Expert Review of Hematology最新文献_第4页

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 : 2025-01-01 Epub 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":"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":"195-200"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","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

Proximal complement inhibitors in paroxysmal nocturnal hemoglobinuria: an abundance of options in a rare disease. 阵发性夜间血红蛋白尿的近端补体抑制剂：一种罕见疾病的丰富选择。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2025-01-06 DOI: 10.1080/17474086.2025.2449864

Taylor S White, Justin R Arnall, Paul Christopher Parish, Jamie Tolerico, Thuy Tran, Donald C Moore

引用次数: 0

Key features of the underlying pathophysiology of Transfusion-related acute lung injury. 输血相关急性肺损伤的基本病理生理特征。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-01-01 Epub Date: 2024-12-03 DOI: 10.1080/17474086.2024.2436972

Rick Kapur

{"title":"Key features of the underlying pathophysiology of Transfusion-related acute lung injury.","authors":"Rick Kapur","doi":"10.1080/17474086.2024.2436972","DOIUrl":"10.1080/17474086.2024.2436972","url":null,"abstract":"Introduction: Transfusion-related acute lung injury (TRALI) remains a leading cause of blood transfusion associated mortality, particularly in the intensive care unit. TRALI is underrecognized, underreported and lacks specific biomarkers and clinical therapies.Areas covered: In this review, the focus will be on the key pathophysiological features of TRALI. This will include the latest insights into the critical importance of complement (in contrast to Fcγ-receptors; FcγRs) as a driver of TRALI, and the role of recipient immune cells such as neutrophils and macrophages, and also the contribution of the pulmonary endothelium.Expert opinion: Increased efforts are needed to stimulate active reporting of TRALI cases. More research into the immuno-cellular pathophysiology of TRALI is required, including the role of the pulmonary endothelium. Heterogeneity in the underlying clinical condition and the different transfusion triggers should be taken into consideration. This will aid in the search for novel biomarkers and therapeutic modalities. At the moment, the most promising potential therapeutic strategies appear to be administration of interleukin (IL)-10, inhibition of complement activation and blockade of Osteopontin (OPN). Follow-up investigations are, however, highly warranted which should pave the way for multicenter international clinical trials, in order to battle the mortality due to TRALI.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"57-64"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142750160","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 : 2025-01-01 Epub 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":"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":"201-214"},"PeriodicalIF":2.3,"publicationDate":"2025-01-01","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

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

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

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