Abstract | OBJECTIVE: METHODS: Fifteen individuals with FXS, ages 6-23, received lithium titrated to levels of 0.8-1.2 mEq/L. The primary outcome measure, the Aberrant Behavior Checklist --Community Edition (ABC-C) Irritability Subscale, secondary outcome measures (other ABC-C subscales, clinical global improvement scale (CGI), visual analog scale for behavior (VAS), Vineland Adaptive Behavior Scale (VABS)), exploratory cognitive and psychophysiological measures and an extracellular signal-regulated kinase (ERK) activation assay were administered at baseline and 2 months of treatment. Side effects were quantified with a standardized checklist and lithium level, complete blood count (CBC), thyroid stimulating hormone (TSH), and chemistry screen were done at baseline, 2 weeks, 4 weeks and 2 months. RESULTS: The only significant treatment-related side effects were polyuria/ polydipsia (n = 7) and elevated TSH (n = 4). Although the ABC-C Irritability Subscale showed only a trend toward improvement, there was significant improvement in the Total ABC-C score (p = 0.005), VAS (p = 0.003), CGI (p = 0.002), VABS Maladaptive Behavior Subscale (p = 0.007), and RBANS List Learning (p = 0.03) and an enhanced ERK activation rate (p = 0.007). Several exploratory tasks proved too difficult for lower-functioning FXS subjects. CONCLUSIONS: Results from this study are consistent with results in mouse and fly models of FXS, and suggest that lithium is well-tolerated and provides functional benefits in FXS, possibly by modifying the underlying neural defect. A placebo-controlled trial of lithium in FXS is warranted.
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Authors | Elizabeth Berry-Kravis, Allison Sumis, Crystal Hervey, Michael Nelson, Stephen W Porges, Ning Weng, Ivan Jeanne Weiler, William T Greenough |
Journal | Journal of developmental and behavioral pediatrics : JDBP
(J Dev Behav Pediatr)
Vol. 29
Issue 4
Pg. 293-302
(Aug 2008)
ISSN: 1536-7312 [Electronic] United States |
PMID | 18698192
(Publication Type: Clinical Trial, Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
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Chemical References |
- Antidepressive Agents
- Receptors, Metabotropic Glutamate
- Lithium Carbonate
- Thyrotropin
- Extracellular Signal-Regulated MAP Kinases
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Topics |
- Administration, Oral
- Adolescent
- Adult
- Antidepressive Agents
(administration & dosage, adverse effects, therapeutic use)
- Blood Cell Count
- Child
- Cognition
(drug effects)
- Extracellular Signal-Regulated MAP Kinases
(drug effects)
- Fragile X Syndrome
(drug therapy, psychology)
- Humans
- Learning
(drug effects)
- Lithium Carbonate
(adverse effects, blood, therapeutic use)
- Pilot Projects
- Psychiatric Status Rating Scales
(statistics & numerical data)
- Psychological Tests
(statistics & numerical data)
- Receptors, Metabotropic Glutamate
(drug effects)
- Thyrotropin
(blood)
- Treatment Outcome
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