{"title":"Emicizumab and unmet needs of patients with hemophilia a who are managed with replacement therapies.","authors":"Johnny Mahlangu","doi":"10.1080/17474086.2024.2402304","DOIUrl":"https://doi.org/10.1080/17474086.2024.2402304","url":null,"abstract":"INTRODUCTIONHemophilia A is managed with coagulation clotting factor VIII (FVIII) therapy that poses significant challenges, such as a high treatment burden, immunogenicity, inconsistent hemostatic cover, poor treatment outcomes, and musculoskeletal progression despite adequate prophylactic treatment. Various non-factor therapies, such as several natural anticoagulant inhibitors and factor FVIII mimetics, have been developed to address these unmet needs. However, the role of emicizumab in addressing these unmet needs remains underexplored.AREAS COVEREDThis review delves into the evolution of hemophilia A replacement clotting therapy from plasma-derived products to recombinant products and, more recently, nonfactor therapies. It underscores the unmet needs of replacement therapy and explores the nonfactor therapies developed to address them. The review then comprehensively summarizes the clinical trial and real-world experience data, demonstrating how emicizumab tackles these unsatisfied demands.EXPERT OPINIONReplacement clotting factor therapies as the standard of care has exposed several needs that have yet to be addressed. However, data from numerous emicizumab clinical trials and real-world experience offer a promising outlook, suggesting that it may effectively address many unmet needs. As hemophilia treatment goals continue to evolve, the role of currently developed nonfactor therapies in hemophilia management is yet to be fully defined.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":"65 1","pages":""},"PeriodicalIF":2.8,"publicationDate":"2024-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142204687","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}
Marina Beltrami-Moreira, Amy Sharma, James B Bussel
{"title":"Immune thrombocytopenia and pregnancy: challenges and opportunities in diagnosis and management.","authors":"Marina Beltrami-Moreira, Amy Sharma, James B Bussel","doi":"10.1080/17474086.2024.2385481","DOIUrl":"10.1080/17474086.2024.2385481","url":null,"abstract":"<p><strong>Introduction: </strong>Immune thrombocytopenia (ITP) affecting pregnancy is a diagnostic and often a therapeutic challenge.</p><p><strong>Areas covered: </strong>We review the current diagnostic criteria for ITP in pregnancy and the potential utility of laboratory tests. We discuss the impact of ITP on pregnancy outcomes and the effects of pregnancy on patients living with chronic ITP. We describe the criteria for intervention, the evidence supporting first-line treatment approaches and the therapeutic decisions and challenges in cases refractory to steroids and IVIG. We review the evidence supporting the potential use of thrombopoietin receptor agonists for refractory thrombocytopenia. Finally, we describe the diagnostic, prognostic, and treatment approaches to neonatal ITP and considerations regarding breastfeeding. We searched the terms 'immune thrombocytopenia' and 'pregnancy' on PubMed to identify the relevant literature published before 31 December 2023, including within cited references.</p><p><strong>Expert opinion: </strong>Decreased platelet production may play a role in pregnancy-related ITP exacerbation. Putative mechanisms include placental hormones, such as inhibin. Although IVIG and prednisone usually suffice to achieve hemostasis for delivery, second-line agents are sometimes required to allow for neuraxial anesthesia. There is growing evidence supporting the use of romiplostim during pregnancy; however, its risk of venous thromboembolism warrants further evaluation.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"595-607"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141893299","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":"What is the future of digital tools to help manage pain in sickle cell disease patients?","authors":"Carlton Dampier","doi":"10.1080/17474086.2024.2388188","DOIUrl":"10.1080/17474086.2024.2388188","url":null,"abstract":"","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"539-541"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141893300","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}
Noomi Vainer, Viktor Rotbain Curovic, Carsten Utoft Niemann, Susan L Slager, Emelie Curovic Rotbain
{"title":"Understanding the interplay between chronic lymphocytic leukemia and type 2 diabetes.","authors":"Noomi Vainer, Viktor Rotbain Curovic, Carsten Utoft Niemann, Susan L Slager, Emelie Curovic Rotbain","doi":"10.1080/17474086.2024.2383417","DOIUrl":"10.1080/17474086.2024.2383417","url":null,"abstract":"<p><strong>Introduction: </strong>Comorbidities play an important role in the management of chronic lymphocytic leukemia (CLL) and may influence survival and treatment outcomes. Considering the aging general population and increasing incidence of type 2 diabetes (T2D), a comprehensive understanding of the interplay between CLL and T2D is essential for optimizing care and outcomes.</p><p><strong>Areas covered: </strong>We present current knowledge on co-existing CLL and T2D including prevalence, shared etiology and risk factors and how the conditions and treatment hereof may influence the outcome of one another. A literature search was performed using PubMed with the cutoff date on 1 February 2024.</p><p><strong>Expert opinion: </strong>The increased mortality observed in persons with CLL who have co-existing T2D is partially ascribed to infections, prompting physicians managing individuals with both conditions to consider closer monitoring during instances of infection and individualized prophylaxis. People with CLL and T2D should be managed for CLL in accordance with the international working group on CLL criteria, and we recommend that physicians exercise particular care not to delay treatment for these individuals. Multidisciplinary approaches with involvement of several specialties may be required for optimal supportive care of co-occurring T2D and CLL.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"617-629"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141747894","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}
Madugodaralalage D S K Gunaratne, Min Shi, Ronald S Go
{"title":"A comprehensive review on gelatinous transformation of the bone marrow.","authors":"Madugodaralalage D S K Gunaratne, Min Shi, Ronald S Go","doi":"10.1080/17474086.2024.2385472","DOIUrl":"10.1080/17474086.2024.2385472","url":null,"abstract":"<p><strong>Introduction: </strong>Gelatinous transformation of the bone marrow (GTBM) represents a clinically significant but often underdiagnosed condition, emphasizing the pivotal role of accurate diagnosis in facilitating appropriate treatment strategies.</p><p><strong>Areas covered: </strong>This special report synthesizes insights gathered from a comprehensive appraisal of clinical and pathology publications on GTBM available on PubMed. By employing search terms such as 'gelatinous,' 'gelatinous transformation,' and 'bone marrow,' this report aims to provide a nuanced understanding of GTBM, elucidating distinctive pathological features while distinguishing it from similar pathologies. The review also discusses currently identified causes of GTBM, clinical, imaging, pathologic, and laboratory findings that are associated with GTBM, and treatment options available.</p><p><strong>Expert opinion: </strong>Contrary to popular belief, we suggest that nutrient deficiency is not solely responsible for the pathogenesis of GTBM and that malignancies, infection, and inflammatory conditions play a critical role in its pathogenesis. We propose that further research on the pathophysiology of GTBM should be performed to unravel the complex interplay of nutritional and inflammatory factors in hematopoiesis, paving the way for innovative treatment approaches in hematopoietic disorders. To better facilitate further research in GTBM, we suggest formulating a pooled patient database with nutritional, genetic, and cytokine markers in a prospective fashion.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"547-554"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141765899","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}
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}