Simvastatin Not Linked to Neurofibromatosis Benefits

July 15, 2008

The use of simvastatin was not associated with cognitive improvements in children with neurofibromatosis type 1, contrary to findings in mouse models suggesting efficacy of this treatment, according to research published in the July 16 issue of the Journal of the American Medical Association.

TUESDAY, July 15 (HealthDay News) -- The use of simvastatin was not associated with cognitive improvements in children with neurofibromatosis type 1 (NF1), contrary to findings in mouse models suggesting efficacy of this treatment, according to research published in the July 16 issue of the Journal of the American Medical Association.

Lianne C. Krab, of the Erasmus MC University Medical Center in Rotterdam, the Netherlands, and colleagues analyzed data from 62 children with NF1 who were randomized to receive daily simvastatin or placebo for 12 weeks. Median age was 13.2 in the treatment group and 11.5 in the placebo group. The primary outcome measures included performance on tests of delayed recall, speed, assessing attention, cerebellar function and a measurement on MRI at baseline and after treatment.

The researchers found no significant differences between the groups on any of the primary outcome measures. On one secondary outcome measure -- an object assembly test -- the treatment group showed significant improvement in scores, specifically in children with poor baseline performance. The authors, however, felt that this finding was suspect.

"The negative outcome of this trial suggests that simvastatin should not be prescribed to ameliorate the cognitive deficits associated with NF1. Further studies to evaluate a longer treatment period and whether the object assembly finding is spurious may be warranted," the authors conclude.

Two of the study co-authors disclosed being co-applicants on a patent for treating learning deficits with HMG-CoA reductase inhibitors.

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