Hematology最新文献

{"title":"Revaccination following CAR-T therapy: a needs assessment.","authors":"Robert Clayden, Barbara Gunka, Brittany Salter, Audrey Y H Dong, Kylie Lepic, Gwynivere Davies, Amaris Balitsky","doi":"10.1080/16078454.2025.2519865","DOIUrl":"https://doi.org/10.1080/16078454.2025.2519865","url":null,"abstract":"<p><strong>Background: </strong>CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy has transformed treatment for relapsed/refractory large B-cell lymphoma, offering promising remission rates. However, it carries significant infectious risks, necessitating revaccination for infection prevention.</p><p><strong>Methods: </strong>This single-center quality improvement study aimed to (1) assess revaccination uptake and barriers in CAR-T recipients through interviews and focus groups, and (2) use qualitative insights to develop an educational handout to improve revaccination adherence. The study was conducted at Juravinski Hospital and Cancer Centre, Hamilton, Ontario, and enrolled 22 patients who received CAR-T between January 2020 and June 2023. Participants completed a survey evaluating their revaccination experience, which informed the development of a patient-facing educational handout. A focus group of survey participants then reviewed the handout and offered feedback on its clarity and usefulness.</p><p><strong>Results: </strong>Only 50% of participants proceeded with revaccination. Key barriers included limited awareness among primary care providers (PCPs), poor communication between hematologists and PCPs, and logistical difficulties. The focus group highlighted gaps in understanding post-CAR-T immunization needs and emphasized the importance of patient and physician education. Participants supported the creation of a concise handout outlining the revaccination schedule and clarified the PCP's role in coordination.</p><p><strong>Conclusion: </strong>This study underscores the need for improved communication across care providers and accessible patient education to support post-CAR-T revaccination. Future efforts should focus on implementing and evaluating targeted educational tools to enhance vaccine uptake in this high-risk population..</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2519865"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144474996","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Analysis of risk factors for relapse after allogeneic hematopoietic stem cell transplantation in acute leukemia.","authors":"Jiawen Wang, Han Zhu, Kourong Miao","doi":"10.1080/16078454.2025.2532915","DOIUrl":"https://doi.org/10.1080/16078454.2025.2532915","url":null,"abstract":"<p><strong>Background: </strong>Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective, and in many cases, the only treatment for curing malignant blood diseases. However, post-transplant relapse is the primary factor affecting survival rates.</p><p><strong>Methods: </strong>We conducted a retrospective analysis of 123 patients with acute leukemia undergoing allo-HSCT at Jiangsu Provincial People's Hospital from August 2017 to June 2023 to discuss the risk factors for relapse in acute leukemia (AL) patients after allo-HSCT.</p><p><strong>Results: </strong>Among the 123 patients, 41experienced relapse, with 30 cases of relapse within one year. Multivariable analysis showed that pre-transplant MRD (+) and NR (HR 2.32, 95%CI 1.18-4.54, <i>P</i> = 0.014), post-transplant MRD (+) (HR 2.13, 95%CI 1.04-4.36, <i>P</i> = 0.016), cGVHD (-) (HR 0.48, 95%CI 0.23-0.97, <i>P</i> = 0.041) and age < 40 (HR 0.44, 95%CI 0.23-0.83, <i>P</i> = 0.011) were independent risk factors for relapse after allo-HSCT.</p><p><strong>Conclusions: </strong>Data from our center indicated that pre-transplant MRD (+) and NR, post-transplant MRD (+), cGVHD (-) and age < 40 are independent risk factors affecting relapse after allo-HSCT.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2532915"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144690061","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic value of miR-106a and miR-20a in AML patients with chemotherapy or allo-HSCT treatment.","authors":"Yue Liu, Jiayu Liu, Yuehua Feng, Xiaoguang Xu, Yingjie Miao, Huijuan Chen, Yu Zhou, Yijun Pan, Yan Liu, Weiying Gu, Yang Cao","doi":"10.1080/16078454.2025.2533577","DOIUrl":"https://doi.org/10.1080/16078454.2025.2533577","url":null,"abstract":"<p><strong>Background: </strong>MiR-106a and miR-20a (miR-17 family members) are frequently dysregulated in carcinogenesis, but their prognostic significance in acute myeloid leukemia (AML) remains unclear.</p><p><strong>Methods: </strong>We analyzed miR-106a and miR-20a expression in bone marrow from 115 AML patients and 45 healthy controls using qRT-PCR. Additionally, we utilized TCGA data (n=188) to assess the association of these miRNAs with clinical factors and outcomes. Prognostic analysis evaluated the impact of miR-106a and miR-20a on overall survival (OS) and event-free survival (EFS). Differentially expressed genes (DEGs) were identified using Limma. GO and KEGG pathway analyses were performed by DAVID. GSEA and PPI were constructed using ClusterProfiler and STRING database.</p><p><strong>Results: </strong>MiR-106a and miR-20a elevated in AML <i>versus</i> healthy controls. In chemotherapy group, miR-106a^high or miR-20a^high predicted poor OS and EFS, with dual-high expression conferring the worst outcome. In allo-HSCT group, miR-106a^high or miR-20a^high predicted poor OS but similar EFS, with dual-high cases showing the worst OS. In the miR-106a^high or miR-20a^high group, allo-HSCT prolonged OS (but not EFS) <i>versus</i> chemotherapy. In the miR-106a^low or miR-20a^low group, there were no obvious differences in OS or EFS between the chemotherapy and allo-HSCT regimens. Multivariable analyses confirmed miR-106a/miR-20a signature as an independent prognostic marker. Moreover, we identified 706 signature-associated DEGs. Bioinformatic analysis illuminated the involvement of miR-106a and miR-20a in regulating diverse biological processes and signaling pathways.</p><p><strong>Conclusions: </strong>MiR-106a and miR-20a are promising AML prognostic biomarkers for adverse outcome. The combined signature improves risk stratification and guides therapy selection (e.g., ⁣allo-HSCT for high-risk cases).</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2533577"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144690062","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Meta-analysis of the prognostic efficacy of daratumumab combined with standard therapy in high-risk multiple myeloma.","authors":"Hongmei Zhou, Min Zhang, Yi Jin, Xianghua Lang","doi":"10.1080/16078454.2025.2532893","DOIUrl":"10.1080/16078454.2025.2532893","url":null,"abstract":"<p><strong>Objective: </strong>This meta-analysis aims to evaluate the efficacy of daratumumab (DAR) in combination with standard therapy for the treatment of high-risk multiple myeloma (HRMM), offering evidence-based insights to guide clinical decision-making.</p><p><strong>Methods: </strong>A comprehensive search was conducted across literature databases to identify studies investigating the use of DAR in HRMM. After removing duplicates, titles, abstracts, and full texts were screened. Study quality was assessed using the Cochrane Handbook for Systematic Reviews of Interventions (version 5.1.0). Studies with high risk of bias were excluded. Data on authors, publication dates, study populations, objective response rate (ORR), progression-free survival (PFS), overall survival (OS), and adverse events were analyzed using RevMan 5.3 software.</p><p><strong>Results: </strong>11 studies were included, comprising 2330 patients in the control group (standard therapy) and 2663 patients in the experimental group (DAR plus standard therapy). All studies were rated as having a low to moderate risk of bias. Meta-analysis showed that ORR, PFS, and OS were significantly higher in the experimental group (<i>P</i> < 0.05). The incidence of anemia was lower in the DAR group, while thrombocytopenia and neutropenia were more frequent (<i>P</i> < 0.05). Funnel plot analysis suggested minimal publication bias.</p><p><strong>Conclusion: </strong>The combination of DAR with standard therapy significantly enhances clinical outcomes in HRMM patients, resulting in prolonged PFS and OS, with manageable adverse effects.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2532893"},"PeriodicalIF":1.6,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144707329","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A rare -α^27.6 deletion compounded with the hemoglobin constant spring mutation identified in a Chinese couple.","authors":"Wei Li, Zhao-Yi Guo, Zi-Han Xiu, Min Long, Yan Xiao, Li-Yi Liu, Yu-Chen Chen, Si-Fan Zeng, Jing Zhang, Min Zhang","doi":"10.1080/16078454.2025.2485694","DOIUrl":"10.1080/16078454.2025.2485694","url":null,"abstract":"<p><strong>Background: </strong>Thalassemia is a common hemoglobin disorder caused by genetic defects in a single autosomal gene. Based on the deficient globin strand, it can be classified as α-thalassemia or β-thalassemia. The 27.6 kb deletion on α-globin related gene cluster (-α^27.6) is a rare α-thalassemia variant discovered in 2011, which could affect the detection of common α-thalassemia variants and cause misdiagnosis.</p><p><strong>Case presentation: </strong>An α-thalassemia variant carrying a Chinese couple was reported in this study. The wife was diagnosed at another hospital as α^CSα/α^CSα but did not manifest corresponding symptoms. After further examinations and in-depth analyses of the results, the genotype of the wife was finally confirmed to be -α^27.6/α^CSα. Meanwhile, the genotype of the husband was diagnosed as α^CSα/αα. The couple requested prenatal diagnosis in the worry of α-thalassemia caused by α^CSα/α^CSα. Genetic tests on the amniotic fluid reported a mild thalassemia-related genotype of α^CSα/αα, on which our suggestion of continuing pregnancy was based.</p><p><strong>Conclusion: </strong>The -α^27.6/α^CSα case and related manifestations were first reported here expanding the gene spectrum of thalassemia. Such genotype can be misdiagnosed as α^CSα/α^CSα causing inaccurate estimations of thalassemia risk. To avoid these misdiagnoses, genetic tests for deletions in the related regions were advised when inconsistencies between the genotype and the phenotype were discovered.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2485694"},"PeriodicalIF":1.6,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144005847","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Emerging molecular targets in deep vein thrombosis: from inflammation to coagulation.","authors":"Zhuying Zhang, Jiaqi Hu, Yanfeng Bai, Wenqi Liang, Yexia Jin","doi":"10.1080/16078454.2025.2548735","DOIUrl":"10.1080/16078454.2025.2548735","url":null,"abstract":"<p><p>Deep vein thrombosis (DVT), a prevalent vascular disorder driven by venous stasis, endothelial injury, and hypercoagulability, imposes a significant global health burden due to life-threatening complications like pulmonary embolism. Recent advances highlight inflammation as a pivotal contributor to DVT pathogenesis, intricately linked with coagulation through immunothrombosis. This review synthesizes emerging molecular targets bridging these pathways, focusing on neutrophil extracellular traps (NETs), peptidylarginine deiminase 4 (PAD4), P-selectin, high-mobility group box 1 (HMGB1), tissue factor (TF), complement C3, and the NLRP3 inflammasome. NETs provide a procoagulant scaffold for fibrin deposition, activate Factor XII, and stabilize thrombi. PAD4 catalyzes NET formation via histone citrullination, while P-selectin mediates leukocyte adhesion and thromboinflammation. HMGB1 amplifies thrombosis by inducing NETosis and TF expression, and leukocyte-derived TF challenges traditional vessel-injury paradigms. Complement C3 and NLRP3 activation further propagate inflammation-coagulation crosstalk. Despite promising preclinical data, therapeutic translation faces challenges, including species differences, off-target effects, and balancing efficacy with immune defense. Innovations in single-cell RNA sequencing and CRISPR screening offer new avenues for target discovery. Targeting these molecules may enable bleeding-sparing therapies, advancing DVT management beyond conventional anticoagulants.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2548735"},"PeriodicalIF":1.6,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144952113","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety and efficacy of CD19-targeted CAR-T-cell therapy for patients with relapsed or refractory TCF3-PBX1 fusion gene-positive B-ALL.","authors":"Can Huang, Yuanyin Teng, Tingting Yang, Mingming Zhang, Yinghui Yu, Shan Fu, Jingjing Feng, He Huang, Yongxian Hu","doi":"10.1080/16078454.2025.2550815","DOIUrl":"10.1080/16078454.2025.2550815","url":null,"abstract":"<p><p><b>Background:</b> Chimeric antigen receptor (CAR)-T therapy has shown significant success in the treatment of relapsed or refractory acute lymphoblastic leukemia (r/r ALL). However, its role in patients with the TCF3-PBX1 fusion gene - which generally exhibit poor prognostic indicators - remains uncertain. <b>Patients and methods:</b> From September 2016 to March 2023, 7 patients with r/r ALL positive for the TCF3-PBX1 fusion gene underwent CD19 CAR-T-cell therapy at the First Affiliated Hospital of Zhejiang University School of Medicine. The safety and efficacy of the treatment were evaluated. <b>Results:</b> Four of the 7 patients experienced CAR-T-cell expansion in vivo, with a median peak percentage of CD3+ T-cell expansion of 64.9% (range: 29.1-78.1%). These 4 patients experienced grade 1 cytokine release syndrome. For the efficacy assessment, 4 patients with CAR-T-cell expansion achieved complete remission (CR), whereas the other 3 did not respond and ultimately died of disease progression. Among the 4 patients who achieved CR, 1 patient with a history of allogeneic stem cell transplantation (allo-HSCT) did not bridge to secondary allo-HSCT and relapsed 7 months after CAR-T-cell infusion. The other 3 CR patients successfully bridged to allo-HSCT; however, 2 of them relapsed post-allo-HSCT. One of the relapsed patients achieved remission after receiving donor-derived CAR-T-cell infusion and has maintained CR to date. Another patient died of disease progression. The remaining patient has achieved sustained remission to date. <b>Conclusion:</b> Our study indicates that CD19 CAR-T therapy is safe and effective in TCF3-PBX1-positive r/r B-ALL. Further studies in larger cohorts are warranted to confirm these observations .</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2550815"},"PeriodicalIF":1.6,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144952264","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Iraqi consensus recommendations for the diagnosis and management of immune thrombocytopenia.","authors":"Ahmed Mjali, Bassam Francis Matti, Waleed Ghanima, Drew Provan, Nareen Tawfeeq Abbas, Alaadin Sahham Naji, Mohammed Saleem Abbas, Alaa Fadhil Alwan, Waseem F Al Tameemi, Tareq Abdullah Saleh, Mazin A Shubber, Ahmed Sabah Noori, Raghad Majid Al-Saeed, Amer Shareef Mohammed, Mohammed Ahmed Al-Anssari, Qutaiba M Dawood","doi":"10.1080/16078454.2025.2555045","DOIUrl":"https://doi.org/10.1080/16078454.2025.2555045","url":null,"abstract":"<p><strong>Background and objectives: </strong>The management of ITP has evolved with evidence-based international guidelines. However, Iraq's unique challenges, including variations in clinical practice and treatment disparities, necessitate localized guidance to bridge global recommendations and real-world practice. This expert consensus aims to provide a comprehensive and practical framework for diagnosing and managing ITP within the Iraqi healthcare setting.</p><p><strong>Methods: </strong>A 16-member multidisciplinary ITP specialist panel (Iraq, Norway, UK), including hematologists, laboratory experts, and related specialists, conducted a systematic PubMed / PubMed Central / Embase review (January 2003-December 2024) using key terms 'primary immune thrombocytopenia' and 'idiopathic thrombocytopenic purpura' (English human studies, excluding conference abstracts). Using a modified Delphi method, consensus was reached on 43 ITP diagnosis/treatment/management statements. After a single voting round, the panel refined the recommendations to ensure their applicability to Iraq's healthcare system. Final recommendations were categorized using evidence grading system.</p><p><strong>Results: </strong>Corticosteroids were the preferred first-line therapy, with intravenous immunoglobulin (IVIG) reserved for urgent platelet elevation. The panel discouraged prolonged corticosteroid use due to adverse effects and defined clear criteria for second-line therapies. Thrombopoietin receptor agonists (TPO-RAs) and rituximab were recommended second-line, while splenectomy was considered a last resort, only after the failure of multiple medical therapies. Special populations received tailored recommendations, including pregnant patients, pediatric cases, and high-thrombosis-risk individuals. Recommendations incorporated quality of life, emphasizing patient-centered care and minimizing unnecessary medication exposure.</p><p><strong>Conclusion: </strong>This expert consensus provides a structured, evidence-informed approach to ITP diagnosis and management in Iraq, balancing best practices with local healthcare realities.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2555045"},"PeriodicalIF":1.6,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145080409","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Identification of RUVBL2 as a novel biomarker to predict the prognosis and drug sensitivity in multiple myeloma based on ferroptosis genes.","authors":"Sishi Tang, Xinyi Long, Fangfang Li, Siyi Jiang, Yunfeng Fu, Jing Liu","doi":"10.1080/16078454.2025.2467499","DOIUrl":"10.1080/16078454.2025.2467499","url":null,"abstract":"<p><strong>Background: </strong>Multiple myeloma (MM) is a hematological malignancy with the proliferation of malignant plasma cells. Numerous studies have highlighted the critical role of ferroptosis in MM. However, how to use ferroptosis-related genes (FRGs) for prognostic prediction and treatment guidance in MM remains unknown.</p><p><strong>Methods: </strong>By analysis of GEO databases, the prognostic gene was identified and a therapeutic strategy for MM patients based on FRGs was explored. A total of 12 FRGs were identified, utilizing the STRING database and Cytoscape software, and the PPI networks were constructed to identify hub genes and further functional enrichment analyses. Based on the aforementioned data, this study analyzed the expression of RUVBL2 in MM patients by qRT-PCR and Western blotting. To validate the functional role of RUVBL2 in the MM cells, cellular experiments were ultimately conducted.</p><p><strong>Results: </strong>The analysis highlighted six hub genes, including TP53, MCM5, TLR4, RUVBL2, GCLM and ITGA6, and functional enrichment analyses indicating enrichment in DNA replication, regulation of apoptotic signaling pathway and PI3K/AKT signaling pathway. Prognostic analysis indicated that TP53, RUVBL2, and MCM5 are associated with MM prognosis, with RUVBL2 displaying a notable area under the curve (AUC) of 0.823 in ROC analysis. The study first determined that RUVBL2 is highly expressed in MM, siRUVBL2-mediated deletion of RUVBL2 inhibited proliferation, promoted apoptosis and increased the sensitivity of BTZ in MM cells, and also overcame BTZ resistance in CD138⁺ primary cells from MM patients.</p><p><strong>Conclusions: </strong>Our study first suggested that RUVBL2 may be regarded as potential therapeutic targets and prognostic value in MM.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2467499"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476081","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost utility analysis of adult patients with severe aplastic anemia: a single-center study.","authors":"Lin Chen, Liwei Fang, Zhexiang Kuang, Jing Xu, Chuan Wang","doi":"10.1080/16078454.2025.2492925","DOIUrl":"https://doi.org/10.1080/16078454.2025.2492925","url":null,"abstract":"<p><strong>Objective: </strong>There is a need for real-world studies in China to help address evidence gaps supporting precise clinical decision-making with respect to the ideal treatment options for patients with severe aplastic anemia (SAA). Accordingly, we objectively evaluated the efficacy of cyclosporine A (CsA) + antilymphocyte globulin (ALG) versus CsA + thrombopoietin receptor agonist (TPO-RA) for such patients.</p><p><strong>Methods: </strong>A cost-utility analysis (CUA) was conducted to compare the quality-adjusted life years (QALY) and total costs associated with the two treatment regimens. Patient utility values were derived from the European Quality-of-Life 5 Dimensions 3 Level Version (EQ-5D-3L) using the Japanese time trade-off conversion method.</p><p><strong>Results: </strong>Patients receiving the CsA + ALG regimen reported higher subjective well-being than those treated with the CsA + TPO-RA regimen from the time of hospital admission through 6 months of follow-up. In addition, the quality-of-life of patients in the CsA + ALG group was significantly higher than that of the patients in the CsA + TPO-RA group, with a difference of 0.08 QALY (<i>P</i> < 0.01). However, the total cost of the CsA + ALG regimen was nearly twice that of the CsA + TPO-RA regimen. The incremental cost per QALY gained with the CsA + ALG regimen relative to the CsA + TPO-RA regimen was 1.63 million yuan.</p><p><strong>Conclusions: </strong>This study utilized CUA to comparatively assess the cost-effectiveness of CsA + ALG and CsA + TPO-RA regimens in the treatment of SAA. Although both regimens were found to be effective, the CsA + TPO-RA regimen presented a viable treatment option with assured therapeutic efficacy while reducing the financial burden, thereby offering greater benefits for patients with SAA and alleviating the societal healthcare costs.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2492925"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144003207","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}