Personalized Medicine for TP53 Mutated Myelodysplastic Syndromes and Acute Myeloid Leukemia
Targeting TP53 mutated myelodysplastic syndromes and acute myeloid leukemia remains a significant unmet need. Recently, new drugs have attempted to improve the outcomes of this poor molecular subgroup. The aim of this article is to review all the current knowledge using active agents including hypom...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2021-09-01
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| Series: | International Journal of Molecular Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1422-0067/22/18/10105 |
| _version_ | 1797518805939781632 |
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| author | Thomas Cluzeau Michael Loschi Pierre Fenaux Rami Komrokji David A. Sallman |
| author_facet | Thomas Cluzeau Michael Loschi Pierre Fenaux Rami Komrokji David A. Sallman |
| author_sort | Thomas Cluzeau |
| collection | DOAJ |
| description | Targeting TP53 mutated myelodysplastic syndromes and acute myeloid leukemia remains a significant unmet need. Recently, new drugs have attempted to improve the outcomes of this poor molecular subgroup. The aim of this article is to review all the current knowledge using active agents including hypomethylating agents with venetoclax, eprenetapopt or magrolimab. We include comprehensive analysis of clinical trials to date evaluating these drugs in TP53 myeloid neoplasms as well as discuss future novel combinations for consideration. Additionally, further understanding of the unique clinicopathologic components of TP53 mutant myeloid neoplasms versus wild-type is critical to guide future study. Importantly, the clinical trajectory of patients is uniquely tied with the clonal burden of TP53, which enables serial TP53 variant allele frequency analysis to be a critical early biomarker in investigational studies. Together, significant optimism is now possible for improving outcomes in this patient population. |
| first_indexed | 2024-03-10T07:34:41Z |
| format | Article |
| id | doaj.art-4757574972614c46965d58eac9cce9f5 |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-10T07:34:41Z |
| publishDate | 2021-09-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-4757574972614c46965d58eac9cce9f52023-11-22T13:32:34ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672021-09-0122181010510.3390/ijms221810105Personalized Medicine for TP53 Mutated Myelodysplastic Syndromes and Acute Myeloid LeukemiaThomas Cluzeau0Michael Loschi1Pierre Fenaux2Rami Komrokji3David A. Sallman4Hematology Department, University Hospital of Nice, Cote d’Azur University, 06200 Nice, FranceHematology Department, University Hospital of Nice, Cote d’Azur University, 06200 Nice, FranceFrench Group of Myelodysplasia, 75010 Paris, FranceMoffit Cancer Center and Research Institute, Tampa, FL 33612, USAMoffit Cancer Center and Research Institute, Tampa, FL 33612, USATargeting TP53 mutated myelodysplastic syndromes and acute myeloid leukemia remains a significant unmet need. Recently, new drugs have attempted to improve the outcomes of this poor molecular subgroup. The aim of this article is to review all the current knowledge using active agents including hypomethylating agents with venetoclax, eprenetapopt or magrolimab. We include comprehensive analysis of clinical trials to date evaluating these drugs in TP53 myeloid neoplasms as well as discuss future novel combinations for consideration. Additionally, further understanding of the unique clinicopathologic components of TP53 mutant myeloid neoplasms versus wild-type is critical to guide future study. Importantly, the clinical trajectory of patients is uniquely tied with the clonal burden of TP53, which enables serial TP53 variant allele frequency analysis to be a critical early biomarker in investigational studies. Together, significant optimism is now possible for improving outcomes in this patient population.https://www.mdpi.com/1422-0067/22/18/10105MDSAMLTP53magrolimabeprenetapopt |
| spellingShingle | Thomas Cluzeau Michael Loschi Pierre Fenaux Rami Komrokji David A. Sallman Personalized Medicine for TP53 Mutated Myelodysplastic Syndromes and Acute Myeloid Leukemia International Journal of Molecular Sciences MDS AML TP53 magrolimab eprenetapopt |
| title | Personalized Medicine for TP53 Mutated Myelodysplastic Syndromes and Acute Myeloid Leukemia |
| title_full | Personalized Medicine for TP53 Mutated Myelodysplastic Syndromes and Acute Myeloid Leukemia |
| title_fullStr | Personalized Medicine for TP53 Mutated Myelodysplastic Syndromes and Acute Myeloid Leukemia |
| title_full_unstemmed | Personalized Medicine for TP53 Mutated Myelodysplastic Syndromes and Acute Myeloid Leukemia |
| title_short | Personalized Medicine for TP53 Mutated Myelodysplastic Syndromes and Acute Myeloid Leukemia |
| title_sort | personalized medicine for tp53 mutated myelodysplastic syndromes and acute myeloid leukemia |
| topic | MDS AML TP53 magrolimab eprenetapopt |
| url | https://www.mdpi.com/1422-0067/22/18/10105 |
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