Topical rapamycin (sirolimus) for the treatment of uncomplicated tufted angiomas in two children and review of the literature

Burleigh, Angela; Kanigsberg, Nordau; Lam, Joseph M.

doi:10.1111/pde.13596

Cited by 13 publications

(9 citation statements)

References 33 publications

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“…Three retrospective studies described the benefits of topical sirolimus in the treatment of tufted angiomas. 10,50,51 A decrease in lesion size was reported for all patients, but no clinical benefit was described for the infantile hemangioma.…”

Section: Vascular Tumorsmentioning

confidence: 93%

Efficacy and safety of sirolimus in the treatment of vascular anomalies: A systematic review

Freixo

Ferreira

Martins

et al. 2020

Journal of Vascular Surgery

142

115

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Objective: The management of vascular anomalies is complex and requires a multidisciplinary team with a combination of medical, surgical, and intervention treatments. Medical treatment is limited and has conflicting results. Off-label use of mammalian target of rapamycin inhibitors shows promising results. The objective of this study was to systematically evaluate the literature published about the efficacy and safety of sirolimus in the treatment of vascular anomalies. Methods: A systematic review of the published literature was conducted using the PubMed database and Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. Results: There were 73 articles included: 2 randomized controlled studies, 2 nonrandomized prospective studies, and 69 retrospective case reports and case series. In total, 373 patients were included. Sirolimus was administered topically to 56 patients and orally to 317 patients. Sirolimus was highly effective in the treatment of vascular tumors associated with Kasabach-Merritt phenomenon (95.5% of the patients clinically improved and 93% had normalization of coagulopathy), venous malformations (size reduction was observed in 88.9% of patients), and lymphatic malformations (clinical improvement in 94.9% of patients). Topical sirolimus results were conflicting. Arteriovenous malformations were not improved by sirolimus. Conclusions: Low-level evidence suggests that sirolimus can improve the prognosis of vascular anomalies, most notably vascular tumors associated with life-threatening coagulopathy and venous and lymphatic malformations. Further research is needed to establish the benefits of sirolimus in the management of vascular anomalies.

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Section: Vascular Tumorsmentioning

confidence: 93%

Efficacy and safety of sirolimus in the treatment of vascular anomalies: A systematic review

Freixo

Ferreira

Martins

et al. 2020

Journal of Vascular Surgery

142

115

View full text Add to dashboard Cite

show abstract

“…Several case reports and case series have reported success using topical sirolimus and tacrolimus ointment in superficial KHE/TA. The investigators have shown a good response of KHE/TA to twice daily topical application of these drugs [130,131]. Tacrolimus is an anti-T-cell immunosuppressive drug that is FDA-approved as a topical gel formulation (available in a concentration of 0.03 and 0.1%) for the treatment of cutaneous inflammatory/fibrotic diseases [132].…”

Section: Topical Treatmentsmentioning

confidence: 99%

Kaposiform hemangioendothelioma: current knowledge and future perspectives

Chen

Yang

et al. 2020

Orphanet J Rare Dis

120

180

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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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“…Therefore, vincristine is now primarily preferred for refractory or recurrent cases. Topical sirolimus or tacrolimus have been shown in small studies to be beneficial for superficial cutaneous lesions but not for KHE with KMP 33,34 . Antiplatelet therapy such as aspirin or ticlopidine in combination with vincristine have also shown success 35–38 .…”

Section: Case 1: Khe With Kmpmentioning

confidence: 99%

How we approach coagulopathy with vascular anomalies

Mack

Crary

2022

Pediatric Blood & Cancer

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Some vascular anomalies can present with challenging hematologic aberrations.Kaposiform hemangioendothelioma (KHE) may be complicated with Kasabach-Merritt phenomenon (KMP) and stagnant blood flow in slow-flow malformations can promote activation and consumption of coagulation factors, which results in bleeding and clotting known as localized intravascular coagulopathy (LIC). These patients can experience significant morbidity secondary to pain due to thrombosis and are at higher risk of hematologic complications during surgical procedures. No standard of care has been established to prevent or manage these complications. This review focuses on the management of coagulopathy in children and adults with vascular anomalies.

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Topical rapamycin (sirolimus) for the treatment of uncomplicated tufted angiomas in two children and review of the literature

Cited by 13 publications

References 33 publications

Efficacy and safety of sirolimus in the treatment of vascular anomalies: A systematic review

Efficacy and safety of sirolimus in the treatment of vascular anomalies: A systematic review

Kaposiform hemangioendothelioma: current knowledge and future perspectives

How we approach coagulopathy with vascular anomalies

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