Khaled M Musallam, Sujit Sheth, Maria Domenica Cappellini, Gian Luca Forni, Aurelio Maggio, Ali T Taher
{"title":"Anemia and iron overload as prognostic markers of outcomes in β-thalassemia.","authors":"Khaled M Musallam, Sujit Sheth, Maria Domenica Cappellini, Gian Luca Forni, Aurelio Maggio, Ali T Taher","doi":"10.1080/17474086.2024.2383420","DOIUrl":"10.1080/17474086.2024.2383420","url":null,"abstract":"<p><strong>Introduction: </strong>Ineffective erythropoiesis and subsequent anemia as well as primary and secondary (transfusional) iron overload are key drivers for morbidity and mortality outcomes in patients with β-thalassemia.</p><p><strong>Areas covered: </strong>In this review, we highlight evidence from observational studies evaluating the association between measures of anemia and iron overload versus outcomes in both non-transfusion-dependent and transfusion-dependent forms of β-thalassemia.</p><p><strong>Expert opinion: </strong>Several prognostic thresholds have been identified with implications for patient management. These have also formed the basis for the design of novel therapy clinical trials by informing eligibility and target endpoints. Still, several data gaps persist in view of the challenge of assessing prospective long-term outcomes in a chronic disease. Pooling insights on the prognostic value of different measures of disease mechanism will be key to design future scoring systems that can help optimize patient management.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"631-642"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141747893","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":"The medication usage pattern and prevalence of polypharmacy among patients with sickle cell disease: a population-based study in southern Iran.","authors":"Abdullah Amini, Erfan Taherifard, Mohammadreza Akbari, Ehsan Taherifard, Niloofar Dehdari Ebrahimi, Mohammad Reza Rajabi, Fahimeh Zamani, Seyed Javad Rekabpour, Razieh Jafaraghaie, Jafar Hassanzadeh, Hossein Molavi Vardanjani","doi":"10.1080/17474086.2024.2385482","DOIUrl":"10.1080/17474086.2024.2385482","url":null,"abstract":"<p><strong>Background: </strong>Due to the numerous complications associated with sickle cell disease (SCD), patients often receive a variety of medications alongside their SCD treatment. However, a notable gap exists in the current literature regarding medication use patterns among them. This study aimed to investigate medication usage patterns in patients with SCD.</p><p><strong>Research design and methods: </strong>This cross-sectional study, conducted in Bushehr Province, employed a stratified random sampling method to select eligible participants with SCD. A thorough interview gathered various information, including details about the medications. The Anatomical Therapeutic Chemical classification system was utilized for drug classification. Polypharmacy was defined as the concurrent use of at least five medications.</p><p><strong>Results: </strong>A total of 300 individuals with SCD were included in this study. Polypharmacy was observed in 26.3% (95% CI: 20.8%-32.8%) of the study population. The analyses revealed positive associations between the use of more concurrent medication use and higher age groups and having multimorbidity. Antianemic preparations (86.7%), antineoplastic agents (58.3%), and vitamins (41.0%) were the most frequent medication classes used by the study participants.</p><p><strong>Conclusions: </strong>Our study revealed notable underutilization of hydroxyurea and a high rate of polypharmacy, associated with age and multimorbidity, among patients with SCD in southern Iran.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"643-650"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141765900","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}
Hilde K Gjelberg, Lars Helgeland, Galina Tsykunova, Håkon Reikvam
{"title":"Bone marrow aspirate or biopsy for multiple myeloma: when percentages matter!","authors":"Hilde K Gjelberg, Lars Helgeland, Galina Tsykunova, Håkon Reikvam","doi":"10.1080/17474086.2024.2383395","DOIUrl":"10.1080/17474086.2024.2383395","url":null,"abstract":"","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"543-546"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141733846","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":"How can we optimize antifungal use and stewardship in the treatment of acute leukemia?","authors":"Thomas Taynton, David Allsup, Gavin Barlow","doi":"10.1080/17474086.2024.2383401","DOIUrl":"10.1080/17474086.2024.2383401","url":null,"abstract":"<p><strong>Introduction: </strong>The global need for antifungal stewardship is driven by spreading antimicrobial and antifungal resistance. Triazoles are the only oral and relatively well-tolerated class of antifungal medications, and usage is associated with acquired resistance and species replacement with intrinsically resistant organisms. On a per-patient basis, hematology patients are the largest inpatient consumers of antifungal drugs, but are also the most vulnerable to invasive fungal disease.</p><p><strong>Areas covered: </strong>In this review we discuss available and forthcoming antifungal drugs, antifungal prophylaxis and empiric antifungal therapy, and how a screening based and diagnostic-driven approach may be used to reduce antifungal consumption. Finally, we discuss components of an antifungal stewardship program, interventions that can be employed, and how impact can be measured. The search methodology consisted of searching PubMed for journal articles using the term antifungal stewardship plus program, intervention, performance measure or outcome before 1 January 2024.</p><p><strong>Expert opinion: </strong>Initial focus should be on implementing effective antifungal stewardship programs by developing and implementing local guidelines and using interventions, such as post-prescription review and feedback, which are known to be effective. Technologies such as microbiome analysis and machine learning may allow the development of truly individualized risk-factor-based approaches to antifungal stewardship in the future.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"581-593"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141733847","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}
Shreyash Dalmia, Brian Harnett, Hanny Al-Samkari, Donald M Arnold
{"title":"Novel treatments for immune thrombocytopenia: targeting platelet autoantibodies.","authors":"Shreyash Dalmia, Brian Harnett, Hanny Al-Samkari, Donald M Arnold","doi":"10.1080/17474086.2024.2385485","DOIUrl":"10.1080/17474086.2024.2385485","url":null,"abstract":"<p><strong>Introduction: </strong>Immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by low platelets and an increased risk of bleeding. Platelet autoantibodies target major platelet glycoproteins and cause Fc-mediated platelet destruction in the spleen and reticuloendothelial systems. As mechanisms of disease, platelet autoantibodies are important therapeutic targets. Neonatal Fc receptor (FcRn) antagonists are a new class of therapeutics that reduce the half-life of immunoglobulin G including pathogenic platelet autoantibodies. Spleen tyrosine kinase (Syk) inhibitors interfere with Fc-mediated platelet clearance. Bruton's tyrosine kinase (BTK) inhibitors and B-cell activating factor (BAFF) inhibitors reduce antibody production. The efficacy of these targeted therapies provides new support for the role of platelet autoantibodies in pathogenesis of ITP even these antibodies can be difficult to detect.</p><p><strong>Areas covered: </strong>This review includes an in-depth exploration of the pathophysiologic mechanisms of ITP, focusing on autoantibodies. Treatments outlined in this review include a) FcRn antagonists, b) complement inhibitors, c) B-cell directed therapies such as BTK inhibitors, and anti-BAFF agents, d) Syk inhibitors, e) plasma-cell directed therapies, and f) novel cellular therapeutic products.</p><p><strong>Expert opinion: </strong>Platelet autoantibodies are often elusive in ITP, yet novel treatments targeting this pathway reinforce their role in the pathogenesis of this autoimmune platelet disorder.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"609-616"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141787681","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":"Gene therapies on the horizon for sickle cell disease: a clinician's perspective.","authors":"Henna Butt, John F Tisdale","doi":"10.1080/17474086.2024.2386366","DOIUrl":"10.1080/17474086.2024.2386366","url":null,"abstract":"<p><strong>Introduction: </strong>Sickle cell disease (SCD) is a monogenic disorder that exerts several detrimental health effects on those affected, ultimately resulting in significant morbidity and early mortality. There are millions of individuals globally impacted by this disease. Research in gene therapy has been growing significantly over the past decade, now with two FDA approved products, aiming to find another cure for this complex disease.</p><p><strong>Areas covered: </strong>This perspective article aims to provide a clinician's insight into the current landscape of gene therapies, exploring the novel approaches, clinical advances, and potential impact on the management and prognosis of SCD. A comprehensive literature search encompassing databases such as PubMed, Web of Science and Google Scholar was employed. The search covered literature published from 1980 to 2024, focusing on SCD and curative therapy.</p><p><strong>Expert opinion: </strong>After careful evaluation of the risks and benefits associated with the use of gene therapy for affected patients, the need for a cure outweighs the risks associated with treatment in most cases of SCD. With advances in current technologies, gene therapies can increase access to cures for patients with SCD.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"555-566"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141792361","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}
Nirija Ranjit Anderson, Dariusz Korczyk, Peter Mollee
{"title":"Improving care for systemic light-chain amyloidosis patients: is a multidisciplinary approach best?","authors":"Nirija Ranjit Anderson, Dariusz Korczyk, Peter Mollee","doi":"10.1080/17474086.2024.2388184","DOIUrl":"10.1080/17474086.2024.2388184","url":null,"abstract":"<p><strong>Introduction: </strong>Light chain (AL) amyloidosis is a rare and complex disease which can affect various systems of the body. In common with many rare and multisystemic diseases, the breadth of diagnostic, clinical, and supportive expertise required to care for such patients is best met by a multidisciplinary team.</p><p><strong>Areas covered: </strong>We outline different phases of the patients' journey, including diagnosis, staging, treatment, and response assessment, to highlight common clinical issues best resolved by a multidisciplinary approach.</p><p><strong>Expert opinion: </strong>To extend the benefit of multidisciplinary care to the majority of patients with AL amyloidosis, innovative healthcare models such as telehealth and multisite multidisciplinary team meetings need to be implemented. The need for a multidisciplinary approach where such a wide array of healthcare skills is required also highlights the shortcomings of our current diagnostic and monitoring assays. Better access to diagnostic and subtyping assays is necessary. The ability to characterize and measure the causative amyloidogenic light chain as well as imaging techniques to accurately diagnose and monitor response to therapy is also needed and is currently an area of research focus.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"567-579"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141999710","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}
Caterina Alati, Martina Pitea, Maria Caterina Mico, Violetta Marafioti, Bruna Greve, Giulia Pratico, Barbara Loteta, Francesca Cogliandro, Gaetana Porto, Giorgia Policastro, Giovanna Utano, Annalisa Sgarlata, Lucrezia Imbalzano, Ilaria Maria Delfino, Elisa Montechiarello, Jessyca Germano, Gianfranco Filippelli, Massimo Martino
{"title":"Optimizing maintenance therapy in acute myeloid leukemia: where do we stand in the year 2024?","authors":"Caterina Alati, Martina Pitea, Maria Caterina Mico, Violetta Marafioti, Bruna Greve, Giulia Pratico, Barbara Loteta, Francesca Cogliandro, Gaetana Porto, Giorgia Policastro, Giovanna Utano, Annalisa Sgarlata, Lucrezia Imbalzano, Ilaria Maria Delfino, Elisa Montechiarello, Jessyca Germano, Gianfranco Filippelli, Massimo Martino","doi":"10.1080/17474086.2024.2382300","DOIUrl":"10.1080/17474086.2024.2382300","url":null,"abstract":"<p><strong>Introduction: </strong>Despite the prognosis of patients affected by acute myeloid leukemia (AML) improved in the last decade, most patients relapse. Maintenance therapy after a chemotherapy approach with or without allogeneic stem cell transplantation could be a way to control the undetectable residual burden of leukemic cells. Several studies are being carried out as maintenance therapy in AML. Some critical points need to be defined, how the physician can choose among the various drugs available.</p><p><strong>Areas covered: </strong>This review discusses the advances and controversies surrounding maintenance therapy for AML patients.</p><p><strong>Expert opinion: </strong>Patients withFLT3-positive AML should receive midostaurin or quizartinib in the first-linesetting. For a patient initially receiving midostaurin, consider switching to sorafenib in the post-transplant setting. Because of the improved safety profile and potency, many experts will lean toward using a second-generation FLT3 inhibitor such as quizartinib or gilteritinib. Finally, no data indicate whether maintenance therapy should be prolonged until progression or for a defined period.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"515-525"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141626431","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}
Sigbjørn Berentsen, Josephine M I Vos, Agnieszka Malecka, Geir E Tjønnfjord, Shirley D'Sa
{"title":"The impact of individual clinical features in cold agglutinin disease: hemolytic versus non-hemolytic symptoms.","authors":"Sigbjørn Berentsen, Josephine M I Vos, Agnieszka Malecka, Geir E Tjønnfjord, Shirley D'Sa","doi":"10.1080/17474086.2024.2372333","DOIUrl":"10.1080/17474086.2024.2372333","url":null,"abstract":"<p><strong>Introduction: </strong>During the last decades, the pathogenesis of cold agglutinin disease (CAD) has been well elucidated and shown to be complex. Several documented or investigational therapies have been made available. This development has resulted in major therapeutic advances, but also in challenges in choice of therapy.</p><p><strong>Areas covered: </strong>In this review, we address each step in pathogenesis: bone marrow clonal lymphoproliferation, composition and effects of monoclonal cold agglutinin, non-complement mediated erythrocyte agglutination, complement-dependent hemolysis, and other effects of complement activation. We also discuss the heterogeneous clinical features and their relation to specific steps in pathogenesis, in particular with respect to the impact of complement involvement. CAD can be classified into three clinical phenotypes with consequences for established treatments as well as development of new therapies. Some promising future treatment approaches - beyond chemoimmunotherapy and complement inhibition - are reviewed.</p><p><strong>Expert opinion: </strong>The patient's individual clinical profile regarding complement involvement and hemolytic versus non-hemolytic features is important for the choice of treatment. Further development of treatment approaches is encouraged, and some candidate drugs are promising irrespective of clinical phenotype. Patients with CAD requiring therapy should be considered for inclusion in clinical trials.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"479-492"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141467350","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}
Maria Elisa Mancuso, Paul McLaughlin, Angela L Forsyth, Leonard A Valentino
{"title":"Joint health and pain in the changing hemophilia treatment landscape.","authors":"Maria Elisa Mancuso, Paul McLaughlin, Angela L Forsyth, Leonard A Valentino","doi":"10.1080/17474086.2024.2378936","DOIUrl":"10.1080/17474086.2024.2378936","url":null,"abstract":"<p><strong>Introduction: </strong>Hemophilia is an inherited bleeding disorder. Bleeding, and in particular joint hemorrhage results in chronic arthropathy and disability. Acute and chronic pain are frequent and limit activity and participation and result in decreased health-related quality of life. Remarkable progress has been made in the diagnosis and treatment of hemophilia but bleeding continues to prove recalcitrant to currently available treatments and joint disease remains problematic. Physiotherapy and pain management are mainstays of current multidisciplinary integrated care of people with hemophilia (PWH). The focus of this review is on preservation of joint health in the era of new and innovative therapies.</p><p><strong>Areas covered: </strong>A search of the PubMed Central was conducted on 1 February 2024 using the MeSH Major Topic terms identified as keywords for the manuscript. This review will highlight what is known and unknown about joint bleeding and arthropathy, including insights on pain as a related complication.</p><p><strong>Expert opinion: </strong>Recent advances in therapeutic interventions aimed at promoting healthy joints in PWH will be discussed, including both the pharmacological treatment landscape and related strategies to promote joint health.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"431-444"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141563157","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}