Expert Review of Hematology最新文献_第5页

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. 输血依赖型地中海贫血患者铁沉积定量MRI评估：临床管理见解。

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

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":"Background: 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.Research design and methods: 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.Results: 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 (MMP9, AQP9, RIN2, EOMES, LCP2, SELPLG, MAL, SOCS5, S1PR3) significantly associated with DLBCL prognosis, suggesting a potential mechanism by which miR-451 suppresses DLBCL development.Conclusions: Our study indicates that a specific set of miR-451 target genes may significantly influence DLBCL patient outcomes.","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":"Introduction: 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.Areas covered: 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).Expert opinion: 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.","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

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":"Introduction: 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.Methods: 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.Results: 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.Conclusions: 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.Registration: This paper was registered with the PROSPERO database (CRD42024499375).","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

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":"Introduction: The development of oral therapies impacts the management of acute myeloid leukemia and myelodysplastic syndromes, especially for targetable mutations including IDH1/2.Areas covered: We discuss IDH1/2 activity and inhibitor therapy in various settings, including monotherapy, combination therapy with hypomethylating agents, and other approaches.Expert opinion: 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.","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