For Children With Neurofibromatosis Type 1, Statin Does Not Improve Cognitive Function

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Main Category: Neurology / Neuroscience
Also Included In: Clinical Trials / Drug Trials;  Cholesterol
Article Date: 16 Jul 2008 - 0:00 PDT

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For children with neurofibromatosis type 1 (NF1), a genetic disorder that often leads to learning disability, cognitive function is not improved by the statin simvastatin, according to an article released on July 15 in JAMA.

NF1 is characterized by the development of tumors in Schwann cells in the nervous system. It is genetically transmitted with an autosomal dominant pattern, which means that it is not linked to sex chromosome, but only one parent needs to contribute the variant gene for a child to develop the disorder.

These tumors can be harmless but can also compress nerve cells thus causing damage. Clinically, the disease symptoms include skin disorders, problems with small and large movement skills, and cognitive disabilities. Some examples of this cognitive disability include loss of visual-spatial skills, nonverbal long-term memory and attention span.

Statins are a class of drugs presently used in adults to lower cholesterol. Previously, some studies in mice have indicated that therapy with statins could improve some cognitive deficits. To investigate the potential effects of a specific statin, simvastatin, on the cognitive function of children with neurofibromatosis type 1, Lianne C. Krab, M.Sc., of Erasmus MC University Medical Center, Sophia Children's Hospital, Rotterdam, The Netherlands, and colleagues performed a randomized trial. A total 62 children received simvastatin or a placebo treatment once daily for 12 weeks.

After the period of treatment, several cognitive tests were performed to address nonverbal long-term memory, attention, and performance on the prism adaptation task, which measures the adaptation of hand movements in response to distortion of a prism glass. No advantage was shown for the test group over the placebo in this respect. Secondary outcomes were evaluated as well, including object assembly scores, which showed some improvement in the test population. Other measures, such as attention fluctuation and motor-visual integration showed no improvement.

The authors conclude that simvastatin has no effect on the improvement of cognitive problems developed in victims of neurofibromatosis type 1. "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," they conclude.

Effect of Simvastatin on Cognitive Functioning in Children With Neurofibromatosis Type 1: A Randomized Controlled Trial
Lianne C. Krab; Arja de Goede-Bolder; Femke K. Aarsen; Saskia M. F. Pluijm; Marlies J. Bouman; Jos N. van der Geest; Maarten Lequin; Coriene E. Catsman; Willem Frans M. Arts; Steven A. Kushner; Alcino J. Silva; Chris I. de Zeeuw; Henriëtte A. Moll; Ype Elgersma
JAMA. 2008;300(3):287-294.
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Written by Anna Sophia McKenney
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