Selumetinib in the Treatment of Symptomatic Intractable Plexiform Neurofibromas in Neurofibromatosis Type 1: A Prospective Case Series with Emphasis on Side Effects.

Author: BaldoFrancesco, BarbiEgidio, BassoLuca, BrunoIrene, Cortellazzo WielLuisa, GrassoAntonio Giacomo, MaestroAlessandra, MagnolatoAndrea, MurruFlora Maria, TrojniakMarta Paulina

Paper Details 
Original Abstract of the Article :
Plexiform neurofibromas (PN) are congenital tumors that affect up to 50% of individuals with neurofibromatosis type 1. Despite their benign nature, they can grow rapidly and cause severe morbidities. Selumetinib, an inhibitor of mitogen-activated protein kinase (MEK) 1 and 2, was reported to induce ...See full text at original site
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引用元:
https://doi.org/10.1007/s40272-020-00399-y

データ提供:米国国立医学図書館(NLM)

Selumetinib: A Potential Hope for Plexiform Neurofibromas?

Plexiform neurofibromas (PNs) can be a real burden for individuals with neurofibromatosis type 1 (NF1), often causing significant morbidity. This research investigates the potential of selumetinib, a MEK inhibitor, as a treatment for symptomatic, intractable PNs. The authors present a prospective case series focusing on the side effects of selumetinib in pediatric patients with inoperable PNs. The study provides valuable insights into the safety and potential efficacy of selumetinib in this context, offering a glimmer of hope for patients with limited treatment options.

Selumetinib: A New Path for Treating Plexiform Neurofibromas?

The study highlights the potential of selumetinib as a treatment option for inoperable PNs. While the results are based on a small case series, the observed clinical responses suggest that selumetinib might offer a new path for managing these challenging tumors. The authors emphasize the need for larger, controlled trials to confirm these findings and to further evaluate the long-term safety and efficacy of selumetinib in this setting.

Navigating the Challenges of Neurofibromatosis Type 1

Living with NF1 can be a challenging experience, especially for those with PNs. This research provides a ray of hope, showcasing the potential of selumetinib as a treatment option. While further research is needed to fully understand its efficacy and safety, the study offers a glimmer of hope for individuals with NF1 who are seeking relief from the debilitating effects of PNs.

Dr.Camel's Conclusion

Just as a camel adapts to the harsh desert environment, individuals with NF1 and PNs must adapt to their condition. This research offers a potential new oasis in the challenging landscape of NF1, suggesting that selumetinib might be a valuable tool for managing PNs. However, like a mirage in the desert, the promise of selumetinib needs to be carefully explored through further research to ensure its true potential for those navigating this challenging condition.

Date :
  1. Date Completed 2020-08-17
  2. Date Revised 2020-08-17
Further Info :

Pubmed ID

32533336

DOI: Digital Object Identifier

10.1007/s40272-020-00399-y

SNS
PICO Info
in preparation
Languages

English

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