Growth hormone replacement in patients with Langerhan's cell histiocytosis.

OBJECTIVES To assess the impact of growth hormone on growth and the underlying disease in children with growth hormone deficiency as a result of Langerhan's cell histiocytosis. STUDY DESIGN Retrospective analysis of data from the Kabi (Pharmacia & Upjohn) international growth database (KIGS) for 82 children with Langerhan's cell histiocytosis treated with recombinant growth hormone. RESULTS At the start of treatment the median (10-90th centile) age was 9.0 (5.2 to 14.7) years, with a median height standard deviation score (SDS) of -2.0 (-3.5 to -0.9). The median pretreatment height velocity (measured in cm/year) was 3.6 (0.9 to 6.4); this increased to 8.8 (3.8 to 12.0) in the first year of treatment with growth hormone, and then remained significantly greater than the pretreatment height velocity at 7.3 (4.4 to 9.7) and 7.1 (4.1 to 9.3) cm/year in the second and third years, respectively. The median height SDS increased from -2.0 to -0.8 (-2.3 to 0.6) by the end of three years of treatment. There was no increase in the recurrence rate of the underlying disease and no adverse event could be directly attributed to growth hormone treatment, apart from one case of benign intracranial hypertension that resolved on stopping treatment with growth hormone. CONCLUSIONS Growth hormone replacement treatment for patients with Langerhan's cell histiocytosis with growth hormone deficiency is beneficial and safe.