Annals of Hematology最新文献

{"title":"Combination of pre-transplant flow cytometry, WT1 expression, and NGS for MRD monitoring is potent in predicting the prognosis of AML receiving allogeneic transplantation.","authors":"Jie Liu, Dan Guo, Hanxi Lian, Peiwen Ding, Xin Liu, Yanqiu Zhao, Huibo Li, Shengjin Fan","doi":"10.1007/s00277-025-06384-0","DOIUrl":"10.1007/s00277-025-06384-0","url":null,"abstract":"<p><p>Minimal residual disease (MRD) monitoring has been demonstrated to important in predicting prognosis in acute myeloid leukemia (AML) receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT), but the ideal time point and method remain unclear. Our study compared the prognostic value of multiparameter flow cytometry (MFC)-based and WT1 expression-based MRD a month before allo-HSCT [HSCT(-1 m)] and after allo-HSCT [HSCT(+ 1 m)], as well as next generation sequencing (NGS)-based MRD at HSCT(-1 m), HSCT(+ 1 m), 3 and 6 months after allo-HSCT [HSCT(+ 3 m) and HSCT(+ 6 m)] among 47 AML patients undergoing allo-HSCT. The MRD status by all the methods at HSCT(-1 m) was proved as a superior indicator with prognostic significance for disease progression, compared to that at HSCT(+ 1 m). For the NGS-based MRD, HSCT(+ 6 m) seemed to be the optimal detection time point, as supported by the optimal prognostic discrimination capability and the relatively high sensitivity for disease progression prediction. Moreover, our data showed that each individual method had some limitations in predicting prognosis; however, pre-transplant MRD monitoring by the combination of MFC, WT1 and NGS could greatly increase the sensitivity (100%) of identifying disease progression and greatly improve prognostic stratification. Our study may provide insights into the optimal time point and methodology for MRD monitoring in AML following allo-HSCT.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":"2915-2926"},"PeriodicalIF":3.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143959419","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":"Emerging treatment approaches for VEXAS syndrome: a systematic review and meta-analysis.","authors":"Berkay Kilic, Efe Sacin, Muhammet Kadir Tanin, Ozgur Can Kilinc, Serdal Ugurlu","doi":"10.1007/s00277-025-06382-2","DOIUrl":"10.1007/s00277-025-06382-2","url":null,"abstract":"<p><p>VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a monogenic autoinflammatory disorder with significant morbidity and mortality. Numerous treatment options including azacitidine, JAK inhibitors, IL-6 inhibitors, anti-IL-1, and anti-TNF agents have been proposed. However, no consensus on optimal treatment algorithm has been reached. This study aims to evaluate the efficacy and safety of medical treatment options through a meta-analysis of existing data to help establish clearer guidelines for managing VEXAS. The study protocol was registered in PROSPERO (CRD42024590134). MEDLINE and EMBASE were screened from inception until March 2025. We included patients with VEXAS syndrome who received treatment with azacitidine, JAK inhibitors, IL-6 inhibitors, anti-IL-1, or anti-TNF agents. The primary outcome was the proportion of complete responders. Partial response and reported adverse events were also evaluated. A total of 16 studies and 367 patients with VEXAS syndrome were included. Concomitant myelodysplastic syndrome (MDS) was reported in 149 (40.6%) patients. Azacitidine treatment resulted in complete and partial response in 67% [95% CI (0.56,0.77)] and in 73% [95% CI (0.64,0.82)] of cases, respectively. JAK inhibitors produced a complete response in 42% [95% CI (0.33,0.52)] and partial response in 79% [95% CI (0.71,0.87)]. IL-6 inhibitors led to a complete response in 24% [95% CI (0.15,0.32)] and partial response in 72% [95% CI (0.64,0.81)]. Adverse events were frequently observed. Azacitidine demonstrated significant efficacy in patients with MDS. JAK inhibitors and IL-6 inhibitors may also be viable treatment options. Prospective clinical trials are needed for further confirmation of the results.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":"2617-2630"},"PeriodicalIF":3.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143966628","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":"A novel compound heterozygous mutation (c.64G > A and c.506-1G > A) associated with congenital coagulation factor VII deficiency: a case report and literature review.","authors":"Yu Jiao, Xiaoyi Lv, Xiaojing Yan","doi":"10.1007/s00277-025-06364-4","DOIUrl":"10.1007/s00277-025-06364-4","url":null,"abstract":"<p><p>Congenital factor VII (FVII) deficiency is a rare autosomal recessive bleeding disorder characterized by prolonged prothrombin time (PT) and reduced FVII coagulant activity (FVII: C). Here, we present the case of a middle-aged male patient with gastrointestinal bleeding, who exhibited prolonged PT and decreased FVII: C levels. Gene sequencing analysis revealed compound heterozygous mutations in the F7 gene: c.64G > A (p.V22I) and c.506-1G > A. Based on the laboratory results and gene sequencing, the patient was diagnosed as FVII deficiency. After adding recombinant activated FVII (rFVIIa) for several days, the laboratory indicators returned to normal and the bleeding symptoms were relieved. In subsequent validation studies, we also identified the c.506-1G > A mutation in his older sister and daughter. Importantly, this represents the first documented case where both mutations coexist concurrently. Additionally, our literature review reveals that approximately 50% of mutation types associated with congenital FVII deficiency are located on exon 9; however, there is no significant correlation between the reduction in FVII: C levels and severity of clinical symptoms based on EAHAD database analysis.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":"2995-3000"},"PeriodicalIF":3.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143960604","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":"Paraneoplastic leukocytoclastic vasculitis mimicking ulcus cruris as rare initial manifestation of smoldering myeloma IgG kappa.","authors":"Christina Brummer, Kirsten Utpatel, Sebastian Geis, Sebastian Haferkamp, Andreas Brosig, Markus Herrmann, Chiara Arnreich, Joachim Hahn, Tobias Pukrop, Wolfgang Herr, Sabine Gaerditz","doi":"10.1007/s00277-025-06366-2","DOIUrl":"10.1007/s00277-025-06366-2","url":null,"abstract":"<p><p>Leukocytoclastic vasculitis (LCV) has been reported as a rare paraneoplastic phenomenon associated with several hematologic disorders, including indolent lymphomas such as Waldenström macroglobulinemia. However, there are very few cases of LCV in the context of plasma cell disorders. We present the case of a 58-year-old female who developed a rapidly progressive, ulceronecrotic skin lesion on her left lower leg due to leukocytoclastic vasculitis. The lesion was initially suspected to be an ulcerative chronic wound (ulcus cruris) but represented an atypical manifestation of leukocytoclastic vasculitis as primary and only clinical sign of smoldering multiple myeloma IgG kappa. After standard induction chemoimmunotherapy with daratumumab, bortezomib, lenalidomide, and dexamethasone, the patient proceeded to high-dose chemotherapy with melphalan, followed by autologous stem cell transplantation for consolidation. Despite a bacterial skin superinfection, myeloma treatment was successfully completed without any major complications. The skin lesion healed concurrently with the reduction in paraprotein levels, and there was no need for plastic surgical intervention. LCV mimicking ulcus cruris can represent a rare and atypical initial manifestation of plasma cell neoplasia. In this case report, systemic myeloma treatment proved to be effective for inducing complete remission of advanced ulceronecrotic skin damage. This case extends the spectrum of reported monocloncal gammopathies of cutaneous significance.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":"3019-3027"},"PeriodicalIF":3.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143974010","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":"Immune responses after one versus two Influenza A/B vaccinations in patients with multiple myeloma.","authors":"Julius C Enssle, Tonio Brinkschmidt, Ralf Dürrwald, Sebastian Wolf, David Zurmeyer, Björn Steffen, Evelyn Ullrich, Thomas Oellerich, Hubert Serve, Ivana von Metzler","doi":"10.1007/s00277-025-06367-1","DOIUrl":"10.1007/s00277-025-06367-1","url":null,"abstract":"<p><p>Seasonal Influenza A/B vaccination is routinely administered in patients with Multiple Myeloma (MM) given their disease-and therapy-associated immunosuppression and risk of infection. Previous data show poor seroconversion rates after one vaccination with an increase to ~ 60% after boosting while the impact of multiple lines of therapy remains unclear. Accordingly, we performed a retrospective single-center study assessing immune responses after single or prime-boosting vaccination in 71 patients with MM treated at our institution during the 2019/20 season. Overall, 63.3% of patients with MM achieved sufficient responses after one or two Influenza A/B vaccinations. In patients receiving a prime-boost approach, significantly higher serological titers but no significant increase in responder rates were observed after the boost vaccination. Complete or very good partial remission and no immunoparesis were identified as independent predictors of sufficient serological response by multivariate regression analysis and responders were characterized by high CD19⁺ B-cell and CD4⁺ T-cell counts. Patients achieving a sufficient response only after the prime-boost approach showed significantly shorter time since high-dose chemotherapy and autologous stem-cell transplantation (HDC-ASCT). Together, this study suggests that single vaccination against Influenza A/B might be sufficient for patients with MM while a prime-boost approach might be necessary for patients with recent HDC-ASCT.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":"2813-2821"},"PeriodicalIF":3.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144075510","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":"Prognostic value of CD47 overexpression measured by flow cytometry in acute myeloid leukemia.","authors":"Vincenzo Sammartano, Anna Sicuranza, Paola Pacelli, Elena Bestoso, Adele Santoni, Corrado Zuanelli Brambilla, Marzia Defina, Alessandra Cartocci, Donatella Raspadori, Monica Bocchia","doi":"10.1007/s00277-025-06401-2","DOIUrl":"10.1007/s00277-025-06401-2","url":null,"abstract":"<p><p>The glycoprotein CD47 is an innate immune checkpoint ubiquitously expressed on all healthy cells to prevent themselves from phagocytosis. CD47 binds to its receptor SIRPα on macrophages, thus producing a signal transduction cascade which inhibits phagocytosis. CD47 is overexpressed on various solid and hematologic malignancies in order to escape the immune system. High expression of CD47 in patients with AML has been associated with poor prognosis, however, there is no standard technique to assess CD47 expression on AML blasts in clinical practice and the real prognostic value of CD47 overexpression varies among studies in the current literature. In this study, CD47 expression was evaluated by flow cytometry on AML blasts from bone marrow samples at diagnosis and reported in terms of median fluorescence intensity (MFI). Flow cytometry analysis demonstrated the expression of CD47 in all AML patients with a median MFI on leukemic blasts of 16.8 (range 2-693.63). CD47 levels on AML blasts correlated with WBC count (rs 0.403, p = 0.016), BM blasts percentage (rs 0.494, p = 0.003), PB blasts percentage (rs 0.482, p = 0.003) and LDH levels (rs 0.382, p = 0.028) and higher expression of CD47 was associated with reduced survival with a hazard ratio of 1.04 (CI: 1.01-1.08, p = 0.047). Further studies with larger sample sizes are necessary to better define the real prognostic value of CD47 overexpression in the complexity of AML tumor microenvironment and, possibly, to identify a subgroup of patients who could derive maximum benefit from emerging CD47-SIRPα blocking therapies.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":"2737-2743"},"PeriodicalIF":3.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144075515","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":"Targeted antineoplastic therapy in critically ill cancer patients: a multicenter analysis of the iCHOP registry.","authors":"Anthea Storck, Gernot Beutel, Matthias Kochanek, Peter Schellongowski, Thomas Staudinger, Nina Buchtele, Julia Cserna, Nicole Brueder, Catherina Lueck, Tobias Liebregts, Asterios Tzalavras, Jakob Hammersen, Frank Kroschinsky, Randolf Forkert, Michael G Kiehl, Michael von Bergwelt-Baildon, Judit Grans-Sibel, Franziska Bach, Jorge Garcia Borrega, Jan-Hendrik Naendrup, Alexander Shimabukuro-Vornhagen, Dennis A Eichenauer, Boris Böll","doi":"10.1007/s00277-025-06400-3","DOIUrl":"10.1007/s00277-025-06400-3","url":null,"abstract":"<p><p>Approximately 20% of intensive care unit (ICU) patients have cancer, and their prognosis has markedly improved in recent years. In addition to improved treatment in the ICU, this is a result of advancements in cancer therapies, including the use of targeted therapies (TTs), such as antibodies and small-molecule kinase inhibitors. Despite the increasing use of TT, there are currently no comprehensive studies examining critically ill cancer patients receiving TT in the ICU. We studied the clinical characteristics of a multicenter cohort of cancer patients who received TT in the ICU. To this end, we extracted data from the iCHOP Registry, comprising critically ill cancer patients from nine centers in Germany and Austria, and analyzed patient characteristics, cancer therapies, and survival outcomes. We then employed Cox proportional hazards regression and Kaplan‒Meier survival analyses to explore factors associated with mortality. Of the 1,762 cancer patients admitted to the ICU who were analyzed for this study, 106 patients (6%) received TT in the ICU, such as antibody-based treatments, kinase inhibitors and proteasome inhibitors. Although the TT recipients were younger, there were several pronounced high-risk features in the TT cohort, as indicated by a greater proportion of hematologic malignancies and autologous stem cell transplantation (SCT), a greater percentage of progressive disease and fewer patients in complete remission at ICU admission than in patients not receiving TT in the ICU. Despite these more pronounced risk features, TT patients had a slightly longer median OS than did the other patients according to Kaplan‒Meier analysis. The factors associated with mortality according to Cox proportional hazards regression analysis included advanced directives, disease progression, SOFA score, invasive mechanical ventilation (IMV), renal replacement therapy, and duration of ICU and hospital stay. Critically ill cancer patients receiving TT in the ICU had distinct characteristics but had comparable survival outcomes compared to patients receiving any other or no antineoplastic therapy in the ICU. While disease status at ICU admission remains crucial, the present study indicates the feasibility and potential benefits of TT in selected ICU patients.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":"2937-2946"},"PeriodicalIF":3.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143957814","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":"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":"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":"2631-2642"},"PeriodicalIF":3.0,"publicationDate":"2025-05-01","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":"Multisystem Langerhans cell histiocytosis in a pediatric patient: a rare case report with literature review and future directions.","authors":"Hamdah Hanifa, Sakhr Alshwayyat, Carolin Maksoud, Alhareth M Amro, Mustafa Alshwayyat, Tala Abdulsalam Alshwayyat, Mesk Alkhatib, Basil Alsaleh, Ramez M Odat, Ammar Ahmad","doi":"10.1007/s00277-025-06375-1","DOIUrl":"10.1007/s00277-025-06375-1","url":null,"abstract":"<p><p>Langerhans cell histiocytosis (LCH) is a rare disorder characterized by the proliferation of Langerhans cells that affects multiple organs. It presents variably in children, complicating diagnosis and treatment strategies. Understanding its genetic and clinical characteristics is crucial for its effective management. We report the case of a 13-year-old male with multisystem LCH involving the temporal bone, parotid gland, and lymph nodes, who presented with auricular pain and swelling. Despite the initial non-specific treatment, advanced imaging and biopsy confirmed the diagnosis. The patient underwent a treatment regimen according to the LCH4 protocol, which included Prednisolone and Vinblastine, and showed significant improvement. This case highlights the necessity of a multidisciplinary approach for diagnosing and managing LCH and illustrates the potential of genetic research and targeted therapies to improve outcomes. Future studies should explore the genetic basis of LCH and the potential links between immunization and disease onset, aiming to refine treatment protocols and enhance patient prognosis.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":"3067-3072"},"PeriodicalIF":3.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143966676","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":"Efficacy and safety of cladribine, low-dose cytarabine and venetoclax in newly diagnosed and relapsed/refractory acute myeloid leukemia: results of a single center study.","authors":"Ting Zhu, Li Wang, Hui Jiang, Shujuan Huang, Haitao Xu, Anyou Wang, Xin Liu","doi":"10.1007/s00277-025-06389-9","DOIUrl":"10.1007/s00277-025-06389-9","url":null,"abstract":"<p><p>The aim of this study was to assess the safety, response, and survival outcomes of cladribine (CLAD) + low-dose cytarabine (LDAC) + venetoclax (CAV) in patients with relapsed/refractory (R/R) and newly diagnosed acute myeloid leukemia (AML). This single-center, retrospective, non-randomized study included 46 adult patients with 29 R/R AML and 17 newly diagnosed AML who were unfit for intensive chemotherapy. All patients received the CAV regimen at our center. In the R/R group (median age 55 years, range 21-77), complete response (CR) was achieved in 44.8%, CR with incomplete blood count recovery (CRi) in 24.1%, composite complete remission (CRc, CR + CRi) and measurable residual disease (MRD) negativity in 51.7%. The median follow-up was 10.9 months, with median overall survival (OS) of 16.4 months (95% CI, 10.9-21.8). Leukopenia (62.1%) was the most common hematologic toxicity, and infection (44.8%) was the most common non-hematologic toxicity. The 30-day mortality rate was 0%, and one patient died within 60 days. In the newly diagnosed group, CR was 76.5%, CRi 17.6%, CRc 94.1%, and MRD negativity 82.3% after one induction cycle. The median OS was 15.5 months (95% CI, 11.1-19.9). Common grade 3/4 hematologic toxicities were leukopenia (76.5%), with infection (52.9%) as the most common non-hematologic toxicity. The CAV regimen demonstrated a high CRc rate and MRD negativity in R/R AML with manageable toxicity. In newly diagnosed acute myeloid leukaemia, this regimen has also demonstrated favourable efficacy, as previously reported, with tolerable haematological toxicity.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":"2745-2753"},"PeriodicalIF":3.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144101003","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}