Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Hypopituitarism
Treatment: Skytrofa
The insurer denied Skytrofa.
The denial is upheld.

The patient is a teenage male. He is followed by his pediatric endocrinologist for short stature. Growth chart shows that his height was tracking close to the 25th-50th percentile between 8.5 and 11.5 years old. The patient's height 2 years ago was 153.69 centimeters (cm); and last year his height was 162.5 centimeters (54th percentile). Midparental height was 67.5 inches. Bone age was read as closest to the 15-15.5-year standard. Predicted adult height was 65-65.5 inches. Insulin-like growth factor-1 (IGF-1) was normal at 383, as well as insulin-like growth factor binding protein-3 (IGFBP-3) at 5.3. The patient did undergo growth hormone (GH) stimulation testing last year and had a peak growth hormone of 10 after clonidine and arginine, by report. Pituitary gland was normal on magnetic resonance imaging (MRI). The patient was started on growth hormone therapy and was now paying out of pocket. The patient's endocrinologist would like to continue with growth hormone therapy. Coverage was denied for not meeting criteria. Appeal letter from the patient's treating physician states that patient has been doing well on Skytrofa (lonapegsomatropin-tcgd).

The requested health service is not medically necessary.

The Consensus Guidelines for the Diagnosis and Treatment of Growth Hormone Deficiency (GHD) in Childhood and Adolescence by The GH (Growth Hormone) Research Society state diagnosing growth hormone deficiency requires a multifaceted process including comprehensive clinical and auxological assessment, combined with biochemical tests of the growth hormone-insulin-like growth factor (IGF) axis and sometimes radiological evaluation which includes:
1. Clinical and Auxological Criteria: Criteria to initiate immediate investigation include: 
1) severe short stature, defined as a height more than three standard deviation (SD) below the mean; 
2) height more than 1.5 standard deviations below the mid-parental height; 
3) height more than two standard deviations below the mean and a height velocity over 1 year more than 1 standard deviation below the mean for chronological age, or a decrease in height standard deviation 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 standard deviations below the mean over 1 year or more than 1.5 
standard deviations sustained over 2 years; this may occur in growth hormone deficiency (GHD), presenting in infancy, or in organic acquired growth hormone deficiency; 
5) signs indicative of an intracranial lesion; 
6) signs of multiple pituitary hormone deficiency (MPHD); and 
7) neonatal symptoms and signs of growth hormone deficiency (GHD). 
2. Biochemical Assessment of growth hormone deficiency: A combination of two provocative growth hormone (GH) testing can be used to test for growth hormone deficiency and failing both tests has traditionally been used to support the diagnosis. An insulin-like growth factor one value less than -2 standard deviations suggests an abnormality in the growth hormone axis. 
3. Radiological evaluation showing evidence of delayed bone age or abnormal pituitary gland. 
This patient was diagnosed with growth hormone deficiency (GHD). Based on medical records submitted, the patient did not meet many of the key criteria for the diagnosis of an isolated 
growth hormone deficiency including the above clinical, some biochemical and radiological criteria. This patient has no significant short stature and there is no clear demonstration of 
persistent linear growth deceleration to suspect growth hormone deficiency. Serum insulin-like growth factor one level (a good surrogate marker of growth hormone status) was noted to be normal despite an attenuated peak growth hormone response to provocative tests. Growth hormone provocative test is not recommended as the sole diagnostic criterion of growth hormone deficiency. Growth hormone stimulation testing has a number of limitations: the tests are non-physiologic; the cutoff level of "normal" is arbitrary; there is marked variability in growth hormone assays; and there is lack of reproducibility of growth hormone stimulation tests. Brain magnetic resonance image (MRI) was normal. These findings are not consistent with growth hormone deficiency per the above Consensus Guidelines for the Diagnosis and Treatment of Growth Hormone Deficiency in Childhood and Adolescence by The GH Research Society. Therefore, the drug is not being requested for a medically accepted indication as supported by current medical literature and any applicable diagnostic/treatment guidelines.