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DRUG:

azacitidine

i
Other names: NS-17, Aza C, NSC-102816, U-18496, Aza-C, NS 17
Company:
Generic mfg.
Drug class:
DNMT inhibitor
1d
New P2 trial • Combination therapy
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Venclexta (venetoclax) • Xospata (gilteritinib) • azacitidine
2d
VENETACIBLE: Plasma Dosage of Venetoclax in the Fup of AML Patients Treated With Aza + Ven (clinicaltrials.gov)
P=N/A, N=20, Active, not recruiting, Centre Hospitalier Universitaire de Nice | Recruiting --> Active, not recruiting
Enrollment closed
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Venclexta (venetoclax) • azacitidine
3d
Venetoclax and Azacitidine Combined With Chidamide (VAC) for the Treatment of Newly Diagnosed Acute Monocytic Leukemia (clinicaltrials.gov)
P2, N=92, Recruiting, The First Affiliated Hospital of Soochow University | Trial primary completion date: Sep 2024 --> Sep 2025
Trial primary completion date
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BCL2 (B-cell CLL/lymphoma 2)
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Venclexta (venetoclax) • azacitidine • Epidaza (chidamide)
4d
Venetoclax and Azacitidine for the Treatment of Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
P1/2, N=34, Active, not recruiting, M.D. Anderson Cancer Center | Recruiting --> Active, not recruiting | N=58 --> 34
Enrollment closed • Enrollment change • Combination therapy
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Venclexta (venetoclax) • azacitidine
5d
Translational Research on Azacitidine Post-Remission Therapy of Acute Myeloid Leukemia in Elderly Patients (QOL-ONE Trans-2). (PubMed, Int J Mol Sci)
FANCA mutations in four patients were linked to a higher relapse risk (HR = 4.96, p = 0.02) for DFS at both 2 and 5 years. Further HLA-specific NGS analyses are ongoing to confirm these results and their therapeutic implications.
Clinical • Journal
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NPM1 (Nucleophosmin 1) • DNMT3A (DNA methyltransferase 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • FANCA (FA Complementation Group A)
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NPM1 mutation • DNMT3A mutation • FANCA mutation
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azacitidine
5d
Replenishing co-downregulated miR-100-5p and miR-125b-5p in malignant germ cell tumors causes growth inhibition through cell cycle disruption. (PubMed, Mol Oncol)
Cells were treated with demethylating 5-azacytidine and pyrosequencing was performed...In summary, combination miR-100-5p/miR-125b-5p mimic replenishment or TRIM71kd caused growth inhibition in malignant GCT cells via cell cycle disruption. Further studies are now warranted, including mimic treatment alongside conventional platinum-based chemotherapy.
Journal
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MIR100 (MicroRNA 100) • MIR99A (MicroRNA 99a) • MIRLET7B (MicroRNA Let-7b) • TRIM7 (Tripartite Motif Containing 7)
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azacitidine
7d
P30CA036727: Geriatric Assessment & Genetic Profiling to Personalize Therapy in Older Adults With Acute Myeloid Leukemia (clinicaltrials.gov)
P2, N=75, Completed, University of Nebraska | Active, not recruiting --> Completed | Trial primary completion date: Oct 2024 --> Mar 2024
Trial completion • Trial primary completion date
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Venclexta (venetoclax) • azacitidine • Rydapt (midostaurin) • decitabine • Mylotarg (gemtuzumab ozogamicin) • Vyxeos (cytarabine/daunorubicin liposomal formulation) • idarubicin hydrochloride • Daurismo (glasdegib) • Starasid (cytarabine ocfosfate)
7d
Antineoplastic therapy affects the in vitro phenotype and functionality of healthy human bone marrow-derived mesenchymal stromal cells. (PubMed, Arch Toxicol)
To address this, we investigated the effects of etoposide, temozolomide, 5-azacitidine, and venetoclax on healthy BM-derived MSC functionality. Our findings show that besides HSPC, also MSC are sensitive to certain antineoplastic agents, resulting in molecular and functional alterations that may contribute to therapy-related myelosuppression. Understanding these interactions could be helpful for the development of strategies to preserve BM MSC functionality during different kinds of anticancer therapies.
Preclinical • Journal • Stroma
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CDKN1A (Cyclin-dependent kinase inhibitor 1A)
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Venclexta (venetoclax) • temozolomide • azacitidine • etoposide IV
7d
Homoharringtonine Added to Venetoclax and Azacitidine Improves Outcome and Mitigates Genetic Impact in Relapsed/Refractory AML: A Multi-center Cohort Study. (PubMed, Clin Cancer Res)
Our findings suggest the addition of HHT to VA might enhance response and mitigate the negative impact of certain genetic patterns in RR-AML while being well tolerated.
Journal
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KRAS (KRAS proto-oncogene GTPase) • FLT3 (Fms-related tyrosine kinase 3) • NPM1 (Nucleophosmin 1) • DNMT3A (DNA methyltransferase 1) • TET2 (Tet Methylcytosine Dioxygenase 2)
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KRAS mutation • FLT3-ITD mutation • FLT3 mutation • DNMT3A mutation • TET2 mutation
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Venclexta (venetoclax) • azacitidine • Synribo (omacetaxine mepesuccinate)
9d
Venetoclax and Azacitidine for Non-Elderly Adult Patients With Acute Myeloid Leukemia (clinicaltrials.gov)
P2, N=36, Active, not recruiting, University of Colorado, Denver | Recruiting --> Active, not recruiting
Enrollment closed
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Venclexta (venetoclax) • azacitidine
9d
Demethylating Agents Combined with Venetoclax for High-risk T-Cell Lymphoma/leukemia Post-Transplant Relapse Prevention (clinicaltrials.gov)
P2, N=59, Recruiting, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
New P2 trial • Post-transplantation
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Venclexta (venetoclax) • azacitidine • decitabine
10d
STIMULUS-AML2: Sabatolimab as a Treatment for Patients With Acute Myeloid Leukemia and Presence of Measurable Residual Disease After Allogeneic Stem Cell Transplantation. (clinicaltrials.gov)
P1/2, N=24, Completed, Novartis Pharmaceuticals | Active, not recruiting --> Completed | Trial completion date: Dec 2024 --> Aug 2024 | Trial primary completion date: Dec 2024 --> Aug 2024
Trial completion • Trial completion date • Trial primary completion date
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azacitidine • sabatolimab (MBG453)
12d
New trial
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Venclexta (venetoclax) • azacitidine • Synribo (omacetaxine mepesuccinate)
12d
A prospective, single-center, single-arm study of mitoxantrone hydrochloride liposome injection combined with venetoclax and azacitidine in the treatment of newly diagnosed and relapsed/refractory acute myeloid leukemia (ChiCTR2400091259)
P4, N=40, Not yet recruiting, General Hospital of Chinese People's Liberation Army Northern Theater Command; General Hospital of Chinese People's Liberation Army Northern Theater C
New P4 trial
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Venclexta (venetoclax) • azacitidine • Duoenda (mitoxantrone liposomal)
15d
5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies (clinicaltrials.gov)
P1, N=20, Recruiting, Benjamin Tomlinson | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2024 --> Dec 2025
Trial completion date • Trial primary completion date
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ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • CD14 (CD14 Molecule) • DCK (Deoxycytidine Kinase 2) • DNMT1 (DNA methyltransferase 1) • ITGAM (Integrin, alpha M) • NT5C (5', 3'-Nucleotidase, Cytosolic)
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Chr del(5q)
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azacitidine • decitabine
16d
PEMAZA: MRD-guided Treatment in NPM1mut AML Patients (clinicaltrials.gov)
P2, N=28, Recruiting, Technische Universität Dresden | Trial completion date: Aug 2024 --> Dec 2026 | Trial primary completion date: Aug 2023 --> Dec 2025
Trial completion date • Trial primary completion date
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NPM1 mutation
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Keytruda (pembrolizumab) • azacitidine
16d
Trial completion date
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Venclexta (venetoclax) • azacitidine • pevonedistat (MLN4924)
16d
Impact of Mutational Landscape and CD47 Levels in the Magrolimab + Azacitidine Vs Placebo + Azacitidine Trial in Patients with Untreated Higher Risk Myelodysplastic Syndrome (ASH 2024)
Given the imbalanced cytogenetic risk between CD47 high and CD47 low in the magrolimab+ aza arm, the observation warrants further investigation into the connection between cytogenetic risk, CD47 levels, and CD47 blockade. Additional data with respect to the mutational landscape will be forthcoming, including central evaluation of TP53 status.
Clinical
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TP53 (Tumor protein P53) • CD47 (CD47 Molecule) • CALR (Calreticulin) • SIRPA (Signal Regulatory Protein Alpha)
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TP53 mutation • CD47 overexpression • CD47 expression
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TruSight Oncology 500 Assay
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azacitidine • magrolimab (ONO-7913)
17d
The implication of TET1, miR-200, and miR-494 expression with tumor formation in colorectal cancer: through targeting Wnt signaling. (PubMed, Mol Biol Rep)
Overall, our findings shed light on the intricate interactions between TET1, Wnt1, and specific miRNAs in colorectal cancer (CRC) and their potential implications for diagnosis and treatment.
Journal
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TET1 (Tet Methylcytosine Dioxygenase 1) • MIR200A (MicroRNA 200a) • MIR494 (MicroRNA 494) • MIR200 (MicroRNA 200)
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miR-200-a expression
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azacitidine
18d
Pevonedistat and Azacitidine in MDS or MDS/MPN Patients Who Fail Primary Therapy With DNA Methyl Transferase Inhibitors (clinicaltrials.gov)
P2, N=71, Completed, Vanderbilt-Ingram Cancer Center | Active, not recruiting --> Completed | Trial completion date: Oct 2024 --> Nov 2023
Trial completion • Trial completion date • Tumor mutational burden
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azacitidine • pevonedistat (MLN4924)
21d
Single-cell analysis of bone marrow CD8+ T cells in Myeloid Neoplasms reveals pathways associated with disease progression and response to treatment with Azacitidine. (PubMed, Cancer Res Commun)
The response to AZA was positively associated with the enrichment of IFN-mediated pathways, whereas an enhanced TGF-β signaling signature was observed in non-responders. Our results suggest that targeting CD8+ T cells with inhibitors of TGF-β signaling in combination with AZA is a potential therapeutic strategy for HR-MDS and AML.
Journal
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CD8 (cluster of differentiation 8) • TGFB1 (Transforming Growth Factor Beta 1) • CXCR3 (C-X-C Motif Chemokine Receptor 3) • B3GAT1 (Beta-1,3-Glucuronyltransferase 1)
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azacitidine
21d
Phase I First-in-Human Dose Escalation Study of the oral Casein Kinase 1α and Cyclin Dependent Kinase 7/9 inhibitor BTX-A51 in advanced MDS and AML. (PubMed, Res Sq)
Ex-vivo studies confirmed higher efficacy of BTX-A51 on RUNX1 -mutated myeloblasts and demonstrate synergy with azacitidine and venetoclax. Although the overall efficacy was modest, this study lays the groundwork for future studies with improved patient selection and combination approaches.
P1 data • Journal • Metastases
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RUNX1 (RUNX Family Transcription Factor 1) • CDK7 (Cyclin Dependent Kinase 7)
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RUNX1 mutation • MCL1 expression • TP53 expression
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Venclexta (venetoclax) • azacitidine • BTX-A51
22d
VERDI: Venetoclax + Azacitidine in Patients with Acute Myeloid Leukemia (clinicaltrials.gov)
P2, N=29, Not yet recruiting, Grupo Cooperativo de Estudio y Tratamiento de las Leucemias Agudas y Mielodisplasias
New P2 trial
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Venclexta (venetoclax) • azacitidine
23d
A review of the isocitrate dehydrogenase inhibitors in management of adult patients with AML and MDS. (PubMed, Expert Rev Hematol)
Olutasidenib, enasidenib, and ivosidenib are approved for relapsed AML...Ivosidenib + azacitidine demonstrated a survival benefit not seen with enasidenib + azacitidine. It is unclear whether newly diagnosed AML should be treated with azacitidine + ivosidenib or azacitidine + venetoclax...Single arm studies show post-transplant maintenance is safe, however, randomized trials are needed. Similarly, IDH inhibitors can be combined with chemotherapy however randomized studies are needed.
Review • Journal
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IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
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Venclexta (venetoclax) • azacitidine • Tibsovo (ivosidenib) • Idhifa (enasidenib) • Rezlidhia (olutasidenib)
23d
Enrollment open • Metastases
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azacitidine • Niktimvo (axatilimab-csfr)
24d
BGB-11417-103: A Study of BGB-11417 in Participants With Myeloid Malignancies (clinicaltrials.gov)
P1/2, N=260, Recruiting, BeiGene | Phase classification: P1b/2 --> P1/2 | Trial completion date: Aug 2025 --> Feb 2028 | Trial primary completion date: Dec 2023 --> Feb 2028
Phase classification • Trial completion date • Trial primary completion date
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azacitidine • sonrotoclax (BGB-11417) • Noxafil (posaconazole)
24d
Enrollment open
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azacitidine • Elzonris (tagraxofusp-erzs)
25d
Testing the Combination of an Anti-Cancer Drug, Iadademstat, With Other Anti-Cancer Drugs (Venetoclax and Azacitidine) for Treating AML (clinicaltrials.gov)
P1, N=45, Recruiting, National Cancer Institute (NCI) | Not yet recruiting --> Recruiting | Initiation date: Oct 2024 --> Jul 2025
Enrollment open • Trial initiation date
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TP53 (Tumor protein P53) • BCL2 (B-cell CLL/lymphoma 2) • NF1 (Neurofibromin 1) • MCL1 (Myeloid cell leukemia 1) • BCL2L1 (BCL2-like 1) • BAX (BCL2-associated X protein)
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BCL2 mutation • MCL1 expression
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Venclexta (venetoclax) • azacitidine • iadademstat (ORY-1001)
25d
Trial initiation date • Combination therapy
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Venclexta (venetoclax) • azacitidine • Elzonris (tagraxofusp-erzs)
25d
Trial completion • Combination therapy
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azacitidine • sabatolimab (MBG453)
26d
Blast Transformation of Chronic Myeloid Leukemia Driven by Acquisition of t(8;21)(q22;q22)/RUNX1::RUNX1T1: Selecting Optimal Treatment Based on Clinical and Molecular Findings. (PubMed, Biomedicines)
We report the case of a 63-year-old woman with a history of CML with poor response to imatinib who progressed to myeloid BP-CML, driven by the acquisition of t(8;21)(q22;q22)/RUNX1::RUNX1T1. The patient received intensive chemotherapy and dasatinib, followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT)...Ponatinib and azacitidine were started as salvage treatment, allowing for the achievement of complete remission with deep molecular response after five cycles. Advances in the knowledge of disease biology and clonal evolution are crucial for optimal treatment selection, which ultimately translates into better patient outcomes.
Journal
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ABL1 (ABL proto-oncogene 1) • RUNX1 (RUNX Family Transcription Factor 1) • RUNX1T1 (RUNX1 Partner Transcriptional Co-Repressor 1)
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ABL1 T315I
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dasatinib • imatinib • Iclusig (ponatinib) • azacitidine
29d
Enrollment change • Combination therapy
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NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A)
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Venclexta (venetoclax) • cytarabine • azacitidine • daunorubicin • idarubicin hydrochloride • bleximenib (JNJ-6617)
1m
Revumenib in Combination With Azacitidine + Venetoclax in Patients NPM1-mutated or KMT2A-rearranged AML (clinicaltrials.gov)
P3, N=415, Not yet recruiting, Stichting Hemato-Oncologie voor Volwassenen Nederland
New P3 trial • Combination therapy
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NPM1 (Nucleophosmin 1)
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NPM1 mutation
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Venclexta (venetoclax) • azacitidine • Revuforj (revumenib)
1m
Azacytidine treatment affects the methylation pattern of genomic and cell-free DNA in uveal melanoma cell lines. (PubMed, BMC Cancer)
This data suggests that DNMT inhibitors cause changes in UM cells that are maintained in cfDNA. The results suggest that targeting methylation in UM treatment and monitoring response to treatment using cfDNA methylation could be a valuable tool.
Preclinical • Journal
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BAP1 (BRCA1 Associated Protein 1)
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azacitidine
1m
Acute myeloid leukemia with NPM1, IDH2, and SETD2 mutations mimicking acute promyelocytic leukemia: A case report and literature review. (PubMed, Medicine (Baltimore))
The comprehensive evaluation of bone marrow morphology, immunology, cytogenetics, and molecular biology is essential for the accurate diagnosis of acute myeloid leukemia.
Review • Journal
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IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • NPM1 (Nucleophosmin 1) • SETD2 (SET Domain Containing 2, Histone Lysine Methyltransferase)
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NPM1 mutation • SETD2 mutation
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Venclexta (venetoclax) • azacitidine
1m
Pitavastatin in Combination With Venetoclax for Chronic Lymphocytic Leukemia or Acute Myeloid Leukemia (clinicaltrials.gov)
P1, N=6, Active, not recruiting, University of California, Irvine | Trial completion date: Jun 2024 --> Jun 2026 | Trial primary completion date: Apr 2023 --> Jul 2024
Trial completion date • Trial primary completion date • Combination therapy
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Venclexta (venetoclax) • azacitidine • pitavastatin
1m
New P3 trial • Combination therapy
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azacitidine • timdarpacept (IMM01)
1m
Ruxolitinib combined with venetoclax and azacitidine in the treatment of refractory T-ALL patients with JAK1, JAK3, and STAT5B gene mutations: a case report and literature review (PubMed, Zhonghua Xue Ye Xue Za Zhi)
Notably, pleural effusion and mediastinal mass significantly improved the post-chest cavity infusion of dexamethasone combined with etoposide at the same stage. The patient also underwent allogeneic hematopoietic stem cell transplantation upon achieving bone marrow remission and was followed up until January 2024. Ruxolitinib combined with venetoclax and azacytidine has shown promising efficacy and safety in treating refractory T-ALL harboring the JAK1, JAK3, and STAT5B mutations, providing a novel therapeutic approach for such patients.
Review • Journal
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JAK1 (Janus Kinase 1) • JAK3 (Janus Kinase 3) • STAT5B (Signal Transducer And Activator Of Transcription 5B)
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JAK3 mutation
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Venclexta (venetoclax) • azacitidine • Jakafi (ruxolitinib) • etoposide IV • dexamethasone