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Growth hormone replacement in patients with Langerhan's cell histiocytosis
S J Howell1, P Wilton, S M Shalet
1Department of Endocrinology, Christie Hospital NHS Trust, Withington, Manchester, UK.
Insights
Growth hormone therapy significantly improves height in children with Langerhan's cell histiocytosis and growth hormone deficiency. Treatment is safe, with no increased disease recurrence or major adverse events.
Area of Science:
- Pediatric Endocrinology
- Pediatric Oncology
- Growth Hormone Therapy
Background:
- Langerhan's cell histiocytosis (LCH) can lead to growth hormone deficiency (GHD) in children.
- Children with GHD experience significantly impaired growth.
- The efficacy and safety of growth hormone (GH) treatment in this specific population require evaluation.
Purpose of the Study:
- To evaluate the impact of recombinant human growth hormone (rhGH) on growth in children with GHD secondary to LCH.
- To assess the safety of rhGH treatment, including effects on LCH recurrence and adverse events.
- To analyze long-term growth outcomes over three years of treatment.
Main Methods:
- Retrospective analysis of data from the Kabi International Growth database (KIGS).
- Inclusion of 82 children diagnosed with LCH and GHD, treated with rhGH.
- Analysis of height standard deviation scores (SDS), height velocity, and adverse events.
Main Results:
- Median height SDS improved from -2.0 to -0.8 after three years of rhGH treatment.
- Median height velocity increased significantly from 3.6 cm/year pretreatment to 8.8 cm/year in the first year, and remained elevated.
- No increase in LCH recurrence was observed; one case of benign intracranial hypertension resolved upon treatment cessation.
Conclusions:
- Recombinant human growth hormone replacement therapy is effective in improving growth in children with GHD due to LCH.
- GH treatment demonstrates a favorable safety profile in this patient cohort.
- GH therapy is a beneficial and safe treatment option for pediatric LCH patients with GHD.
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.