Summary

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• Selumetinib (Koselugo) is recommended for the treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). This medication has shown significant efficacy, with an Objective Response Rate (ORR) of up to 73.8% and a Disease Control Rate (DCR) of up to 96.8%. The most common adverse events associated with its use are gastrointestinal reactions.
• Four systematic reviews/meta-analyses provided comprehensive analysis on Selumetinib's safety and efficacy across various conditions, primarily NF1 and non-small cell lung cancer (NSCLC).
• Compared to other therapies for late-stage/metastatic NSCLC, Selumetinib showed better efficacy when used alone rather than in combination with other therapies, although its overall effectiveness was not superior. However, it demonstrated a significantly better safety profile compared to combined therapy regimens.
• In the treatment of different types of neurofibromatosis-related tumors, particularly NF1-associated ones in children aged two years or older who have progressive or inoperable PN, Selumetinib presented high effectiveness rates between 68%-71%, surpassing several other medications targeting similar pathways or tumor types.
• The medication demonstrates robust performance, especially among pediatric patients suffering from NF1-associated PNs, indicating that this subgroup can greatly benefit from this medication while maintaining manageable side effects.
• While the potential exists for cross-disease application, such as NSCLC treatment among broader age groups, including average ages around sixty-two years old, comparative efficiency may not surpass combined therapy options despite having a safer profile, thus emphasizing the need for patient-specific considerations during the clinical decision-making process.