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Role of Sirolimus in Advanced Kaposiform Hemangioendothelioma
Vikash S Oza1, Mark D Mamlouk2, Christopher P Hess2
1Ronald O. Perelman Department of Dermatology, New York University School of Medicine, New York, New York.
Abstract:
Kaposiform hemangioendothelioma (KHE) is an infiltrative vascular tumor that classically presents in infancy. Management typically focuses on treating Kasabach-Merritt phenomenon (KMP), a disorder of severe and at times life-threatening platelet trapping. However, the morbidity of KHE extends beyond KMP. The infiltrative nature of the tumor can lead to long-term disability and often makes complete surgical resection impossible. We report the case of a 10-year-old boy with a KHE of his right distal thigh who was unable to walk without assistance due to fibrotic change and right knee contracture. He had no laboratory evidence of KMP at the time of representation. Rapamycin was started in hopes of reducing the tumor burden. Within 2 months of therapy, fibrotic areas softened, his contracture nearly resolved, and there was marked improvement in his mobility. Rapamycin has been previously reported to be effective in managing cases of KHE complicated by KMP. Our report emphasizes the role for rapamycin in the treatment of KHE in the absence of KMP through the inhibition of vasculogenesis and fibrotic pathways.
Insights
Rapamycin effectively treated Kaposiform hemangioendothelioma (KHE) in a child without Kasabach-Merritt phenomenon (KMP). The treatment improved mobility by reducing tumor burden and fibrotic changes.
Area of Science:
- Vascular biology
- Pediatric oncology
- Pharmacology
Background:
- Kaposiform hemangioendothelioma (KHE) is an infiltrative vascular tumor typically seen in infants.
- Management often targets Kasabach-Merritt phenomenon (KMP), a serious complication involving platelet trapping.
- KHE's infiltrative nature can cause long-term disability and surgical challenges.
Observation:
- A 10-year-old boy presented with KHE in his right thigh, causing mobility issues due to fibrosis and knee contracture.
- He showed no signs of KMP at the time of presentation.
- Rapamycin treatment was initiated to reduce tumor burden.
Findings:
- Within 2 months, rapamycin therapy softened fibrotic areas and resolved the knee contracture.
- Significant improvement in the patient's mobility was observed.
- This suggests rapamycin's efficacy beyond KMP management.
Implications:
- Rapamycin may be a viable treatment for KHE even in the absence of KMP.
- The drug's mechanism may involve inhibiting vasculogenesis and fibrotic pathways.
- This case highlights rapamycin's potential to address KHE-related morbidity and functional impairment.
