Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Short Stature 
Treatment: Pharmacy/Prescription Drugs 

The patient is seeking growth hormone (GH) treatment under the indication of idiopathic short stature (ISS). Patient was born after 39 weeks of gestation; birth weight was 6 lbs. 10 oz and birth length was 19 inches. Mother's height is 59 inches and father's height is 67 inches (mid parental height of 60.5+/-2''). Mother was late in her puberty and reached menarche at age 14 years. Father received growth hormone (GH) as a child, also late in his puberty. 

Patient was initially evaluated in the pediatric endocrinology clinic. Growth points showed that the weight had dropped from 50% tile to less than the 5% tile at while growth velocity was calculated as 3.3 inches per year. The  comprehensive evaluation was unremarkable including normal  karyotype and SHOX gene testing was negative for mutation or deletion. 

Growth was followed and height was 100.4 cm (-3.1 SD) and her weight was 15.5 kg (-2.2 SD). Evaluation was unremarkable, including a normal IGF-1 and IGFBP-3. For poor growth velocity, the patient underwent GH stimulation testing on and the peak GH response was 11 nanograms/milliliter (ng/ml) after Aginine and Clonidine. Patient was then evaluated for a second opinion. The height at that time was 106 cm (2.7 SD) and  weight was 15.36 kg (-2.3 SD). At a most recent follow up visit, the height was 113.8 (-2.5 SD) and weight was 18.2 kg (-2.5 SD). 

The health plan's determination of medical necessity is overturned. This patient is seeking GH treatment under the indication of idiopathic short stature (ISS)ISS. In 2003, the US Food and Drug Administration (FDA) approved injectable somatropin (Humatrope) for the treatment of idiopathic short stature (ISS), also called non-GH-deficient short stature, defined by a height standard deviation score (SDS) of SDS <= -2.25 and associated with growth rates unlikely to permit attainment of adult height in the normal range, in pediatric patients for whom diagnostic evaluation excludes other causes of short stature that should be observed or treated by other means. A meta-analysis of the efficacy of growth hormone treatment in children with idiopathic short stature including 10 controlled trials and 28 uncontrolled trials suggested an average gain in adult height of approximately 4-6 cm from GH treatment. This patient is quite short for age and although not from tall parents and mid parental height is only 60.5+/-2 inches, the patient is not growing well. However, weight was affected even more than height and had dropped from 50% tile to 2% tile in about 2.0 years which was consistent with nutritional growth failure. 

As this patient's height percentiles have progressively dropped over the period of years, the case of ISS can be made. Overall, this patient's height has dropped from -1.8 SD to 2.52 SD and weight from -2.0 SD to -2.5 SD in little less than 5.5 years. As this patient's height is 2.5 SD or more below the mean for the age, the patient qualifies for GH treatment under ISS. ISS is a heterogeneous condition and not all children with ISS will respond equally to GH treatment. The Pediatric Endocrine Society Guidelines for Growth Hormone and Insulin-Like Growth Factor-1 Treatment in Children and Adolescents recommend GH treatment for ISS on a case-by-case basis after an assessment of physical and psychological burdens, and discussion of risks and benefits.