Case report: Successful use of sotatercept to treat pulmonary arterial hypertension in a patient with hereditary hemorrhagic telangiectasia.

Abinaya Ramakrishnan, Scott Olitsky, Rajan Saggar, Richard N Channick, Justin P McWilliams

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Article in Respiratory medicine case reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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5 · Who and what money

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5 authors.

Abinaya RamakrishnanDivision of Interventional Radiology, Department of Radiology, UCLA Medical Center, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.

Scott OlitskyCureHHT, HHT Foundation International, Monkton, MD, USA.

Rajan SaggarPulmonary Vascular Disease Program, Division of Pulmonary, Critical Care, and Sleep Medicine, UCLA Medical Center, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.

Richard N ChannickPulmonary Vascular Disease Program, Division of Pulmonary, Critical Care, and Sleep Medicine, UCLA Medical Center, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.

Justin P McWilliamsDivision of Interventional Radiology, Department of Radiology, UCLA Medical Center, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

This case report describes a 45-year-old female with HHT type 1 (ENG mutation) and congenital heart disease (sinus venosus atrial septal defect and partial anomalous pulmonary venous return), diagnosed with severe PAH at age 16. Despite long-term treatment with epoprostenol, treprostinil, macitentan, and tadalafil, her condition progressed, leading to evaluation for lung transplantation. In May 2024, sotatercept, a novel TGF-β superfamily ligand trap, was initiated, resulting in significant improvement in exertional capacity and quality of life. However, the patient experienced increased epistaxis frequency (2-4 episodes/week), though hemoglobin levels rose from 13.0 to 15.4 g/dL. No new telangiectasias or other adverse events were noted. This is the first reported case of sotatercept use in HHT-associated PAH, highlighting its potential efficacy but also the need for careful monitoring due to increased bleeding risk. Further studies are required to establish the safety and efficacy of sotatercept in this population.

Indexed as

EpistaxisHereditary hemorrhagic telangiectasiaPulmonary arterial hypertensionSotatercept

Identifiers

PMID41229706

DOI10.1016/j.rmcr.2025.102321

PMCPMC12603696

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.