Hematology. American Society of Hematology. Education Program最新文献_第5页

How to approach the high-risk myeloma from induction through relapse? 高危骨髓瘤从诱导到复发如何处理？

IF 3.7 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2025-12-05 DOI: 10.1182/hematology.2025000762

Katja C Weisel, Christoph Schaefers, Lisa B Leypoldt

{"title":"How to approach the high-risk myeloma from induction through relapse?","authors":"Katja C Weisel, Christoph Schaefers, Lisa B Leypoldt","doi":"10.1182/hematology.2025000762","DOIUrl":"10.1182/hematology.2025000762","url":null,"abstract":"Despite the enormous progress made in the treatment of multiple myeloma (MM) with various treatment choices in frontline and relapse settings, treatment of high-risk (HR)MM remains a therapeutic challenge. Definition of HR disease is dynamic and mainly based on cytogenetic assessment showing typical cytogenetic abnormalities. Most recently, the International Myeloma Society and the International Myeloma Working Group published a revision of HR criteria, IMS-IMWG Consensus Genomic Staging. Immediate identification of HRMM at primary diagnosis is crucial to refer the patients to optimal treatment. Emerging data, especially from clinical trials designed explicitly for HR patients, demonstrated that upfront quadruplet treatment followed by consolidation and continuous combination maintenance, including high dose melphalan and autologous stem cell transplantation for patients who qualify, is the current best approach leading to a markedly improved prognosis. The primary treatment goal is the rapid achievement of sustained minimal residual disease- negative response with treatment continuation beyond to avoid early relapses and evolvement of resistant clones. A distinct subgroup of patients, the so-called functional HR patients, are defined as relapsing between 12 and 18 months despite optimal frontline treatment. The unmet need for effective treatment options in this challenging situation is now partially mitigated by the introduction of B-cell maturation antigen-directed T-cell-engaging immunotherapies. Early data show favorable outcomes, with treatments with chimeric antigen receptor T-cells achieving durable responses.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2025 1","pages":"652-658"},"PeriodicalIF":3.7,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12891410/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145677389","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Tailored treatment of acute immune-mediated thrombotic thrombocytopenic purpura. 急性免疫介导的血栓性血小板减少性紫癜的量身定制治疗。

IF 3.7 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2025-12-05 DOI: 10.1182/hematology.2025000757

Linus Alexander Völker, Paul Thomas Brinkkötter

{"title":"Tailored treatment of acute immune-mediated thrombotic thrombocytopenic purpura.","authors":"Linus Alexander Völker, Paul Thomas Brinkkötter","doi":"10.1182/hematology.2025000757","DOIUrl":"10.1182/hematology.2025000757","url":null,"abstract":"Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare but life-threatening disease caused by an autoantibody-mediated mechanism of deficiency of the von Willebrand factor (VWF)-cleaving protease ADAMTS13. Novel therapeutic principles have challenged the standard of care, consisting of therapeutic plasma exchange (TPE), glucocorticoids, and adjunct immunosuppressants. Anti-VWF therapy with caplacizumab has been shown to be effective in halting thrombotic microangiopathy earlier, preventing early relapses, and potentially lowering TTP-associated mortality. It has also opened avenues toward a more tailored treatment approach guided by ADAMTS13 activity levels and a treatment algorithm forgoing TPE. This article highlights the importance of an early diagnosis and early therapeutic interventions indiscriminate of the initial clinical presentation that ultimately enable a TPE-free treatment. Moreover, it discusses the approach to handling immunosuppression and anti-VWF therapy in cases with prolonged autoimmunity. Timely diagnosis and therapy are based on on-site ADAMTS13 testing and the immediate availability of anti-VWF therapy in an emergency situation while close monitoring of the clinical and laboratory response to anti-VWF-therapy informs the decision to forgo TPE. A novel standard of care comprising anti-VWF-therapy, glucocorticoids, and early immunosuppression with anti-CD20-agents with or without TPE in selected cases based on cases series and the MAYARI trial (NCT05468320) is currently being established.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2025 1","pages":"614-620"},"PeriodicalIF":3.7,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12891330/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145677537","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

CAR T-cell therapy and bispecific antibodies in the management of multiple myeloma. CAR - t细胞疗法和双特异性抗体在多发性骨髓瘤治疗中的应用。

IF 3.7 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2025-12-05 DOI: 10.1182/hematology.2025000721

Danai Dima, Rahul Banerjee, Doris K Hansen

{"title":"CAR T-cell therapy and bispecific antibodies in the management of multiple myeloma.","authors":"Danai Dima, Rahul Banerjee, Doris K Hansen","doi":"10.1182/hematology.2025000721","DOIUrl":"10.1182/hematology.2025000721","url":null,"abstract":"The introduction of CAR (chimeric antigen receptor) T-cell therapy and bispecific antibodies into clinical practice has revolutionized the treatment landscape of relapsed/refractory multiple myeloma (RRMM). Both modalities have shown impressive clinical efficacy with slightly different but overall manageable toxicity profiles. At present, two B-cell maturation antigen (BCMA)-targeted CAR T-cell therapies, idecabtagene vicleucel and ciltacabtagene autoleucel, are approved for standard use in the United States. There are currently 4 commercially approved bispecific antibodies: teclistamab, elranatamab, and linvoseltamab, which target BCMA, as well as talquetamab, which targets the GPRC5D antigen on the surface of plasma cells. In this review, we explore (a) the advantages and challenges of integrating CAR T-cell therapy earlier in the RRMM treatment course; (b) the safety and efficacy of bispecific antibodies and their evolving role in the current RRMM treatment paradigm; (c) practical considerations for both modalities, focusing on patient selection and supportive care strategies; and (d) recommendations for sequencing of T-cell redirecting therapies to maximize long-term outcomes.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2025 1","pages":"324-333"},"PeriodicalIF":3.7,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12891446/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145677558","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Transplant in myeloma: what is its role? 骨髓瘤移植：它的作用是什么？

IF 3.7 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2025-12-05 DOI: 10.1182/hematology.2025000730

Cesar Rodriguez

引用次数: 0

Maintenance after CAR T? Are we there yet? Reducing the risk of relapse after loss of anti-CD19 CAR T-cell persistence in ALL. CAR - T后的维护？我们到了吗？降低ALL中抗cd19 CAR - t细胞持久性丧失后复发的风险。

IF 3.7 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2025-12-05 DOI: 10.1182/hematology.2025000734

Maria Gabelli, Sara Ghorashian

{"title":"Maintenance after CAR T? Are we there yet? Reducing the risk of relapse after loss of anti-CD19 CAR T-cell persistence in ALL.","authors":"Maria Gabelli, Sara Ghorashian","doi":"10.1182/hematology.2025000734","DOIUrl":"10.1182/hematology.2025000734","url":null,"abstract":"Anti-CD19 chimeric antigen receptor (CAR) T cells can induce complete remission in the majority of children and young adults affected by multiple relapsed/refractory B-cell precursor acute lymphoblastic leukemia (ALL) and provide potential long-term cure through continuous surveillance against leukemic recurrence. The presence of B-cell aplasia represents an indirect marker of CAR T functional persistence. Patients treated with tisagenlecleucel who present with early (within 6 months from infusion) B-cell recovery (BCR) have an increased risk of relapse and merit further treatment. In this work, we describe a pediatric clinical scenario and discuss the possible interventions-that is, hematopoietic stem cell transplantation (HSCT), second CAR T infusion, and maintenance chemotherapy-for similar patients with early BCR after CAR T, based on the available literature. We advocate for HSCT with total body irradiation (TBI) in children who had never received transplantation and can safely undergo TBI, while those who already had a first HSCT with TBI or present with a contraindication to TBI, in the absence of available clinical trials, can be considered for maintenance chemotherapy, given early indications of good tolerability and promising noninferior outcomes. Patients with Philadelphia-positive ALL should receive tyrosine kinase inhibitors in the context of either HSCT or maintenance chemotherapy. For young adults, 3 different commercial CAR T are available, but so far, clinical data are insufficient to support any specific consolidation strategy.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2025 1","pages":"432-442"},"PeriodicalIF":3.7,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12891365/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145677420","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Molecular surprises in evaluations of red cell disorders. 红细胞紊乱评估中的分子惊喜。

IF 3.7 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2025-12-05 DOI: 10.1182/hematology.2025000726

Yasmin Elgammal, Wenying Zhang, Theodosia A Kalfa

{"title":"Molecular surprises in evaluations of red cell disorders.","authors":"Yasmin Elgammal, Wenying Zhang, Theodosia A Kalfa","doi":"10.1182/hematology.2025000726","DOIUrl":"10.1182/hematology.2025000726","url":null,"abstract":"Red cell disorders may present with overlapping clinical presentation and laboratory findings; in addition, complete phenotypic characterization of the patients' red cells is more challenging in the most severe cases, which are typically transfusion-dependent. The increasing availability of next-generation sequencing over the past 2 decades, initially with focused gene panels for certain disease groups, optimized to include the known coding and noncoding pathogenic variants for those diseases, has improved the accuracy and timeliness of diagnosis. The ongoing expansion to whole-exome and genome sequencing has been revealing unexpected, rare, overlooked, or previously unknown genetic disorders and expands our knowledge on the pathophysiology of known and novel human diseases. The vast information gained by genetic sequencing should still be checked against the phenotype to confirm agreement. A positive result does not always guarantee that the cause of the patient's symptoms has been identified; phenotype-genotype correlation is critical. In our era of targeted treatments and progress in gene therapy, utilization of genetic workup to improve the timing and precision of diagnosis is crucial to ensure that patients receive effective management, improving their outcome.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2025 1","pages":"370-376"},"PeriodicalIF":3.7,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12891333/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145677466","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Refractory ITP: revisiting definitions, diagnostics, and management paradigms. 难治性ITP：重新审视定义、诊断和管理范例。

IF 3.7 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2025-12-05 DOI: 10.1182/hematology.2025000720

Sandhya R Panch, Ganesh Raman, James B Bussel

引用次数: 0

Optimizing the "right" patient selection for treatment for sickle cell disease. 优化镰状细胞病治疗的“正确”患者选择。

IF 3.7 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2025-12-05 DOI: 10.1182/hematology.2025000751

Raffaella Colombatti, Giulia Reggiani

{"title":"Optimizing the \"right\" patient selection for treatment for sickle cell disease.","authors":"Raffaella Colombatti, Giulia Reggiani","doi":"10.1182/hematology.2025000751","DOIUrl":"10.1182/hematology.2025000751","url":null,"abstract":"Sickle cell disease (SCD), the most prevalent hemoglobinopathy globally, is a complex, systemic disorder with significant phenotypic variability and evolving clinical burden. As the therapeutic landscape expands-ranging from hydroxyurea and red blood cell transfusions to novel disease-modifying agents and curative approaches, such as hematopoietic stem cell transplantation and gene therapy-patient selection for each strategy becomes increasingly multifaceted. This article explores how clinical severity, genotype, age, organ involvement, access to care, and patient preferences interact in shaping therapeutic decisions. Two illustrative cases highlight how disease trajectory, individual choices, and real-world barriers influence eligibility and response to treatment. We propose a multidimensional framework for patient selection, incorporating both clinical indicators and patient-centered outcomes. While monotherapy with a single disease-modifying therapy remains the cornerstone of SCD treatment, all therapeutic decisions should be individualized. However, combination and curative approaches are more complex and require a comprehensive evaluation of efficacy, feasibility, and patient values. Emerging trends, including biomarker-driven stratification, inclusive clinical trials, and shared decision-making tools, coupled with the use of artificial intelligence, offer opportunities to better align therapy with the needs of diverse patients across settings. Ultimately, defining the \"right\" patient demands dynamic, context-sensitive evaluation, ensuring that therapeutic advances translate into equitable and valuable care for individuals with SCD globally.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2025 1","pages":"571-576"},"PeriodicalIF":3.7,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12891557/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145677502","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

IF 3.7 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2025-12-05 DOI: 10.1182/hematology.2025000728

Arnaud Jaccard, Laurent Magy, Bertrand Arnulf, Frank Bridoux, Murielle Roussel

{"title":"Monoclonal-related neuropathies: diagnosis, prognosis, and outcomes.","authors":"Arnaud Jaccard, Laurent Magy, Bertrand Arnulf, Frank Bridoux, Murielle Roussel","doi":"10.1182/hematology.2025000728","DOIUrl":"10.1182/hematology.2025000728","url":null,"abstract":"Monoclonal gammopathy is observed in approximately 10% of patients with peripheral neuropathy, which is particularly common in those with the immunoglobulin M monoclonal component. The diagnostic challenge lies in determining whether the neuropathy is causally related to the paraprotein or merely coincidental. Physicians must be familiar with the spectrum of monoclonal gammopathy-associated neuropathies, including anti-myelin-associated glycoprotein neuropathy, amyloid light chain amyloidosis, POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome, CANOMAD (chronic ataxic neuropathy, ophthalmoplegia, IgM paraprotein, cold agglutinins, and disialosyl antibodies), and cryoglobulinemia, as well as their clinical, electrophysiologic, and immunologic characteristics. Initial workup includes serum protein electrophoresis, immunofixation, free light chain analysis, and targeted antibody testing based on the paraprotein and neuropathy phenotype. Close collaboration between neurologists and hematologist-oncologists is essential for accurate diagnosis and appropriate treatment, which may involve both immunotherapy and targeted therapies for the underlying hematologic disorder.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2025 1","pages":"385-392"},"PeriodicalIF":3.7,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12891631/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145677520","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Diagnosis and management of cold agglutinin disease. 感冒凝集素病的诊断和治疗。

IF 3.7 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2025-12-05 DOI: 10.1182/hematology.2025000718

Sigbjørn Berentsen

{"title":"Diagnosis and management of cold agglutinin disease.","authors":"Sigbjørn Berentsen","doi":"10.1182/hematology.2025000718","DOIUrl":"10.1182/hematology.2025000718","url":null,"abstract":"Cold agglutinin disease (CAD) is an autoimmune hemolytic anemia, a specific clonal B-cell disorder of the bone marrow, and a monoclonal gammopathy of clinical significance. Thus, CAD should be distinguished from cold agglutinin syndrome, a more heterogeneous cold hemolytic syndrome that occurs secondary to other clinical disease. Cold agglutinins in CAD are usually of the immunoglobulin M kappa class with a heavy chain variable region encoded by the IGHV4-34 gene segment. The hemolytic anemia is entirely mediated by classical complement activation, which also explains some additional clinical features, such as fatigue and acute exacerbations. Non-complement-mediated steps in pathogenesis are also essential, such as erythrocyte agglutination and, probably, coexistent cryoglobulin activity in some patients, resulting in cold-induced circulatory symptoms. Based on this heterogeneity, different clinical phenotypes can be defined and used to guide individualized treatment. Established therapies aim at targeting the pathogenic B-cell clone or the classical complement activation pathway. Novel and investigational therapies include Bruton's tyrosine kinase inhibitors, plasma cell-directed therapies, novel complement inhibitors, and entirely new approaches such as cytokine inhibitors and, possibly, antibodies specific for the VH4-34 protein sequence. Patients with CAD requiring therapy should be considered for clinical trials.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2025 1","pages":"295-304"},"PeriodicalIF":3.7,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12891375/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145677524","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0