2026-08-14 | Bevacizumab Reduces Epistaxis-Related Operative Burden in Hereditary Hemorrhagic Telangiectasia.
Bevacizumab reduced epistaxis-related ablation frequency by 55.6% in HHT patients. Over half of HHT patients (57.7%) required no ablation intervention after bevacizumab initiation. Maintenance bevacizumab dosing remains highly variable, underscoring the need for standardization.
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2026-08-14 | Brain Abscess as the Initial Manifestation of Hereditary Hemorrhagic Telangiectasia in a Child.
Brain abscess secondary to pulmonary arteriovenous malformation is rare in children and may be the first manifestation of hereditary hemorrhagic telangiectasia (HHT). We retrospectively reviewed the clinical presentation, laboratory findings, imaging examinations, and diagnostic process of a child with brain abscess associated with pulmonary arteriovenous malformation and HHT. A 12-year-old boy presented with fever, headache, and limb weakness. Brain abscess was diagnosed, and antimicrobial therapy was initiated. Laboratory testing revealed fungal infection and erythrocytosis. Further evaluation identified a pulmonary arteriovenous malformation. Although the family history was initially unremarkable, physical examination revealed facial telangiectasia, recurrent epistaxis, and digital clubbing in his father, fulfilling the Curaçao criteria for HHT. Right-to-left shunt should be considered in children with brain abscess of unclear origin. Early recognition of underlying HHT is critical for timely diagnosis and management.
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2026-08-12 | Clinical characteristics of pulmonary hypertension associated with hereditary hemorrhagic telangiectasia treated with bevacizumab: a single-center case series.
Pulmonary hypertension (PH) is a severe complication of hereditary hemorrhagic telangiectasia (HHT). The experience with bevacizumab in HHT-PH is limited. Therefore, the aim was to provide our single-center experience with the use of bevacizumab in patients with HHT-PH. The clinical data of HHT-PH patients treated with bevacizumab were retrospectively reviewed between September 2021 and June 2024 at Beijing Anzhen Hospital, Capital Medical University. To describe the clinical features of these patients, we recruited pulmonary arterial hypertension (PAH) patients and PH associated with left heart diseases (PH-LHD) patients, matched for age and gender. The symptoms, underlying diseases, medication history, laboratory test results, treatments, and therapeutic responses were investigated retrospectively in all patients. In total, six cases of HHT-PH that were treated with bevacizumab, of which only five received follow-up after completing the 3-month induction therapy. Compared with PAH and PH-LHD patients, HHT-PH patients were characterized by high cardiac index (CI) [HHT-PH vs. PAH vs. PH-LHD: 6.10 (3.77, 7.60) vs. 2.37 (1.92, 3.03) vs. 2.38 (1.91, 2.63) L/min/m2, P=0.001] in conjunction with low systemic vascular resistance [740.93±333.06 vs. 1,836.07±673.64 vs. 1,667.78±453.84 dyn·s·cm-5, P=0.001] and PVR [189.94 (54.46, 289.89) vs. 886.70 (705.18, 1278.68) vs. 229.48 (179.15, 416.60) dyn·s·cm-5, P=0.002]. All the HHT-PH patients experienced significant improvements in symptoms (World Health Organization functional class), hemoglobin, brain natriuretic peptide levels and tricuspid annular plane systolic excursion/systolic pulmonary arterial pressure after the induction therapy with bevacizumab. Of note, two of the five patients had a baseline CI <4 L/min/m2, yet still experienced clinically meaningful improvement after the bevacizumab induction therapy. Four out of five patients experienced improvements in hemodynamics and 6-minute walking distance after the induction therapy. Our study found that the hemodynamic profile of HHT-PH patients treated with bevacizumab differed significantly from that of PAH and PH-LHD. There might be patients with a CI below 4 L/min/m2 who were still in a high-output state and could benefit from bevacizumab treatment in clinical practice.
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2026-08-11 | Targeting KIT prevents brain arteriovenous malformations driven by ALK1-deficient angiogenic endothelial cells.
Hereditary Hemorrhagic Telangiectasia type 2 (HHT2), caused by mutations in ACVRL1 ( also known as ALK1), is characterized by brain arteriovenous malformations (bAVMs), abnormal artery-vein connections for which treatment options remain limited. Despite evidence of endothelial cell (EC) heterogeneity, its role in bAVM pathogenesis remains poorly defined. Using endothelial-specific inducible Alk1 knockout mice (Alk1iECKO) and regionally resolved single-cell RNA sequencing, we show that loss of ALK1 signaling induces bAVMs predominantly in the perineural vascular plexus (PNVP). This process is driven by the emergence of a KIT+ angiogenic EC population with human AVM-like transcriptional features, including tip-cell markers and activation of PI3K and KRAS signaling pathways. Cross-species analyses and validation in human samples demonstrate that KIT expression is conserved in endothelial cells from both sporadic and HHT2 brain AVMs. Drug repurposing analysis identified KIT as a top actionable target, and we show that Kit is directly repressed by BMP9-ALK1-SMAD4 signaling. Pharmacological inhibition of KIT reduced angiogenic reprogramming and vascular malformations in vivo without affecting normal vasculature. These findings identify a pathogenic angiogenic EC state and position KIT signaling as a therapeutically actionable pathway in brain AVMs.
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2026-08-02 | Intranasal bleomycin sclerotherapy versus local Avastin® therapy as an adjunct to laser treatment for epistaxis in hereditary hemorrhagic telangiectasia
Hereditary hemorrhagic telangiectasia is a rare disease of vascular development, often accompanied by severe epistaxis. Multimodality treatment may improve epistaxis control, but the optimal combination of modalities has not yet been established. To study and compare the effects of adding intranasal bleomycin versus local Avastin ® therapy to laser treatment for epistaxis in patients with hereditary hemorrhagic telangiectasia. We present a case study of all hereditary hemorrhagic telangiectasia patients who underwent laser therapy with or without bleomycin sclerotherapy or local Avastin ® therapy for epistaxis during 8 consecutive years. We calculated the mean modified posttreatment epistaxis severity score changes, proportions of procedures after which a minimal important difference in modified epistaxis severity score was reached, and intervals between subsequent treatments. A higher mean modified epistaxis severity score change was seen after laser treatment combined with bleomycin, compared to laser treatment alone. There was a longer mean treatment interval after both laser combined with Avastin ® , and laser combined with bleomycin, compared to laser treatment alone. Adding bleomycin to laser treatment shows a higher modified epistaxis severity score improvement compared to laser treatment only or laser combined with Avastin ® in patients with hereditary hemorrhagic telangiectasia. Adding bleomycin or Avastin ® to laser may lengthen treatment intervals compared to laser therapy alone.
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