Expert Review of Hematology最新文献

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 : 2025-03-17 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

Modulation of the endocannabinoid system in chronic conditions: a potential therapeutic intervention yet to be explored in sickle cell disease.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-03-04 DOI: 10.1080/17474086.2025.2471864

Lucas Bibiano de Oliveira Souza, Juliana Paiva Gouvea Sicoli, Sara Teresinha Olalla Saad, Bruno Deltreggia Benites

{"title":"Modulation of the endocannabinoid system in chronic conditions: a potential therapeutic intervention yet to be explored in sickle cell disease.","authors":"Lucas Bibiano de Oliveira Souza, Juliana Paiva Gouvea Sicoli, Sara Teresinha Olalla Saad, Bruno Deltreggia Benites","doi":"10.1080/17474086.2025.2471864","DOIUrl":"10.1080/17474086.2025.2471864","url":null,"abstract":"Introduction: Individuals living with Sickle Cell Disease (SCD) are subject to numerous chronic complications, including disabling chronic pain, often dependent on opioids and with important repercussions on the quality of life. The use of Medicinal Cannabis in this scenario may be a promising strategy for mitigating this impact.Areas covered: This work compiles current knowledge regarding the endocannabinoid system in humans and the role of this system in various organic functions. Articles were retrieved through a comprehensive search of the PubMed NCBI database, covering relevant studies up to 2024. These data bring important speculations on the potential role of the use of medicinal cannabis in modulating SCD chronic complications, and the preliminary results of clinical trials carried out in this condition are discussed.Expert opinion: The search for understanding the role of cannabis-derived products in the management of chronic complications of sickle cell disease could add resources to the serious challenge of dealing with the multiple aspects of the disease faced by patients. They range from the management of chronic pain itself to the risks of opioid dependence, in addition to other difficult scenarios, such as leg ulcers and chronic inflammation and its consequences.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-10"},"PeriodicalIF":2.3,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143482579","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 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 : 2025-02-27 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":"1-7"},"PeriodicalIF":2.3,"publicationDate":"2025-02-27","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

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 : 2025-02-26 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":"1-13"},"PeriodicalIF":2.3,"publicationDate":"2025-02-26","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

The influence of lymphoid enhancer binding factor-1 expression on the outcome of adult acute promyelocytic leukemia patients.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-02-23 DOI: 10.1080/17474086.2025.2467870

Thoraya M Abdelhamid, Yasmine Y Hassaneen, Mohamed Ghareeb, Eman O Rasekh

{"title":"The influence of lymphoid enhancer binding factor-1 expression on the outcome of adult acute promyelocytic leukemia patients.","authors":"Thoraya M Abdelhamid, Yasmine Y Hassaneen, Mohamed Ghareeb, Eman O Rasekh","doi":"10.1080/17474086.2025.2467870","DOIUrl":"10.1080/17474086.2025.2467870","url":null,"abstract":"Background: Acute promyelocytic leukemia (APL) is considered one of the greatest success stories in cancer therapy. However, early deaths remain the leading cause of treatment failure. This study aimed to analyze LEF1 expression in adult APL patients to evaluate its impact on survival outcomes, particularly early deaths.Research design and methods: LEF1 expression was analyzed by RT-qPCR in 78 denovo adult APL samples and 20 bone marrow samples from healthy matched donors as a control group. The cutoff for LEF1 fold change was set at 0.2250 using the receiver operating characteristic curve.Results: LEF1 expression was down regulated in APL patients as compared to the control group with statistically significant difference between the two groups (p < 0.001). The incidence of early deaths was higher in the low-LEF1 expressers than in high expressers (p = 0.018). LEF1 was determined to be an independent factor affecting early deaths. The high-risk patient group with low LEF1 expression had the worst overall survival.Conclusions: This study supports the potential of LEF1 to be a prognostic parameter in APL and a predictor of early deaths. Incorporating LEF1 into risk stratification could help to minimize early deaths. Future studies should explore combined risk factor analyses for improved prognosis in APL patients.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-9"},"PeriodicalIF":2.3,"publicationDate":"2025-02-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143440496","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 past, the present and the future of immune checkpoints inhibitors in multiple myeloma.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-02-23 DOI: 10.1080/17474086.2025.2469720

Valeria Tomarchio, Anna Crescenzi, Mariantonietta Tafuri, Martina Verri, Monica Di Cecca, Luigi Rigacci, Ombretta Annibali

{"title":"The past, the present and the future of immune checkpoints inhibitors in multiple myeloma.","authors":"Valeria Tomarchio, Anna Crescenzi, Mariantonietta Tafuri, Martina Verri, Monica Di Cecca, Luigi Rigacci, Ombretta Annibali","doi":"10.1080/17474086.2025.2469720","DOIUrl":"https://doi.org/10.1080/17474086.2025.2469720","url":null,"abstract":"Introduction: Myeloma genesis is a very complex mechanism in which the interaction between plasma cells and microenvironments with immune cells, cytokines and chemokines have a central role. In the last years, the improved knowledge of immune checkpoint models led to the development of new drugs (anti-PD1/PD-L1 axis or anti-TIGIT) that now have a crucial role in the treatment of many hematological malignancies.Areas covered: In this review, the current significant literature was discussed. In the past, initial trials combining immune checkpoint inhibitors (ICIs) with immunomodulatory drugs or proteasome inhibitors demonstrated suboptimal results in terms of efficacy and safety. On the other hand, recent trials based on the combination of ICIs with immunotherapies, such as CAR-T cells or bispecific antibodies, are a particularly promising area of investigation.Expert opinion: Our idea after the evaluation of scientific literature is that despite the past, ICIs may represent a promising therapeutic approach for myeloma, particularly when combined with CAR-T cells or bispecific antibodies. By targeting immune evasion mechanisms, ICIs may enhance the efficacy of these treatments and provide new hope for patients with resistant disease. Future research will be crucial to further elucidate their optimal use in myeloma and to develop personalized treatment strategies.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-02-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143482581","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

Problems faced by people with hemophilia aged 18-35 years in social life: a qualitative study.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-02-21 DOI: 10.1080/17474086.2025.2468386

Selin Davun, Zeynep Meva Altaş, Mahmut Batman, Melda Karavuş, Mehmet Mert Baykara, Beyza Doğan, Nabila Salehin, Sarp Kayauman, Ahmet Celal Demirel, Seyhan Hıdıroğlu

{"title":"Problems faced by people with hemophilia aged 18-35 years in social life: a qualitative study.","authors":"Selin Davun, Zeynep Meva Altaş, Mahmut Batman, Melda Karavuş, Mehmet Mert Baykara, Beyza Doğan, Nabila Salehin, Sarp Kayauman, Ahmet Celal Demirel, Seyhan Hıdıroğlu","doi":"10.1080/17474086.2025.2468386","DOIUrl":"10.1080/17474086.2025.2468386","url":null,"abstract":"Background: The aim of this qualitative study is to determine and evaluate the problems faced by people with hemophilia aged 18-35 years in their social lives.Research design and methods: In this qualitative type research, 13 individuals aged 18-35 years with hemophilia were reached with snowball sampling and online interviews with them. Participants were asked about their sociodemographic characteristics and the problems they encountered in various fields related to hemophilia using a semi-structured question guide with open-ended questions. Thematic content analysis was carried out.Results: Most participants were diagnosed with hemophilia in early childhood and have maintained knowledge of the condition since then. Hemophilia-related complications often resulted in significant educational delays, with two participants unable to complete formal schooling. Experiences of stigmatization in school were commonly reported. Some families faced migration or job changes due to the financial burden of hemophilia. While the majority viewed current healthcare services in Türkiye as sufficient, access issues in rural areas were noted. Nearly all participants belonged to a hemophilia-related non-governmental organization, which they felt had greatly enhanced their quality of life.Conclusions: To improve the social well-being of people with hemophilia, awareness campaigns to reduce stigma and foster a more supportive social environment are needed.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-8"},"PeriodicalIF":2.3,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143440494","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

Effect of erythropoiesis-stimulating agents on malignant neoplasms: FAERS database and Mendelian randomization.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-02-20 DOI: 10.1080/17474086.2025.2468400

Cuilv Liang, Qiaohong Wang, Peihong Wang, Yin Zhang

{"title":"Effect of erythropoiesis-stimulating agents on malignant neoplasms: FAERS database and Mendelian randomization.","authors":"Cuilv Liang, Qiaohong Wang, Peihong Wang, Yin Zhang","doi":"10.1080/17474086.2025.2468400","DOIUrl":"10.1080/17474086.2025.2468400","url":null,"abstract":"Background: The relationship between erythropoiesis-stimulating agents (ESAs) and malignant neoplasms (MNs) has been controversial in previous studies. Our study aimed to explore the correlation between ESAs and MNs.Research design and methods: Drug-target Mendelian randomization (MR) analyses were conducted to evaluate the causal associations of ESAs for 12 classifications of MNs. Meanwhile, a pharmacovigilance study was performed by extracting adverse events (AEs) from the FDA Adverse Event Reporting System (FAERS) database to validate and complement our findings. MR analysis revealed negative association of ESAs with MN of ovary (p = 0.047), liposarcoma (p = 0.001), small cell lung cancer (p = 0.017), colorectal cancer (p = 0.004), and brain meningioma (p = 0.004) and revealed positive association of ESAs with MN of bladder (p = 0.001), eye and adnexa (p = 0.012), heart, mediastinum, and pleura (p = 0.032), lip (p = 0.041), larynx (p = 0.015), non-small cell lung cancer (p = 0.009), and malignant melanoma (p = 0.001). Positive signals were found in MN of hematological system, digestive organs, central nervous system, eye and adnexa, head and neck cancer, lung cancer, and mucinous and mucinous cystic tumor in FAERS database (all reporting odds ratio and proportional reporting ratio >1).Conclusion: ESAs were causally correlated with many types of MNs. The use of ESAs in these tumors needs more attention.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-9"},"PeriodicalIF":2.3,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143432042","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

Navigating the gap between guidelines and practical challenges in selecting first-line therapy for chronic lymphocytic leukemia.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-02-20 DOI: 10.1080/17474086.2025.2469719

Stefano Molica

{"title":"Navigating the gap between guidelines and practical challenges in selecting first-line therapy for chronic lymphocytic leukemia.","authors":"Stefano Molica","doi":"10.1080/17474086.2025.2469719","DOIUrl":"https://doi.org/10.1080/17474086.2025.2469719","url":null,"abstract":"Introduction: Chronic lymphocytic leukemia (CLL) management has shifted from chemotherapy to targeted therapies like BTK and BCL-2 inhibitors, significantly improving patients' survival and quality of life. Current treatment guidelines often fail to fully address the real-world challenges of patient preferences, comorbidities, and logistical constraints. Consequently, a more personalized approach is essential to enhance decision-making and optimize treatment outcomes.Areas covered: This report delves into the challenges associated with frontline CLL therapy, emphasizing BTK inhibitors and venetoclax-based regimens. It examines patient stratification based on genetic markers, such as TP53 mutations and/or del(17p), the use of geriatric assessments for older patients, and the influence of comorbidities like cardiovascular disease and renal dysfunction. Furthermore, it highlights the need for a practical decision-making framework that integrates patient-specific considerations and addresses the limitations of existing treatment guidelines.Expert opinion: The proposed framework emphasizes a patient-centered approach that integrates clinical, genetic, and logistical factors to guide CLL treatment decisions. By addressing real-world challenges such as patient preferences for all-oral regimens and quality-of-life considerations, this approach aims to deliver truly personalized care. Future updates to CLL treatment guidelines should prioritize models that align with the unique needs and priorities of CLL patients.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-6"},"PeriodicalIF":2.3,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143467462","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 : 2025-02-17 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":"1-8"},"PeriodicalIF":2.3,"publicationDate":"2025-02-17","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