Appeal type: Experimental/Investigational
Decision: Overturned

Physician 1: The patient is a nine-year-old female with a long history of growth delay. She has been diagnosed with Noonans syndrome. She has been treated with growth hormone therapy since September 2003 with marked improvement in her growth, and her physician has recommended further treatment with growth hormone therapy. The Health Plan has denied authorization and coverage for growth hormone therapy on the basis it is considered experimental for treatment of the patients medical condition. Most academic centers in the United States use growth hormone for treatment of patients with Noonans syndrome. In the case of this patient, there is potential for ultimate short stature. The patient has had a very positive response to initial growth hormone therapy. Use of growth hormone for treatment of patients such as this patient is standard of care in the national community. Therefore, I have determined that the requested therapy is likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be overturned. Physician 2: The patient is a nine-year-old female with short stature. The patient had normal birth weight and length, but had poor growth during the initial year of life onwards. She had constipation problems over the initial two to three years of life. Initially, her appearance and poor growth led to suspicions she had Williamss syndrome and a chromosome test undertaken was normal. The patient appears to have had delayed growth through the age of two years to present, but at a relatively normal rate and with some bone age retardation. Her bone age was 5 to 5.5 years at age 9 years. Somatomedin-C and IGFBP-3 levels performed years before were normal. It does not appear that a growth hormone (GH) workup was performed. A geneticist evaluation argued the case for a diagnosis of Noonans syndrome supported by the presence of a persistent cardiac murmur. The patient was given a trial of GH therapy at 0.3mg/kg/week and apparently grew one inch in the first month. The response after that is not known. At an insurance change, GH was discontinued. The patient has been seen by a pediatric endocrinologist who recommended GH therapy. The Health Plan has denied coverage for GH therapy on the basis it is considered investigational for the patients diagnosis. Review of the submitted clinical information demonstrates this is not an SGA (small for gestational age) related growth failure. The patients growth fell by the first year of life and she has not caught up since. Her considerably retarded bone age suggests that she may have a reasonable adult height prediction, assuming that her rate of growth is not abnormally low. The diagnosis of Noonans syndrome is not firm, but appears plausible. Unless there is documentation that her present rate of growth is abnormally slow, growth hormone therapy is not medically indicated at this time.  The situation should be revisited at onset of puberty when a more accurate adult height prediction can be made. Therefore, I have determined that the requested therapy is not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be upheld. Physician 3: The patient is a nine-year-old female with Noonans syndrome. Her parents have requested authorization and coverage for growth hormone (GH) therapy to treat the patients short stature and growth retardation associated with this disorder. The patient received GH therapy in September 2003 and experienced excellent results with growth acceleration. The Health Plan has denied coverage for GH therapy. This patient was treated successfully in September 2003 with GH resulting in rapid growth acceleration. She gained one inch in height during the first month of GH therapy. Review of the submitted literature reveals there is evidence of the efficacy of growth hormone for treatment of patients with Noonans syndrome. Though Noonans syndrome is not specifically listed as an indication for GH therapy by the Health Plan, the Health Plan indicates GH therapy is indicated for treatment of Turners syndrome, which is substantially similar to Noonans syndrome.  Furthermore, in the case of this patient it is already known that she responds well to GH therapy. There is no evidence that any alternative therapy will be of benefit. Based upon the information set forth above, I have determined that the requested therapy is likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. Therefore, the Health Plans denial should be overturned.