The U.S. FDA Approved Selumetinib (Koselugo) for the Treatment of Pediatric Patients 2 years of age and older with Neurofibromatosis Type 1 (NF1) and Symptomatic, Inoperable Plexiform Neurofibromas (PN).

Adverse reactions caused by Selumetinib bisulfate capsules

The adverse reactions of Selumetinib bisulfate capsules mainly include the following.

Digestive system reactions

The most common adverse reactions include vomiting, abdominal pain, diarrhea and nausea.

Skin reactions

including itching, rash, acne-like rash, dry skin and paronychia.

Systemic reactions

fatigue and fever are also common side effects.

Musculoskeletal system reactions

Musculoskeletal pain is also a common side effect.

Side effects of Selumetinib also include heart problems, ocular toxicity, headaches, stomatitis, etc. These side effects vary in severity, some are mild, while others may require medical intervention. When patients use Selumetinib bisulfate capsules, they should do it under the guidance of a doctor and report any adverse reactions in a timely manner so that appropriate dose adjustments or treatments can be made.

How to treat the adverse reactions caused by Selumetinib sulfate capsules?

For the side effects of Selumetinib bisulfate capsules, here are some treatments:

Diarrhea

It is recommended that patients start using antidiarrheal drugs immediately after the first occurrence of loose stools, and increase fluid intake, depending on the severity of adverse reactions, it may be necessary to discontinue the drug, reduce the dose, or permanently discontinue Selumetinib.

Nausea and vomiting

The diet should be light and easy to digest, and the doctor may prescribe anti-nausea drugs to control the symptoms.

Skin rash

Keep the skin clean and hygienic, and do not use harsh toiletries.

Cardiac 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. Depending on the severity of the adverse effects, discontinuation, dose reduction, or permanent discontinuation of Selumetinib may be required.

The above measures can help manage and alleviate the side effects of Selumetinib bisulfate capsules, but all treatments and adjustments should be made under the guidance of a doctor.

Efficacy of Selumetinib bisulfate capsules

In an open-label, multicenter, single-arm trial, paediatric patients with NF1 and measurable target PN were at risk for serious morbidity without surgical resection. Patients in the efficacy population (N=50) must also have at least one serious morbidity associated with the target PN. Patients receive 25mg of Selumetinib bisulfate capsules orally twice daily until disease progression or unacceptable toxicity.

The primary efficacy outcome measure is the overall response rate (ORR) as assessed by the NCI, defined as the percentage of patients with a ≥20% reduction in tumor volume over 3-6 months confirmed by subsequent MRI magnetic resonance imaging (MRI). The ORR was 66 percent, with all patients having a partial response, and 82 percent of responders had sustained remission for at least 12 months. An independent, focused review of ORR using the same mitigation criteria showed an ORR of 44%.