Skip to main content
Erschienen in:

18.07.2024 | short review

Trends in MDS and AML from ASH 2023

verfasst von: Prof. Dr. Michael Pfeilstöcker

Erschienen in: memo - Magazine of European Medical Oncology | Ausgabe 3/2024

Einloggen, um Zugang zu erhalten

Summary

Presentations in the field of clonal myeloid diseases from the 65th American Society of Hematology conference provided insights into future therapeutic approaches especially for the subset of older patients. While new drugs and concepts for acute myeloid leukemia need further work before clinical implementation, some data on myelodysplastic neoplasms providing new treatment options for transfusion-dependent low-risk patients are already practice-changing.
Literatur
1.
Zurück zum Zitat Issa GC, Ravandi F, DiNardo C, et al. Therapeutic implications of menin inhibition in acute leukemias. Leukemia. 2021;35:2482–95.CrossRefPubMed Issa GC, Ravandi F, DiNardo C, et al. Therapeutic implications of menin inhibition in acute leukemias. Leukemia. 2021;35:2482–95.CrossRefPubMed
2.
Zurück zum Zitat Aldos I, Issa GC, Thirman M, et al. Revumenib monotherapy in patients with relapsed/refractory KMT2Ar acute leukemia: topline efficacy and safety results from the pivotal augment-101 phase 2 study. LBA‑5. ASH 2023. Aldos I, Issa GC, Thirman M, et al. Revumenib monotherapy in patients with relapsed/refractory KMT2Ar acute leukemia: topline efficacy and safety results from the pivotal augment-101 phase 2 study. LBA‑5. ASH 2023.
3.
Zurück zum Zitat Issa GC, Cuglievan B, DiNardo CD, et al. Early results of the phase I/II study investigating the all-oral combination of the menin inhibitor revumenib (SNDX-5613) with Decitabine/Cedazuridine (ASTX727) and Venetoclax in acute Myeloid leukemia (SAVE). Blood. 2023;142(Supplement 1):58.CrossRef Issa GC, Cuglievan B, DiNardo CD, et al. Early results of the phase I/II study investigating the all-oral combination of the menin inhibitor revumenib (SNDX-5613) with Decitabine/Cedazuridine (ASTX727) and Venetoclax in acute Myeloid leukemia (SAVE). Blood. 2023;142(Supplement 1):58.CrossRef
4.
Zurück zum Zitat Jabbour E, Searle E, Abdul-Hay M, et al. A first-in-human phase 1 study of the Menin-KMT2A (MLL1) inhibitor JNJ-75276617 in adult patients with relapsed/refractory acute leukemia harboring KMT2A or NPM1 alterations. Blood. 2023;142(Supplement 1):57.CrossRef Jabbour E, Searle E, Abdul-Hay M, et al. A first-in-human phase 1 study of the Menin-KMT2A (MLL1) inhibitor JNJ-75276617 in adult patients with relapsed/refractory acute leukemia harboring KMT2A or NPM1 alterations. Blood. 2023;142(Supplement 1):57.CrossRef
5.
Zurück zum Zitat Yang L, Chen F, Liang H, et al. Selinexor in combination with venetoclax and azacitidine for newly diagnosed (ND) unfit acute myeloid leukemia (AML): a multicenter, open-label prospective study. Blood. 2023;142(Supplement 1):55.CrossRef Yang L, Chen F, Liang H, et al. Selinexor in combination with venetoclax and azacitidine for newly diagnosed (ND) unfit acute myeloid leukemia (AML): a multicenter, open-label prospective study. Blood. 2023;142(Supplement 1):55.CrossRef
6.
Zurück zum Zitat Angelucci E, Deconinck E, Manteigas D, et al. Durable outcomes with manageable safety leading to prolonged survival with tagraxofusp for treatment-naive patients with blastic plasmacytoid dendritic cell neoplasm: updated results from a European named patient program. Blood. 2023;142(Supplement 1):547.CrossRef Angelucci E, Deconinck E, Manteigas D, et al. Durable outcomes with manageable safety leading to prolonged survival with tagraxofusp for treatment-naive patients with blastic plasmacytoid dendritic cell neoplasm: updated results from a European named patient program. Blood. 2023;142(Supplement 1):547.CrossRef
7.
Zurück zum Zitat Minetto P, Rosellini S, Guolo F, et al. Single agent Tagraxofusp in relapsed/refractory CD123-positive acute myeloid leukemia: a preliminary analysis of Italian Gimema AML2020 trial. Blood. 2023;142(Supplement 1):2918.CrossRef Minetto P, Rosellini S, Guolo F, et al. Single agent Tagraxofusp in relapsed/refractory CD123-positive acute myeloid leukemia: a preliminary analysis of Italian Gimema AML2020 trial. Blood. 2023;142(Supplement 1):2918.CrossRef
8.
Zurück zum Zitat Lane AA, Garcia JS, Raulston EG, et al. Tagraxofusp in combination with Azacitidine and Venetoclax in newly diagnosed CD123+ acute Myeloid leukemia, expansion cohort of a phase 1b multicenter trial. Blood. 2023;142(Supplement 1):4277.CrossRef Lane AA, Garcia JS, Raulston EG, et al. Tagraxofusp in combination with Azacitidine and Venetoclax in newly diagnosed CD123+ acute Myeloid leukemia, expansion cohort of a phase 1b multicenter trial. Blood. 2023;142(Supplement 1):4277.CrossRef
9.
Zurück zum Zitat Nguyen D, Ravandi F, Wang SA, et al. A phase II study of Vibecotamab, a CD3-CD123 Bispecific T‑cell engaging antibody, for MDS or CMML after Hypomethylating failure and in MRD-positive AML. Blood. 2023;142(Supplement 1):322.CrossRef Nguyen D, Ravandi F, Wang SA, et al. A phase II study of Vibecotamab, a CD3-CD123 Bispecific T‑cell engaging antibody, for MDS or CMML after Hypomethylating failure and in MRD-positive AML. Blood. 2023;142(Supplement 1):322.CrossRef
10.
Zurück zum Zitat Choe H, Tomlinson BK, Gyurkocza B, et al. 131 I‑Apamistamab-led Allogeneic hematopoietic cell transplant significantly improves OS in patients with TP53 mutated R/R AML. Blood. 2023;142(Supplement 1):469.CrossRef Choe H, Tomlinson BK, Gyurkocza B, et al. 131 I‑Apamistamab-led Allogeneic hematopoietic cell transplant significantly improves OS in patients with TP53 mutated R/R AML. Blood. 2023;142(Supplement 1):469.CrossRef
11.
Zurück zum Zitat Yilmaz M, Muftuoglu M, DiNardo CD, et al. Phase I/II study of Quizartinib, Venetoclax, and Decitabine triple combination in FLT3-ITD mutated AML. Blood. 2023;142(Supplement 1):158.CrossRef Yilmaz M, Muftuoglu M, DiNardo CD, et al. Phase I/II study of Quizartinib, Venetoclax, and Decitabine triple combination in FLT3-ITD mutated AML. Blood. 2023;142(Supplement 1):158.CrossRef
12.
Zurück zum Zitat Ruhnke L, Schliemann C, Mikesch J‑H, et al. Venetoclax plus high-dose Cytarabine and Mitoxantrone (HAM-Ven) as salvage treatment for relapsed/refractory AML: updated results of the phase-I/II SAL relax trial. Blood. 2023;142(Supplement 1):160.CrossRef Ruhnke L, Schliemann C, Mikesch J‑H, et al. Venetoclax plus high-dose Cytarabine and Mitoxantrone (HAM-Ven) as salvage treatment for relapsed/refractory AML: updated results of the phase-I/II SAL relax trial. Blood. 2023;142(Supplement 1):160.CrossRef
13.
Zurück zum Zitat Bazinet A, Garcia-Manero G, Short NJ, et al. A phase 2 study of the fully oral combination of ASTX727 (Decitabine/Cedazuridine) plus Venetoclax for older and/or unfit patients with acute Myeloid leukemia. Blood. 2023;142(Supplement 1):833.CrossRef Bazinet A, Garcia-Manero G, Short NJ, et al. A phase 2 study of the fully oral combination of ASTX727 (Decitabine/Cedazuridine) plus Venetoclax for older and/or unfit patients with acute Myeloid leukemia. Blood. 2023;142(Supplement 1):833.CrossRef
14.
Zurück zum Zitat Atluri H, Mullin J, Takahashi K, et al. Phase Ib/2 study of oral decitabine/cedazuridine (ASTX727) and Venetoclax in combination with the targeted mutant IDH1 inhibitor Ivosidenib or the targeted mutant IDH2 inhibitor Enasidenib: 2023 update. Blood. 2023;142(Supplement 1):968.CrossRef Atluri H, Mullin J, Takahashi K, et al. Phase Ib/2 study of oral decitabine/cedazuridine (ASTX727) and Venetoclax in combination with the targeted mutant IDH1 inhibitor Ivosidenib or the targeted mutant IDH2 inhibitor Enasidenib: 2023 update. Blood. 2023;142(Supplement 1):968.CrossRef
15.
Zurück zum Zitat Othman J, Potter N, Ivey A, et al. The benefit of allogeneic transplant in 1st complete remission in NPM1 mutated AML with or without FLT3 ITD is restricted to those testing MRD positive after induction—an analysis of the UK NCRI AML17 and AML19 studies. Blood. 2023;142(Supplement 1):425.CrossRef Othman J, Potter N, Ivey A, et al. The benefit of allogeneic transplant in 1st complete remission in NPM1 mutated AML with or without FLT3 ITD is restricted to those testing MRD positive after induction—an analysis of the UK NCRI AML17 and AML19 studies. Blood. 2023;142(Supplement 1):425.CrossRef
16.
Zurück zum Zitat Orvain C, Bertoli S, Peterlin P, et al. Molecular relapse after first-line intensive therapy in patients with core-binding factor and NPM1—mutated acute myeloid leukemia—a Filo study. Blood. 2023;142(Supplement 1):421.CrossRef Orvain C, Bertoli S, Peterlin P, et al. Molecular relapse after first-line intensive therapy in patients with core-binding factor and NPM1—mutated acute myeloid leukemia—a Filo study. Blood. 2023;142(Supplement 1):421.CrossRef
17.
Zurück zum Zitat Guolo F, Fianchi L, Martelli MP, et al. Optimal duration of CPX-351 treatment and best timing for consolidation with Allogeneic stem cell transplantation: evidence from a large real-world Italian study. Blood. 2023;142(Supplement 1):731.CrossRef Guolo F, Fianchi L, Martelli MP, et al. Optimal duration of CPX-351 treatment and best timing for consolidation with Allogeneic stem cell transplantation: evidence from a large real-world Italian study. Blood. 2023;142(Supplement 1):731.CrossRef
18.
Zurück zum Zitat Mims A, Xie Z, Vasconcelos A, et al. Intensive chemotherapy (IC) followed by oral Azacitidine (AZA) maintenance versus venetoclax (VEN) plus AZA for patients (pts) with acute myeloid leukemia (AML): retrospective analysis of an electronic medical record (EMR) database in the United States. Blood. 2023;142(Supplement 1):550.CrossRef Mims A, Xie Z, Vasconcelos A, et al. Intensive chemotherapy (IC) followed by oral Azacitidine (AZA) maintenance versus venetoclax (VEN) plus AZA for patients (pts) with acute myeloid leukemia (AML): retrospective analysis of an electronic medical record (EMR) database in the United States. Blood. 2023;142(Supplement 1):550.CrossRef
19.
Zurück zum Zitat Smith BD, Lachowiez CA, Ambinder AJ, et al. A comparison of acute myeloid leukemia (AML) regimens: hypomethylating agents combined with Ivosidenib or Venetoclax in newly diagnosed patients with IDH1 mutations: a real-world evidence study. Blood. 2023;142(Supplement 1):971.CrossRef Smith BD, Lachowiez CA, Ambinder AJ, et al. A comparison of acute myeloid leukemia (AML) regimens: hypomethylating agents combined with Ivosidenib or Venetoclax in newly diagnosed patients with IDH1 mutations: a real-world evidence study. Blood. 2023;142(Supplement 1):971.CrossRef
21.
Zurück zum Zitat Garcia JS, Platzbecker U, Odenike O, et al. Efficacy and safety of Venetoclax in combination with Azacitidine for the treatment of patients with treatment-naive, higher-risk myelodysplastic syndromes. Blood. 2023;142(Supplement 1):319.CrossRef Garcia JS, Platzbecker U, Odenike O, et al. Efficacy and safety of Venetoclax in combination with Azacitidine for the treatment of patients with treatment-naive, higher-risk myelodysplastic syndromes. Blood. 2023;142(Supplement 1):319.CrossRef
22.
Zurück zum Zitat Tentori CA, Gregorio C, Robin M, et al. Clinical and genomic-based decision support system to define the optimal timing of Allogeneic hematopoietic stem cell transplantation in patients with Myelodysplastic syndromes (MDS). Blood. 2023;142(Supplement 1):197.CrossRef Tentori CA, Gregorio C, Robin M, et al. Clinical and genomic-based decision support system to define the optimal timing of Allogeneic hematopoietic stem cell transplantation in patients with Myelodysplastic syndromes (MDS). Blood. 2023;142(Supplement 1):197.CrossRef
23.
Zurück zum Zitat Mittelman M, Henry DH, Glaspy J, et al. Efficacy and safety of Roxadustat for treatment of anemia in patients with lower-risk Myelodysplastic syndrome (LR-MDS) with low red blood cell (RBC) transfusion burden: results of phase III Matterhorn study. Blood. 2023;142(Supplement 1):195.CrossRef Mittelman M, Henry DH, Glaspy J, et al. Efficacy and safety of Roxadustat for treatment of anemia in patients with lower-risk Myelodysplastic syndrome (LR-MDS) with low red blood cell (RBC) transfusion burden: results of phase III Matterhorn study. Blood. 2023;142(Supplement 1):195.CrossRef
24.
Zurück zum Zitat Komrokji RS, Santini V, Fenaux P, et al. Efficacy of Imetelstat in Achieving Red Blood Cell Transfusion Independence (RBC-TI) across Different Risk Subgroups in Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis-Stimulating Agents (ESAs) in IMerge Phase 3 Study. Blood. 2023;142(Supplement 1):194.CrossRef Komrokji RS, Santini V, Fenaux P, et al. Efficacy of Imetelstat in Achieving Red Blood Cell Transfusion Independence (RBC-TI) across Different Risk Subgroups in Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis-Stimulating Agents (ESAs) in IMerge Phase 3 Study. Blood. 2023;142(Supplement 1):194.CrossRef
25.
Zurück zum Zitat Garcia-Manero G, Platzbecker U, Santini V, et al. Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent (ESA)-naive patients (pts) with transfusion-dependent (TD) lower-risk Myelodysplastic syndromes (LR-MDS): full analysis of the COMMANDS trial. Blood. 2023;142(Supplement 1):193.CrossRef Garcia-Manero G, Platzbecker U, Santini V, et al. Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent (ESA)-naive patients (pts) with transfusion-dependent (TD) lower-risk Myelodysplastic syndromes (LR-MDS): full analysis of the COMMANDS trial. Blood. 2023;142(Supplement 1):193.CrossRef
Metadaten
Titel
Trends in MDS and AML from ASH 2023
verfasst von
Prof. Dr. Michael Pfeilstöcker
Publikationsdatum
18.07.2024
Verlag
Springer Vienna
Erschienen in
memo - Magazine of European Medical Oncology / Ausgabe 3/2024
Print ISSN: 1865-5041
Elektronische ISSN: 1865-5076
DOI
https://doi.org/10.1007/s12254-024-00984-8