Sotatercept for the Treatment of Pulmonary Arterial Hypertension

Abstract

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Platelets, extracellular vesicles and coagulation in pulmonary arterial hypertension

“…87 However, serious adverse events were common and included subdural hematoma in patients who were Sotatercept is a novel, first-in-class recombinant fusion protein that is undergoing evaluation in PAH. 89 Sotatercept binds activins and growth differentiation factors to restore homeostasis between pro-and anti-proliferative signals in the TGFβ superfamily pathway, which are imbalanced in PAH. In the PULSAR trial, treatment with sotatercept was associated with a significant reduction in PVR.…”

“…Haematological adverse events were anticipated and noted, including thrombocytopaenia and polycythaemia. 89 Indeed sotatercept has previously been studied as a potential treatment of anaemia in patients with haematological malignancy. 90 The precise mechanism of thrombocytopaenia and associated clinical implications require exploration.…”

World Pulmonary Hypertension Day: reflections and planning

“…O primeiro estudo clínico nacional com medicamentos para HAP teve início em 2002, e a participação em ensaios clínicos multicêntricos internacionais se iniciou já em 2003. Desde então, praticamente o desenvolvimento de todos os novos fármacos contou com a participação de pesquisadores nacionais, não apenas com a inclusão de um número significativo de pacientes em estudos clínicos de grande relevância, (8,9) mas também no desenvolvimento de vários dos protocolos desses mesmos estudos. Portanto, nosso país contribuiu de forma objetiva para que hoje o mundo tenha 16 medicamentos aprovados para o tratamento da HAP.…”

Progenitor/Stem Cells in Vascular Remodeling during Pulmonary Arterial Hypertension

“…This signaling pathway dysregulation plays a major role, and some studies have indeed demonstrated that it also controls progenitor and stem cell behavior. Thus recent results of a protector effect of a BMP treatment in Bmpr2 mutated mice [2] or of a proteic compound acting as a Transforming Growth Factor β (TGF-β) decay molecule designed to counterbalance the deficient BMP pathway [3] could also involve regulation of vascular progenitor and stem cells. Pulmonary hypertension is characterized by excessive pulmonary vascular occlusive remodeling with an increased medial thickness of normally muscularized arterioles and muscularization of previously non-muscularized arterioles.…”