Luspatercept in the treatment of lower-risk myelodysplastic syndromes.
Future Oncol
; 17(12): 1473-1481, 2021 Apr.
Article
em En
| MEDLINE
| ID: mdl-33511859
ABSTRACT
Transforming growth factor beta (TGF-ß) signaling pathway is key to hematopoiesis regulation. Increased activation of this pathway contributes to ineffective terminal erythroid differentiation in myelodysplastic syndromes (MDS). Luspatercept is a novel fusion protein that traps TGF-ß ligands preventing them from binding to Type II TGF-ß receptors, thereby decreasing phosphorylated SMAD2/3 resulting in the downstream effect of promoting erythropoiesis. Seminal clinical trials using luspatercept, PACE-MD and MEDALIST, demonstrated impressive efficacy in the treatment of transfusion-dependent anemia in intermediate risk or lower MDS had led to the US FDA approval for this indication. This review summarizes luspatercept mechanisms of action, efficacy/safety data supporting its use and ongoing clinical trials in MDS.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Síndromes Mielodisplásicas
/
Proteínas Recombinantes de Fusão
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Fragmentos Fc das Imunoglobulinas
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Fator de Crescimento Transformador beta
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Receptores de Activinas Tipo II
Tipo de estudo:
Etiology_studies
/
Risk_factors_studies
Limite:
Humans
Idioma:
En
Revista:
Future Oncol
Ano de publicação:
2021
Tipo de documento:
Article
País de afiliação:
Estados Unidos