Understanding complex disease-related mechanisms: Rational therapies for Diamond-Blackfan anaemia.
Br J Haematol
; 204(5): 1598-1599, 2024 May.
Article
em En
| MEDLINE
| ID: mdl-38485153
ABSTRACT
The rich history surrounding Diamond-Blackfan anaemia (DBA), originally described in 1938 as congenital hypoplastic anaemia2 reflects the evolution of paediatric haematology. In their paper, the authors1 present the results of a clinical trial using the thrombopoietin-mimetic agent eltrombopag to treat red cell failure in DBA. A low response rate belies the importance of this work. Commentary on Duncan et al. Treatment of refractory/relapsed Diamond-Blackfan anaemia with eltrombopag. Br J Haematol 2024;2042077-2085.
Palavras-chave
Texto completo:
1
Bases de dados:
MEDLINE
Assunto principal:
Pirazóis
/
Benzoatos
/
Anemia de Diamond-Blackfan
/
Hidrazinas
Limite:
Humans
Idioma:
En
Revista:
Br J Haematol
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Estados Unidos