Expert Review of Hematology最新文献_第2页

Hairy cell leukemia (HCL) and HCL-like disorders: present, emergent treatment options and future directions. 毛细胞白血病（HCL）和 HCL 类疾病：现状、紧急治疗方案和未来方向。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-12-01 Epub Date: 2024-11-14 DOI: 10.1080/17474086.2024.2427660

Xavier Troussard

{"title":"Hairy cell leukemia (HCL) and HCL-like disorders: present, emergent treatment options and future directions.","authors":"Xavier Troussard","doi":"10.1080/17474086.2024.2427660","DOIUrl":"10.1080/17474086.2024.2427660","url":null,"abstract":"Introduction: Hairy cell leukemia accounts for less than 2% of leukemias. The hairy cells express CD11c, CD25, CD103, and CD123 markers. The BRAFV600E mutation was detected in 95% of HCL cases. Patients achieve high complete response rate with purine analogues with or without rituximab, but relapses are inevitable. HCL-like disorders including HCL variant, splenic diffuse red pulp lymphoma, and splenic marginal zone lymphoma are BRAFV600E negative. The CD25 expression is negative. The absence of BRAFV600E mutation in HCL variant contrasts with the presence of mitogen-activated protein kinase kinase 1 (MAP2K1) mutations in 50% of cases.Areas covered: We investigated the criteria used to distinguish HCL from HCL-like disorders. Recent discoveries in molecular biology have enabled the introduction of several new drugs in HCL patients. We explore the investigational agents: inhibitors of BRAF, MEK, and Bruton tyrosine kinase and potential future strategies we will use in the future in patients with relapsed/refractory HCL. We also discuss the clinical trials in progress.Expert opinion: The association of Cladribine (CDA) with rituximab (R) is the standard first-line treatment in fit HCL and HCL variant patients. BRAF and BTK inhibitors are options in relapsed/refractory HCL patients. The optimal treatment sequences remain to be determined.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"907-915"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142616816","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 oral health promotion on the quality of life of children with bleeding disorders: fighting misconceptions. 促进口腔健康对出血性疾病儿童生活质量的影响：消除误解。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-12-01 Epub Date: 2024-11-27 DOI: 10.1080/17474086.2024.2432354

Nayera H K El Sherif, Mariem O M Wassel, Salwa Mostafa, Fatma R R Abd El Maged, Sally E Nathan, Dina Hamdy, Fatma S E Ebeid

{"title":"The impact of oral health promotion on the quality of life of children with bleeding disorders: fighting misconceptions.","authors":"Nayera H K El Sherif, Mariem O M Wassel, Salwa Mostafa, Fatma R R Abd El Maged, Sally E Nathan, Dina Hamdy, Fatma S E Ebeid","doi":"10.1080/17474086.2024.2432354","DOIUrl":"10.1080/17474086.2024.2432354","url":null,"abstract":"Background: Understanding the disease-specific risks and patient-related barriers of children with bleeding disorders is necessary for primary oral health promotion. Our goal was to assess the oral health status and the impact of oral health promotion among patients with bleeding disorders.Research design and methods: At baseline, 70 patients with inherited and acquired bleeding disorders had a complete intraoral examination, completed the oral health-related quality of life (OHRQoL) questionnaires, and an oral health education was given. After 6 months, the effectiveness of the oral hygiene promotion was evaluated clinically and through the OHRQoL questionnaires.Results: Our cohort included 33 patients with chronic immune thrombocytopenia (cITP), 27 hemophilia A patients, and 10 with inherited thrombasthenia. Forty patients (57.1%) had dental caries, 90.0% showed fair oral hygiene status with variable degrees of gingivitis. The baseline self-image score was significantly inferior among patients with inherited bleeding disorders, while the psychological domain for family was greatly affected among cITP patients. After 6 months, there was a significant reduction in the oral debris, the modified gingival indexes, the percentages of cases with oral bleeding, and a significantly improved mean OHRQoL total score.Conclusions: After the oral health education, the OHRQoL scores had significantly improved, and oral hygiene status were acceptable among patients with bleeding disorders.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"967-975"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142738830","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

Late effects of hemopoietic stem cell transplant for sickle cell disease: monitoring and management. 造血干细胞移植治疗镰状细胞病的后期影响：监测与管理。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-12-01 Epub Date: 2024-11-05 DOI: 10.1080/17474086.2024.2423368

Marti Goldenberg, Sophie Lanzkron, Lydia H Pecker

引用次数: 0

BCL-2 inhibition in acute myeloid leukemia: resistance and combinations. 急性髓性白血病中的 BCL-2 抑制剂：抗药性与联合用药。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-12-01 Epub Date: 2024-11-18 DOI: 10.1080/17474086.2024.2429604

Qi Zhang Tatarata, Zhe Wang, Marina Konopleva

{"title":"BCL-2 inhibition in acute myeloid leukemia: resistance and combinations.","authors":"Qi Zhang Tatarata, Zhe Wang, Marina Konopleva","doi":"10.1080/17474086.2024.2429604","DOIUrl":"10.1080/17474086.2024.2429604","url":null,"abstract":"Introduction: The introduction of venetoclax has revolutionized the treatment landscape of acute myeloid leukemia, offering new therapeutic opportunities. However, the clinical response to venetoclax varies significantly between patients, with many experiencing limited duration of response.Areas covered: Identified resistance mechanisms include both intrinsic and acquired resistance to VEN. The former is associated with cell lineage and differentiation state. The latter includes dependency on alternative BCL-2 family anti-apoptotic protein(s) mediated by genetic, epigenetic, or post-translational mechanisms, mitochondrial and metabolic involvement, as well as microenvironment. Understanding these mechanisms is crucial for optimizing venetoclax-based therapies and enhancing treatment outcomes for patients with acute myeloid leukemia. This review aims to elucidate the primary mechanisms underlying resistance to venetoclax and explore current therapeutic strategies to overcome this challenge.Expert opinion: In patients with venetoclax resistance, alternative options include targeted combination therapies tailored to individual cases based on cytogenetics and prior treatments. Many of these therapies require further clinical investigation to validate their safety and efficacy.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"935-946"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142647291","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

CRISPR-based gene therapy for the induction of fetal hemoglobin in sickle cell disease. 基于 Crispr 的基因疗法诱导镰状细胞病中的胎儿血红蛋白。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-12-01 Epub Date: 2024-12-04 DOI: 10.1080/17474086.2024.2429605

Meghann McManus, Haydar Frangoul, Martin H Steinberg

{"title":"CRISPR-based gene therapy for the induction of fetal hemoglobin in sickle cell disease.","authors":"Meghann McManus, Haydar Frangoul, Martin H Steinberg","doi":"10.1080/17474086.2024.2429605","DOIUrl":"10.1080/17474086.2024.2429605","url":null,"abstract":"Introduction: Sickle cell disease is ameliorated and perhaps can be 'cured' if enough fetal hemoglobin is present in most erythrocytes. Hydroxyurea, which increases fetal hemoglobin levels, is widely available and effective, especially in children. Nevertheless, only cell-based gene therapy can achieve a 'curative' fetal hemoglobin threshold.Areas covered: We cover the path to modulating fetal hemoglobin gene expression and the use of CRISPR/Cas9 gene editing as a viable clinical modality for treating severe sickle cell disease relying on references obtained from PubMed. Mobilized autologous hematopoietic stem and progenitor cells are engineered with vectors that derepress genes that regulate fetal hemoglobin gene expression. Following myeloablative conditioning, gene-edited cells are reinfused, engrafted, and make large amounts of fetal hemoglobin. Within months, fetal hemoglobin forms more than 40% of the total hemoglobin and hemoglobin levels normalize; symptoms of sickle cell disease disappear.Expert opinion: Optimistically, these patients are 'cured,' but long term follow-up is needed. Although approved by regulatory agencies and highly efficacious, because of its technical imperatives and cost, this first gene editing therapeutic will be unavailable to most people with severe sickle cell disease. It is highly likely that improved methods of genomic editing will simplify gene therapy, reduce its costs, and lead to its wider applicability.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"957-966"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142616287","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

Ibrutinib and venetoclax combination therapy for mantle cell lymphoma: are two better than one? 伊布替尼和 venetoclax 联合疗法治疗套细胞淋巴瘤：两种疗法比一种疗法更好吗？

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-12-01 Epub Date: 2024-11-10 DOI: 10.1080/17474086.2024.2427663

Jian Li, Constatine S Tam

{"title":"Ibrutinib and venetoclax combination therapy for mantle cell lymphoma: are two better than one?","authors":"Jian Li, Constatine S Tam","doi":"10.1080/17474086.2024.2427663","DOIUrl":"10.1080/17474086.2024.2427663","url":null,"abstract":"Introduction: Mantle cell lymphoma (MCL) is a non-Hodgkin B-cell lymphoma typically regarded as incurable with standard chemotherapy. Ibrutinib has become an accepted second-line treatment for relapsed or refractory MCL. Although venetoclax has shown activity against the disease, these results have not been consistently seen in the post-ibrutinib era. Therefore, there is growing evidence that supports using ibrutinib and venetoclax together in patients with chronic lymphocytic leukemia.Areas covered: This article looks at the evidence for combining ibrutinib and venetoclax in treating relapsed or refractory MCL, particularly from the AIM and SYMPATICO studies. The phase II AIM study evaluated the ongoing combination of ibrutinib and venetoclax following a run-in period of ibrutinib. The phase III SYMPATICO study started both drugs without a run-in period and included a fixed two-year duration of venetoclax.Expert opinion: The combination of ibrutinib and venetoclax has shown effectiveness in patients with relapsed/refractory MCL, indicating potential for fixed-duration therapy. The emerging use of measurable residual disease and molecular data may help identify which patients are suitable for stopping treatment. As new information becomes available on ibrutinib in first-line settings and chimeric T-cell therapy, the optimal timing for combining ibrutinib and venetoclax may require further refinement.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"885-889"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142603894","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

Rivaroxaban plus aspirin after lower-extremity revascularization. 下肢血运重建术后利伐沙班加阿司匹林。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-12-01 Epub Date: 2024-11-29 DOI: 10.1080/17474086.2024.2432358

Graham R McClure, John Eikelboom

{"title":"Rivaroxaban plus aspirin after lower-extremity revascularization.","authors":"Graham R McClure, John Eikelboom","doi":"10.1080/17474086.2024.2432358","DOIUrl":"10.1080/17474086.2024.2432358","url":null,"abstract":"Introduction: Patients undergoing revascularization of the lower extremities have unacceptably high rates of major adverse cardiac and limb events despite the routine use of antiplatelet therapy. Optimization of antithrombotic therapy provides an opportunity to reduce this risk. Recent large, randomized trials have demonstrated substantial benefit from the combination of low-dose rivaroxaban and aspirin compared with aspirin alone. Despite this new evidence, uptake remains limited.Areas covered: This review will outline the drug profile of rivaroxaban, summarize the key efficacy and safety data for the combination of low-dose rivaroxaban and aspirin following lower extremity revascularization, and examine barriers to therapy uptake.Expert opinion: Combination of low-dose rivaroxaban and aspirin is the only antithrombotic regimen that has been shown to reduce both cardiac and limb events following peripheral revascularization while maintaining an acceptable bleeding profile. Single and dual antiplatelet therapy have limited randomized evidence for this indication, but are commonly used. An important contributor is the failure of major societal guidelines to incorporate this new evidence. Moving forward, there is an urgent need to update these guidelines. Further evaluation of the efficacy and safety of dual antiplatelet therapy will help to inform optimal antithrombotic therapy after lower extremity revascularization.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"877-884"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142667630","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

Global prophylaxis trends in hemophilia: a macroeconomic analysis and its association with world development indicators. 全球血友病预防趋势：宏观经济分析及其与世界发展指标的关联。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-12-01 Epub Date: 2024-11-18 DOI: 10.1080/17474086.2024.2429606

M Joseph John, Jeff Stonebraker, Aikaj Jindal, Ellia Tootoonchian, Glenn P Pierce, Emna Gouider, Arihant Jain, Donna Coffin

{"title":"Global prophylaxis trends in hemophilia: a macroeconomic analysis and its association with world development indicators.","authors":"M Joseph John, Jeff Stonebraker, Aikaj Jindal, Ellia Tootoonchian, Glenn P Pierce, Emna Gouider, Arihant Jain, Donna Coffin","doi":"10.1080/17474086.2024.2429606","DOIUrl":"10.1080/17474086.2024.2429606","url":null,"abstract":"Introduction: Prophylaxis is the recommended management strategy for all persons with hemophilia (PwH), yet its adoption is uneven worldwide.Areas covered: This analysis examines global disparities in hemophilia care, focusing on global prophylactic coverage and its correlation with the World Bank's world development indicators. It outlines the disproportionate consumption of clotting factors and non-factor concentrates in high-income countries compared to lower-income counterparts and the challenges of expanding prophylaxis coverage in under-resourced settings. The analysis integrates socioeconomic data with global health indicators to understand these disparities and advocates for increased distribution of treatment resources across all income levels, emphasizing the need for policy changes to improve hemophilia care worldwide. Studies addressing the prophylaxis perspectives in hemophilia were selected using PubMed and Google Scholar platforms (unlimited time frame). Articles were supplemented with WFH's annual surveys and guidelines, including the WFH Global Survey 2022, WFH Guidelines for the Management of Hemophilia 2020 and World Bank data.Expert opinion: Significant disparities in hemophilia care and factor usage exist between high-income and lower-income countries. Standardized, harmonized metrics for different types of factor consumption are critical to accurately assess and compare hemophilia care on an international basis.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"947-956"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142616667","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

Quantitative MRI evaluation of iron deposition in patients with transfusion-dependent thalassemia: clinical management insights.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-12-01 Epub Date: 2024-12-03 DOI: 10.1080/17474086.2024.2435353

Xiaojing Ning, Fei Peng, Siyu Tan, Cheng Tang, Chaotian Luo, Fangyan Xiao, Chen Zhao, Peng Peng

{"title":"Quantitative MRI evaluation of iron deposition in patients with transfusion-dependent thalassemia: clinical management insights.","authors":"Xiaojing Ning, Fei Peng, Siyu Tan, Cheng Tang, Chaotian Luo, Fangyan Xiao, Chen Zhao, Peng Peng","doi":"10.1080/17474086.2024.2435353","DOIUrl":"10.1080/17474086.2024.2435353","url":null,"abstract":"Background: In patients with thalassemia, different organs are affected differently by iron overload. Nevertheless, the reasons for this could be the same key transporters. This study investigated the iron deposition in different organs of transfusion-dependent thalassemia (TDT) patients and its correlation.Research design and methods: This cross-sectional study involved 54 TDT patients who underwent MRI T2* examinations of the heart, liver, pancreas, spleen, kidneys, and pituitary. The study analyzed the iron deposition in each organ and evaluated the correlation of iron deposition using Spearman's test.Results: Among the 54 patients with TDT, liver iron overload was found in 49/54 (90.7%) cases, pancreas iron overload in 43/54 (79.6%) cases, spleen iron overload in 18/26 (69.2%) patients, heart iron overload in 20/54 (37.0%) cases, and kidney iron overload in 8/54 (14.8%) patients. Most patients (66.7%) with iron overload in the liver but not in the heart exhibited spleen iron abnormalities. Pituitary T2* and pancreas T2* (r = 0.790), pituitary T2* and kidney T2* (r = 0.692), kidney T2* and pancreas T2* (r = 0.672) showed positive correlation (all p < 0.05).Conclusions: Patients with TDT exhibited significant organ-specific iron overload. These findings highlight the importance of routine MRI screening for monitoring and managing iron overload in patients with TDT. Pituitary, pancreas, and kidney may have similar iron-loading mechanisms.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"977-983"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142767797","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

Current and emerging therapies as potential treatment for people with von Willebrand disease. 作为治疗冯-威廉氏病患者的潜在方法的现有疗法和新兴疗法。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-12-01 Epub Date: 2024-11-18 DOI: 10.1080/17474086.2024.2429611

Emmanuel J Favaloro, Leonardo Pasalic, Jennifer Curnow

{"title":"Current and emerging therapies as potential treatment for people with von Willebrand disease.","authors":"Emmanuel J Favaloro, Leonardo Pasalic, Jennifer Curnow","doi":"10.1080/17474086.2024.2429611","DOIUrl":"10.1080/17474086.2024.2429611","url":null,"abstract":"Introduction: Von Willebrand disease (VWD) reflects the most common inherited bleeding disorder, arising from defects or deficiencies in the von Willebrand factor (VWF). VWD treatment mostly relies on the replacement of missing or defective VWF, but additional ('adjunct') therapies are useful in select patients/situations. Patients with VWD are often misdiagnosed and therefore non-optimally managed.Areas covered: We provide a narrative review, following relevant literature searches in PubMed related to the topic up to September 2024. After an overview of VWF, VWD, and current treatments, we explore the use of nonstandard or emerging therapies for VWD. For example, FVIII replacement or antibody-based FVIII bypassing strategies (e.g. emicizumab) may prove useful in some cases or in initial treatment of certain VWD patients, including those with type 2N or 3 VWD, or those with inhibitors. Additional emerging therapies may also be useful, including hemostasis rebalancing agents.Expert opinion: Just as hemophilia is experiencing a renaissance of treatment options, so too will the landscape of VWD treatment change over time. This will be fueled by the concept of personalized treatment, meaning potentially different treatments for different VWD patients, or for given patients according to treatment aims.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"917-933"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142616293","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