Neurofibromatosis type 1 (NF1): treatment

Written and reviewed by Dr Ibrahim Imam, Consultant Neurologist. Last reviewed .

Neurofibromatosis type 1 (NF1): treatment. Carboplatin, Vincristine. Fully referenced neurology checklist.

Treatments of optic pathway gliomas

  • Carboplatin
  • Vincristine

Treatments of cutaneous neurofibromas

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Treatments of vascular abnormalities

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Treatments of scoliosis

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Investigational drugs: for tumours

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Investigational drugs: others

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References

  1. Saleh M, Dib A, Beaini S, et al. Neurofibromatosis type 1 system-based manifestations and treatments: a review. Neurol Sci 2023; 44:1931-1947.
  2. Dombi E, Baldwin A, Marcus LJ, et al. Activity of selumetinib in neurofibromatosis type 1-related plexiform neurofibromas. N Engl J Med 2016; 375:2550-2560.
  3. Robertson KA, Nalepa G, Yang FC, et al. Imatinib mesylate for plexiform neurofibromas in patients with neurofibromatosis type 1: a phase 2 trial. Lancet Oncol 2012; 13:1218-1224. 
  4. Weiss B, Widemann BC, Wolters P, et al. Sirolimus for progressive neurofibromatosis type 1-associated plexiform neurofibromas: a neurofibromatosis Clinical Trials Consortium phase II study. Neuro Oncol 2015; 17:596-603.
  5. Slopis JM, Arevalo O, Bell CS, et al. Treatment of disfiguring cutaneous lesions in neurofibromatosis-1 with everolimus: a phase ii, open-label, single-arm trial. Drugs R D 2018; 18:295-302.
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