Diagnostic performance of desmopressin stimulation test in pediatric cushing's disease.
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OBJECTIVE: To evaluate the diagnostic performance of the desmopressin (DDAVP) stimulation test in pediatric patients with Cushing’s disease (CD), and to compare its accuracy and safety profile to the ovine corticotropin-releasing hormone (oCRH) stimulation test. DESIGN: A retrospective cohort study. METHODS: Pediatric patients with CD who underwent peripheral or bilateral inferior petrosal sinus sampling (BIPSS) stimulation testing with either DDAVP or oCRH were included. Patients were matched 1:1 for age, sex, and tumor size. The performance of each test was assessed by evaluating ACTH and cortisol responses and calculating test sensitivities. RESULTS: In peripheral stimulation testing, DDAVP demonstrated 96.9% sensitivity for cortisol and 81.3% for ACTH, while oCRH showed 93.8% and 96.9% sensitivities respectively (p > 0.05). Percentage change of ACTH was higher in the CRH group compared to DDAVP. In BIPSS, the DDAVP stimulation showed sensitivity 73.3% for baseline and 80% for post-stimulation results, while oCRH had sensitivity 93.3% and 100% respectively. Central-to-peripheral ACTH ratios were similar across groups. No major adverse events were reported, and both tests were well tolerated. CONCLUSION: Although the DDAVP stimulation test demonstrates lower diagnostic accuracy compared to the CRH test, it still provides sufficient sensitivity and given its availability and lower cost, it represents a pragmatic alternative to CRH stimulation. SIGNIFICANCE STATEMENT: The diagnosis of pediatric CD is challenging due to the rarity of the condition and limited access to dynamic testing agents such as ovine corticotropin-releasing hormone (oCRH). This study provides the largest pediatric evaluation of the desmopressin (DDAVP) stimulation test, demonstrating its diagnostic accuracy and safety profile comparable to oCRH stimulation. The findings support the use of DDAVP as a reliable and practical alternative for diagnosing CD in children, particularly in settings where oCRH is unavailable. This work addresses a critical gap in pediatric endocrinology and has the potential to improve diagnostic pathways and outcomes in this population.