Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1 With MEK Inhibitor Selumetinib: A Nonrandomized Clinical Trial.

Summary: Scientists tested a drug called selumetinib to help adults with Neurofibromatosis Type 1 (NF1), a condition that causes bumpy skin tumors. These tumors can be painful, itchy, and embarrassing. Eleven patients took the medicine for up to two years. The good news is that the tumors shrank by about 28% for most people. The bad news is that the drug caused bothersome side effects like itchy rashes and dry skin. Because of these skin problems, many patients had to stop the treatment early. While the drug shrinks the tumors, the side effects make it difficult for people to keep taking it.