Azacitidine combined with venetoclax alleviates AML-MR with TP53 mutation in SDS: a case report and literature review.
Anticancer Drugs
; 35(6): 548-555, 2024 Jul 01.
Article
en En
| MEDLINE
| ID: mdl-38502829
ABSTRACT
Shwachman-Diamond syndrome (SDS) is an autosomal recessive genetic disease, which is prone to transform into myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). TP53 mutation is a driving factor involved in the transformation of SDS into MDS/AML, and in the evolution of MDS to AML. Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) is the only curable approach, however, challenge remains regarding the balance between efficacy and the high risk from treatment-related toxicity and mortality to achieve temporary disease control before transplantation to gain time and opportunities for transplantation. At present, pre-transplant bridging therapy has emerged as one of the important options with improved efficacy, reduced tumor burden, and less treatment-related toxicity. Here we reported azacitidine combined with venetoclax was used as pre-transplant bridging regimen in a TP53-mutant AML-MR case developed from SDS. He achieved complete remission with incomplete recovery and proceeded to Allo-HSCT. We hope to provide some evidence and insight for in-depth research and clinical treatment by presenting this case.
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Sulfonamidas
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Azacitidina
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Leucemia Mieloide Aguda
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Protocolos de Quimioterapia Combinada Antineoplásica
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Proteína p53 Supresora de Tumor
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Compuestos Bicíclicos Heterocíclicos con Puentes
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Mutación
Límite:
Humans
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Male
Idioma:
En
Revista:
Anticancer Drugs
Asunto de la revista:
ANTINEOPLASICOS
Año:
2024
Tipo del documento:
Article