Growth hormone replacement and the risk of malignancy in children with neurofibromatosis.

OBJECTIVE

To assess the efficacy and safety of growth hormone (GH) therapy in children with GH deficiency in association with neurofibromatosis.

METHODS

Retrospective analysis of data from the Pharmacia and Upjohn International Growth Database (KIGS) in a total of 102 GH-deficient children with neurofibromatosis treated with recombinant GH.

RESULTS

Median pretreatment height velocity was 4.2 cm/yr (1.7 to 6.4 cm/yr), increased to 7.1 cm/yr (4.6 to 10.0 cm/yr) in the first year of GH therapy, and remained significantly greater than pretreatment at 5.7 cm/yr (2.9 to 8.3 cm/yr) and 5.7 cm/yr (2.6 to 7.9 cm/yr) in the second and third years, respectively. The median height SD score increased from -2.4 to -1.8 by the end of 3 years of treatment. Five patients had either a recurrence of an intracranial tumor or a second intracranial tumor; this incidence of tumor occurrence is comparable to that reported previously in similar patients with neurofibromatosis. Other adverse events were relatively minor and unlikely to be attributable to GH therapy

CONCLUSIONS

The data indicate that GH replacement therapy, per se, for patients with neurofibromatosis and GH deficiency is likely to be beneficial and unassociated with excessive malignant risk.