Tamibarotene-based therapy is a novel targeted approach for the treatment of relapsed/refractory (R/R) acute myeloid leukemia (AML) with retinoic acid receptor alpha () gene overexpression. Approximately, 50% of higher-risk myelodysplastic syndrome (MDS) patients and approximately 30% of AML patients are positive for overexpression using a blood-based biomarker test that measures expression in peripheral blasts. A phase 2 study investigating the activity of tamibarotene in patients with overexpression was conducted in patients with AML and MDS (NCT02807558). In 28 patients with R/R AML and overexpression treated with tamibarotene in combination with azacitidine, the median overall survival was 5.9 months. In 21 response-evaluable patients, the complete remission/complete remission with incomplete hematologic recovery (CR/CRi) rate was 19%, and median time to initial CR/CRi was 1.2 months. The favorable safety profile and preliminary clinical activity support the development of combination therapies with tamibarotene in myeloid malignancies with overexpression.
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http://dx.doi.org/10.1080/10428194.2023.2243356 | DOI Listing |
Br J Haematol
December 2024
Division of Hematology, Department of Medicine, University of Miami Sylvester Comprehensive Cancer Center, Miami, Florida, USA.
Acute myeloid leukaemia (AML) arising from a myeloproliferative neoplasm (MPN) is more aggressive and less responsive to therapies compared to de novo AML. Olutasidenib, an oral small-molecule inhibitor of mutated IDH1 (mIDH1), showed encouraging and durable responses in a phase 1/2 study of adults with post-MPN mIDH1 AML. Patients received olutasidenib 150 mg BID monotherapy or in combination with azacitidine.
View Article and Find Full Text PDFCancer Manag Res
December 2024
Afyonkarahisar Health Sciences University Department of Medical Services and Techniques, Afyonkarahisar, Türkiye.
Objective: Myeloid leukemia is a stem cell disease with high mortality due to the challenges of high-dose treatments and side effects. Innovative nanoparticle drug delivery systems are being explored to enhance efficacy and tissue-targeted therapy. This study investigates the potential of Bentonite (BNT)-based nanoparticles (NPs) as drug carriers for azacitidine (AZA) in treating THP-1 and K562 myeloid leukemia (AML) cell lines, aiming to improve drug stability, bioavailability, and therapeutic efficacy while ensuring controlled release.
View Article and Find Full Text PDFCancer Rep (Hoboken)
December 2024
Department of Otolaryngology, Shidong Hospital, Yangpu District, Shidong Hospital Affiliated to University of Shanghai for Science and Technology, Shanghai, China.
Background: Methyl-CpG-binding domain 2 (MBD2) attaches to methylated DNA, which mediates methylated gene transcription, leading to gene silencing and affecting tumor progression. The molecular mechanisms of MBD2 in head and neck squamous cell carcinoma (HNSCC) remain insufficiently characterized.
Aims: This study sought to assess the clinical relevance of MBD2 expression in HNSCC, with a particular focus on elucidating its functional role in tumor progression and its regulatory influence on p21 expression and cellular proliferation.
Ann Hematol
December 2024
National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Shizi Street 188, Suzhou, 215006, P. R. China.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is theoretically the only curative option for high-risk myelodysplastic syndrome (HR MDS) patients. However, the management of patients with relapsed disease post allo-HSCT remains a challenge with few standard treatments. Chidamide, a new selective histone deacetylase, has shown synergistic anti-leukemia effect combined with azacitidine in acute myeloid leukemia patients.
View Article and Find Full Text PDFHematology
December 2024
Department of Hematology, Taixing People's Hospital Affiliated to Yangzhou University, Taixing, People's Republic of China.
Objectives: A combination of hypomethylation agents (HMA) and the HAG regimen (homoharringtonine, cytarabine, G-CSF) shows promise as a treatment for Acute Myeloid Leukemia (AML). Nevertheless, the clinical efficacy of this combined therapy in contrast to the HAG regimen alone remains uncertain.
Methods: We conducted a meta-analysis of eligible studies comparing the clinical efficacy of these two regimens.
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