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Case Reports
. 2023 Aug 17;1(4):luad101.
doi: 10.1210/jcemcr/luad101. eCollection 2023 Jul.

Successful Management of Cushing Syndrome From Ectopic ACTH Secretion in an Adolescent With Osilodrostat

Kathryn Blew  1 , David Van Mater  2 , Laura Page  1
Affiliations
Case Reports

Successful Management of Cushing Syndrome From Ectopic ACTH Secretion in an Adolescent With Osilodrostat

Kathryn Blew et al. JCEM Case Rep. .

Abstract

A previously healthy 11-year-old male was found to have a mass in the pancreatic head after several months of abdominal pain and jaundice. Pathology was consistent with a World Health Organization grade 2 pancreatic neuroendocrine tumor. He developed refractory hypertension and was found to have Cushing syndrome from ectopic ACTH secretion, with oligometastatic liver disease. He underwent surgical resection of the pancreatic tumor and metastases. Postoperatively, his Cushing syndrome resolved, but it reemerged 1 year later in the setting of disease recurrence. He was not a candidate for bilateral adrenalectomy. Ketoconazole therapy was inadequate and he was started on metyrapone, lanreotide, cabergoline, and spironolactone. Although this regimen was well-tolerated, his Cushing syndrome recurred 4 months later as his metastatic disease burden increased. Osilodrostat was begun and the dose was gradually increased in response to his uncontrolled Cushing syndrome. Osilodrostat resulted in rapid improvement and eventual normalization of his urinary free cortisol at a dose of 18 mg twice daily. He had no adverse effects. This rare case highlights the successful off-label use of osilodrostat, a medication intended for refractory Cushing disease in adult patients, in a pediatric patient with Cushing syndrome caused by ectopic ACTH secretion.

Keywords: Cushing syndrome; ectopic ACTH secretion; osilodrostat; pediatric.

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Figures

Figure 1.
Figure 1.
Gross specimen of pancreatic neuroendocrine tumor (A), microscopic specimen with hematoxylin and eosin stain of well-differentiated tumor (B), microscopic specimen with immunohistochemistry staining (brown), indicating a minority of cells positive for ACTH (C).
Figure 2.
Figure 2.
Positron emission tomography scan with Ga-68 DOTATATE before (A and B) and after (C and D) bland hepatic embolizations. Following bland embolization, the size and enhancement of hepatic metastases decreased (A and C) and nodal metastases advanced (B and D) with indication of enhancing metastatic activity with arrows.
Figure 3.
Figure 3.
Impact of bland embolization procedures and medical therapy on ACTH (A) and 24-hour urinary free cortisol (B) levels.
Figure 4.
Figure 4.
Impact of the patient's Cushing syndrome and his subsequent treatments on his body mass index percentile.
See this image and copyright information in PMC

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