{"title":"Maintenance therapy after ASCT in newly diagnosed multiple myeloma patients: single agent <i>versus</i> 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":"<p><strong>Introduction: </strong>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.</p><p><strong>Areas covered: </strong>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.</p><p><strong>Expert opinion: </strong>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.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"719-734"},"PeriodicalIF":2.1,"publicationDate":"2025-09-01","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}
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":"<p><strong>Background: </strong>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.</p><p><strong>Research design and methods: </strong>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 (<i>p=</i>0.004).</p><p><strong>Results: </strong>SORA maintenance significantly improved OS and RFS probability when compared to the control group (36.3 <i>vs</i> 11.6 months; <i>p</i> = 0.01 and 95.5% and 66.7%; <i>p</i> = 0.004; respectively). SORA maintenance effectively reduced the risk of death by 93.6% for patients in complete remission with detectable measurable residual disease (<i>p</i> = 0.001). At the last follow-up, 4.2% of patients died in SORA-group when compared with 50% in the control group (<i>p</i> < 0.001).</p><p><strong>Conclusions: </strong>SORA maintenance after HSCT diminishes the cumulative incidence of relapse as well as prolongs OS and RFS in FLT3-mutated AML.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"753-760"},"PeriodicalIF":2.1,"publicationDate":"2025-09-01","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}
François Depasse, Nikolaus B Binder, Mariona Bono, Matthias Germer, Michael Heins, Martina Leplatoni, Jürgen Patzke, Stephan Schwers, Michael Spannagl, Peter L Turecek
{"title":"Von Willebrand factor is a multifaceted player in hemostasis requiring a diverse array of analytical and diagnostic approaches.","authors":"François Depasse, Nikolaus B Binder, Mariona Bono, Matthias Germer, Michael Heins, Martina Leplatoni, Jürgen Patzke, Stephan Schwers, Michael Spannagl, Peter L Turecek","doi":"10.1080/17474086.2025.2525458","DOIUrl":"10.1080/17474086.2025.2525458","url":null,"abstract":"<p><strong>Introduction: </strong>Von Willebrand factor (VWF) plays a crucial role in hemostasis: its interactions with endothelial matrices, platelets, and factor VIII make it a key player in both primary hemostasis and coagulation. Pathology associated with VWF spans mild to severe bleeding manifestations in the case of inherited von Willebrand disease (VWD), the most common congenital bleeding disorder, or acquired von Willebrand syndrome (AVWS). Conversely, VWF can be associated with thrombotic manifestations in situations related to inflammation, infection, or inherited or acquired ADAMTS13 defects.</p><p><strong>Areas covered: </strong>This review article aims to provide guidance on the use and interpretation of clinical laboratory assays available to measure VWF and other factors related to VWD. Different VWF tests can be used in different clinical settings for efficient diagnosis and patient management. Assay limitations are also addressed.</p><p><strong>Expert opinion: </strong>A myriad of laboratory assays, from first line to esoteric assays, exist to enable adequate diagnosis of VWD and other diseases influenced by VWF. Clinical investigations of VWF are complicated because VWF has multiple functions which variably depend on the patient's pathophysiological status. The right choice of assays is therefore critical to provide adequate diagnosis in due time and with reasonable analytical efforts.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"701-718"},"PeriodicalIF":2.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144483756","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}
{"title":"An interview with Dr. Jean A. Yared on his experience as an investigator for obecabtagene autoleucel - by Reegan Burnell-Clarke (Commissioning Editor).","authors":"Jean A Yared","doi":"10.1080/17474086.2025.2524219","DOIUrl":"10.1080/17474086.2025.2524219","url":null,"abstract":"","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"697-699"},"PeriodicalIF":2.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144474371","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}
{"title":"Incorporating novel therapies into the treatment of older patients with classic Hodgkin lymphoma.","authors":"Aditya Ravindra, Eric Mou","doi":"10.1080/17474086.2025.2526685","DOIUrl":"10.1080/17474086.2025.2526685","url":null,"abstract":"<p><strong>Introduction: </strong>Outcomes of older patients with classic Hodgkin lymphoma (cHL) are inferior to those of younger patients secondary to their distinct clinical presentation, unique disease biology, increased burden of medical comorbidity, and poorer tolerance to conventional therapies. Greater attention to the objective parameters underlying patient fitness has led to the recognition of comprehensive geriatric assessments (CGA) as an important method to optimize selection for appropriate therapy intensity. Given the magnified risk associated with traditional chemotherapy in older patients with cHL, the incorporation of the novel agents brentuximab vedotin (BV) and immune checkpoint inhibitors (ICIs) into modern treatment paradigms offers promise for improved outcomes in this population.</p><p><strong>Areas covered: </strong>We describe historical outcomes in older patients with cHL, the underpinnings of traditional treatment strategies, the evolving therapeutic landscape via integration of novel therapies into the frontline and relapsed/refractory settings, and the importance of contextualizing therapy selection via formal CGAs.</p><p><strong>Expert opinion: </strong>Novel therapies have broadened the array of therapeutic options for older patients with cHL eligible for either curative or palliative intent therapy. Further investigation into rational combinations of these drugs, together with ongoing efforts to validate cHL-specific CGAs, aim to improve outcomes for older patients across the spectrum of fitness.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"735-751"},"PeriodicalIF":2.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144505312","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}
Walaa M Alsafi, Ola A El-Gendy, Ahmad I Al-Shafei, Ahmed A Hassan, Ishag Adam
{"title":"The association between hemoglobin level, anemia, and hypertension among adolescents in Sudan: a multicenter cross-sectional study.","authors":"Walaa M Alsafi, Ola A El-Gendy, Ahmad I Al-Shafei, Ahmed A Hassan, Ishag Adam","doi":"10.1080/17474086.2025.2554641","DOIUrl":"https://doi.org/10.1080/17474086.2025.2554641","url":null,"abstract":"<p><strong>Background: </strong>Limited data exist on the association between hemoglobin levels/anemia, C-reactive protein (CRP), and hypertension in adolescents. This study aimed to examine the associations between hemoglobin levels/anemia and hypertension among adolescents in two regions of Sudan (River Nile State and Gadarif).</p><p><strong>Research design and methods: </strong>A multicenter cross-sectional study. Sociodemographic characteristics were evaluated using a questionnaire. Standardized procedures were used to measure adolescents' weight, height, hemoglobin levels, and CRP. Multivariate binary analyses were conducted.</p><p><strong>Results: </strong>This study included 738 adolescents; 44.0% weremales and 56.0% were females. The median age of the adolescents was 14.8 (interquartile range, IQR: 13.1-16.3) years. Of the 738 adolescents, 69 (9.4%) had hypertension, and 222 (30.1%) had anemia. In multivariate binary analysis, increasing body mass index (BMI) (adjusted odd ratio, AOR = 1.12, 95% confidenceinterval [CI]: 1.05-1.18) and male sex (AOR = 1.84, 95% CI: 1.15-3.24) were positively associated with hypertension, where as anemia (AOR = 0.36, 95% CI:0.15-0.84) demonstrated an inverse association with hypertension. No associations were found between age, CRP, location, and hypertension.</p><p><strong>Conclusions: </strong>This study reported an inverse association between anemia and hypertension. Further research is necessary to investigate this population's complex association between hemoglobin levels/anemia and hypertension.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-6"},"PeriodicalIF":2.1,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144948417","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}
Kelsey Uminski, Lindsay Cowley, Tzu-Fei Wang, Alan Tinmouth, Roy Khalife
{"title":"Clinical decisions and factors influencing the management of persons with hemophilia requiring antithrombotic therapy: a qualitative study.","authors":"Kelsey Uminski, Lindsay Cowley, Tzu-Fei Wang, Alan Tinmouth, Roy Khalife","doi":"10.1080/17474086.2025.2554652","DOIUrl":"https://doi.org/10.1080/17474086.2025.2554652","url":null,"abstract":"<p><strong>Background: </strong>Persons with hemophilia face challenges when requiring antithrombotic therapy due to competing bleeding and thrombosis risks. The absence of robust evidence complicates clinical decision-making, relying on expert opinions and consensus.</p><p><strong>Research design and methods: </strong>To explore the decision-making processes of physicians managing antithrombotic therapy in persons with hemophilia, identify key factors shaping clinical judgment, and develop a decision-making framework to improve patient care and research. We conducted a qualitative study grounded in constructivist methodology, recruiting seven Canadian physicians with expertise in hemophilia and/or thromboembolic disorders. Three virtual focus groups were held and analyzed using reflexive thematic analysis. Themes were developed iteratively to identify key components.</p><p><strong>Results: </strong>Participants described five themes involving initial and continuous risk assessment of bleeding and thrombosis, selection of safe antithrombotic therapies or alternatives, and development of hemophilia-specific treatment plans. They highlighted the need for periodic reassessment of strategies and emphasized individualized, co-produced care. Each framework element encompassed multiple factors influencing decision-making toward patient-centered care.</p><p><strong>Conclusions: </strong>This study provides a decision-making framework to guide antithrombotic therapy in persons with hemophilia. By integrating risk assessments, individualized care, and shared decision-making, the framework addresses this high-risk context. Future research should validate the framework and incorporate patient perspectives to enhance practice.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-7"},"PeriodicalIF":2.1,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144948388","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}
{"title":"Complex interaction of δ-globin variant HbA<sub>2</sub>-Famagusta combined with β-globin variant Hb Monroe in <i>cis</i> with the HBB:c.-92C>G mutation: hematological characteristics and molecular diagnostic insights.","authors":"Sitthichai Panyasai, Wibhasiri Srisuwan, Thitima Sumphanapai, Surada Satthakarn","doi":"10.1080/17474086.2025.2549379","DOIUrl":"10.1080/17474086.2025.2549379","url":null,"abstract":"<p><strong>Background: </strong>Hemoglobin (Hb) variants can cause clinical conditions like thalassemia. Understanding their molecular phenotypes and clinical profiles is essential for accurate diagnosis and genetic counseling.</p><p><strong>Research design and methods: </strong>Blood samples from a 67-year-old Thai female and four relatives were analyzed using HPLC and capillary electrophoresis. PCR techniques and sequencing were used to identify globin gene mutations and β-globin haplotypes. Pathogenicity and transcription factor-binding sites were predicted. A rapid diagnostic method was developed and validated using 1,414 samples to assess the geographic distribution of the variants.</p><p><strong>Results: </strong>Hb analysis revealed HbA, HbA<sub>2</sub>, and a slower-migrating Hb fraction. Mutations identified included HBB:c.92 G > C(HbMonroe) in <i>cis</i> with HBB:c.-92C > G, and HBD:c.60C > A(HbA<sub>2</sub>-Famagusta). This novel combination exhibited normal HbA<sub>2</sub> levels, potentially masking β-thalassemia. Combining HbMonroe with HbE leads to severe clinical symptoms. HbA<sub>2</sub>-Famagusta was predicted to be benign, while HbMonroe was deleterious. The geographic distribution of HbMonroe was found to be 0.09% with an allele frequency of 0.00042 and 1.30% with an allele frequency of 0.00649 in β-thalassemia carriers and EF syndrome, respectively.</p><p><strong>Conclusions: </strong>HbMonroe is a β<sup>0</sup>-thalassemic variant affecting splicing and structure, undetectable by standard Hb analysis. Including HbA<sub>2</sub> variants in total HbA<sub>2</sub> quantification is essential for accurate diagnosis and improved genetic counseling in thalassemia-endemic areas.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-15"},"PeriodicalIF":2.1,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144859073","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}
Fatimah S Alshaikh, Abdelhalim Deifalla, Reginald P Sequeira, Alexander Woodman
{"title":"Gene polymorphisms predicting response to hydroxyurea treatment in Bahraini patients with sickle cell disease.","authors":"Fatimah S Alshaikh, Abdelhalim Deifalla, Reginald P Sequeira, Alexander Woodman","doi":"10.1080/17474086.2025.2546575","DOIUrl":"10.1080/17474086.2025.2546575","url":null,"abstract":"<p><strong>Background: </strong>This study investigated the association between response to hydroxyurea (HU) treatment and fetal hemoglobin (HbF), and the prevalence of mutations that regulate HbF synthesis, drug transport and biotransformation in sickle cell disease (SCD) patients.</p><p><strong>Research design and methods: </strong>Study included <i>n</i> = 390 Bahrainis with a history of sickle cell crises. Responders (<i>n</i> = 127; 68%) were patients achieving HbF ≥ 15% along with other improvements. Non-responders (<i>n</i> = 60; 32%) failed to achieve this threshold despite maximum tolerated dose treatment.</p><p><strong>Results: </strong>Hydroxyurea treated patients had decreased frequency of painful crises and hospitalizations, increased Hb and HbF and decreased sickle cell hemoglobin (HbS), and white blood cells (WBCs). The minor allele frequency of ARG2 (rs10483801), HBS1L-MYB (rs4895441), CYP2C19 (rs4986893) CYP2C19 (rs4244285), and OATP1B3 (rs3711358) gene was significantly higher in non-responders compared to responders. A negative correlation was found between the number of pain crises and hospitalizations per year and HbF%. No significant correlation was reported between the dosage and the number of hospitalizations per year. No significant correlation was found between the duration of treatment and HbF%.</p><p><strong>Conclusions: </strong>Findings highlight the importance of a personalized treatment approach to maximize the benefits and minimize the side effects of HU, thereby improving clinical outcomes.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-14"},"PeriodicalIF":2.1,"publicationDate":"2025-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144803930","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}
{"title":"Mucosal toxicity in hematological malignancies: prevention, management, and novel therapeutic insights.","authors":"Pasquale Niscola, Marco Giovannini","doi":"10.1080/17474086.2025.2546118","DOIUrl":"10.1080/17474086.2025.2546118","url":null,"abstract":"<p><strong>Introduction: </strong>Mucosal toxicities remain a longstanding and challenging concern in the treatment of hematopoietic malignancies (HM). In addition to the classic oral (OM) and gastrointestinal mucositis (GIM) induced by chemotherapy (CHT) and/or radiotherapy (RT), novel targeted treatments and immunotherapies may cause other forms of mucosal disorders.</p><p><strong>Areas covered: </strong>This overview provides updated insights into the pathobiology and management strategies for mucosal toxicities induced by treatments for HMs. Additionally, it reappraises classic forms of mucositis and novel mucosal toxicities induced by new treatments for HMs.</p><p><strong>Expert opinion: </strong>Although significant progress has been made in the pathophysiologic pathways of conventional CHT/RT-associated OM, much remains to be discovered. Indeed, OM and GIM have a multifactorial etiopathogenesis that includes direct effects, oxidative injury, upregulation of immunologic molecules, and changes in the microbiome. Preventive measures remain the cornerstone of management, mainly palliative in clinically established mucositis. However, new therapeutic insights, primarily related to mesenchymal cells and cytokine inhibitors, are emerging, and ongoing research is critical for translating these new findings into clinical practice.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-15"},"PeriodicalIF":2.1,"publicationDate":"2025-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144803931","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}