Dermatologic Effects of Selumetinib in Pediatric Patients with Neurofibromatosis Type 1: Clinical Challenges and Therapeutic Management

    March 2024 in “ Journal of Clinical Medicine
    Paola Borgia, Gianluca Piccolo, Andrea Santangelo, Cristina Chelleri, Gianmaria Viglizzo, C. Occella, Carlo Minetti, Pasquale Striano, Maria Cristina Diana
    TLDR Selumetinib causes skin and hair side effects in kids with NF1, affecting treatment and quality of life.
    The study on selumetinib in 20 pediatric patients with Neurofibromatosis Type 1 (NF1) reveals significant dermatologic adverse effects, including xerosis, paronychia, acneiform rash, and hair abnormalities, impacting treatment adherence and quality of life. Xerosis affected 70% of patients, acneiform rash 55%, and hair abnormalities 70%, with females more prone to hair issues. These side effects led to treatment suspension in 20% of cases, causing tumor rebound in 75% of those. The study underscores the need for tailored management strategies to ensure effective treatment, highlighting the importance of addressing these adverse effects to maintain therapy efficacy and patient quality of life.
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