Expert Review of Hematology最新文献_第3页

Clinical laboratory diagnostics and monitoring of ADAMTS13 deficiency and of related diseases: an integrated approach. 临床实验室诊断和监测ADAMTS13缺乏和相关疾病：综合方法

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-09-01 Epub Date: 2026-07-20 DOI: 10.1080/17474086.2026.2702550

Nikolaus B Binder, François Depasse, Michael Spannagl, Jürgen Patzke, Stephan Schwers, Peter L Turecek

{"title":"Clinical laboratory diagnostics and monitoring of ADAMTS13 deficiency and of related diseases: an integrated approach.","authors":"Nikolaus B Binder, François Depasse, Michael Spannagl, Jürgen Patzke, Stephan Schwers, Peter L Turecek","doi":"10.1080/17474086.2026.2702550","DOIUrl":"10.1080/17474086.2026.2702550","url":null,"abstract":"Introduction: ADAMTS13, a crucial enzyme in hemostasis, that specifically cleaves von Willebrand factor (VWF), plays a pivotal role in the pathogenesis and diagnosis of thrombotic thrombocytopenic purpura (TTP), a microcirculatory form of thrombotic microangiopathy (TMA). Its deficiency leads to the accumulation of ultra-large VWF multimers, causing microvascular thrombosis. Understanding and measuring ADAMTS13 activity are essential for diagnosis and management of TTP and other thrombotic diseases associated with ADAMTS13 deficiency.Areas covered: This review explores the evolution of ADAMTS13 assays from labor-intensive methods to rapid, automated platforms. It examines congenital and acquired ADAMTS13 deficiencies, highlighting diagnostic strategies and therapeutic interventions. A comprehensive literature search was conducted between September 2025 and June 2026 using PubMed and GoogleScholar, focusing on recent advancements and extending to foundational studies when necessary.Expert opinion: The integration of rapid ADAMTS13 testing has the potential to improve clinical decision-making by shortening diagnostic uncertainty and supporting earlier differentiation of TTP from other TMAs. Despite progress, challenges remain in assay standardization, sensitivity, and interpretation. Future developments may include point-of-care technologies and conformation-sensitive assays to enhance relapse prediction and monitoring. Collaboration between clinicians and laboratories is crucial to optimize patient outcomes and advance the field of ADAMTS13 diagnostics.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1037-1057"},"PeriodicalIF":2.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148469836","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Efficacy of pacritinib versus momelotinib in patients with thrombocytopenic myelofibrosis: a matching-adjusted indirect treatment comparison. 帕西替尼与莫美洛替尼在血小板减少性骨髓纤维化患者中的疗效：一项匹配调整的间接治疗比较。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-09-01 Epub Date: 2026-06-30 DOI: 10.1080/17474086.2026.2686813

Naveen Pemmaraju, Alessandro Lucchesi, Stephen T Oh, Koo Wilson, Zalmai Hakimi, Sarah A Buckley, Purvi Suthar, Mikolaj Parkitny, Piotr Wojciechowski, Klaudia Kolonko, John O Mascarenhas

{"title":"Efficacy of pacritinib versus momelotinib in patients with thrombocytopenic myelofibrosis: a matching-adjusted indirect treatment comparison.","authors":"Naveen Pemmaraju, Alessandro Lucchesi, Stephen T Oh, Koo Wilson, Zalmai Hakimi, Sarah A Buckley, Purvi Suthar, Mikolaj Parkitny, Piotr Wojciechowski, Klaudia Kolonko, John O Mascarenhas","doi":"10.1080/17474086.2026.2686813","DOIUrl":"10.1080/17474086.2026.2686813","url":null,"abstract":"Background: Thrombocytopenia is a prognostic marker in patients with myelofibrosis, associated with increased morbidity and reduced survival. We conducted a matching-adjusted-indirect-comparison (MAIC) of pacritinib-vs-momelotinib for treatment of patients with thrombocytopenic myelofibrosis.Research design and methods: Data were from Phase 3 randomized controlled trials of patients with thrombocytopenic myelofibrosis treated with pacritinib 200 mg BID (PERSIST-2 [NCT02055781]) with baseline platelet count 24‒99 × 109/L matched on prognostic indicators with patients on momelotinib 200 mg QD (pooled post-hoc analysis of patients with platelet count <100 × 109/L (MOMENTUM [NCT04173494]; SIMPLIFY-1 [NCT01969838]). Week-24 endpoints included % patients achieving: ≥50% total symptom score reduction (TSS-50), ≥35% spleen volume reduction (SVR-35) and red-blood-cell-transfusion-independence (RBC-TI). Reweighted effects were compared with momelotinib using logistic regression/odds ratio (OR [95% confidence interval, CI]) and Cox regression/hazard ratio (HR [95% CI]) for overall survival (OS).Results: After matching, baseline characteristics balanced; 76‒81% of matched pacritinib patients had moderate thrombocytopenia (platelet count 50‒99 × 109/L). All outcomes favored pacritinib on ORs (95% CI) for TSS-50: 1.75 (0.60, 5.09); SVR-35: 1.40 (0.32, 6.04); RBC-TI: 1.19 (0.36, 3.97); and OS: HR (95% CI) 0.35 (0.12, 1.05); however, none had statistical significance.Conclusions: This MAIC revealed consistent, nominal trends toward pacritinib efficacy benefits and OS in patients with myelofibrosis, most with moderate thrombocytopenia.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1107-1115"},"PeriodicalIF":2.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148276313","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Sutimlimab for cold agglutinin disease: an updated perspective from approval to real-world clinical treatment. Sutimlimab用于感冒凝集素疾病：从批准到实际临床治疗的最新视角

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-09-01 Epub Date: 2026-07-09 DOI: 10.1080/17474086.2026.2699806

Catherine M Broome, Bruno Fattizzo

{"title":"Sutimlimab for cold agglutinin disease: an updated perspective from approval to real-world clinical treatment.","authors":"Catherine M Broome, Bruno Fattizzo","doi":"10.1080/17474086.2026.2699806","DOIUrl":"10.1080/17474086.2026.2699806","url":null,"abstract":"Introduction: Sutimlimab, a classical complement pathway (CP) inhibitor, was approved in 2022 in the US, EU, and Japan for the treatment of cold agglutinin disease (CAD), a rare form of autoimmune hemolytic anemia (AIHA) characterized by CP‑mediated extravascular hemolysis and circulatory symptoms related to IgM‑mediated red blood cell (RBC) agglutination. This review reexamines the clinical trial data and compares those findings with published real‑world experience (RWE), providing clinicians with efficacy and safety data that extend beyond the clinical trial experience in this rare AIHA.Areas covered: The first-in-human trials, the two seminal clinical trials (CARDINAL and CADENZA), and the published post‑marketing RWE are presented. Literature searches for CAD and sutimlimab were conducted in PubMed and in abstracts from ASH and EHA from 2016 to present. All articles and abstracts regarding sutimlimab and CAD were included.Expert opinion: Long-term data from clinical trials and published RWE support the safety and efficacy of sutimlimab. Targeting the CP as primary therapy for CAD offers a unique, targeted management strategy that minimizes exposure to immunosuppressive regimens. The rapid onset of sutimlimab's activity provides a potentially lifesaving treatment option in situations where immediate control of hemolysis is critical. Opportunities remain to increase our understanding of the role of complement inhibition combined with immunosuppressive therapy in CAD. The impact of complement inhibition on morbidity and mortality in CAD remains to be determined.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1027-1036"},"PeriodicalIF":2.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148404253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Advances in treatments for hemophilia A/B with inhibitors: current treatment gaps and promising therapies. 用抑制剂治疗血友病A/B的进展：目前的治疗差距和有希望的治疗方法。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-09-01 Epub Date: 2026-07-11 DOI: 10.1080/17474086.2026.2701252

Johnny Mahlangu

{"title":"Advances in treatments for hemophilia A/B with inhibitors: current treatment gaps and promising therapies.","authors":"Johnny Mahlangu","doi":"10.1080/17474086.2026.2701252","DOIUrl":"10.1080/17474086.2026.2701252","url":null,"abstract":"Introduction: Hemophilia A and B are inherited bleeding disorders caused by deficiencies of coagulation factors VIII and IX. The development of neutralizing alloantibodies (inhibitors) remains the most serious treatment complication, rendering factor replacement ineffective and substantially increasing morbidity, treatment complexity, and healthcare costs.Areas covered: A literature search was conducted using PubMed/MEDLINE, Embase, and Google Scholar for publications from January 2000 to March 2026 using combinations of the terms 'hemophilia A,' 'hemophilia B,' 'inhibitors,' 'immune tolerance induction,' 'bypassing agents,' 'emicizumab,' 'concizumab,' 'marstacimab,' 'fitusiran,' and 'gene therapy.' Relevant clinical trials, observational studies, guidelines, and review articles were evaluated. This review examines current and emerging therapies for inhibitor patients, including bypassing agents, immune tolerance induction, emicizumab, anti-tissue factor pathway inhibitor therapies, antithrombin suppression, next-generation factor mimetics, gene therapy, and immune-modulatory approaches. Pivotal clinical trials demonstrate major reductions in bleeding rates and significant advances in prophylactic care.Expert opinion: Despite transformative progress, important unmet needs remain, including incomplete hemostatic control, lack of predictive biomarkers, limited laboratory standardization, and inequitable global access. Future advances will depend on biomarker-guided personalized therapy, rational combination strategies, and integration of immune-modulatory and gene-based approaches to achieve durable tolerance and potentially curative outcomes.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1059-1076"},"PeriodicalIF":2.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148404197","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Prognostic factors for patients with relapsed or refractory multiple myeloma who are initiating a fourth or later line car T-cell or bispecific antibody therapy: a systematic literature review and meta-analysis. 复发或难治性多发性骨髓瘤患者开始第四线或更晚的car - t细胞或双特异性抗体治疗的预后因素：系统文献综述和荟萃分析

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-09-01 Epub Date: 2026-06-22 DOI: 10.1080/17474086.2026.2688385

Krina Patel, Ken Hasegawa, Jie Ting, Surbhi Sidana, Michael J Zoratti, David Wennersbusch, Steve Kanters, Ana Kostic, Taha Itani, Doris K Hansen

{"title":"Prognostic factors for patients with relapsed or refractory multiple myeloma who are initiating a fourth or later line car T-cell or bispecific antibody therapy: a systematic literature review and meta-analysis.","authors":"Krina Patel, Ken Hasegawa, Jie Ting, Surbhi Sidana, Michael J Zoratti, David Wennersbusch, Steve Kanters, Ana Kostic, Taha Itani, Doris K Hansen","doi":"10.1080/17474086.2026.2688385","DOIUrl":"10.1080/17474086.2026.2688385","url":null,"abstract":"Introduction: Multiple myeloma is associated with substantial disease burden and frequent relapses, despite therapeutic advances. As evidence for chimeric antigen receptor (CAR) T-cell and bispecific antibody (BsAb) therapies is dominated by single-arm trials and real-world studies, understanding key prognostic factors is critical for informing indirect treatment comparisons (ITCs).Methods: A systematic literature review of MEDLINE, EMBASE, CENTRAL, and pertinent conferences for trials and observational studies evaluating CAR T-cell and/or BsAb therapies. Eligible studies presented potential factors in relation to efficacy, survival, and/or safety outcomes. Meta-analyses summarized factor-outcome associations, overall and by treatment class.Results: From 102 publications (43 studies), analyses were feasible for 15 potential factors. Statistically significant factor-outcome dyads were identified, including factors of age, cytogenetic risk, ECOG PS, ISS/R-ISS stage, bone marrow plasma cell burden, extramedullary disease, ferritin, prior anti-BCMA exposure, and triple-class refractory status. Associations varied by outcome and, in some cases, by treatment class.Conclusions: This study highlights prognostic factors, identified through a systematic and transparent process, that may influence ITCs and policy decision-making in later-line multiple myeloma.Registration: PROSPERO (CRD42023446880). Only pilot work and no formal review or data collection occurred prior to the registration.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1091-1106"},"PeriodicalIF":2.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148217187","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Are electrical stimulation devices the way forward for addressing heavy menstrual bleeding in women with von Willebrand disease? 电刺激装置是治疗血管性血友病女性月经大量出血的可行方法吗？

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-09-01 Epub Date: 2026-07-07 DOI: 10.1080/17474086.2026.2699801

Maureen K Baldwin

引用次数: 0

The emerging and evolving role of flow cytometry in myelodysplastic syndromes diagnosis. 流式细胞术在骨髓增生异常综合征诊断中的新兴和发展作用。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-09-01 Epub Date: 2026-06-14 DOI: 10.1080/17474086.2026.2688383

Emre Akar, Seval Akpınar, Mehmet Baysal

引用次数: 0

Supportive care, prevention, and emerging therapies for acute chest syndrome in sickle cell disease. 镰状细胞病急性胸综合征的支持性护理、预防和新疗法

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-09-01 Epub Date: 2026-07-14 DOI: 10.1080/17474086.2026.2702549

Rachel R Rice, Shaina M Willen

{"title":"Supportive care, prevention, and emerging therapies for acute chest syndrome in sickle cell disease.","authors":"Rachel R Rice, Shaina M Willen","doi":"10.1080/17474086.2026.2702549","DOIUrl":"10.1080/17474086.2026.2702549","url":null,"abstract":"Introduction: Acute chest syndrome (ACS) is the leading cause of death and a major cause of hospitalization in sickle cell disease (SCD). Despite advances in care, ACS remains a life-threatening complication with no FDA-approved targeted therapy for established disease. This review examines the current evidence base for supportive care, preventive strategies, and emerging therapies.Areas covered: We conducted a comprehensive literature search of PubMed, clinical trial registries, and major hematology conferences through December 2025. This review synthesizes data on supportive measures (fluid management, analgesia, antibiotics, oxygen/respiratory support, and transfusion), preventive interventions (incentive spirometry and nocturnal bilevel positive airway pressure), disease-modifying therapies (hydroxyurea, L-glutamine), investigational agents (therapeutic anticoagulation, inhaled nitric oxide, and pyruvate kinase activators), and recently approved gene therapies (exagamglogene autotemcel, lovotibeglogene autotemcel). Pathophysiology, biomarkers, and barriers to implementation in resource-limited settings are also addressed.Expert opinion: Supportive care has reduced ACS mortality but remains reactive and non-curative. Hydroxyurea remains first-line prevention, while L-glutamine offers a complementary option. Gene therapies represent a paradigm shift toward durable or potentially curative prevention of ACS. With expanding access to these therapies, ACS may become a rare complication of SCD, although significant barriers related to cost, infrastructure, and global equity must be overcome.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1009-1017"},"PeriodicalIF":2.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148435892","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The impact of a cancer diagnosis for myeloproliferative neoplasm (MPN) patients. 癌症诊断对骨髓增生性肿瘤（MPN）患者的影响。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-09-01 Epub Date: 2026-07-02 DOI: 10.1080/17474086.2026.2696287

Jon Mathias

引用次数: 0

Myelofibrosis treatment sequencing: mutation-informed risk and optimal JAK inhibitor discontinuation. 骨髓纤维化治疗测序：突变知情风险和最佳JAK抑制剂停药。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-09-01 Epub Date: 2026-07-13 DOI: 10.1080/17474086.2026.2688872

Giulia Benevolo, Giuseppe Lanzarone, Benedetta Rosti, Eloise Beggiato

{"title":"Myelofibrosis treatment sequencing: mutation-informed risk and optimal JAK inhibitor discontinuation.","authors":"Giulia Benevolo, Giuseppe Lanzarone, Benedetta Rosti, Eloise Beggiato","doi":"10.1080/17474086.2026.2688872","DOIUrl":"10.1080/17474086.2026.2688872","url":null,"abstract":"Introduction: Myelofibrosis (MF) is a heterogeneous myeloproliferative neoplasm characterized by splenomegaly, constitutional symptoms, cytopenias, and risk of leukemic transformation. As therapeutic options expand, treatment discontinuation and sequencing are increasingly recognized as clinically relevant determinants of patient outcomes.Areas covered: A literature search was performed using PubMed/MEDLINE and major hematology conference proceedings (ASH and EHA), covering publications from January 2010 through February 2026. This review discusses mutation-informed risk stratification, current and emerging JAK inhibitor treatment sequencing strategies, and practical approaches to treatment discontinuation and therapeutic transition in MF.Expert opinion: Optimizing outcomes in MF increasingly requires proactive transition management rather than reactive treatment interruption. Early identification of treatment failure, phenotype-guided therapeutic sequencing, and integration of molecular risk assessment may improve long-term disease management and could contribute to better clinical outcomes, although prospective confirmation remains necessary.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1077-1089"},"PeriodicalIF":2.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148435837","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0