Efficacy and Safety of Sirolimus for Blue Rubber Bleb Nevus Syndrome: A Prospective Study

    January 2021 in “ ˜The œAmerican journal of gastroenterology ”
    Jiaolin Zhou, Zichen Zhao, Tao Sun, Wei Liu, Zi-Hao Yu, Jingjuan Liu, Yiqi Yu, Shou-Bin Ning, Hongbing Zhang
    TLDR Sirolimus effectively reduces lesions and improves quality of life in Blue Rubber Bleb Nevus Syndrome with manageable side effects.
    The prospective study evaluated the efficacy and safety of sirolimus in 11 patients (4 males, 7 females) with Blue Rubber Bleb Nevus Syndrome (BRBNS), aged 5-49 years. Sirolimus treatment at 1.0 mg/m² significantly reduced the average lesion size by 7.4%, 9.3%, and 13.0% at 3, 6, and 12 months, respectively (P < 0.05). Hemoglobin levels increased significantly after 6 and 12 months (P = 0.006 and 0.019), and only 1 patient required a blood transfusion. Quality of life and coagulation function improved, with mild and self-limited adverse effects such as oral ulcers (81.8%), acne (27.3%), transient liver enzyme elevation (18.2%), and hair loss (9.1%). The study concluded that sirolimus effectively reduces VM size, alleviates GI bleeding, and eliminates transfusion dependence in BRBNS patients, supporting its use as a first-line treatment.
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