Expert Review of Hematology最新文献_第8页

How clinicians and persons with hemophilia may approach shared decision-making. 临床医生和血友病患者如何共同决策。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2024-06-01 Epub Date: 2024-05-11 DOI: 10.1080/17474086.2024.2353761

Courtney D Thornburg, Donna Coffin

引用次数: 0

Advances in our understanding of genetic markers and targeted therapies for pediatric LCH. 我们对小儿 LCH 遗传标记和靶向疗法的认识取得了进展。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2024-06-01 Epub Date: 2024-05-11 DOI: 10.1080/17474086.2024.2353772

Aban Bahabri, Oussama Abla

{"title":"Advances in our understanding of genetic markers and targeted therapies for pediatric LCH.","authors":"Aban Bahabri, Oussama Abla","doi":"10.1080/17474086.2024.2353772","DOIUrl":"10.1080/17474086.2024.2353772","url":null,"abstract":"Introduction: Langerhans cell histiocytosis (LCH) is a rare myeloid neoplasm, encompassing a diverse clinical spectrum ranging from localized bone or skin lesions to a multisystemic life-threatening condition. Over the past decade, there has been an expansion in understanding the molecular biology of LCH, which translated into innovative targeted therapeutic approaches.Areas covered: In this article, we will review the molecular alterations observed in pediatric LCH and the relationship between these molecular changes and the clinical phenotype, as well as targeted therapies in LCH.Expert opinion: Mitogen-activated protein kinase (MAPK) pathway mutation is a hallmark of LCH and is identified in 80% of the cases. Notably, BRAFV600E mutation is seen in ~50-60% of the cases, ~30% has other MAPK pathway mutations, while 15-20% have no detected mutations. While the first line therapeutic approach is vinblastine and prednisone, targeted therapies - specifically BRAF/MEK inhibitors - emerged as a promising second-line salvage strategy, particularly when a mutation is identified. Most patients respond to BRAF/MEK inhibitors but at least 75% reactivate after stopping, however, most patients respond again when restarting inhibitors.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"223-231"},"PeriodicalIF":2.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140891797","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

Elranatamab vs. teclistamab: battle of the BCMA bispecifics in relapsed/refractory multiple myeloma. 艾拉他单抗与替卡单抗：复发性/难治性多发性骨髓瘤中的BCMA双特异性之争。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2024-06-01 Epub Date: 2024-05-11 DOI: 10.1080/17474086.2024.2353751

Donald C Moore, Katelynn Granger, Hailey Hill, Allison Karabinos, James A Davis

引用次数: 0

Olutasidenib: a novel mutant IDH1 inhibitor for the treatment of relapsed or refractory acute myeloid leukemia. Olutasidenib：治疗复发或难治性急性髓性白血病的新型突变IDH1抑制剂。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2024-06-01 Epub Date: 2024-05-21 DOI: 10.1080/17474086.2024.2354486

Jorge E Cortes

{"title":"Olutasidenib: a novel mutant IDH1 inhibitor for the treatment of relapsed or refractory acute myeloid leukemia.","authors":"Jorge E Cortes","doi":"10.1080/17474086.2024.2354486","DOIUrl":"10.1080/17474086.2024.2354486","url":null,"abstract":"Introduction: Recurrent mutations in isocitrate dehydrogenase 1 (mIDH1) occur in about 7% to 14% of all cases of acute myeloid leukemia (AML). The discovery of targetable mutations in AML, including IDH mutations, expanded the therapeutic landscape of AML and led to the development of targeted agents. Despite significant advances in current treatment options, remission and overall survival rates remain suboptimal. The IDH1 inhibitor, olutasidenib, demonstrated encouraging safety and clinical benefits as monotherapy in patients with relapsed or refractory (R/R) mIDH1 AML.Areas covered: This review outlines the olutasidenib drug profile and summarizes key safety and efficacy data, focusing on the 150 mg twice daily dose from the pivotal registrational cohort of the phase 2 trial that formed the basis for the US Food and Drug Administration approval of olutasidenib in patients with R/R AML with a susceptible IDH1 mutation.Expert opinion: Olutasidenib offers patients with R/R mIDH1 AML a new treatment option, with improved complete remission and a longer duration of response than other targeted mIDH1 treatment options. Olutasidenib provided clinical benefit with a manageable safety profile. Additional analyses to further characterize the safety and efficacy of olutasidenib in frontline and R/R settings as monotherapy and as combination therapy are ongoing.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"211-221"},"PeriodicalIF":2.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140921265","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

Disruption of the Hippo pathway promotes the proliferation of childhood acute lymphoblastic leukemia cells, inhibits apoptosis and chemosensitivity. hippo 通路的中断会促进儿童急性淋巴细胞白血病细胞的增殖，抑制细胞凋亡和化疗敏感性。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2024-06-01 Epub Date: 2024-05-20 DOI: 10.1080/17474086.2024.2356255

Hao Jiang, Rui-Bo Zhang, Juan Peng, Lan Ren, Heng-Dong Wang

{"title":"Disruption of the Hippo pathway promotes the proliferation of childhood acute lymphoblastic leukemia cells, inhibits apoptosis and chemosensitivity.","authors":"Hao Jiang, Rui-Bo Zhang, Juan Peng, Lan Ren, Heng-Dong Wang","doi":"10.1080/17474086.2024.2356255","DOIUrl":"10.1080/17474086.2024.2356255","url":null,"abstract":"Background: Despite advancements in chemotherapy and stem cell transplantation, the recurrence and chemoresistance of childhood acute lymphoblastic leukemia (cALL) remain a significant challenge, thus indicating the need for novel therapeutic targets.Research design and methods: The protein levels of YAP1, p-YAP1, TAZ, and Cyr61 of cALL patients and healthy volunteers were measured by western blot analysis. Then the leukemic cell line SUP-B15 was transfected with sh-YAP1 and pcDNA3.1-YAP1 to knockdown or overexpress YAP1. The viability, chemosensitivity, apoptosis, migration, and invasion of SUP-B15 cells were determined by MTT, flow cytometry, and Transwell assay.Results: The cALL patients had higher YAP1, TAZ, and Cyr61 protein expression and lower p-YAP1 protein expression in bone marrow tissues compared with healthy volunteers (p < 0.01). In SUP-B15 cells, YAP1 knockdown upregulated p-YAP1 protein expression (p < 0.01) and downregulated TAZ and Cyr61 protein expression (p < 0.01). In addition, knocking down YAP1 significantly inhibited cell viability, migration, and invasion, and induced apoptosis (p < 0.01). YAP1 knockdown also reduced the IC50 value following treatment with vincristine, daunorubicin, cyclophosphamide, and dexamethasone (p < 0.05).Conclusions: Disruption of the Hippo pathway attenuates the development of cALL by promoting cell proliferation while suppressing apoptosis and drug sensitivity.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"269-274"},"PeriodicalIF":2.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140957097","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

Evaluation of the effect of voxelotor and darbepoetin alfa on hemoglobin levels in patients with sickle cell disease. 评估Voxelotor和darbepoetin alfa对镰状细胞病患者血红蛋白水平的影响。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2024-06-01 Epub Date: 2024-05-16 DOI: 10.1080/17474086.2024.2352497

Salome Bwayo Weaver, Helen Akinwale, Nkem P Nonyel, La'marcus T Wingate

{"title":"Evaluation of the effect of voxelotor and darbepoetin alfa on hemoglobin levels in patients with sickle cell disease.","authors":"Salome Bwayo Weaver, Helen Akinwale, Nkem P Nonyel, La'marcus T Wingate","doi":"10.1080/17474086.2024.2352497","DOIUrl":"10.1080/17474086.2024.2352497","url":null,"abstract":"Background: To date, there is limited evidence on patients utilizing both voxelotor and darbepoetin alfa and its impact on hemoglobin levels. The objective is to evaluate the effect of voxelotor and darbepoetin alfa on hemoglobin levels in patients with SCD.Research design and methods: This was a retrospective chart review study that assessed the primary independent variable as the utilization of either voxelotor alone, darbepoetin alfa alone, or the concurrent administration of voxelotor and darbepoetin alfa. Descriptive statistics were utilized to obtain the mean standard deviation for numerical variables and proportions for categorical variables.Results: A total of 23 participants were included in this study. When comparing baseline to 2 months and 3 months, participants on voxelotor alone experienced a 3% decrease and a 6.6% increase in hemoglobin, darbepoetin alfa alone group a 4.3% decrease and a 0.6% increase in hemoglobin and voxelotor and darbepoetin group a 4.4% decrease and a 0.5% decrease in hemoglobin levels. Fifty percent of the participants in the voxelotor group and 6 (66.7%) participants in the voxelotor plus darbepoetin alfa group experienced adverse drug events.Conclusions: Voxelotor resulted in a clinically significant difference in the percent change of hemoglobin from baseline to 3 months.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"255-260"},"PeriodicalIF":2.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140957101","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

Frontline combination of dasatinib and low-intensity chemotherapy in adults with de novo Philadelphia chromosome-positive acute lymphoblastic leukemia 达沙替尼与低强度化疗的前线组合治疗新发费城染色体阳性急性淋巴细胞白血病成人患者

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2024-04-14 DOI: 10.1080/17474086.2024.2343885

Yizhou Peng, Jinjin Huang, Jin Yin, Fankai Meng, Yang Cao, Liang Huang, Dengju Li, Yicheng Zhang, Donghua Zhang, Li Meng, Zhiqiang Han, Zhenya Hong

引用次数: 0

Curative effect of motherwort combined with ethinylestradiol-cyproterone acetate on dysfunctional uterine bleeding 益母草联合醋酸炔雌醇-环丙孕酮对功能失调性子宫出血的治疗效果

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2024-04-13 DOI: 10.1080/17474086.2024.2343878

Qiaoyun Tang, Xueyuan Chen

引用次数: 0

Hemophagocytic lymphohistiocytosis in Egyptian children: diagnosis, treatment challenges and outcome 埃及儿童嗜血细胞淋巴组织细胞增多症：诊断、治疗挑战和结果

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2024-04-10 DOI: 10.1080/17474086.2024.2341044

Azza A Tantawy, Nayera H.K. Elsherif, Solaf M Elsayed, Heba G.A. Ali, Sara M Makkeyah, Hisham I.E Elsantiel, Geneviève de Saint Basile, Iman A Ragab

引用次数: 0

Clinical characteristics of HFE C282Y/H63D compound heterozygotes identified in a specialty practice: key differences from HFE C282Y homozygotes. 在专科诊所发现的 HFE C282Y/H63D 复合杂合子的临床特征：与 HFE C282Y 同合子的主要区别。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-04-01 Epub Date: 2024-04-03 DOI: 10.1080/17474086.2024.2337950

Mukul Singal, Amir Mahmoud, Pradyumna Dinkar Phatak

{"title":"Clinical characteristics of HFE C282Y/H63D compound heterozygotes identified in a specialty practice: key differences from HFE C282Y homozygotes.","authors":"Mukul Singal, Amir Mahmoud, Pradyumna Dinkar Phatak","doi":"10.1080/17474086.2024.2337950","DOIUrl":"10.1080/17474086.2024.2337950","url":null,"abstract":"Background: Patients with p.C282Y homozygous (p.C282Y) HFE mutations are more likely to develop hemochromatosis (HC) than p.C282Y/p.H63D compound heterozygotes (p.C282Y/H63D).Research design and methods: We conducted a retrospective chart review of 90 p.C282Y and 31 p.C282Y/H63D patients at a referral practice to illustrate the differences in the natural history of the disease in these two HC cohorts.Results: Over a median follow-up of 17 years, p.C282Y had higher mean serum ferritin (1105 mg/dL vs. 534 mg/dL, p = 0.001) and transferrin saturations (75.3% vs. 49.5%, p = 0.001) at diagnosis. p.C282Y underwent more therapeutic phlebotomies (TP) till de-ironing (mean 24 vs. 10), had higher mean mobilized iron stores (4759 mg vs. 1932 mg), and required more annual maintenance TP (1.9/year vs. 1.1/year, p = 0.039). p.C282Y/H63D were more likely to have obesity (45.2% vs. 20.2%, p = 0.007) at diagnosis, with a non-significant trend toward consuming more alcohol. There was no significant difference in the development of HC-related complications between the two cohorts.Conclusions: p.C282Y have a higher mobilizable iron and require more TP. p.C282Y/H63D likely require additional insults such as obesity or alcohol use to develop elevated ferritin. De-ironing may mitigate the risk of developing HC-related complications.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"145-152"},"PeriodicalIF":2.3,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140326523","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