Sirolimus for treatment of Kaposiform haemangioendothelioma with Kasabach-Merritt phenomenon: a retrospective cohort study

Zhang, G.; Chen, H.; Gao, Ya; Liu, Y.; Wang, J.; Liu, X.Y.

doi:10.1111/bjd.16400

Cited by 17 publications

(16 citation statements)

References 7 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%

“…A total of 15 studies 3,6,11,12,14,[17][18][19][20][21][22][23][24][25][26] met the final inclusion criteria and were selected for meta-analysis (Fig. 1).…”

Section: Eligible Studies and Study Characteristicsmentioning

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%

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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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“…6,9,10 Currently, sirolimus alone or combined with a short course of oral corticosteroids appears to be the best therapeutic option. [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.…”

Section: Low Dose Sirolimus Treatment For Refractory Tufted Angioma Amentioning

confidence: 99%

“…A 2013 consensus recommended steroids associated with vincristine for KMP . Currently, sirolimus alone or combined with a short course of oral corticosteroids appears to be the best therapeutic option . The optimal sirolimus dose in patients with KMF has not been established, but serum levels should be maintained between 5 and 15 ng/mL.…”

mentioning

confidence: 99%

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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Long‐term outcomes of sirolimus treatment for kaposiform hemangioendothelioma: Continuing successes and ongoing challenges

Zhou

Qiu

et al. 2023

Intl Journal of Cancer

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Treatment with sirolimus, an inhibitor of the mammalian target of rapamycin pathway, has improved the prognosis of patients with kaposiform hemangioendothelioma (KHE). However, the efficacy, durability and tolerability of long-term sirolimus treatment in patients with KHE have not been well elucidated. We performed efficacy and safety assessments based on more than 4.5 years of follow-up in patients receiving sirolimus therapy for KHE. One hundred sixtyseven patients were analyzed, including 102 (61.1%) patients with the Kasabach-Merritt phenomenon (KMP). Follow-up was conducted after a median of 56.0 months. A total of 154 (92.2%) patients had a durable response to sirolimus treatment. No difference in durable response was found between patients without KMP and patients with KMP (95.4% vs 90.2%; difference, 5.2%; 95% confidence interval [CI], À4.0% to 13.1%). Rebound growth occurred in 17.3% of patients upon sirolimus discontinuation. Early treatment discontinuation (odds ratio [OR]: 3.103; 95% CI: 1.529-6.299; P = .002) and mixed lesion type (OR: 2.271; 95% CI: 0.901-5.727; P = .047) were associated with tumor rebound growth. No KHE-related deaths occurred in this cohort. At the last follow-up, approximately 17.4% of patients had active disease and/or changes in body structures to a variable extent. Serious adverse events occurred most commonly during the first year of sirolimus therapy. Follow-up of almost 4.5 years demonstrated that the efficacy of sirolimus persisted over time and that long-term treatment Abbreviations: CI, confidence interval; KHE, kaposiform hemangioendothelioma; KMP, Kasabach-Merritt phenomenon; MRI, magnetic resonance imaging; mTOR, mammalian target of rapamycin; OR, odds ratio; ROM, range of motion.Jiangyuan Zhou, Yanan Li and Tong Qiu have contributed equally to this work.

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Sirolimus for treatment of Kaposiform haemangioendothelioma with Kasabach-Merritt phenomenon: a retrospective cohort study

Cited by 17 publications

References 7 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

Long‐term outcomes of sirolimus treatment for kaposiform hemangioendothelioma: Continuing successes and ongoing challenges

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