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| Article authors | Article title | Article journal | Article year | Patient | Response to Sirolimus | Sirolimus dose | Sirolimus level | Time to improvement |
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| Yuksekkaya H, Ozbek O, keser M, Toy H [4] | Blue rubber bleb nevus syndrome: successful treatment with Sirolimus | Pediatrics | 2012 | 8 yo F; GI bleeding, not responsive to prednisolone, propranolol, aminocaproic acid, and alpha-interferon therapy | Hemoglobin improved from 7 gm/dL to 14 gm/dL (with iron supplement); | 0.05–0.1 mg/kg | 1–5 ng/mL | 2 months. Symptoms returned if sirolimus discontinued |
| Taddio A, Benelli E, Pierobon C, Martelossi S, Berti I, Ventura A [6] | From skin to gut | J Pediatr | 2013 | 3 yo M; severe iron deficiency anemia, large subcutaneous swelling on right ankle, and multiple skin lesions | Hemoglobin improved from 6.4 gm/dL to 'stable' | not available (NA) | NA | 6 months |
| Ozgonenel B, Martin A [9] | Low-dose sirolimus controls recurrent iron deficiency in a patient with blue rubber bleb nevus syndrome | Pediatr Blood Cancer | 2015 | 18 yo F; GI bleeding and iron deficiency anemia | Hemoglobin improved from 5 gm/dL to ∼14 gm/dL | 1.6 mg/m2/day divided BID; reduced to 0.6 mg/m2/day | 10–15 ng/mL, then goal levels reduced <2.0–3.1 ng/mL | “after starting sirolimus” |
| Ferrés-Ramis L, knöpfel N, Salinas-Sanz J, Martín-Santiago A [8] | Rapamycin in the treatment of blue rubber bleb nevus syndrome | Actas Dermo-Sifiliográficas (English Edition) | 2015 | 8 yo with congenital cutaneous and GI vascular malformations | Decreased size of lesions, normalized hemoglobin | Initial dose 0.05 mg/kg, reduced to 0.02 mg/kg | | Within a month |
| Warner B, Butt A, cairns S [7] | Sirolimus is a successful treatment for recurrent iron deficiency anemia in blue rubber bleb nevus syndrome | J Pediatr Gastroenterol Nutr | 2015 | 18 yo M; hemangioma, tracheostomy, anemic; thalidomide—discontinued due to side effects; multiple bowel resections to remove vascular malformations | Hemoglobin 6.9 gm/dL, anemia resolved | 4 mg daily | | |
| Salloum R, Fox CE, Alvarez-Allende CR, et al. [3] | Response of blue rubber bleb nevus syndrome to sirolimus treatment. | Pediatr Blood Cancer | 2016 | 2–16 yo, cutaneous, GI tract, visceral and muscular lesions | Decreased size of lesions, decreased pain, normalized hemoglobin | 0.8 mg/m2 every 12 hour | dose titrated to target trough level between 10 and 13 ng/mL | less than 3 months |
| Cardoso H, Dias JA, Silva M, et al. [10] | Gastrointestinal: successful treatment with sirolimus of a patient with blue rubber bleb nevus syndrome | J Gastroenterol Hepatol | 2016 | 19 yo M; multiple visceral, muscular, and subcutaneous vascular lesions, complicated with chronic local pain and GI bleeding requiring RBC transfusion (total of 74); treatment with thermal argon ablation and sclerosis and segmental jejunoileal surgical resections, propranolol and ferric carboxymaltose | improved blood loss, asthenia, and decreased size of lesions; hemoglobin improved by 6 gm/dL | 2 gm/day | | about 5 months |
| Ünlüsoy Aksu A, Sari S, Eğritaş Gürkan Ö, Dalgiç B [11] | Favorable response to sirolimus in a child with blue rubber bleb nevus syndrome in the gastrointestinal tract | J Pediatr Hematol Oncol | 2017 | 11 yo M with vascular malformation in GI tract | Normalized hemoglobin in 2.5 months, decreased lesions in 5 months | 0.1 mg/kg/d | 1–5 ng/mL | 2.5–5 months |
| Akyuz C, Susam-Sen H, Aydin B [12] | Blue rubber bleb nevus syndrome: promising response to sirolimus | INDIAN Pediatr | 2017 | 6 yo F with skin lesions, GI tract and consumptive coagulopathy (platelets 77K, fibrinogen 104 mg/dL, d-dimer >40 mg/dL); oral steroids without success; bleeding requiring transfusions (hemoglobin 6.1 gm/dL) | improved size and number of lesions, no further GI bleeding or anemia | 1.6 to 2 mg/m2/day | 5–12 ng/mL | Less than 1 month. Disconintued sirolimus and at 4 months off therapy- no evidence of microscopic blood in stool and normal hemaglobin levels, stable lesions |
| Wang KL, Ma SF, Pang LY, Zhang MN, Hu LY, Liu MJ, Zou LP [13] | Sirolimus alternative to blood transfusion as a life saver in blue rubber bleb nevus syndrome | Medicine (Baltimore) | 2018 | 12 yo F with multiple hemangiomas on head and neck, limbs and trunk, tip of tongue and digestive tract. Severe anemia, requiring red cell transfusion every 2 weeks. Mutation in exon 15 of TEK gene | Improved hemoglobin, skin and digestive tract hemangiomas, no further transfusions | 1 mg/m2/d, average 0.7 mg/d | 6.2–11.89 ug/L | 1 month |
| Kizilocak H, Dikme G, celkan T [14] | Sirolimus experience in blue rubber bleb nevus syndrome | J Pediatr Hematol Oncol | 2018 | Four children, ages 4–15 years. Three with GI lesions, one with respiratory tract lesions | Normalized hemoglobin, decreased pain, decreased size of lesions | 1.2 mg/m2/d | | 2 months |
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