Journal
JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY
Volume 38, Issue 8, Pages E322-E325Publisher
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1097/MPH.0000000000000509
Keywords
mechanistic target of rapamycin (mTOR); Kasabach-Merritt phenomenon (KMP); kaposiform hemangioendothelioma (KHE); sirolimus; everolimus
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Funding
- Ministry of Education, Culture, Sports, Science and Technology of Japan [25461587]
- Health and Labour Science Research Grant for Research on Intractable Diseases from the Ministry of Health, Labour and Welfare of Japan
- Practical Research Project for Rare/Intractable Diseases from Japan's Agency for Medical Research and Development, AMED [15Aek0109057h0102]
- Grants-in-Aid for Scientific Research [16K10023, 25461587] Funding Source: KAKEN
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Kasabach-Merritt phenomenon (KMP) is a life-threatening consumptive coagulopathy associated with underlying kaposiform hemangioendothelioma (KHE) in infancy. We describe the case of a 3-month-old girl with KHE complicated by KMP who responded dramatically to treatment with everolimus, a mechanistic target of rapamycin (mTOR) inhibitor. Immunohistochemical expression of mTOR was found in the KHE biopsy specimens, which may explain the improvement of KMP and reduction in KHE tumor size with mTOR inhibitor treatment. This effective use of everolimus may shed light on the emerging role of mTOR signaling in the development and pathogenesis of KHE and KMP.
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