Octreotide Therapy of Pediatric Hypothalamic Obesity: A Double-Blind, Placebo-Controlled Trial
Robert H. Lustig,
Pamela S. Hinds,
Karen Ringwald-Smith,
Robbin K. Christensen,
Sue C. Kaste,
Randi E. Schreiber,
Shesh N. Rai,
Shelly Y. Lensing,
Shengjie Wu and
Xiaoping Xiong
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 childrensurviving brain tumors and/or cranial irradiation. These subjectsare thought to exhibit autonomic dysregulation of the ß-cell,with insulin hypersecretion in response to oral glucose tolerancetesting (OGTT). We report the results of a randomized, double-blind,placebo-controlled trial of octreotide therapy for pediatrichypothalamic obesity. Eighteen subjects [weight, 100.6 ±5.6 kg; body mass index (BMI), 37.1 ± 1.3 kg/m2] receivedoctreotide (5–15 µg/kg·d sc) or placebo for6 months.
With octreotide, weight (mean ± SEM) was +1.6 ±0.6 vs. +9.1 ± 1.7 kg for placebo (P < 0.001). BMIwas -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 ± 215pM after placebo (P = 0.034). Improvement in physical activityby parent report was noted with octreotide, but not placebo(P = 0.03). For the octreotide group, changes in quality oflife positively correlated with changes in insulin response(P = 0.041). Complications and adverse events were mild andself-limited.
These data demonstrate the beneficial effects of octreotidein pediatric hypothalamic obesity. Octreotide suppressed insulin,and stabilized weight and BMI. Improved quality of life correlatedwith the degree of insulin suppression. Octreotide was safeand well tolerated.
This work was supported in part by the Cancer Center SupportCore Grant P30-CA-12765 and the American Lebanese Syrian AssociatedCharities. The work was presented in part at the 6th Joint Meetingof the Lawson Wilkins Pediatric Endocrine Society/European Societyfor Pediatric Endocrinology, Montréal, Québec,Canada, July 2001.
Present address for R.H.L.: Department of Pediatrics, Universityof California San Francisco, California 94143-0136.
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weight (mean ± SEM) was +1.6 ± 0.6 vs. +9.1 ± 1.7 kg for placebo (P < 0.001). 
