Expert Review of Hematology最新文献_第3页

A retrospective single-center study on the effectiveness and tolerability of emicizumab in patients with hemophilia a with and without inhibitors. 一项关于埃米珠单抗对使用或未使用抑制剂的 a 型血友病患者的有效性和耐受性的回顾性单中心研究。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2025-02-27 DOI: 10.1080/17474086.2025.2471862

Sheikh Bilal Ahmad, Asifa Amin, Malik Rohi, Sarwat Muzaffar, Sheikh Fahad

{"title":"A retrospective single-center study on the effectiveness and tolerability of emicizumab in patients with hemophilia a with and without inhibitors.","authors":"Sheikh Bilal Ahmad, Asifa Amin, Malik Rohi, Sarwat Muzaffar, Sheikh Fahad","doi":"10.1080/17474086.2025.2471862","DOIUrl":"10.1080/17474086.2025.2471862","url":null,"abstract":"Background: Hemophilia A (HA) is an X-linked disorder due to deficient/defective coagulation factor VIII (FVIII), which causes excessive bleeding either traumatic or spontaneous. Frequent FVIII replacement is complicated by the development of inhibitors. The bispecific monoclonal antibody emicizumab, offers a novel approach by bridging activated factors IXa and X for effective hemostasis.Research design and methods: This retrospective study analyzed emicizumab treatment outcomes in 73 patients with severe HA at a tertiary care center in Jammu and Kashmir, India. Data included demographic profiles, bleeding rates, joint health scores, and adverse events (AEs).Results: Emicizumab demonstrated significant efficacy and safety in the study with median patient age of 14 years, with 17/73 patients having FVIII inhibitors. It resulted in a 100% reduction in annualized bleeding rate (ABR) for patients with inhibitors (from 20.7 to 0.0) and a 99.5% reduction for those without inhibitors (from 7.7 to 0.04). All inhibitor-negative patients showed ABR improvements and significant reduction in joint bleeds and resolution of all target joints. Tolerability was favorable with only one patient reporting a non-significant AE.Conclusions: Emicizumab prophylaxis effectively reduces ABRs and enhances joint health in severe HA patients, irrespective of inhibitor presence, providing a convenient, well-tolerated alternative to FVIII therapy.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"275-281"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143515094","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

CD8+CD25+ Tregs as a predictor of glucocorticoid sensitivity in patients with immune thrombocytopenia.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2025-02-17 DOI: 10.1080/17474086.2025.2467386

Sara El-Sayed Abd El-Ghani, Lina Ayman Mahmoud Mohamed, Mohamed Roshdi El-Masry, Hala M Abdelhamid, Mohamed Fateen, Marwa Salah Mohamed

{"title":"CD8+CD25+ Tregs as a predictor of glucocorticoid sensitivity in patients with immune thrombocytopenia.","authors":"Sara El-Sayed Abd El-Ghani, Lina Ayman Mahmoud Mohamed, Mohamed Roshdi El-Masry, Hala M Abdelhamid, Mohamed Fateen, Marwa Salah Mohamed","doi":"10.1080/17474086.2025.2467386","DOIUrl":"10.1080/17474086.2025.2467386","url":null,"abstract":"Background: ITP is an immune-mediated disorder showing increased platelet destruction and reduced platelet production. Glucocorticoids are the first-line treatment, but 10-20% of patients are insensitive to them. We should avoid the irrational use of steroids for treating ITP. No markers exist to predict treatment responses in ITP. Recently, a few studies showed abnormal ratio and function of Tregs in ITP patients and that the levels of Tregs change after glucocorticoid treatment. We aimed to study the value of CD8+ CD25+ Tregs in predicting glucocorticoid sensitivity in newly diagnosed ITP patients.Research design and methods: This cohort (longitudinal) study included 60 ITP patients. The baseline platelet counts and levels of CD8+ CD25+ Tregs were measured at diagnosis. The response to steroids was evaluated after 2 weeks of treatment, then after 3 months and 6 months.Results: The ROC curve was plotted, and the p value was not significant; therefore, the optimal critical value, specificity and sensitivity could not be determined.Conclusions: This study couldn't confirm the value of CD8+CD25+ Tregs in predicting glucocorticoid sensitivity in ITP, but there was a significant positive correlation between the CD8+CD25+ Tregs percentage and the steroids' response after 2 weeks. Therefore, further studies are recommended.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"257-264"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143406523","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

Pegcetacoplan: the first and only C3-targeted therapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria. Pegcetacoplan：治疗成人阵发性夜间血红蛋白尿症的首个也是唯一的 C3 靶向疗法。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2024-12-16 DOI: 10.1080/17474086.2024.2440101

Tze Wei Chan, Hein Than, Tertius Tuy, Yeow Tee Goh

{"title":"Pegcetacoplan: the first and only C3-targeted therapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria.","authors":"Tze Wei Chan, Hein Than, Tertius Tuy, Yeow Tee Goh","doi":"10.1080/17474086.2024.2440101","DOIUrl":"10.1080/17474086.2024.2440101","url":null,"abstract":"Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of hematopoietic stem cells, characterized by somatic mutations of the Phosphatidylinositol Glycan Class A Gene, resulting in increased hemolysis. The advent of complement inhibitors has since changed the way clinicians approach treating PNH. Pegcetacoplan is a C3 inhibitor that has shown promise in this field and improved outcomes for patients who have been diagnosed with PNH.Areas covered: This review article will aim to examine the pathophysiology of PNH and the current treatments available, with a focus on pegcetacoplan. It will focus on the pharmacodynamics, pharmocokinetics and evidence in the use of pegcetacoplan in PNH. Electronic sources including PubMed, MEDLINE, were utilized with studies in the last 5 years prioritized, especially the phase 3 Prince and Pegasus studies.Expert opinion: The results from phase 3 studies for pegcetacoplan have been promising, showing good efficacy and improvements in patients' conditions. More research is required to evaluate the use of pegcetacoplan, especially in combination with existing treatment in patients who are having suboptimal results. Nonetheless, with more results on the way and new agents to treat PNH in the vicinity, this remains a very exciting time for both clinicians and patients.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"11-20"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142799950","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

Extracellular vesicles secreted by leukemic cells as mediators of dysregulated hematopoiesis: acute myeloid leukemia as a case in point.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2025-02-26 DOI: 10.1080/17474086.2025.2471860

Vishakha Kasherwal, Vaijayanti Kale, Anuradha Vaidya

{"title":"Extracellular vesicles secreted by leukemic cells as mediators of dysregulated hematopoiesis: acute myeloid leukemia as a case in point.","authors":"Vishakha Kasherwal, Vaijayanti Kale, Anuradha Vaidya","doi":"10.1080/17474086.2025.2471860","DOIUrl":"10.1080/17474086.2025.2471860","url":null,"abstract":"Introduction: Acute myeloid leukemia (AML) cells exhibit a profound capacity for resistance to conventional chemotherapeutic agents, posing a substantial challenge to existing therapeutic paradigms. Interestingly, this happens in the face of a luxuriant proliferation of leukemic blasts in the peripheral blood. This paradox of concurrent proliferative activity and cellular quiescence underscores a complex biological phenomenon that is intricately mediated by AML-derived Extracellular vesicles (EVs).Areas covered: An extensive literature review search was done on PubMed/Scopus/Web of Sciences databases to identify studies published between 2013 and 2024 elucidating and demonstrating the effect of AML-derived EVs, Microvesicles (MVs) and Exosomes (Exos) in regulating the normal and dysregulated bone marrow (BM) niche.Expert opinion: The review delves into understanding the molecular mechanisms underlying the dual behavior of AML cells - proliferation and quiescence, with a special focus on the role of the EVs and their subtypes viz. Exos and MVs in establishing a discrete BM microenvironment that is subversive to chemotherapy. It offers a novel perspective on the intricate interplay between the leukemic cells and their microenvironment, with implications for therapeutic interventions targeting AML persistence and drug resistance.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"225-237"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143499825","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

Understanding and overcoming resistance to tyrosine kinase inhibitors (TKIs) in Chronic myeloid leukemia (CML). 了解和克服慢性髓性白血病（CML）对酪氨酸激酶抑制剂（TKIs）的耐药性。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2024-12-15 DOI: 10.1080/17474086.2024.2440776

Alessandro Laganà, Emilia Scalzulli, Maria Laura Bisegna, Claudia Ielo, Maurizio Martelli, Massimo Breccia

{"title":"Understanding and overcoming resistance to tyrosine kinase inhibitors (TKIs) in Chronic myeloid leukemia (CML).","authors":"Alessandro Laganà, Emilia Scalzulli, Maria Laura Bisegna, Claudia Ielo, Maurizio Martelli, Massimo Breccia","doi":"10.1080/17474086.2024.2440776","DOIUrl":"10.1080/17474086.2024.2440776","url":null,"abstract":"Introduction: Chronic myeloid leukemia (CML) represents one of the first neoplasms whose molecular pathogenesis was successfully unraveled, with tyrosine kinase inhibitors (TKIs) representing one of the first-targeted therapies. TKIs have revolutionized long-term outcomes of CML patients and their life expectancy. Nonetheless, a minority of patients will develop TKI resistance due to a complex and multifactorial process that ultimately leads to the emergence of an unresponsive cancer clone. Overcoming TKI resistance is considered one of the major challenges in CML management.Areas covered: In this review, the main findings extrapolated from published research, guidelines, and clinical trials regarding TKI resistance (published before October 2024) are discussed. Data have been obtained through broad research on Medline, Embase, Pubmed, and archives from EHA and ASH congresses.Expert opinion: Nowadays, asciminib and ponatinib have expanded the therapeutic arsenal for resistant-CML management and allogenic transplant still represents an important alternative in the context of multiple TKI failures. Off-label use of TKIs combination therapies, although theoretically appealing, lacks robust clinical evidence and regulatory approval. Looking ahead, the introduction of novel technologies such as digital PCR (dPCR) and next generation sequencing (NGS) holds great potential to revolutionize the management of TKI-resistant CML cases.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"65-79"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142794456","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

Spontaneous regression in mature T-cell non-Hodgkin lymphoma. 成熟t细胞非霍奇金淋巴瘤的自发消退。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2024-12-07 DOI: 10.1080/17474086.2024.2439469

Akihiro Ohmoto, Shigeo Fuji

{"title":"Spontaneous regression in mature T-cell non-Hodgkin lymphoma.","authors":"Akihiro Ohmoto, Shigeo Fuji","doi":"10.1080/17474086.2024.2439469","DOIUrl":"10.1080/17474086.2024.2439469","url":null,"abstract":"Introduction: Spontaneous regression (SR) is observed in some patients with mature T-cell non-Hodgkin lymphoma (MTCL), including adult T-cell leukemia/lymphoma (ATL), although the incidence is rare.Area covered: We extracted 31 cases with MTCL who experienced SR based on a literature search and summarized the patient characteristics and clinical outcomes.Expert opinion: MTCL with SR included various subtypes, the most common being ATL (n = 17). Five of 24 cases (21%) maintained SR for more than 5 years, and the median duration of SR was 2 years. Sixteen of 31 cases (52%) experienced tumor relapse after SR. Two retrospective studies including ATL cases showed SR rates of 18% and 4%, respectively. Representative triggers are infection and surgical biopsies, and possible mechanisms include immunological mechanisms such as cross-reactivity of virus-specific T cells with tumor antigens. The diagnostic criteria for SR are not standardized among reports, and the clinical outcomes are not fully described. Practically, observation is the only accepted strategy after SR was achieved. In the era of molecular targeted therapy or immunotherapy, new strategies including maintenance therapy after SR could be discussed, although clinical data are lacking.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"47-55"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791485","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

A plain language summary on indirectly comparing bleeding after valoctocogene roxaparvovec gene therapy to bleeding with emicizumab prophylaxis.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2025-03-17 DOI: 10.1080/17474086.2025.2467861

Jan Astermark, Tyler W Buckner, Laurent Frenzel, Anthony J Hatswell, David Hinds, Sandra Santos, Robert Klamroth, Tobias Becker, Deon York

引用次数: 0

NSAIDS/COXIBS for the treatment of musculoskeletal pain secondary to hemophilic arthropathy. 非甾体抗炎药/COXIBS治疗血友病关节病继发的肌肉骨骼疼痛。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2024-12-04 DOI: 10.1080/17474086.2024.2438237

E Carlos Rodriguez-Merchan, Hortensia De la Corte-Rodriguez

{"title":"NSAIDS/COXIBS for the treatment of musculoskeletal pain secondary to hemophilic arthropathy.","authors":"E Carlos Rodriguez-Merchan, Hortensia De la Corte-Rodriguez","doi":"10.1080/17474086.2024.2438237","DOIUrl":"10.1080/17474086.2024.2438237","url":null,"abstract":"Introduction: Cyclooxygenase-2 (COX-2) inhibitors (COXIBS) are considered a suitable option for the treatment of hemophilic arthropathy.Areas covered: The role of traditional non-steroidal anti-inflammatory drugs; (NSAIDS) and COXIBS in people with hemophilia (PWH) and in the non-hemophiliac population has been reviewed in order to know which of them is more advisable in PWH and whether they should be used as the first or second therapeutic step for the treatment of musculoskeletal pain (since there is a discrepancy between what is advised by the WFH and the WHO).Expert opinion: For the treatment of chronic musculoskeletal pain related to hemophilic arthropathy, it is reasonable to use as a first step a combination of oral paracetamol (650 mg per every 6 h) or metamizole (575 mg per every 6 h), one of the COXIBS (e.g. celecoxib 200 mg per once a day) and a proton pump inhibitor (e.g. omeprazole 20 mg per once a day). The possible side effects of COXIBS should never be forgotten. For the treatment of hemophilic arthropathy pain, the risk/benefit ratio of COXIBS should be carefully assessed on an individual basis, although they are more advisable than traditional NSAIDS.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"33-38"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142767796","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

Radioimmunotherapy of acute myeloid leukemia: a critical assessment of its prospects and limitations. 急性髓性白血病的放射免疫治疗：对其前景和局限性的关键评估。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2025-01-10 DOI: 10.1080/17474086.2025.2449863

Donald Bunjes

引用次数: 0

Novel approaches for preventing COVID-19 infection in immunocompromised patients with hematologic malignancies. 预防血液系统恶性肿瘤免疫功能低下患者COVID-19感染的新方法

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2024-12-03 DOI: 10.1080/17474086.2024.2436973

Shmuel Shoham, Daniele Focosi, Massimo Franchini, Alaa Atamna

{"title":"Novel approaches for preventing COVID-19 infection in immunocompromised patients with hematologic malignancies.","authors":"Shmuel Shoham, Daniele Focosi, Massimo Franchini, Alaa Atamna","doi":"10.1080/17474086.2024.2436973","DOIUrl":"10.1080/17474086.2024.2436973","url":null,"abstract":"Introduction: COVID-19 is a continuing challenge for immunocompromised patients with hematological malignancies. Such patients are at increased risk for complications, including hospitalization, respiratory failure, delayed anti-cancer therapies, and even death. In addition to non-pharmacologic interventions, the main strategies for prevention in such patients are vaccination and pre-exposure prophylaxis.Areas covered: In this narrative review, which relied on a review of the PubMed and bioRxiv databases (starting 1 November 2019), we summarize the epidemiology of COVID-19 and vaccine responses in patients with hematological malignancies and the use of antiviral agents as prophylaxis. A limitation to vaccination is suboptimal immune responses in immunocompromised patients, particularly those with abnormalities in lymphocyte count and function. A limitation to prophylaxis, which has only been proven effective for antiviral monoclonal antibodies (mAbs), is the emergence of resistant strains in the general population.Expert opinion: For immunocompromised patients with hematological malignancies, we recommend vaccinations as guided by evolving US Centers for Disease Control and Prevention (CDC) recommendations, consideration of pre-exposure prophylaxis with antiviral mAbs, providing that they are effective against circulating viral strains, and rapid diagnostic testing linked to early therapy for the prevention of severe complications of COVID-19 in those who have broken through the prevention strategies.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"39-46"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142750233","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