Hypochondroplasia

CARMEL, CALIF. -- To date, the Food and Drug Administration has approved the use of growth hormone only for the indication of growth hormone deficiency Turner's syndrome, and chronic renal failure.

But a 4-year review of data from the Genentech National Cooperative Growth Study suggests that growth hormone is also effective in treating short stature associated with precocious puberty hypothyroidism, Down syndrome, and 15 other genetic syndromes, Dr. Caroline Buckway said at the annual Western regional meeting of the American Federation for Medical Research.

Although the findings are too preliminary to advocate treatment with growth hormone for these conditions, the potential clinical impact is substantial, said Dr. Buckway, a fellow in pediatric endocrinology at Oregon Health Sciences University in Portland.

"These other conditions are worthy of consideration," she said. Prospective, randomized studies are necessary to confirm these observations.

The Genentech National Cooperative Growth Study is a large surveillance database of children who have been treated with growth hormone both on and off label. More than 31,000 patients were enrolled in the registry at the time of the study.

Dr. Buckway and Dr. Ron Rosenfeld, who is also of the university, analyzed the growth data from patients who were pre-pubertal at the initiation of their treatment and followed these patients for up for 4 years.

They measured the effect of growth hormone on FDA-approved indications as well its effect on 18 other conditions. (See box for complete list of conditions treated.) Every condition showed a growth rate in year 1 of greater than or equal to 6.8 cm, they said.

This was similar to the average growth rate in year 1 of 7.7 cm seen in patients with Turner's syndrome.

All conditions except achondroplasia (4.9 cm) had a mean growth rate in year 2 of greater than 6 cm.

In year 3, the mean growth velocity was greater than 5 cm in all conditions except achondroplasia (4 cm) and cystic fibrosis (4.4 cm).

In year 4, the growth rate exceeded 5 cm in all conditions except Turner's syndrome (4.9 cm), achondroplasia, (4.5 cm), cystic fibrosis (4.5 cm), and thalassemia (3.3 cm). There was a net gain in height standard deviation scores for all conditions over 4 years.

The data used in the analysis were provided by Genentech.

Conditions Treated

These 18 conditions were treated with growth hormone in the Genentech National Cooperative Growth Study:

* Achondroplasia

* Congenital adrenal hyperplasia

* Crohn's disease

* Cystic fibrosis

* Down syndrome

* Familial hypophosphatemic rickets

* Fetal alcohol syndrome

* Hypochondroplasia

* Hypothyroidism

* Intrauterine growth retardation

* Juvenile rheumatoid arthritis

* Neural tube defects

* Neurofibromatosis

* Precocious puberty

* Prader-Willi Syndrome

* Russell-Silver Syndrome

* Sickle cell anemia

* Thalassemia

Source: Dr. Caroline Buckway

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