sirolimus + propranolol

Sirolimus + propranolol is a combination therapy being investigated for the treatment of refractory infantile hepatic hemangioendothelioma (IHHE). Sirolimus, an mTOR inhibitor, works by binding to the cytosolic protein FKBP12 to form a complex that inhibits the mechanistic target of rapamycin (mTOR) signaling pathway, thereby suppressing vascular endothelial cell proliferation and angiogenesis. Propranolol is a non-selective beta-adrenergic receptor antagonist that is thought to cause vasoconstriction, down-regulate angiogenic factors like VEGF and bFGF, and induce apoptosis in capillary endothelial cells. This combination is specifically being studied by the Shanghai Children's Medical Center for infants who do not respond adequately to propranolol monotherapy, aiming to improve therapeutic outcomes while reducing the need for more invasive interventions like surgery or interferon therapy.

Other names

sirolimus and propranolol combination therapypropranolol + sequential sirolimus treatment