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A chance to cut is a chance to cure: complete resection of an atypical neurofibroma prevents further progression to malignancy

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A Correction to this article was published on 29 July 2023

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Abstract

Plexiform neurofibromas are the hallmark of neurofibromatosis type 1 (NF1) and significantly contribute to the overall burden of disease. While surgical excision has long been the only available therapy, the MEK inhibitor (MEKi) selumetinib has been approved as a non-surgical treatment option for these tumors in 2020 (USA) and 2021 (Europe), respectively. However, selumetinib will result in tumor shrinkage only after several months of therapy and might not prevent malignant transformation of a plexiform neurofibroma that occurs with a frequency of 10–15%. Here, we demonstrate that surgical excision might be the therapy of choice in some plexiform neurofibromas despite the availability of MEKi therapy.

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Additional data on the molecular mechanisms underlaying MPNST development and increased clinical experience with MEK inhibition in NF1 are necessary to determine the role of surgery in a multimodal treatment concept of NF1 associated plexiform neurofibromas.

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Authors and Affiliations

  1. Department of Pediatrics, Sana Kliniken, Duisburg, Germany

    Pia Vaassen, Axel Feldkamp & Thorsten Rosenbaum

  2. Department of Neurosurgery, Sana Kliniken, Duisburg, Germany

    Martin Scholz

  3. Department of Neuropathology, Universitätsklinikum, Essen, Germany

    Tobias Blau

  4. Department of Radiology and Neuroradiology, Sana Kliniken, Duisburg, Germany

    Nikola Reinhard Dürr

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  1. Pia Vaassen
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  2. Axel Feldkamp
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  3. Martin Scholz
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  4. Tobias Blau
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  5. Nikola Reinhard Dürr
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  6. Thorsten Rosenbaum
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Contributions

PV and TR wrote the main manuscript text. NR D, AF and TB prepared figures and figure legends 1-3. All authors reviewed the manuscript.

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Correspondence to Pia Vaassen.

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TR and PV received consulting fees from Alexion-AstraZeneca Rare Diseases. NRD, AF, MS, and TB declare no conflict of interests.

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Vaassen, P., Feldkamp, A., Scholz, M. et al. A chance to cut is a chance to cure: complete resection of an atypical neurofibroma prevents further progression to malignancy. Childs Nerv Syst 39, 3301–3304 (2023). https://doi.org/10.1007/s00381-023-06029-5

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