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Challenges of Selumetinib Therapy for Neurofibromatosis in a Resource-Limited Setting

Year: 2025

Type: article

Abstract: Neurofibromatosis type 1 (NF1) is a genetic disorder driven by dysregulated RAS/MAPK signaling, leading to plexiform neurofibromas (PNs). Selumetinib, an MEK inhibitor, is an effective non-surgical treatment for inoperable PNs, but continuous therapy is essential to sustain tumor control. We report the first documented use of selumetinib in Iraq for an NF1 patient with an inoperable PN. After nine months of treatment, the tumor showed significant regression. However, a six-month interruption due to drug inaccessibility led to rapid regrowth. This case highlights the efficacy of selumetinib in NF1-associated PNs and the consequences of treatment interruption. It underscores the need for reliable access to targeted therapies in resource-limited settings to ensure sustained clinical benefit.

Source: Cureus

DOI: 10.7759/cureus.81071

Authors: Tamara M Khalaf, Aya A Alqadhi

Language: English

Topic: Neurofibromatosis and Schwannoma Cases

Subfield: Neurology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: diamond

OpenAlex ID: W4408773326

FWCI:

Cites: 14

Cited by:

Related to: 10