Annals of Hematology最新文献

{"title":"Exploring perceptions in the management and treatment of polycythaemia vera in the UK","authors":"Becky Bagnall,&nbsp;Claire Woodley,&nbsp;Rozinder Bains,&nbsp;Katherine Gibson,&nbsp;Amir Nawaz,&nbsp;Jacqueline Ryan,&nbsp;Mary Frances McMullin","doi":"10.1007/s00277-025-06352-8","DOIUrl":"10.1007/s00277-025-06352-8","url":null,"abstract":"<div><p>Approximately 1140 people are diagnosed with polycythaemia vera (PV) annually in the United Kingdom (UK). Adherence to the British Society of Haematology (BSH) guidelines for PV diagnosis and management is not well understood. To explore UK’s PV diagnosis, management practices and unmet needs. A structured survey, co-developed with a UK haematology consultant, an advanced nurse practitioner and a pharmacist, was completed by 57/332 invited healthcare practitioners from July to October 2023 through 1:1 interviews conducted by Novartis Medical Science Liaisons. Results were analysed descriptively. Most respondents (68%) follow the BSH 2018 guidelines for diagnosing PV. Treatment goals are to reduce thromboembolic event risk and control haematocrit and symptoms. Most patients (68%) were receiving cytoreductive therapy (typically first-line hydroxycarbamide); 28% received antiplatelet medication and/or venesection alone. Stable patients are usually monitored every 3 months through telephone (68%), increasing to monthly when uncontrolled, mainly in-person (54%). General practitioners (56%) manage cardiovascular risks, but there is doubt over referral response. All respondents monitor symptoms, with only 19% regularly using MPN10. The greatest educational need was identifying hydroxycarbamide resistance and intolerance (58%). This survey offers insights into therapeutic approaches and areas for improvement in the UK’s PV clinical practice.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 3","pages":"1623 - 1631"},"PeriodicalIF":3.0,"publicationDate":"2025-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06352-8.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143810273","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Blinatumomab in combination with olverembatinib and concurrent intrathecal chemotherapy successfully treated a chronic myeloid leukemia relapse with central nervous system blast crisis: case report and literature review.","authors":"Ying Wang, Qiong Liu, Chun-Li Xu, Dong-Ping Huang, Yu Chen","doi":"10.1007/s00277-025-06351-9","DOIUrl":"https://doi.org/10.1007/s00277-025-06351-9","url":null,"abstract":"<p><strong>Objective: </strong>Central nervous system (CNS) blast crisis in chronic myeloid leukemia (CML) is rare and presents a significant treatment challenge due to the limited ability of many systemic therapies to penetrate the blood-brain barrier (BBB). This case highlights the need for effective treatment strategies.</p><p><strong>Case report: </strong>This case report describes a 43-year-old man diagnosed with CML who developed a CNS blast crisis followed by bone marrow relapse while receiving flumatinib.</p><p><strong>Discussion: </strong>The patient achieved complete remission (CR) in both the bone marrow and CNS after receiving a combination of blinatumomab and olverembatinib, along with weekly intrathecal chemotherapy.</p><p><strong>Conclusions: </strong>The combination of blinatumomab, olverembatinib, and concurrent intrathecal chemotherapy may be an effective treatment strategy for CML progression, particularly in cases with CNS involvement.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143810253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immunoglobulin heavy/light chain assay in the diagnosis, monitoring and follow-up of renal AL amyloidosis patients at different disease stages.","authors":"Yujie Wang, Feng Liu, Yunyun Liu, Shihui Dong, Yao Lin, Xumeng Liu, Shaoshan Liang, Dandan Liang, Feng Xu, Xiaodong Zhu, Fan Yang, Lei Ma, Xinchen Yao, Xiaoyu Wang, Caihong Zeng","doi":"10.1007/s00277-025-06345-7","DOIUrl":"https://doi.org/10.1007/s00277-025-06345-7","url":null,"abstract":"<p><p>Immunoglobulin light chain (AL) amyloidosis is a rare clonal plasma cell disorder with high rate of missed diagnosis, misdiagnosis and mortality. Conventional assays, such as serum immunofixation electrophoresis (IFE) and serum free light chain (FLC) assay, are unable to accurately detect low concentrations of monoclonal protein (M protein), especially as a patient's renal function deteriorates. The heavy/light chain (HLC) assay, a relatively new method, can quantify intact immunoglobulins in serum and has proven to be valuable in the diagnosis and monitoring of multiple myeloma (MM). However, there is limited research on its application in AL amyloidosis. In this study, we evaluate the value of HLC assay in AL amyloidosis patients at different disease stages, and compare it to the performance of IFE and FLC assay. Among 40 untreated patients, 34 (85%) were positive for IFE, 34 (85%) had an abnormal free light chain ratio (FLCr), and 31 (78%) had an abnormal heavy light chain ratio (HLCr). Among 67 serum samples obtained from 44 treated patients, 57 (85%) were positive for IFE, 9 (13%) had abnormal FLCr, and 45 (67%) had abnormal HLCr. There were 1 (14%) of 7 patients in complete response (CR), 17 (68%) of 25 patients in very good partial response (VGPR), 9 (82%) of 11 patients in partial response (PR) and 6 (75%) of 8 patients in no response (NR) showed an abnormal HLCr. Our findings identified the potential value of the HLC assay in the detection of M proteins and response and serologic residual disease monitoring.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143810187","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mutational heterogeneities in STAT3 and clonal hematopoiesis-related genes in acquired pure red cell aplasia","authors":"Toru Kawakami,&nbsp;Fumihiro Kawakami,&nbsp;Shuji Matsuzawa,&nbsp;Taku Yamane,&nbsp;Yuga Mizuno,&nbsp;Ami Asakura,&nbsp;Daigo Higano,&nbsp;Shotaro Miyairi,&nbsp;Kaoko Sakai,&nbsp;Sayaka Nishina,&nbsp;Hitoshi Sakai,&nbsp;Yasushi Kubota,&nbsp;Yumiko Higuchi,&nbsp;Hideyuki Nakazawa,&nbsp;Fumihiro Ishida","doi":"10.1007/s00277-025-06356-4","DOIUrl":"10.1007/s00277-025-06356-4","url":null,"abstract":"<div><p>Dysregulation of T cell-mediated immunity is considered a major pathophysiological mechanism in acquired pure red cell aplasia (PRCA), including idiopathic PRCA, large granular lymphocytic leukemia-associated PRCA, and thymoma-associated PRCA. Although <i>STAT3</i> mutations are frequently detected in PRCA patients, the roles of other mutational profiles and their impact on clinical characteristics remain unclear. In this study, whole-exome sequencing and targeted sequencing using a custom-designed panel were performed on 53 PRCA patients. The most frequently mutated genes were <i>STAT3</i> (36%), <i>PCLO</i> (9%), <i>TET2</i> (9%), <i>NEB</i> (6%), <i>DNMT3A</i> (6%), and <i>POT1</i> (6%). Based on genetic profiles, patients were classified into three groups: those with <i>STAT3</i> variants (group S), those without <i>STAT3</i> variants but with variants in clonal hematopoiesis (CH)-related genes (group C), and those without variants in either <i>STAT3</i> or CH-related genes (group O). Patients in group O had a higher median age compared to group S, while group S exhibited milder anemia severity than group C. Additionally, <i>POT1</i> variants were associated with the idiopathic subtype of PRCA in females, often co-occurring with <i>STAT3</i> variants. Variants in CH-related genes and other genes, including <i>STAT3</i> and <i>POT1</i>, may play crucial roles in the pathophysiology of PRCA.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 3","pages":"1471 - 1479"},"PeriodicalIF":3.0,"publicationDate":"2025-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06356-4.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143810217","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of BCOR/BCORL1 mutation on outcomes of allogeneic hematopoietic stem cell transplantation in acute myeloid leukemia patients.","authors":"YunXia Zhou, Haixiao Zhang, Xinhui Zheng, Rongli Zhang, Xin Chen, Qiaoling Ma, Donglin Yang, Jialin Wei, Aiming Pang, Yi He, Sizhou Feng, Mingzhe Han, Weihua Zhai, Erlie Jiang","doi":"10.1007/s00277-025-06346-6","DOIUrl":"https://doi.org/10.1007/s00277-025-06346-6","url":null,"abstract":"<p><p>BCOR alteration is a well-established adverse-risk marker for acute myeloid leukemia (AML) in 2022 ELN risk stratification. However, outcomes of BCOR- or BCORL1-mutated AML after allogeneic hematopoietic stem cell transplantation (allo-HSCT) are as yet poorly defined. In an 877-patient consecutive AML transplantation cohort, we found 83 (9.5%) patients with BCOR or BCORL1 mutation (BCOR/BCORL1^mut). We retrospectively evaluated the clinical characteristics and transplant outcomes of BCOR/BCORL1^mut patients and compared them with 276 patients with normal karyotype (BCOR/BCORL1^wt). Frameshift mutation was the predominant alteration of BCOR (n = 22, 39.3%), and the majority of BCORL1 was missense mutation (n = 25, 65.8%). The most common co-mutated gene of BCOR/BCORL1^mut was DNMT3A (n = 23, 27.7%). BCOR/BCORL1^mut was also associated with lower WBC counts at diagnosis (P = 0.003), shorter interval from diagnosis to transplantation (P = 0.037), and fewer achieved minimal residual disease negativity pre-transplantation (P < 0.001), compared to BCOR/BCORL1^wt. Three-year OS, DFS and CIR of BCOR/BCORL1^wt and BCOR/BCORL1^mut groups were 75.2% (95% CI, 70.0-80.8%) vs. 76.0% (95% CI, 66.0-87.5%) (HR, 0.92; 95% CI, 0.54-1.57; P = 0.77), 74.5% (95% CI, 69.4-80.1%) vs. 67.7% (95%CI, 57.0-80.4%) (HR, 1.20; 95% CI, 0.75-1.91; P = 0.46), and 12.6% (95% CI, 8.9-17.0%) vs. 24.0% (95% CI, 14.1-35.4%) (HR, 1.85; 95% CI, 1.04-3.3; P = 0.03), respectively. We also investigated the impact of the type and location of BCOR/BCORL1^mut on transplant outcomes, but no significant effect was observed. Our findings suggest that BCOR/BCORL1^mut is associated with relapse after allo-HSCT, despite no observed difference in OS, and that allo-HSCT could help to overcome the impact of BCOR/BCORL1^mut characteristics on outcomes.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143810209","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Molecular profiling of cell-free DNA from classic Hodgkin lymphoma patients identifies potential prognostic clusters and corresponds with disease dynamics","authors":"Nick Veltmaat,&nbsp;Geok-Wee Tan,&nbsp;Yujie Zhong,&nbsp;Sophie Teesink,&nbsp;Martijn Terpstra,&nbsp;Johanna Bult,&nbsp;Marcel Nijland,&nbsp;Joost Kluiver,&nbsp;Arjan Diepstra,&nbsp;Anke van den Berg,&nbsp;Wouter J. Plattel","doi":"10.1007/s00277-025-06328-8","DOIUrl":"10.1007/s00277-025-06328-8","url":null,"abstract":"<div><p>Cell-free DNA (cfDNA) analysis has advantages over tissue analysis for molecular profiling of classic Hodgkin lymphoma (cHL) at diagnosis and offers additional opportunities for sensitive non-invasive disease tracking during treatment. The aim of this study is to correlate cfDNA based molecular profiling with disease characteristics including serum Thymus and Activation Regulated Chemokine (TARC) levels and FDG-PET imaging, which are established markers of disease assessment. cfDNA isolated from plasma samples of 42 cHL patients was analyzed using low coverage whole genome and targeted next-generation sequencing. Patients were clustered in three groups based on Epstein-Barr virus (EBV) and <i>SOCS1</i> mutational status. Patients in the EBV-negative (EBV−) &amp; <i>SOCS1</i> mutated (m) cluster had more extensive disease based on significantly higher serum TARC (sTARC) levels, higher metabolic tumor volume and increased risk of treatment failure. Additionally, the median variant allele frequency and mutational load was highest in the EBV− &amp; <i>SOCS1</i>m cluster, which was validated in two external cohorts. The estimated tumor fraction and median variant allele frequency of the single nucleotide variants correlated with sTARC levels. Disease tracking over time demonstrated cfDNA level dynamics that partly resembled sTARC levels and imaging results. In conclusion, we show that cfDNA based clustering on EBV status and <i>SOCS1</i> mutational status correlates with adverse disease characteristics and increased risk of treatment failure. CfDNA-based disease tracking has the potential to serve as a sensitive tool that can complement existing response assessment methods in cHL patients.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 3","pages":"1789 - 1800"},"PeriodicalIF":3.0,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06328-8.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143810211","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The impact of obesity and its related underlying diseases on cytokine release syndrome and the efficacy of CAR-T therapy in treating B-cell malignancies.","authors":"Mengyi Du, Rosanna Tryphene Massounga Mayombo, Jiachen Liu, Yinqiang Zhang, Danying Liao, Yu Hu, Heng Mei","doi":"10.1007/s00277-025-06338-6","DOIUrl":"https://doi.org/10.1007/s00277-025-06338-6","url":null,"abstract":"<p><p>Chimeric Antigen Receptor T-cell (CAR-T) therapy has revolutionized treatment for relapsed/refractory B-cell malignancies, including B-cell Acute Lymphoblastic Leukemia (B-ALL) and Diffuse Large B-Cell Lymphoma (DLBCL). However, the influence of obesity and related comorbidities on treatment outcomes and toxicity profiles remains unclear. This retrospective study included 115 patients treated with CAR-T therapy at Union Hospital, Tongji Medical College, Huazhong University of Science and Technology from 2017 to October 2023. Patients were stratified into high-risk and low-risk groups based on Body Mass Index (BMI) and the presence of obesity-related comorbidities. Clinical outcomes, including Cytokine Release Syndrome (CRS) and Immune effector Cell-Associated Neurotoxicity Syndrome (ICANS) severity, treatment efficacy, Overall Survival (OS), and Progression-Free Survival (PFS), were analyzed. Logistic regression models assessed the relationships between covariates and clinical outcomes. The median BMI was 21.91 (IQR 19.265-24.365). Among the patients, 32 were overweight, and only one had a BMI over 30. Severe CRS occurred in 16 patients, with a higher proportion in those with obesity or related conditions (10.4% vs. 3.5%, p = 0.01). Hyperlipidemia significantly increased the risk of severe CRS (OR = 3.730, CI [1.204-11.556], p = 0.022). However, being overweight, having diabetes, hypertension, coronary heart disease, or fatty liver were not significantly associated with severe CRS. Elevated total cholesterol was moderately correlated with increased Interleukin 6 (IL-6) levels (R = 0.637, p < 0.001) and weakly with Interferon gamma (IFN-γ) (R = 0.337, p < 0.001). Besides, overweight patients had a lower proportion of CAR-T cells post-infusion (OR = 0.98, CI [0.961-1.0], p = 0.048). Obesity and related comorbidities did not significantly impact treatment efficacy. However, hyperlipidemia was associated with an increased risk of severe CRS, emphasizing the need for tailored risk management strategies in CAR-T therapy. Clinical trial: NCT02965092/ NCT03366350/ NCT04008251(ClinicalTrials.gov).</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143802246","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Molecular response with asciminib 20 mg QD in a patient intolerant to multiple TKIs.","authors":"Ruth Stuckey, Laura Navarrete Bullón, Asunción Borrero Borrego, Violeta Vidal Ballester, Carlos Rodríguez-Medina, Alejandro Morales Curbelo, Leslie González Pinedo, Melissa Torres Ochando, Alvaro Veiga Vaz, María Del Mar Perera, Cristina Bilbao-Sieyro, Juan Francisco López Rodríguez, Adrián Segura-Díaz, María Teresa Gómez-Casares","doi":"10.1007/s00277-025-06339-5","DOIUrl":"https://doi.org/10.1007/s00277-025-06339-5","url":null,"abstract":"<p><p>This case highlights a young CML patient who achieved a major molecular response (MMR) after just four months with a subtherapeutic dose of asciminib. The patient could not tolerate full-dose asciminib, or the full dose of two previous tyrosine kinase inhibitors (TKI) due to myelotoxicity, but blood counts recovered rapidly upon TKI suspension and reduced dosing enabled sustained treatment. This is the second reported case of the use of 20 mg QD asciminib, four times below the recommended dose, and the first to demonstrate efficacy at this dose. The study emphasizes asciminib's potential for overcoming treatment challenges associated with multi-resistant/intolerant CML patients.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143794651","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Incidence and risk factors of severe infections in diffuse large B-cell lymphoma patients undergoing immunochemotherapy.","authors":"Haiqiu Zhao, Rong Su, Jie Luo, Lin Liu","doi":"10.1007/s00277-025-06296-z","DOIUrl":"https://doi.org/10.1007/s00277-025-06296-z","url":null,"abstract":"<p><p>To determine the incidence and risk factors of severe infection (SI) in patients with diffuse large B-cell lymphoma (DLBCL) receiving rituximab-chemotherapy (R-C). This retrospective study was conducted at a tertiary care center in China. Patients with newly diagnosed DLBCL who were treated with R-C between January 1, 2022, and July 1, 2023, at our center were included in this study. SI was defined as an infection resulting in hospitalization. COVID-19 infections were excluded from the study. Patients were categorized as having SI or not by analyzing clinical data, comparing the characteristics of the two groups, and exploring the independent risk factors for SI using multivariate logistic analyses. A total of 300 patients were enrolled in this study, with a male-to-female ratio of 1:1.03 and a median age of 62 years (range: 20-88 years). A total of 117 patients (39.0%) developed SIs, with most (86.3%) occurring within six months of the first treatment. One-third of the infections were identified as opportunistic. Advanced stage disease (OR 2.814, 95% CI 1.603-4.939, P < 0.001), smoking history (OR 9.379, 95% CI 3.901-22.547, P < 0.001), concomitant autoimmune diseases (OR 3.730, 95% CI 1.349-10.311, P = 0.011), dexamethasone equivalent dose ≥ 15 mg/d (OR 2.436, 95% CI 1.213-4.894, P = 0.012) and prophylactic treatment with granulocyte-stimulating factors (OR 0.319, 95% CI 0.178-0.574, P < 0.001) were independently associated with the development of SI in DLBCL patients receiving R-C. Infection is a relatively common complication following R-C treatment in patients with DLBCL. Independent risk factors for SI were identified, which may aid clinicians in developing individualized infection prevention and treatment strategies.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143794649","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The efficacy of immunosuppressive therapy with or without thrombopoietin receptor agonist in elderly patients with severe aplastic anemia","authors":"Jianping Li,&nbsp;Yimeng Shi,&nbsp;Baohang Zhang,&nbsp;Wenrui Yang,&nbsp;Liping Jing,&nbsp;Li Zhang,&nbsp;Bing Han,&nbsp;Jun Shi,&nbsp;Weiping Yuan,&nbsp;Sidan Li,&nbsp;Fengkui Zhang,&nbsp;Xin Zhao","doi":"10.1007/s00277-025-06335-9","DOIUrl":"10.1007/s00277-025-06335-9","url":null,"abstract":"<div><p>Old individuals are at a high risk of developing aplastic anemia, and immunosuppressive therapy (IST) based on anti-human T-lymphocyte immunoglobulin (ATG) and cyclosporine (CsA) is recommended for treating severe aplastic anemia (SAA). Adding the thrombopoietin receptor agonist (TPO-RA) to IST could improve hematologic responses in patients with SAA; however, limited data exist for elder patients. Here, we report on the efficacy and prognostic factors associated with porcine ATG and CsA with or without TPO-RA as first-line therapy in elderly patients with SAA. Porcine ATG was administered intravenously at a dose of 20 mg/kg/d for 5 days. CsA was administered orally, maintaining plasma trough concentrations of 150–250 µg/L. Eltrombopag was administered at a dose of 75–150 mg/day, and hetrombopag was administered orally at a dose of 15 mg/day. One hundred and twenty-eight SAA patients, with a median age of 63 (60–73) years old, were included in this study, including 44 very severe aplastic anemia (VSAA) patients. All patients completed the porcine ATG treatment, and mild serum sicknesses were observed. Ten patients (2 SAA patients and 8 VSAA patients) died within 3 months of ATG initiation (early death), with severe infections being the main cause of death. Sixty-nine patients achieved a hematologic response at 6 months, with an overall response (OR) rate of 53.9%. The complete hematologic response (CR) rate at 6 months was 18.0%. The addition of TPO-RA to IST did not improve the OR and CR rates; nonetheless, early death was significantly lower (0%) in patients receiving TPO-RA compared to those not receiving TPO-RA (12%). Patients were followed up for a median of 28 (0.1–117) months. The OR rate at the last follow-up was 62.5%, while the CR rate was 36.7%. One patient progressed to myelodysplastic syndrome, one to leukemia, one to hemolytic paroxysmal nocturnal hemoglobinuria, and five developed clonal chromosomal abnormalities. The 2-year overall survival rate and failure-free survival rate were 86.0% (95% CI: 78.1-91.3%) and 70.5% (95% CI: 61.3-77.9%), respectively. A baseline neutrophil count of &lt; 0.05 × 10⁹/L was identified as an independent prognostic factor for early death. Age younger than 65 years, a baseline reticulocyte count of ≥ 7 × 10⁹/L, and the absence of fever before and within 3 months following ATG treatment were independent predictors for hematologic response at 6 months. In conclusion, treatment with porcine ATG and CsA was safe and effective in elderly patients with SAA. An extremely low baseline neutrophil count indicated a high risk of early death.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 3","pages":"1515 - 1525"},"PeriodicalIF":3.0,"publicationDate":"2025-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06335-9.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143794653","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}