From bench to bedside: The promise of sotatercept in hematologic disorders

Lan, Zehao; Lv, Zhibao; Zuo, Wanyun; Xiao, Yichao

doi:10.1016/j.biopha.2023.115239

Biomedicine & Pharmacotherapy

2023

DOI: 10.1016/j.biopha.2023.115239

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From bench to bedside: The promise of sotatercept in hematologic disorders

Zehao Lan

Zhibao Lv

Wanyun Zuo

et al.

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2024

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Cited by 2 publications

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Pharmacological Agents and Potential New Therapies in Pulmonary Arterial Hypertension

Santos,

de Sá Freire Onofre,

de Assis Fernandes Caldeira

et al. 2024

CVP

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Pulmonary arterial hypertension (PAH) is a progressive disease characterized by an imbalance between vasoactive mediators, which causes vascular remodeling, increased pulmonary vascular resistance, and right ventricular overload, ultimately leading to heart failure and death. A metabolic theory has been suggested to explain the pathophysiology of PAH whereby abnormalities in mitochondrial biogenesis can trigger a hyperproliferative and apoptosis-resistant phenotype in cardiopulmonary and malignant cells, leading to mitochondrial dysfunction, which in turn causes the Warburg effect. This can culminate in the mitophagy of pulmonary vessels and cardiomyocytes. The present narrative review focuses on the pathophysiology of PAH, the pharmacological agents currently available for its treatment, and promising and challenging areas of therapeutic investigation.

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Pharmacological Agents and Potential New Therapies in Pulmonary Arterial Hypertension

Santos,

de Sá Freire Onofre,

de Assis Fernandes Caldeira

et al. 2024

CVP

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Treatment Options for Congenital Dyserythropoietic Anemias (CDAs): Advances in Bone Marrow Transplantation, Gene Therapy, and Targeted Therapies

Sophia,

Maria,

Elena

et al. 2024

J Hematol Clin Res

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Congenital Dyserythropoietic Anaemia (CDA) is a rare genetic disorder that affects the maturation of red blood cells. The disorder is classified into different types, with a prevalence ranging from 1 in 100,000 to 1 in 1,000,000 individuals. Treatment strategies are designed with the primary focus on symptom management, the prevention and treatment of complications, and the underlying disease pathophysiology. The advent of bone marrow transplantation, gene therapy, and targeted therapies has considerably expanded the scope for therapeutic intervention in CDAs. Supportive care, including blood transfusions and iron chelation therapy, has demonstrated efficacy in managing iron overload and improving overall survival rates. The potential of gene therapy, targeted therapies, and hematopoietic growth factors in the treatment of CDA is currently being investigated. Further research and clinical trials are required to develop more effective and personalized therapeutic interventions.

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From bench to bedside: The promise of sotatercept in hematologic disorders

Cited by 2 publications

References 68 publications

Pharmacological Agents and Potential New Therapies in Pulmonary Arterial Hypertension

Pharmacological Agents and Potential New Therapies in Pulmonary Arterial Hypertension

Treatment Options for Congenital Dyserythropoietic Anemias (CDAs): Advances in Bone Marrow Transplantation, Gene Therapy, and Targeted Therapies

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