Pituitary function and the response to GH therapy in patients with Langerhans cell histiocytosis: analysis of the KIMS database.

The majority of adult LCH patients with GHD in the KIMS database were female and had childhood-onset disease in a substantial proportion.

• 81 adults with LCH were studied at baseline
• 27 patients had childhood onset GHD
• 56% of patients were female
• Mean age at GHD onset was 29 (±15) years

Diabetes insipidus was highly prevalent among LCH patients with GHD in the KIMS database.

• Diabetes insipidus was diagnosed in 86% of LCH patients
• This reflects the well-known propensity of LCH to affect the hypothalamic-pituitary axis

GH replacement therapy significantly reduced total cholesterol in LCH patients after 1 year of treatment.

• Analysis of 1 year of GH treatment was possible in 37 patients
• Total cholesterol decreased by 0.9 mmol/L (95% CI: -1.5 to -0.3 mmol/L; P < 0.05)
• The mean GH dose at 1 year was 0.39 (±0.21) mg
• IGF-1 SDS at 1 year was -0.5 (95% CI: -1.2 to 0.2)

Quality of life, as measured by the AGHDA score, improved significantly after 1 year of GH replacement in LCH patients.

• AGHDA-QoL score was assessed in 20 patients
• AGHDA score improved by 2.8 points (95% CI: -5.6 to 0.0; P < 0.05)
• A decrease in AGHDA score indicates improvement in quality of life

BMI increased modestly but not significantly after 1 year of GH treatment in LCH patients.

• Mean BMI increased by 0.6 ± 3 kg/m² (95% CI: -0.2 to 1.4)
• This change was not statistically significant based on the confidence interval crossing zero

The metabolic and quality of life effects of GH therapy in LCH patients did not differ significantly from those in patients with granulomatous or lymphocytic hypophysitis.

• The LCH population was compared to two groups: granulomatous hypophysitis and lymphocytic hypophysitis
• All treatment effects were adjusted for age, gender, and baseline values
• No statistically significant differences were found between the three groups after adjustment

Serious adverse events were reported in a substantial proportion of LCH patients during GH therapy, but no new safety signals were identified.

• 77 patients were included in the safety analysis
• 20 of 77 patients had serious adverse events reported
• 36 serious adverse events were reported during 435 patient-years of follow-up
• The rate of serious adverse events was 82.8 per 1000 patient-years
• No new safety signals were reported