Annals of Hematology最新文献

{"title":"Impact of secondary-type mutations on the prognosis of AML patients with NPM1 mutation: a systematic review and meta-analysis.","authors":"Tao Wang, Lei Zhao, Hongbing Ma","doi":"10.1007/s00277-025-06431-w","DOIUrl":"https://doi.org/10.1007/s00277-025-06431-w","url":null,"abstract":"<p><p>Nucleophosmin 1 (NPM1) mutation is commonly associated with a favorable prognosis in acute myeloid leukemia (AML). Conversely, secondary mutations such as those in ASXL1, RUNX1, EZH2, and SRSF2 are generally linked to poor outcomes. The combined prognostic impact of NPM1 and secondary mutations in AML patients remains unclear. This meta-analysis aimed to evaluate the prognostic significance of secondary mutations in AML patients harboring NPM1 mutation. A systematic literature search was conducted following PRISMA guidelines, identifying studies published up to June 2024 from databases such as PubMed, Web of Science, and the Cochrane Library. The inclusion criteria included adult AML patients with confirmed NPM1 mutation, detailed reporting of secondary mutations, and comparative prognostic outcomes. Fourteen high-quality studies from twelve publications were included, encompassing 4,022 patients who all carried NPM1 mutations; among these, 618 also harbored secondary mutations. Data extraction and quality assessment were performed independently by two researchers via the Newcastle-Ottawa Scale (NOS). Statistical analyses involved fixed-effects models due to low heterogeneity (I²=0% for OS and I²=35% for EFS/RFS). Publication bias and sensitivity analyses confirmed the robustness of the findings. Secondary mutations were not significantly associated with OS (HR = 1.16, 95% CI: 0.99-1.35, p = 0.07) or EFS/RFS (HR = 1.15, 95% CI: 0.96-1.38, p = 0.14) in the overall NPM1-mutated AML population. However, within the European LeukemiaNet (ELN) favorable prognosis group, the presence of secondary mutations was significantly associated with reduced OS (HR = 1.95, 95% CI: 1.39-2.73, p < 0.01). Subgroup analyses based on median age, geographical region, and allogeneic hematopoietic stem cell transplantation (allo-HSCT) rates did not reveal significant modifiers of the prognostic impact of secondary mutations. Secondary mutations do not significantly adversely affect OS or EFS/RFS in the general population of AML patients with NPM1 mutation. Notably, within the ELN favorable prognosis group, secondary mutations are associated with markedly poorer OS, highlighting the need for careful prognostic assessment and potential treatment strategy adjustments in this subset of patients.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144155662","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":"Correction to: Mutational heterogeneities in STAT3 and clonal hematopoiesis-related genes in acquired pure red cell aplasia.","authors":"Toru Kawakami, Fumihiro Kawakami, Shuji Matsuzawa, Taku Yamane, Yuga Mizuno, Ami Asakura, Daigo Higano, Shotaro Miyairi, Kaoko Sakai, Sayaka Nishina, Hitoshi Sakai, Yasushi Kubota, Yumiko Higuchi, Hideyuki Nakazawa, Fumihiro Ishida","doi":"10.1007/s00277-025-06435-6","DOIUrl":"https://doi.org/10.1007/s00277-025-06435-6","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144155619","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 expression patterns and prognostic significance of PD-1 and TIM-3 on T cells and the differentiated subsets in acute myeloid leukemia.","authors":"Kai Sun, Zong-Yan Shi, Ya-Zhe Wang, Dai-Hong Xie, Yan-Rong Liu, Qian Jiang, Hao Jiang, Xiao-Jun Huang, Ya-Zhen Qin","doi":"10.1007/s00277-025-06418-7","DOIUrl":"https://doi.org/10.1007/s00277-025-06418-7","url":null,"abstract":"<p><p>Researches on the immune checkpoint molecule and its clinical application in acute myeloid leukemia (AML) fall behind solid tumors. The expression patterns and prognostic significance of immune checkpoint molecules across T cells and T-cell differentiated subsets in AML require further elucidation. In the present study, bone marrow (BM) samples from 165 newly diagnosed AML patients and 12 healthy donors (HDs) were tested PD-1 and TIM-3 expression on CD4⁺ and CD8⁺T cells as well as their differentiated subsets by multi-parameter flow cytometry. Compared with HDs, PD-1 was significantly overexpressed on total CD4⁺T cells and their major constituent subsets (naïve, central memory, and effector memory T cells; all P < 0.05), whereas TIM-3 was overexpressed on both total CD4⁺ and CD8⁺T cells, as well as all differentiated subsets (all P < 0.05). Both high expressions of PD-1 on CD8⁺T and TIM-3 on CD4⁺T cells were associated with poor relapse-free survival (RFS) and event-free survival (EFS) (all P < 0.05). In addition, high PD-1 expression on CD8⁺T cells independently predicted poorer RFS and EFS (P = 0.010 and 0.0011). Further stratification by T-cell subsets revealed that high PD-1 expression on naïve CD4⁺T cells and TIM-3 on effector CD4⁺T cells emerged as independent adverse prognostic factors for RFS (P = 0.018 and P = 0.034, respectively), replacing the prognostic impact of PD-1 on total CD8⁺T cells. However, high PD-1 expression on CD8⁺T cells remained the sole independent predictor of EFS (P = 0.0065). In conclusion, the expression patterns of PD-1 and TIM-3 in the BM T cells and their differentiated sub-populations in newly diagnosed AML patients were distinct from HDs, and predicted outcomes.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144149030","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":"Relapses and outcomes of systemic chemo-free therapies combined with intravitreous methotrexate in isolated primary vitreoretinal lymphoma: an analysis based on two prospective cohort studies.","authors":"Hongyun Chen, Zhe Zhuang, Xiao Zhang, Daobin Zhou, Meifen Zhang, Wei Zhang","doi":"10.1007/s00277-025-06414-x","DOIUrl":"https://doi.org/10.1007/s00277-025-06414-x","url":null,"abstract":"<p><p>The definitive therapeutic paradigm for primary vitreoretinal lymphoma (PVRL) remains controversial, with ongoing debates surrounding the comparative efficacy of localized ocular therapy, high-dose systemic chemotherapy, and combined-modality approaches. The benefit of systemic administration of methotrexate is unambiguous and severe adverse events related to intense chemotherapy are reported. Immunomodulators such as lenalidomide and Bruton's tyrosine kinase (BTK) inhibitors, like zanubrutinib, have not been explored for their safety and efficacy as first-line treatments for PVRL. This study aimed to analyze the safety and efficacy of chemo-free combination regimens in newly diagnosed PVRL patients. In our study, systemic chemo-free regimens were administrated as induction therapy, including the ZR regimen (zanubrutinib and rituximab), and the R2 regimen (lenalidomide and rituximab). Of the 44 patients, 41 (93.2%) achieved complete remission following induction therapy. No severe systemic adverse events were observed. After a median follow-up of 25.1 months, 17 patients (38.6%) suffered relapses, including one intraocular relapse and 17 brain relapses. By the final follow-up, 5 patients (11.4%) had died from disease progression. The median PFS and OS were 22.1 and 71.5 months, respectively. The ZR regimen demonstrated a longer PFS compared to the R2 regimen (10.9 months vs. not reached; log-rank test, P = 0.00044), positioning it as a promising treatment option for PVRL patients.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144141051","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":"Matching-adjusted indirect comparison of CPX- 351 in secondary Acute Myeloid Leukemia between the registrative trial and a real-life study.","authors":"Luana Fianchi, Alfonso Piciocchi, Fabio Guolo, Francesco Marchesi, Giovanni Marsili, Chiara Cattaneo, Michele Gottardi, Francesco Restuccia, Anna Candoni, Elettra Ortu La Barbera, Rita Fazzi, Crescenza Pasciolla, Olimpia Finizio, Nicola Fracchiolla, Mario Delia, Federica Lessi, Michela Dargenio, Valentina Bonuomo, Maria Ilaria Del Principe, Patrizia Zappasodi, Marco Picardi, Claudia Basilico, Monica Piedimonte, Paola Minetto, Patrizia Chiusolo, Lucia Prezioso, Caterina Buquicchio, Lorella Melillo, Daniele Zama, Francesca Farina, Valentina Mancini, Michela Rondoni, Alessandro Busca, Livio Pagano","doi":"10.1007/s00277-025-06381-3","DOIUrl":"https://doi.org/10.1007/s00277-025-06381-3","url":null,"abstract":"<p><p>A real-life study on CPX-351 and the standard arm ('7 + 3') of the CPX-351 registrative trial in adults with secondary Acute Myeloid Leukemia were compared by an unanchored Matching-adjusted indirect comparison (MAIC), in order to evaluate the efficacy and toxicity of CPX-351. Results of this study are important to confirm the role of CPX-351 in significantly improving survival and remission rates compared with '7 + 3' with a good safety profile in AML patients with high-risk features, a target group traditionally with a very poor prognosis. Moreover, this pilot analysis underlines the potentiality of the statistical method to compare studies with strong differences.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144135948","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":"Platelet-to-Neutrophil ratio as a predictor of risk of complications in sickle cell disease: a valuable insight for resource-limited settings.","authors":"Chilota Efobi, Nnanna Ukpai, Onyinye Ezinne Eze, Bruno Basil","doi":"10.1007/s00277-025-06419-6","DOIUrl":"https://doi.org/10.1007/s00277-025-06419-6","url":null,"abstract":"<p><p>Sickle cell disease (SCD) presents significant clinical challenges, particularly in resource-limited settings where early identification of high-risk patients remains difficult, necessitating the use of simple, cost-effective biomarkers to improve risk stratification and guide timely interventions. This study investigates the potential of platelet-to-neutrophil ratio (PNR) to serve as a predictor for risk of specific SCD complications in a population of Nigerian patients. This hospital-based cross-sectional analytical study was conducted over 7 months and included a total of 73 adult patients with haemoglobin SS genotype in a steady state. Data on socio-demographics, medical history, clinical characteristics, and laboratory parameters, including platelet-to-neutrophil ratio (PNR) were obtained. Data analysis was performed using the Statistical Package for the Social Sciences (SPSS) version 25. Comparative statistical analyses, binary logistic regression, and receiver operating characteristic (ROC) curve evaluations were performed to determine the predictive value of PNR for risk of SCD complications. Patients with avascular necrosis (AVN) had significantly lower median PNR values compared to those without the condition (151.0 IQR: 311.2 vs. 77.5 IQR: 61.0, p = 0.004). Multivariate logistic regression analysis identified PNR as an independent predictor of AVN (p = 0.034, OR = 1.003, 95% CI: 1.000-1.006), with an ROC-derived optimal cutoff of 96.6 yielding a sensitivity of 69.6% and specificity of 76.5% (AUC = 0.733, p = 0.004). This study reveals that RNR demonstrates reasonable potential as a predictor of AVN in Nigerian SCD patients and could serve as an easily accessible biomarker for SCD risk stratification, particularly in resource-limited settings. Further studies are needed for validation of its clinical utility and possible integration into SCD management protocols.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144135953","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":"Subclonal emergence of polycythemia vera, chronic myelomonocytic leukemia, and chronic myeloid leukemia.","authors":"Violaine Tran Quang, Jules Cretin, Romain Loyaux, Bilel Ben Jedidia, Sihem Tarfi, Guillaume Gricourt, Quentin Barathon, Corine Joy, Pascaline Etancelin, Orianne Wagner-Ballon, Cécile Pautas, Lydia Roy, Ivan Sloma","doi":"10.1007/s00277-025-06417-8","DOIUrl":"https://doi.org/10.1007/s00277-025-06417-8","url":null,"abstract":"<p><p>The present longitudinal study reports a unique patient followed over almost three decades who sequentially developed polycythemia vera, chronic myelomonocytic leukemia, and chronic myeloid leukemia. The patient received successive hydroxyurea, ruxolitinib, and a combination of ruxolitinib and nilotinib. The clonal architecture dynamic was reconstructed using targeted high throughput asymmetric capture sequencing, allowing detection and quantification of mutations in 43 myeloid genes and BCR::ABL1 fusion in multiple bone marrow or peripheral blood samples and in single cell-derived colonies obtained from bone marrow colony-forming cell assays. This analysis has uncovered an unexpected subclonal link between three myeloid malignancies, all stemming from a DNMT3A/TET2 double mutant clone. Over a period of more than 30 years, this clone underwent major telomere shortening. However, a striking sustained major molecular response of the terminal dominant clone carrying all driver mutations was achieved by combination therapy with nilotinib and ruxolitinib. The remaining clone driving both polycythemia and chronic myelomonocytic leukemia remained unaffected and evolved to myelofibrosis and proliferative CMML.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144135966","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":"Epidemiology and survival of patients with primary lymphoma of bone: a large retrospective cohort study.","authors":"Xiaojie Liang, Weixiang Lu, Tong Li, Baiwei Luo, Yuzhe Wu, Chaoran Lin, Yang Liu, Liang Wang","doi":"10.1007/s00277-025-06413-y","DOIUrl":"https://doi.org/10.1007/s00277-025-06413-y","url":null,"abstract":"<p><p>Primary lymphoma of bone (PLB) is a rare extranodal lymphoma, and its epidemiology and prognosis remain controversial. We conducted a retrospective analysis of 1,222 patients with PLB in the Surveillance, Epidemiology, and End Results (SEER) database to investigate its epidemiology and prognostic factors. The incidence of PLB peaked in 1992 with an average annual percent change of 1.72 after a significant rise from 1975 to 1992, followed by a general decline. The risk of death from PLB involves both patient and treatment factors. Survival analysis revealed that age, stage, laterality, chemotherapy, and primary site significantly influence both overall survival and disease-specific survival. We integrated and compared 99 machine learning algorithms, and identified the Random Survival Forest (RSF) model as the most effective for predicting PLB outcomes. Patients were stratified into low- and high-risk groups according to the RSF model score. The incidence of PLB began to decrease after 1992, with variations by age, race, and gender. The factors influencing the prognosis of PLB are multifaceted. And the RSF model showed promising performance, aiding clinicians in early prognosis identification and improving clinical outcomes through revised management strategies and patient care.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144135935","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 effect of impaired renal function, increased red cell mass and plasma volume on thrombotic risk in PV patients.","authors":"Marko Lucijanic, Danijela Lekovic, Andrija Bogdanovic, Ivan Krecak","doi":"10.1007/s00277-025-06348-4","DOIUrl":"https://doi.org/10.1007/s00277-025-06348-4","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144126168","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":"Health-related quality of life with daratumumab, bortezomib, melphalan, and prednisone versus bortezomib, melphalan, and prednisone alone in transplant-ineligible patients with newly diagnosed multiple myeloma: analysis of the phase 3 OCTANS study.","authors":"Weijun Fu, Soo-Mee Bang, Honghui Huang, Kihyun Kim, Wei Li, Gang An, Je-Jung Lee, Zhen Cai, Jie Jin, Yafei Wang, Chor Sang Chim, Robin Carson, Rui Liu, Man Zhao, Xi Chen, Canchan Cui, Jian Hou, Jianxiang Wang","doi":"10.1007/s00277-025-06303-3","DOIUrl":"https://doi.org/10.1007/s00277-025-06303-3","url":null,"abstract":"<p><p>In the phase 3 OCTANS study, daratumumab plus bortezomib/melphalan/prednisone (D-VMP) significantly improved response rates and progression-free survival versus VMP alone in transplant-ineligible Asian patients with newly diagnosed multiple myeloma (NDMM). Understanding the impact of treatment on patient-reported outcomes (PROs) is of increasing interest. Here, we report on the impact of D-VMP and VMP on PROs in OCTANS. PROs were a secondary endpoint and were assessed using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30-item (EORTC QLQ-C30) and the EuroQol 5-dimensional descriptive system (EQ-5D-5L) questionnaire, administered at screening, every 3 months (Year 1), every 6 months thereafter (until disease progression), and 8 and 16 weeks post disease progression. Treatment effects were estimated using a mixed-effects model with repeated measures. Overall, 220 patients were randomized (D-VMP, n = 146; VMP, n = 74). Compliance rates were 100% at baseline in both treatment groups and remained relatively high by Month 12 (D-VMP, 82.6%; VMP, 67.4%). Comparable improvements from baseline were generally observed between treatment groups across most PRO scores. Significant improvements were observed with D-VMP versus VMP in Global Health Status (GHS) score at 9 months (p = 0.0443) and in social functioning and nausea and vomiting symptom scores at 12 months (p = 0.0042 and p = 0.0012, respectively). In summary, transplant-ineligible Asian patients with NDMM demonstrated improvements in PROs following treatment with D-VMP and VMP; however, greater improvements were observed in GHS, social functioning, and nausea and vomiting symptoms with D-VMP.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144126166","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}