☆ 4.4 Review

Proposals for Clinical Trials in Chronic Myelomonocytic Leukemia

CURRENT TREATMENT OPTIONS IN ONCOLOGY (2023)

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Article Hematology

The mutational landscape in chronic myelomonocytic leukemia and its impact on allogeneic hematopoietic cell transplantation outcomes: a Center for Blood and Marrow Transplantation Research (CIBMTR) analysis

Matthew Mei et al.

Summary: Somatic mutations are recognized as an important prognostic factor in chronic myelomonocytic leukemia (CMML). However, limited data are available regarding their impact on outcomes after allogeneic hematopoietic cell transplantation (HCT).

HAEMATOLOGICA (2023)

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Letter Hematology

Hypomethylating agent and venetoclax in patients with chronic myelomonocytic leukemia: Is the combination indeed better?

Somedeb Ball et al.

AMERICAN JOURNAL OF HEMATOLOGY (2022)

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Article Hematology

Chronic myelomonocytic leukemia: 2022 update on diagnosis, risk stratification, and management

Mrinal M. Patnaik et al.

Summary: Chronic myelomonocytic leukemia (CMML) is a clonal hematopoietic stem cell disorder with overlapping features of myelodysplastic syndromes and myeloproliferative neoplasms. Diagnosis is based on the presence of peripheral blood monocytosis and bone marrow dysplasia. Mutations and risk factors affect prognosis, and hypomethylating agents are commonly used for treatment. Allogeneic stem cell transplant is a potential curative option.

AMERICAN JOURNAL OF HEMATOLOGY (2022)

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Article Hematology

A randomised phase II study of azacitidine (AZA) alone or with Lenalidomide (LEN), Valproic acid (VPA) or Idarubicin (IDA) in higher-Risk MDS or low blast AML: GFM's pick a winner trial, with the impact of somatic mutations

Lionel Ades et al.

Summary: The combination of AZA with other drugs did not improve the outcome observed in higher-risk MDS patients. There was no significant difference in survival and progression-free survival among the different treatment arms. Some combinations led to increased infection rates and hospitalization due to myelosuppression. Factors associated with better treatment response and survival included certain clinical indicators and gene mutations.

BRITISH JOURNAL OF HAEMATOLOGY (2022)

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Review Hematology

How I diagnose and treat chronic myelomonocytic leukemia

Mrinal M. Patnaik

Summary: CMML is a myelodysplastic syndrome/myeloproliferative overlap neoplasm with potential risk of transformation to acute myeloid leukemia. While allogeneic stem cell transplant is the potential cure, it is limited by age and comorbidities of affected patients. DNA methyltransferase inhibitors are approved for management, but their efficacy is limited and personalized/targeted therapies are needed.

HAEMATOLOGICA (2022)

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Article Cardiac & Cardiovascular Systems

TET2-Driven Clonal Hematopoiesis and Response to Canakinumab An Exploratory Analysis of the CANTOS Randomized Clinical Trial

Eric C. Svensson et al.

Summary: This study investigated whether individuals with CHIP have a greater reduction in cardiovascular events in response to IL-1 beta neutralization therapy.

JAMA CARDIOLOGY (2022)

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Article Hematology

International Consensus Classification of Myeloid Neoplasms and Acute Leukemias: integrating morphologic, clinical, and genomic data

Daniel A. Arber et al.

Summary: In 2016, the WHO, Society for Hematopathology, and European Association for Haematopathology collaborated to update the classification of myeloid neoplasms and acute leukemias, advancing the field of myeloid neoplasms and acute leukemias.

BLOOD (2022)

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Review Oncology

The 5th edition of the World Health Organization Classification of Haematolymphoid Tumours: Myeloid and Histiocytic/Dendritic Neoplasms

Joseph D. Khoury et al.

Summary: This paper summarizes the new WHO classification scheme for myeloid and histiocytic/dendritic neoplasms, emphasizing the refinement of diagnostic criteria and the importance of actionable biomarkers, while maintaining global applicability.

LEUKEMIA (2022)

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Article Oncology

The ABNL-MARRO 001 study: a phase 1-2 study of randomly allocated active myeloid target compound combinations in MDS/MPN overlap syndromes

Tamara K. Moyo et al.

Summary: Myelodysplastic/myeloproliferative neoplasms (MDS/MPN) are rare hematologic malignancies that have a significant impact on patient quality of life. Current treatment options have poor outcomes, making it a critical area of unmet clinical need. The ABNL-MARRO project aims to advance the treatment of MDS/MPN and explore clinical and pathologic markers of disease severity, prognosis, and treatment response.

BMC CANCER (2022)

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Review Oncology

ASXL1/2 mutations and myeloid malignancies

Edward A. Medina et al.

Summary: Myeloid malignancies are caused by the accumulation of genetic and epigenetic alterations that interfere with hematopoietic stem cell (HSC) self-renewal, increase HSC proliferation, and cause differentiation defects. The polycomb group (PcG) and trithorax group (TrxG) of epigenetic regulators play opposing roles in regulating the expression of genes essential for stem cell functions. The genes encoding these proteins and the proteins that interact with them or affect their presence on chromatin are frequently mutated in myeloid malignancies. Enhancers of Trithorax and Polycomb (ETP) proteins regulate PcG and TrxG proteins. ASXL1 and ASXL2, which are ETP proteins, form chromatin modification complexes and transcription factors. ASXL1 mutations are often found in myeloid malignancies and are associated with a poor prognosis, while ASXL2 mutations are more common in AML with t(8;21)/RUNX1-RUNX1T1 and less frequent in other subtypes of myeloid malignancies.

JOURNAL OF HEMATOLOGY & ONCOLOGY (2022)

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Article Oncology

Real-World Data on Chronic Myelomonocytic Leukemia: Clinical and Molecular Characteristics, Treatment, Emerging Drugs, and Patient Outcomes

Sandra Castano-Diez et al.

Summary: Despite emerging molecular information, the outcome of patients with chronic myelomonocytic leukemia (CMML) remains unsatisfactory. This study aimed to explore the potential correlation between clinical features, gene mutations, and treatment regimens with overall survival (OS) and transformation into acute myeloid leukemia (AML). The most commonly detected mutations were TET2, SRSF2, ASXL1, and RUNX1. Hypomethylating agents and allogeneic stem cell transplantation (alloSCT) were administered to patients, with varying response rates. Targeted therapies with IDH or FLT3 inhibitors showed promise in specific gene mutation cases. AML transformation occurred in a significant proportion of patients, with changes in gene mutation status observed. Complete molecular characterization can help improve risk stratification and personalize therapy.

CANCERS (2022)

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Meeting Abstract Hematology

Response Rates to Venetoclax Based Therapies in Chronic Phase and Blast Transformed Chronic Myelomonocytic Leukemia

Clifford M. Csizmar et al.

BLOOD (2022)

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Meeting Abstract Hematology

Therapeutic Targeting of EP300/CBP By Bromodomain Inhibition in Acute Myeloid Leukemia

Luciano Nicosia et al.

BLOOD (2022)

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Meeting Abstract Hematology

Phase 1b Trial of Anti-Immunoglobulin-like Transcript 3 Monoclonal Antibody MK-0482 Monotherapy in Relapsed/ Refractory Acute Myeloid Leukemia and Chronic Myelomonocytic Leukemia

Ghayas C. Issa et al.

BLOOD (2022)

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Meeting Abstract Hematology

Clinical and Biological Effects of Canakinumab in Lower-Risk Myelodysplastic Syndromes (MDS): Results from a Phase 2 Clinical Trial

Guillermo Garcia-Manero et al.

BLOOD (2022)

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Meeting Abstract Hematology

Pelabresib (CPI-0610) As Add-on to Ruxolitinib in Myelofibrosis: Durability of Response and Safety Beyond Week 24 in the Phase 2 MANIFEST Study

Claire Harrison et al.

BLOOD (2022)

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Meeting Abstract Hematology

Efficacy and Safety of Ruxolitinib for Treatment of Symptomatic Chronic Myelomonocytic Leukemia (CMML): Results of a Multicenter Phase II Clinical Trial

Eric Padron et al.

BLOOD (2022)

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Meeting Abstract Hematology

Concerto (NCT04409639): A Phase 2 Trial of Cobimetinib in Newly Diagnosed and HMA-Treated CMML Patients with RAS Pathway Mutations

Ami B. Patel et al.

BLOOD (2022)

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Meeting Abstract Hematology

INCB057643 Monotherapy in Patients with Relapsed or Refractory Myelofibrosis: A Phase 1 Study

Justin Watts et al.

BLOOD (2022)

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Meeting Abstract Hematology

ACUTE MYELOID LEUKEMIAS: INVESTIGATIONAL THERAPIES, EXCLUDING TRANSPLANTATION AND CELLULAR IMMUNOTHERAPIES

Richard F. Little et al.

BLOOD (2022)

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Meeting Abstract Hematology

BMS-986158, a Potent BET Inhibitor, As Monotherapy and in Combination with Ruxolitinib or Fedratinib in Intermediate- or High-Risk Myelofibrosis: First Results from a Phase 1/2 Study

Rosa Ayala et al.

BLOOD (2022)

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Meeting Abstract Hematology

Enasidenib (ENA) Is Effective in Patients with IDH2 Mutated Myelodysplastic Syndrome (MDS) : The Ideal Phase 2 Study By the GFM Group

Lionel Ades et al.

BLOOD (2021)

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Meeting Abstract Hematology

Ivosidenib Monotherapy Is Effective in Patients with IDH1 Mutated Myelodysplastic Syndrome (MDS): The Idiome Phase 2 Study By the GFM Group

Marie Sebert et al.

BLOOD (2021)

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Article Hematology

Outcomes of patients with chronic myelomonocytic leukaemia treated with non-curative therapies: a retrospective cohort study

Lisa Pleyer et al.

Summary: The study suggests that hypomethylating agents are preferred therapy for patients with higher-risk chronic myelomonocytic leukemia and those with myeloproliferative CMML. CPSS is a valuable tool to identify patients most likely to benefit from hypomethylating agents. Further evidence from prospective cohorts is needed to confirm these findings.

LANCET HAEMATOLOGY (2021)

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Article Multidisciplinary Sciences

RAS mutations drive proliferative chronic myelomonocytic leukemia via a KMT2A-PLK1 axis

Ryan M. Carr et al.

Summary: The research focuses on the relationship between proliferative chronic myelomonocytic leukemia (pCMML) and RAS pathway mutations, revealing that RAS mutations lead to a unique gene expression profile enriched in mitotic kinases such as PLK1. The study suggests that inhibiting RAS-driven PLK1 expression could be a viable therapeutic target for pCMML.

NATURE COMMUNICATIONS (2021)

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Article Multidisciplinary Sciences

Mutant ASXL1 induces age-related expansion of phenotypic hematopoietic stem cells through activation of Akt/mTOR pathway

Takeshi Fujino et al.

Summary: ASXL1 mutations are frequently detected in age-related clonal hematopoiesis (CH), but their mechanism in driving CH remains unclear. The study demonstrates that expression of C-terminal truncated ASXL1 in hematopoietic stem cells leads to Akt de-ubiquitination, activated Akt/mTOR signaling, and aberrant HSC proliferation.

NATURE COMMUNICATIONS (2021)

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Review Oncology

Risk-Adapted, Individualized Treatment Strategies of Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML)

Jan Philipp Bewersdorf et al.

Summary: MDS and CMML are two distinct blood cancers with variable symptoms and risks. Management decisions should be individualized and based on validated risk stratification tools. Supportive care is the mainstay for lower-risk patients, while bone marrow transplant is the only curative option for higher-risk patients.

CANCERS (2021)

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Meeting Abstract Oncology

Phase 3 VERONA study of venetoclax with azacitidine to assess change in complete remission and overall survival in treatment-naive higher-risk myelodysplastic syndromes.

Amer Methqal Zeidan et al.

JOURNAL OF CLINICAL ONCOLOGY (2021)

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Article Hematology

Adjunctive Volasertib in Patients With Acute Myeloid Leukemia not Eligible for Standard Induction Therapy: A Randomized, Phase 3 Trial

Hartmut Dohner et al.

Summary: This phase 3 trial did not demonstrate a survival benefit with the polo-like kinase inhibitor volasertib combined with low-dose cytarabine in older patients with acute myeloid leukemia who were ineligible for intensive chemotherapy, potentially due to increased early mortality in the volasertib group from myelosuppression and infections.

HEMASPHERE (2021)

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Article Oncology

Epigenetic targeted therapy of stabilized BAP1 in ASXL1 gain-of-function mutated leukemia

Lu Wang et al.

Summary: The study uncovered gain-of-function mutations in ASXL1 that stabilize BAP1 protein and induce widespread epigenetic changes in myeloid neoplasms. By developing a chemical inhibitor targeting ASXL1-altered leukemia, the research provides a potential therapeutic strategy for this type of cancer. This breakthrough sheds light on the molecular mechanisms of ASXL1 mutations in leukemia pathogenesis and highlights small-molecular catalytic inhibitors of BAP1 as a promising targeted therapy.

NATURE CANCER (2021)

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Article Hematology

Impact of clinical, cytogenetic, and molecular profiles on long-term survival after transplantation in patients with chronic myelomonocytic leukemia

Janghee Woo et al.

HAEMATOLOGICA (2020)

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Article Oncology

LILRB4 expression in chronic myelomonocytic leukemia and myelodysplastic syndrome based on response to hypomethylating agents

Kelly S. Chien et al.

LEUKEMIA & LYMPHOMA (2020)

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Article Hematology

Phase 1 study of lenzilumab, a recombinant anti-human GM-CSF antibody, for chronic myelomonocytic leukemia

Mrinal M. Patnaik et al.

BLOOD (2020)

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Article Medicine, General & Internal

Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia

C. D. DiNardo et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

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Meeting Abstract Hematology

Activity of Venetoclax-Based Therapy in CMML and CMML with Blast Transformation

Danielle Hammond et al.

BLOOD (2020)

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Article Medicine, General & Internal

Lysozyme nephropathy in chronic myelomonocytic leukemia

Ami B. Patel et al.

CLINICAL CASE REPORTS (2019)

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Review Biochemistry & Molecular Biology

The role of ASXL1 in hematopoiesis and myeloid malignancies

Shuhei Asada et al.

CELLULAR AND MOLECULAR LIFE SCIENCES (2019)

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Article Multidisciplinary Sciences

ASXL1 and SETBP1 mutations promote leukaemogenesis by repressing TGFβ pathway genes through histone deacetylation

Makoto Saika et al.

SCIENTIFIC REPORTS (2018)

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Article Hematology

Diagnosis and Treatment of Chronic Myelomonocytic Leukemias in Adults Recommendations From the European Hematology Association and the European LeukemiaNet

Raphael Itzykson et al.

HEMASPHERE (2018)

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Article Hematology

Gain of function of ASXL1 truncating protein in the pathogenesis of myeloid malignancies

Hui Yang et al.

BLOOD (2018)

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Article Hematology

A phase II trial of ruxolitinib in combination with azacytidine in myelodysplastic syndrome/myeloproliferative neoplasms

Rita Assi et al.

AMERICAN JOURNAL OF HEMATOLOGY (2018)

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Article Oncology

Better transplant outcome with pre-transplant marrow response after hypomethylating treatment in higher-risk MDS with excess blasts

Seung-Ah Yahng et al.

ONCOTARGET (2017)

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Article Hematology

CMML: Clinical and molecular aspects

Raphael Itzykson et al.

INTERNATIONAL JOURNAL OF HEMATOLOGY (2017)

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Article Hematology

How I treat chronic myelomonocytic leukemia

Eric Solary et al.

BLOOD (2017)

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Review Oncology

Turning the tide in myelodysplastic/myeloproliferative neoplasms

Michael W. N. Deininger et al.

NATURE REVIEWS CANCER (2017)

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Review Hematology

Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel

Theo de Witte et al.

BLOOD (2017)

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Article Hematology

Response to erythropoietic-stimulating agents in patients with chronic myelomonocytic leukemia

Blanca Xicoy et al.

EUROPEAN JOURNAL OF HAEMATOLOGY (2016)

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Article Oncology

Age-related mutations and chronic myelomonocytic leukemia

C. C. Mason et al.

LEUKEMIA (2016)

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Article Hematology

Integrating clinical features and genetic lesions in the risk assessment of patients with chronic myelomonocytic leukemia

Chiara Elena et al.

BLOOD (2016)

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Editorial Material Oncology

Unplugging JAK/STAT in Chronic Myelomonocytic Leukemia

Eric Solary

CLINICAL CANCER RESEARCH (2016)

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Article Oncology

A Multi-Institution Phase I Trial of Ruxolitinib in Patients with Chronic Myelomonocytic Leukemia (CMML)

Eric Padron et al.

CLINICAL CANCER RESEARCH (2016)

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Article Oncology

Eltrombopag Use in Patients With Chronic Myelomonocytic Leukemia (CMML): A Cautionary Tale

Hanadi Ramadan et al.

CLINICAL LYMPHOMA MYELOMA & LEUKEMIA (2016)

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Letter Oncology

A phase II study of the efficacy, safety, and determinants of response to 5-azacitidine (Vidaza®) in patients with chronic myelomonocytic leukemia

Srinivas K. Tantravahi et al.

LEUKEMIA & LYMPHOMA (2016)

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Article Hematology

Achievement of complete remission predicts outcome of allogeneic haematopoietic stem cell transplantation in patients with chronic myelomonocytic leukaemia. A study of the Chronic Malignancies Working Party of the European Group for Blood and Marrow Transplantation

Argiris Symeonidis et al.

BRITISH JOURNAL OF HAEMATOLOGY (2015)

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Letter Oncology

Ex vivo activity of BCL-2 family inhibitors ABT-199 and ABT-737 combined with 5-azacytidine in myeloid malignancies

James M. Bogenberger et al.

LEUKEMIA & LYMPHOMA (2015)

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Article Genetics & Heredity

Juvenile myelomonocytic leukaemia and Noonan syndrome

Marion Strullu et al.

JOURNAL OF MEDICAL GENETICS (2014)

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Article Hematology

FLT3 mutations in myelodysplastic syndrome and chronic myelomonocytic leukemia

Naval Daver et al.

AMERICAN JOURNAL OF HEMATOLOGY (2013)

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Article Hematology

GM-CSF-dependent pSTAT5 sensitivity is a feature with therapeutic potential in chronic myelomonocytic leukemia

Eric Padron et al.

BLOOD (2013)

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Article Hematology

Development and validation of a prognostic scoring system for patients with chronic myelomonocytic leukemia

Esperanza Such et al.

BLOOD (2013)

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Article Oncology

Mayo prognostic model for WHO-defined chronic myelomonocytic leukemia: ASXL1 and spliceosome component mutations and outcomes

M. M. Patnaik et al.

LEUKEMIA (2013)

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Article Oncology

Predictive factors of response and survival among chronic myelomonocytic leukemia patients treated with azacitidine

Lionel Ades et al.

LEUKEMIA RESEARCH (2013)

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Article Hematology

Applicability of a Pick a Winner trial design to acute myeloid leukemia

Robert K. Hills et al.

BLOOD (2011)

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Article Hematology

ASXL1 mutation is associated with poor prognosis and acute transformation in chronic myelomonocytic leukaemia

Veronique Gelsi-Boyer et al.

BRITISH JOURNAL OF HAEMATOLOGY (2010)

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Review Biochemistry & Molecular Biology

The Inflammasomes

Kate Schroder et al.

CELL (2010)

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Article Oncology

RAS Mutations Contribute to Evolution of Chronic Myelomonocytic Leukemia to the Proliferative Variant

Clara Ricci et al.

CLINICAL CANCER RESEARCH (2010)

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Article Oncology

Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study

Pierre Fenaux et al.

LANCET ONCOLOGY (2009)

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Article Oncology

Single-cell profiling identifies aberrant STAT5 activation in myeloid malignancies with specific clinical and biologic correlates

Nikesh Kotecha et al.

CANCER CELL (2008)

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Article Oncology

Prognostic factors and risk assessment in chronic myelomonocytic leukemia: Validation study of the M. D. Anderson prognostic scoring system

Miloslav Beran et al.

LEUKEMIA & LYMPHOMA (2007)

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Article Oncology

Decitabine improves patient outcomes in myelodysplastic syndromes - Resuits of a Phase III randomized study

H Kantarjian et al.

CANCER (2006)

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Article Oncology

Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: A study of the cancer and leukemia group B

LR Silverman et al.

JOURNAL OF CLINICAL ONCOLOGY (2002)

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