Current Hematologic Malignancy Reports最新文献

{"title":"Highlights from MPN Asia 2025: Advances in Molecular Pathogenesis and Therapeutic Strategies in Myeloproliferative Neoplasms.","authors":"Prithviraj Bose, Zhijian Xiao, Hans C Hasselbalch, Josef T Prchal, Minghui Duan, Abdulraheem Yacoub, Raajit Rampal, Jean-Jacques Kiladjian, Gabriela S Hobbs, Tsewang Tashi, Kazuya Shimoda, Keita Kirito, Harinder Gill, Hsin-An Hou, Sung-Eun Lee, Jian Huang, Bing Li, Albert Qin, Lennex Hsueh-Lin Yu, John O Mascarenhas, Ruben A Mesa","doi":"10.1007/s11899-025-00752-3","DOIUrl":"10.1007/s11899-025-00752-3","url":null,"abstract":"<p><strong>Purpose of review: </strong>This report summarizes key insights from the 8th Annual International Symposium on Myeloproliferative Neoplasms (MPN Asia 2025). The symposium brought together global experts to discuss advancements in MPN biology, diagnostics, and therapeutics, with a focus on emerging molecular understanding, novel treatment strategies and real-world data.</p><p><strong>Recent findings: </strong>Molecular profiling has become essential in MPN risk stratification and therapeutic decision-making. High-risk mutations (e.g., ASXL1, TP53) and inflammatory pathways (e.g., IL-17, NF-κB) were shown to correlate with disease progression and transformation. Interferon-based therapy is increasingly used in younger, low-risk, or treatment-naïve patients, and is also being investigated in myelofibrosis and essential thrombocythemia. Ropeginterferon alfa-2b, a novel interferon-based therapy, demonstrated durable clinical efficacy in polycythemia vera. Its high initial-dose and accelerated titration (HIDAT) regimen led to fast achievement of complete hematologic response, rapid reductions in JAK2V617F allele burden, and high complete molecular response rate. Combination regimens involving ruxolitinib and agents such as pelabresib, selinexor, and interferon showed potential for enhanced efficacy. Population-based studies from Asia contributed regional epidemiological and treatment data, reinforcing the role of real-world evidence. Modern prognostic models such as MIPSS70+ v2.0 and GIPSS were discussed for more precise risk prediction. Preliminary findings also suggest ropeginterferon alfa-2b may be a safe option during pregnancy. MPN Asia 2025 highlighted the growing role of molecular diagnostics and targeted therapeutics in the management of MPNs. Ropeginterferon alfa-2b has emerged as a therapeutic potential across the MPN spectrum. Its early use and personalized strategies are increasingly recognized. Real-world data and regional insights are shaping a more nuanced, globally informed approach to MPN care.</p>","PeriodicalId":10852,"journal":{"name":"Current Hematologic Malignancy Reports","volume":"20 1","pages":"9"},"PeriodicalIF":2.7,"publicationDate":"2025-07-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12254180/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144616612","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Contemporary CMML Risk Stratification and Management.","authors":"Dahniel Sastow, Arjun Syal, Douglas Tremblay","doi":"10.1007/s11899-025-00753-2","DOIUrl":"https://doi.org/10.1007/s11899-025-00753-2","url":null,"abstract":"<p><strong>Purpose of review: </strong>Chronic myelomonocytic leukemia (CMML), a rare myelodysplastic/myeloproliferative neoplasm (MDS/MPN) overlap syndrome, poses diagnostic and therapeutic challenges due to its heterogeneity and rarity. This review highlights updates to CMML diagnostic criteria, examines risk stratification models and their clinical implications, and outlines both established and emerging therapies for this rare and likely underrecognized hematologic malignancy.</p><p><strong>Recent findings: </strong>Updated diagnostic criteria from 2022 reduce the monocyte threshold to 0.5 × 10⁹/L for CMML diagnosis, which will reclassify many cases previously diagnosed as MDS. Risk stratification models continue to be refined allowing for improved prediction and may help select appropriate patients for allogeneic stem cell transplantation, the only curative therapeutic modality in CMML. Although trials in CMML were long relegated to subpopulation of MDS studies, there has recently been a flourishing of novel therapies being tested specifically in CMML. These include lenzilumab (anti-GM-CSF) and IO-202 (anti-LILRB4), which have demonstrated promising early efficacy signals but require further study. Established treatments, which include hypomethylating agents and hydroxyurea as well as the JAK1/2 inhibitor ruxolitinib, provide limited survival benefits in CMML, underscoring the urgent need for novel therapeutic development. Coordinated dedicated research efforts have started to evaluate new agents in CMML. Along with further diagnostic and prognostic refinement, these advances are welcomed for this rare and heterogenous disease.</p>","PeriodicalId":10852,"journal":{"name":"Current Hematologic Malignancy Reports","volume":"20 1","pages":"8"},"PeriodicalIF":2.7,"publicationDate":"2025-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144559473","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Emerging Therapeutic Approaches for Anemia in Myelofibrosis.","authors":"Helen T Chifotides, Andrea Duminuco, Elena Torre, Calogero Vetro, Patrick Harrington, Giuseppe A Palumbo, Prithviraj Bose","doi":"10.1007/s11899-025-00751-4","DOIUrl":"https://doi.org/10.1007/s11899-025-00751-4","url":null,"abstract":"<p><strong>Purpose of review: </strong>In this review, we highlight conventional agents and novel emerging therapeutic strategies to treat anemia in MF.</p><p><strong>Recent findings: </strong>Anemia is a common and challenging feature of myelofibrosis (MF). The pathobiology of anemia is multifactorial, including progressive bone marrow fibrosis, decreased erythropoiesis due to high hepcidin levels leading to iron sequestration in the reticuloendothelial system, hypersplenism, erythropoiesis inhibition by myelosuppressive JAK inhibitors (ruxolitinib, fedratinib), and others. MF-associated anemia has a negative impact on survival. Conventional agents to manage anemia include erythropoiesis-stimulating agents, danazol, corticosteroids, and immunomodulatory agents, but responses are infrequent and lack durability. Notable advancements have emerged in developing novel treatments for anemia in MF, including the regulatory approval of momelotinib (ACVR1/JAK1/2 inhibitor) in 2023 and development of novel promising agents targeting hemojuvelin and activins. Momelotinib and pacritinib (ACVR1/JAK2 inhibitor) are the preferred JAK inhibitors for patients with cytopenias (anemia, thrombocytopenia). Luspatercept and elritercept are activin receptor ligand traps, promoting erythroid maturation and late-stage erythropoiesis. Currently, luspatercept is being evaluated in a phase 3 trial (INDEPENDENCE™) for anemia in MF patients who are on a JAK2 inhibitor and require transfusions, and in a phase 2 trial (ODYSSEY) in combination with momelotinib in MF patients who are transfusion dependent, whether or not on a JAK inhibitor. Interim results of the RESTORE trial demonstrated that elritercept significantly decreased transfusions in MF patients. DISC-0974 is a first-in-class anti-hemojuvelin (positive hepcidin regulator) monoclonal antibody that decreased hepcidin expression, increased serum iron, and enhanced erythropoiesis in anemic patients with MF in a phase 1b/2 study. Burgeoning studies of novel anemia-targeted agents and combinations are significantly improving the quality of life and outcomes of patients with MF. The recent approval of momelotinib to treat MF with anemia and the emerging novel anemia-directed strategies in early and advanced clinical development have ushered in a new era in the treatment of MF-related anemia.</p>","PeriodicalId":10852,"journal":{"name":"Current Hematologic Malignancy Reports","volume":"20 1","pages":"7"},"PeriodicalIF":2.7,"publicationDate":"2025-05-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143967688","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Role of Population Based Studies in Advancing our Knowledge of Myelodysplastic Syndromes.","authors":"Diego A Adrianzen-Herrera, Aneta Strumlowska","doi":"10.1007/s11899-025-00750-5","DOIUrl":"10.1007/s11899-025-00750-5","url":null,"abstract":"<p><strong>Purpose of the review: </strong>Myelodysplastic syndromes (MDS) are myeloid neoplasms characterized by high molecular and genomic heterogeneity. Accordingly, efforts in risk assessment and therapeutic intervention mostly target unique profiles that individualize specific MDS subtypes. In this review, we explored the contributions of population based studies accounting for MDS as a group.</p><p><strong>Recent findings: </strong>Large population based studies have been critical to define important details of our current knowledge of the disease. We summarized the most important population research contributions in MDS, focusing on its epidemiology, population risk factors, and relevant clinical associations. We discuss how these population data can provide vital insights to inform prevention measures, testing strategies, and treatment decisions. Population studies play an important role in guiding clinical and research efforts in MDS. Despite its complex molecular and genomic landscape, population data is integral to define the burden of disease, identify risk factors and clinical associations, and can help elucidate pathogenic mechanisms.</p>","PeriodicalId":10852,"journal":{"name":"Current Hematologic Malignancy Reports","volume":"20 1","pages":"6"},"PeriodicalIF":2.7,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143001567","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Advances in Novel Systemic Therapies for the Management of Cutaneous T Cell Lymphoma (CTCL).","authors":"Katherine B Case, Pamela B Allen","doi":"10.1007/s11899-024-00746-7","DOIUrl":"10.1007/s11899-024-00746-7","url":null,"abstract":"<p><strong>Purpose of review: </strong>Cutaneous T cell lymphomas (CTCLs) are comprised of a heterogenous group of non-Hodgkin lymphomas that can be difficult to treat and are often refractory to standard therapies. Mycosis fungoides (MF) and Sezary syndrome (SS) are the most common subtypes, accounting for the majority of CTCLs. There is no standard of care, and no treatments are curative. In this review, we summarize the promising, recently reported data describing novel systemic agents for the management of MF/SS.</p><p><strong>Recent findings: </strong>Clinical trials are currently exploring a number of agents, including novel chemotherapies, antibodies and antibody drug conjugates (ADCs), immunotherapy, and cellular therapies. These promising novel agents may expand the treatment landscape for MF/SS.</p>","PeriodicalId":10852,"journal":{"name":"Current Hematologic Malignancy Reports","volume":"20 1","pages":"5"},"PeriodicalIF":2.7,"publicationDate":"2025-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142969857","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mutant Calreticulin in MPN: Mechanistic Insights and Therapeutic Implications.","authors":"Mifra Faiz, Merle Riedemann, Jonas S Jutzi, Ann Mullally","doi":"10.1007/s11899-024-00749-4","DOIUrl":"10.1007/s11899-024-00749-4","url":null,"abstract":"<p><strong>Purpose of review: </strong>More than a decade following the discovery of Calreticulin (CALR) mutations as drivers of myeloproliferative neoplasms (MPN), advances in the understanding of CALR-mutant MPN continue to emerge. Here, we summarize recent advances in mehanistic understanding and in targeted therapies for CALR-mutant MPN.</p><p><strong>Recent findings: </strong>Structural insights revealed that the mutant CALR-MPL complex is a tetramer and the mutant CALR C-terminus is exposed on the cell surface. Targeting mutant CALR utilizing antibodies is the leading therapeutic approach, while mutant CALR-directed vaccines are also in early clinical trials. Additionally, chimeric antigen receptor (CAR) T-cells directed against mutant CALR are under evaluation in preclinical models. Approaches addressing the cellular effects of mutant CALR beyond MPL-JAK-STAT activation, such as targeting the unfolded protein response, proteasome, and N-glycosylation pathways, have been tested in preclinical models. In CALR-mutant MPN, the path from discovery to mechanistic understanding to direct therapeutic targeting has advanced rapidly. The longer-term goal remains clonally-selective therapies that modify the disease course in patients.</p>","PeriodicalId":10852,"journal":{"name":"Current Hematologic Malignancy Reports","volume":"20 1","pages":"4"},"PeriodicalIF":2.7,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12085081/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142946111","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Navigating the Economic Burden of Multiple Myeloma: Insights into Cost-effectiveness of CAR-T and Bispecific Antibody Therapies.","authors":"Praneeth Reddy Keesari, Diana Samuels, Charan Thej Reddy Vegivinti, Yashwitha Sai Pulakurthi, Revathi Kudithi, Meekoo Dhar, Murali Janakiram","doi":"10.1007/s11899-024-00748-5","DOIUrl":"10.1007/s11899-024-00748-5","url":null,"abstract":"<p><strong>Purpose of review: </strong>Multiple myeloma is a chronic malignancy and with evolving treatment options, understanding the economic burden and cost-effectiveness of therapies is crucial for clinicians and researchers.</p><p><strong>Recent findings: </strong>In this, we review the recent approval of Bispecific antibodies and CAR-T for myeloma and their cost implications, including direct and indirect costs. We compare this to current regimens and provide cost comparisons in this review. We conclude that the use of more effective therapies such as CAR-T in earlier lines of therapies may be more cost-effective depending on the country and model used. Further studies are essential to better understand the cost-effectiveness of bispecific antibodies including head-to-head comparisons to CAR-T therapy.</p>","PeriodicalId":10852,"journal":{"name":"Current Hematologic Malignancy Reports","volume":"20 1","pages":"3"},"PeriodicalIF":2.7,"publicationDate":"2025-01-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142926796","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Monoclonal Gammopathy-Associated Neuropathy.","authors":"Shayna Sarosiek, Christopher T Doughty, Jorge J Castillo","doi":"10.1007/s11899-024-00745-8","DOIUrl":"10.1007/s11899-024-00745-8","url":null,"abstract":"<p><strong>Purpose of review: </strong>Peripheral neuropathy (PN) is more commonly seen in individuals with monoclonal gammopathies, especially in patients with an IgM monoclonal gammopathy or Waldenström macroglobulinemia.</p><p><strong>Recent findings: </strong>There are multiple potential ways that the paraprotein may result in peripheral neuropathy. The diagnosis and management of monoclonal gammopathy-associated PN are challenging and necessitate a concerted effort between the hematologist/oncologist and the neurologist. This review describes the most common PN syndromes associated with monoclonal gammopathy, such as anti-myelin-associated glycoprotein neuropathy, light chain amyloidosis, cryoglobulinemia, POEMS, CANOMAD, and others. We also review the therapies used to treat these conditions.</p>","PeriodicalId":10852,"journal":{"name":"Current Hematologic Malignancy Reports","volume":"20 1","pages":"2"},"PeriodicalIF":2.7,"publicationDate":"2024-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142827443","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Disparities in Clinical Trial Enrollment- Focus on CAR-T and Bispecific Antibody Therapies.","authors":"Nadia Islam, Laura Budvytyte, Nandita Khera, Talal Hilal","doi":"10.1007/s11899-024-00747-6","DOIUrl":"10.1007/s11899-024-00747-6","url":null,"abstract":"<p><strong>Purpose of review: </strong>Recent studies show that unresolved disparities hinder enrollment to clinical trials, equitable distribution of treatments, and impact the generalizability of trials, compromising health outcomes across different populations. This review aims to examine the persistent disparities noted in clinical trial enrollment, with particular focus on lymphoid malignancies, CAR-T cell and bispecific antibody therapies.</p><p><strong>Recent findings: </strong>Targeted interventions can enhance recruitment of underrepresented groups in clinical trials and address the complex barriers hindering participation, which are essential for achieving healthcare access equity and treatment outcomes. Improvement must be multifaceted, addressing socioeconomic, geographic, and biologic factors contributing to underrepresentation. This includes more lenient eligibility criteria, improving outreach and education, as well as using technology to diversify trial participation.</p>","PeriodicalId":10852,"journal":{"name":"Current Hematologic Malignancy Reports","volume":"20 1","pages":"1"},"PeriodicalIF":2.7,"publicationDate":"2024-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11618314/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142767046","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pulmonary, Hepatic, and Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Telomere Biology Disorders.","authors":"Kelly M Pennington, Douglas Simonetto, Timucin Taner, Abhishek A Mangaonkar","doi":"10.1007/s11899-024-00724-z","DOIUrl":"10.1007/s11899-024-00724-z","url":null,"abstract":"<p><strong>Purpose of the review: </strong>This study aimed to summarize evidence and provide consensus-based guidelines for management of transplantation in patients with telomere biology disorders (TBD). Specifically, this review focuses on clinical management of lung, liver, and bone marrow transplantation in TBD patients.</p><p><strong>Recent findings: </strong>TBD patients have specific unique biological vulnerabilities such as T cell immunodeficiency, susceptibility to infections, hypersensitivity to chemotherapy and radiation, and cytopenias. Furthermore, multiple organ involvement at diagnosis makes clinical management especially challenging due to higher degree of organ damage, and stress-induced telomeric crisis. Sequential and combined organ transplants, development of novel radiation and alkylator-free conditioning regimen, and use of novel drugs for graft-versus-host disease prophylaxis are some of the recent updates in the field. Multidisciplinary management is essential to optimize transplant outcomes in patients with TBD. In this review, we provide consensus-based transplant management guidelines for clinical management of transplant in TBD.</p>","PeriodicalId":10852,"journal":{"name":"Current Hematologic Malignancy Reports","volume":" ","pages":"293-299"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139691449","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}