Novel Therapies in Myelofibrosis: Beyond JAK Inhibitors
AbstractPurpose of ReviewTo discuss the current treatment paradigm, review novel targets, and summarize completed and ongoing clinical trials that may lead to a paradigm shifts in the management of myelofibrosis (MF).Recent FindingsIn addition to the recent approval and ongoing late-stage development of multiple novel JAK inhibitors, recent clinical studies demonstrate therapeutic potential of targeting multiple alternate proteins and pathways including BET, MDM2, telomerase, BCL2, LSD1, PI3K, SMAC, and PTX2 in patients with MF.SummaryMF is a myeloproliferative neoplasm characterized by clonal proliferation of myeloid cell...
Source: Current Hematologic Malignancy Reports - October 1, 2022 Category: Hematology Source Type: research

Management of Chronic Myeloid Leukemia in Children and Young Adults
AbstractPurpose of ReviewDue to lack of pediatric-specific data, the management of chronic myeloid leukemia (CML) in pediatric, adolescents, and young adults is guided by adult CML evidence-based recommendations. Pediatric CML presents differently than adult CML and is often a more aggressive disease with different biological and host factors, yet there is sparse literature on how to address those differences.Recent FindingsOver the past two decades, tyrosine kinase inhibitors (TKIs) have changed the way CML is treated. There are currently three FDA-approved TKIs (imatinib, dasatinib, and nilotinib) for pediatric patients....
Source: Current Hematologic Malignancy Reports - October 1, 2022 Category: Hematology Source Type: research

Advances in Risk Stratification and Treatment of Polycythemia Vera and Essential Thrombocythemia
AbstractPurpose of ReviewEstimating and modifying thrombotic risk is currently the mainstay of care for patients with polycythemia vera (PV) and essential thrombocythemia (ET). In recent years, however, increased attention has shifted towards quality of life and disease modification. In this review, we discuss recent advances in risk stratification, present updated results for ruxolitinib and interferon randomized clinical trials, discuss new approaches in antiplatelet and anticoagulant treatment, and summarize early phase trials of novel agents and emerging therapeutic concepts for the treatment of PV and ET.Recent Findin...
Source: Current Hematologic Malignancy Reports - October 1, 2022 Category: Hematology Source Type: research

Towards a Personalized Definition of Prognosis in Philadelphia-Negative Myeloproliferative Neoplasms
AbstractPurpose of ReviewPhiladelphia-negative myeloproliferative neoplasms (MPNs) include polycythemia vera (PV), essential thrombocythemia (ET), prefibrotic (pre-), and overt-primary myelofibrosis (primary MF, PMF). PV and ET could evolve into secondary MF (SMF), whose early diagnosis relies on monitoring signs of possible progression. All MPNs have a risk of blast phase (BP), that is associated with a very dismal outcome. Overall survival (OS) is different among MPNs, and disease-specific prognostic scores should be applied for a correct clinical management. In this review, an overview of current prognostic scores in MP...
Source: Current Hematologic Malignancy Reports - October 1, 2022 Category: Hematology Source Type: research

Progress and Challenges in Survivorship After Acute Myeloid Leukemia in Adults
AbstractPurpose of ReviewAcute myeloid leukemia (AML) survivors face unique challenges affecting long-term outcomes and quality of life. There is scant literature on the long-term impact of AML treatment in physical and mental health, disease recurrence, and financial burden in survivors.Recent FindingsFatigue, mental health concerns, infections, sexual dysfunction, and increase cancer recurrence occur after AML treatment. Chronic graft-versus-host disease (GVHD) and infections are common concerns in AML after hematopoietic stem cell transplantation (HCT). Survivorship guidelines encompass symptoms and complications but fa...
Source: Current Hematologic Malignancy Reports - September 19, 2022 Category: Hematology Source Type: research

Pediatric Germline Predisposition to Myeloid Neoplasms
AbstractPurpose of ReviewAdvances in the understanding of germline predisposition to pediatric cancers, particularly myeloid neoplasms, have increased rapidly over the last 20  years. Here, we highlight the most up-to-date knowledge regarding known pathogenic germline variants that contribute to the development of myeloid neoplasms in children.Recent FindingsThis discussion enumerates the most notable myeloid neoplasm-causing germline mutations. These mutations may be organized based on their molecular underpinnings —transcriptional control, splicing and signal transduction control, and a group of heterogeneous bone mar...
Source: Current Hematologic Malignancy Reports - September 19, 2022 Category: Hematology Source Type: research

Progress and Challenges in Survivorship After Acute Myeloid Leukemia in Adults
AbstractPurpose of ReviewAcute myeloid leukemia (AML) survivors face unique challenges affecting long-term outcomes and quality of life. There is scant literature on the long-term impact of AML treatment in physical and mental health, disease recurrence, and financial burden in survivors.Recent FindingsFatigue, mental health concerns, infections, sexual dysfunction, and increase cancer recurrence occur after AML treatment. Chronic graft-versus-host disease (GVHD) and infections are common concerns in AML after hematopoietic stem cell transplantation (HCT). Survivorship guidelines encompass symptoms and complications but fa...
Source: Current Hematologic Malignancy Reports - September 19, 2022 Category: Hematology Source Type: research

Pediatric Germline Predisposition to Myeloid Neoplasms
AbstractPurpose of ReviewAdvances in the understanding of germline predisposition to pediatric cancers, particularly myeloid neoplasms, have increased rapidly over the last 20  years. Here, we highlight the most up-to-date knowledge regarding known pathogenic germline variants that contribute to the development of myeloid neoplasms in children.Recent FindingsThis discussion enumerates the most notable myeloid neoplasm-causing germline mutations. These mutations may be organized based on their molecular underpinnings —transcriptional control, splicing and signal transduction control, and a group of heterogeneous bone mar...
Source: Current Hematologic Malignancy Reports - September 19, 2022 Category: Hematology Source Type: research

Towards a Personalized Definition of Prognosis in Philadelphia-Negative Myeloproliferative Neoplasms
AbstractPurpose of ReviewPhiladelphia-negative myeloproliferative neoplasms (MPNs) include polycythemia vera (PV), essential thrombocythemia (ET), prefibrotic (pre-), and overt-primary myelofibrosis (primary MF, PMF). PV and ET could evolve into secondary MF (SMF), whose early diagnosis relies on monitoring signs of possible progression. All MPNs have a risk of blast phase (BP), that is associated with a very dismal outcome. Overall survival (OS) is different among MPNs, and disease-specific prognostic scores should be applied for a correct clinical management. In this review, an overview of current prognostic scores in MP...
Source: Current Hematologic Malignancy Reports - September 1, 2022 Category: Hematology Source Type: research

Value in Myeloma Care: Myth or Reality
AbstractPurpose of ReviewDespite tremendous advances in multiple myeloma (MM) care, the disease maintains considerable morbidity and requires long-term treatment associated with significant financial toxicity to patients and high costs to society. In this review, we explore why — despite treatment advances — value in MM treatment is largely a myth, then explain some ways the myth might become a reality.Recent FindingsWe discuss how value-based care in MM should include patient-centered outcomes such as financial toxicity and quality of life, which are heavily impacted by cost of drugs and the indefinite duration of the...
Source: Current Hematologic Malignancy Reports - August 30, 2022 Category: Hematology Source Type: research

Bispecific Antibodies for the Treatment of Multiple Myeloma
AbstractPurpose of ReviewAdvances in multiple myeloma therapies have greatly improved outcomes for patients living with the disease, although to date there is yet to be a cure. Cellular and immunotherapies, approved or in development, offer the promise of significantly advancing toward that possibility. The aim of this review is to provide a synopsis and commentary on the current and future states of bispecific agents aimed at harnessing the antineoplastic potential of T-cells in treating and eradicating myeloma.Recent FindingsNumerous bispecific agents are in clinical development with some on the precipice of regulatory a...
Source: Current Hematologic Malignancy Reports - August 27, 2022 Category: Hematology Source Type: research

Role of Germline Predisposition to Therapy-Related Myeloid Neoplasms
AbstractPurpose of ReviewTherapy-related myeloid neoplasms (t-MNs) are aggressive leukemias that develop following exposure to DNA-damaging agents. A subset of patients developing t-MN may have an inherited susceptibility to develop myeloid neoplasia. Herein, we review studies reporting t-MN and their association with a germline or inherited predisposition.Recent FindingsEmerging evidence suggests that development of t-MN is the result of complex interactions including generation of somatic variants in hematopoietic stem cells and/or clonal selection pressure exerted by the DNA-damaging agents, and immune evasion on top of...
Source: Current Hematologic Malignancy Reports - August 20, 2022 Category: Hematology Source Type: research