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Review
. 2015 Mar;28(2):107-12.
doi: 10.1097/YCO.0000000000000131.

Treatment of the psychiatric problems associated with fragile X syndrome

Randi J Hagerman  1 , Jonathan Polussa
Affiliations
Review

Treatment of the psychiatric problems associated with fragile X syndrome

Randi J Hagerman et al. Curr Opin Psychiatry. 2015 Mar.

Abstract

Purpose of review: This work reviews recent research regarding treatment of fragile X syndrome (FXS), the most common inherited cause of intellectual disability and autism spectrum disorder. The phenotype includes anxiety linked to sensory hyperarousal, hyperactivity, and attentional problems consistent with attention deficit hyperactivity disorder and social deficits leading to autism spectrum disorder in 60% of boys and 25% of girls with FXS.

Recent findings: Multiple targeted treatments for FXS have rescued the phenotype of the fmr1 knockout mouse, but few have been beneficial to patients with FXS. The failure of the metabotropic glutamate receptor 5 antagonists falls on the heels of the failure of Arbaclofen's efficacy in children and adults with autism or FXS. In contrast, efficacy has been demonstrated in a controlled trial of minocycline in children with FXS. Minocycline lowers the abnormally elevated levels of matrix metalloproteinase 9 in FXS. Acamprosate and lovastatin have been beneficial in open-label trials in FXS. The first 5 years of life may be the most efficacious time for intervention when combined with behavioral and/or educational interventions.

Summary: Minocycline, acamprosate, lovastatin, and sertraline are treatments that can be currently prescribed and have shown benefit in children with FXS. Use of combined medical and behavioral interventions will likely be most efficacious for the treatment of FXS.

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Conflict of interest statement

Conflicts of interest

Randi Hagerman has received funding from Novartis, Roche/Genentech, Seaside Therapeutics Neurpharm, Curemark, Forest, and Alcobra for treatment studies in fragile X syndrome, autism and Down’s syndrome. She has also consulted with Roche/Genentech, Novartis, and Alcobra regarding treatment studies in Fragile X syndrome. Jonathan Polussa does not have any conflicts.

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References

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