Blue rubber bleb nevus syndrome: Promising response to sirolimus

Akyüz, Canan; Şen, Hilal Susam; Aydın, Burça

doi:10.1007/s13312-017-0998-1

Cited by 18 publications

(19 citation statements)

References 5 publications

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“…The majority of patients continued sirolimus until the publication date. Two patients were able to discontinue treatment without symptom recurrence . Three patients who terminated treatment experience a relapsed and were reported to have continued treatment till the time of publication …”

Section: Resultsmentioning

confidence: 98%

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Sirolimus in blue rubber bleb naevus syndrome: A systematic review

Wong

Phan

Bandera

et al. 2018

J Paediatrics Child Health

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The aim of this study is to review sirolimus as a treatment for blue rubber bleb naevus syndrome (BRBNS). A literature search of Medline, Embase, CINAHL, SCOPUS and Google Scholar was conducted for publications reporting treatment of patients with BRBNS with sirolimus. Of 46 articles identified, 17 studies reporting 23 patients met inclusion criteria. Sirolimus was well tolerated in all but one patient who required treatment cessation; 17/18 patients noted an improvement in gastrointestinal disease where this was reported; 21/22 patients noted an improvement in cutaneous disease where this was reported. Based on these results, sirolimus may be considered a first-line treatment of BRBNS depending on patient morbidity.

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Section: Resultsmentioning

confidence: 98%

“…Four articles were omitted as they lacked sufficient clinical detail . After applying inclusion and exclusion criteria, 17 studies (two retrospective reviews and 15 case reports) with 23 patients were selected for inclusion . The clinical detail of the patients included is outlined in Table S1 (Supporting Information).…”

Section: Resultsmentioning

confidence: 99%

Sirolimus in blue rubber bleb naevus syndrome: A systematic review

Wong

Phan

Bandera

et al. 2018

J Paediatrics Child Health

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“…Ten case reports describe the effect of sirolimus orally for the treatment of venous malformations. 12,18,[56][57][58][59][60][61][62][63] Efficacy was considered a nonspecified reduction in lesion size, which was observed in 88.9% of patients. The median age of patients in these reports was 12 years (1.4-23.0 years).…”

Section: Vascular Malformationsmentioning

confidence: 99%

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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“…Clinical data on the use of sirolimus in patients with vascular anomalies are rare and most of them are case reports. 27,28 Since our patient did not respond to propranolol, prednisolone and vincristine treatments and the lesion showed slight progression, it was decided to try sirolimus treatment after embolization.…”

Section: Discussionmentioning

confidence: 95%

Temporal bone hemangioendothelioma as a rare vascular tumor in childhood: case report and review of the literature

Gündoğan¹,

Çıtak²,

Sağcan³

et al. 2020

Turk J Pediatr

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Background. Hemangiondothelioma is a rare vascular tumor that can occur in the bone. Temporal bone involvement has been reported extremely rare in the literature. Case. Radiological examination of a one-year-old girl who was admitted due to facial paralysis revealed vascular tumor of the temporal bone and Galen vein aneurysm. Pathological examination showed retiform hemangioendothelioma. She was treated with propranolol, prednisolone, vincristine, and endovascular embolization followed by oral sirolimus. With sirolimus treatment, a partial response was obtained first, then the tumor remained stable and sirolimus treatment was discontinued. No progression was observed in the disease after discontinuation of treatment. Conclusion. In this article, a case of hemangioendothelioma originating from the temporal bone is discussed in the light of other case reports in the literature.

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Blue rubber bleb nevus syndrome: Promising response to sirolimus

Abstract: Sirolimus may be a valuable option for reducing bleeding complications and cosmetic sequelae for the patients with this syndrome.

Cited by 18 publications

References 5 publications

Sirolimus in blue rubber bleb naevus syndrome: A systematic review

Sirolimus in blue rubber bleb naevus syndrome: A systematic review

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

Temporal bone hemangioendothelioma as a rare vascular tumor in childhood: case report and review of the literature

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