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Effect of pioglitazone treatment on behavioral symptoms in autistic children

INTRODUCTION: Autism is complex neuro-developmental disorder which has a symptomatic diagnosis in patients characterized by disorders in language/communication, behavior, and social interactions. The exact causes for autism are largely unknown, but is has been speculated that immune and inflammatory...

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Autores principales: Boris, Marvin, Kaiser, Claudia C, Goldblatt, Allan, Elice, Michael W, Edelson, Stephen M, Adams, James B, Feinstein, Douglas L
Formato: Texto
Lenguaje:English
Publicado: BioMed Central 2007
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1781426/
https://www.ncbi.nlm.nih.gov/pubmed/17207275
http://dx.doi.org/10.1186/1742-2094-4-3
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author Boris, Marvin
Kaiser, Claudia C
Goldblatt, Allan
Elice, Michael W
Edelson, Stephen M
Adams, James B
Feinstein, Douglas L
author_facet Boris, Marvin
Kaiser, Claudia C
Goldblatt, Allan
Elice, Michael W
Edelson, Stephen M
Adams, James B
Feinstein, Douglas L
author_sort Boris, Marvin
collection PubMed
description INTRODUCTION: Autism is complex neuro-developmental disorder which has a symptomatic diagnosis in patients characterized by disorders in language/communication, behavior, and social interactions. The exact causes for autism are largely unknown, but is has been speculated that immune and inflammatory responses, particularly those of Th2 type, may be involved. Thiazolidinediones (TZDs) are agonists of the peroxisome proliferator activated receptor gamma (PPARγ), a nuclear hormone receptor which modulates insulin sensitivity, and have been shown to induce apoptosis in activated T-lymphocytes and exert anti-inflammatory effects in glial cells. The TZD pioglitazone (Actos) is an FDA-approved PPARγ agonist used to treat type 2 diabetes, with a good safety profile, currently being tested in clinical trials of other neurological diseases including AD and MS. We therefore tested the safety and therapeutic potential of oral pioglitazone in a small cohort of children with diagnosed autism. CASE DESCRIPTION: The rationale and risks of taking pioglitazone were explained to the parents, consent was obtained, and treatment was initiated at either 30 or 60 mg per day p.o. A total of 25 children (average age 7.9 ± 0.7 year old) were enrolled. Safety was assessed by measurements of metabolic profiles and blood pressure; effects on behavioral symptoms were assessed by the Aberrant Behavior Checklist (ABC), which measures hyperactivity, inappropriate speech, irritability, lethargy, and stereotypy, done at baseline and after 3–4 months of treatment. DISCUSSION AND EVALUATION: In a small cohort of autistic children, daily treatment with 30 or 60 mg p.o. pioglitazone for 3–4 months induced apparent clinical improvement without adverse events. There were no adverse effects noted and behavioral measurements revealed a significant decrease in 4 out of 5 subcategories (irritability, lethargy, stereotypy, and hyperactivity). Improved behaviors were inversely correlated with patient age, indicating stronger effects on the younger patients. CONCLUSION: Pioglitazone should be considered for further testing of therapeutic potential in autistic patients.
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spelling pubmed-17814262007-01-25 Effect of pioglitazone treatment on behavioral symptoms in autistic children Boris, Marvin Kaiser, Claudia C Goldblatt, Allan Elice, Michael W Edelson, Stephen M Adams, James B Feinstein, Douglas L J Neuroinflammation Case Study INTRODUCTION: Autism is complex neuro-developmental disorder which has a symptomatic diagnosis in patients characterized by disorders in language/communication, behavior, and social interactions. The exact causes for autism are largely unknown, but is has been speculated that immune and inflammatory responses, particularly those of Th2 type, may be involved. Thiazolidinediones (TZDs) are agonists of the peroxisome proliferator activated receptor gamma (PPARγ), a nuclear hormone receptor which modulates insulin sensitivity, and have been shown to induce apoptosis in activated T-lymphocytes and exert anti-inflammatory effects in glial cells. The TZD pioglitazone (Actos) is an FDA-approved PPARγ agonist used to treat type 2 diabetes, with a good safety profile, currently being tested in clinical trials of other neurological diseases including AD and MS. We therefore tested the safety and therapeutic potential of oral pioglitazone in a small cohort of children with diagnosed autism. CASE DESCRIPTION: The rationale and risks of taking pioglitazone were explained to the parents, consent was obtained, and treatment was initiated at either 30 or 60 mg per day p.o. A total of 25 children (average age 7.9 ± 0.7 year old) were enrolled. Safety was assessed by measurements of metabolic profiles and blood pressure; effects on behavioral symptoms were assessed by the Aberrant Behavior Checklist (ABC), which measures hyperactivity, inappropriate speech, irritability, lethargy, and stereotypy, done at baseline and after 3–4 months of treatment. DISCUSSION AND EVALUATION: In a small cohort of autistic children, daily treatment with 30 or 60 mg p.o. pioglitazone for 3–4 months induced apparent clinical improvement without adverse events. There were no adverse effects noted and behavioral measurements revealed a significant decrease in 4 out of 5 subcategories (irritability, lethargy, stereotypy, and hyperactivity). Improved behaviors were inversely correlated with patient age, indicating stronger effects on the younger patients. CONCLUSION: Pioglitazone should be considered for further testing of therapeutic potential in autistic patients. BioMed Central 2007-01-05 /pmc/articles/PMC1781426/ /pubmed/17207275 http://dx.doi.org/10.1186/1742-2094-4-3 Text en Copyright © 2007 Boris et al; licensee BioMed Central Ltd. http://creativecommons.org/licenses/by/2.0 This is an Open Access article distributed under the terms of the Creative Commons Attribution License ( (http://creativecommons.org/licenses/by/2.0) ), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Case Study
Boris, Marvin
Kaiser, Claudia C
Goldblatt, Allan
Elice, Michael W
Edelson, Stephen M
Adams, James B
Feinstein, Douglas L
Effect of pioglitazone treatment on behavioral symptoms in autistic children
title Effect of pioglitazone treatment on behavioral symptoms in autistic children
title_full Effect of pioglitazone treatment on behavioral symptoms in autistic children
title_fullStr Effect of pioglitazone treatment on behavioral symptoms in autistic children
title_full_unstemmed Effect of pioglitazone treatment on behavioral symptoms in autistic children
title_short Effect of pioglitazone treatment on behavioral symptoms in autistic children
title_sort effect of pioglitazone treatment on behavioral symptoms in autistic children
topic Case Study
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1781426/
https://www.ncbi.nlm.nih.gov/pubmed/17207275
http://dx.doi.org/10.1186/1742-2094-4-3
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