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    Case Report: Safety and Efficacy of Denosumab in Four Children With Noonan Syndrome With Multiple Giant Cell Lesions of the Jaw

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    Author
    Ferriero, Kristen
    Shah, Biraj
    Yan, Yun
    Khatri, Surya
    Caccamese, John
    Napoli, Joseph A.
    Bober, Michael B.
    Crane, Janet L.
    Date
    2020-09-18
    Journal
    Frontiers in Pediatrics
    Publisher
    Frontiers Media S.A.
    Type
    Article
    
    Metadata
    Show full item record
    See at
    https://doi.org/10.3389/fped.2020.00515
    Abstract
    Noonan syndrome is a genetic disorder caused by mutations in the RAS/MAPK pathway. Multiple giant cell lesions are a rare sequelae of disruptions in this pathway, termed Noonan-like multiple giant cell lesions (NL/MGCLs). Medical management of these tumors rather than surgical intervention is preferential as the lesions are benign but locally destructive and recurring. This case series describes four male pediatric patients with Noonan syndrome and multiple giant cell lesions of the jaw treated with denosumab, a monoclonal antibody to receptor activator of nuclear factor kappa B ligand (RANKL), which has been approved for the treatment of malignant giant cell tumors in adults but not evaluated for safety or efficacy in children. All four pediatric patients responded clinically and radiographically to the treatment. Adverse events occurred in a predictable pattern and included hypocalcemia and joint pain during the initiation of treatment and symptomatic hypercalcemia after the cessation of treatment. Growth was not significantly impaired in these skeletally immature patients. This case series demonstrates how a weight-adjusted denosumab dose can effectively treat NL/MGCLs and provides laboratory data for consideration of the timing of monitoring for known side effects.
    Sponsors
    National Institutes of Health
    Keyword
    child
    denosumab
    jaw
    multiple giant cell lesions
    Noonan syndrome
    Identifier to cite or link to this item
    http://hdl.handle.net/10713/13859
    ae974a485f413a2113503eed53cd6c53
    10.3389/fped.2020.00515
    Scopus Count
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