Selumetinib is the first and only drug approved in China for the treatment of pediatric patients with neurofibromatosis type 1 (NF1) with symptomatic and inoperable plexiform neurofibromas (PN), providing a new treatment option for NF1 patients.

The efficacy of the neurofibromatosis type 1 drug Selumetinib and the management of precautions

Inhibition of MEK1/2

As a highly efficient and selective, non-ATP-competitive MEK1/2 inhibitor, Selumetinib can selectively inhibit MEK1 and MEK2, thereby normalizing the dysregulated signaling pathway and alleviating the condition of NF1 patients.

Anti-tumor effect

Selumetinib blocks the signaling pathway related to tumor growth by inhibiting MEK1/2, thereby inhibiting the growth and spread of tumor cells.

Potential for the treatment of other tumors

Although Selumetinib is currently mainly used for the treatment of NF1, it has also shown certain efficacy against other tumors, such as low-grade serous ovarian cancer and BRAFV600E-mutant melanoma.

Trial efficacy

In a phase II clinical trial, Selumetinib monotherapy achieved an objective response rate of 66% in pediatric patients with NF1 and symptomatic, inoperable plexiform neurofibromas. Eighty-two percent of children had a duration of remission of 12 months or longer. In studies conducted in China, Selumetinib has shown good efficacy and safety in the treatment of NF1-associated plexiform neurofibromas, providing a new treatment option for NF1 patients.

Precautions

Heart problems

Ejection fraction should be assessed before starting treatment, every 3 months in the first year, and every 6 months thereafter, and according to the clinical situation.

Ophthalmotoxicity

Before starting Selumetinib, regular ophthalmologic evaluations should be performed during treatment to check for new or worsening visual changes.

Gastrointestinal toxicity

It is recommended that patients start using antidiarrheal drugs immediately after the first occurrence of loose stools and increase fluid intake.

Skin toxicity

monitor severe rashes, and depending on the severity of adverse reactions, it may be necessary to discontinue the drug, reduce the dose, or permanently discontinue Selumetinib.

Creatine phosphokinase (CPK) increase

Serum CPK levels should be monitored regularly before, during and clinically, and rhabdomyolysis or other causes should be evaluated if CPK elevation occurs.

Vitamin E levels and bleeding risk

Selumetinib capsules contain vitamin E, and excessive amounts may increase the risk of bleeding, especially when used in combination with vitamin K antagonists or antiplatelet drugs.

Storage

Selumetinib should be stored at room temperature, with a specific temperature range of 15°C to 30°C (59°F to 86°F). Direct exposure of the drug to sunlight or other strong light sources should be avoided in case the composition of the drug changes. It needs to be placed out of the reach of children to prevent accidental ingestion or improper contact by children. It is recommended to store in the original bottle and keep the desiccant included in the original package to avoid moisture. Follow the expiration date instructions on the package and do not use expired medicines.

Selumetinib has significant efficacy in the treatment of patients with NF1-related plexiform neurofibroma, and it is important to follow the doctor's guidance when using Selumetinib and maintain communication with the medical team during the treatment to ensure the safety and efficacy of the treatment. If you have any adverse reactions or other questions, you should consult your doctor promptly.