Low dose sirolimus treatment for refractory tufted angioma and congenital kaposiform hemangioendothelioma, both with Kasabach‐Merritt phenomenon

Mariani, Letícia Gaertner; Schmitt, Ismael R.; Garcia, Clotilde Druck; Kiszewski, Ana Elisa

doi:10.1002/pbc.27810

Cited by 15 publications

(17 citation statements)

References 22 publications

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“…Many authors have reported maintaining the serum levels between 8 and 15 ng/ml [114,116,120]. However, low-dose sirolimus (2-3 ng/ml serum levels) may be associated with low toxicity and has been demonstrated to be effective for treating patients without KMP [127]. Interestingly, there is mounting evidence that low-dose sirolimus markedly ameliorates the development of inflammation and fibrogenesis in animal models, providing a theoretical basis for its use in KHE with musculoskeletal disorders [128,129].…”

Section: Sirolimusmentioning

confidence: 99%

Kaposiform hemangioendothelioma: current knowledge and future perspectives

Chen

Yang

et al. 2020

Orphanet J Rare Dis

111

157

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Kaposiform hemangioendothelioma (KHE) is a rare vascular neoplasm with high morbidity and mortality. The initiating mechanism during the pathogenesis of KHE has yet to be discovered. The main pathological features of KHE are abnormal angiogenesis and lymphangiogenesis. KHEs are clinically heterogeneous and may develop into a lifethreatening thrombocytopenia and consumptive coagulopathy, known as the Kasabach-Merritt phenomenon (KMP). The heterogeneity and the highly frequent occurrence of disease-related comorbidities make the management of KHE challenging. Currently, there are no medications approved by the FDA for the treatment of KHE. Multiple treatment regimens have been used with varying success, and new clinical trials are in progress. In severe patients, multiple agents with variable adjuvant therapies are given in sequence or in combination. Recent studies have demonstrated a satisfactory efficacy of sirolimus, an inhibitor of mammalian target of rapamycin, in the treatment of KHE. Novel targeted treatments based on a better understanding of the pathogenesis of KHE are needed to maximize patient outcomes and quality of life. This review summarizes the epidemiology, etiology, pathophysiology, clinical features, diagnosis and treatments of KHE. Recent new concepts and future perspectives for KHE will also be discussed.

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Section: Sirolimusmentioning

confidence: 99%

Kaposiform hemangioendothelioma: current knowledge and future perspectives

Chen

Yang

et al. 2020

Orphanet J Rare Dis

111

157

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“…Sirolimus has shown excellent prospects for the treatment of hemangioma accompanied by KMS. [ 2 , 5 ] After 1-year treatment with sirolimus, the infant in this study showed stable vital signs, with all laboratory indicators as well as his pancreas size returning to normal. Therefore, sirolimus is effective in the treatment of hemangioma with KMS, but further prospective trials are needed to assess the efficacy of this drug and to determine the scientific therapeutic dose and duration of treatment.…”

mentioning

confidence: 66%

“…It is characterized by thrombocytopenia and intravascular coagulation that occasionally develops into disseminated intravascular coagulation. [ 1 , 2 ] A rare incidence of diffuse infiltrative pancreatic hemangioma with a high possibility of missed diagnosis of KMS makes it a fatal disease. Here, we described a case diagnosed with diffuse infiltrative hemangioma (DIH) of pancreas accompanied by KMS and provided ultrasonic data for early and accurate diagnosis, and successful treatment experiences with sirolimus.…”

mentioning

confidence: 99%

Diffuse infiltrative hemangioma of pancreas accompanied by Kasabach-Merritt Syndrome: A case report

Zhang

Luo

Wang

2020

Chinese Medical Journal

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“…No hay reportes de afectos adversos graves. Aunque se obtuvo una respuesta positiva con toxicidad limitada en los casos publicados, no existe una estandarización de los criterios de respuesta ni de toxicidad (Tabla 1) [29][30][31][32][33][34][35][36][37] . Los HEK son tumores vasculares infrecuentes y localmente agresivos en la edad pediátrica 1 .…”

Section: Discussionunclassified

Hemangioendotelioma kaposiforme refractario en población pediátrica: reporte de caso y revisión de la literatura

Gómez-Villegas¹,

Pérez-Téllez²,

Ochoa-Gaviria³

et al. 2021

BMHIM

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Introducción: El hemangioendotelioma kaposiforme (HEK) es un tumor vascular poco frecuente caracterizado por una invasión local agresiva y un síndrome de atrapamiento de plaquetas conocido como fenómeno de Kasabach-Merritt. Aunque muchos casos de HEK se tratan con éxito con control local o quimioterapia de baja intensidad, otros son resistentes y se cuenta con pocas opciones terapéuticas. El objetivo de este reporte es mostrar la experiencia del tratamiento con sirolimus por vía oral en un paciente pediátrico con HEK asociado a fenómeno de Kasabach-Merritt refractario al tratamiento de primera línea, quien mostró excelente respuesta al tratamiento. Caso clínico: Paciente de sexo masculino de 3 meses con un HEK refractario al manejo de primera línea (corticoides, propranolol, vincristina), sin posibilidad de hacer control local, por lo que se decide terapia combinada con sirolimus, presentando control local y resolución de la coagulopatía desde la primera semana de iniciado el manejo y con resolución de la malformación vascular después de 12 meses de seguimiento. Conclusiones: Aunque no existen pautas claras para el tratamiento del HEK refractario en la edad pediátrica, la evidencia actual demuestra que el sirolimus es un medicamento eficaz que puede ser considerado como opción terapéutica de primera línea en estos pacientes.

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Low dose sirolimus treatment for refractory tufted angioma and congenital kaposiform hemangioendothelioma, both with Kasabach‐Merritt phenomenon

Cited by 15 publications

References 22 publications

Kaposiform hemangioendothelioma: current knowledge and future perspectives

Kaposiform hemangioendothelioma: current knowledge and future perspectives

Diffuse infiltrative hemangioma of pancreas accompanied by Kasabach-Merritt Syndrome: A case report

Hemangioendotelioma kaposiforme refractario en población pediátrica: reporte de caso y revisión de la literatura

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