Expert Review of Hematology最新文献_第5页

Maintenance therapy after ASCT in newly diagnosed multiple myeloma patients: single agent versus combination drugs. 新诊断多发性骨髓瘤患者ASCT后的维持治疗：单药与联合药物。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-27 DOI: 10.1080/17474086.2025.2525457

Enrica Antonia Martino, Ernesto Vigna, Caterina Labanca, Antonella Bruzzese, Francesco Mendicino, Giulio Caridà, Eugenio Lucia, Virginia Olivito, Noemi Puccio, Nicola Amodio, Antonino Neri, Fortunato Morabito, Massimo Gentile

{"title":"Maintenance therapy after ASCT in newly diagnosed multiple myeloma patients: single agent versus combination drugs.","authors":"Enrica Antonia Martino, Ernesto Vigna, Caterina Labanca, Antonella Bruzzese, Francesco Mendicino, Giulio Caridà, Eugenio Lucia, Virginia Olivito, Noemi Puccio, Nicola Amodio, Antonino Neri, Fortunato Morabito, Massimo Gentile","doi":"10.1080/17474086.2025.2525457","DOIUrl":"10.1080/17474086.2025.2525457","url":null,"abstract":"Introduction: Maintenance therapy plays a crucial role in prolonging progression-free survival and overall survival in multiple myeloma. Lenalidomide remains the gold standard, as demonstrated in phase 3 trials, consistently showing superior survival compared to observation or placebo. However, both established and novel agents - such as thalidomide and pomalidomide, proteasome inhibitors (PIs), monoclonal antibodies (moAbs), and bispecific antibodies - have been investigated as alternatives to assess their efficacy and safety.Areas covered: This review delivers a comprehensive analysis of the current landscape of maintenance strategies in MM and presents the available evidence supporting the efficacy of novel agents, both as monotherapy and in combination.Expert opinion: Maintenance therapy is a critical component of MM management, capable of improving disease control and survival. Lenalidomide has demonstrated its ability to extend patients' survival, but cumulative toxicity remains a significant concern. For high-risk patients, maintenance therapy with PIs and CD38-targeting moAbs has proven to improve outcomes. However, challenges such as quality of life, cost, accessibility, and treatment resistance persist. A minimal residual disease (MRD)-adapted maintenance strategy is desirable, particularly to enable personalized treatment approaches in clinical practice.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-16"},"PeriodicalIF":2.3,"publicationDate":"2025-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144483754","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

Post-transplant sorafenib effectively prevents relapse in FLT3-mutated acute myeloid leukemia. 移植后索拉非尼可有效预防flt3突变的急性髓系白血病复发。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-27 DOI: 10.1080/17474086.2025.2525453

Adrianna Spałek, Agata Wieczorkiewicz-Kabut, Patrycja Zielińska, Anna Kopińska, Krzysztof Woźniczka, Anna Koclęga, Aleksandra Butrym, Jarosław Czyż, Grzegorz Helbig

{"title":"Post-transplant sorafenib effectively prevents relapse in FLT3-mutated acute myeloid leukemia.","authors":"Adrianna Spałek, Agata Wieczorkiewicz-Kabut, Patrycja Zielińska, Anna Kopińska, Krzysztof Woźniczka, Anna Koclęga, Aleksandra Butrym, Jarosław Czyż, Grzegorz Helbig","doi":"10.1080/17474086.2025.2525453","DOIUrl":"10.1080/17474086.2025.2525453","url":null,"abstract":"Background: Almost 50% of patients with FLT3-ITD AML relapse despite undergoing allogeneic hematopoietic stem cell transplantation (HSCT). FLT-3 inhibitors (FLT3i) can be used in a post-HSCT setting as a relapse prevention.Research design and methods: We retrospectively compared 24 FLT3-mutated AML patients receiving post-HSCT SORA with a control group of 24 FLT3-mutated AML SORA-free individuals. SORA was initiated at a median of 2.9 months after transplantation. Median dosage was 600 mg daily with median treatment duration of 8.8 months. Due to toxicities, 16/24 patients required dose modifications with 6 early SORA withdrawals. After median follow-up of 20.7 months, 1 patient relapsed in SORA-group whereas 9 relapses were observed in the control group (p=0.004).Results: SORA maintenance significantly improved OS and RFS probability when compared to the control group (36.3 vs 11.6 months; p = 0.01 and 95.5% and 66.7%; p = 0.004; respectively). SORA maintenance effectively reduced the risk of death by 93.6% for patients in complete remission with detectable measurable residual disease (p = 0.001). At the last follow-up, 4.2% of patients died in SORA-group when compared with 50% in the control group (p < 0.001).Conclusions: SORA maintenance after HSCT diminishes the cumulative incidence of relapse as well as prolongs OS and RFS in FLT3-mutated AML.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-8"},"PeriodicalIF":2.3,"publicationDate":"2025-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144511770","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

Non- MPL-W515K/L mutations in myeloproliferative neoplasms: insights from two case reports and a review of the literature. 骨髓增殖性肿瘤中的非MPL-W515K/L突变：来自两个病例报告和文献综述的见解

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-27 DOI: 10.1080/17474086.2025.2527345

Ane Sofie Tønne Nesse, Hilde Kollsete Gjelberg, Miriam Sandnes, Rakel Brendsdal Forthun, Håkon Reikvam

{"title":"Non- MPL-W515K/L mutations in myeloproliferative neoplasms: insights from two case reports and a review of the literature.","authors":"Ane Sofie Tønne Nesse, Hilde Kollsete Gjelberg, Miriam Sandnes, Rakel Brendsdal Forthun, Håkon Reikvam","doi":"10.1080/17474086.2025.2527345","DOIUrl":"https://doi.org/10.1080/17474086.2025.2527345","url":null,"abstract":"Background: Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs) result from clonal proliferation of hematopoietic stem cells, and include polycythemia vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF). Key driver mutations in the JAK2, CALR, and MPL genes are important for diagnosis and differentiation of triple-negative cases. The MPL gene, particularly exon 10, harbors mutation hotspots influencing pathogenesis and prognosis.Research designand methods: Thisstudy presents two cases of atypical MPLmutations in MPN-patients and investigates the prevalence ofnon-canonical MPLmutationsin the literature.Results: Wereport two MPN cases with non-canonical MPLmutations (S204P and W515R) detected by next-generation sequencing.We also conducted a systematic review of the PubMed database,identifying 68 cases of non-W515L/K MPLmutations. A total of 86 mutations were identified, comprised of 32unique non-canonical mutations. W515R/S/A were the most frequent(31%), followed by V501A/M (15%) and S505N/C (13%). 59% of patientshad ET, 24% PMF and 13% post-ET/PV MF. Most mutations (71%) occurredin exon 10. 26% harbored concurrent JAK2,CALR andMPLmutations.Conclusions: Ourfindings highlight the importance of non-canonical mutations indiagnosis of MPN to prevent misclassification and improve patientmanagement. Understanding these mutations could lead to more tailoredtreatments and better outcomes in MPN patients.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144527119","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

An interview with Dr. Jean A. Yared on his experience as an investigator for obecabtagene autoleucel - by Reegan Burnell-Clarke (Commissioning Editor). 对Jean博士的采访讲述了他们作为obbecabtagene自燃汽油研究者的经历——regan Burnell（委托编辑）。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-27 DOI: 10.1080/17474086.2025.2524219

Jean A Yared

引用次数: 0

Is the early use of ketamine effective as an opioid sparing adjunct for treating pain in sickle cell disease patients? 早期使用氯胺酮作为阿片类药物保留辅助治疗镰状细胞病患者疼痛有效吗？

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-05-11 DOI: 10.1080/17474086.2025.2500605

Vineetha Reddy Nallagatla, Natasha M Archer

引用次数: 0

Treating sickle cell disease in resource-limited sub-Saharan Africa: recent strategies and recommendations in addressing the gaps for the provision of evidence-based management. 在资源有限的撒哈拉以南非洲治疗镰状细胞病：在提供循证管理方面弥补差距的最新战略和建议。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-05-11 DOI: 10.1080/17474086.2025.2500599

Miguel Brito, Catarina Ginete, Akinyemi Ofakunrin, Ijeoma Diaku-Akinwumi, Baba Psalm Duniya Inusa

{"title":"Treating sickle cell disease in resource-limited sub-Saharan Africa: recent strategies and recommendations in addressing the gaps for the provision of evidence-based management.","authors":"Miguel Brito, Catarina Ginete, Akinyemi Ofakunrin, Ijeoma Diaku-Akinwumi, Baba Psalm Duniya Inusa","doi":"10.1080/17474086.2025.2500599","DOIUrl":"10.1080/17474086.2025.2500599","url":null,"abstract":"Introduction: The management of Sickle cell disease (SCD) in sub-Saharan Africa (SSA) suffers from the lack of universal infant and population screening, inadequate access to standard treatment and poor public health prioritization amidst unstable political systems.Areas covered: The state of evidencebased management of SCD in SSA was investigated including sustainability of international funding agencies.Expert opinion: Current efforts are fragmentary along languages lines; sometimes driven by the funder's objectives and not the national agenda. The review highlighted the role of internal and external partnerships such as SPARCO, ARISE, CONSA, as well as technology-based support for the implementation of evidence-based care for SCD. We advocate for increased funding to implement SCD comprehensive care in line with the WHO SCD Framework for Primary, Secondary, Tertiary and Specialist Comprehensive Care at state and national level. To achieve this objective, it is important that SCD, as a leading non-communicable disease in Africa, be mandated as a standing agenda for the National Council of Ministers at the African Union, WHO and other regional bodies in Africa.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"447-462"},"PeriodicalIF":2.3,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143964168","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

How can we improve healthcare outcomes for hemophilia B patients with inhibitors? 我们如何改善使用抑制剂的B型血友病患者的医疗结果？

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-04-29 DOI: 10.1080/17474086.2025.2499524

Kristin N Maher, Allison P Wheeler

引用次数: 0

Kompetitive allele specific PCR (KASP) based genotyping of sickle gene in the selected sub-ethnic tribal population of Gujarat and Madhya Pradesh. 基于竞争等位基因特异性PCR （KASP）的古吉拉特邦和中央邦亚民族部落人群镰刀基因分型研究

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-05-08 DOI: 10.1080/17474086.2025.2499530

Urvi Budhbhatti, Bhumika Prajapati, Bhagirath Dave, Aman Tripathi, Chaitanya Joshi, Madhvi Joshi

{"title":"Kompetitive allele specific PCR (KASP) based genotyping of sickle gene in the selected sub-ethnic tribal population of Gujarat and Madhya Pradesh.","authors":"Urvi Budhbhatti, Bhumika Prajapati, Bhagirath Dave, Aman Tripathi, Chaitanya Joshi, Madhvi Joshi","doi":"10.1080/17474086.2025.2499530","DOIUrl":"10.1080/17474086.2025.2499530","url":null,"abstract":"Background: Indian tribal population is more vulnerable to sickle cell disease (SCD), an autosomal recessive disease caused by a single A>T transversion mutation in the HBB gene. Current study aims to develop a novel Kompetitive allele specific PCR (KASP) assay for genotyping of sickle gene in selected tribal communities of India.Patients and methods: The study was conducted from 2021 to 2023 involving 583 self-declared healthy individuals from four tribal communities, i.e. Korku, Kol, and Sahariya of Madhya Pradesh and Dongri bhil of Gujarat. The sickle cell genotyping was performed using KASP and PCR-RFLP methods and further validated by Sanger sequencing. Various hematological and biochemical parameters were also studied and compared for their association with identified genotypes.Results: Prevalence of sickle cell trait (SCT) was observed as 4.67%, 9.33%, and 16.41% in Kol, Korku, and Dongri bhil, respectively. The SCD prevalence of 0.7% was observed in Dongri bhil. The KASP assay reflects as a novel, high-throughput, and cost-effective method for effective screening. RBC, folic acid, fetal Hb (HBF), and bilirubin have shown significant association with sickle gene mutation.Conclusions: KASP assay offers a rapid, precise, and cost-effective method for screening in tribal populations, which can help in early identification, management, and elimination of disease.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"491-499"},"PeriodicalIF":2.3,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143981915","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

To enhance the detection of aplastic anemia in primary care settings: a population-based study in Italy. 加强在初级保健机构再生障碍性贫血的检测：在意大利的一项基于人群的研究。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-05-10 DOI: 10.1080/17474086.2025.2500604

Francesco Lapi, Ettore Marconi, Iacopo Cricelli, Adriana Masotti, Alessandro Rossi, Claudio Cricelli

{"title":"To enhance the detection of aplastic anemia in primary care settings: a population-based study in Italy.","authors":"Francesco Lapi, Ettore Marconi, Iacopo Cricelli, Adriana Masotti, Alessandro Rossi, Claudio Cricelli","doi":"10.1080/17474086.2025.2500604","DOIUrl":"10.1080/17474086.2025.2500604","url":null,"abstract":"Background: Aplastic Anemia (AA) is a rare, life-threatening condition featured by hypocellular bone marrow without leukemia or myelodysplastic syndromes. Early diagnosis is vital because of the effectiveness of the existing treatments. This study examined AA epidemiology and clinical correlates to aid general practitioners (GPs) in recognizing potential AA cases.Research design and methods: The Italian Health Search database (HSD) was used. A cohort study on individuals aged 18 years and older registered in the HSD between 1 January 1998, and 31 December 2022, estimated the prevalence and incidence rate of AA. Cases were operationally classified as 'certain,' 'probable,' and 'possible.' A case-control study was conducted to examine the clinical correlates of AA.Results: The cumulative prevalence was 3.8-4.8 per 100,000. The AA incidence rates for certain, certain/probable, and certain/probable/possible diagnoses were 0.3, 0.7, and 6 cases/million person-years, respectively. Increased infections (OR = 2.5), higher comorbidity burden (Charlson Index 1: OR = 2.14; 2+: OR = 2.43), and immunosuppressants use (OR = 14.9) were strongly associated with an AA diagnosis.Conclusions: Our findings indicate that AA is often underdiagnosed in primary care, but these data could help raise the suspicion of AA. Efforts are needed to utilize GPs' healthcare records for early AA identification and to enhance GP-hematologist collaboration.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"463-469"},"PeriodicalIF":2.3,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143989716","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

Vasculopathy among children and adolescents with sickle cell disease: the crosstalk with annexin A1, vitamin D, and myocardial iron overload. 儿童和青少年镰状细胞病患者的血管病变：与膜联蛋白A1、维生素D和心肌铁超载的串扰

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-04-18 DOI: 10.1080/17474086.2025.2495670

Mohsen Saleh ElAlfy, Eman Abdel Rahman Ismail, Sara Mostafa Makkeyah, Ahmed Samir, Dina Husseiny Salama, Nanis Mohammed Salah Eldin, Dina Mohamed Fathy ElMaghraby, Nada Ayman Gad, Marwa Fath Ahmed Ali, Fatma Soliman Elsayed Ebeid

{"title":"Vasculopathy among children and adolescents with sickle cell disease: the crosstalk with annexin A1, vitamin D, and myocardial iron overload.","authors":"Mohsen Saleh ElAlfy, Eman Abdel Rahman Ismail, Sara Mostafa Makkeyah, Ahmed Samir, Dina Husseiny Salama, Nanis Mohammed Salah Eldin, Dina Mohamed Fathy ElMaghraby, Nada Ayman Gad, Marwa Fath Ahmed Ali, Fatma Soliman Elsayed Ebeid","doi":"10.1080/17474086.2025.2495670","DOIUrl":"10.1080/17474086.2025.2495670","url":null,"abstract":"Background: Annexin A1 plays an important role in myocardial defense against ischemia-reperfusion injury. We aimed to evaluate the role of annexin A1 as a potential marker of vasculopathy in children and adolescents with sickle cell disease (SCD) and its relation to myocardial iron content (MIC) and vitamin D status.Research design and methods: Forty-one patients with SCD were compared with 40 age- and sex-matched healthy controls, and underwent assessment of serum annexin A1, vitamin D, Doppler echocardiography and cardiac magnetic resonance (CMR).Results: Six (14.6%) SCD patients had cardiac disease, five (12.2%) had abnormal MIC (≥1.16) and 10 (24.4%) had pulmonary hypertension risk. Annexin A1 levels were significantly lower among patients with SCD compared with healthy controls (p < 0.001). SCD patients with pulmonary hypertension risk, evidence of diastolic dysfunction, and nephropathy as well as those with serum ferritin ≥ 2500 µg/L and vitamin D deficiency had lower Annexin A1 levels than those without. Serum annexin A1 levels were negatively correlated to urinary albumin creatinine ratio (UACR) and Tei index while positively correlated to vitamin D among SCD patients.Conclusions: Annexin A1 could be a promising marker of vasculopathy and may provide a biochemical explanation for vitamin D deficiency in SCD.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"481-490"},"PeriodicalIF":2.3,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144001681","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