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CANCER:

Myelodysplastic Syndrome

1d
HM2023-05: GTB-3650 Trike for High Risk MDS and R/R AML (clinicaltrials.gov)
P1, N=45, Recruiting, Masonic Cancer Center, University of Minnesota | Not yet recruiting --> Recruiting
Enrollment open • Trispecific
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GTB-3650
2d
Financial Navigation Study Among Young Adult Blood Cancer Survivors (clinicaltrials.gov)
P=N/A, N=130, Completed, Tufts Medical Center | Active, not recruiting --> Completed | N=260 --> 130 | Trial completion date: Jun 2024 --> Nov 2023
Trial completion • Enrollment change • Trial completion date
2d
225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome (clinicaltrials.gov)
P1, N=15, Recruiting, City of Hope Medical Center | Not yet recruiting --> Recruiting | Trial completion date: Feb 2027 --> May 2028 | Initiation date: Oct 2024 --> Jan 2025 | Trial primary completion date: Feb 2027 --> May 2028
Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date
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Darzalex (daratumumab) • sirolimus • melphalan • fludarabine IV
3d
RAPID: Safety of MT-401-OTS in Patients with Relapsed AML or MDS (clinicaltrials.gov)
P1, N=40, Not yet recruiting, Marker Therapeutics, Inc. | Trial completion date: May 2029 --> Sep 2029 | Initiation date: Oct 2024 --> Jan 2025 | Trial primary completion date: May 2029 --> Sep 2029
Trial completion date • Trial initiation date • Trial primary completion date
3d
Alpha-fetoprotein-producing intramucosal gastric cancer found during examination of metastatic lymph nodes. (PubMed, Clin J Gastroenterol)
The patient died of exacerbation of myelodysplastic syndrome 5 years 8 months postoperatively with no recurrence. Our experience suggests the need for further studies to validate whether the indications for endoscopic resection can apply to alpha-fetoprotein-producing gastric cancer in the same manner as to conventional gastric cancer.
Journal • Metastases
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AFP (Alpha-fetoprotein)
4d
NCI-2017-02205: Infusion of Expanded Cord Blood Cells in Addition to Single Cord Blood Transplant in Treating Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplastic Syndromes (clinicaltrials.gov)
P2, N=35, Recruiting, Fred Hutchinson Cancer Center | N=20 --> 35 | Trial completion date: Oct 2026 --> Mar 2027 | Trial primary completion date: Dec 2024 --> Mar 2025
Enrollment change • Trial completion date • Trial primary completion date
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HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule) • HLA-B (Major Histocompatibility Complex, Class I, B)
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cyclophosphamide • fludarabine IV • thiotepa • dilanubicel (DVX101)
5d
Phoenix: Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to MDS With del5q, Refractory/Resistant/Intolerant to Prior Treatments, RBC-TD (clinicaltrials.gov)
P2, N=22, Recruiting, Associazione Qol-one | Trial completion date: Nov 2028 --> Dec 2029 | Trial primary completion date: Apr 2024 --> Dec 2024
Trial completion date • Trial primary completion date
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Reblozyl (luspatercept-aamt)
5d
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
6d
A line in shifting sand: Can we define and target TP53 mutated MDS? (PubMed, Semin Hematol)
Our current understanding is that TP53 mutated MDS and AML are globally quite similar, but as a group have unique features compared to TP53 wildtype (WT) disease. Optimizing immunotherapy and targeting vulnerabilities due to co-mutations and/or chromosome abnormalities should be the focus of future research.
Journal • IO biomarker
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TP53 (Tumor protein P53)
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TP53 mutation • TP53 wild-type
6d
RNA splicing as a therapeutic target in myelodysplastic syndromes. (PubMed, Semin Hematol)
Emerging evidence shows that splicing factor-mutant cells are more sensitive to perturbations targeting the spliceosome, aberrantly spliced genes and/or their regulated molecular pathways. This review summarizes current therapeutic strategies and ongoing efforts targeting splicing factor mutations for the treatment of MDS.
Journal
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SF3B1 (Splicing Factor 3b Subunit 1) • SRSF2 (Serine and arginine rich splicing factor 2) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
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SF3B1 mutation • SRSF2 mutation • U2AF1 mutation
6d
The tubulin polymerization inhibitor gambogenic acid induces myelodysplastic syndrome cell apoptosis through upregulation of Fas expression mediated by the NF-κB signaling pathway. (PubMed, Cancer Biol Ther)
GNA combined with the MCL-1 inhibitor MIK665 potently suppressed the proliferation of MDS cells...GNA-induced apoptosis was attenuated in either p65 KO or Fas KO cells. These results demonstrate that GNA inhibited tubulin polymerization and induced apoptosis of MDS cells through upregulation of Fas expression mediated by the NF-κB signaling pathway, suggesting a chemotherapeutic strategy for MDS by microtubule dynamics disruption.
Journal
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CASP3 (Caspase 3) • CASP7 (Caspase 7)
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MCL1 expression
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MIK665
6d
Rapid Disease Progression of Myelodysplastic Syndrome is Reflected in Transcriptomic and Functional Abnormalities of Bone Marrow MSCs. (PubMed, Stem Cells)
Together, our data point towards a degree of co-development between MSCs and HSPCs during the progression of MDS, where changes in MDS-MSCs take place mainly at the transcriptomic and functional levels. These unique differences in MDS-MSCs can be utilized to improve disease prognostication and implement targeted therapy for unmet clinical needs.
Journal
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NFKB1 (Nuclear factor of kappa light polypeptide gene enhancer in B-cells 1)
6d
Long-term outcomes of PARP inhibitors in ovarian cancer: survival, adverse events, and post-progression insights. (PubMed, ESMO Open)
We emphasize the importance of long-term follow-up and real-world data coming from international registries to define the efficacy and safety of stopping PARPi at relapse at a pre-specified time. To this point, biomarkers able to identify the patients who will experience long-term remission with PARPi maintenance or develop early resistance are urgently needed to guide treatment decision and duration.
Journal • Adverse events • BRCA Biomarker • PARP Biomarker
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HRD (Homologous Recombination Deficiency) • BRCA (Breast cancer early onset)
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HRD
7d
GPR68 supports AML cells through the calcium/calcineurin pro-survival pathway and confers chemoresistance by mediating glucose metabolic symbiosis. (PubMed, Biochim Biophys Acta Mol Basis Dis)
As glucose metabolic symbiosis and the heterogeneous dependencies on aerobic glycolysis and cellular respiration tremendously impact chemosensitivity, the inhibition of GPR68 potentiated the tumoricidal effect of first-line chemotherapeutic agents, including BCL-2 inhibitors targeting OxPhos and cytarabine (AraC) targeting glycolysis...The overexpression of GPR68 drives a Ca2+/CaN pro-survival pathway and mediates glucose metabolic symbiosis in AML cells, suggesting the diagnostic and therapeutic potential of GPR68 in AML. (GPR68, G proton-coupled receptor 68; PLCβ, phospholipase C beta; CaN, Calcineurin; IDH, isocitrate dehydrogenase; HIF-1α, Hypoxia-inducible factor alpha subunit; GLUT1, Glucose transporter type 1; HK-1, Hexokinase 1).
Journal • IO biomarker
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HIF1A (Hypoxia inducible factor 1, alpha subunit) • CAPN1 (Calpain 1) • SLC2A1 (Solute Carrier Family 2 Member 1)
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cytarabine
7d
The IL-1β inhibitor canakinumab in previously treated lower-risk myelodysplastic syndromes: a phase 2 clinical trial. (PubMed, Nat Commun)
Sequential post-hoc prospective single-cell RNA sequencing analyses of HSPCs and bone marrow mononuclear cells at different time points during therapy showed that canakinumab's on-target effects in hematopoietic populations expressing the IL-1β receptor decreased the TNF-mediated inflammatory signaling pathway but rescued ineffective erythropoiesis only in the context of lower genetic complexity. This study demonstrates that better stratification strategies could target lower-risk MDS patients more effectively.
P2 data • Journal
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IL1B (Interleukin 1, beta)
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Ilaris (canakinumab)
7d
Diagnosis of TP53-mutated myeloid disease by the ICC and WHO 5th Edition Classifications. (PubMed, Blood Adv)
However, when compared to a separate cohort of patients with AML, MR without TP53 mutations, patients with TP53-mutated AML showed a distinct genetic profile and significantly worse overall survival, supporting the inclusion of AML with mutated TP53 as a distinct disease category. Overall, our results show that a significant percentage of TP53-mutated myeloid disease is classified differently by the ICC and WHO5 and highlight areas to address in future classification systems.
Journal
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TP53 (Tumor protein P53)
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TP53 mutation
7d
Clonal hematopoiesis in patients with autoimmune thrombocytopenia: an international multicenter study. (PubMed, Blood Adv)
Additionally, clonal hematopoiesis was associated with increased thrombotic risk (26% vs 8% in NGS-negative cases, p=0.01), independently from TPO-RA exposure, though with an age effect. These data demonstrated the prevalence of clonal hematopoiesis in 18% of adult ITP patients, being associated with older age, relapsed/refractory disease, and high risk of thrombotic complications.
Clinical • Journal
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DNMT3A (DNA methyltransferase 1) • ASXL1 (ASXL Transcriptional Regulator 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • SRSF2 (Serine and arginine rich splicing factor 2)
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DNMT3A mutation • TET2 mutation • SRSF2 mutation
7d
Novel Scoring System for Ranking Hematopoietic Stem Cell Transplantation. (PubMed, Clin Transplant)
This study suggests that HLA mismatches that do not alter peptide binding or orientation (Peptide Score = 0) could expand the number of permissive HLA mismatches. Further investigation is needed to confirm this observation and to explore alternative scoring systems for ranking HLA mismatched donors.
Retrospective data • Journal
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HLA-C (Major Histocompatibility Complex, Class I, C)
8d
Characterization of the Molecular Signature of Human Monocytes in Aging and Myelodysplastic Neoplasms. (PubMed, Eur J Immunol)
Aging boosts inflammatory genes upregulation, whereas MDS favors antigen presentation, reflecting distinct immune and disease-specific adaptations. • MDS shows reduced inflammatory activity in CD14+ cells, whereas CMML exhibits heightened inflammation, highlighting distinct disease mechanisms.
Journal
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CD14 (CD14 Molecule)
8d
Journal
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MYC (V-myc avian myelocytomatosis viral oncogene homolog)
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Vyxeos (cytarabine/daunorubicin liposomal formulation)
8d
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)
9d
Elevated serum direct bilirubin is predictive of a poor prognosis for primary myelodysplastic syndrome. (PubMed, BMC Cancer)
An elevated DBIL level was identified as an independent adverse prognostic factor for MDS patients. An individualized prediction model was established and validated to improve prediction of OS and LFS.
Retrospective data • Journal
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KRAS (KRAS proto-oncogene GTPase) • ASXL1 (ASXL Transcriptional Regulator 1) • B2M (Beta-2-microglobulin)
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KRAS mutation • ASXL1 mutation
9d
VYSION: VYxeoS Liposomal Italian Observational Study iN the Real Practice (clinicaltrials.gov)
P=N/A, N=112, Active, not recruiting, Jazz Pharmaceuticals | Recruiting --> Active, not recruiting
Enrollment closed
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Vyxeos (cytarabine/daunorubicin liposomal formulation)
10d
DNA and RNA NGS for Myeloid Neoplasms Using Oncomine Myeloid Assay GX v2 on GeneXus: An Assessment of Clinical Utility (AMP 2024)
This DNA- and RNA-based 80-gene panel has proven to be a powerful tool for genomic profiling of myeloid neoplasms. The results were provided to hematopathologists/oncologists in timely fashion with the critical information for diagnosis confirmation, and disease classification, as well as assessment of patient response to treatment.
Clinical • Next-generation sequencing
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KRAS (KRAS proto-oncogene GTPase) • TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • NRAS (Neuroblastoma RAS viral oncogene homolog) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • NPM1 (Nucleophosmin 1) • DNMT3A (DNA methyltransferase 1) • JAK2 (Janus kinase 2) • RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • KMT2A (Lysine Methyltransferase 2A) • TET2 (Tet Methylcytosine Dioxygenase 2) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11) • SRSF2 (Serine and arginine rich splicing factor 2) • BCOR (BCL6 Corepressor) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • CEBPA (CCAAT Enhancer Binding Protein Alpha) • STAG2 (Stromal Antigen 2) • DDX41 (DEAD-Box Helicase 41) • CALR (Calreticulin) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
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FLT3-ITD mutation • NPM1 mutation • U2AF1 mutation • CEBPA mutation • JAK2 V617F
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Oncomine Myeloid Assay GX
11d
NCI-2019-03057: Using the Anticancer Drug Olaparib to Treat Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome With an Isocitrate Dehydrogenase (IDH) Mutation (clinicaltrials.gov)
P2, N=14, Active, not recruiting, National Cancer Institute (NCI) | N=94 --> 14 | Trial completion date: Dec 2024 --> Nov 2025 | Trial primary completion date: Dec 2024 --> Jan 2024
Enrollment change • Trial completion date • Trial primary completion date
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IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
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IDH2 mutation • IDH1 R132H • IDH2 R172K • IDH1 R132C • IDH wild-type • IDH1 R132 • IDH1 R132G • IDH2 R140Q • IDH1 R132L • IDH1 R132S • IDH1 R132V • IDH2 R172 • IDH2 R172G
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Lynparza (olaparib)
11d
A Safety, Tolerability and Efficacy Study of NC525 in Subjects with Advanced Myeloid Neoplasms (clinicaltrials.gov)
P1, N=63, Active, not recruiting, NextCure, Inc. | Recruiting --> Active, not recruiting
Enrollment closed
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NC525
13d
New trial
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decitabine
13d
Efficacy of HLA epitope-matched platelet transfusion (ChiCTR2400091253)
P=N/A, N=700, Not yet recruiting, Qilu Hospital of Shandong University; Qilu Hospital of Shandong University
New trial
13d
Study of an application-delivered mindfulness-based intervention to manage fear of progression for patients with hematologic malignancies (ChiCTR2400090963)
P=N/A, N=90, Completed, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College; Institute of Hematology
New trial
13d
A novel myeloablative pretreatment scheme for single arm, single center, exploratory study of umbilical cord blood transplantation (ChiCTR2400090099)
P=N/A, N=30, Not yet recruiting, The Seventh Affiliated Hospital Sun Yat-sen University; The?Seventh?Affiliated?Hospital?Sun?Yat-sen?University
New trial
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cyclophosphamide • fludarabine IV
13d
Phase II clinical study on the efficacy, safety, and pharmacokinetics of HS-20106 in IPSS-R extremely low-risk, low-risk, or moderate risk myelodysplastic syndrome (MDS) anemia subjects (ChiCTR2400090140)
P2, N=176, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College.; Institute of Hematolog
New P2 trial
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elritercept (KER-050)
16d
MCC-20963: Fedratinib in Myelodysplastic /Myeloproliferative Neoplasms (MDS/MPNs) and Chronic Neutrophilic Leukemia (CNL) (clinicaltrials.gov)
P2, N=25, Active, not recruiting, H. Lee Moffitt Cancer Center and Research Institute | Recruiting --> Active, not recruiting
Enrollment closed
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ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase)
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Inrebic (fedratinib)
16d
Trial suspension • Combination therapy
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Venclexta (venetoclax)
16d
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
Genetic abnormalities in bone marrow failure (PubMed, Rinsho Ketsueki)
Notably, the presence of PNH-type cells and HLA class I allele-lacking cells represent two major instances of escape hematopoiesis, which indicate the presence of HSCs evading autoimmune T cell attacks. These findings provide crucial insights into the immune pathophysiology of BM failure.
Journal
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DNMT3A (DNA methyltransferase 1) • ASXL1 (ASXL Transcriptional Regulator 1) • BCOR (BCL6 Corepressor) • BCORL1 (BCL6 Corepressor Like 1)
17d
Validation of the Revised 2022 European LeukemiaNet Risk Stratification in Adult Patients with Acute Myeloid Leukemia. (PubMed, Blood Adv)
We aimed to validate the prognostic value of the 2022 ELN classification (ELN22) evaluating 1,570 newly diagnosed AML patients (median age, 56 years) treated with cytarabine-based intensive chemotherapy regimens...In conclusion, the ELN22 risk stratification improves prognostic discrimination in a large cohort of intensively treated AML patients. Given the heterogeneous outcome in patients with MR gene alterations, ranging between those of intermediate and adverse risk patients, we suggest reevaluation of risk allocation in these patients.
Journal
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TP53 (Tumor protein P53) • EZH2 (Enhancer of zeste 2 polycomb repressive complex 2 subunit) • STAG2 (Stromal Antigen 2) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
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TP53 mutation • EZH2 mutation • STAG2 mutation • ZRSR2 mutation
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cytarabine
17d
BoHemE: Study Investigating the Interactions of Bone and Hematopoiesis in the Elderly (clinicaltrials.gov)
P=N/A, N=297, Completed, Technische Universität Dresden | Recruiting --> Completed | Trial completion date: Dec 2023 --> Aug 2024 | Trial primary completion date: Dec 2023 --> Aug 2024
Trial completion • Trial completion date • Trial primary completion date
17d
Alpha/Beta CD19+ Depleted Haploidentical Transplantation + Zometa for Pediatric Hematologic Malignancies and Solid Tumors (clinicaltrials.gov)
P1, N=22, Recruiting, University of Wisconsin, Madison | Suspended --> Recruiting | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2024 --> Dec 2025
Enrollment open • Trial completion date • Trial primary completion date
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melphalan • zoledronic acid
17d
Trial completion date
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Venclexta (venetoclax) • azacitidine • pevonedistat (MLN4924)
17d
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)