Expert Review of Hematology最新文献

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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":"<p><strong>Introduction: </strong>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.</p><p><strong>Areas covered: </strong>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.</p><p><strong>Expert opinion: </strong>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.</p>","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
Identification of miR-451 target genes as prognostic markers in diffuse large B-cell lymphoma. 将 miR-451 靶基因鉴定为弥漫大 B 细胞淋巴瘤的预后标志物。
IF 2.3 4区 医学
Expert Review of Hematology Pub Date : 2024-11-04 DOI: 10.1080/17474086.2024.2422019
Yi Zhang, Zichen Wei, Han Xu, Xin Wang, Ting Gu, Hongliang Dong, Hongzhuan Chang, Lei Pang
{"title":"Identification of miR-451 target genes as prognostic markers in diffuse large B-cell lymphoma.","authors":"Yi Zhang, Zichen Wei, Han Xu, Xin Wang, Ting Gu, Hongliang Dong, Hongzhuan Chang, Lei Pang","doi":"10.1080/17474086.2024.2422019","DOIUrl":"https://doi.org/10.1080/17474086.2024.2422019","url":null,"abstract":"<p><strong>Background: </strong>B-cell lymphoma, a diverse malignancy, is intricately regulated by multiple factors. MicroRNAs (miRNAs) have been demonstrated to be important regulators of the initiation and progression of human B-cell lymphoma, but their functions need to be further explored.</p><p><strong>Research design and methods: </strong>Two B-cell lymphoma cell lines, Romas and HBL-1, were engineered to overexpress miR-451, with cell proliferation assessed via cell counting assays. Flow cytometry was used to study the effects of miR-451 on cell cycle and apoptosis. Bioinformatics analyses were performed using GEO datasets (GSE181063, GSE32918, GSE56315) to identify DEGs, and potential miR-451 target genes were predicted using tools like ENCORI, TargetScan, and R packages. A risk model for DLBCL prognosis was developed using Cox and LASSO regression. qRT-PCR validated the expression of these target genes.</p><p><strong>Results: </strong>This study revealed that miR-451 inhibited cell proliferation, arrested the cell cycle, and induced apoptosis in human DLBCL cell lines. Bioinformatics analysis identified 9 target genes (<i>MMP9</i>, <i>AQP9</i>, <i>RIN2</i>, <i>EOMES</i>, <i>LCP2</i>, <i>SELPLG</i>, <i>MAL</i>, <i>SOCS5</i>, <i>S1PR3</i>) significantly associated with DLBCL prognosis, suggesting a potential mechanism by which miR-451 suppresses DLBCL development.</p><p><strong>Conclusions: </strong>Our study indicates that a specific set of miR-451 target genes may significantly influence DLBCL patient outcomes.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-12"},"PeriodicalIF":2.3,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142567416","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
What are the therapeutic options for previously treated myelofibrosis? 对于曾接受过治疗的骨髓纤维化,有哪些治疗方案?
IF 2.3 4区 医学
Expert Review of Hematology Pub Date : 2024-11-04 DOI: 10.1080/17474086.2024.2423367
Cassandre Petit, Hugues de Lavallade, Claire Harrison
{"title":"What are the therapeutic options for previously treated myelofibrosis?","authors":"Cassandre Petit, Hugues de Lavallade, Claire Harrison","doi":"10.1080/17474086.2024.2423367","DOIUrl":"https://doi.org/10.1080/17474086.2024.2423367","url":null,"abstract":"<p><strong>Introduction: </strong>The disruption of the JAK/STAT signaling pathway is a defining feature of myelofibrosis (MF). The introduction of JAK inhibitors (JAKi) has transformed the therapeutic approach to MF, becoming essential to treatment and reshaping the management landscape. While JAKi are now the preferred first-line treatment for most patients, various management options are available for those who do not respond to initial therapy.</p><p><strong>Areas covered: </strong>This review focuses on management options for patients with MF, with particular emphasis on therapeutic strategies following the failure of first-line JAKi. It provides a comprehensive overview of the current treatment landscape, including alternative JAKi and other approaches. The review is based on an extensive literature search using available databases (PubMed, Cochrane …) and relevant web resources (clinicaltrials.gov).</p><p><strong>Expert opinion: </strong>Ruxolitinib benefits in MF often diminish after 3-4 years, with complications like thrombocytopenia and anemia. Three newer JAKi offer alternatives with similar efficacy and varied side effects. Stem cell transplantation is a curative option for a minority, ideally timed at peak response to JAKi. Research aims to enhance first-line treatments and restore responses in resistant patients. Future therapies may include novel combinations or immunotherapies targeting specific mutations, requiring collaboration between patient, clinical, and pharmaceutical communities.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-12"},"PeriodicalIF":2.3,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142567336","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 review of the isocitrate dehydrogenase inhibitors in management of adult patients with AML and MDS. 综述异柠檬酸脱氢酶抑制剂在治疗急性髓细胞白血病和骨髓增生异常综合症成年患者中的应用。
IF 2.3 4区 医学
Expert Review of Hematology Pub Date : 2024-11-01 Epub Date: 2024-11-07 DOI: 10.1080/17474086.2024.2422554
Mallory Norman, Katelyn Yamartino, Rachel Gerstein, Rory Shallis, Lourdes Mendez, Nikolai Podoltsev, Maximilian Stahl, William Eighmy, Amer M Zeidan
{"title":"A review of the isocitrate dehydrogenase inhibitors in management of adult patients with AML and MDS.","authors":"Mallory Norman, Katelyn Yamartino, Rachel Gerstein, Rory Shallis, Lourdes Mendez, Nikolai Podoltsev, Maximilian Stahl, William Eighmy, Amer M Zeidan","doi":"10.1080/17474086.2024.2422554","DOIUrl":"10.1080/17474086.2024.2422554","url":null,"abstract":"<p><strong>Introduction: </strong>The development of oral therapies impacts the management of acute myeloid leukemia and myelodysplastic syndromes, especially for targetable mutations including <i>IDH1/2</i>.</p><p><strong>Areas covered: </strong>We discuss <i>IDH1/2</i> activity and inhibitor therapy in various settings, including monotherapy, combination therapy with hypomethylating agents, and other approaches.</p><p><strong>Expert opinion: </strong>Olutasidenib, enasidenib, and ivosidenib are approved for relapsed AML. Ivosidenib is approved for relapsed MDS and alone or with azacitidine in newly diagnosed AML. However, unanswered questions exist. In newly diagnosed AML, ivosidenib + azacitidine shows a survival benefit compared to azacitidine, but it is unknown whether ivosidenib + azacitidine demonstrates improved survival compared to ivosidenib. Ivosidenib + azacitidine demonstrated a survival benefit not seen with enasidenib + azacitidine. It is unclear whether newly diagnosed AML should be treated with azacitidine + ivosidenib or azacitidine + venetoclax. Azacitidine + venetoclax shows excellent response rates in IDH mutated disease. Retrospective data show low response rates of IDH inhibitor therapy post-venetoclax whereas HMA + venetoclax retains activity post IDH inhibition. The role of IDH inhibition post-transplant is unclear. Single-arm studies show post-transplant maintenance is safe; however, randomized trials are needed. Similarly, IDH inhibitors can be combined with chemotherapy however randomized studies are needed.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"755-767"},"PeriodicalIF":2.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142544657","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 danicopan in patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis. 阵发性夜间血红蛋白尿患者服用达尼可潘的安全性和疗效:系统综述和荟萃分析。
IF 2.3 4区 医学
Expert Review of Hematology Pub Date : 2024-11-01 Epub Date: 2024-10-28 DOI: 10.1080/17474086.2024.2422558
Emir Muvaffak, Muhammed Edib Mokresh, Abdullah Varda, Mahmoud Lakmoush, Merve Kabasakal Ilter
{"title":"Safety and efficacy of danicopan in patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis.","authors":"Emir Muvaffak, Muhammed Edib Mokresh, Abdullah Varda, Mahmoud Lakmoush, Merve Kabasakal Ilter","doi":"10.1080/17474086.2024.2422558","DOIUrl":"10.1080/17474086.2024.2422558","url":null,"abstract":"<p><strong>Introduction: </strong>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated intravascular hemolysis (IVH). Current treatments like Eculizumab and Ravulizumab have limitations, with many patients still requiring transfusions. This study aimed to investigate the safety and efficacy of the new emergent treatment called Danicopan.</p><p><strong>Methods: </strong>We systematically searched five electronic databases - Epistemonikos, Web of Science, Medline, Scopus, and ClinicalTrials - to ensure comprehensive coverage. The systematic review was conducted following the PRISMA guidelines, ensuring methodological rigor.</p><p><strong>Results: </strong>Four studies were eligible for inclusion, all of them were multicenter trials with 79 patients studied. Treatment with Danicopan led to a notable improvement in hemoglobin levels and a decrease in reticulocyte counts. However, LDH levels did not significantly change after treatment. Additionally, there was a significant increase in GPI-deficient erythrocytes but not in GPI-deficient granulocytes. Total and direct bilirubin levels showed significant differences between treatment groups, and there was an improvement in FACIT scores from baseline.</p><p><strong>Conclusions: </strong>Our systematic review and meta-analysis support the potential of Danicopan as a viable therapeutic option for PNH patients. The targeted inhibition of factor D within the complement system by Danicopan demonstrates both safety and efficacy in managing PNH, as evidenced by our findings.</p><p><strong>Registration: </strong>This paper was registered with the PROSPERO database (CRD42024499375).</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"819-831"},"PeriodicalIF":2.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142497813","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
Etiological spectrum of pancytopenia in adults based on hematological parameters and bone marrow studies. 基于血液学参数和骨髓研究的成人全血细胞减少症病因谱。
IF 2.3 4区 医学
Expert Review of Hematology Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1080/17474086.2024.2421374
Umera Saleem, Hamza Tariq, Shahroz Alam, Ahmed Faraz Zafar, Urwah Tanveer, Muhammad Hamza Khan
{"title":"Etiological spectrum of pancytopenia in adults based on hematological parameters and bone marrow studies.","authors":"Umera Saleem, Hamza Tariq, Shahroz Alam, Ahmed Faraz Zafar, Urwah Tanveer, Muhammad Hamza Khan","doi":"10.1080/17474086.2024.2421374","DOIUrl":"10.1080/17474086.2024.2421374","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to investigate the demographic, clinical, and diagnostic aspects of adult pancytopenia while exploring its etiological spectrum through hematological parameters and bone marrow studies.</p><p><strong>Research design and methods: </strong>This observational study involved 117 adult individuals ranging from 13 to 85 years who presented with pancytopenia. A comprehensive examination of demographic features, hematological parameters, clinical presentations, and physical findings, including liver and spleen characteristics, was conducted. Additionally, serological analyses for HBsAg and Anti HCV were performed. The diagnostic spectrum was determined through bone marrow studies.</p><p><strong>Results: </strong>Pancytopenia manifested with varied clinical symptoms, with generalized weakness (72.65%), fever (64.1%), dyspnea (54.70%), bleeding (34.2%), and weight loss (25.6%) being prominent. Physical examination revealed a range of liver and spleen characteristics, with hepatomegaly observed in 32.48% and splenomegaly in 44.4% of cases. Serological findings indicated HBsAg positivity in 8.5% and Anti HCV positivity in 21.37% of cases. The diagnostic distribution encompassed diverse conditions, with aplastic anemia (17.1%), megaloblastic anemia (12.8%), and myelodysplastic syndromes (12.8%) being prevalent.</p><p><strong>Conclusions: </strong>This study provides a comprehensive overview of the demographic, clinical, and diagnostic aspects of pancytopenia. The observed prevalence of different diagnoses underscores the necessity of a thorough evaluation, including bone marrow studies, for accurate diagnosis and appropriate management.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"861-869"},"PeriodicalIF":2.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142497811","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 role of interleukin-20 and vascular endothelial growth factor-A biomarkers in the detection of renal impairment in patients with multiple myeloma. 白细胞介素-20和血管内皮生长因子-A生物标志物在检测多发性骨髓瘤患者肾功能损害中的作用。
IF 2.3 4区 医学
Expert Review of Hematology Pub Date : 2024-11-01 Epub Date: 2024-10-17 DOI: 10.1080/17474086.2024.2417085
Dina Samir Elsaid, Tamer Abd Elhamid Elbedewy, Mona Ashraf Hamza, Riham Abdel-Hamid Haroun
{"title":"The role of interleukin-20 and vascular endothelial growth factor-A biomarkers in the detection of renal impairment in patients with multiple myeloma.","authors":"Dina Samir Elsaid, Tamer Abd Elhamid Elbedewy, Mona Ashraf Hamza, Riham Abdel-Hamid Haroun","doi":"10.1080/17474086.2024.2417085","DOIUrl":"10.1080/17474086.2024.2417085","url":null,"abstract":"<p><strong>Background: </strong>Multiple myeloma (MM) is a malignant incurable disease characterized by monoclonal plasma cell increase associated with renal impairment. Evaluation of neutrophil-to-lymphocyte ratio (NLR), monocyte-to-lymphocyte ratio (MLR), platelet-to-lymphocyte ratio (PLR), hemoglobin/red cell distribution width (HB/RDW), interleukin-20 (IL-20), and vascular endothelial growth factor-A (VEGFA) in patients with MM (with or without renal impairment) as prognostic and severity indicators.</p><p><strong>Research design and methods: </strong>A cross-sectional study was conducted on sixty MM patients with renal impairment, sixty MM patients without renal impairment, and sixty subjects (control group). Complete blood count, IL-20 immunoassay, and gene expression of IL-20, and VEGFA were evaluated.</p><p><strong>Results: </strong>Higher levels of NLR, MLR, and IL-20, and moreover lower levels of PLR, HB/RDW, as well as upregulation of IL-20, and VEGFA gene expression were detected in MM patients, especially those with renal impairment. Receiver operating characteristic curves analysis of NLR, MLR, PLR, and IL-20 showed high sensitivity and specificity in the diagnosis of MM and disease stages.</p><p><strong>Conclusions: </strong>NLR, MLR, PLR, HB/RDW, IL-20, and VEGFA may be implicated in the inflammatory process of MM and renal impairment pathogenesis. NLR, MLR, and IL-20 can be used as prognostic markers in MM stages.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"841-853"},"PeriodicalIF":2.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461603","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
Evolving strategies to overcome barriers in CAR-T cell therapy for acute myeloid leukemia. 克服急性髓性白血病 CAR-T 细胞疗法障碍的不断发展的战略。
IF 2.3 4区 医学
Expert Review of Hematology Pub Date : 2024-11-01 Epub Date: 2024-10-30 DOI: 10.1080/17474086.2024.2420614
Chanukya K Colonne, Erik L Kimble, Cameron J Turtle
{"title":"Evolving strategies to overcome barriers in CAR-T cell therapy for acute myeloid leukemia.","authors":"Chanukya K Colonne, Erik L Kimble, Cameron J Turtle","doi":"10.1080/17474086.2024.2420614","DOIUrl":"10.1080/17474086.2024.2420614","url":null,"abstract":"<p><strong>Introduction: </strong>Acute myeloid leukemia (AML) is a complex and heterogeneous disease characterized by an aggressive clinical course and limited efficacious treatment options in the relapsed/refractory (R/R) setting. Chimeric antigen receptor (CAR)-modified T (CAR-T) cell immunotherapy is an investigational treatment strategy for R/R AML that has shown some promise. However, obstacles to successful CAR-T cell immunotherapy for AML remain.</p><p><strong>Areas covered: </strong>In analyses of clinical trials of CAR-T cell therapy for R/R AML, complete responses without measurable residual disease have been reported, but the durability of those responses remains unclear. Significant barriers to successful CAR-T cell therapy in AML include the scarcity of suitable tumor-target antigens (TTA), inherent T cell functional deficits, and the immunoinhibitory and hostile tumor microenvironment (TME). This review will focus on these barriers to successful CAR-T cell therapy in AML, and discuss scientific advancements and evolving strategies to overcome them.</p><p><strong>Expert opinion: </strong>Achieving durable remissions in R/R AML will likely require a multifaceted approach that integrates advancements in TTA selection, enhancement of the intrinsic quality of CAR-T cells, and development of strategies to overcome inhibitory mechanisms in the AML TME.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"797-818"},"PeriodicalIF":2.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142497812","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
BCL-2 and BTK inhibitors for chronic lymphocytic leukemia: current treatments and overcoming resistance. 治疗慢性淋巴细胞白血病的 BCL-2 和 BTK 抑制剂:目前的治疗方法和克服耐药性的方法。
IF 2.3 4区 医学
Expert Review of Hematology Pub Date : 2024-11-01 Epub Date: 2024-10-06 DOI: 10.1080/17474086.2024.2410003
Tadeusz Robak, Magdalena Witkowska, Anna Wolska-Washer, Paweł Robak
{"title":"BCL-2 and BTK inhibitors for chronic lymphocytic leukemia: current treatments and overcoming resistance.","authors":"Tadeusz Robak, Magdalena Witkowska, Anna Wolska-Washer, Paweł Robak","doi":"10.1080/17474086.2024.2410003","DOIUrl":"10.1080/17474086.2024.2410003","url":null,"abstract":"<p><strong>Introduction: </strong>In the last decade, BTK inhibitors and the BCL-2 inhibitor venetoclax have replaced immunochemotherapy in the treatment of CLL.</p><p><strong>Areas covered: </strong>This review describes the use of BTK inhibitors and BCL2 inhibitors in the treatment of naive and relapsed or refractory CLL, with particular attention to the mechanisms of resistance. It also addresses the management of double-refractory patients, and the discovery of novel drugs. The corpus of papers was obtained by a search of the PubMed and Google Scholar databases for articles in English.</p><p><strong>Expert opinion: </strong>Covalent BTK inhibitors and venetoclax are commonly recommended for previously-untreated and relapsed/refractory CLL. However, resistance to both drug classes can develop over time. As such, double-refractory patients are difficult to manage and novel approaches are urgently needed.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"781-796"},"PeriodicalIF":2.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142364981","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
Combination therapy of dexamethasone, rituximab, and cyclosporine for adults with primary immune thrombocytopenia: a prospective observational study. 地塞米松、利妥昔单抗和环孢素联合治疗成人原发性免疫性血小板减少症:一项前瞻性观察研究。
IF 2.3 4区 医学
Expert Review of Hematology Pub Date : 2024-11-01 Epub Date: 2024-10-28 DOI: 10.1080/17474086.2024.2420633
Ying Liang, Cong Tang, Chunyu Liu, Lu Zhang, Yongguang Fang, Aixiang Zhao, Nannan Chen
{"title":"Combination therapy of dexamethasone, rituximab, and cyclosporine for adults with primary immune thrombocytopenia: a prospective observational study.","authors":"Ying Liang, Cong Tang, Chunyu Liu, Lu Zhang, Yongguang Fang, Aixiang Zhao, Nannan Chen","doi":"10.1080/17474086.2024.2420633","DOIUrl":"10.1080/17474086.2024.2420633","url":null,"abstract":"<p><strong>Background: </strong>To assess the effectiveness of the combination of dexamethasone, rituximab, and cyclosporine in treating adults with primary immune thrombocytopenia (ITP).</p><p><strong>Research design and methods: </strong>This prospective study enrolled consecutive adult patients diagnosed with ITP at the 967th Hospital of the Chinese People's Liberation Army Joint Service Support Force Hospital between November 2019 and February 2023.</p><p><strong>Results: </strong>Twenty-eight patients (13 males, median age 43.5 years) were included. All patients previously experienced ineffective or relapsed ITP, with a median disease duration of 26.5 (range, 7-72) months. At baseline, the median platelet (PLT) count was 13.5 × 10<sup>9</sup>/L (8.25-20 × 10<sup>9</sup>/L). Following treatment, the PLT counts were significantly increased at weeks 1, 3, and 4. The early response rates at weeks 1 and 4 were 82.1% (23/28 patients) and 71.4% (20/28 patients), respectively. The 1-, 3-, and 6-month response rates were 71.4% (20/28), 67.9% (19/28), and 75% (21/28). The treatment-free survival rates at 12 and 24 months were 82.35% (14/17) and 71.43% (10/14), respectively. Six patients experienced transient adverse reactions.</p><p><strong>Conclusions: </strong>The combination of dexamethasone, rituximab, and cyclosporine may present a promising therapeutic option for patients with refractory ITP, with good tolerability and mild adverse reactions.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"855-860"},"PeriodicalIF":2.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142497809","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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