Hematology, Transfusion and Cell Therapy最新文献

{"title":"Dysmegakaryopoiesis in myelodysplastic syndrome: Beyond cell dysplasia","authors":"Maria Mirele da Silva Ribeiro , Francisco Dário Rocha Filho , Howard Lopes Ribeiro Junior , Priscila da Silva Mendonça , Ronald Feitosa Pinheiro , Silvia Maria Meira Magalhães","doi":"10.1016/j.htct.2024.09.2485","DOIUrl":"10.1016/j.htct.2024.09.2485","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103725"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143420844","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":"Chronic lymphocytic leukemia diagnosis: how many more algorithms and scoring systems do we need?","authors":"Daniel Mazza Matos","doi":"10.1016/j.htct.2024.05.007","DOIUrl":"10.1016/j.htct.2024.05.007","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103668"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142094237","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":"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":"Is galactomannan a useful tool for triage and diagnosis of oral invasive aspergillosis?","authors":"Maria Júlia Pagliarone ,&nbsp;Lara Maria Alencar Ramos Innocentini ,&nbsp;Fernanda Bortolotto ,&nbsp;Vanessa Tonetto Marques Galves ,&nbsp;Hilton Marcos Alves Ricz ,&nbsp;Tatiane Cristina Ferrari ,&nbsp;Renato Luiz Guerino Cunha ,&nbsp;Belinda Pinto Simões ,&nbsp;Leandro Dorigan de Macedo","doi":"10.1016/j.htct.2024.06.005","DOIUrl":"10.1016/j.htct.2024.06.005","url":null,"abstract":"<div><h3>Objective</h3><div>To evaluate the accuracy of the galactomannan serum test in diagnosing oral invasive aspergillosis.</div></div><div><h3>Methods</h3><div>This prospective observational study included oncohematological neutropenic patients with suspected invasive aspergillosis, but without signs of pulmonary involvement. These patients underwent nasofibroscopy, biopsy, galactomannan serum testing, and maxillofacial high-resolution computed tomography to diagnose invasive aspergillosis. Patients were divided into two groups: Group 1 consisted of those with proven invasive aspergillosis, while Group 2 included patients without proven invasive aspergillosis. Sensitivity, specificity, positive predictive value, and negative predictive value were calculated.</div></div><div><h3>Results</h3><div>Thirteen patients were included in Group 1 and four in Group 2. The sensitivity, specificity, positive predictive and negative predictive values were 0.69, 1.0, 1.0 and 0.5, respectively. Sensitivity was higher in cases with <em>Aspergillus</em> sinusitis than in cases with exclusive oral lesions (0.77 versus 0.5, respectively). The galactomannan serum test optical density index was higher in Group 1 (2.4; range 0.2–3.5) than in Group 2 (0.2; range: 0.1–0.3; <em>P</em>-value = 0.007.</div></div><div><h3>Conclusions</h3><div>The galactomannan serum test is a valuable tool for screening invasive aspergillosis, especially in cases with nasal or sinus involvement, but biopsy is still the gold standard for diagnosis.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103687"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142335172","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":"Unraveling thalassemia intermedia: Novel insights of a hemoglobin Jax [HBA2:c.44G>C] and deletional α0-thalassemia interaction phenotype","authors":"Sitthichai Panyasai ,&nbsp;Kanokwan Jaiping ,&nbsp;Pisuttinee Khantarag ,&nbsp;Patcharee Nochod ,&nbsp;Surada Satthakarn","doi":"10.1016/j.htct.2025.103739","DOIUrl":"10.1016/j.htct.2025.103739","url":null,"abstract":"<div><h3>Objective</h3><div>To elucidate the molecular basis, hematological features, and electrophoretic and chromatographic mobility behavior of an unstable α₂-globin chain variant, and to describe the diagnostic approach.</div></div><div><h3>Methods</h3><div>A Thai patient with unexplained chronic anemia and her daughter were investigated. Hematological data were analyzed using a standard automated cell counter. Hemoglobin was analyzed using high-performance liquid chromatography (HPLC) and capillary electrophoresis (CE). Mutational analysis was performed using appropriate polymerase chain reaction (PCR) techniques and direct sequencing. Additionally, α-globin haplotype analysis was conducted. Simple and rapid diagnostic methods were developed.</div></div><div><h3>Results</h3><div>Hemoglobin analysis in the patient revealed anomalous peaks separated from normal hemoglobin visible using the HPLC technique. These peaks were virtually absent in the daughter. DNA analysis identified a G to C mutation at codon 14 of the α₂-globin gene responsible for hemoglobin Jax in <em>trans</em> to the α⁰-thalassemia gene in the patient. Heterozygosity of this mutation was identified in her daughter. Hematological analysis showed mild thalassemia-like changes in simple heterozygotes and exhibited a hemoglobin H-like phenotype when combined with α⁰-thalassemia. Isopropanol stability testing and bioinformatic software indicated that the variant was unstable and potentially damaging. This mutation was confirmed using allele-specific PCR. Hemoglobin Jax was strongly associated with the haplotype [+ - <em>S</em> + - + -].</div></div><div><h3>Conclusions</h3><div>Hemoglobin Jax, a pathological α-globin variant, is asymptomatic in simple heterozygotes and demonstrates more pronounced clinical effects when associated with deletional α-thalassemia. This knowledge can help develop strategies to prevent hemoglobinopathies in regions of high prevalence. Accurate identification requires DNA level analysis.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103739"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143420840","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":"STAT5B::RARα-positive acute promyelocytic leukemia: Role of next generation sequencing in detection of a rare malignancy","authors":"Indranil Dey ,&nbsp;Sushant Vinarkar ,&nbsp;Mayur Parihar ,&nbsp;Deepak Kumar Mishra","doi":"10.1016/j.htct.2024.07.009","DOIUrl":"10.1016/j.htct.2024.07.009","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103726"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143349524","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":"Effect of blood groups on acquired and congenital thrombotic thrombocytopenic purpura and clinical correlation: Multi-center Turkish cohort study","authors":"Cevat İlteriş Kıkılı,&nbsp;Damla Ortaboz,&nbsp;Melek Yanaşık,&nbsp;Muhlis Cem Ar,&nbsp;Sevgi Kalayoğlu Beşışık","doi":"10.1016/j.htct.2024.09.2483","DOIUrl":"10.1016/j.htct.2024.09.2483","url":null,"abstract":"<div><h3>Background</h3><div>Thrombotic thrombocytopenic purpura (TTP) is a microangiopathic hemolytic anemia associated with ADAMTS-13 deficiency, a cleaving protease of von Willebrand factor (vWF). According to the literature, blood group O tends to be less common among these patients than in the general population. This study aimed to investigate whether the decreasing trend of blood group O in thrombotic thrombocytopenic purpura patients is observed in a Turkish cohort and to analyze the relationship between clinical outcomes and blood groups.</div></div><div><h3>Patients and Methods</h3><div>A total of 65 patients with acquired and five patients with congenital thrombotic thrombocytopenic purpura from two university hospitals were enrolled in this study. As a control group, the blood group data of 136,231 individuals who were not diagnosed without anemia were obtained from the archives of the Istanbul Medical Faculty Blood Centre. The blood groups were compared between cases and the Control Group using the chi-square test. Subsequently, the clinical outcomes of patients and categorized blood groups were compared by the chi-square test, Mann Whitney U test and Cox regression with Kaplan Meier analysis.</div></div><div><h3>Results</h3><div>This study shows that the decreasing trend of blood group O was not observed in this Turkish cohort. Regarding the relationship between blood groups and clinical outcomes, the AB blood group is associated with a good prognosis and blood group O is associated with a poor prognosis. In addition, relapses were more common with blood group A patients but less common in blood group B.</div></div><div><h3>Conclusion</h3><div>The current study shows the association between thrombotic thrombocytopenic purpura and blood groups in the Turkish cohort. This study also contributes by analyzing the relationship between blood groups and clinical outcomes.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103723"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143437928","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":"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}