Medical Information

Selumetinib is a MEK inhibitor mainly used to treat type 1 neurofibromatosis related plexiform neurofibromatosis. Its common side effects include skin reactions, gastrointestinal symptoms, and cardiac toxicity, which require close monitoring of adverse reactions and individualized adjustment of medication regimens.

1. Indications

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 accelerated approval of objective response rate (ORR) and duration of response (DOR), and further validation of clinical benefits is required.

2. Common side effects

(1) Skin reaction

Including rash (80%), acne like dermatitis (63%), and paronychia (36%), mostly of grade 1-2. Keep the skin clean and moist, and suspend or reduce medication in severe cases.

(2) Gastrointestinal symptoms

Common diarrhea (70%), nausea (50%), and vomiting (40%) can be alleviated through dietary adjustments. Symptoms above level 3 require interruption of treatment until recovery, followed by a 25% reduction in dosage and re administration.

(3) Cardiac toxicity

May cause a decrease in left ventricular ejection fraction (15%) and hypertension (10%). Assess cardiac function before medication and regularly monitor blood pressure and electrocardiogram during treatment.

3. Serious side effects

Including retinal vein occlusion (1.4%), cardiomyopathy (1.1%), and rhabdomyolysis (0.7%). Seek medical attention immediately if there are changes in vision, chest pain, or muscle pain. Serious adverse reactions require permanent discontinuation of medication.

4. Medication for special populations

(1) Child patients

Children over 2 years old should be administered according to their body surface area (25mg/m ² bid). Monitoring of growth and development indicators is necessary, and attention should be paid to the toxicity risk of bone growth plates.

(2) Liver and kidney dysfunction

Mild liver damage does not require dose adjustment, but there is a lack of data for moderate to severe cases. Mild kidney damage (GFR ≥ 60) does not require adjustment, moderate to severe cases should be used with caution.

5. Medication management

It should be taken on an empty stomach (2 hours before or 1 hour after meals) and swallowed whole. Skip the dose if missed for more than 6 hours. Avoid receiving live vaccines during treatment and regularly monitor CPK levels.