From Skin to Gut

Taddio, Andrea; Benelli, Elisa; Pierobon, Chiara; Martelossi, Stefano; Berti, Irene; Ventura, Alessandro

doi:10.1016/j.jpeds.2013.03.045

Cited by 5 publications

(7 citation statements)

References 3 publications

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“…The first case report describing response of BRBNS to sirolimus was published in 2012 by Yussekkaya et al [ 4 ]. Following that there have been several other reports also describing response to sirolimus [ 3 , 6 – 14 ]. Table 1 details the various reports in the literature so far that have described the use of sirolimus for BRBNS.…”

Section: Discussionmentioning

confidence: 99%

Successful Management of Blue Rubber Bleb Nevus Syndrome (BRBNS) with Sirolimus

Ogu

Abusin

Abu‐Arja

et al. 2018

Case Reports in Pediatrics

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Blue rubber bleb nevus syndrome (BRBNS) is a rare disease with vascular malformations in several systems of the body, most commonly the skin and gastrointestinal tract. Bleeding from the gastrointestinal (GI) tract is a major complication, which may lead to chronic iron deficiency anemia and the need for frequent blood transfusions due to ongoing gastrointestinal blood loss. In this case report, we describe a now 19-year-old female with BRBNS who required six blood transfusions per year and after starting sirolimus is symptom- and transfusion-free.

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Section: Discussionmentioning

confidence: 99%

Successful Management of Blue Rubber Bleb Nevus Syndrome (BRBNS) with Sirolimus

Ogu

Abusin

Abu‐Arja

et al. 2018

Case Reports in Pediatrics

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“…32 Sirolimus blocks mTOR, thereby serving as a targeted therapy for BRBNS. Its efficacy was first demonstrated in 2012 31 and since then there have been multiple reports describing its use [12][13][14][15][16][17][18][19][20][21][22][23][24][25][26][27][28][29][30][31] (Table S1).…”

Section: Discussionmentioning

confidence: 99%

“…Steroids, interferon α‐2a, propranolol, and thalidomide have not demonstrated efficacy and exhibit unfavorable side effects 9–11 . Sirolimus has anti‐angiogenic properties, with multiple reports documenting its success in BRBNS 12–31 . To date, one prospective study exists reporting the efficacy and safety of sirolimus in BRBNS, 12 though questions remain regarding dosing, duration, and long‐term side effects.…”

Section: Introductionmentioning

confidence: 99%

“…[9][10][11] Sirolimus has antiangiogenic properties, with multiple reports documenting its success in BRBNS. [12][13][14][15][16][17][18][19][20][21][22][23][24][25][26][27][28][29][30][31] To date, one prospective study exists reporting the efficacy and safety of sirolimus in BRBNS, 12 though questions Abbreviations: BRBNS, blue rubber bleb nevus syndrome; CT, computed tomography; EGD, esophagogastroduodenoscopy; GI, gastrointestinal. remain regarding dosing, duration, and long-term side effects.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Sirolimus for management of GI bleeding in blue rubber bleb nevus syndrome: A case series

Duong

Geddis

Carlberg

et al. 2022

Pediatric Blood & Cancer

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Blue rubber bleb nevus syndrome (BRBNS) commonly presents with anemia from bleeding gastrointestinal (GI) vascular malformations. Management is highly variable, as no consensus guidelines for medical treatment currently exist. Sirolimus has been used in BRBNS to decrease GI bleeding and seems well tolerated, though questions remain regarding dosing, duration of therapy, and adverse effects. Here, we report our single‐center experience of four pediatric patients with BRBNS who were successfully treated with sirolimus and review the existing literature regarding sirolimus for treatment of GI bleeding in BRBNS. Further prospective studies are needed to establish optimal dosage, drug monitoring, and duration.

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“…Multiple reports have shown interferon-α and corticosteroids to be unsuccessful in long-term treatment of multi focal, large GI lesions [ 4 ], [ 7 ]. However, recent publications have suggested that sirolimus may be beneficial in the treatment of gastrointestinal bleeds and anemia [ 7 ], [ 8 ]. Therapy with sirolimus can cause many side effects including hypertension, immunosuppression, renal failure, anemia and hyperlipidemia.…”

Section: Discussionmentioning

confidence: 99%