PSAT043 Liraglutide Ameliorates Symptoms of Cushing Syndrome in a Pediatric Patient

BACKGROUND: Cushing Syndrome (CS) is a rare disease characterized by hypercortisolism. Pituitary adenomas resulting in ACTH-dependent disease may be difficult to visualize radiologically, resulting in a prolonged diagnostic course and delay in definitive treatment. Adjuvant medical treatment with pharmacologic agents, including steroidogenesis inhibitors and adrenolytics, can be utilized for symptom management. However, hypercortisolemia may be refractory to medical therapy making it necessary to consider symptomatic treatments. We present a case of a pediatric patient with Cushing syndrome in whom treatment with liraglutide, a GLP-1 agonist approved for the treatment of hyperglycemia and obesity, resulted in improvement of symptoms of hypercortisolemia. CASE: A 13 year old Caucasian female presented with a 2 year history of linear growth arrest, accelerated weight gain and delayed puberty. Her physical examination was remarkable for overt signs of hypercortisolemia. Initial laboratory evaluation showed normal thyroid function, hyperglycemia with HbA1c 7.2% (normal < 5.7%), dyslipidemia and elevated midnight salivary cortisol 0.898 (normal < 0.112 mcg/mL). Hypercortisolemia was confirmed when morning serum cortisol was 25.6 mcg/dL after 1 mg overnight dexamethasone suppression test (DST). This along with an elevated morning ACTH level, 72.8 (7.2- 63.3 pg/mL), confirmed a diagnosis of ACTH-dependent Cushing syndrome. Magnetic Resonance Imaging of the pituitary gland with contrast showed a 4 mm non- enhancing lesion consistent with a Rathke's cleft cyst which was unlikely to be the source of excess ACTH. Results from high dose (8 mg overnight) DST, Corticotropin Releasing Hormone (CRH) stimulation test, and Inferior Petrosal Sinus Sampling for ACTH post CRH stimulation were partially inconsistent with pituitary disease. Further workup for ectopic source(s) of ACTH showed normal imaging of the neck, chest, abdomen and pelvis. A DOTATATE PET scan failed to identify an ACTH source. Adrenolytic adjuvant treatment with mitotane was started but was discontinued due to intolerable gastrointestinal effects. Treatment was changed to ketoconazole, however, hypercortisolemia remained refractory to treatment as evidenced by continued elevation of 24- hour urine free cortisol levels. Metformin was initiated for hyperglycemia treatment but was not tolerated due to persistent nausea and vomiting. The patient was switched to liraglutide and within 3 months she had 10% weight loss, normalization of fasting blood glucose levels and reduction of HbA1c to 5.7%. Additionally, the patient's energy level and baseline hidradenitis suppurativa improved. CONCLUSION: In some pediatric patients with CS, definitive treatment of hypercortisolemia may be delayed or not feasible due to a lack of biochemical or radiological evidence showing the specific etiology for CS. Medical treatment with pharmacologic agents can be utilized for symptom management, however, hypercortisolemia may persist and even be recalcitrant to therapy. In this setting, clinicians should consider using liraglutide to ameliorate some of the cardinal symptoms of CS in pediatric patients. Presentation: Saturday, June 11, 2022 1:00 p.m. - 3:00 p.m.