Primary treatment regimen and diabetes insipidus as predictors of health outcomes in adults with childhood-onset craniopharyngioma.
BACKGROUND
Craniopharyngiomas are often associated with significant morbidity due to their location and treatment effects. Little is known of the effects of primary treatment regimen and diabetes insipidus (DI), a clinical surrogate of hypothalamic obesity, on health outcomes in adults with childhood-onset craniopharyngioma (COCP).


OBJECTIVE
The objective of the study was to examine health outcomes of adults with COCP based on primary treatment regimens and the presence of DI.


METHODS
This study included a retrospective KIMS (Pfizer International Metabolic Database) data analysis of 180 adults with COCP according to the primary treatment regimen [one surgery (1Surg) vs complex treatment regimen (CTrR) of more than 1Surg and/or radiotherapy] and the presence of DI.


RESULTS
The majority of COCP patients underwent transcranial surgery (77%) without receiving radiotherapy (84%). Compared with the 1Surg group, more CTrR patients developed visual field defects and ophthalmoplegia (all P < .01). Compared with patients without DI, those with DI had higher rates of anterior pituitary hormone deficits, body mass index, and fat mass (all P < .01). By contrast, fasting glucose, hemoglobin A1c, lipid panel, and quality of life were comparable among 1Surg vs CTrR patients, and patients with vs without DI. Regardless of primary treatment received, the presence of DI in either group was associated with higher rates of anterior pituitary hormone deficits and obesity.


CONCLUSIONS
CTrR and DI predicted health outcomes differently. CTrR predisposed to the development of visual dysfunction, whereas DI was associated with higher rates of anterior pituitary dysfunction and weight gain. Higher body mass index and fat mass in patients with DI further implicate the role of hypothalamic damage as an important causal factor of obesity in these patients.
Division of Endocrinology, Diabetes, and Clinical Nutrition (K.C.J.Y., D.M.C.), Department of Medicine, Oregon Health and Science University, Portland, Oregon 97239; Pfizer Endocrine Care (M.K.-H., P.J.J.), Pfizer Inc, SE-190 91 Sollentuna, Sweden; Pfizer Endocrine Care (J.L.F.), Pfizer, Inc, New York, New York 10017; The Saban Research Institute (M.E.G.), Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, California 90027; and Antwerp Centre for Endocrinology (R.A.), Antwerp B-2600, Belgium.
Kevin C J
KC
Yuen
Maria
M
Kołtowska-Häggström
David M
DM
Cook
Janet L
JL
Fox
Peter J
PJ
Jönsson
Mitchell E
ME
Geffner
Roger
R
Abs
D000293:Adolescent; D000328:Adult; D017668:Age of Onset; D002648:Child; D002675:Child, Preschool; D003397:Craniopharyngioma / Q000150:complications* / Q000175:diagnosis* / Q000628:therapy; D003430:Cross-Sectional Studies; D003919:Diabetes Insipidus / Q000150:complications* / Q000453:epidemiology; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D020360:Neoadjuvant Therapy / Q000379:methods* / Q000706:statistics & numerical data; D010911:Pituitary Neoplasms / Q000150:complications* / Q000175:diagnosis* / Q000628:therapy; D011379:Prognosis; D016896:Treatment Outcome; D055815:Young Adult