Medical Management of Tumors Associated With Kasabach-Merritt Phenomenon

Tlougan, Brook E.; Lee, Margaret T.; Drolet, Beth A.; Frieden, Ilona J.; Adams, Denise M.; Garzon, María C.

doi:10.1097/mph.0b013e318298ae9e

Cited by 56 publications

(65 citation statements)

References 18 publications

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“…5 Our stablished protocol for KMP involves a combination of chemotherapy with vincristine (0.05 mg/kg given weekly) and double antiaggregation with ticlopidine (10 mg/kg/day) and aspirin (10 mg/kg/day). 6 7 Sirolimus treatment in cutaneous KHE has been described as successful in the literature, as well as in our own experience; however, it failed in our patient with visceral KHE. 8 9 Side effects of medical therapy and recurrence rates of KHE are not described well; therefore, long-term follow-up is mandatory in these patients.…”

Section: Discussionsupporting

confidence: 47%

Pancreatic Kaposiform Hemangioendothelioma Not Responding to Sirolimus

Triana

Dore

Nuñez

et al. 2017

European J Pediatr Surg Rep

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Background Kaposiform hemangioendothelioma (KHE) is a vascular tumor frequently associated with Kasabach–Merritt phenomenon (KMP), characterized by severe thrombocytopenia and consumptive coagulopathy. Visceral involvement in KHE is rare. In our recent experience, sirolimus has shown to be an effective treatment in cutaneous KHE, becoming indeed the treatment of choice in KMP. We report a case of pancreatic KHE associated with KMP and refractory to sirolimus. Case Report A 4-month-old infant is referred for obstructive jaundice (10 mg/dL conjugated bilirubin) secondary to vascular pancreatic tumor. Magnetic resonance (MR) and immunohistochemistry were compatible with KHE, but the tumor was considered unresectable. We initiated sirolimus (0.8 mg/m 2 /12 h) to treat KMP, and interventional radiology was performed for percutaneous biliary diversion. This procedure prompted KMP (platelets: 51,000/µL). Sirolimus treatment for 7 days showed no effect; therefore, we started our VAT protocol (vincristine/aspirine/ticlopidin) with great response after 10 days (platelets: 3,70,000/µL). Three months later, percutaneous biliary diversion was replaced by a biliary stent. The tumor disappeared leaving fibrosis and dilatation of biliary tract needing hepaticojejunostomy 6 months later. Discussion It is difficult to establish protocols for an unusual presentation of a tumor with different targets. This is a reason collaborative multicenter studies should be performed. Management of obstructive jaundice secondary to a tumor that usually regresses in 10 years is an added challenge; therefore, the management should be led by a multidisciplinary team. Sirolimus treatment in cutaneous KHE has been described as successful in the literature, as well as in our own experience; however, it failed in our first patient with visceral KHE. We need to investigate the different response to pharmacological agents in tumors with similar histopathology, but with visceral involvement.

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Section: Discussionsupporting

confidence: 47%

Pancreatic Kaposiform Hemangioendothelioma Not Responding to Sirolimus

Triana

Dore

Nuñez

et al. 2017

European J Pediatr Surg Rep

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“…Coagulopathy and thrombocytopenia in KHE are typically associated with more aggressive presentations and poorer outcomes. Therefore, the chosen treatment regimen should be based on the individual patient and his or her treatment response . In cases of active bleeding, severe coagulopathy and/or thrombocytopenia, fresh frozen plasma and/or cryoprecipitate can be used in the management of KMP.…”

Section: Discussionmentioning

confidence: 99%

“…Consensus treatment guidelines from a multidisciplinary expert panel were published in 2013. Medical treatments with corticosteroids and/or vincristine have been recommended for the management of KHE . However, first‐line treatment with corticosteroids is successful in only 10–27% of all cases, and treatment with vincristine is successful in 60–70% of patients .…”

mentioning

confidence: 99%

Sirolimus for the treatment of progressive kaposiform hemangioendothelioma: A multicenter retrospective study

Chen

Xiang

et al. 2017

Intl Journal of Cancer

112

126

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Kaposiform hemangioendothelioma (KHE) is an aggressive disease with high morbidity and mortality. The aim of this study was to retrospectively evaluate the efficacy and safety of sirolimus for the treatment of progressive KHE. A multicenter, retrospective cohort study was conducted in patients with progressive KHE treated with sirolimus. A total of 52 patients were analyzed. Thirty-seven (71%) patients exhibited Kasabach-Merritt phenomenon (KMP) and were significantly younger than the patients without KMP [95% confidence interval (CI), 14.39-41.61; p < 0.001]. Patients without KMP were all treated with sirolimus alone, whereas 21 KMP patients with severe symptoms received short-term combination therapy with prednisolone. Overall, 96% and 98% of patients showed improved relief of notable symptoms and/or improved complications at 6 and 12 months after treatment, respectively. After sirolimus treatment, significant decreases in mean severity scores occurred at 6 months (95% CI, 2.23-2.54, p < 0.001) and 12 months (95% CI, 1.53-1.90, p < 0.001). Compared to KMP patients, patients without KMP showed a response that was similar to but less pronounced during the 12 months of treatment (95% CI, 40.87-53.80; p < 0.001). For subgroup analysis of KMP patients, there were no significant differences in tumor shrinkage between those treated with combination therapy and those receiving sirolimus alone (95% CI, 18.11-25.02; p > 0.05). No patients permanently discontinued treatment due to toxicity-related events, and no drug-related deaths occurred. Sirolimus was effective and safe for the treatment of progressive KHE. Sirolimus may be considered as a first-line therapy or as part of a multidisciplinary approach for the treatment of KHE.

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“…27 The group recommends a regimen of systemic corticosteroids and weekly vincristine as standard of care for KHE associated with KMP. 27,28 …”

Section: Managementmentioning

confidence: 99%

Kasabach-Merritt Phenomenon: Classic Presentation and Management Options

Mahajan

Margolin

Iacobas

2017

Clinical Medicine Insights: Blood Disorders

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Kasabach-Merritt phenomenon (KMP) is a rare consumptive coagulopathy associated with specific vascular tumors, kaposiform hemangioendothelioma, and tufted angioma. Kasabach-Merritt phenomenon, characterized by profound thrombocytopenia, hypofibrinogenemia, elevated fibrin split products, and rapid tumor growth, can be life-threatening. Severe symptomatic anemia may also be present. With prompt diagnosis and management, KMP can resolve and vascular tumors have been shown to regress. This review highlights the clinical presentation, histopathology, management, and treatment of KMP associated with kaposiform hemangioendothelioma, and less frequently tufted angioma. A classic clinical case is described to illustrate the presentation and our management of a patient with KMP.

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Medical Management of Tumors Associated With Kasabach-Merritt Phenomenon

Cited by 56 publications

References 18 publications

Pancreatic Kaposiform Hemangioendothelioma Not Responding to Sirolimus

Pancreatic Kaposiform Hemangioendothelioma Not Responding to Sirolimus

Sirolimus for the treatment of progressive kaposiform hemangioendothelioma: A multicenter retrospective study

Kasabach-Merritt Phenomenon: Classic Presentation and Management Options

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