Hematology, Transfusion and Cell Therapy最新文献

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Howell-Jolly-like inclusions in granulocytes of a liver transplant recipient 肝移植受者粒细胞中的howell - jolly样包涵体
IF 1.6
Hematology, Transfusion and Cell Therapy Pub Date : 2025-09-10 DOI: 10.1016/j.htct.2025.103978
Verónica Roldán Galiacho, Sara Hormaza de Jauregui, Lourdes Elicegui Fernández
{"title":"Howell-Jolly-like inclusions in granulocytes of a liver transplant recipient","authors":"Verónica Roldán Galiacho, Sara Hormaza de Jauregui, Lourdes Elicegui Fernández","doi":"10.1016/j.htct.2025.103978","DOIUrl":"10.1016/j.htct.2025.103978","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 4","pages":"Article 103978"},"PeriodicalIF":1.6,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145026397","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
An enigmatic tale of macrophages in bone marrow causing inflammation of the brain: A case report on CNS HLH 骨髓中巨噬细胞引起脑炎症的神秘故事:一例中枢神经系统HLH报告
IF 1.6
Hematology, Transfusion and Cell Therapy Pub Date : 2025-09-08 DOI: 10.1016/j.htct.2025.103981
Ivy E. Verriet , Jessica Liu , Adrienne Fulford , Uday Deotare
{"title":"An enigmatic tale of macrophages in bone marrow causing inflammation of the brain: A case report on CNS HLH","authors":"Ivy E. Verriet ,&nbsp;Jessica Liu ,&nbsp;Adrienne Fulford ,&nbsp;Uday Deotare","doi":"10.1016/j.htct.2025.103981","DOIUrl":"10.1016/j.htct.2025.103981","url":null,"abstract":"<div><h3>Background</h3><div>Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening immune disorder characterized by excessive inflammation and multiorgan involvement. Rarely, HLH can manifest with signs and symptoms isolated to the central nervous system (CNS). This case report highlights the unique clinical course of CNS-isolated HLH in a 19-year-old female who, despite a nine-year delay in diagnosis, achieved disease remission following a hematopoietic stem cell transplant (HSCT).</div></div><div><h3>Case</h3><div>The patient initially presented at 9 years old with seizures, ataxia, and progressive cognitive decline. Over the next nine years, extensive diagnostic evaluations were performed, including neuroimaging, cerebrospinal fluid analysis, and genetic testing. Genetic testing identified a compound heterozygous mutation in the PRF1 gene, confirming a diagnosis of familial HLH (FHL). The patient underwent hematopoietic stem cell transplant (HSCT) from an HLA-matched unrelated donor. Despite significant complications, including multiple infections and renal failure, she achieved remission. Six years post-transplant, the patient exhibited stabilization of neurological function, cessation of seizures, and absence of active HLH.</div></div><div><h3>Conclusion</h3><div>This case underscores the importance of considering genetic testing in patients with unexplained CNS symptoms and atypical radiological findings. Timely HSCT, even in cases with delayed diagnosis, can lead to remission and improved quality of life.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 4","pages":"Article 103981"},"PeriodicalIF":1.6,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145010124","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Daratumumab-EPOCH for transformed anaplastic multiple myeloma Daratumumab-EPOCH治疗转化间变性多发性骨髓瘤
IF 1.6
Hematology, Transfusion and Cell Therapy Pub Date : 2025-09-06 DOI: 10.1016/j.htct.2025.103977
Espen Stormorken , Anders Erik Astrup Dahm
{"title":"Daratumumab-EPOCH for transformed anaplastic multiple myeloma","authors":"Espen Stormorken ,&nbsp;Anders Erik Astrup Dahm","doi":"10.1016/j.htct.2025.103977","DOIUrl":"10.1016/j.htct.2025.103977","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 4","pages":"Article 103977"},"PeriodicalIF":1.6,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145004250","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Post-thaw dimethyl sulfoxide reduction in autologous peripheral blood progenitor cell suspensions 解冻后自体外周血祖细胞悬浮液中二甲亚砜的还原
IF 1.6
Hematology, Transfusion and Cell Therapy Pub Date : 2025-08-25 DOI: 10.1016/j.htct.2025.103965
Miroslava Jandová , Pavel Měřička , Jiří Gregor , Miriam Lánská , Aleš Bezrouk , Dana Čížková , Jakub Radocha
{"title":"Post-thaw dimethyl sulfoxide reduction in autologous peripheral blood progenitor cell suspensions","authors":"Miroslava Jandová ,&nbsp;Pavel Měřička ,&nbsp;Jiří Gregor ,&nbsp;Miriam Lánská ,&nbsp;Aleš Bezrouk ,&nbsp;Dana Čížková ,&nbsp;Jakub Radocha","doi":"10.1016/j.htct.2025.103965","DOIUrl":"10.1016/j.htct.2025.103965","url":null,"abstract":"<div><h3>Background and objectives</h3><div>Dimethyl sulfoxide has become the most common cryoprotectant used for cryopreservation of hematopoietic progenitor cells because of its efficiency, regardless of its potentially toxic side effects. Its application is considered safe, provided that the daily dose administered does not exceed 1 gram per kilogram of patient weight. Indications for its reduction after thawing are limited to patients with high risk of malignant arrhythmia and those with severely impaired renal function. However, dimethyl sulfoxide reduction can lead to the loss of viable progenitors.</div></div><div><h3>Methods</h3><div>A retrospective study of viable hematopoietic progenitor cell recovery after dimethyl sulfoxide reduction was performed with 13 patients (nine men, four women) with secondary amyloidosis in multiple myeloma (<em>n</em> = 9), primary amyloid light chain amyloidosis (<em>n</em> = 3), or severe adverse reaction at the beginning of the hematopoietic progenitor cell concentrate infusion (<em>n</em> = 1). The Wilcoxon signed-rank test was used.</div></div><div><h3>Results</h3><div>The results of the dimethyl sulfoxide reduction process showed a high recovery of viable nucleated cells (median: 120.85 %), and of viable mononuclear cells (median: 104.53 %). There was a significant decrease in total number of viable CD34<sup>+</sup> cells in comparison with data obtained after original collection (median: 51.49 %). No significant decrease in colony-forming unit capacity was observed after dimethyl sulfoxide reduction (median: 93.37 %).</div></div><div><h3>Conclusion</h3><div>The dimethyl sulfoxide removal process and total process recoveries revealed considerable individual variability. To minimize the risk of prolonged engraftment or non-engraftment, it is important to apply this process only to high-risk patients.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 4","pages":"Article 103965"},"PeriodicalIF":1.6,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144892872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
COVID-19 Microangiopathy: Insights into plasma exchange as a therapeutic strategy COVID-19微血管病:血浆置换作为治疗策略的见解
IF 1.6
Hematology, Transfusion and Cell Therapy Pub Date : 2025-08-23 DOI: 10.1016/j.htct.2025.103963
Yigit Baykara , Kaan Sevgi , Yamac Akgun
{"title":"COVID-19 Microangiopathy: Insights into plasma exchange as a therapeutic strategy","authors":"Yigit Baykara ,&nbsp;Kaan Sevgi ,&nbsp;Yamac Akgun","doi":"10.1016/j.htct.2025.103963","DOIUrl":"10.1016/j.htct.2025.103963","url":null,"abstract":"<div><div>COVID-19-associated thrombotic microangiopathy has emerged as a severe complication that exacerbates morbidity and mortality in critical cases. Thrombotic microangiopathy, characterized by microvascular thrombosis and endothelial injury, includes conditions like thrombotic thrombocytopenic purpura and atypical hemolytic uremic syndrome. This review investigates therapeutic plasma exchange as a potential strategy to mitigate COVID-19-induced thrombotic microangiopathy, examining its role in removing pro-inflammatory cytokines, immune complexes, and pro-thrombotic factors. Additionally, it highlights the synergistic effects when therapeutic plasma exchange is combined with treatments such as complement inhibitors and immunosuppressants. Preliminary evidence, drawn from case reports and early trials, supports the efficacy of therapeutic plasma exchange in improving outcomes for COVID-19-associated thrombotic microangiopathy. However, larger randomized controlled trials are necessary to definitively establish its place in COVID-19 management, particularly for high-risk and transplant patients with underlying immunological vulnerabilities.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 4","pages":"Article 103963"},"PeriodicalIF":1.6,"publicationDate":"2025-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144889793","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Updating the Brazilian clinical practice guidelines for sickle cell disease: Recommendations and development process 更新巴西镰状细胞病临床实践指南:建议和发展过程
IF 1.6
Hematology, Transfusion and Cell Therapy Pub Date : 2025-08-23 DOI: 10.1016/j.htct.2025.103964
Ludmila Peres Gargano , Mariana Millan Fachi , Layssa Andrade Oliveira , Clarisse Lobo , Katharina Nelly Tobos Melnikoff , Selma Soriano , Marta da Cunha Lobo Souto Maior , Meline Rossetto Kron-Rodrigues , Dalila Fernandes Gomes , Haliton Alves Oliveira Junior , Rosa Camila Lucchetta
{"title":"Updating the Brazilian clinical practice guidelines for sickle cell disease: Recommendations and development process","authors":"Ludmila Peres Gargano ,&nbsp;Mariana Millan Fachi ,&nbsp;Layssa Andrade Oliveira ,&nbsp;Clarisse Lobo ,&nbsp;Katharina Nelly Tobos Melnikoff ,&nbsp;Selma Soriano ,&nbsp;Marta da Cunha Lobo Souto Maior ,&nbsp;Meline Rossetto Kron-Rodrigues ,&nbsp;Dalila Fernandes Gomes ,&nbsp;Haliton Alves Oliveira Junior ,&nbsp;Rosa Camila Lucchetta","doi":"10.1016/j.htct.2025.103964","DOIUrl":"10.1016/j.htct.2025.103964","url":null,"abstract":"<div><h3>Background</h3><div>Sickle cell disease is a hereditary blood disorder that significantly impacts morbidity and mortality, requiring comprehensive care. In Brazil, its management in the National Health Service follows the Brazilian Clinical Practice Guidelines, based on evidence and expert consensus. Periodic updates ensure alignment with new scientific findings.</div></div><div><h3>Objectives</h3><div>This study describes the methodology for updating the clinical guidelines for sickle cell disease and provides an overview of recommendations for diagnosis, treatment and monitoring, emphasizing the evidence and health technology assessments for prioritized technologies.</div></div><div><h3>Methods</h3><div>The update followed the technical guide of the Brazilian Ministry of Health, and the Gradings of Recommendation, Assessment, Development and Evaluation (GRADE) approach. All the recommendations were assessed by the National Committee for Health Technology Incorporation (Conitec). The clinical guidelines panel included health technology assessment researchers, clinical experts, and policymakers. Systematic reviews assessed new evidence with stakeholder contributions being incorporated through public consultation. Cost-effectiveness analysis was applied to support new technology coverage or changes.</div></div><div><h3>Results</h3><div>The updated clinical guidelines provide structured recommendations for screening, diagnosis, prophylaxis, vaccination, and treatment, covering pharmacological and non-pharmacological approaches. It emphasizes patient and caregiver education to promote early recognition of complications. Expected benefits include fewer pain crises, fewer hospitalizations and transfusions, and improved fetal hemoglobin level, quality of life and survival rates. Key updates include listing epoetin alfa and 100 mg hydroxyurea tablets, expanding hydroxyurea eligibility criteria and revising monitoring protocols.</div></div><div><h3>Conclusion</h3><div>The updated clinical practice guidelines standardize sickle cell disease care in the Brazilian NHS aligned with current evidence. Dissemination and integration aim to enhance healthcare delivery, while future assessments should optimize real-world implementation.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 4","pages":"Article 103964"},"PeriodicalIF":1.6,"publicationDate":"2025-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144889740","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Electroacupuncture as a tool to stimulate bone marrow megakaryocytes in mice: A pilot study 电针作为刺激小鼠骨髓巨核细胞的工具:一项初步研究
IF 1.6
Hematology, Transfusion and Cell Therapy Pub Date : 2025-08-23 DOI: 10.1016/j.htct.2025.103966
Luiza P.R. dos Santos Mariani , Rita M.V.M. Rocha , Lidiane M.B. Leite , Alexandra C. Senegaglia , Pedro V. Michelotto
{"title":"Electroacupuncture as a tool to stimulate bone marrow megakaryocytes in mice: A pilot study","authors":"Luiza P.R. dos Santos Mariani ,&nbsp;Rita M.V.M. Rocha ,&nbsp;Lidiane M.B. Leite ,&nbsp;Alexandra C. Senegaglia ,&nbsp;Pedro V. Michelotto","doi":"10.1016/j.htct.2025.103966","DOIUrl":"10.1016/j.htct.2025.103966","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 4","pages":"Article 103966"},"PeriodicalIF":1.6,"publicationDate":"2025-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144889749","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Incidence of hepatocellular carcinoma in beta thalassemia: a systematic review and meta-analysis -地中海贫血中肝细胞癌的发生率:一项系统回顾和荟萃分析
IF 1.6
Hematology, Transfusion and Cell Therapy Pub Date : 2025-07-01 DOI: 10.1016/j.htct.2025.103934
Marcella Adisuhanto , Alver Prasetya , Alius Cahyadi , Amaylia Oehadian
{"title":"Incidence of hepatocellular carcinoma in beta thalassemia: a systematic review and meta-analysis","authors":"Marcella Adisuhanto ,&nbsp;Alver Prasetya ,&nbsp;Alius Cahyadi ,&nbsp;Amaylia Oehadian","doi":"10.1016/j.htct.2025.103934","DOIUrl":"10.1016/j.htct.2025.103934","url":null,"abstract":"<div><h3>Background</h3><div>Current evidence indicates that iron overload increases the risk of hepatocellular carcinoma. However, the incidence of hepatocellular carcinoma in thalassemia is still unclear. This review aims to summarize the current evidence regarding the incidence of hepatocellular carcinoma in thalassemia patients.</div></div><div><h3>Methods</h3><div>Detailed searches were conducted in several databases, including PubMed, Europe PMC, EBSCOHost, and ProQuest. Keywords such as “thalassemia” and “hepatocellular carcinoma,” along with other relevant synonyms, were used. Articles investigating the incidence of hepatocellular carcinoma in thalassemia patients were included. Pooled estimates were calculated using the DerSimonian Laird inverse-variance random effect model and presented as incidence (%) along with their 95 % confidence intervals and 95 % prediction intervals.</div></div><div><h3>Results</h3><div>From a total of 318 articles, five studies encompassing a total of 9592 thalassemia patients were included in this study. The cumulative incidence of hepatocellular carcinoma in thalassemia patients was 1.96 % (95 % confidence interval: 0.88 %–4.27 %; prediction interval: 0.12 %–24.74 %; I<sup>2</sup> = 86.8 %). Of the 139 hepatocellular carcinoma patients, 121 were reported positive for anti-HCV, 78 for HCV RNA, three for HbsAg, and 50 positive for anti-HBV or had past infections. The liver iron concentration and ferritin level ranges in all studies were 2.95–10.5 mg/g and 3.1–2950 µg/L, respectively.</div></div><div><h3>Conclusions</h3><div>The present meta-analysis demonstrates that the incidence of hepatocellular carcinoma in thalassemia patients was high (1.96 %). It might be caused by liver infection, iron overload, or something else.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 3","pages":"Article 103934"},"PeriodicalIF":1.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144739164","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
EFFICACY OF ROXADUSTAT IN CHRONIC KIDNEY DISEASE PATIENTS NOT ON DIALYSIS WITH ANEMIA: SYSTEMATIC REVIEW AND META-ANALYSIS OF RANDOMIZED CONTROLLED TRIALS 罗沙司他对非透析性贫血慢性肾病患者的疗效:随机对照试验的系统评价和荟萃分析
IF 1.8
Hematology, Transfusion and Cell Therapy Pub Date : 2025-07-01 DOI: 10.1016/j.htct.2025.103924
Lokman Hekim Tanriverdi , Ayşe Uysal , Arzu Akyay , Yurday Öncül , Ahmet Sarici
{"title":"EFFICACY OF ROXADUSTAT IN CHRONIC KIDNEY DISEASE PATIENTS NOT ON DIALYSIS WITH ANEMIA: SYSTEMATIC REVIEW AND META-ANALYSIS OF RANDOMIZED CONTROLLED TRIALS","authors":"Lokman Hekim Tanriverdi ,&nbsp;Ayşe Uysal ,&nbsp;Arzu Akyay ,&nbsp;Yurday Öncül ,&nbsp;Ahmet Sarici","doi":"10.1016/j.htct.2025.103924","DOIUrl":"10.1016/j.htct.2025.103924","url":null,"abstract":"<div><h3>Objective</h3><div>Anemia is a common complication in patients with Chronic Kidney Disease (CKD), particularly in those not receiving dialysis. Roxadustat, a Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitor (HIF-PHI), has been investigated as a therapeutic option for anemia management in this population. This study aimed to evaluate the efficacy of Roxadustat compared to control interventions in Non-Dialysis-Dependent CKD (NDD-CKD) patients.</div></div><div><h3>Methodology</h3><div>A comprehensive literature search was conducted in Cochrane CENTRAL, Ovid Medline, PubMed, and Web of Science up to December 14, 2024. Randomized Controlled Trials (RCTs) directly comparing Roxadustat with a control group were included. Data were pooled using an inverse variance-weighted random-effects model. The primary efficacy outcome was the change in Hemoglobin (Hb) levels at weeks 24–28 and during follow-up. Subgroup analyses were performed based on the type of control intervention (Erythropoiesis-Stimulating Agents [ESAs] vs. placebo) and prior ESA use.</div></div><div><h3>Results</h3><div>A total of six RCTs, including 5,330 patients, from 520 unique records from the databases were included. Roxadustat significantly increased Hb levels during follow-up compared to the control group (Mean Difference [MD = 1.21 g/dL], 95% confidence interval [95% CI 0.45 to 1.97], I² = 99%, p = 0.0017). However, at weeks 24–28, the increase in Hb levels was not statistically significant (MD = 0.86 g/dL, 95% CI -0.11 to 1.83, I² = 99.4%, p = 0.0833). Iron-related parameters showed mixed results. Roxadustat was associated with a significant reduction in ferritin levels (MD = -38.54 ng/mL, 95% CI -68.21 to -8.87, I² = 84.1%, p = 0.0109). Conversely, Total Iron-Binding Capacity (TIBC) was significantly increased with Roxadustat treatment (MD = 20.33 μg/dL, 95% CI 1.15 to 39.51, I² = 98.5%, p = 0.0377). No significant difference was observed in serum iron (MD = 3.1 μg/dL, 95% CI -0.39 to 6.6, I² = 93.1%, p = 0.0820) and Transferrin Saturation (TSAT) levels (MD = -1.08%, 95% CI -2.42 to 0.26, I² = 40.1%, p = 0.1151) between the two groups. Subgroup analyses revealed that in placebo-controlled trials, Roxadustat significantly increased Hb levels at both weeks 24–28 and during follow-up. However, in trials comparing Roxadustat with ESAs, the changes in Hb levels were not significant at either time point.</div></div><div><h3>Conclusion</h3><div>Roxadustat reduced ferritin but increased TIBC without significantly affecting free iron and TSAT levels compared to the control group in patients with NDD-CKD.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103924"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534312","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
LOW DOSE CYTARABINE PLUS SORAFENIB IN AN ELDERLY PATIENT WITH ACUTE MYELOID LEUKEMIA 低剂量阿糖胞苷加索拉非尼治疗老年急性髓性白血病1例
IF 1.8
Hematology, Transfusion and Cell Therapy Pub Date : 2025-07-01 DOI: 10.1016/j.htct.2025.103886
Tural Mahmudov
{"title":"LOW DOSE CYTARABINE PLUS SORAFENIB IN AN ELDERLY PATIENT WITH ACUTE MYELOID LEUKEMIA","authors":"Tural Mahmudov","doi":"10.1016/j.htct.2025.103886","DOIUrl":"10.1016/j.htct.2025.103886","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Introduction&lt;/h3&gt;&lt;div&gt;Acute Myeloid Leukemia (AML) is the most common acute leukemia in adults and is generally associated with a poor prognosis. The failure of therapeutic approaches in AML treatment is attributed to various clinical characteristics of patients, disease biology, and treatment intensity. Mutations in the Fms-Like Tyrosine kinase-3 (FLT3) receptor have been reported in approximately one-third of AML cases. The most common FLT3 mutation is Internal Tandem Duplication (ITD), which has been identified in approximately 25% of adult AML patients and in 3%–5% of newly diagnosed Myelodysplastic Syndromes (MDS). FLT3-ITD is associated with high White Blood Cell (WBC) counts, elevated Lactate Dehydrogenase (LDH) levels, increased percentages of blast cells in the blood and bone marrow, and poor clinical outcomes. However, it does not appear to significantly affect the ability of adult patients to achieve Complete Remission (CR).&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Objective&lt;/h3&gt;&lt;div&gt;This case report presents the response of a 71-year-old AML patient to low-dose Cytarabine (Ara-C) combined with sorafenib treatment.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Case presentation&lt;/h3&gt;&lt;div&gt;A 71-year-old female patient presented in February 2024 with complaints of excessive thirst, fatigue, weakness, and loss of appetite. At diagnosis, leukocytosis, anemia, and thrombocytopenia were observed. Peripheral blood smear analysis revealed blast cell infiltration, and immunophenotypic studies identified markers consistent with the AML M5 subtype: CD34+/− (3.4%), CD123+, CD33+, CD13+, CD14+, CD36+, CD64+, HLA-DR+, and cMPO+. Conventional karyotyping was normal, whereas molecular analysis detected FLT3-ITD (51%, 27 bp mutant). Given the patient’s overall health status, a treatment regimen of low-dose Ara-C (20 mg BID on days 1–10) and sorafenib (400 mg on days 11–28) was initiated. Due to hematologic toxicity, dose reductions were necessary during treatment. After four cycles, bone marrow aspiration revealed a blast percentage of 0.8%, FLT3-ITD mutation was no longer detectable, and Minimal Residual Disease (MRD) negativity was achieved, confirming Complete Remission (CR). This treatment protocol was selected based on the patient's clinical condition, leading to a successful outcome.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;div&gt;FLT3-ITD-positive AML patients may benefit from low-dose Ara-C and sorafenib therapy, particularly when carefully selected based on clinical criteria. However, further comprehensive randomized prospective studies are required to better evaluate the efficacy and safety of this approach.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Discussion&lt;/h3&gt;&lt;div&gt;This case highlights the efficacy of low-dose cytarabine and sorafenib combination therapy in an elderly AML patient with FLT3-ITD mutation. FLT3-ITD mutation is a well-established marker of poor prognosis in AML and is associated with resistance to conventional therapies. In elderly AML patients, treatment decisions are often challenging due to com","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103886"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534468","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","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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