Efficacy and safety of sirolimus in the treatment of gastrointestinal angiodysplasias.

BACKGROUND

Gastrointestinal angiodysplasias (GIAD) causes recurrent bleeding, and current treatments have limitations. Sirolimus, a mammalian target of rapamycin inhibitor, shows promise in inhibiting abnormal angiogenesis.

AIM: To evaluate the efficacy and safety of sirolimus in reducing bleeding episodes and improving clinical outcomes in patients with GIAD.

METHODS

We conducted a self-controlled study with 11 patients taking oral sirolimus. Retrospective data were collected prior to treatment, and prospective data were gathered during the study. Efficacy was assessed primarily by comparing bleeding episodes before and after sirolimus, with measurements at 3 and 6 months post-administration. The initial dose was 0.8 mg/m² once daily, adjusted to maintain trough blood concentrations between 5-10 ng/mL. Secondary outcomes included hemoglobin (Hb) levels, blood transfusion volume, and vascular lesions. Safety was monitored by tracking adverse reactions.

RESULTS

The average number of bleeding episodes decreased significantly from 2.09 ± 1.04 to 1.00 ± 0.75 in the 3 months preceding treatment, and from 3.80 ± 1.93 to 2.00 ± 1.63 in the 6 months preceding treatment. Sirolimus also increased Hb levels, reduced the need for transfusions, and decreased vascular lesions, improving clinical outcomes. All adverse effects were mild and resolved or improved within 1 week to 1 month without stopping sirolimus or needing lipid-lowering treatment.

CONCLUSION

Sirolimus reduced bleeding and transfusion needs while improving Hb levels in GIAD patients. Although these findings are encouraging, the limited sample size and lack of a control group warrant caution. Future controlled trials with larger populations are needed to validate sirolimus's potential in GIAD.