Rapamycin as Novel Treatment for Refractory-to-Standard-Care Slow-Flow Vascular Malformations

Boon, Laurence M.; Hammer, Jennifer; Seront, Emmanuel; Dupont, Sophie; Hammer, Frank; Clapuyt, Philippe; Vikkula, Miikka

doi:10.1097/01.prs.0000472322.66501.f6

Cited by 11 publications

(17 citation statements)

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“…Therapy is usually multifaceted and includes physical therapies, anti‐coagulant medication and interventions such as sclerotherapy, endovascular laser, surgery and, more recently, sirolimus (rapamycin) . Indications for each are often subjective and individualised and there is only low level evidence (retrospective cohort series, case studies and unpublished ongoing studies) that such interventions may reduce or delay the onset of symptoms, particularly pain, swelling and haemorrhagic complications …”

Section: Introductionmentioning

confidence: 99%

Localised intravascular coagulation complicating venous malformations in children: Associations and therapeutic options

Zhuo

Russell

Wargon

et al. 2017

J Paediatrics Child Health

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Venous malformations are slow-flow congenital vascular malformations that enlarge as the child ages and may be associated with localised intravascular coagulation, a consumptive coagulopathy characterised by elevated D-dimer and decreased fibrinogen levels. The authors review the known correlations between localised intravascular coagulation and venous malformation number, size and planes involved, and call attention to the concept of the progression of localised intravascular coagulopathy as the child ages and their venous malformations enlarge. The authors also discuss the identified therapeutic options for its investigation, management and treatment, including compression garments, anti-coagulation therapy, sclerotherapy, endovascular laser, surgical excision and sirolimus (rapamycin). Evidence for protocol improvements that may be instigated for the optimal physical and medical therapy of venous malformations complicated by localised intravascular coagulopathy is reviewed.

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Section: Introductionmentioning

confidence: 99%

Localised intravascular coagulation complicating venous malformations in children: Associations and therapeutic options

Zhuo

Russell

Wargon

et al. 2017

J Paediatrics Child Health

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“…Nevertheless, some of these complicated VAs are refractory to the abovementioned treatment modalities. In these cases, rapamycin (mTOR) inhibitors are found to be more promising (9)(10)(11)(12)(13)(14)(15)(16).…”

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confidence: 99%

“…Since this initial success, the use of sirolimus has expanded rapidly (8). While initially explored for KHE and lymphatic malformation (LM) (26)(27)(28), sirolimus is now being used broadly throug hout the VA spectrum and is showing promising results in both systemic administration (10,(29)(30)(31) and topical administration (32)(33)(34).…”

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confidence: 99%

Oral and Topical Sirolimus for Vascular Anomalies: A Multicentre Study and Review

Sandbank¹,

Molho‐Pessach²,

Farkas³

et al. 2019

Acta Derm Venerol

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This is an open access article under the CC BY-NC license. www.medicaljournals.se/acta Acta Derm Venereol 2019; 99: 990-996 990 SIGNIFICANCEIn recent years, sirolimus, an mTOR inhibitor, has become a new therapeutic option for patients with vascular anomalies that do not respond to other treatments. We report here a retrospective series of 19 young adults, children and neonates with complex vascular anomalies treated with sirolimus. Overall, clinical improvement was demonstrated in 15 patients (79%). In addition, an up-to-date literature review was performed and 150 cases of vascular anomalies treated with sirolimus were analysed. The results suggest that sirolimus is an effective and safe treatment. Further study is needed into the early use of sirolimus in cases of low-flow lesions, and overgrowth syndromes with low-flow components.Vascular anomalies (VAs) may be associated with significant morbidity and mortality. The aim of this study was to evaluate the efficacy and safety of sirolimus (rapamycin) in the treatment of children and young adults with complicated VAs. A retrospective chart was created that included 19 patients treated with sirolimus for complicated VAs. Concurrently, a search of the PubMed database for VA cases treated with sirolimus was conducted. Descriptive analysis was performed and the efficiency rate of sirolimus was calculated. This retrospective study included 19 patients, 17 of whom were treated with oral sirolimus and 2 with topical sirolimus. Clinical improvement occurred in 15 patients (79%). One patient experienced near-complete resolution. Only 2 patients showed poor response and discontinued treatment. The literature review analysed 150 cases of VA treated with sirolimus. Sirolimus was efficient in 85% of cases, including 5 cases of complete resolution. Sirolimus appears to be an effective and safe treatment for children and young adults with complicated VAs.

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“…Six patients with complicated vascular anomalies that had been refractory to other measures were successfully treated using sirolimus . mTOR inhibition is also being studied in a cohort of venous malformations . Finally, in a murine model, the use of sirolimus prevented self‐renewal and vascular differentiation of patient‐derived infantile hemangioma stem cells .…”

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confidence: 99%

Tuberous Sclerosis Complex Associated with Vascular Anomalies or Overgrowth

Jenkins

McCuaig

Drolet

et al. 2016

Pediatric Dermatology

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Rapamycin as Novel Treatment for Refractory-to-Standard-Care Slow-Flow Vascular Malformations

Cited by 11 publications

References 0 publications

Localised intravascular coagulation complicating venous malformations in children: Associations and therapeutic options

Localised intravascular coagulation complicating venous malformations in children: Associations and therapeutic options

Oral and Topical Sirolimus for Vascular Anomalies: A Multicentre Study and Review

Tuberous Sclerosis Complex Associated with Vascular Anomalies or Overgrowth

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