Expert Review of Hematology最新文献

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Therapeutic options for relapsed acute myeloid leukemia after hematopoietic stem cell transplantation. 造血干细胞移植后复发性急性髓性白血病的治疗选择。
IF 2.1 4区 医学
Expert Review of Hematology Pub Date : 2025-08-13 DOI: 10.1080/17474086.2025.2547866
Alkim Yolcu, Elifcan Aladag Karakulak, Hakan Goker
{"title":"Therapeutic options for relapsed acute myeloid leukemia after hematopoietic stem cell transplantation.","authors":"Alkim Yolcu, Elifcan Aladag Karakulak, Hakan Goker","doi":"10.1080/17474086.2025.2547866","DOIUrl":"https://doi.org/10.1080/17474086.2025.2547866","url":null,"abstract":"<p><strong>Introduction: </strong>Acute myeloid leukemia (AML) is a malignant clonal hematopoietic cell disorder, characterized by impaired hematopoiesis and bone marrow failure. Allogeneic hematopoietic stem cell transplantation (allo-HCT) is an established therapy with curative potential. Post-transplant relapse does occur and has dismal prognosis, which is the main cause of death after allo-HCT. Relapses after allo-HCT in AML do cause standard treatment approaches challenging, which often necessitate individualized treatment modalities and large prospective clinical trials are needed to delineate standardized therapies. The management of AML relapse after allo-HCT remains a clinical challenge, and no standardized treatment approach currently exists.</p><p><strong>Areas covered: </strong>This review provides an overview of the available therapeutic options for patients with relapsed AML after allo-HCT. It provides a beneficial framework on the optimal treatment approach, including factors that influence drug preferences. A search of papers published up to March 2025 was conducted on PubMed using the keywords.</p><p><strong>Expert opinion: </strong>Combining chemotherapy, targeted agents, and immunotherapy can increase the rate of response and survival for relapsed AML after allo-HCT. Future research is needed to develop strategies that can reduce the risk of relapse after allo-HCT. Individualization of treatments and exploration of combination therapies are potential approaches for improving efficacy.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":""},"PeriodicalIF":2.1,"publicationDate":"2025-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144834685","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
Identifying high-risk patients with MALT lymphoma undergoing first-line therapy for disease progression. 识别因疾病进展而接受一线治疗的高危MALT淋巴瘤患者。
IF 2.1 4区 医学
Expert Review of Hematology Pub Date : 2025-08-12 DOI: 10.1080/17474086.2025.2547834
John Sharp, Adam J Olszewski, Narendranath Epperla
{"title":"Identifying high-risk patients with MALT lymphoma undergoing first-line therapy for disease progression.","authors":"John Sharp, Adam J Olszewski, Narendranath Epperla","doi":"10.1080/17474086.2025.2547834","DOIUrl":"https://doi.org/10.1080/17474086.2025.2547834","url":null,"abstract":"<p><strong>Introduction: </strong>Marginal zone lymphoma (MZL) is an indolent B-cell non-Hodgkin lymphoma (iNHL) with mucosa-associated lymphoid tissue (MALT) lymphoma being the most common subtype. Typical of other iNHLs, a fraction of patients will experience more aggressive disease marked by early relapse despite effective front-line therapy.</p><p><strong>Area covered: </strong>In this review, we discuss existing prognostic scoring systems for patients with MALT lymphoma undergoing first-line therapy, including the MALT-IPI, the Revised MALT-IPI, the MZL-IPI, and the EMZL-MPI. We review strengths and limitations of these systems and discuss advances in the current understanding of molecular and genomic drivers of lymphomagenesis in this disease, which are not currently reflected in prognostic scoring systems. We review evolving first-line treatment approaches and discuss how there may be opportunities to refine our ability to prognosticate outcomes because of these recent advances.</p><p><strong>Expert opinion: </strong>Existing prognostic scoring systems for MALT lymphoma each have their own merit and are valuable for informing risk assessment in both clinical and research contexts, however, as understanding of the molecular and genetic drivers of MALT lymphomas improves and first-line treatment approaches incorporate novel targeted and immune-based therapies, there will likely be opportunities to enhance the current prognostic models.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":""},"PeriodicalIF":2.1,"publicationDate":"2025-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144821055","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
Indicators of an increased risk of therapy-related myeloid neoplasms in lymphoma patients: how can we best evaluate severe impairment of bone marrow function? 淋巴瘤患者治疗相关髓系肿瘤风险增加的指标:我们如何最好地评估骨髓功能的严重损害?
IF 2.1 4区 医学
Expert Review of Hematology Pub Date : 2025-08-09 DOI: 10.1080/17474086.2025.2545344
Tadeusz Kubicki, Anna Puła, Aleksandra Gołos, Łukasz Bołkun, Bartosz Puła
{"title":"Indicators of an increased risk of therapy-related myeloid neoplasms in lymphoma patients: how can we best evaluate severe impairment of bone marrow function?","authors":"Tadeusz Kubicki, Anna Puła, Aleksandra Gołos, Łukasz Bołkun, Bartosz Puła","doi":"10.1080/17474086.2025.2545344","DOIUrl":"10.1080/17474086.2025.2545344","url":null,"abstract":"<p><strong>Introduction: </strong>The development of new lymphoma therapies in recent years has led to a significant increase in patient survival. However, in some cases, despite disease remission, the outcome of the applied therapy is diminished by the development of secondary cancers. These often have dismal outcomes and are unresponsive to standard therapies. Both therapy-related myelodysplastic syndrome (t-MDS) and therapy-related acute myeloid leukemia (t-AML) are well-known side effects of cytotoxic chemotherapy and/or radiation therapy.</p><p><strong>Areas covered: </strong>This review summarizes the key factors associated with an increased risk of therapy-related neoplasms in lymphoma patients, focusing on the various elements that may contribute to this susceptibility. The major factors described in detail include the impact of different anti-lymphoma therapies, disturbances in the bone marrow microenvironment, the presence of clonal hematopoiesis, and germline predispositions. The review is based on a PubMed database search for articles published up to 1 May 2025, covering the above mentioned topics, as well as the authors' clinical and research experience.</p><p><strong>Expert opinion: </strong>Considering the progress in lymphoma therapy and the prospect of long-term survival, efforts should be made to identify patients at higher risk of developing therapy-related myeloid neoplasms. A better understanding of germline predispositions and the role of clonal hematopoiesis should support therapy tailored to individual risk profiles.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-12"},"PeriodicalIF":2.1,"publicationDate":"2025-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144798625","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
Cartilage degeneration in hemophilic arthropathy: to focus on prevention or regeneration? 血友病关节软骨退行性变:重点是预防还是再生?
IF 2.1 4区 医学
Expert Review of Hematology Pub Date : 2025-08-08 DOI: 10.1080/17474086.2025.2545339
Emerito Carlos Rodriguez-Merchan
{"title":"Cartilage degeneration in hemophilic arthropathy: to focus on prevention or regeneration?","authors":"Emerito Carlos Rodriguez-Merchan","doi":"10.1080/17474086.2025.2545339","DOIUrl":"10.1080/17474086.2025.2545339","url":null,"abstract":"<p><strong>Introduction: </strong>One of the problems that remain unsolved in people with hemophilia (PwH) is how to regenerate articular cartilage.</p><p><strong>Areas covered: </strong>In this article, we have reviewed the existing information on articular cartilage regeneration in the general population and in PwH. In the general population, it has been reported that the clinical results of different types of commercial products are better than those of microfractures and that the results of products that combined microfractures and allograft products are better than those of autologous chondrocyte products. In PwH iron chelators, anti-inflammatory drugs, and anti-fibrinolytics drugs have shown beneficial effects, predominantly in preclinical studies. Hemarthrosis models have shown that mesenchymal stem cells (MSCs) intraarticular injections have some beneficial effects on cartilage structure and function. An <i>in vitro</i> preliminary study has highlighted the opportunity of using hemophilic chondrocytes for autologous cartilage implants in PwH. In a clinical study, bone marrow-derived cells transplantation (BMDCT) seemed to be a promising regenerative treatment for osteochondral lesions in mild ankle hemophilic arthropathy in PwH.</p><p><strong>Expert opinion: </strong>Today it is not possible to regenerate articular cartilage. It is essential to perform primary hematologic prophylaxis throughout the life of PwH to prevent the articular cartilage from degeneration.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-5"},"PeriodicalIF":2.1,"publicationDate":"2025-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144788594","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
Cognitive impairment in hematology patients planned for chimeric antigen receptor T-cell therapy. 血液病患者的认知障碍计划进行嵌合抗原受体t细胞治疗。
IF 2.1 4区 医学
Expert Review of Hematology Pub Date : 2025-08-08 DOI: 10.1080/17474086.2025.2542867
Valeriya Kuznetsova, Hannah Rosenfeld, Carmela Sales, Sam van der Linde, Izanne Roos, Stefanie Roberts, Fiore D'Aprano, Samantha M Loi, Mark Dowling, Michael Dickinson, Tomas Kalincik, Simon J Harrison, Charles B Malpas, Mary Ann Anderson
{"title":"Cognitive impairment in hematology patients planned for chimeric antigen receptor T-cell therapy.","authors":"Valeriya Kuznetsova, Hannah Rosenfeld, Carmela Sales, Sam van der Linde, Izanne Roos, Stefanie Roberts, Fiore D'Aprano, Samantha M Loi, Mark Dowling, Michael Dickinson, Tomas Kalincik, Simon J Harrison, Charles B Malpas, Mary Ann Anderson","doi":"10.1080/17474086.2025.2542867","DOIUrl":"10.1080/17474086.2025.2542867","url":null,"abstract":"<p><strong>Background: </strong>Chimeric antigen receptor T-cell (CAR-T) therapy is used to treat several types of relapsed and refractory hematological malignancies and is associated with cognitive side-effects. The accurate diagnosis of cognitive impairment following CAR-T requires knowledge of baseline cognitive status prior to the therapy.</p><p><strong>Research design and methods: </strong>Adult patients with advanced hematologic or solid organ malignancies underwent cognitive assessment, including a self-report questionnaire of psychopathology and subjective cognitive function, prior to receiving CAR-T. A subset of individuals also completed the Montreal Cognitive Assessment (MoCA) to examine utility of cognitive screening.</p><p><strong>Results: </strong>Of 60 patients included, 16 (27%) had cognitive impairment, with six unique patterns of dysfunction. Memory impairment was the most common finding (15%). Impaired patients were more likely to have B-cell acute lymphoblastic leukemia (<i>p</i> = 0.024, BF<sub>10</sub> = 9.30), be younger (<i>p</i> = 0.007, BF<sub>10</sub> = 7.76), have bone marrow involvement (<i>p</i> = 0.037, BF<sub>10</sub> = 5.18), or have evidence of psychopathology (<i>p</i> = 0.004, BF<sub>10</sub> = 31.30). Analyses did not support the utility of cognitive screening. Of those patients who completed a self-report measure of psychopathology, nine (16%) were elevated on at least one symptom domain.</p><p><strong>Conclusions: </strong>The findings demonstrate a broad spectrum of cognitive and psychological symptoms, emphasizing the importance of baseline evaluation for detecting cognitive symptoms that might arise after CAR-T.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-11"},"PeriodicalIF":2.1,"publicationDate":"2025-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144759595","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
Reduced-intensity conditioning for allogeneic transplantation in classical Hodgkin lymphoma. 经典霍奇金淋巴瘤同种异体移植的低强度调节。
IF 2.1 4区 医学
Expert Review of Hematology Pub Date : 2025-08-08 DOI: 10.1080/17474086.2025.2545336
Hiba Narvel, Gulrayz Ahmed, Mehdi Hamadani
{"title":"Reduced-intensity conditioning for allogeneic transplantation in classical Hodgkin lymphoma.","authors":"Hiba Narvel, Gulrayz Ahmed, Mehdi Hamadani","doi":"10.1080/17474086.2025.2545336","DOIUrl":"10.1080/17474086.2025.2545336","url":null,"abstract":"<p><strong>Introduction: </strong>Hodgkin lymphoma (HL) is a curable disease; however, 10-20% of patients experience relapsed/refractory disease. While autologous hematopoietic cell transplantation (auto-HCT) remains standard, a substantial proportion relapse, necessitating alternative strategies. Allogeneic HCT (allo-HCT) remains a potentially curative option and here we emphasize the role of reduced-intensity conditioning (RIC) in HL.</p><p><strong>Areas covered: </strong>In this review, we compare the feasibility, efficacy, and safety of myeloablative conditioning (MAC) and RIC allo-HCT in relapsed/refractory HL. Additionally, we describe the evolving landscape of transplantation in HL with the use of novel agents, especially immune checkpoint inhibitors, the role of alternative donors especially for ethnic minorities, and the evolving literature on the role of post-transplant Cyclophosphamide (PTC) in improving outcomes.</p><p><strong>Expert opinion: </strong>Allo-HCT remains a potentially curative option for patients with relapsed/refractory HL. RIC allo-HCT has emerged as the preferred platform for most patients, offering a favorable balance between efficacy and tolerability by leveraging graft-versus-malignancy (GVM) effects while minimizing non-relapse mortality over myeloablative conditioning. The use of ICI in the first line has significantly altered post-transplant outcomes by enhancing GVM effects but also increasing the risk of graft-versus-host disease (GVHD). PTCy-based prophylaxis and optimized donor selection now enable the safer use of alternative donors without compromising outcomes.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-10"},"PeriodicalIF":2.1,"publicationDate":"2025-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144788596","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
Is antithrombotic therapy necessary after stenting of nonthrombotic iliac vein lesions? 非血栓性髂静脉病变支架置入术后是否需要抗血栓治疗?
IF 2.1 4区 医学
Expert Review of Hematology Pub Date : 2025-08-06 DOI: 10.1080/17474086.2025.2545343
Sergey G Gavrilov
{"title":"Is antithrombotic therapy necessary after stenting of nonthrombotic iliac vein lesions?","authors":"Sergey G Gavrilov","doi":"10.1080/17474086.2025.2545343","DOIUrl":"10.1080/17474086.2025.2545343","url":null,"abstract":"","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-2"},"PeriodicalIF":2.1,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144788595","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
Determining treatment pathways for older patients with acute myeloid leukemia: patient and clinician perspectives. 确定老年急性髓性白血病患者的治疗途径:患者和临床医生的观点。
IF 2.1 4区 医学
Expert Review of Hematology Pub Date : 2025-08-01 Epub Date: 2025-06-22 DOI: 10.1080/17474086.2025.2521397
Esther N Oliva, Antonio Almeida
{"title":"Determining treatment pathways for older patients with acute myeloid leukemia: patient and clinician perspectives.","authors":"Esther N Oliva, Antonio Almeida","doi":"10.1080/17474086.2025.2521397","DOIUrl":"10.1080/17474086.2025.2521397","url":null,"abstract":"<p><strong>Introduction: </strong>Achieving remission and prolonging duration of response are the primary treatment objectives for patients with acute myeloid leukemia (AML). However, identifying the best approach for older patients poses a significant challenge. This review explores the treatment pathways for older patients, especially those not eligible for stem cell transplantation and emphasizes the importance of optimizing outcomes by actively involving patients in their care plans.</p><p><strong>Areas covered: </strong>There is currently no clinical consensus on when to use intensive or less-intensive induction chemotherapy for older patients, and more empirical evidence is needed. Meanwhile, this decision must still account for patients' preferences and circumstances in addition to the benefits and risks of therapy. Survey data have shown that patients want to be involved in their treatment decisions and that higher patient engagement improves patient-reported quality of care and satisfaction. While the importance of patient engagement is widely recognized, clinicians can work toward bridging the gap between patients' preferred and perceived levels of involvement in their treatment decisions.</p><p><strong>Expert opinion: </strong>Patient engagement in treatment decisions is particularly important for older patients with AML. Understanding patients' perspectives and expectations for clinical and quality-of-life outcomes is essential to tailoring the most appropriate and effective treatment plan.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"595-604"},"PeriodicalIF":2.1,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144316289","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
MiR-106b-5p, MiR-200c-3p, and MiR-146a-5p expression as putative biomarkers for disease state in primary immune thrombocytopenia. MiR-106b-5p, MiR-200c-3p和MiR-146a-5p表达作为原发性免疫性血小板减少症疾病状态的推定生物标志物。
IF 2.1 4区 医学
Expert Review of Hematology Pub Date : 2025-08-01 Epub Date: 2025-06-25 DOI: 10.1080/17474086.2025.2522298
Reham Salah El Zaiat, Mai A H Abouelenin, Amany A Saleh, Mahmoud El-Hawy, Iman Aly Ahmedy, Manal Monir Mansour
{"title":"MiR-106b-5p, MiR-200c-3p, and MiR-146a-5p expression as putative biomarkers for disease state in primary immune thrombocytopenia.","authors":"Reham Salah El Zaiat, Mai A H Abouelenin, Amany A Saleh, Mahmoud El-Hawy, Iman Aly Ahmedy, Manal Monir Mansour","doi":"10.1080/17474086.2025.2522298","DOIUrl":"10.1080/17474086.2025.2522298","url":null,"abstract":"<p><strong>Background: </strong>The pathophysiology of primary immune thrombocytopenia (ITP) is complicated and multifactorial, including platelet antibody formation and T cell imbalance. Emerging evidence has revealed differential miRNA expression in autoimmune disorders, including ITP. Nevertheless, the role of miR-106b-5p, miR-200c-3p, and miR-146a-5p in ITP remains unclear. Herein, we explored the potential role of these miRNAs in pediatric ITP and examined how their plasma levels influenced response to therapy.</p><p><strong>Research design and methods: </strong>Three groups were recruited in this study: newly diagnosed ITP children (<i>n</i> = 25) in group I, chronic ITP children (<i>n</i> = 25) in group II, and normal controls (<i>n</i> = 25) in group III. Plasma levels of miR-106p-5p, miR-200c-3p, and miR-146a-5p were measured by polymerase chain reaction.</p><p><strong>Results: </strong>MiR-106b-5p and miR-200c-3p were upregulated, whereas miR-146a-5p was downregulated in newly diagnosed and chronic ITP versus controls. MiR-200c-3p and miR-146a-5p were much higher in chronic ITP than newly diagnosed ITP. Lower miR-106b-5p levels were associated with complete response.</p><p><strong>Conclusions: </strong>MiR-106b-5p and miR-200c-3p were elevated, while miR-146a-5p was suppressed in ITP versus controls. Reduced miR-106b-5p indicated a full response to therapy. These markers may be useful as diagnostic ITP biomarkers. Moreover, miR-106b-5p level can be used to monitor response to therapy and as a predictor for complete response.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"689-696"},"PeriodicalIF":2.1,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144316299","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
Safety and efficacy of marstacimab in patients with hemophilia A and B: a systematic review and meta-analysis. 马司他单在血友病A和B患者中的安全性和有效性:一项系统评价和荟萃分析。
IF 2.1 4区 医学
Expert Review of Hematology Pub Date : 2025-08-01 Epub Date: 2025-06-21 DOI: 10.1080/17474086.2025.2522296
Muhammed Edib Mokresh, Omar Alomari, Sena Mokresh, Cagla Sumeyye Caliskan, Merve Kabasakal Ilter
{"title":"Safety and efficacy of marstacimab in patients with hemophilia A and B: a systematic review and meta-analysis.","authors":"Muhammed Edib Mokresh, Omar Alomari, Sena Mokresh, Cagla Sumeyye Caliskan, Merve Kabasakal Ilter","doi":"10.1080/17474086.2025.2522296","DOIUrl":"10.1080/17474086.2025.2522296","url":null,"abstract":"<p><strong>Background: </strong>Hemophilia A and B are life-threatening congenital bleeding disorders traditionally managed with frequent factor replacement therapies, often complicated by breakthrough bleeds and inhibitor development. Marstacimab, a monoclonal antibody targeting TFPI, has emerged as a novel prophylactic treatment to reduce bleeding episodes in patients without inhibitors.</p><p><strong>Methods: </strong>A systematic review was conducted following PRISMA guidelines. A comprehensive literature search was performed across multiple databases. Meta-analysis was conducted using R, applying a random-effects model.</p><p><strong>Results: </strong>Nine manuscripts were included. Marstacimab significantly reduced the annualized bleeding rate (mean difference: -16.30; 95% CI: [-18.46, -14.15], <i>p</i> < 0.001) and showed a favorable safety profile with most adverse events being mild or moderate, and no thrombotic events reported. The use of a prefilled pen device further highlighted the therapeutic benefits and ease-of-use of Marstacimab.</p><p><strong>Conclusions: </strong>This meta-analysis reinforces the efficacy and safety of Marstacimab in reducing bleeding rates in severe hemophilia A and B. The findings support its role as a promising, transformative alternative to conventional factor replacement therapies.</p><p><strong>Registration: </strong>The study protocol for this systematic review was registered in the International Prospective Registry of Systematic Reviews (PROSPERO) database (www.crd.york.ac.uk/prospero/), and it was allocated the PROSPERO identification number CRD42024620215.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"649-660"},"PeriodicalIF":2.1,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144316301","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
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