Combination therapy with venetoclax and azacitidine for the treatment of myelodysplastic syndromes with DDX41 mutations.
Hematology
; 29(1): 2338509, 2024 Dec.
Article
em En
| MEDLINE
| ID: mdl-38597818
ABSTRACT
Myelodysplastic syndromes (MDS) patients with DEAD-box helicase 41 (DDX41) mutations have been reported to be treated effectively with lenalidomide; however, there are no randomized studies to prove it. Venetoclax and azacitidine are safe and effective in high-risk MDS/AML. In this study, we evaluated the efficacy of venetoclax and azacitidine combination therapy in eight consecutive MDS patients with DDX41 mutations at our centre from March 2021 to November 2023. We retrospectively analyzed the genetic features and clinical characteristics of these patients. Our findings suggest that MDS patients with DDX41 mutation may benefit from the therapy, for six subjects received this regimen as initial therapy and five of the six subjects achieved complete remission.
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Texto completo:
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Base de dados:
MEDLINE
Assunto principal:
Sulfonamidas
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Síndromes Mielodisplásicas
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Compostos Bicíclicos Heterocíclicos com Pontes
Limite:
Humans
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article