Hematology, Transfusion and Cell Therapy最新文献

{"title":"Validation of an automated quality control method to test sterility of two advanced therapy medicinal products: Mesenchymal stromal cells and their extracellular vesicles","authors":"Carolina Kymie Vasques Nonaka ,&nbsp;Zaquer Suzana Munhoz Costa-Ferro ,&nbsp;Ana Carolina Palmeira Arraes ,&nbsp;Thamires Lopes Weber ,&nbsp;Luciana Souza de Aragão França ,&nbsp;Katia Nunes Silva ,&nbsp;Bruno Solano de Freitas Souza","doi":"10.1016/j.htct.2024.09.2486","DOIUrl":"10.1016/j.htct.2024.09.2486","url":null,"abstract":"<div><div>Mesenchymal stromal cells are multipotent cells present in various tissues that are widely studied for relevant therapeutic potential due to their paracrine immunomodulatory and tissue regenerating properties. Many mesenchymal stromal cell-based products are under investigation for the treatment of different clinical conditions. Recently, the therapeutic potential of the extracellular vesicles released by these cells has been under focus, with emphasis on clinical translation. Sterility testing during manufacture and before the final release of the advanced therapy medicinal products to markets is a critical quality control measure. Therefore, analytical methods for sterility testing in addition to complying with pharmacopeial standards must validate the adequacy of each product and evaluate matrix interference. Here, an automated system for sterility control of reagents used in the bioprocessing of mesenchymal stromal cells and their extracellular vesicles was validated. Reagents (culture media, antibiotics, and excipients in the final product) were inoculated with 10 or 50 colony forming units of microorganisms in BACTEC™ Peds Plus™ T/F aerobic/anaerobic bottles. Under aerobic conditions (BACTEC™ Peds Plus™ T/F aerobic bottles), microbial growth was detected within an acceptable incubation time according to regulatory guidelines. The results of this study corroborate other studies that use automated sterility testing as an alternative to the manual USP&lt;71&gt; compendial method to detect microorganisms close to the limit of detection within an acceptable incubation time.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103727"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143043894","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Imetelstat as a novel therapeutic approach for myelodysplastic syndrome","authors":"Ammara Waheed Arain,&nbsp;Anushka Andani,&nbsp;Aayat Kashif,&nbsp;Radeyah Waseem","doi":"10.1016/j.htct.2024.09.2484","DOIUrl":"10.1016/j.htct.2024.09.2484","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103724"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143378708","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Complex somatic mutation landscape in myeloid cells in a patient with VEXAS syndrome: First Brazilian case report","authors":"Fabíola Reis de Oliveira ,&nbsp;Adriane Souza Lima ,&nbsp;Carlos Roberto Faria Jr ,&nbsp;Thaise Oliveira Quaresma ,&nbsp;Marcio M. Mourani ,&nbsp;Lauro Wichert-Ana ,&nbsp;Paulo Louzada Jr ,&nbsp;Fernanda Gutierrez-Rodrigues ,&nbsp;Neal S. Young ,&nbsp;Rodrigo T. Calado","doi":"10.1016/j.htct.2024.05.013","DOIUrl":"10.1016/j.htct.2024.05.013","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103686"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304626","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ponatinib in the treatment of patients with chronic myeloid leukemia and increased cardiovascular risk: A review of management strategies","authors":"Tomasz Sacha,&nbsp;Katarzyna Krawczyk","doi":"10.1016/j.htct.2024.04.124","DOIUrl":"10.1016/j.htct.2024.04.124","url":null,"abstract":"<div><div>The introduction of tyrosine kinase inhibitors has revolutionized the treatment of chronic myeloid leukemia vastly improving the prognosis and clinical outcome of most patients. It was estimated that approximately 40–50 % of patients treated with imatinib will require treatment with a second-generation or third-generation tyrosine kinase inhibitor to achieve an optimal response. The treatment duration, increased patient survival, and aging of the population receiving tyrosine kinase inhibitors raise concerns as to long-term toxicities, such as an elevated cardiovascular risk and a higher rate of comorbidities. Ponatinib is a highly potent third-generation tyrosine kinase inhibitor that was shown to be effective in patients with a wide range of ABL mutations, including T315I. The use of ponatinib is associated with significant vascular toxicity, including peripheral arterial occlusive disease, ischemic heart disease, cerebrovascular accidents, and venous thromboembolism. This review discusses the vascular toxicity of ponatinib and presents a comprehensive panel of tests for the evaluation of patients requiring ponatinib therapy. Moreover, the management of patients with cardiovascular risk factors who receive ponatinib is discussed. Finally, the strategy for establishing the optimal dose of ponatinib in patients with chronic myeloid leukemia is described.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103675"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142515299","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Transmitted blood infections and emerging vector-borne diseases in blood donors in northern Portugal","authors":"Ana Mota ,&nbsp;Margarida Fonseca Cardoso","doi":"10.1016/j.htct.2025.103734","DOIUrl":"10.1016/j.htct.2025.103734","url":null,"abstract":"<div><h3>Background</h3><div>Screening of transfusion-transmissible infectious agents of blood components is carried out in order to guarantee the safety of the transfusion process. The objective of this investigation was to characterize cases positive for transfusion-transmissible infectious agents in blood donations in the North of Portugal.</div></div><div><h3>Method</h3><div>Data from 2010 to 2022 of the Local Health Unit-Santo Antonio were used for this study. In specific epidemiological situations, malaria, Chagas disease and West Nile virus were screened.</div></div><div><h3>Main results</h3><div>Over 12 years, the health unit, received 137,751 donations with 108 positive tests. The proportions of human immunodeficiency viruses, syphilis, human hepatitis viruses type B and C varied between 0 and 44/100,000 donations. In this period, two cases of malaria were detected in 2020–2021, and 21 were detected in 2022 corresponding to 52.1/1000 donations screened. In 2022, two cases of Chagas disease and no cases of West Nile virus were detected.</div></div><div><h3>Conclusion</h3><div>These results highlight the importance of a rigorous investigation at the time of donation in which the donor's history, including origin and movement in areas of greater geographic risk, are assessed. The recent and increasing detection of cases of malaria and Chagas disease confirms the presence of emerging infectious diseases transmitted by vectors, including mosquitoes, in blood donors. The increased risk of vector-borne diseases in Europe is a public health problem and represents a new challenge in screening donations.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103734"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143351157","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Congenital thrombotic thrombocytopenic purpura and human leukocyte antigen analysis–an amazing clue","authors":"Cevat İlteriş Kıkılı,&nbsp;Demet Kıvanç,&nbsp;Hayriye Şentürk Çiftçi,&nbsp;Mustafa M. Özbalak,&nbsp;Mustafa N. Yenerel,&nbsp;Meliha Nalçacı,&nbsp;Fatma S. Oğuz,&nbsp;Sevgi K. Beşışık","doi":"10.1016/j.htct.2025.103746","DOIUrl":"10.1016/j.htct.2025.103746","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103746"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143420843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Twice or once a day? Filgrastim dosing schedule for peripheral hematopoietic stem cells mobilization","authors":"Mehmet Bakırtaş ,&nbsp;Bahar Uncu Ulu ,&nbsp;Tuğçe Nur Yiğenoğlu ,&nbsp;Semih Başcı ,&nbsp;Ali Kılınç ,&nbsp;Tahir Darçın ,&nbsp;Fatma Nurbüke Şarkışla ,&nbsp;Derya Şahin ,&nbsp;Nuran Ahu Baysal ,&nbsp;Dicle İskender ,&nbsp;Merih Kızıl Çakar ,&nbsp;Mehmet Sinan Dal ,&nbsp;Fevzi Altuntaş","doi":"10.1016/j.htct.2025.103738","DOIUrl":"10.1016/j.htct.2025.103738","url":null,"abstract":"<div><h3>Introduction</h3><div>Granulocyte colony-stimulating factor (G-CSF) is the most prevalently used growth factor for peripheral blood hematopoietic stem cell (HSC) mobilization. Most centers split the granulocyte colony-stimulating factor in two daily doses, whereas some centers administer one dose per day. This study aims to investigate the effect of the filgrastim dosing schedule on the quantity of hematopoietic stem cells collected after mobilization in healthy donors.</div></div><div><h3>Methods</h3><div>A total of 251 healthy donors mobilized in our center were included in the study. Mobilization was either once a day (filgrastim 1 × 10 mg/kg/day) or twice a day (filgrastim 2 × 5 mg/kg/day).</div></div><div><h3>Results</h3><div>White blood cell and peripheral CD34⁺ cell numbers were significantly higher in the Twice-a-day Group on the fifth day compared to the Once-a-day Group. No statistically significant difference was shown between the two groups regarding the number of CD34⁺ cells collected on the first apheresis day or the number of apheresis procedures needed to achieve the targeted number of CD34⁺ cells.</div></div><div><h3>Conclusion</h3><div>This study revealed that one daily dose of 10 mg/kg filgrastim is as effective as administering the same dose split on two days for an adequate amount of CD34⁺ cells in healthy donors.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103738"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143420841","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"From the mechanism of action to clinical management: A review of cardiovascular toxicity in adult treated with CAR-T therapy","authors":"Frank Nunes ,&nbsp;Breno Moreno de Gusmão ,&nbsp;Franciely Bueno Wiginesk ,&nbsp;Euler Manenti ,&nbsp;Juliana Soares ,&nbsp;Mizianne Garcia Freitas ,&nbsp;Juliane Dantas Seabra-Garcez ,&nbsp;Alexandre Manoel Varela ,&nbsp;João Pedro Passos Dutra ,&nbsp;Bruno Cesar Bacchiega ,&nbsp;Tânia Félix Lorenzato da Fonseca Peixoto ,&nbsp;Carolina Maria Pinto Domingues de Carvalho e Silva ,&nbsp;Renato D. Lopes ,&nbsp;Ariane Vieira Scarlatelli Macedo","doi":"10.1016/j.htct.2024.06.008","DOIUrl":"10.1016/j.htct.2024.06.008","url":null,"abstract":"<div><div>Chimeric antigen receptor T-cell therapy represents an innovative approach to immunotherapy and currently stands out, particularly for oncohematological patients refractory to traditional treatments. Ongoing trials are further expanding its clinical use for new oncological and non-oncological indications, potentially leading to newer treatment options soon.</div><div>This new approach, however, also presents challenges, including cardiovascular toxicity. Little is reported in pivotal studies, and some recent retrospective observations suggest a non-negligible incidence of side effects with presentation ranging from mild adverse cardiovascular events to fatal complications in which, in most cases, there is a direct or indirect association with cytokine release syndrome.</div><div>In this literature review, the hypotheses of an important interface between cytokine release syndrome and cardiotoxicity by chimeric antigen receptor T-cell therapy will be addressed, as will current knowledge about risk factors for cardiotoxicity and recommendations for pre-therapy evaluation, post-infusion monitoring and clinical management of these complications.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103693"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304629","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessing the contribution of myelofibrosis to a leukoerythroblastic blood picture","authors":"Stephen E. Langabeer","doi":"10.1016/j.htct.2025.103735","DOIUrl":"10.1016/j.htct.2025.103735","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103735"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143349525","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of blueberry extract on hematological response in phenylhydrazine-induced hemolytic anemia","authors":"Daniela Drosdowski,&nbsp;Patrick Türck,&nbsp;Silvio Tasca,&nbsp;Gabriel de Lima Rosa,&nbsp;Edson Fernando Muller Guzzo,&nbsp;Sara Elis Bianchi,&nbsp;Adriana Simon Coitinho,&nbsp;Cristina Campos Carraro,&nbsp;Adriane Belló-Klein,&nbsp;Alexandre Luz de Castro,&nbsp;Valquiria Linck Bassani,&nbsp;Alex Sander da Rosa Araujo","doi":"10.1016/j.htct.2025.103744","DOIUrl":"10.1016/j.htct.2025.103744","url":null,"abstract":"<div><div>The objective of this study was to explore the therapeutic effect of blueberries on hematological parameters, oxidative stress, and interleukin-10 levels in acute hemolytic anemia induced by the administration of an intraperitoneal injection of 40 mg/kg phenylhydrazine. Male Wistar rats were divided into three groups: Control, anemia (PHZ), and anemia plus blueberries (PHZ+BB). Blueberries were administered via oral gavage (250 mg/day). The erythrocyte osmotic fragility, splenomegaly, iron metabolism, hematological analysis, reactive oxygens species, sulfhydryl group, and interleukin-10 levels were evaluated. The erythrocyte osmotic fragility (in 0.85% and 0.55% sodium chloride solution) and spleen weight-to-body weight ratio (∼400%) were elevated in the PHZ and PHZ+BB Groups compared to the controls (<em>p</em>-value &lt; 0.05). Increased transferrin and reactive oxygens species levels were found in the PHZ (15%) compared to the Control Group (<em>p</em>-value &lt; 0.05). There was an immune inflammatory response in the PHZ Group due to increases in the total leukocyte (300%), lymphocyte (100%), and neutrophil (400%) counts compared to the Control Group (<em>p</em>-value &lt; 0.05); the PHZ Group showed increased interleukin-10 levels (100%) compared to the Control Group (<em>p</em>-value &lt; 0.05). Blueberries showed a partial protective effect on these parameters, since there were lower neutrophil and lymphocyte counts and diminished interleukin-10 levels in the PHZ+BB Group compared to the PHZ Group (<em>p</em>-value &lt; 0.05). In addition, blueberries increased sulfhydryl group levels (<em>p</em>-value &lt; 0.05). These data suggest a protective role of blueberries against inflammatory response and oxidative stress in an acute hemolytic anemia model.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103744"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143509797","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}