66.6 inches compared to his midparental target height of 65.75 inches. At issue is the medical necessity of Nutropin AQ.

Yes, the Nutropin is medically necessary for this patient.
Clinical and auxological criteria: The evaluation for growth hormone deficiency (GHD) in a short child, where short stature is defined as a height more than 2 standard deviation (SD) below the population mean, should not be initiated until other causes of growth failure, such as hypothyroidism, chronic systemic disease, Turner syndrome, or skeletal disorder, have been considered and appropriately excluded. It is recognized that short stature is often the only feature present. (Brod, M., Alolga, S. L., & Beck, J. F., et al., 2017). He does not have the diagnosis of growth hormone deficiency (GHD), Prader Willi, growth failure in small for gestational age (SGA), Turner syndrome or Noonan Syndrome, SHOX deletion or growth failure associated with chronic renal insufficiency.
Criteria to initiate immediate investigation include: 1) severe short stature, defined as a height more than 3 SD below the mean; 2) height more than 1.5 standard deviation (SD) below the mid-parental height; 3) height more than 2 SD below the mean and a height velocity over 1 year more than 1 SD below the mean for chronological age, or a decrease in height SD of more than 0.5 over 1 year in children over 2 years of age; 4) in the absence of short stature, a height velocity more than 2 SD below the mean over 1 year or more than 1.5 SD sustained over 2 years; this may occur in GHD, presenting in infancy, or in organic acquired GHD; 5) signs indicative of an intracranial lesion; 6) signs of multiple pituitary hormone deficiency (MPHD); and 7) neonatal symptoms and signs of GHD... Biochemical assessment of GHD: A combination of 2 Provocative GH testing can be used to test for GHD and failing both tests has traditionally been used to support the diagnosis. IGF-1 value less than -2 SD suggest an abnormality in the GH axis... Radiological evaluation: An evidence of delayed bone age and presence of structural or developmental anomaly in the pituitary gland. (GH Research Society, 2000).
This patient did not fail 2 Provocative GH tests but has a low IGF-1. However, provocative GH testing has a number of limitations. The tests are non-physiological, the cutoff level of "normal" is arbitrary, and there is marked variability in GH assay and a lack of reproducibility of GH stimulation tests. Thus, having a low IGF-1 in addition to poor growth (dropping percentiles) could be considered features of GH deficiency
The patient's height is greater than (>) 2 standard deviations. Furthermore, the patient's height is more than 2 SD below the mid-parental height, and there is also a history of prior suboptimal linear growth- that would raise the concern for GHD. This patient's recent bone age was delayed with a small pituitary gland. The current medical literature does support the use of GH in this patient. Therefore, the request for GH is medically necessary.