Hematology, Transfusion and Cell Therapy最新文献

{"title":"Assessing the role of bronchoscopy in the management of patients with acute leukemia—a transversal study and proposal of evaluation","authors":"José Vanildo Rodrigues de Oliveira ,&nbsp;Carlos Wilson de Alencar Cano ,&nbsp;José Vivaldo Moreira Feitosa Júnior ,&nbsp;Guilherme Carneiro Barreto ,&nbsp;Fernanda Rodrigues Mendes ,&nbsp;Raphael Costa Bandeira de Melo ,&nbsp;Elvira Deolinda Rodrigues Pereira Velloso ,&nbsp;Vanderson Rocha ,&nbsp;Eduardo Magalhães Rego ,&nbsp;Wellington Fernandes da Silva","doi":"10.1016/j.htct.2025.103741","DOIUrl":"10.1016/j.htct.2025.103741","url":null,"abstract":"<div><h3>Introduction</h3><div>Bronchoscopy is frequently performed in the management of patients with acute leukemia due to their high susceptibility to infections. In this setting, it is performed in the context of lung infiltrates on imaging and persistent fever in immunocompromised subjects. This study aimed to evaluate the utility of bronchoscopy in patients with acute leukemia, its diagnostic yield, and its impact on management decisions.</div></div><div><h3>Methods</h3><div>This is a single-center cross-sectional study that included patients diagnosed with acute leukemia of any phenotype who received intensive chemotherapy. Consecutive patients who underwent bronchoscopy as part of a work-up for associated infections were selected, while patients who had undergone bone marrow transplant were excluded. This study investigated patient characteristics and the impact of bronchoscopy on changes in clinical management.</div></div><div><h3>Results</h3><div>Seventy-nine patients who underwent bronchoscopy at various stages of treatment were analyzed. The most frequent type of acute leukemia was acute myeloid leukemia, accounting for 68.3 % of cases. The induction phase was the most prevalent (29.1 %) treatment stage. Bacterial cultures were positive in 17 out of the 74 patients evaluated, with <em>Pseudomonas aeruginosa</em> being the most frequently identified microorganism<em>.</em> A change in medical management was observed in 18.2 % of cases, and only six patients experienced secondary complications.</div></div><div><h3>Conclusions</h3><div>This is the first Brazilian study to evaluate the utility of bronchoscopy in managing infectious complications in patients with acute leukemia. The impact of bronchoscopy on clinical management was less than anticipated, largely due to its low yield in identifying causative agents. Nevertheless, it remains a safe procedure and can be useful in specific situations.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103741"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143437300","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":"A rare case of circulating anaplastic lymphoma kinase-negative anaplastic large cell lymphoma","authors":"Radu Chiriac,&nbsp;Lucile Baseggio","doi":"10.1016/j.htct.2025.103743","DOIUrl":"10.1016/j.htct.2025.103743","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103743"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143420823","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":"Evaluation of the role of serum soluble ST2 as a diagnostic biomarker for cancer-associated venous thromboembolism","authors":"Eman Mohamed Abdellatif,&nbsp;Emad Hamdy Hamouda Mohammed,&nbsp;Azza Mohamed Amin Darwish","doi":"10.1016/j.htct.2025.103740","DOIUrl":"10.1016/j.htct.2025.103740","url":null,"abstract":"<div><h3>Introduction</h3><div>Venous thromboembolism, a common complication associated with cancer, causes increased morbidity and mortality. D-dimer levels are often increased non-specifically in cancer which limits their use to diagnose venous thromboembolism. The current study aimed to investigate the role of the serum soluble suppression of tumorigenicity 2 (sST2) as a new biomarker to diagnose venous thromboembolism in cancer.</div></div><div><h3>Methods</h3><div>Eighty-eight patients with different types of cancer were enrolled and divided into two groups: Group I: 44 cancer patients with confirmed diagnosis of venous thromboembolism and Group II: 44 age- and sex-matched cancer patients without any thrombotic complications. The D-dimer test and sST2 measurement were performed for all study subjects.</div></div><div><h3>Results</h3><div>Serum sST2 levels were significantly higher in Group I than in Group II (<em>p</em>-value &lt; 0.001); the median serum sST2 was 13.02 ng/mL (range: 7.65–117.9 ng/mL) in Group I versus 8.56 ng/mL (range: 5.59–10.33 ng/mL) in Group II. There was a significant positive correlation between serum sST2 and the D-dimer level. Using a receiver operating characteristic curve, sST2 had a greater area under the curve than the D-dimer test (0.974 versus 0.869, respectively). Although the D-dimer test was more sensitive, sST2 had a greater specificity than D-dimer (95.45 % versus 27.3 %, respectively) and a higher positive predictive value (95.3 % versus 56.8 %, respectively).</div></div><div><h3>Conclusion</h3><div>The results of the current study support a potential role of soluble ST2 to aid in diagnosing venous thromboembolism in cancer patients.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103740"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143437299","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":"When innovation meets patient blood management – a new way to see bleeding","authors":"Guilherme Rabello ,&nbsp;Rosangela Monteiro ,&nbsp;Bianca Meneghini ,&nbsp;Fabio Biscegli Jatene","doi":"10.1016/j.htct.2024.07.002","DOIUrl":"10.1016/j.htct.2024.07.002","url":null,"abstract":"<div><div>The first step in innovation is to identify a problem of real relevance and systematically address it to deliver a sophisticated and viable solution. Disruptive innovation is a process where technology, products, or services are transformed or replaced by a better innovative solution. This superiority must be perceived by users as being more accessible, simple, or convenient. Patient Blood Management (PBM) suggests the notion of the timely application of evidence-based medical and surgical concepts designed to maintain hemoglobin concentration, optimize hemostasis and minimize blood loss thus improving patient outcomes, that is, they are aimed at changing patient care, assisting healthcare professionals in disease treatment and cure as well as risk reduction. Thus, innovation in PBM is a new frontier to be pursued. The management of patient's blood and preparation for surgical procedures is an enormous challenge that helps minimize anemia and control blood loss during hospitalization, ensuring they are discharged in adequate clinical conditions. Until 2016, there was no standard definition or classification for the severity of intraoperative bleeding or hemostasis. The development of a PBM program when combined to the development of a bleeding scale such as the validated Intraoperative Bleeding (VIBe) Scale, represents a new solution that balances perioperative blood loss and more importantly, enables a critical cultural change which can be useful to help surgeons communicate anticipated hemostatic needs throughout a case and therefore enhance efficiency leading to better outcomes.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 1","pages":"Article 103691"},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304649","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":"Oral calcium supplementation versus placebo in mitigating citrate reactions during apheresis: an open-label randomized control trial","authors":"Masaya Abe ,&nbsp;Keiko Fujii ,&nbsp;Nobuharu Fujii ,&nbsp;Toshiharu Mitsuhashi ,&nbsp;Takuya Fukumi ,&nbsp;Yuichi Sumii ,&nbsp;Maiko Kimura ,&nbsp;Tomohiro Urata ,&nbsp;Takumi Kondo ,&nbsp;Fumio Otsuka ,&nbsp;Yoshinobu Maeda","doi":"10.1016/j.htct.2024.06.010","DOIUrl":"10.1016/j.htct.2024.06.010","url":null,"abstract":"<div><h3>Background</h3><div>Citrate-related hypocalcemia is the most common adverse event linked with peripheral blood progenitor cell apheresis. A previous retrospective study highlighted the prophylactic effectiveness of oral calcium drinks before apheresis, supplemented with intravenous calcium gluconate. Consequently, this study is a randomized controlled trial comparing oral calcium with placebo drinks</div></div><div><h3>Study Design and Methods</h3><div>Healthy donors were randomized to receive either oral calcium (Cohort A) or placebo (Cohort B) drinks. If symptoms emerged, all donors were given calcium drinks to counteract hypocalcemia. The primary endpoint centered on the incidence of Grade 1 or higher citrate-related symptoms. Analyses were performed using the crude model and doubly robust estimation.</div></div><div><h3>Results</h3><div>Forty-two healthy donors participated from January 2021 to July 2022. Case distribution (Cohort A: Cohort B) stood at 3:7 (Grade 1), 2:2 (Grade 2), and 1:0 (Grade 3); no Grade 4 cases were identified. There was no statistical significance in the incidence of Grade 1 or higher and Grade 3 citrate-related symptoms.</div></div><div><h3>Discussion</h3><div>The cumulative incidence of citrate-related side effects was less pronounced than in the previous research. This could stem from absence of blinding, and the decision to administer calcium drinks to the untreated group upon symptom detection. Although preemptive oral calcium intake before peripheral blood progenitor cell apheresis is not wholly effective, providing calcium-rich beverages to symptomatic donors may stave off symptom intensification.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"46 ","pages":"Pages S234-S240"},"PeriodicalIF":1.8,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11726075/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142368027","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":"Venetoclax with low-dose cytarabine, a forgotten combination in patients with acute myeloid leukemia ineligible for intensive chemotherapy: a systematic review","authors":"Lauro Fabián Amador-Medina ,&nbsp;Erick Crespo-Solís ,&nbsp;Francisco Javier Turrubiates-Hernández ,&nbsp;Karla Edith Santibañez-Bedolla","doi":"10.1016/j.htct.2024.07.006","DOIUrl":"10.1016/j.htct.2024.07.006","url":null,"abstract":"<div><h3>Background</h3><div>Based on the VIALE-A and VIALE-C studies, the Food and Drug Administration approved venetoclax in 2020 in combination with azacitidine or low-dose cytarabine for the treatment of patients with acute myeloid leukemia ineligible for intensive chemotherapy. After the publication of these studies, venetoclax/azacitidine was assumed to be superior to venetoclax/low-dose cytarabine; however, these studies were not designed to demonstrate superiority between these combinations. Therefore, we conducted a systematic review to describe overall survival, complete remission rate, and composite complete remission rate to assess response of these two regimens in patients with newly diagnosed acute myeloid leukemia who are ineligible for intensive chemotherapy.</div></div><div><h3>Materials and methods</h3><div>The PubMed and Web of Science databases were searched for retrospective studies and complete remission, composite complete remission, and overall survival rates were recorded.</div></div><div><h3>Results</h3><div>Only 11 of the 815 publications identified were eligible to be included n this review, ten studies evaluated the venetoclax/azacitidine combination and one study evaluated the venetoclax/low-dose cytarabine combination. The median overall survival for venetoclax/azacitidine was 10.75 months, whereas for venetoclax/low-dose cytarabine the median overall survival had not been reached at the time of publication. Composite complete remission was 63.3 % for venetoclax/azacitidine and 90 % for venetoclax/low-dose cytarabine. Adverse events were similar for both combinations.</div></div><div><h3>Conclusions</h3><div>A limited number of studies investigating the venetoclax/low-dose cytarabine combination exist. Based on the available data, the superiority of venetoclax/azacitidine over venetoclax/low-dose cytarabine cannot be assumed for all acute myeloid leukemia patients who are ineligible for intensive chemotherapy. Venetoclax/low-dose cytarabine can still be considered as an option for the drug combinations currently under investigation.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"46 ","pages":"Pages S322-S331"},"PeriodicalIF":1.8,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11726088/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142376481","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":"Comparison of plasma cell bone marrow counts by different methods in patients diagnosed with plasma cell disorders","authors":"Claudia Monteiro,&nbsp;Paulo Campregher,&nbsp;Denise Pasqualin,&nbsp;Nydia Bacal,&nbsp;Liliana Suganuma,&nbsp;Elvira Velloso","doi":"10.1016/j.htct.2024.06.002","DOIUrl":"10.1016/j.htct.2024.06.002","url":null,"abstract":"<div><h3>Introduction</h3><div>Plasma cell quantification in bone marrow is important for diagnosis, prognosis, and treatment of plasma cell diseases. It can be performed by several methods such as aspiration, imprint and flow cytometry, and biopsy.</div></div><div><h3>Objectives</h3><div>To compare plasma cell counts at diagnosis of plasma cell diseases using different methods.</div></div><div><h3>Methods</h3><div>An observational study was carried out of laboratory results of adult patients with plasma cell diseases, who underwent aspiration, imprint cytology, flow cytometry (CD38, C138) and biopsy in a single institution between January 2015 and May 2021. The intraclass correlation coefficient was used to assess agreement between different methods with results stratified into three groups: &lt;10%; 10–59% and ≥60% of infiltration.</div></div><div><h3>Results</h3><div>Sixty-seven cases were studied: 59.7% were men with a median age of 70 (range: 32–85) years. The diagnoses were multiple myeloma in 61%, gammopathy of undetermined significance in 25.4%, smoldering myeloma in 6% and other plasma cell dyscrasias in 7.6%. Less than 10% infiltration was found in 32 (47.7%), 35 (52.2%), 44 (65.7%) and 25 (37.3%) of patients, respectively by aspiration, imprint cytology, flow cytometry and biopsy. Infiltration ≥60% was detected in 7 (10.4%), 4 (6.0%), 2 (3.0%) and 21 (31.3%) cases, respectively. There was disagreement between the results in 37 (55.2%) of patients. Of these, 28 had greater infiltration in biopsies. The concordance (Kappa index) of biopsy with aspiration, imprint and flow cytometry was 0.501, 0.408 and 0.17; of aspiration with imprint and flow cytometry, it was 0.738 and 0.541 and between imprint and flow cytometry, it was 0.573%.</div></div><div><h3>Conclusions</h3><div>Only aspiration and imprint cytology results agreed. Biopsy showed greater infiltrations than the other methods, but aspiration, and imprint and flow cytometry provided additional data in the diagnosis and thus should also be performed.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"46 ","pages":"Pages S202-S207"},"PeriodicalIF":1.8,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11726111/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142570807","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":"The effect of modulating platelet reactive oxygen species by the addition of antioxidants to prevent clearance of cold-stored platelets","authors":"Rufeng Xie,&nbsp;Yiming Yang,&nbsp;Xueyu Jiang,&nbsp;Li Gao,&nbsp;Juan Sun,&nbsp;Jie Yang","doi":"10.1016/j.htct.2024.09.2479","DOIUrl":"10.1016/j.htct.2024.09.2479","url":null,"abstract":"<div><h3>Background</h3><div>It is known that the rapid clearance of cold-stored platelets is attributed to various storage lesions, including an abnormal increase in reactive oxygen species when platelets are exposed to cold temperatures. As an antioxidant, N-acetylcysteine exhibits some significant effects on scavenging various reactive oxygen species and inhibiting cell damage and apoptosis.</div></div><div><h3>Aims</h3><div>This study aimed to investigate the effects of N-acetylcysteine on reducing reactive oxygen species production and protecting cold-stored platelets from phagocytosis and clearance, and to determine the optimal concentration of N-acetylcysteine.</div></div><div><h3>Methods</h3><div>Platelet concentrates were divided into three groups: room-temperature-stored platelets, cold-stored platelets, and cold-stored platelets with the addition of different concentrations of N-acetylcysteine. After five days of storage, reactive oxygen species production, lipid peroxidation levels, activation marker expressions, GPIb/ɑ desialylation with exposure of glycan residues and other quality parameters of platelets were measured and compared between the groups. Phagocytosis of platelets was detected by phorbol 12-myristate 13-acetate-activated THP-1 or Hep G2 cells. Moreover, the recovery of infused platelets was measured in severe combined immunodeficient mice at different timepoints.</div></div><div><h3>Results</h3><div>After 5 days of storage, cytoplasmic reactive oxygen species significantly increased in chilled compared to non-chilled platelets; they were notably reduced with the addition of N-acetylcysteine, particularly at a concentration of 5 mM. Compared with chilled platelets, the P-selectin and phosphatidylserine expressions, as well as exposure of GPIb/ɑ glycan residues, were significantly reduced with 5 mM of N-acetylcysteine. Phagocytosis of platelets by THP-1 or Hep G2 cells was significantly lower in 5 mM of N-acetylcysteine compared to cold-stored platelets without N-acetylcysteine.</div></div><div><h3>Conclusions</h3><div>This study demonstrated correlations between reactive oxygen species production and their pro-oxidant effects on platelet clearance after cold storage. The addition of N-acetylcysteine at an appropriate concentration do not only protects chilled platelets from storage lesions caused by reactive oxygen species overproduction but also prevents platelet phagocytosis <em>in vitro</em> and clearance <em>in vivo</em>, thereby extending circulating time.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"46 ","pages":"Pages S272-S283"},"PeriodicalIF":1.8,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11726102/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142585411","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":"Development and validation of the self-regulation of blood donation scale for blood donors","authors":"Fahimeh Ranjbar Kermani ,&nbsp;Sedigheh Amini Kafi-Abad ,&nbsp;Mahtab Maghsudlu ,&nbsp;Kamran Mousavi Hosseini ,&nbsp;Fatemeh Mohammadali ,&nbsp;Atefeh MohammadJafari","doi":"10.1016/j.htct.2024.09.2482","DOIUrl":"10.1016/j.htct.2024.09.2482","url":null,"abstract":"<div><h3>Background</h3><div>Because knowledge of blood donor motivation is crucial in guiding recruitment and retention efforts, the present study aimed at developing and validating a new scale as a multidimensional measure of blood donation motivation from the perspective of the self- determination theory.</div></div><div><h3>Methods</h3><div>This study was conducted in three phases from September 2022 to May 2023. The first phase involved developing a draft scale based on a literature review. In the second phase, face and content validity were performed. The third phase used a cross-sectional design to assess the construct validity and internal consistency of the initial scale following administration to blood donors with a history of at least one previous whole blood donation who visited the largest blood transfusion center in Iran. Of the 420 subjects who were recruited using a mixed sampling method, 343 who fully completed the initial version of the scale were subjected to an construct validity assessment using exploratory factor analysis with Equamax rotation and internal consistency using McDonald's omega coefficient.</div></div><div><h3>Main results</h3><div>The initial version of the scale consisted of 30 survey items with both a content validity ratio and a content validity index of 0.99. Exploratory factor analysis identified 24 items; grouped in six regulation factors (non-regulation, external regulation, introjected regulation, identified regulation, integrated regulation, and intrinsic regulation). The factors demonstrated adequate internal consistency with ω values ranging from 0.60 to 0.79.</div></div><div><h3>Conclusion</h3><div>The present study provides psychometric support for the newly developed questionnaire to evaluate donation motivation among blood donors in Iran or in other countries with similar language, and religious and cultural values.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"46 ","pages":"Pages S299-S305"},"PeriodicalIF":1.8,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11726103/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142635094","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":"CAR-T CELL THERAPY IN ACUTE LEUKEMIAS","authors":"Serhat Çelik","doi":"10.1016/j.htct.2024.11.102","DOIUrl":"10.1016/j.htct.2024.11.102","url":null,"abstract":"&lt;div&gt;&lt;div&gt;Acute leukemias, particularly acute lymphoblastic leukemia (ALL) and, to a lesser extent, acute myeloid leukemia (AML), remain among the most challenging hematologic malignancies due to high mortality rates and limited treatment options. In this context, Chimeric Antigen Receptor T (CAR-T) cell therapy has emerged as a promising approach for patients with refractory or relapsed disease.&lt;/div&gt;&lt;div&gt;CAR-T cells are generated by genetically engineering the patient's T cells to express synthetic receptors targeting specific tumor-associated antigens. In ALL, CD19-targeted CAR-T cell therapies have demonstrated complete remission (CR) rates of 70–90%. For AML, ongoing research is exploring alternative targets.&lt;/div&gt;&lt;div&gt;Clinical Studies and Outcomes&lt;/div&gt;&lt;div&gt;ELIANA Trial&lt;/div&gt;&lt;div&gt;The ELIANA trial, the largest global multicenter study of CD19-targeted CAR-T therapy, focused on pediatric and young adult ALL patients Tisagenlecleucel was infused into 75 ALL patients and evaluated for efficacy. The overall remission rate at 3 months was 81%, and all patients who responded to treatment were found to be negative for minimal residual disease by flow cytometry. Event-free survival and overall survival rates were 73% and 90% at 6 months and 50% and 76% at 12 months, respectively. Median duration of remission was not achieved. Tisagenlecleucel persisted in the blood for up to 20 months. Grade 3 or 4 adverse events thought to be related to tisagenlecleucel occurred in 73% of patients. Cytokine release syndrome occurred in 77% of patients, 48% of whom received tocilizumab. Neurological events occurred in 40% of patients and were managed with supportive care, and no brain edema was reported.&lt;/div&gt;&lt;div&gt;ZUMA-3 Trial&lt;/div&gt;&lt;div&gt;The ZUMA-3 an international, multicentre, single-arm, open-label study evaluating the efficacy and safety of the autologous anti-CD19 CAR-T-cell therapy KTE-X19 in adult patients with relapsed or refractory B-precursor acute lymphoblastic leukaemia. KTE-X19 was administered to 55 (77%) patients. At a median follow-up of 16.4 months (13.8-19.6), 39 patients (71%; 95% CI 57-82, p&lt;0.0001) had CR or CRi and 31 (56%) achieved CR. Median duration of remission was 12.8 months (95% CI 8.7 - not estimable), median relapse-free survival was 11.6 months (2.7-15.5) and median overall survival was 18.2 months (15.9 - not estimable). Among responders, median overall survival was not reached and 38 (97%) patients had MRD negativity. Ten (18%) patients received allo-SCT consolidation after KTE-X19 infusion. The most common adverse events of grade 3 or higher were anemia (27 [49%] patients) and pyrexia (20 [36%] patients). 14 (25%) patients had grade 3 or higher infections. Two grade 5 KTE-X19-related events occurred (cerebral herniation and septic shock). Grade 3 or higher cytokine release syndrome occurred in 13 (24%) patients, and grade 3 or higher neurologic events occurred in 14 (25%) patients.&lt;/div&gt;&lt;div&gt;AML Target Studies&lt;/div&gt;&lt;div&gt;AML poses","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"46 ","pages":"Pages S10-S11"},"PeriodicalIF":1.8,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143327107","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}