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Open-label memantine in fragile X syndrome
Craig A Erickson1, Jennifer E Mullett, Christopher J McDougle
1Department of Psychiatry, Indiana University School of Medicine, 702 Barnhill Drive, Room 4300, Indianapolis, IN 46202, USA. crericks@iupui.edu
Journal of Autism and Developmental Disorders
|July 18, 2009
Summary
Memantine showed modest effectiveness for fragile X syndrome (FXS) symptoms in a pilot study. While some patients improved globally, specific symptom scales did not show significant changes, and irritability was a side effect.
Area of Science:
- Neuroscience
- Pharmacology
- Genetics
Background:
- Glutamatergic dysfunction is a key factor in fragile X syndrome (FXS) pathophysiology.
- FXS is a genetic disorder associated with intellectual disability and behavioral challenges.
Purpose of the Study:
- To evaluate the effectiveness and tolerability of memantine in patients with FXS.
- To assess memantine's impact on target symptoms in FXS, particularly in those with comorbid Pervasive Developmental Disorder (PDD).
Main Methods:
- A pilot study involving a review of medical records from 6 patients with FXS and PDD.
- Open-label treatment with memantine was administered over a mean duration of 34.7 weeks.
Main Results:
- Four out of six (67%) patients demonstrated global clinical benefit as assessed by the Clinical Global Impression-Improvement (CGI-I) scale.
- No statistically significant improvements were observed on symptom-specific rating scales.
- Two patients experienced treatment-limiting irritability while on memantine.
Conclusions:
- Memantine demonstrated modest effectiveness in some patients with FXS.
- Further rigorous investigation is necessary to confirm these findings and establish memantine's role in FXS treatment.
