Expert Review of Hematology最新文献

Comprehensive review of morphological diagnosis of adult T-cell leukemia-lymphoma.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-04-09 DOI: 10.1080/17474086.2025.2490759

Shigeo Fuji, Masakazu Muta, Tomoko Hisakata, Noriaki Kawano, Eiichirou Ikeda, Hiroyoshi Kouno, Yuuya Tanabe, Kayoko Nakanish

{"title":"Comprehensive review of morphological diagnosis of adult T-cell leukemia-lymphoma.","authors":"Shigeo Fuji, Masakazu Muta, Tomoko Hisakata, Noriaki Kawano, Eiichirou Ikeda, Hiroyoshi Kouno, Yuuya Tanabe, Kayoko Nakanish","doi":"10.1080/17474086.2025.2490759","DOIUrl":"https://doi.org/10.1080/17474086.2025.2490759","url":null,"abstract":"Introduction: Adult T-cell leukemia-lymphoma (ATL) is a rare hematological malignancy associated with human T-cell leukemia virus type 1 (HTLV-1) infection. Accurate diagnosis and classification of ATL clinical subtypes rely on the identification of abnormal lymphocytes in peripheral blood smears. However, the diverse morphology of ATL cells and lack of standardized evaluation criteria worldwide can pose challenges.Area covered: This review consolidates expert opinions from clinical laboratory technologists experienced in the diagnosis of ATL. Here, we present the characteristic features of ATL cells and the common pitfalls encountered in clinical practice. In acute-type ATL, the 'flower cell' with its deeply lobulated, petal-like nucleus is a hallmark feature. While highly diagnostic, flower cells may not be abundant in all samples, and cells with minimal nuclear atypia can coexist. Chronic-type ATL is often characterized by small ATL cells with nuclear indentations or twists and hyperchromatic chromatin. Smoldering-type ATL cells share a similar morphology but generally exhibit milder nuclear atypia.Expert opinion: This review would facilitate the identification of ATL cells in routine clinical practice and serves as a foundation for future discussion and standardization of morphological criteria for ATL diagnosis.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143810889","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

The challenges of iron chelation therapy in thalassemia: how do we overcome them?

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-04-08 DOI: 10.1080/17474086.2025.2489562

Lauren E Wang, Sara Muttar, Sherif M Badawy

{"title":"The challenges of iron chelation therapy in thalassemia: how do we overcome them?","authors":"Lauren E Wang, Sara Muttar, Sherif M Badawy","doi":"10.1080/17474086.2025.2489562","DOIUrl":"10.1080/17474086.2025.2489562","url":null,"abstract":"Introduction: Packed red blood cell (pRBC) transfusions are the primary treatment for thalassemia. However, chronic transfusions ultimately result in iron overload, causing heart, liver, and endocrine complications along with other comorbidities. Although iron chelation is routinely initiated to remove excess iron, adherence remains a challenge, and iron overload still contributes to significant morbidity and early mortality in thalassemia.Areas covered: We review the evidence for iron overload and its complications in thalassemia. We also assess iron chelation strategies with possible adherence challenges categorized as patient-, medication-, and system-related barriers. Evidence suggests that lower adherence rates have been associated with more endorsed barriers. Further, patient-related barriers could be internal or external, and taking a patient-centered approach is key to addressing these challenges. Choosing the right iron chelator could help overcome some medication-related barriers. Finally, insurance coverage and access to specialized centers could affect initiation of iron chelation.Expert opinion: A critical and routine assessment of adherence barriers is key to optimizing patients' adherence to iron chelation. Adherence is often a multifactorial process, and it varies over time. Shared decision making with patients and/or caregivers is an important next step to improving adherence to iron chelation, and ultimately health outcomes.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-7"},"PeriodicalIF":2.3,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143779580","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

Correlation between hematocrit and the risk of common human cancers: results of a 1999-2020 observational survey and Mendelian randomization analysis.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-04-03 DOI: 10.1080/17474086.2025.2486381

Guo-Sheng Li, Tao Huang, Jing-Xiao Li, Jun Liu, Xiang Gao, Guan-Qiang Yan, Nuo Yang, Hua-Fu Zhou

{"title":"Correlation between hematocrit and the risk of common human cancers: results of a 1999-2020 observational survey and Mendelian randomization analysis.","authors":"Guo-Sheng Li, Tao Huang, Jing-Xiao Li, Jun Liu, Xiang Gao, Guan-Qiang Yan, Nuo Yang, Hua-Fu Zhou","doi":"10.1080/17474086.2025.2486381","DOIUrl":"10.1080/17474086.2025.2486381","url":null,"abstract":"Background: Previous studies have underscored the significance of hematocrit (HCT) in different tumors. However, further research is needed to explore its causal relationship with various tumors.Research design and methods: A retrospective analysis and a Mendelian randomization analysis were conducted on a sample of 3,580,275 samples to investigate the causal relationships between high HCT levels and the risks of developing the 15 prevalent malignant tumors. Additionally, using an in-house cohort, Kaplan-Meier curves were utilized to examine the relationship between HCT levels and the prognosis of patients with prostate cancer (PCa).Results: An elevated HCT level was identified as a protective factor against six types of cancers, including PCa (odds ratio [OR] < 1.000, p < 0.05), and a risk factor for cervical cancer, melanoma, and non-melanoma skin cancer (OR > 1.000, p < 0.05). A potential causal association was found between high HCT levels and decreased risks of developing PCa (OR = 0.887, p < 0.05) and breast cancer (OR = 0.893, p < 0.05). HCT was identified as a protective factor against PCa (p < 0.05).Conclusions: This study identified significant associations between HCT and nine tumors. HCT levels may serve as a protective factor against PCa.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-12"},"PeriodicalIF":2.3,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751936","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

Five decades of successive establishment of hemophilia care in Thailand.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-04-02 DOI: 10.1080/17474086.2025.2486378

Ampaiwan Chuansumrit, Thip Sriphaisal, Triroj Krutvecho, Nongnuch Sirachainan, Pimpun Kitpoka, Monratta Pitakcharoen, Suphaneewan Jaovisidha, Panyu Panburana, Monthon Suwannuraks, Nantana Sri-Udomporn, Chusak Kijkunasathian, Rungrote Nateserinilkul, Bunchoo Pongtanakul, Chanchai Traivaree, Patcharee Komvilaisak, Darintr Sosothikul, Yujinda Lektrakul, Thunyakorn Nuntiyagul, Pacharapan Surapolchai, Natsaruth Songthawee, Ekawat Suwantaroj, Pantep Angchaisuksiri, Theera Ruchutrakul, Ponlapat Rojnuckarin

{"title":"Five decades of successive establishment of hemophilia care in Thailand.","authors":"Ampaiwan Chuansumrit, Thip Sriphaisal, Triroj Krutvecho, Nongnuch Sirachainan, Pimpun Kitpoka, Monratta Pitakcharoen, Suphaneewan Jaovisidha, Panyu Panburana, Monthon Suwannuraks, Nantana Sri-Udomporn, Chusak Kijkunasathian, Rungrote Nateserinilkul, Bunchoo Pongtanakul, Chanchai Traivaree, Patcharee Komvilaisak, Darintr Sosothikul, Yujinda Lektrakul, Thunyakorn Nuntiyagul, Pacharapan Surapolchai, Natsaruth Songthawee, Ekawat Suwantaroj, Pantep Angchaisuksiri, Theera Ruchutrakul, Ponlapat Rojnuckarin","doi":"10.1080/17474086.2025.2486378","DOIUrl":"10.1080/17474086.2025.2486378","url":null,"abstract":"Introduction: Management of patients with hemophilia in healthcare resource limited countries is challenging for healthcare providers.Area covered: Thai patients with hemophilia initially received episodic treatment of cryoprecipitate and fresh frozen plasma (FFP) in 1965. Home treatment using cryoprecipitate at early bleeding episodes was initiated in 1979 and replaced with fresh dry plasma in 1982. Heat-treated, lyophilized cryoprecipitate commenced in 1997, and factor concentrate was imported for clinical use in 2000. A healthy diet, limit weight gain and exercise for the prominent vein constitute a commitment among every hemophiliac. National advocacy of home treatment using factor concentrates at early bleeding episodes started in 2006 and nationwide low-dose prophylaxis in 2018. Immune tolerance induction (ITI) was initially attempted in 1997. The dental splint was invented as a local measure for dental procedures in 1979, and fibrin glue has been applied to surgical sites. A computerized program for hereditary bleeding disorders registry was initiated in 1999 and became nationwide in 2020. Finally, nationwide nonfactor therapy of monthly low-dose emicizumab prophylaxis will be established soon.Expert opinion: Hemophilia care in Thailand constitutes the real-world experience passing from generation to generation for five decades using our limited resources efficiently.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-13"},"PeriodicalIF":2.3,"publicationDate":"2025-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751885","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

Oral decitabine in acute myeloid leukemia: assessing efficacy, safety, and future implications for older patients.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-04-02 DOI: 10.1080/17474086.2025.2487605

Pasquale Niscola

{"title":"Oral decitabine in acute myeloid leukemia: assessing efficacy, safety, and future implications for older patients.","authors":"Pasquale Niscola","doi":"10.1080/17474086.2025.2487605","DOIUrl":"10.1080/17474086.2025.2487605","url":null,"abstract":"Introduction: Older patients with acute myeloid leukemia (AML) are often unsuitable for standard treatments and traditionally have a dismal prognosis. For 20 years, hypomethylating agents (HMAs), as single agents and recently as a backbone with venetoclax, have been used in this setting. The oral combination of decitabine and cedazuridine (C-DEC), which is therapeutically and pharmacologically equivalent to the intravenous (IV) formulation (IV-DEC), has expanded the therapeutic arsenal for AML, allowing for better convenience of administration. This review provides an overview of C-DEC, current clinical applications, and ongoing clinical studies, highlighting its potential role in managing AML in older patients.Areas covered: For the aim of this review, the authors searched the current English literature on C-DEC in AML patients through PubMed, using several pertinent keywords. To summarize the most recent developments as up-to-date as possible, we considered, with a few exceptions, only papers published over the last three years until March 2025.Expert opinion: Compared to IV-DEC, C-DEC provides similar therapeutic exposure and efficacy with an optional safety profile, enhanced treatment adherence, reduced treatment burden, and required healthcare facilities compared to those associated with IV-DEC, providing a precious tool to manage older and unfit AML patients.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-9"},"PeriodicalIF":2.3,"publicationDate":"2025-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751836","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

What is the importance of monitoring iron levels in different organs over time with magnetic resonance imaging in transfusion-dependent thalassemia patients?

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-03-31 DOI: 10.1080/17474086.2025.2486379

Antonella Meloni, Vincenzo Positano, Paolo Ricchi, Alessia Pepe, Riccardo Cau

{"title":"What is the importance of monitoring iron levels in different organs over time with magnetic resonance imaging in transfusion-dependent thalassemia patients?","authors":"Antonella Meloni, Vincenzo Positano, Paolo Ricchi, Alessia Pepe, Riccardo Cau","doi":"10.1080/17474086.2025.2486379","DOIUrl":"10.1080/17474086.2025.2486379","url":null,"abstract":"Introduction: Iron overload is the main pathophysiological driver of organ damage in transfusion-dependent thalassemia (TDT). Magnetic resonance imaging (MRI) provides detailed insights into the distribution and severity of iron accumulation in the different organs.Areas covered: This special report describes the impact of MRI on clinical and therapeutic management and short- and long-term outcomes in TDT patients. PubMed, Scopus, and Google Scholar databases were searched to identify the relevant studies published before November 2024.Expert opinion: Cardiac and hepatic MRI are now well-established modalities, integrated into the clinical practice. They have become essential for tailoring iron chelation therapies to the specific patient's needs and for monitoring treatment efficacy. The improved control of cardiac iron burden has translated into reduced morbidity and mortality. The MRI accessibility remains limited in resource-limited settings and progress in this field relies on educating and training centers to ensure accurate execution and interpretation. The clinicopathological significance, prognostic value, and reproducibility of pancreatic iron levels assessment have been established, charting a path toward its clinical use. There are limited data about renal, adrenal, and pituitary iron deposition, and more research is needed to fully establish the functional significance and to standardize and validate the MRI protocols.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-9"},"PeriodicalIF":2.3,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143729649","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

Anemia and its predictors among patients on hemodialysis, at Felege Hiote Referral Hospital, Northwest Ethiopia, 2022: a retrospective cohort study.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-02-01 Epub Date: 2025-01-29 DOI: 10.1080/17474086.2025.2458272

Mihretie Gedfew, Addisu Getie

{"title":"Anemia and its predictors among patients on hemodialysis, at Felege Hiote Referral Hospital, Northwest Ethiopia, 2022: a retrospective cohort study.","authors":"Mihretie Gedfew, Addisu Getie","doi":"10.1080/17474086.2025.2458272","DOIUrl":"10.1080/17474086.2025.2458272","url":null,"abstract":"Background: Anemia is a critical complication among hemodialysis patients, often leading to poor outcomes. This study aimed to assess anemia prevalence and identify predictors among hemodialysis patients at Felege Hiote Referral Hospital, Northwest Ethiopia, in 2022.Research design and methods: A retrospective cohort study analyzed 410 hemodialysis patients from January 2018 to February 2022. Data was collected from medical records, entered using Epi-data Version 4.1, and analyzed with STATA Version 14. Kaplan-Meier survival curves assessed survival time, while Cox regression identified anemia predictors.Results: Of 410 patients, 66 (16.1%) developed anemia, with an incidence rate of 7.9 per 100 person-years (95% CI: 3.1-13.5). Significant predictors included female sex (IRR: 1.5, p = 0.04), rural residence (IRR: 2.5, p = 0.01), low body mass index (IRR: 1.6, p = 0.02), and congestive heart failure (IRR: 6.9, p = 0.02).Conclusions: Anemia prevalence among hemodialysis patients is high, with key predictors including gender, residence, body mass index, and comorbidities. Interventions targeting these factors, especially in rural areas, could reduce anemia incidence. Study limitations include single-center data, incomplete variables, and lack of causality.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"143-153"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143046051","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

Platelet indices in preeclampsia: comparative analysis with normotensive pregnant women.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-02-01 Epub Date: 2025-01-30 DOI: 10.1080/17474086.2025.2458262

Hamza Tariq, Muhammad Hamza Khan, Fnu Poombal, Muhammad Saad Khan, Minahil Mateen Ahmad, Mehreen Khalid, Umera Saleem

{"title":"Platelet indices in preeclampsia: comparative analysis with normotensive pregnant women.","authors":"Hamza Tariq, Muhammad Hamza Khan, Fnu Poombal, Muhammad Saad Khan, Minahil Mateen Ahmad, Mehreen Khalid, Umera Saleem","doi":"10.1080/17474086.2025.2458262","DOIUrl":"10.1080/17474086.2025.2458262","url":null,"abstract":"Background: To compare platelet count (PC), mean platelet volume (MPV), and platelet distribution width (PDW) between women with preeclampsia (PE) and normotensive pregnant women, and evaluate their effectiveness as predictors of PE.Research design and methods: This cross-sectional study at Nishtar Hospital, Multan, included 141 women: 74 normotensive and 67 preeclamptic. Data was collected using an automated hematology analyzer and analyzed with SPSS version 26 and ROC curves.Results: Mean age was 27.45 ± 5.18 years for cases and 28.41 ± 5.28 years for controls (p = 0.280). Gestational age was lower in the preeclamptic group (31.97 ± 4.07 weeks) compared to controls (33.92 ± 3.30 weeks) (p = 0.002). Blood pressures were higher in preeclamptic women (p < 0.001). Platelet count was lower in preeclamptic women (183.42 ± 95.69) vs. controls (256.42 ± 77.98) (p < 0.001). MPV (10.98 ± 1.55 vs. 9.79 ± 1.59, p < 0.001) and PDW (16.82 ± 5.70vs. 14.20 ± 2.40, p < 0.001) were higher in preeclamptic women. ROC analysis showed PDW had an AUC of 0.73 and MPV an AUC of 0.71.Conclusions: PDW and MPV are significantly altered in preeclamptic women and can aid in early detection, potentially enhancing management.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"135-142"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143037784","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

Sports practice should be supported in people with severe hemophilia, but patient counseling and tailoring prophylactic treatment with clotting factors and non-replacement therapy is essential.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-02-01 Epub Date: 2025-01-27 DOI: 10.1080/17474086.2025.2459271

Emerito Carlos Rodriguez-Merchan, Hortensia De la Corte-Rodriguez

{"title":"Sports practice should be supported in people with severe hemophilia, but patient counseling and tailoring prophylactic treatment with clotting factors and non-replacement therapy is essential.","authors":"Emerito Carlos Rodriguez-Merchan, Hortensia De la Corte-Rodriguez","doi":"10.1080/17474086.2025.2459271","DOIUrl":"10.1080/17474086.2025.2459271","url":null,"abstract":"Introduction: This article discusses the current role of sports practice in people with hemophilia (PWH).Areas covered: On 11 January 2025, a bibliographic search was carried out in PubMed using 'hemophilia sports' as keywords. A total of 411 articles were found, of which only 22 were finally analyzed because they were directly related to the title of this article (inclusion criterion). The remaining 389 were eliminated because they were not directly related to the title of the article (exclusion criterion). It was found that sports activity is essential for patients with severe hemophilia to maintain joints' range of motion, diminish joint bleeding, enhance muscle mass and strength, improve proprioception, muscular trophism and bone mineral density, and avert secondary joint degeneration. PWH with factor levels < 10% during sports had a bleeding risk of 41% versus 20% in those with higher factor levels (>10%).Expert opinion: Prophylaxis therapy can permit patients with severe hemophilia to participate in vigorous activities or high-impact sports. Moreover, such activities or sports have a positive effect on patient's psychosocial health. Therefore, sports practice should be supported in people with severe hemophilia but patient counseling and tailoring prophylaxis therapy with clotting factors and non-replacement therapy is essential.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"123-134"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143032782","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

The future of siRNA-mediated approaches to treat von Willebrand disease.

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-02-01 Epub Date: 2025-01-29 DOI: 10.1080/17474086.2025.2459259

Noa A Linthorst, Bart J M van Vlijmen, Jeroen C J Eikenboom

{"title":"The future of siRNA-mediated approaches to treat von Willebrand disease.","authors":"Noa A Linthorst, Bart J M van Vlijmen, Jeroen C J Eikenboom","doi":"10.1080/17474086.2025.2459259","DOIUrl":"10.1080/17474086.2025.2459259","url":null,"abstract":"Introduction: The clinical management of the inherited bleeding disorder von Willebrand disease (VWD) focuses on normalizing circulating levels of von Willebrand factor (VWF) and factor VIII (FVIII) to prevent or control bleeding events. The heterogeneous nature of VWD, however, complicates effective disease management and development of universal treatment guidelines.Areas covered: The current treatment modalities of VWD and their limitations are described and why this prompts the development of new treatment approaches. In particular, RNA-based therapeutics have gained significant interest because of their ability to reversibly alter gene expression with long-term efficacy. In the field of VWD, small-interfering RNAs (siRNAs) have been explored through various strategies to improve disease phenotypes. These different approaches are discussed as well as their potential impact on reshaping the future therapeutic landscape.Expert opinion: Current treatments for VWD often require frequent intravenous administration of VWF concentrates or desmopressin, with only short-term benefits. Moreover, remaining circulating mutant VWF can cause detrimental effects. Allele-selective siRNA-based therapies could provide more reliable and long-term disease correction by specifically targeting mutant VWF. This approach could be applied to a large part of the population aligning with the growing emphasis on personalized treatment and patient-centered care in VWD management.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"109-122"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11854048/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143046154","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0