Oral sirolimus for the treatment of juvenile xanthogranuloma: Report of two pediatric cases

Juvenile xanthogranuloma (JXG) with extensive cutaneous or visceral organ involvement is often associated with high morbidity and treatment commonly involves surgical excision, radiotherapy, systemic steroids, or chemotherapy. Sirolimus, a mammalian target of rapamycin (mTOR) inhibitor, is an oral a...

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Published inPediatric dermatology
Main Authors Toker, Michelle, Hassonjee, Fatema Esaa, Amodio, John, Edelman, Morris C, Emeghebo, Kristina I, Levy, Carolyn Fein, Shaigany, Sheila
Format Journal Article
LanguageEnglish
Published United States 05.03.2024
Subjects
mammalian target of rapamycin (mTOR)
histiocytic disorders
juvenile xanthogranuloma (JXG)
pediatric dermatology
sirolimus
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Summary:Juvenile xanthogranuloma (JXG) with extensive cutaneous or visceral organ involvement is often associated with high morbidity and treatment commonly involves surgical excision, radiotherapy, systemic steroids, or chemotherapy. Sirolimus, a mammalian target of rapamycin (mTOR) inhibitor, is an oral antitumor and immunosuppressive therapy used to treat various neoplastic disorders, including histiocytic disorders. We report two pediatric cases of JXG successfully treated with oral sirolimus monotherapy, and postulate that sirolimus may induce rapid disease resolution and long-term remission for patients with both skin-limited and multisystemic JXG. Our findings warrant further investigation of the relationship between the mTOR pathway and JXG.
ISSN:0736-8046
1525-1470
DOI:10.1111/pde.15579