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February 8, 20262 citations

Neurofibromatosis type 1 plexiform neurofibromas: Integrating treatment across pediatric and adult populations.

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AAAmy E. ArmstrongSt. Louis Children's HospitalAGAndrea M. GrossCincinnati Children's Hospital Medical CenterLKLaura KlesseTexas Scottish Rite Hospital for Children

Key Points

  • The aim is to explore effective treatments for plexiform neurofibromas in pediatric and adult neurofibromatosis type 1 patients.
  • Review current therapeutic strategies including surgery and medical therapies.
  • Evaluate the impacts of disease variability and age on treatment tolerability.
  • Highlight the need for individualized care and multidisciplinary management.
  • About 50% of individuals with neurofibromatosis type 1 develop plexiform neurofibromas.
  • MEK inhibitors show promise but require further study for long-term efficacy in pediatric patients.
  • Age-specific treatment planning is critical due to different clinical presentations and comorbidities.

Abstract

Plexiform neurofibromas (PNs) are a hallmark of neurofibromatosis type 1 (NF1), affecting ∼50% of individuals with the condition. Originating from Schwann cells and other peripheral nerve sheath components, these tumors can cause significant morbidity, including functional impairment, diminished health-related quality of life, chronic pain, and malignant transformation. Managing NF1-PNs is challenging because of disease variability, differing growth rates, and age-related differences in clinical presentation and treatment tolerability. This review examines current therapeutic strategies, including surgery, medical therapies, and emerging treatments, emphasizing individualized care. Highlighted here is the need for age-specific treatment planning, particularly as disease progression, comorbidities, and side-effect profiles differ between pediatric and adult patients. Optimizing outcomes requires personalized surveillance and coordinated multidisciplinary management across all age groups. While MEK inhibitors (MEKi) provide therapeutic benefit, their long-term efficacy and safety, particularly in pediatric patients who may receive these agents for extended periods, warrant further investigation. Additionally, adult patients face unique comorbidities that may complicate therapy. Superficial PNs and potential MEK inhibitor resistance remain underexplored. Growing interest in combination therapies and adjuvant strategies may improve outcomes. Ongoing research is crucial to personalize treatment regimens, to identify effective combinations, and to refine surveillance protocols, ultimately enhancing long-term quality of life for individuals living with NF1-PN.

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Cite This Study

Armstrong et al. (2026) studied this question.

synapsesocial.com/papers/698827c90fc35cd7a8846b9ahttps://doi.org/10.1093/neuonc/noag023
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