{"title":"Discrepancies in Treatment Goals and Concerns Regarding Disease Management between Patients with Myeloproliferative Neoplasms and Hematologists in China: Analysis from a Multicenter Cross-Sectional Survey.","authors":"Junling Zhuang, Hongxia Shi, Xiaoli Liu, Minghui Duan, Xin Du, Ling Qin, Wuhan Hui, Rong Liang, Meifang Wang, Ye Chen, Dongyun Li, Wei Yang, Gusheng Tang, Weihua Zhang, Xia Kuang, Wei Su, Yanqiu Han, Xialu Lan, Limei Chen, Jihong Xu, Zhuogang Liu, Jian Huang, Chunting Zhao, Hongyan Tong, Jianda Hu, Chunyan Chen, Xiequn Chen, Zhijian Xiao, Qian Jiang","doi":"10.1159/000547173","DOIUrl":"https://doi.org/10.1159/000547173","url":null,"abstract":"<p><strong>Introduction: </strong>This study was aimed to identify the discrepancies in treatment goals and concerns regarding disease management between patients with myeloproliferative neoplasms (MPNs) and hematologists.</p><p><strong>Methods: </strong>A study was conducted among patients with MPNs, including polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF), and hematologists in China.</p><p><strong>Results: </strong>Data from 1,645 respondents with ET, PV, and MF and 715 hematologist respondents were analyzed. Cure of disease and healthy blood counts as treatment goals were reported more by almost half of the respondents with MPNs than by hematologists. However, prevention of thrombotic events, delayed transformation of disease, improvement of symptoms and better quality of life, and reduction in spleen size were less reported by respondents with MPNs than by hematologists. In multivariate analyses, education, comorbidities, symptom burden, disease duration, and annual out-of-pocket expenses for treatment were significantly associated with the treatment goals of respondents with MPNs. However, female physicians and senior professors paid more attention to these goals. Regarding concerns on MPN-related issues, more respondents with MPNs paid more attention to disease knowledge and restrictions in daily life compared to hematologists, whereas the majority of physicians attached importance to medication-related issues.</p><p><strong>Conclusion: </strong>The perceptions of patients with MPNs and hematologists differed in terms of treatment goals and concerns of management of MPNs. Sociodemographic and clinical variables were associated with the respondents' perspectives on MPNs. Therefore, sufficient patient-physician communication is suggested to improve treatment satisfaction and compliance.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-11"},"PeriodicalIF":1.1,"publicationDate":"2025-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145342471","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"The Causal Relationship between Lipid Metabolites and Multiple Myeloma Risk: A Mendelian Randomization Study.","authors":"Jian Tao, Ling Wang, Zheyun Gu","doi":"10.1159/000548566","DOIUrl":"https://doi.org/10.1159/000548566","url":null,"abstract":"<p><strong>Introduction: </strong>Research has demonstrated a potential link between lipid metabolites and multiple myeloma (MM); however, the causal relationship remains uncertain. This Mendelian randomization (MR) study aimed to explore the potential causal relationship between lipid metabolites and MM.</p><p><strong>Methods: </strong>In this study, data on lipid metabolites were obtained from a genome-wide association study of metabolites in blood samples from 7,824 Europeans. Genetic information related to MM came from the UK Biobank database, encompassing 601 patients with MM and 372,016 control samples. In this MR analysis, inverse-variance weighted method was used as the primary analysis method; MR Egger and weighted median were employed as complementary approaches. Sensitivity analyses were conducted using the Cochran Q test, MR-Egger intercept, MR-PRESSO, and leave-one-out analysis.</p><p><strong>Results: </strong>A total of 121 human lipid metabolites were analyzed in this MR study. The analysis result revealed that 1-docosahexaenoyl-glycerophosphocholine [odds ratio (OR) = 1.0059, 95% confidence interval (CI) 1.0043-1.0076, P < 0.01 , FDR = 0.12], tetradecanedioate (OR = 1.0007, 95% CI 1-1.0013, P = 0.0498 , FDR = 0.23), and X-12990-docosapentaenoic acid (OR = 1.0029, 95% CI 1.0015-1.0044, P < 0.01 , FDR = 0.15) were linked to an increased risk of MM. As for palmitoleate (OR = 0.9972, 95% CI 0.9947-0.9997, P = 0.0299 , FDR = 0.19) , a nominal inverse association was observed. None of these associations reached statistical significance after FDR correction (all FDR > 0.05). Sensitivity analyses verified the robustness of these nominally significant results.</p><p><strong>Conclusion: </strong>Genetic evidence demonstrated nominal associations of 1-docosahexaenoyl-sn-glycero-3-phosphocholine, tetradecanedioate, X-12990-eicosapentaenoic acid, and palmitoleate with MM risk, though these did not survive FDR correction. While these findings suggest potential metabolic pathways in MM pathogenesis, further validation is required before considering these compounds as biomarkers for clinical screening or drug target selection.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-14"},"PeriodicalIF":1.1,"publicationDate":"2025-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145306820","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Annatina Sarah Schnegg-Kaufmann, Ulrike Bacher, Alicia Rovó, Martin Andres, Gertrud Wiedemann, Naomi Porret, Bijan Moshaver, Nicolas Kaufmann, Joëlle Tchinda, Sara C Meyer, Anne Angelillo-Scherrer
{"title":"Artificial intelligence in haematologic diagnostics: Current applications and future perspectives.","authors":"Annatina Sarah Schnegg-Kaufmann, Ulrike Bacher, Alicia Rovó, Martin Andres, Gertrud Wiedemann, Naomi Porret, Bijan Moshaver, Nicolas Kaufmann, Joëlle Tchinda, Sara C Meyer, Anne Angelillo-Scherrer","doi":"10.1159/000548753","DOIUrl":"https://doi.org/10.1159/000548753","url":null,"abstract":"<p><p>Clinical researchers and laboratory specialists are striving to explore artificial intelligence (AI) to facilitate and optimise haematological diagnostics in response to the growing demand for more efficient and accurate diagnoses. This review summarises current approaches integrating AI into blood and bone marrow cytomorphology, flow cytometry (FC), genetics, and haemostasis. Efforts include automated cell differentiation in peripheral blood and bone marrow aspirates, algorithms for identifying causes of anaemia, tools for rapid diagnosis of acute leukaemia and other haematological entities. AI in FC may reduce subjectivity and variability, while in genomics, machine learning (ML) is increasingly implemented for processing high-throughput sequencing data, and may enable automated detection of karyotypes in the future. In haemostasis, AI allows for automation in quality control, the establishment of personalised reference ranges, and potentially automated result interpretation. AI has, however, limitations such as cross-platform compatibility and often lacks sufficient validation. Ethical concerns include risks of bias and regulations are lagging behind the rapid developments. Despite this, AI shows promise for automating and improving many steps in hematological diagnostics, though final interpretation still needs expert haematologists.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-25"},"PeriodicalIF":1.1,"publicationDate":"2025-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145278637","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Letian Shao, Zhen Kou, Renaguli Abulaiti, Qiping Shi, Xiaolong Qi, Zengsheng Wang, Shunsheng Zhai, Li An, Qin Huang, Guzailinuer Wufuer, Yan Li
{"title":"Establishment and Evaluation of Prognostic Prediction Model for Diffuse Large B-cell Lymphoma Patients Based on International Prognostic Index and FAT4, TP53 Mutation.","authors":"Letian Shao, Zhen Kou, Renaguli Abulaiti, Qiping Shi, Xiaolong Qi, Zengsheng Wang, Shunsheng Zhai, Li An, Qin Huang, Guzailinuer Wufuer, Yan Li","doi":"10.1159/000548543","DOIUrl":"https://doi.org/10.1159/000548543","url":null,"abstract":"<p><p>Diffuse large B-cell lymphoma (DLBCL) is a common type of non-Hodgkin lymphoma with clinical and genetic heterogeneity, leading to significant differences in patient prognosis. In this study, a retrospective analysis was conducted on relevant data from 155 DLBCL patients treated at our hospital. Single-factor and further multivariate COX analysis were used to identify independent prognostic factors associated with progression-free survival (PFS) and overall survival (OS) in DLBCL patients. The predictive performance and clinical application value of the risk prediction model were evaluated using receiver operating characteristic (ROC) curves and decision curve analysis (DCA). The consistency between the actual and predicted event rates was assessed using calibration plots, and validation was performed using bootstrapping resampling. It was demonstrated that the integration of FAT4 mutation and TP53 mutation into the FAT4-TP53-IPI model can enhance the prognostic value of the IPI scoring system.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-28"},"PeriodicalIF":1.1,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145211280","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kyoung Il Min, Daehun Kwag, Gi June Min, Sung-Soo Park, Silvia Park, Sung-Eun Lee, Byung-Sik Cho, Ki-Seong Eom, Yoo-Jin Kim, Hee-Je Kim, Chang-Ki Min, Seok-Goo Cho, Jae-Ho Yoon
{"title":"Ponatinib Monotherapy in adult patients with Relapsed or Refractory Philadelphia-Positive Acute Lymphoblastic Leukemia: A Real-World Retrospective Analysis Including MRD Relapse.","authors":"Kyoung Il Min, Daehun Kwag, Gi June Min, Sung-Soo Park, Silvia Park, Sung-Eun Lee, Byung-Sik Cho, Ki-Seong Eom, Yoo-Jin Kim, Hee-Je Kim, Chang-Ki Min, Seok-Goo Cho, Jae-Ho Yoon","doi":"10.1159/000548544","DOIUrl":"https://doi.org/10.1159/000548544","url":null,"abstract":"<p><strong>Introduction: </strong>The treatment of relapsed or refractory (R/R) Philadelphia chromosome (Ph)-positive acute lymphoblastic leukemia (ALL) remains challenging after failure to several tyrosine kinase inhibitors. This study evaluated the clinical outcomes of ponatinib in a real-world cohort with R/R Ph-positive ALL, including those treated at the stage of measurable residual disease (MRD) relapse.</p><p><strong>Methods: </strong>We retrospectively analyzed 79 adults with R/R Ph-positive ALL treated with ponatinib monotherapy. At the start of treatment, 55 patients (69.6%) were in hematologic relapse, while 24 (30.4%) were in MRD relapse. We evaluated CR rate, MRD response, survival outcomes, and predictors of response and survival according to various clinical and genetic parameters.</p><p><strong>Results: </strong>CR was achieved in 48 (60.7%) patients, and 22 of 46 with MRD data (47.8%) achieved CMR. Ponatinib initiation at MRD relapse was associated with higher odds of better molecular response. In multivariate analysis, age under 60 and MRD response better than MMR were linked to improved OS. However, 2-year OS remained poor at 29.5% (95% CI: 18.9-40.9%). Allo-HCT was performed in 38 patients (48.1%), with a 2-year post-transplant OS of 29.1% (95% CI: 12.9-47.6%). Prior allo-HCT was associated with inferior OS and DFS.</p><p><strong>Conclusion: </strong>Ponatinib achieved an acceptable CR rate and MRD response in R/R Ph-positive ALL, but long-term survival remained poor despite allo-HCT. These results support earlier use of ponatinib in the salvage setting and highlight the need for combination strategies to overcome the limited durability of monotherapy in patients with R/R Ph-positive ALL.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-22"},"PeriodicalIF":1.1,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145211332","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Bochra Sedaki, Alexandre Iat, Sami Benachour, Michael Loschi, Karine Risso, Ghada Abichou, Corinne Ferrero-Vacher, Neila De Pooter, Joy Mouanes-Abelin, Berengere Dadone-Montaudie, Thomas Cluzeau
{"title":"Real World Experience of Azacitidine + Venetoclax in AML rigorously following management guidelines from clinical trial.","authors":"Bochra Sedaki, Alexandre Iat, Sami Benachour, Michael Loschi, Karine Risso, Ghada Abichou, Corinne Ferrero-Vacher, Neila De Pooter, Joy Mouanes-Abelin, Berengere Dadone-Montaudie, Thomas Cluzeau","doi":"10.1159/000548334","DOIUrl":"https://doi.org/10.1159/000548334","url":null,"abstract":"<p><p>Azacitidine + venetoclax is a new standard of care for acute myeloid leukemia not eligible for intensive chemotherapy. This combination is associated with hematological toxicities and some drug interactions. Real-world studies have shown a decrease in results and outcomes when compared to VIALE-A study. We report here our single center experience using AZA + VEN rigorously following management guidelines from the VIALE-A study, applied to a real-life population. Forty-two AML patients were analyzed. CR/CRi/MLFS rate was 73,8 % after 1 cycle with 51% CR/CRi and 80,9% after 2 cycles. Median overall survival was 18 months [95% CI, 10.3-25.7]. Our retrospective study showed that rigorous use of AZA + VEN in real-world setting was associated with results close to clinical trial observations. We showed the need to rigorously follow AZA+VEN dosage but also toxicity guidelines and practice recommendations edited in the clinical trial even in real-world.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-13"},"PeriodicalIF":1.1,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145190677","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Adrian Schwarzer, Christian Späth, Thomas Neumann, Jan Krönke, William H Krüger
{"title":"Case report: Donor-derived clonal haematopoiesis after allogeneic stem cell transplantation.","authors":"Adrian Schwarzer, Christian Späth, Thomas Neumann, Jan Krönke, William H Krüger","doi":"10.1159/000548473","DOIUrl":"https://doi.org/10.1159/000548473","url":null,"abstract":"<p><p>The accidental transmission of malignant and non-malignant hematological diseases through allogeneic hematopoietic cell transplantation has been described. Next-generation sequencing facilitates the detection of a wide range of mutations in suspected myeloid diseases. The transfer of clonal haematopoiesis is not uncommon and raises important medical and ethical considerations. We present two cases of donor-derived clonal haematopoiesis (CHIP) following allogeneic stem cell transplantation. In one case, the establishment of donor-derived CHIP was stabilized by donor-lymphocyte infusions given to counteract declining donor chimerism.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-13"},"PeriodicalIF":1.1,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145084777","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Luise Froessl, Theo Sottero, L Steven Brown, Hsiao C Li, Radhika Kainthla, Navid Sadeghi
{"title":"Patient Characteristics and Outcomes of Nodular Lymphocyte-Predominant Hodgkin's Lymphoma at a Safety-Net System Compared to an Academic Comprehensive Cancer Center.","authors":"Luise Froessl, Theo Sottero, L Steven Brown, Hsiao C Li, Radhika Kainthla, Navid Sadeghi","doi":"10.1159/000548357","DOIUrl":"10.1159/000548357","url":null,"abstract":"<p><strong>Introduction: </strong>Nodular lymphocyte-predominant Hodgkin's lymphoma (NLPHL) often follows an indolent course but carries a risk of late recurrence and transformation. Given its rarity, there is significant variability in the treatment patterns at various healthcare centers.</p><p><strong>Methods: </strong>This retrospective chart review aimed to compare the patient characteristics and outcomes of NLPHL patients >18 years of age diagnosed between January 1st, 2007, and December 31st, 2022, at Parkland Health, the safety-net system for uninsured/underinsured patients in Dallas County, with patients treated at the neighboring NCI-designated Harold C. Simmons Comprehensive Cancer Center (SCCC).</p><p><strong>Results: </strong>Our cohort included 53 adult patients (25 at PH vs. 28 at SCCC). PH patients were more likely to belong to racial/ethnic minority groups (black non-Hispanic 84% at PH vs. 32% at SCCC, Hispanic 16% at PH vs. 0% at SCCC, p < 0.01) and to be uninsured (60% at PH vs. 0% at SCCC, p < 0.01). Site of care (PH vs. SCCC) or race/ethnicity did not impact the treatment choice. At a median follow-up of 60 months (IQR 21-83), 3 deaths occurred, resulting in an overall 5-year restricted mean overall survival of 57 months. Overall survival and progression-free survival were not statistically different between the two sites of treatment.</p><p><strong>Conclusion: </strong>Despite health inequities that typically impact safety-net patients, we did not observe differences in treatment patterns or outcomes of Nodular lymphocyte-predominant Hodgkin's lymphoma between patients treated at PH compared to SCCC.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-6"},"PeriodicalIF":1.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145028706","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Janine Briggeler-Mani, Emmanuel Häfliger, Annatina Sarah Schnegg-Kaufmann, Katarzyna Aleksandra Jalowiec, Nicola Andina, Nada Agbariah, Adrian Dante De Angelis, Bastien Grandjean, Linet Njue, Allam Ramanjaneyulu, Ulrike Bacher, Yara Banz, Naomi Azur Porret, Alicia Rovó
{"title":"Paroxysmal Nocturnal Hemoglobinuria with Large Clones in Non-Hypoplastic Myelodysplastic Syndrome: Report of Two Cases.","authors":"Janine Briggeler-Mani, Emmanuel Häfliger, Annatina Sarah Schnegg-Kaufmann, Katarzyna Aleksandra Jalowiec, Nicola Andina, Nada Agbariah, Adrian Dante De Angelis, Bastien Grandjean, Linet Njue, Allam Ramanjaneyulu, Ulrike Bacher, Yara Banz, Naomi Azur Porret, Alicia Rovó","doi":"10.1159/000548287","DOIUrl":"10.1159/000548287","url":null,"abstract":"<p><strong>Introduction: </strong>Paroxysmal nocturnal hemoglobinuria (PNH) clones are frequently found in hypoplastic myelodysplastic syndromes (hMDS), though less commonly than in aplastic anemia. In contrast, the coexistence of hemolytic PNH with large clones and classical, hypercellular MDS (non-hMDS) is rare and likely underrecognized in clinical practice. Since 2014, 229 MDS patients have been seen at our department. Here, we report two cases with this association and discuss their particular diagnostic and treatment challenges.</p><p><strong>Case presentations: </strong>The first case is a 68-year-old woman with a hemolytic PNH of 59 years duration. We first saw her in June 2021; she had pancytopenia, with values stable over the past 25 years. After a complete work-up, MDS with low blasts and SF3B1 mutation was diagnosed. She was subsequently diagnosed with symptomatic pulmonary hypertension, and in 2023, she started therapy with ravulizumab, achieving good disease control. The second case concerns a 76-year-old man diagnosed with MDS at age 74. One year later, his anemia worsened, and hemolytic PNH with large clones was diagnosed. The patient showed initial benefit from ravulizumab, and he was later switched to pegcetacoplan, which led to effective disease control.</p><p><strong>Conclusion: </strong>We want to emphasize the importance of assessing PNH clones in the diagnosis of non-hMDS, especially in cases with significant anemia. Regarding PNH treatment in such patients, we found that they are underrepresented in studies investigating complement inhibitor. However, standard doses recommended for PNH appear effective and safe regardless of the underlying disease.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-8"},"PeriodicalIF":1.1,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145022547","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Yating Li, Jie Chen, Yunqi Cui, Na Hu, Wanting Ying, Hongming Huang, Xiaoyan Qu, Zhengxu Sun
{"title":"A Nomogram for Predicting Progression-Free Survival in Primary Extramedullary Multiple Myeloma Using Routine Laboratory Variables.","authors":"Yating Li, Jie Chen, Yunqi Cui, Na Hu, Wanting Ying, Hongming Huang, Xiaoyan Qu, Zhengxu Sun","doi":"10.1159/000548028","DOIUrl":"10.1159/000548028","url":null,"abstract":"<p><strong>Introduction: </strong>Extramedullary involvement in multiple myeloma represents aggressive disease, with clinical stratification of primary extramedullary disease (EMD) remaining a challenge. In this study, we aimed to develop a credible nomogram utilizing routine laboratory variables to predict individual survival for primary EMD patients.</p><p><strong>Methods: </strong>We retrospectively analyzed a cohort of 60 primary EMD patients, from January 2006 to December 2022. Independent risk factors were identified and subsequently incorporated to generate a nomogram using the Cox proportional hazard regression model. Then, we classified patients into two risk groups based on the nomogram model risk score and compared their survival time using the Kaplan-Meier method.</p><p><strong>Results: </strong>After a median follow-up of 25.4 months, the median progression-free survival (PFS) of primary EMD patients was 29.4 months. Three independent prognostic factors, namely Ki67, endothelial activation stress index (EASIX), and monocyte count, were identified and subsequently incorporated to generate a nomogram using the Cox proportional hazard regression model. Nomogram performance was assessed using various metrics. Then, we classified patients into two risk groups based on the nomogram model risk score, and the Kaplan-Meier curve showed that the median PFS was significantly longer in the low-risk group compared to the high-risk group (37.1 months versus 2.6 months, p < 0.001).</p><p><strong>Conclusion: </strong>A predictive nomogram was developed and validated to evaluate the outcome of primary EMD patients.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-11"},"PeriodicalIF":1.1,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144938378","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}