Vox Sanguinis最新文献

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Management of massive haemorrhage in transfusion medicine services in the Middle East and North Africa 中东和北非输血医疗服务机构对大出血的管理
IF 2.7 4区 医学
Vox Sanguinis Pub Date : 2024-06-20 DOI: 10.1111/vox.13701
Arwa Z. Al‐Riyami, Suha Hejres, Sanaa Abd Elshafy, Hind Al Humaidan, Hanady Samaha
{"title":"Management of massive haemorrhage in transfusion medicine services in the Middle East and North Africa","authors":"Arwa Z. Al‐Riyami, Suha Hejres, Sanaa Abd Elshafy, Hind Al Humaidan, Hanady Samaha","doi":"10.1111/vox.13701","DOIUrl":"https://doi.org/10.1111/vox.13701","url":null,"abstract":"Background and ObjectivesMassive transfusion protocols (MTPs) are critical in managing haemorrhage, yet their utilization varies. There is lack of data on the utilization of MTPs in the Middle East and North Africa (MENA) region. This study aims to assess the degree of utilization of MTPs in the region.Materials and MethodsWe conducted a survey to collect data on MTP use, inviting medical directors of transfusion services from various hospitals. Data were analysed to determine the prevalence of MTP utilization, their compositions, challenges in application and areas of future need.ResultsEighteen respondents participated, representing 11 countries in the region. Thirteen hospitals implemented MTP, and eight included paediatrics. Eleven institutions used more than one definition of massive haemorrhage, with the most common being ≥10 red blood cell (RBC) units transfused for adults and replacement of >50% total blood volume in paediatrics. The majority of sites with MTPs utilized 1:1:1 RBCs:platelets:plasma ratio (70%). Variations were observed in the types and blood groups of components used. Two sites utilized whole blood, while six are considering it for future use. Utilization of adjunctive agents and frequency of laboratory testing varied among the sites. Challenges included the lack of medical expertise in protocol development, adherence and paediatric application. The need assessment emphasized the need for developing regional guidelines, standardized protocols and training initiatives.ConclusionAlthough several hospitals have adopted MTPs, variations exist in activation criteria, blood product ratios and monitoring. Challenges include the lack of medical expertise, protocol adherence and addressing paediatric needs. Standardizing protocols, enhancing training and paediatric application are crucial for improving massive transfusion management in the region.","PeriodicalId":23631,"journal":{"name":"Vox Sanguinis","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141502249","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
Limited evidence, lasting decisions: How voluntary non-remunerated plasma donations can avoid the commercial one-way street. 有限的证据,持久的决定:自愿无偿捐献血浆如何避免商业单行道。
IF 1.8 4区 医学
Vox Sanguinis Pub Date : 2024-06-01 Epub Date: 2024-03-14 DOI: 10.1111/vox.13613
Kelsey J MacKay, Fritz Schiltz, Philippe Vandekerckhove
{"title":"Limited evidence, lasting decisions: How voluntary non-remunerated plasma donations can avoid the commercial one-way street.","authors":"Kelsey J MacKay, Fritz Schiltz, Philippe Vandekerckhove","doi":"10.1111/vox.13613","DOIUrl":"10.1111/vox.13613","url":null,"abstract":"","PeriodicalId":23631,"journal":{"name":"Vox Sanguinis","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140120758","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
Transfusion Camp Rwanda 2023: A train-the-trainer workshop establishing locally driven leadership in knowledge translation and sustainability in transfusion medicine education. 2023 年卢旺达输血营:输血医学教育中的知识转化和可持续发展方面建立本地驱动领导力的培训师培训讲习班。
IF 2.7 4区 医学
Vox Sanguinis Pub Date : 2024-06-01 Epub Date: 2024-02-29 DOI: 10.1111/vox.13611
Francoise Nizeyimana, Jacob Pendergrast, Angelique Ntegerejuwampayee, Yulia Lin, Aimable Kanyamuhunga, Christopher Gashaija, Florence Masaisa, Claudine Uzamukunda, Grace Mutuyimana, Fabrice Ndicunguye, Alexis Gaseminari, Bruce Mulindwa, Thomas Muyombo, Sophie Chargé, Teresa Skelton
{"title":"Transfusion Camp Rwanda 2023: A train-the-trainer workshop establishing locally driven leadership in knowledge translation and sustainability in transfusion medicine education.","authors":"Francoise Nizeyimana, Jacob Pendergrast, Angelique Ntegerejuwampayee, Yulia Lin, Aimable Kanyamuhunga, Christopher Gashaija, Florence Masaisa, Claudine Uzamukunda, Grace Mutuyimana, Fabrice Ndicunguye, Alexis Gaseminari, Bruce Mulindwa, Thomas Muyombo, Sophie Chargé, Teresa Skelton","doi":"10.1111/vox.13611","DOIUrl":"10.1111/vox.13611","url":null,"abstract":"<p><strong>Background and objectives: </strong>Blood transfusion is performed daily in hospitals. Gaps exist between transfusion guidelines and day-to-day clinical care. These gaps are prevalent in resource-limited settings due to scarce continuing medical education. Transfusion Camp Rwanda aims to bridge this gap by (1) delivering context-appropriate up-to-date education, (2) teaching participants how to independently deliver a case-based curriculum and (3) identifying strategies to promote change in transfusion practice in Rwanda.</p><p><strong>Materials and methods: </strong>In May 2023, a multidisciplinary team from Canada and Rwanda carried out a Transfusion Camp train-the-trainer workshop for clinicians from all five provinces in Rwanda. Participants attended in-person lectures, seminars and workshop group discussions on the implementation of the Rwanda National Directives on Rational Use of Blood and Blood Components. Course feedback was based on the Kirkpatrick Model of Training and Evaluation.</p><p><strong>Results: </strong>Fifty-one physicians and laboratory technicians participated in the course. Confidence in caring for patients based on transfusion guidelines was self-rated as 'excellent' by 23% of participants before and 77% after, while 84% reported they planned to teach Transfusion Camp to others and 100% responded that they will apply course content to clinical practice. Workshop groups recommended strategies to improve transfusion medicine practice in Rwanda in four domains: Communication, Institutional Approval, Practice Audits and Education.</p><p><strong>Conclusion: </strong>Transfusion medicine education in Rwanda using a train-the-trainer approach was well-received by participants and allowed for a more detailed understanding of the local medical and educational environment. These observations can inform the further expansion of the Transfusion Camp Rwanda project.</p>","PeriodicalId":23631,"journal":{"name":"Vox Sanguinis","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139997615","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
Has the frequency of ABO RhD blood groups in Australian blood donors changed as a result of the removal of the variant Creutzfeldt-Jakob disease-based deferral? 澳大利亚献血者中 ABO RhD 血型的频率是否因取消基于变异克雅氏症的延迟而发生变化?
IF 1.8 4区 医学
Vox Sanguinis Pub Date : 2024-06-01 Epub Date: 2024-03-07 DOI: 10.1111/vox.13614
Rena Hirani, Veronica C Hoad, Iain B Gosbell, David O Irving
{"title":"Has the frequency of ABO RhD blood groups in Australian blood donors changed as a result of the removal of the variant Creutzfeldt-Jakob disease-based deferral?","authors":"Rena Hirani, Veronica C Hoad, Iain B Gosbell, David O Irving","doi":"10.1111/vox.13614","DOIUrl":"10.1111/vox.13614","url":null,"abstract":"<p><strong>Background and objectives: </strong>Until 25 July 2022, Australians who had spent more than 6 months in the United Kingdom or territories between 1980 and 1996 were deferred from blood donation due to the risk of variant Creutzfeldt-Jakob disease. Removal of this geography-based donor deferral on RhD-negative blood availability has not been reported.</p><p><strong>Materials and methods: </strong>All donors who donated at least once from 25 July 2022 to 25 July 2023 were included. UK donor status, first-time donor and ABO RhD data were extracted from the National Blood Management System.</p><p><strong>Results: </strong>Data from 566,447 blood donors with a valid ABO RhD result were analysed. Of these, 34,560 were new or returning lapsed donors following removal of the UK donor deferral. The median age [range] in years for all donors was 43 [75] with UK donors being older 53 [70]. There was a higher prevalence of RhD-negative status in UK donors (20.2%) compared with first-time blood donors (15.7%).</p><p><strong>Conclusion: </strong>UK donors were generally older, female and more likely to be RhD-negative. Although UK donors provided a boost to RhD-negative blood collections, the overall prevalence of ABO RhD blood groups in the total Australian blood donor panel remained similar to previous estimates.</p>","PeriodicalId":23631,"journal":{"name":"Vox Sanguinis","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140050410","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
Routine results of an algorithm for managing the production of blood components. 血液成分生产管理算法的常规结果。
IF 2.7 4区 医学
Vox Sanguinis Pub Date : 2024-06-01 Epub Date: 2024-02-27 DOI: 10.1111/vox.13609
Ana Isabel Pérez-Aliaga, Irene Ayerra, Javier Sánchez-Guillén, F Javier López, Fernando Puente, Alfonso Aranda, José María Domingo, Carmen Garcés
{"title":"Routine results of an algorithm for managing the production of blood components.","authors":"Ana Isabel Pérez-Aliaga, Irene Ayerra, Javier Sánchez-Guillén, F Javier López, Fernando Puente, Alfonso Aranda, José María Domingo, Carmen Garcés","doi":"10.1111/vox.13609","DOIUrl":"10.1111/vox.13609","url":null,"abstract":"<p><strong>Background and objectives: </strong>The variability in the number of donations together with a growing demand for platelet concentrates and plasma-derived medicines make us seek solutions aimed at optimizing the processing of blood. Some mathematical models to improve efficiencies in blood banking have been published. The goal of this work is to validate and evaluate an algorithm's impact in the production of blood components in the Blood and Tissues Bank of Aragon (BTBA).</p><p><strong>Materials and methods: </strong>A mathematical algorithm was designed, implemented and validated through simulations with real data. It was incorporated into the fractionation area, which uses the Reveos® fractionation system (Terumo BCT) to split blood into its components. After 9 months of daily routine validation, retrospective activity data from the Blood Bank and Transfusion Services before and during the use of the algorithm were compared.</p><p><strong>Results: </strong>Using the algorithm, the outdating rate of platelet concentrates (PC) decreased by 87.8% in the blood bank. The average shelf life remaining of PC supplied to Transfusion Services increased by almost 1 day. As a consequence, the outdating rate in the Aragon Transfusion Network decreased by 33%. In addition, extra 100 litres of plasma were obtained in 9 months.</p><p><strong>Conclusions: </strong>The algorithm improves the blood establishment's workflow and facilitates the decision-making process in whole blood processing. It resulted in a decrease in PC outdating rate, increase in PC shelf life and finally an increase in the volume of recovered plasma, leading to significant cost savings.</p>","PeriodicalId":23631,"journal":{"name":"Vox Sanguinis","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139973703","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
Stigmatizing deferrals disproportionally reduce donor return rates: Evidence from Brazil. 污名化延期会不成比例地降低捐赠者的回报率:来自巴西的证据。
IF 2.7 4区 医学
Vox Sanguinis Pub Date : 2024-06-01 Epub Date: 2024-02-29 DOI: 10.1111/vox.13607
Yan Vieites, Claudio M Ferreira, Rafael Goldszmidt, Eduardo B Andrade
{"title":"Stigmatizing deferrals disproportionally reduce donor return rates: Evidence from Brazil.","authors":"Yan Vieites, Claudio M Ferreira, Rafael Goldszmidt, Eduardo B Andrade","doi":"10.1111/vox.13607","DOIUrl":"10.1111/vox.13607","url":null,"abstract":"<p><strong>Background and objectives: </strong>Prior research has shown that temporary deferrals negatively influence donor return rates, but it remains unknown the extent to which these effects vary across reasons for deferral. We investigate whether deferrals differ in their degree of perceived stigmatization and, if so, how being deferred for stigmatizing (vs. non-stigmatizing) reasons affects subsequent donation behaviour.</p><p><strong>Materials and methods: </strong>We examined whether reasons for deferral vary on their perceived level of stigmatization through an online survey (n = 400). Furthermore, we used a dataset encompassing 25 years of donation records from the state-run blood collection agency (BCA) from Rio de Janeiro, Brazil, to investigate how stigmatizing (vs. non-stigmatizing) reasons for deferral affected return rates of 82,648 donors over a 60-month follow-up period.</p><p><strong>Results: </strong>Being deferred for sex- and drug-related reasons was perceived as much more stigmatizing than other reasons for deferral (odds ratio = 3.14, 95% confidence interval [CI]: 2.33-4.25). Controlling for multiple observables, prospective donors were less likely to return to the BCA when deferred for stigmatizing (vs. non-stigmatizing) reasons (adjusted hazard ratio = 0.87, 95% CI: 0.83-0.93).</p><p><strong>Conclusion: </strong>Donors perceive deferrals motivated by sex- and drug-related reasons as particularly stigmatizing, which is negatively associated with donor return rates. BCAs may want to pay special attention when communicating stigmatizing reasons for deferral to prospective donors.</p>","PeriodicalId":23631,"journal":{"name":"Vox Sanguinis","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139997614","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
Abstracts of the 38th International Congress of the ISBT, Barcelona, Spain, 23-27 June 2024. 2024 年 6 月 23 日至 27 日在西班牙巴塞罗那举行的第 38 届 ISBT 国际大会摘要。
IF 1.8 4区 医学
Vox Sanguinis Pub Date : 2024-06-01 DOI: 10.1111/vox.13652
{"title":"Abstracts of the 38th International Congress of the ISBT, Barcelona, Spain, 23-27 June 2024.","authors":"","doi":"10.1111/vox.13652","DOIUrl":"https://doi.org/10.1111/vox.13652","url":null,"abstract":"","PeriodicalId":23631,"journal":{"name":"Vox Sanguinis","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141459533","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
Decreasing parvovirus B19 and hepatitis A nucleic acid test positivity rates in Canadian plasma donors following the initiation of COVID-19 restriction in March 2020. 2020 年 3 月开始限制 COVID-19 之后,加拿大血浆捐献者的副病毒 B19 和甲型肝炎核酸检测阳性率下降。
IF 2.7 4区 医学
Vox Sanguinis Pub Date : 2024-06-01 Epub Date: 2024-03-14 DOI: 10.1111/vox.13616
Steven J Drews, Carmen Charlton, Sheila F O'Brien, Samantha Burugu, Gregory A Denomme
{"title":"Decreasing parvovirus B19 and hepatitis A nucleic acid test positivity rates in Canadian plasma donors following the initiation of COVID-19 restriction in March 2020.","authors":"Steven J Drews, Carmen Charlton, Sheila F O'Brien, Samantha Burugu, Gregory A Denomme","doi":"10.1111/vox.13616","DOIUrl":"10.1111/vox.13616","url":null,"abstract":"<p><strong>Background and objectives: </strong>In Canada, plasma sent for fractionation is tested for both parvovirus B19 (B19V) and hepatitis A virus (HAV). This study compared positivity rates of B19 and HAV nucleic acid tests (NATs) in Canadian plasma samples for the pre-COVID-19 restriction era (2015 to end of February 2020 [Q1] 2020) and the post-COVID-19 restriction era.</p><p><strong>Materials and methods: </strong>Pooled EDTA plasma specimens were tested within 24 months of blood draw using the Procleix Panther System (Grifols Diagnostic Solutions Inc, San Diego, CA, USA) for B19V and HAV detection. Reactive pools were resolved by individual specimen testing.</p><p><strong>Results: </strong>Between 1 January 2015, and 31 March 2022, 3,928,619 specimens from Canadian plasma donors were tested for B19V. For the same period, 3,922,954 specimens were tested for HAV. To account for a lag in specimen testing for up to 24 months, the data were divided into: (1) a pre-pandemic period (1 January 2015-31 March 2020; B19V tested n = 2,412,701, B19V NAT-positive n = 240 [0.01%], HAV tested n = 2,407,036, HAV NAT-positive n = 26 [0.001%]); (2) a two-year mixed-impact period (1 April 2020-31 March 2022; B19V tested n = 968,250, B19V NAT-positive n = 14 [0.001%], HAV tested n = 968,250, HAV NAT-positive n = 2 [0.0002%]); and (3) a pandemic-impact period (1 April 2022-31 March, 2023; B19V tested n = 597,668, B19V NAT-positive n = 3 [0.0005%], HAV tested n = 597,668, HAV NAT-positive n = 1 [0.0002%]).</p><p><strong>Conclusion: </strong>The percentage of B19V- and HAV-positive donations was significantly reduced from the pre-pandemic period to the pandemic-impact period.</p>","PeriodicalId":23631,"journal":{"name":"Vox Sanguinis","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140120757","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
Evaluation of new haematology analyser, XN-31, for malaria detection in blood donors: A single-centre study from India. 新型血液分析仪 XN-31 用于献血者疟疾检测的评估:印度的一项单中心研究。
IF 2.7 4区 医学
Vox Sanguinis Pub Date : 2024-06-01 Epub Date: 2024-03-24 DOI: 10.1111/vox.13621
Aseem Kumar Tiwari, Shalini Goel, Ganesh Singh, Pawan Kumar Gahlot, Renu Saxena, Vaibhav Jadhav, Monisha Sethi
{"title":"Evaluation of new haematology analyser, XN-31, for malaria detection in blood donors: A single-centre study from India.","authors":"Aseem Kumar Tiwari, Shalini Goel, Ganesh Singh, Pawan Kumar Gahlot, Renu Saxena, Vaibhav Jadhav, Monisha Sethi","doi":"10.1111/vox.13621","DOIUrl":"10.1111/vox.13621","url":null,"abstract":"<p><strong>Background and objectives: </strong>Malaria continues to be a significant public health concern in India, with several regions experiencing endemicity and sporadic outbreaks. The prevalence of malaria in blood donors, in India, varies between 0.02% and 0.07%. Common techniques to screen for malaria, in blood donors and patients, include microscopic smear examination and rapid diagnostic tests (RDTs) based on antigen detection. The aim of this study was to evaluate a new fully automated analyser, XN-31, for malaria detection, as compared with current practice of using RDT.</p><p><strong>Materials and methods: </strong>Cross-sectional analytical study was conducted to evaluate clinical sensitivity and specificity of new automated analyser XN-31 among blood donors' samples and clinical samples (patients with suspicion of malaria) from outpatient clinic collected over between July 2021 and October 2022. No additional sample was drawn from blood donor or patient. All blood donors and patients' samples were processed by malaria rapid diagnostic test, thick-smear microscopy (MIC) and the haematology analyser XN-31. Any donor blood unit incriminated for malaria was discarded. Laboratory diagnosis using MIC was considered the 'gold standard' in the present study. Clinical sensitivity and specificity of XN-31 were compared with the gold standard.</p><p><strong>Results: </strong>Fife thousand and five donor samples and 82 diagnostic samples were evaluated. While the clinical sensitivity and specificity for donor samples were 100%, they were 72.7% and 100% for diagnostic samples.</p><p><strong>Conclusion: </strong>Automated haematology analysers represent a promising solution, as they can deliver speedy and sensitive donor malaria screening assessments. This method also has the potential to be used for pre-transfusion malaria screening along with haemoglobin estimation.</p>","PeriodicalId":23631,"journal":{"name":"Vox Sanguinis","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140207687","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
Current state of gene therapy in sickle cell disease. 镰状细胞病的基因治疗现状。
IF 2.7 4区 医学
Vox Sanguinis Pub Date : 2024-06-01 Epub Date: 2024-03-15 DOI: 10.1111/vox.13612
Mei San Tang, Hua Shan
{"title":"Current state of gene therapy in sickle cell disease.","authors":"Mei San Tang, Hua Shan","doi":"10.1111/vox.13612","DOIUrl":"10.1111/vox.13612","url":null,"abstract":"<p><p>Sickle cell disease (SCD) is a type of hemoglobinopathy due to an autosomal recessive genetic defect, causing significant red cell sickling, multi-organ damage and long-term severe morbidities. Due to its complicated care and the impact on quality of life, a curative treatment for SCD is highly desirable. In recent years, gene therapy is emerging as a curative option for SCD, where autologous haematopoietic stem cells are collected from SCD patients and genetically modified ex vivo to reduce its sickling tendency before reinfusion. Although still largely investigational, a limited number of gene therapy options have been recently granted approval for SCD patients. Published data are still currently limited, but early studies have so far demonstrated the intended outcomes of less vaso-occlusive crisis and haemolysis. Nonetheless, despite its curative potential, larger clinical trials and longer follow-up period are still necessary to evaluate the safety of this treatment option, especially the risk of unintended genetic modifications. Furthermore, SCD patients frequently have limited access to specialty care; hence, the issues of affordability and accessibility to SCD gene therapy must also be addressed for it to benefit the appropriate patient population.</p>","PeriodicalId":23631,"journal":{"name":"Vox Sanguinis","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140132707","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
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