Journal of Blood Medicine最新文献

{"title":"Optimizing Care in Patients with Paroxysmal Nocturnal Hemoglobinuria: Managing Suboptimal Response and Uncontrolled Disease.","authors":"Marc Bienz, Monika Oliver, Catherine Sperlich, Christopher J Patriquin","doi":"10.2147/JBM.S561117","DOIUrl":"10.2147/JBM.S561117","url":null,"abstract":"<p><p>The treatment of patients with PNH has been revolutionized by terminal complement C5 inhibitors, which control intravascular hemolysis and thrombosis, reduce morbidity and mortality, and improve life expectancy to that approaching people without PNH. In recent years, approval of proximal inhibitors provides clinicians and patients with additional treatment options such that patients who have residual anemia, ongoing symptoms affecting quality of life, or are intolerant to terminal C5 inhibition now have options to optimize treatment. Here, we provide five questions to guide clinicians involved in the care of patients with PNH in assessing treatment response on terminal inhibitors and identifying patients who might benefit from therapy adjustments. We also provide insights into additional treatment options.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"561117"},"PeriodicalIF":2.1,"publicationDate":"2026-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12967866/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147433178","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":"Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders.","authors":"Rebecca Winkler, Ishan Herath, Radoslaw Kaczmarek, Weidong Xiao, Roland W Herzog, Paige E Severeid","doi":"10.2147/JBM.S576017","DOIUrl":"10.2147/JBM.S576017","url":null,"abstract":"<p><p>In the last few years, gene therapy, holding the promise for long-term disease correction through a one-time treatment, has transitioned from experimental research to approved medicine. Several gene therapies are now available for congenital blood disorders, notably for hemophilia and hemoglobinopathies. In this review, we discuss each of the six therapies that now have regulatory approval for treatment in the United States: Roctavian (valoctocogene roxaparvovec) for hemophilia A, Beqvez (fidanacogene elaparvovec) and Hemgenix (etranacogene dezaparvovec) for hemophilia B, Lyfgenia (lovotibeglogene autotemcel) for sickle cell disease, Zynteglo (betibeglogene autotemcel) for β-thalassemia, and Casgevy (exagamglogene autotemcel) for either sickle cell disease or β-thalassemia. The underlying principles for these treatments vary and include both in vivo and ex vivo methods, lentiviral and adeno-associated viral (AAV) vectors, and gene silencing by the CRISPR-Cas9 gene editing system. Consequently, they pose correspondingly disparate risks and benefits when compared to other treatment modalities and to each other. Although long-term effects are not yet entirely understood, given the novelty of these therapies, knowledge on patient outcomes is continuously increasing. Overall, results are very encouraging, often freeing patients from the need for coagulation factor or red blood cell (RBC) infusions, albeit that for some of these diseases there is room for further improvement in terms of safety and therapeutic durability, which may be achieved with next-generation gene therapy products. However, improvements are needed to address issues with durability of results, side effects, and accessibility of these therapies.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"576017"},"PeriodicalIF":2.1,"publicationDate":"2026-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12949972/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147344289","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":"Haematological Profile of Patients with Sickle Cell Disease in the Acholi Sub-Region, Uganda.","authors":"Silvia Awor, Jimmyy Opee, Denis Ocaya, Benard Abola, Geoffrey Maxwell Malinga, Christine Oryema, Beatrice Arwenyo, Acaye Ongwech, Proscovia Nnamuyomba, Jackie Epila, David Musoke","doi":"10.2147/JBM.S558610","DOIUrl":"10.2147/JBM.S558610","url":null,"abstract":"<p><strong>Background: </strong>Sickle cell disease (SCD) is a genetic blood disorder most prevalent in Eastern and Western Africa. With the high prevalence of SCD in northern Uganda, we set out to document the haematological profile of patients with SCD in Acholi sub-region of northern Uganda.</p><p><strong>Methods: </strong>This was a cross-sectional study at Gulu University Teaching Hospitals from February to May 2025. Patients with SCD gave blood, which was analysed at GRRH, and the results were shared with their healthcare providers. Logistic regression was done to determine the association between the haematological parameters and hydroxyurea use.</p><p><strong>Results: </strong>Four hundred eighteen blood samples were analysed. The mean age of the participants was seven years of age, and the median was 5 years of age, ranging from 1 to 28 years of age. About 95% of participants had anaemia, 92.1% erythropenia, and 92.6% low haemtocrit levels. Meanwhile, 47.9% of participants had leucocytosis and 49.1% thrombocytosis. Hydroxyurea use was associated with a normal platelet count (OR=0.35, 95% CI 0.18-0.65, p-value=0.001).</p><p><strong>Conclusion: </strong>In patients with sickle cell disease, there were increased white blood cells, platelets, and low red blood cells. That may reflect increased haemolytic activities that destroy the sickled red blood cells in low oxygen tension. Hydroxyurea use was associated with normal platelet counts.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"558610"},"PeriodicalIF":2.1,"publicationDate":"2026-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12927789/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147283833","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 an Interpretable Machine Learning Model for Predicting Thrombocythemia Risk During Third Generation Cephalosporin Therapy.","authors":"Kailei Du, Maofeng Wang, Ping Yu","doi":"10.2147/JBM.S567773","DOIUrl":"10.2147/JBM.S567773","url":null,"abstract":"<p><strong>Objective: </strong>Third-generation cephalosporins are widely used for severe infections but carry thrombocythemia risks complicating therapeutic decisions. Current predictive tools lack accuracy and clinical interpretability. This study aimed to develop an interpretable machine learning (ML) model for thrombocythemia risk stratification during cephalosporin therapy.</p><p><strong>Methods: </strong>A retrospective cohort of 45,779 adults treated with third-generation cephalosporins (2019-2023) was analyzed. After exclusions (age <18, missing data, baseline platelet anomalies), 25,707 patients were included. Thrombocythemia was defined as platelet count >400×10⁹/L within 30 days post-treatment. Predictors encompassed demographics, comorbidities, medications, and laboratory parameters. Data preprocessing included multiple imputation and stratified partitioning (70% training, 30% testing). Three ML algorithms (XGBoost, Random Forest, LightGBM) were evaluated using ROC-AUC, Brier score, and clinical utility metrics. SHAP analysis provided model interpretability.</p><p><strong>Results: </strong>XGBoost demonstrated superior performance, achieving the highest test-set discrimination (AUC=0.858, 95% CI:0.814-0.902) and calibration (Brier score=0.0088). SHAP analysis identified Baseline platelet count (PLT), red blood cell count (RBC), creatinine (CRE), daily usage frequency, and sex as key drivers. PLT was the strongest predictor (SHAP range: -1.67 to +1.48), with lower PLT exerting protective effects. RBC and CRE ranked second and third in importance, showing nonlinear risk relationships. Key clinical interactions included amplified risk from malignancies (SHAP=-0.215) and protective effects of female sex (SHAP=-0.194).</p><p><strong>Conclusion: </strong>This interpretable ML framework enables precise thrombocythemia risk prediction during cephalosporin therapy, balancing algorithmic performance with clinical actionability.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"567773"},"PeriodicalIF":2.1,"publicationDate":"2026-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12912166/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146219972","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":"Research Progress on Transitional Care for Hemophilia Patients from Pediatric to Adult Services: Focusing on Disease Severity and Care Continuity.","authors":"Siting Wu, Chunli Wang, Dan Li, Dongrong Shi","doi":"10.2147/JBM.S558200","DOIUrl":"10.2147/JBM.S558200","url":null,"abstract":"<p><p>Hemophilia is a lifelong coagulation disorder with varying severity levels, which significantly impacts management strategies. The transition period from pediatric to adult medical system represents a critical stage for ensuring continuous and tailored care. This narrative review synthesizes current evidence on transitional care for hemophilia patients, focusing on nursing-led interventions. A structured search of literature from 2010 to 2024 was conducted, drawing from key databases including PubMed, Embase, and CINAHL, as well as major guideline repositories and professional society publications. The findings from this analysis systematically summarize the challenges and intervention strategies in the transitional period. At the patient level, there are problems such as lack of self-management ability, psychosocial adaptation disorder and treatment compliance decline, particularly among those with severe disease; this period also requires a deliberate shift in responsibility from parents to the young person, a process that requires careful support for both. At the level of medical system, the transition difficulty is exacerbated by the differences between pediatric and adult medical models, the lack of multidisciplinary cooperation and the uneven distribution of resources, all of which hinder seamless care continuity. At present, the effective intervention strategies include: the implementation of structured transition plan, the training of self injection and emergency treatment skills, the provision of psychosocial support, and the establishment of nurse led multidisciplinary cooperation mode. During the transition period, nurses play the core role of assessment planning, education empowerment, coordination advocacy and psychological support. In the future, it is necessary to strengthen the development of digital tools, research on long-term health outcomes and policy support, so as to optimize the transitional nursing system and ensure the continuity of life-long health management of patients across all severity levels. While this review synthesizes global evidence, it is pertinent to note that challenges of resource access, particularly the urban-rural divide in availability of factor concentrates, novel therapies, and home-treatment programs, are often more pronounced in many developing countries, including China. The insights gathered underscore the need for standardized, nurse-coordinated transitional care protocols to inform both clinical practice and healthcare policy, aiming to bridge current gaps in service delivery and improve long-term patient outcomes.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"558200"},"PeriodicalIF":2.1,"publicationDate":"2026-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12912012/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146219970","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":"Detection of IDH1, IDH2, and NPM1 Mutations in Acute Myeloid Leukemia by High-Resolution Melting Analysis in Comparison with Direct Sequencing and MRD Detection.","authors":"Atefeh Soltani, Razieh Sharifmoghadasi, Ali Noroozi-Aghideh, Najmeh Lashgari, Alireza Moradabadi","doi":"10.2147/JBM.S567789","DOIUrl":"10.2147/JBM.S567789","url":null,"abstract":"<p><strong>Background: </strong>Acute Myeloid Leukemia (AML) is a heterogeneous type of acute leukemia. Genetic mutations are the most important prognostic factors in AML. Mutations that affect isocitrate dehydrogenase 1 and 2 (IDH1 and 2) genes are associated with poor prognosis, but the nucleophosmin 1 (NPM1) mutation is favorable. In this study, we investigated the efficiency of the High-Resolution Melting (HRM) method for the detection of NPM1, IDH1, and IDH2 mutations, and used it for minimal residual disease detection.</p><p><strong>Methods: </strong>Formalin-fixed, paraffin-embedded (FFPE) marrow biopsies of 78 patients with AML were analyzed. Mutation detection was performed using the HRM method and the results were compared with those obtained by direct sequencing. The melting temperatures (Tm) were analyzed using the blast cell percentage.</p><p><strong>Results: </strong>NPM1, IDHs mutations were detected in 28 (35.8%) and 21 (26.9%) samples, respectively. Among all IDH mutations, 13 (16.6%) and eight (10.2%) samples were harboring the IDH-1 and -2 gene mutations, respectively. Based on the HRM results, samples with higher blast cells had a minimal difference in Tm compared to samples with higher blast cells.</p><p><strong>Conclusion: </strong>Our study is generally distinguished from previous studies based on the desirable characteristics of the HRM method. Regarding mutation detection in low amounts of extracted DNA, the relationship between Tm and the blast percentage is an advantageous characteristic of the HRM method. As shown in a previous study, the use of NPM1 and IDHs for the detection of Minimal Residual Disease (MRD) by HRM is a sensitive method.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"567789"},"PeriodicalIF":2.1,"publicationDate":"2026-02-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13012557/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147512401","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":"Antioxidant Effects of CoQ10 in Transfusion-Dependent β-Thalassemia Major Patients: Implications for Ferroptosis-Related Pathways.","authors":"Kiavash Fekri, Mohammad Mehdi Mirforoughi, Yaser Salehinajafabadi, Hadi Raeisi Shahraki, Raziyeh Ershadi, Shima Rahmati","doi":"10.2147/JBM.S573174","DOIUrl":"10.2147/JBM.S573174","url":null,"abstract":"<p><strong>Background: </strong>The role of oxidative stress and ferroptosis in the pathogenesis of thalassemia major have been established and have been shown to cause tissue damage and disease progression. The lipophilic antioxidant coenzyme Q10 (CoQ10) can protect against tissue damage by restoring antioxidant enzyme function and decreasing oxidative damage. This study evaluated the effect of CoQ10 supplementation on biomarkers of ferroptosis in patients with thalassemia major.</p><p><strong>Methods: </strong>In this single-arm pre-post study, patients with confirmed thalassemia major (48) received oral CoQ10 (100 mg/day) for 8 weeks. Peripheral blood samples were collected before and after the study period for assays to measure antioxidant enzyme activity: superoxide dismutase (SOD), catalase (CAT), and glutathione peroxidase (GPx). Statistical analyses were conducted using paired <i>T</i>-test and Wilcoxon.</p><p><strong>Results: </strong>CoQ10 supplementation significantly increased SOD and GPx activity compared to baseline, (p < 0.05). No significant change was seen in CAT activity. No statistically significant differences were observed in hematological parameters and ferritin level after using CoQ10.</p><p><strong>Conclusion: </strong>CoQ10 supplementation appears to exert protective effects against ferroptosis in patients with thalassemia major, primarily by enhancing antioxidant defenses. Our study findings support the hypothesis that CoQ10 may represent a potential adjunctive therapy in mitigating oxidative stress and ferroptotic damage.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"573174"},"PeriodicalIF":2.1,"publicationDate":"2026-01-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13007963/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147512395","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":"Nafamostat Mesilate as an Anticoagulation Strategy for Heparin-Induced Thrombocytopenia: A Case Report.","authors":"Shuqin Mei, Cheng Xue, Lingling Liu, Linlin Cui, Jiayi Lv, Mengna Ruan, Zhiguo Mao, Chunlai Lu, Lijun Sun, Bing Dai","doi":"10.2147/JBM.S564615","DOIUrl":"10.2147/JBM.S564615","url":null,"abstract":"<p><p>Heparin-induced thrombocytopenia (HIT) is a serious and potentially life-threatening reaction to heparin, affecting 0.1% to 5% of patients. Those with end-stage renal disease face an and even higher risk because of repeated exposure to heparin during dialysis. HIT Type II, an immune-mediated condition, results from antibodies against heparin/platelet factor 4 (PF4) complexes, leading to platelet activation and thromboembolism. We present a 78-year-old woman with end-stage kidney disease (ESRD) who developed HIT Type II after low-molecular-weight heparin (LMWH) and unfractionated heparin (UFH) exposure during hemodialysis. Despite a negative PF4/heparin ELISA, a 4T score of 7 confirmed the presence of clinical HIT. She experienced thrombocytopenia (30×10⁹/L) and severe thrombotic events. Heparin was discontinued, and anticoagulation transitioned to argatroban and later nafamostat mesilate (NM) due to argatroban shortage. Platelet counts normalized (223×10⁹/L), and NM was effective without clotting complications. This case highlights the challenges in HIT diagnosis, emphasizing the role of clinical evaluation over laboratory tests, and underscores the utility of NM as an alternative anticoagulant in resource-limited settings. Key questions remain regarding heparin rechallenge safety, causative agent identification in multi-heparin exposure, and rapid differentiation of HIT from anaphylactoid reactions. Further studies are needed to optimize HIT management in high-risk populations.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"564615"},"PeriodicalIF":2.1,"publicationDate":"2026-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13005218/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147498925","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 a Novel Thrombosis Prediction Model for Adult Immune Thrombocytopenia (ITP-THROMBO).","authors":"Huiling Yan, Xing Hu, Lijun Zhu, Yuhan Jiang, Chen Luo, Mengya Lv, Yan Wang, Juan Tong, Changcheng Zheng","doi":"10.2147/JBM.S570135","DOIUrl":"10.2147/JBM.S570135","url":null,"abstract":"<p><strong>Purpose: </strong>Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by bleeding, yet paradoxically, it can also predispose patients to thrombotic events; therefore, investigating high-risk factors for thrombosis in ITP patients and developing a predictive model is essential.</p><p><strong>Patients and methods: </strong>A total of 1112 patients were diagnosed with ITP between January 2018 and December 2023. Excluding 216 patients under the age of 18, a total of 896 adult ITP patients were included in this study (of whom 101 developed thrombosis). Patients were randomly allocated to the training set (n=628) and validation set (n=268) in a 7:3 ratio.</p><p><strong>Results: </strong>Atrial fibrillation, peripheral vascular disease, venous thromboembolism history, pneumonia < 1 month, cerebrovascular events history, and D-dimer levels were identified as predictive factors for thrombosis in ITP patients. The six factors formed 57 unique combinations, providing robust predictive power for thrombosis in ITP under different clinical scenarios. In the training set, the area under the curve (AUC) for the nomogram was 0.656 (95% CI: 0.578-0.735) to 0.931 (95% CI: 0.902-0.960) and the AUC in the validation set was 0.539 (95% CI: 0.425-0.653) to 0.893 (95% CI: 0.828-0.957). The calibration curve demonstrated good concordance between the model's predicted probabilities and actual observed probabilities, and the decision curve analysis indicated that the model had significant clinical utility.</p><p><strong>Conclusion: </strong>This cohort study developed a simple and practical predictive model (ITP-THROMBO) for estimating thrombosis risk in ITP patients; this model facilitates rapid identification of ITP patients at high thrombotic risk, enabling timely decision-making support for personalized treatment planning.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"570135"},"PeriodicalIF":2.1,"publicationDate":"2026-01-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13003822/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147498950","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":"Empiric Imipenem/Cilastatin/Relebactam for Febrile Neutropenia After Allogeneic Hematopoietic Stem Cell Transplantation: Two Case Reports.","authors":"Dao-Xing Deng, Ting Wang, Hui Sun, Jun Kong, Yuan-Yuan Zhang, Le-Qing Cao, Xiao-Dong Mo","doi":"10.2147/JBM.S565949","DOIUrl":"10.2147/JBM.S565949","url":null,"abstract":"<p><p>Febrile neutropenia develops in approximately three-quarters of recipients of allogeneic hematopoietic stem cell transplantation (allo-HSCT). Among them, carbapenem-resistant Enterobacterales (CRE) associated mortality reaches up to 70%. Thus, rapid identification of CRE-high-risk patients and prompt empiric CRE coverage are critical. Two allo-HSCT recipients whose febrile neutropenia persisted on empiric carbapenem were promptly escalated to imipenem/cilastatin/relebactam, alone or combined with aztreonam and tigecycline-before culture confirmation. Defervescence occurred within 24-48 h, inflammatory markers declined progressively, and clinical infection control was achieved. Successful neutrophil and platelet engraftment followed. Immediate empiric imipenem/cilastatin/relebactam for CRE-high-risk allo-HSCT recipients with febrile neutropenia rapidly controlled infections and permitted successful hematopoietic recovery. Prospective studies are warranted.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"565949"},"PeriodicalIF":2.1,"publicationDate":"2026-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13003795/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147498864","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}