Blood Reviews最新文献

{"title":"Programmed death inhibitors in CNS B-cell lymphoma: A literature review.","authors":"Mostafa Mohammed Saleh, Walid Rasheed, Saud Alhayli, Alfadel Alshaibani, Mahmoud Aljurf, Riad El Fakih","doi":"10.1016/j.blre.2025.101321","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101321","url":null,"abstract":"<p><p>Checkpoint inhibitors emerged as a promising therapeutic approach in the management of central nervous system lymphomas, particularly primary and secondary B-cell CNS lymphomas. These are aggressive malignancies and carry a poor prognosis with limited treatment options. Advances in tumor immunology highlighted the role of immune checkpoint pathways, especially PD-1/PD-L1 axis, in facilitating immune evasion in CNS lymphomas. Preclinical studies demonstrated a suppressive tumor microenvironment, often with expression of checkpoint ligands and infiltration by exhausted T cells, suggesting potential sensitivity to immune checkpoint blockade. Clinical experience, including early phase studies, showed encouraging response rates and durable remissions in patients treated with PD-1 inhibitors. Combination strategies incorporating checkpoint inhibitors with targeted therapies are under investigation and may enhance treatment efficacy while maintaining manageable toxicity profiles. As evidence accumulates, checkpoint blockade is increasingly viewed as a potential treatment strategy in CNS lymphomas, warranting further exploration in prospective trials and biomarker-driven studies.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101321"},"PeriodicalIF":6.9,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692581","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Current trends and advances in the management of acquired Hemophilia A.","authors":"Emilie Zuner, Stéphanie Désage, Hamdi Rezigue, Yesim Dargaud, Anne Lienhart, Christophe Nougier","doi":"10.1016/j.blre.2025.101320","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101320","url":null,"abstract":"<p><p>Acquired hemophilia A (AHA), a rare and life-threatening bleeding disorder of adults, caused by anti-factor VIII (FVIII) autoantibodies, is often underestimated, particularly in patients receiving antithrombotic therapy. AHA is often associated with autoimmune disease, cancer, infection or pregnancy. AHA, characterized by severe spontaneous bleeding, particularly in muscles and subcutaneous tissues, requires a timely and accurate diagnosis. Delayed diagnosis worsens the prognosis and increases the risk of complications. Disease confirmation requires identification of FVIII deficiency and anti-FVIII antibodies. Treatment focuses on managing acute bleeding episodes, addressing the underlying condition and eradicating auto-antibodies through immunosuppressive therapy. Bypassing agents are used for treatment, but promising new therapeutic options such as emicizumab are under evaluation. AHA remains a serious condition with high mortality from bleeding complications and adverse effects of immunosuppression. This review provides a comprehensive overview of current knowledge on AHA, including epidemiology, pathophysiology, diagnosis, prognostic factors and therapeutic strategies.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101320"},"PeriodicalIF":6.9,"publicationDate":"2025-07-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144669093","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluation of current mouse in vivo models and advanced in vitro models for leukaemia research.","authors":"Xenia Bubnova, Lauren Hope, Helen Wheadon","doi":"10.1016/j.blre.2025.101318","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101318","url":null,"abstract":"<p><p>Leukaemia is caused by genetic mutations within haematopoietic stem and progenitor cells, leading to the production of immature blasts. While mouse models have been instrumental in studying disease mechanisms and testing therapies, their limitations contribute to 90 % failure rate of new therapies in clinical trials. This is often attributed to the choice of model utilised, and failure of mouse models to accurately replicate the complexity of the human disease. This review examines different leukaemia mouse models, including transgenic, syngeneic and xenografts, discussing their phenotype, advantages and limitations. Finally, we describe advanced technologies for in vitro modelling of haematopoiesis and leukaemia. These models provide a promising platform for tumour microenvironment research, and a robust human-relevant pipeline for drug screening, reducing our reliance on in vivo testing. The information in this review will enable researchers to make informed decisions on the most appropriate models to carry out pre-clinical testing in the future.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101318"},"PeriodicalIF":6.9,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144531303","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Iron chelation therapy in myelodysplastic syndromes and allogeneic hematopoietic cell transplantation, a delicate balance.","authors":"Fieke W Hoff, Eduard Schulz, Steven Pavletic, Alain Mina","doi":"10.1016/j.blre.2025.101319","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101319","url":null,"abstract":"<p><p>Anemia is a hallmark of myelodysplastic syndromes/neoplasms (MDS) and most patients with MDS chronically require red blood cell transfusions. Due to the body's inability to excrete excess iron, patients are at increased risk of iron overload, often defined by ferritin levels >1000 ng/mL. Iron overload can cause progressive organ damage from iron deposition in tissues and has been linked to increased mortality. In MDS patients undergoing allogeneic hematopoietic cell transplantation (HCT), iron overload has also been associated with increased non-relapse mortality, decreased overall survival, and a higher incidence of relapse. Prospective and retrospective studies have demonstrated the safety and clinical benefit of iron chelation therapy (ICT) in lower-risk MDS. Despite some common adverse effects associated with ICT, such as renal toxicity and gastro-intestinal symptoms, managing iron levels remains essential in transfusion-dependent MDS patients, and those who are undergoing HCT to optimize pre-transplant conditions, and enhance post-transplant outcomes.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101319"},"PeriodicalIF":6.9,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144531304","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Novel therapies for acute myeloid leukemia. Does age still matter?","authors":"Baher Krayem, Avraham Frisch, Netanel Horowitz","doi":"10.1016/j.blre.2025.101317","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101317","url":null,"abstract":"<p><p>The prognosis of patients with AML varies significantly with age, driven by biological heterogeneity and age-associated factors such as comorbidities, functional status, and hospitalization burden. Many novel therapies have been approved in recent years; however, pivotal trials often include patients within a restricted age range, limiting the extrapolation of their findings across the broader AML population. For example, the FLT3 inhibitor midostaurin was added to chemotherapy for patients aged 18-60 years, while the BCL-2 inhibitor venetoclax was combined with azacitidine in patients aged 75 years and older, leaving important knowledge gaps regarding their efficacy and safety in other age groups. Moreover, for several novel therapies, particularly in populations outside the original trial age range, supporting evidence is derived primarily from single-arm studies or real-world experience rather than randomized controlled trials, further complicating clinical decision-making. This review explores the efficacy and safety of widely used traditional and novel therapies for AML, with particular focus on the impact of age on these different therapeutic regimens.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101317"},"PeriodicalIF":6.9,"publicationDate":"2025-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144303630","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exploring the depths of hypogammaglobulinemia in lymphoid malignancies: Pathophysiology, clinical implications, management options, and future directions.","authors":"Alex Wonnaparhown, Talal Hilal, Albert Chong, Rafael Fonseca","doi":"10.1016/j.blre.2025.101316","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101316","url":null,"abstract":"<p><p>The treatment of lymphoid malignancies is rapidly advancing with recognition of hypogammaglobulinemia (HG) and increased infection risk with the use of cellular-targeted therapies, such as chimeric antigen receptor (CAR) T cell and bispecific antibody (BsAb) therapy. Underlying adaptive immune dysfunction seen in malignancy, immunoparesis, and prior lines of B cell-targeting therapies also predispose patients to HG and infections prior to more advanced cellular therapies. Clinicians should become familiar with how to evaluate adaptive immunity and causes of HG. Various strategies exist to reduce infections in immunosuppressed patients, including risk-reducing practices, vaccination, prophylactic antibiotics, and IgG replacement therapy (IgG-RT). Future research should focus on identifying biomarkers to help with infectious risk-stratification and identify populations that would best benefit from IgG-RT.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101316"},"PeriodicalIF":6.9,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144327885","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Advances in the critical care management for patients with hematological malignancies.","authors":"Elie Azoulay, Lara Zafrani, Joseph Nates, Alexis Maillard, Dara Chean, Bruno Ferreyro, Judith E Nelson, Philippe R Bauer, Kathryn Puxty, Cristina Gutierrez, Naike Bigé, Eric Mariotte, Sandrine Valade, Boris Boell, Kathleen Puntillo, Antoine Lafarge, Marcio Soares, Peter Schellongowski, Emmanuel Canet, Pedro Castro, Alexandre Demoule, Frédéric Pène, Laveena Munshi, Alexander Shimabukuro-Vornhagen, Thomas Staudinger, Lene Russell, Sara Fernandez, Matthias Kochanek, Virginie Lemiale, Michael von Bergwelt-Baildon, Michael Darmon, Ignacio Martin-Loeches","doi":"10.1016/j.blre.2025.101306","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101306","url":null,"abstract":"<p><p>Hematological malignancies (HMs) are increasingly associated with life-threatening complications requiring intensive care unit (ICU) management. Recent advancements in therapies, diagnostics, and critical care protocols have improved outcomes for these patients, yet significant challenges persist. This manuscript explores the evolving landscape of critical care in hematology, emphasizing the unique complications, management strategies, and future directions in the field. Patients with HMs are particularly vulnerable to infections, sepsis, organ dysfunction, and treatment-related toxicities such as cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and coagulopathies. Innovations in the management of acute respiratory failure, septic shock, and invasive fungal infections have contributed to better survival rates, yet outcomes remain suboptimal for certain high-risk groups. Furthermore, new therapies, including CAR-T cells, bispecific antibodies, and immune checkpoint inhibitors, present both opportunities and challenges in the ICU setting due to their potential toxicities. Emerging trends emphasize the importance of early ICU admission, multidisciplinary collaboration, and precision medicine in improving patient care. The integration of biomarker-driven strategies, advanced diagnostics, and artificial intelligence holds promise for optimizing therapeutic interventions and enhancing antimicrobial stewardship. Additionally, patient-centered approaches, including time-limited trials and goal-oriented discussions, aim to balance aggressive care with quality-of-life considerations. This review underscores the need for continued research to address disparities in access to care, improve long-term outcomes, and develop standardized protocols for managing critically ill hematology patients. By advancing the integration of oncology and critical care, clinicians can better navigate the complexities of modern therapies and provide holistic, evidence-based care that aligns with patient values and priorities.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101306"},"PeriodicalIF":6.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144555965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Tackling myeloma bone disease: From pathophysiology to cutting-edge therapies.","authors":"Sophie Roux, Françoise Debiais, Marie-Hélène Vieillard","doi":"10.1016/j.blre.2025.101305","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101305","url":null,"abstract":"<p><p>Bone involvement in multiple myeloma (MM) is marked by osteolysis, driven by excessive bone resorption and a profound suppression of bone formation. Interactions between MM cells and the bone microenvironment-mediated by integrins,chemokines, and bone marrow stromal cells-play a critical role in the development of myeloma bone disease (MBD). Key players include osteoclasts, osteoblasts, and osteocytes. Osteoclast activation is mainly driven by RANK/RANKL and other pro-osteoclastogenic factors that disrupt bone remodeling, while impaired bone formation involves Wnt signaling inhibition and Runx2/Cbfa1 suppression. Emerging therapeutic strategies are focused on addressing both the tumor burden and the bone remodeling imbalance, with advances in molecular targeting and microRNA-based approaches. Similarly, novel anti-myeloma therapies show promise for MBD, though their full impact is not yet defined. This review highlights recent findings in MM-associated bone disease and discusses current and prospective therapies aimed at improving patient outcomes.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101305"},"PeriodicalIF":6.9,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144182785","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Unmet needs in hemophilic arthropathy.","authors":"Yesim Dargaud, Sebastien Lobet, Nathalie Roussel, Leonard A Valentino","doi":"10.1016/j.blre.2025.101304","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101304","url":null,"abstract":"<p><p>Hemophilia A and B are rare X-linked bleeding disorders caused by coagulation factor deficiencies, leading to joint bleeding, synovial hypertrophy and chronic hemophilic arthropathy marked by progressive cartilage and bone damage. Musculoskeletal issues remain the primary source of morbidity in people with hemophilia (PwH). Despite significant advances in prophylactic therapies, joint pain, functional limitations, and deterioration persist. The long-term impact of novel treatments on joint health and physical activity levels remains incompletely understood. Early detection and prevention of damage is challenging, highlighting the need for highly sensitive diagnostic tools to identify subclinical changes before irreversible damage occurs. Pain management, currently adapted from other conditions, does not fully meet the unique needs of PwH. Research into targeted pain relief, synovial hypertrophy management, and cartilage regeneration is crucial. Addressing unmet needs in diagnosis, treatment, and management requires collaboration between clinical and research communities to improve care effectiveness and enhance the quality of life for PwH.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101304"},"PeriodicalIF":6.9,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144144599","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient-reported outcomes - the missing link to advancing light chain (AL) amyloidosis clinical research.","authors":"Tobias Dittrich, Lina Weinert, Anita D'Souza","doi":"10.1016/j.blre.2025.101303","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101303","url":null,"abstract":"<p><p>Systemic light chain (AL) amyloidosis often results in multi-organ dysfunction and significant morbidity. Clinical assessments may not capture the full impact of disease and treatment on patients. Patient-reported outcomes (PROs) can help fill this gap. Although evidence suggests that the use of PROs provides additional predictive value beyond established cardiac staging systems, their integration into standard AL amyloidosis management remains limited. Our review examines the prognostic and therapeutic value of PROs and their current use as endpoints in clinical trials. We also discuss practical considerations, including instrument selection and administration, data interpretation, and reporting. Finally, we present a roadmap for integrating PROs into routine AL amyloidosis management, focusing on the selection of appropriate ePRO platforms and implementation strategies. We advocate a framework for data sharing and a coordinated research agenda. By addressing evidence gaps and prioritizing the patient perspective, PROs have the potential to advance AL amyloidosis care and research.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101303"},"PeriodicalIF":6.9,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144144584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}