Annals of Hematology最新文献

{"title":"Validity of the criteria for clinical transformation of follicular lymphoma: analysis of a mostly biopsied cohort","authors":"Hiro Tatetsu, Takafumi Shichijo, Kisato Nosaka, Yusuke Higuchi, Dai Maruyama, Masao Matsuoka, Jun-ichirou Yasunaga","doi":"10.1007/s00277-025-06357-3","DOIUrl":"10.1007/s00277-025-06357-3","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 4","pages":"2575 - 2578"},"PeriodicalIF":3.0,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06357-3.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143904803","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":"Role of IRF4 in mediating plasmablast differentiation in diffuse large B-cell lymphomas via mTORC1 pathway","authors":"Jingjing Gao,&nbsp;Chuntuan Li,&nbsp;Xingzhi Lin,&nbsp;Yanling Zhuang,&nbsp;Mingquan Wang,&nbsp;Hongjun Lin,&nbsp;Xiongpeng Zhu","doi":"10.1007/s00277-025-06273-6","DOIUrl":"10.1007/s00277-025-06273-6","url":null,"abstract":"<div><p>Autoimmune haemolytic anaemia (AIHA) is common secondary to diffuse large B-cell lymphoma (DLBCL). However, there are no reports on tumour B-cells differentiating into plasmablasts in DLBCL secondary AIHA. To state impact of the interferon regulatory factor 4 (IRF4) on DLBCL and explore the mechanism of IRF4 on plasmablast differentiation.We analysed the expression of immunity-related genes from the Gene Expression Omnibus and correlated predictors from clinical and laboratory data using R package and various statistical tools. Western blotting (WB) was used to detect protein levels in DLBCL cell lines of different subtypes to investigate the plasmablast and activation of mTORC1. To furtherly validate mTOR regulation of plasmablast differentiation, mTOR-activated and -inhibited cell models were constructed by CCK8 and flow cytometry (FCM) was used to assess the proportion of CD38 positive cells. We found that <i>IRF4</i> was highly expressed in activated B-cell-like (ABC) DLBCL vs. germinal centre B-cell-like (GCB) DLBCL. Positive MUM-1and low haemoglobin values were corrected to non-CGB patients. Plasmablast indictors (BLIMP-1, ARF4, IRE1α, and IRF4) and mTORC1 pathway indictors (mTOR, p70S6K and phosphorylated-p70S6K) were different level in ABC cell lines. After successfully constructing cell models, the proportion of CD38⁺ cells changed in mTOR-activated and -inhibited ABC-DLBCL cell models. We first pointed out that the role of the IRF4 invovling in DLBCL cell plasmablast differentiation via mTORC1 pathway. These findings could be extended to provide experimental evidence for novel treatments of secondary AIHA in ABC-DLBCL.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 4","pages":"2449 - 2459"},"PeriodicalIF":3.0,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06273-6.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143904783","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":"Follicular lymphoma grade 3A: aggressiveness and curability in an Asian cohort of 781 patients","authors":"Yaoyao Jing,&nbsp;Donghui Xing,&nbsp;Bo Yang,&nbsp;Yong Yu,&nbsp;Haifeng Zhao,&nbsp;Hongliang Yang,&nbsp;Yafei Wang,&nbsp;Bei Sun,&nbsp;Xiaofang Wang","doi":"10.1007/s00277-025-06349-3","DOIUrl":"10.1007/s00277-025-06349-3","url":null,"abstract":"<div><p>Follicular lymphoma grade 3A (FL3A) is an uncommon subtype of FL with histology falling between FL grade 1/2 (FL1/2) and FL grade 3B (FL3B). The behavior of FL3A is currently debated, with some studies suggesting it may be potentially curable while others deem it incurable. However, the lack of large-scale studies with prolonged follow-up and changes in prognosis due to the use of rituximab (R) call for further investigation into the clinical course, prognosis, and treatment of FL3A in the R era. We conducted a follow-up for a median of 7.3 years (30% exceeded 10 years) on 104 FL3A cases who received R-CHOP/R-CHOP-like treatment. We compared the characteristics, clinical behavior, and prognosis of the FL3A group with control groups of DLBCL (<i>n</i> = 478), FL3B (<i>n</i> = 45), and FL1/2 (<i>n</i> = 154). Based on R-CHOP/like therapy, FL3A had similar outcomes to FL3B and DLBCL, with comparable 5-year and 10-year PFS and OS rates. Follow-up revealed that both FL3A and FL3B showed PFS platform 6 years after treatment initiation, while DLBCL had low recurrence rates (&lt; 2%) after 5 years. FL1/2 showed persistent recurrence. No significant difference was observed in PFS among the four groups (<i>p</i> = 0.955) (Fig. 2<b>A</b>). Based on R-CHOP/R-CHOP-like regimen treatment, the OS of FL3A, FL3B and DLBCL manifested no statistically significant differences (<i>p</i> = 0.812), although FL3A appears to exhibit the poorest long-term survival in the curve, but FL1/2 showed better OS than the other three groups (<i>p</i> &lt;0.001) (Fig. 2<b>B</b>). The period most at risk for recurrence was within the first year after initial treatment. Follicular Lymphoma International Prognostic Index (FLIPI) predicted PFS and OS for low-risk patients in FL3A, while FLIPI-2 was more effective in describing prognosis for high-risk patients in terms of PFS and OS. Based on treatment with R-CHOP/like, the prognosis of FL3A is comparable to that of FL3B and DLBCL. FL3A exhibits a PFS platform at 6 years post-initial treatment. FL3A had potential disease aggressiveness and curability.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 4","pages":"2437 - 2448"},"PeriodicalIF":3.0,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06349-3.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143904784","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":"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,&nbsp;Feng Liu,&nbsp;Yunyun Liu,&nbsp;Shihui Dong,&nbsp;Yao lin,&nbsp;Xumeng Liu,&nbsp;Shaoshan Liang,&nbsp;Dandan Liang,&nbsp;Feng Xu,&nbsp;Xiaodong Zhu,&nbsp;Fan Yang,&nbsp;Lei Ma,&nbsp;Xinchen Yao,&nbsp;Xiaoyu Wang,&nbsp;Caihong Zeng","doi":"10.1007/s00277-025-06345-7","DOIUrl":"10.1007/s00277-025-06345-7","url":null,"abstract":"<div><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></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 4","pages":"2287 - 2295"},"PeriodicalIF":3.0,"publicationDate":"2025-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06345-7.pdf","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":"OA","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":"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}