Octreotide Therapy of Pediatric Hypothalamic Obesity: A Double-Blind, Placebo-Controlled Trial

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 β-cell, with insulin hypersecretion in response to oral glucose tolerance testing (OGTT). We report the results of a...

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Veröffentlicht in:The journal of clinical endocrinology and metabolism 2003-06, Vol.88 (6), p.2586-2592
Hauptverfasser: Lustig, Robert H., Hinds, Pamela S., Ringwald-Smith, Karen, Christensen, Robbin K., Kaste, Sue C., Schreiber, Randi E., Rai, Shesh N., Lensing, Shelly Y., Wu, Shengjie, Xiong, Xiaoping
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container_issue 6
container_start_page 2586
container_title The journal of clinical endocrinology and metabolism
container_volume 88
creator Lustig, Robert H.
Hinds, Pamela S.
Ringwald-Smith, Karen
Christensen, Robbin K.
Kaste, Sue C.
Schreiber, Randi E.
Rai, Shesh N.
Lensing, Shelly Y.
Wu, Shengjie
Xiong, Xiaoping
description 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 β-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, Δweight (mean ± sem) was +1.6 ± 0.6 vs. +9.1 ± 1.7 kg for placebo (P < 0.001). ΔBMI was −0.2 ± 0.2 vs. +2.2 ± 0.5 kg/m2, respectively (P < 0.001). OGTT documented Δinsulin 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.
doi_str_mv 10.1210/jc.2002-030003
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These subjects are thought to exhibit autonomic dysregulation of the β-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, Δweight (mean ± sem) was +1.6 ± 0.6 vs. +9.1 ± 1.7 kg for placebo (P &lt; 0.001). ΔBMI was −0.2 ± 0.2 vs. +2.2 ± 0.5 kg/m2, respectively (P &lt; 0.001). OGTT documented Δinsulin 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). 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These subjects are thought to exhibit autonomic dysregulation of the β-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, Δweight (mean ± sem) was +1.6 ± 0.6 vs. +9.1 ± 1.7 kg for placebo (P &lt; 0.001). ΔBMI was −0.2 ± 0.2 vs. +2.2 ± 0.5 kg/m2, respectively (P &lt; 0.001). OGTT documented Δinsulin 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.</abstract><cop>United States</cop><pub>Endocrine Society</pub><pmid>12788859</pmid><doi>10.1210/jc.2002-030003</doi><tpages>7</tpages><oa>free_for_read</oa></addata></record>
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source MEDLINE; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; Oxford University Press Journals All Titles (1996-Current)
subjects Adolescent
Blood Glucose - analysis
Body Height - drug effects
Body Mass Index
Body Weight - drug effects
Child
Double-Blind Method
Energy Intake - drug effects
Female
Hormones - adverse effects
Hormones - therapeutic use
Humans
Hypothalamic Diseases - complications
Insulin - blood
Insulin-Like Growth Factor I - metabolism
Leptin - blood
Male
Obesity - drug therapy
Obesity - etiology
Obesity - pathology
Obesity - physiopathology
Octreotide - adverse effects
Octreotide - therapeutic use
Placebos
Quality of Life
Safety
title Octreotide Therapy of Pediatric Hypothalamic Obesity: A Double-Blind, Placebo-Controlled Trial
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