Sotatercept therapy in a patient with TBX4-associated heritable pulmonary arterial hypertension led to removal from the lung transplant waiting list and return to sporting activities at 1 year.
Case Report (n=1)
Does sotatercept improve clinical status in a patient with TBX4-associated heritable pulmonary arterial hypertension?
Sotatercept demonstrated exceptional clinical efficacy in a patient with TBX4-associated heritable PAH, leading to removal from the lung transplant waiting list.
ABSTRACT Pulmonary arterial hypertension (PAH) is a life‐threatening disease of the pulmonary arteries. Progressively increasing pulmonary artery pressure may lead to fatal failure of the right ventricle, necessitating aggressive drug treatment. Sotatercept, a novel drug for PAH‐targeted therapy, shows promising results in clinical trials. Nevertheless, efficacy in the heterogeneous PAH population and long‐term outcomes have yet to be investigated. This case report illustrates the life‐changing effects of sotatercept in a patient with heritable PAH associated with a TBX4 mutation. Our patient faced near immobility, oxygen dependency, and was listed for lung transplantation in November 2023. Following sotatercept administration from July 2024 onward, she started sporting activities and has been removed from the transplantation waiting list. Thereby, this case illustrates an exceptional response to sotatercept in a patient with TBX4‐associated heritable PAH. Positive effects persist 1 year after initiation without significant side effects.
Schneijdenberg et al. (Wed,) conducted a case report in TBX4-associated heritable pulmonary arterial hypertension (n=1). Sotatercept was evaluated. Sotatercept therapy in a patient with TBX4-associated heritable pulmonary arterial hypertension led to removal from the lung transplant waiting list and return to sporting activities at 1 year.
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