What is the treatment of selumetinib? How is the effect?
Selumetinib is a MEK1/2 inhibitor mainly used to treat type 1 neurofibromatosis related plexiform neurofibromatosis. It exerts anti-tumor effects by inhibiting the abnormally activated RAS/RAF/MEK/ERK signaling pathway, and has been clinically shown to significantly reduce tumor volume and improve symptoms.
1. Treat diseases
Smetinib has been approved for the treatment of symptomatic, non-surgical plexiform neurofibromatosis (PN) in patients aged 2 years and older with neurofibromatosis type 1 (NF1). This indication is based on FDA accelerated approval and is applicable to patients whose tumors cause significant incidence rate or complex complications.
2. Mechanism of action
By selectively inhibiting the activity of MEK1 and MEK2 kinases, abnormal activation of the RAS/RAF/MEK/ERK signaling pathway is blocked. This pathway is continuously activated in NF1 patients due to neurofibrillin deficiency, leading to tumor growth.
3. Therapeutic effect
(1) Objective relief rate
Key clinical trials have shown that the objective response rate of NF1 related PN patients receiving treatment with sematinib is 66%, with a median response time of 1.3 months, and 82% of patients have sustained response for ≥ 1 year.
(2) Symptom improvement
It can significantly alleviate tumor related pain, functional impairment, and disfigurement, with 67% of patients reporting a decrease in pain intensity and 58% of patients reporting improvement in motor function.
(3) Long term therapeutic effect
Continuous treatment can maintain the tumor shrinkage effect, with a 3-year progression free survival rate of 84%. However, tumor regeneration may occur after discontinuation of the medication, and individualized evaluation of the treatment cycle is required.
4. Medication characteristics
As the first oral MEK inhibitor, the twice daily dosing regimen is convenient for home treatment. It should be taken on an empty stomach to optimize absorption, and the recommended starting dose is 25mg/m ²/time.
5. Limitations
Completely resected PN has no preventive effect and is not suitable for asymptomatic patients. Some patients may develop drug resistance and require monitoring of tumor progression and timely adjustment of treatment plans.
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