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Departments of Endocrinology (R.H.L., R.E.S.), Nursing (P.S.H.), Clinical Nutrition (K.R.-S.), Pharmacy (R.K.C.), Diagnostic Imaging (S.C.K.), and Biostatistics (S.N.R., S.Y.L., S.W., X.X.), St. Jude Children’s Research Hospital, Memphis, Tennessee 38105
Address all correspondence and requests for reprints to: Robert H. Lustig, M.D., Division of Pediatric Endocrinology, Box 0136, University of California San Francisco, 500 Parnassus Avenue, San Francisco, California 94143-0136. E-mail: rlustig{at}peds.ucsf.edu.
Hypothalamic obesity is a devastating complication in children surviving brain tumors and/or cranial irradiation. These subjects are thought to exhibit autonomic dysregulation of the ß-cell, with insulin hypersecretion in response to oral glucose tolerance testing (OGTT). We report the results of a randomized, double-blind, placebo-controlled trial of octreotide therapy for pediatric hypothalamic obesity. Eighteen subjects [weight, 100.6 ± 5.6 kg; body mass index (BMI), 37.1 ± 1.3 kg/m2] received octreotide (5–15 µg/kg·d sc) or placebo for 6 months.
With octreotide, 
weight (mean ± SEM) was +1.6 ± 0.6 vs. +9.1 ± 1.7 kg for placebo (P < 0.001). BMI was -0.2 ± 0.2 vs. +2.2 ± 0.5 kg/m2, respectively (P < 0.001). OGTT documented insulin response (peak - basal) of -417 ± 304 pM after octreotide vs. +216 ± 215 pM after placebo (P = 0.034). Improvement in physical activity by parent report was noted with octreotide, but not placebo (P = 0.03). For the octreotide group, changes in quality of life positively correlated with changes in insulin response (P = 0.041). Complications and adverse events were mild and self-limited.
These data demonstrate the beneficial effects of octreotide in pediatric hypothalamic obesity. Octreotide suppressed insulin, and stabilized weight and BMI. Improved quality of life correlated with the degree of insulin suppression. Octreotide was safe and well tolerated.
This work was supported in part by the Cancer Center Support Core Grant P30-CA-12765 and the American Lebanese Syrian Associated Charities. The work was presented in part at the 6th Joint Meeting of the Lawson Wilkins Pediatric Endocrine Society/European Society for Pediatric Endocrinology, Montréal, Québec, Canada, July 2001.
Present address for R.H.L.: Department of Pediatrics, University of California San Francisco, California 94143-0136.
Abbreviations: ALL, Acute lymphoblastic leukemia; BMI, body mass index; HbA1c, hemoglobin A1c; HV, height velocity; OGTT, oral glucose tolerance testing; QoL, quality of life; SJCRH, St. Jude Children’s Research Hospital; U.T., University of Tennessee; VMH, ventromedial hypothalamus.
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