• Growth hormone and neurofibromatosis.

      Howell, Simon J; Wilton, Patrick; Lindberg, Anders; Shalet, Stephen M; Department of Endocrinology, Christie Hospital NHS Trust, Manchester, UK. (2000)
      Data collected from 102 neurofibromatosis (NF) children with growth hormone (GH) deficiency (GHD) who were receiving GH replacement therapy were reviewed to assess the efficacy and safety of GH therapy in this condition. GH was administered at a mean dose of 0. 18 mg/kg/week. During the 1st year the median height velocity increased significantly from 4.2 cm/year before treatment to 7.1 cm/year, and the median height standard deviation score increased from -2.4 to -1.9. The response to therapy, however, was not as good as that observed in patients with idiopathic GHD. GH therapy did not influence the progression of any of the features of NF, including intracranial tumours, and was not associated with an excess of other adverse events. We conclude that GH treatment of NF patients with GHD is beneficial in terms of improved growth rate and is well tolerated.
    • Growth hormone replacement and the risk of malignancy in children with neurofibromatosis.

      Howell, Simon J; Wilton, Patrick; Lindberg, Anders; Shalet, Stephen M; Department of Endocrinology, Christie Hospital NHS Trust, Withington, Manchester, United Kingdom. (1998-08)
      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.