High importance
Aug 21, 2026
To evaluate current evidence on systemic therapies for Hereditary Hemorrhagic Telangiectasia (HHT) and identify emerging therapeutic targets.
The review synthesizes findings from clinical trials and studies involving various systemic therapies, including antifibrinolytics, anti-angiogenic agents, mTOR and AKT inhibitors, and immunomodulatory drugs, assessing their efficacy in HHT management.
Existing therapies have shown variable effectiveness in specific patient cohorts, with promising results that indicate potential for systemic treatment to complement traditional approaches. Current clinical trials are underway to explore novel agents targeting the dysregulated BMP9-BMP10/ENG/ALK1 pathway.
The review notes a lack of specific FDA-approved systemic agents for HHT, and variability in patient responses complicates drawing definitive conclusions about treatment efficacy and the integration of systemic therapies into standard care.
Understanding and developing systemic therapies for HHT is crucial as it addresses underlying disease mechanisms, potentially improving patient outcomes beyond current palliative interventions. This shift could lead to a change in clinical practice and better overall management of HHT-induced complications.
Hereditary hemorrhagic telangiectasia (HHT) is a rare autosomal dominant vascular disorder characterized by mucocutaneous telangiectases and visceral vascular malformations, leading to recurrent bleeding, iron-deficiency anemia, and organ complications. Pulmonary, cerebral, and hepatic involvement are major determinants of morbidity and mortality. Management has traditionally relied on local and interventional approaches, which are largely palliative and do not address underlying disease mechanisms. New insights in HHT pathophysiology, particularly in the dysregulated BMP9-BMP10/ENG/ALK1 pathway, which is fundamental to the angiogenesis process, have enabled the development of targeted systemic therapies. Although no systemic drug is specifically approved for HHT, antifibrinolytics, anti-angiogenic agents, mTOR and AKT inhibitors, and immunomodulatory drugs have shown variable efficacy in selected patients. This review summarizes current evidence on systemic therapies and ongoing clinical trials, highlighting their potential to complement existing strategies and move toward disease-modifying approaches.