At-Risk Phenotype of Neurofibromatose-1 Patients: A Multicentre Case-Control Study
2011

At-Risk Phenotype of Neurofibromatosis Type 1 Patients

Sample size: 220 publication 10 minutes Evidence: high

Author Information

Author(s): Sbidian Emilie, Bastuji-Garin Sylvie, Valeyrie-Allanore Laurence, Ferkal Salah, Lefaucheur Jean P, Drouet Alain, Brugière Pierre, Vialette Cédric, Combemale Patrick, Barbarot Sébastien, Wolkenstein Pierre

Primary Institution: Université Paris Est (UPEC)

Hypothesis

The association between subcutaneous neurofibromas and internal neurofibromas in patients with neurofibromatosis type 1 is related to peripheral neuropathy.

Conclusion

Subcutaneous neurofibromas are significantly associated with internal neurofibromas and axonal neuropathies in neurofibromatosis type 1 patients.

Supporting Evidence

  • Patients with at least ten subcutaneous neurofibromas had a significantly higher risk of internal neurofibromas.
  • Axonal neuropathies with slowed conduction velocities were more common in patients with at least ten subcutaneous neurofibromas.
  • Subcutaneous neurofibromas were associated with a higher risk of malignant transformation.

Takeaway

People with a certain type of skin tumor called subcutaneous neurofibromas are more likely to have other tumors inside their bodies and nerve problems.

Methodology

This was a prospective multicentre case-control study involving 110 NF-1 patients with subcutaneous neurofibromas and 110 matched controls.

Potential Biases

Potential assessment bias was minimized by having independent reviewers for MRI and electrophysiological data.

Limitations

Selection bias cannot be excluded, and the study was limited to patients from specific hospital centers.

Participant Demographics

Mean age of participants was 41 years, with 59% being female.

Statistical Information

P-Value

p<0.05

Confidence Interval

95% CI provided for various odds ratios.

Statistical Significance

p<0.05

Digital Object Identifier (DOI)

10.1186/1750-1172-6-51

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