Successful management of steroid‐resistant vascular tumors associated with the Kasabach–Merritt phenomenon using sirolimus

Tan, Xiaohong; Zhang, Jing; Zhou, Shaoyi; Liu, Zhenyin; Zhang, Tao; Xià, Jì

doi:10.1111/1346-8138.14231

Cited by 22 publications

(31 citation statements)

References 15 publications

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“…The 15 studies described 202 cases of KHE. Of the 15 studies, 5 studies chose vincristine as the definitive treatment . The remaining 10 studies used sirolimus …”

Section: Resultsmentioning

confidence: 99%

“…In terms of the safety of sirolimus, the side effects have been described in detail in eight studies and include bronchitis (1/25), lymphopenia (1/25), elevation of AST and ALT (3/25), hyperlipidaemia (3/25), opportunistic infection (2/25), mild reversible leukopenia (2/25), mucositis (7/25), and platelet elevation (1/25), fever (1/25), pain (1/25), skin rash/vomiting (1/25) and diarrhoea (1/25) . There were no instances of toxicity‐related permanent treatment discontinuation or drug‐related deaths (Table ).…”

Section: Resultsmentioning

confidence: 99%

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Vincristine and sirolimus in the treatment of kaposiform haemangioendothelioma

Peng

Yang

et al. 2019

J Paediatrics Child Health

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Aim Kaposiform haemangioendothelioma (KHE) is a rare, potentially life‐threatening vascular tumour that is often associated with thrombocytopenia and coagulopathy, known as the Kasabach‐Merritt phenomenon (KMP). Because of the rarity and complexity of KHE, the optimal paradigm for treating KHE has yet to be elucidated. We aim to assess the efficacy and safety of vincristine and sirolimus for the treatment of KHE. Methods A comprehensive review of the literature was conducted from January 1993 to June 2018. A total of 15 studies were selected for the meta‐analysis. Five studies included 75 individuals and reported the response and side effects to vincristine in the treatment of KHE with or without KMP. A total of 10 studies that included 127 individuals reported the response and safety of sirolimus for treating KHE with or without KMP. Results The pooled odds ratio (OR) for the effectiveness of vincristine was 0.72. The pooled OR for the effectiveness of sirolimus was 0.91. The side effects associated with vincristine during the treatment included neuropathy, abdominal pain, loss of appetite and mild elevations of aspartate aminotransferase (AST) and alanine aminotransferase (ALT). The side effects associated with sirolimus therapy included bronchitis; lymphopenia; elevated AST, ALT and platelets; hyperlipidaemia; opportunistic infection; mild reversible leukopenia; mucositis; fever; pain and skin rash/vomiting and diarrhoea. Conclusions This systematic review showed a high efficacy of vincristine and sirolimus in the treatment of KHE. Based on the available data in the literature, it appears that sirolimus is potentially an efficacious and safe treatment option for KHE. Further randomised, controlled trials are recommended.

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“…The 15 studies described 202 cases of KHE. Of the 15 studies, 5 studies chose vincristine as the definitive treatment . The remaining 10 studies used sirolimus …”

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Vincristine and sirolimus in the treatment of kaposiform haemangioendothelioma

Peng

Yang

et al. 2019

J Paediatrics Child Health

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“…3,4 KMP mainly appears in newborns and infants, with mortality estimated at 10-30%. 5,6 We present two cases of refractory KMP that improved with low-dose of sirolimus.…”

Section: Low Dose Sirolimus Treatment For Refractory Tufted Angioma Amentioning

confidence: 99%

“…[11][12][13][14][15][16][17][18] The optimal sirolimus dose in patients with KMF has not been established, but serum levels should be maintained between 5 and 15 ng/mL. 5,11,15 Low-dose sirolimus, using levels of 2-3 ng/mL, is associated with low toxicity. 19,20 Sirolimus is highly effective and easy to use; however, its cost may limit the use.…”

Section: Low Dose Sirolimus Treatment For Refractory Tufted Angioma Amentioning

confidence: 99%

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

Mariani¹,

Schmitt²,

Garcia

et al. 2019

Pediatric Blood & Cancer

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Splenic kaposiform hemangioendothelioma presenting as insidious consumptive coagulopathy

et al. 2021

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A healthy 2-year-old male with a history of mild persistent asthma visited the outpatient hematology clinic to evaluate intermittent epistaxis. There was no history of recent illness. His exam was notable for left upper extremity bruising secondary to recent venipuncture, but no lymphadenopathy or hepatosplenomegaly. Complete blood count (CBC) revealed hemoglobin 9.3 g/dL, leukocyte count 20.7 10 9 /L, and platelet count 33 k 10 9 /L. Peripheral blood smear exhibited polychromasia, numerous large platelets, and a few reactive lymphocytes.The differential diagnosis for both thrombocytopenia and anemia include processes leading to impaired hematopoiesis, increased cell destruction, or abnormal cell pooling. Impaired hematopoiesis could stem from infectious suppression, infiltrative processes (leukemia, metabolic disorders, etc.), or inherited and acquired bone marrow failure syndromes. Possible destructive or consumptive processes included microangiopathy, disseminated intravascular coagulation (DIC), or immune destruction. Sequestration within the liver or spleen was considered, but he was without hepatosplenomegaly to suggest a site of abnormal pooling. We approached his work-up with a stepwise approach. Screening tests were normal for common infectious diseases implicated in marrow suppression (Table 1). Since the patient appeared well and did not exhibit any findings that would prompt an immediate evaluation for malignancy or sepsis, we decided to repeat a short interval CBC to see if his cytopenias self-resolved. When they did not, further evaluation of the bone marrow was warranted. Bone marrow biopsy and aspirate did not demonstrate marrow infiltration or failure (Table 1). Lab work was sent to investigate consumptive processes. Lactate dehydrogenase levels and reticulocyte counts were elevated (Table 1), suggesting a hemolytic process. A hemoglobinopathy screen and glucose-6-phosphate dehydrogenase testing were normal, though active hemolysis can skew results. In an ill-appearing child, the presence of hemolysis and thrombocytopenia should prompt an investigation for DIC or microangiopathy, but in this well appearing-child, an immune process was thought likelier. We, therefore, pursued an immunologic work-up, including screening for autoimmune lymphoproliferative syndrome (ALPS) (Table 1). The results were unrevealing except for a low IgG level at 228 (505-1280 mg/dL) with normal lymphocyte subsets.Low IgG is often associated with systemic autoimmune disorders, vasculitides, atopy, and asthma. We attributed the low IgG and eosinophilia to the patient's known history of asthma. A consumptive autoimmune process was high on the differential, and the involvement of two cell lines would be classified as Evans syndrome. Evans syndrome, though never a primary diagnosis, is treated upfront with

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Successful management of steroid‐resistant vascular tumors associated with the Kasabach–Merritt phenomenon using sirolimus

Cited by 22 publications

References 15 publications

Vincristine and sirolimus in the treatment of kaposiform haemangioendothelioma

Vincristine and sirolimus in the treatment of kaposiform haemangioendothelioma

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

Splenic kaposiform hemangioendothelioma presenting as insidious consumptive coagulopathy

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