Blood advances最新文献

{"title":"Patterns of progression among 427 Smoldering Myeloma patients diagnosed after 2014: importance of monitoring.","authors":"Efstathios Kastritis, Irene Solia, Panagiotis Malandrakis, Foteini Theodorakakou, Ioannis Ntanasis-Stathopoulos, Nikolaos Kanellias, Despina V Fotiou, Magdalini Migkou, Evangelos Eleutherakis Papaiakovou, Vassiliki Spiliopoulou, Nikoletta-Aikaterini Kokkali, Asimina Papanikolaou, Stavroula Giannouli, Maria Gavriatopoulou, Evangelos Terpos, Meletios-Athanasios A Dimopoulos","doi":"10.1182/bloodadvances.2025016083","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025016083","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751273","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Spatial transcriptomics of progression gene signature and tumor microenvironment leading to progression in mycosis fungoides.","authors":"Myoung Eun Choi, Gyeonghoon Kim, Hwa Jeong Shin, Chong Hyun Won, Sung Eun Chang, Mi Woo Lee, Woo Jin Lee","doi":"10.1182/bloodadvances.2024014495","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024014495","url":null,"abstract":"<p><p>Mycosis fungoides (MF) is characterized by stepwise evolution from patch to plaque, and sometimes to tumor. Identifying patients with early-stage MF who could progress to an advanced stage is challenging. This study investigated changes in transcriptomic expression and the tumor microenvironment associated with MF progression. Spatially resolved transcriptomic profiling was conducted using CD3, CD4, and CD30 morphology markers. In the lymphoma cell area, genes linked to collagen fibril assembly and regulation of interleukin-4 were upregulated as the disease progressed from patch-MF to plaque-MF. Genes involved in the cell cycle and glutamate catabolism were upregulated during the transition from plaque-MF to tumor-MF. 46 significant genes that consistently increased in expression during progression were identified. Stage 1 MF patients with high progression signatures showed significantly increased cancer-associated fibroblast (CAF) (p = 0.008), were more likely to progress (p < 0.001), receive radiation (p = 0.023) during follow-up. Furthermore, M2 macrophages significantly increased in lymphoma cell areas (p < 0.001) and immune cell areas (p = 0.031) in plaque MF compared with patch MF. CD163 expression was significantly correlated with most of the progression signatures and T cell exhaustion makers (LAG-3, TIM-3). Immunohistochemical staining revealed that several CAF markers and CD163 significantly increased during progression, and these markers were more frequently observed in progressive stage 1 MF compared with indolent stage 1 MF. In conclusion, this study identified significant transcriptomic changes during MF progression and found that tumor microenvironment, particularly M2 macrophages and CAFs, could contribute to progression in early-stage MF.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751284","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Autologous Stem Cell Transplantation for Relapsed/Refractory Large B Cell Lymphoma: Multicenter GETH-TC/GELTAMO Study.","authors":"Leyre Bento, Antonio Gutiérrez, Carmen Martinez, María Consejo Ortí Verdet, Marina Sorribes Portella, Ana Carolina Caballero Gonzalez, Marta Peña, Ariadna Pérez, Ana Jiménez-Ubieto, Lucía Medina, Mariana Beatriz Bastos Oreiro, Paula Fernández Caldas-González, Maria Belen Navarro, Isabel Salcedo, Pau Abrisqueta, Ignacio Español, Javier Cornago Navascués, Fernando Martin-Moro, Lucía García Tomás, Pilar Gómez-Prieto, María Rosario Varela, María Puente, Joud Zanabili, Teresa Zudaire, Izaskun Zeberio, Raquel Del Campo, Leslie González Pinedo, Pedro Antonio Gonzalez-Sierra, Cristina Blázquez-Goñi, Jordina Rovira, Marta Sitges, Mireia Franch-Sarto, Almudena Cabero Martínez, Alberto Mussetti, Juan Montoro, Antonia Sampol, Anna Sureda, Dolores Caballero-Barrigon, Alejandro Martin Martin Garcia-Sancho","doi":"10.1182/bloodadvances.2024015415","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015415","url":null,"abstract":"<p><p>We performed a retrospective multicenter study including 791 patients with relapsed/refractory (R/R) large B cell lymphoma (LBCL) who underwent ASCT from 2010-2021. All the patients received rituximab and anthracycline-based frontline therapy. After a median follow-up of 74 months (95%CI 68-81) from infusion, 65% of the patients were alive and 84% of them free of disease. Progression-free survival (PFS) and overall survival (OS) at 6 years were 51% (95%CI 47-54) and 63% (95%CI 60-67), respectively. Non-relapse mortality (NRM) at 1 year was 9% (95%CI 7-11). Age >60 years at ASCT [HR 1.31 (95%CI 1.06-1.62), p=0.011], ASCT as ≥3rd line [HR 1.81 (95%CI 1.42-2.31), p<0.001] and partial response (PR) versus complete response (CR) at ASCT [HR 1.46 (95%CI 1.18-1.81), p<0.001] were the independent variables influencing PFS. Age >60 years at ASCT [HR 1.62 (95%CI 1.24-2.12), p<0.001], time period before first of November 2012 [HR 1.40 (95%CI 1.07-1.83), p=0.014], ASCT as ≥3rd line [HR 1.77 (95%CI 1.32-2.37), p<0.001], PR versus CR [HR 1.58 (95%CI 1.22-2.05), p<0.001] and stable disease (SD) versus CR pre-ASCT [HR 3.41 (95%CI 1.81-6.45), p<0.001] were the variables associated with worse OS. Refractory or early relapse did not significantly influence survival (6y-PFS and OS in patients with refractory, early and late relapse was 54% and 64%; 46% and 62% and 49% and 63%, respectively). To our knowledge, this is the largest series analyzing the efficacy of ASCT in patients with R/R LBCL after rituximab-containing frontline therapy. Our results indicate that ASCT is a curative option for patients with chemosensitive disease.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751251","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"AhR activation mitigates graft-versus-host disease of the central nervous system by reducing microglial NF-κB signaling.","authors":"Alexander Zähringer, Ines Morgado, Daniel Erny, Florian Ingelfinger, Jana Gawron, Sangya Chatterjee, Valentin Wenger, Dominik Schmidt, Lennard Frederik Schwöbel, Rachael C Adams, Marlene Langenbach, Alina Hartmann, Natascha Osswald, Julian Wolf, Günther Schlunck, Priscilla S Briquez, Kathleen Grueter, Dietrich A Ruess, Ian Frew, Ann-Cathrin Burk, Verena Holzmüller, Bodo Grimbacher, David Michonneau, Geoffroy Andrieux, Gérard Socié, Julia Kolter, Melanie Börries, Marie Follo, Franziska Blaeschke, Lisa Sevenich, Marco Prinz, Robert Zeiser, Janaki Manoja Vinnakota","doi":"10.1182/bloodadvances.2024015000","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015000","url":null,"abstract":"<p><p>Acute Graft-versus-Host Disease (GVHD) that occurs after allogeneic hematopoietic cell transplantation (allo-HCT) can affect the central nervous system (CNS). The majority of allo-HCT patients receive antibiotic treatment, which alters the microbiome and essential microbiome-derived metabolites. We investigated the impact of microbiome modifications on CNS-GVHD and therapeutic strategies to overcome the microbiome-derived metabolite depletion. Antibiotic treatment of mice undergoing allo-HCT increased microglia numbers in the brain, indicating increased inflammation. In addition, microglia morphology shifted towards a highly branched phenotype. Consistent with a pro-inflammatory phenotype microglia exhibited increased NF-κB and Src activity. Antibiotic treatment caused the depletion of the bacteria-derived arylhydrocarbon receptor (AhR) ligand indole-3-acetate in the brain. Conversely, treatment of primary microglia with the AhR-ligand- 6-formylindolo (3, 2-b) carbazole (FICZ) reduced NF-κB activity and phagocytic potential. Microglia expansion and morphological changes were reversed by AhR-ligand-FICZ-treatment. Moreover, the AhR-ligand indole-3-acetate was also reduced in the CNS of patients that developed acute GVHD concomitant with increased microglial NF-κB expression. In summary, we demonstrated that antibiotic treatment and a subsequent decrease of AhR-ligands resulted in increased microglia activation during CNS-GVHD. FICZ-treatment hampered CNS inflammation by inhibiting NF-κB activity, thereby providing a metabolic modifier to interfere with pathogenic microglia signaling and CNS-GVHD in vivo.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751241","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hematological phenotypes in GATA2 deficiency syndrome arise from aging maladaptation to proliferation and somatic events.","authors":"Juncal Fernandez-Orth, Cansu Koyunlar, Julia Miriam Weiss, Emanuele Gioacchino, Hans W J de Looper, Geoffroy Andrieux, Mariette Ter Borg, Joke Zink, Irene Gonzalez Menendez, Remco Hoogenboezem, Baris Ismail Yigit, Kirsten Gussinklo, Roger Mulet-Lazaro, Charlotte Wantzen, Sophie Pfeiffer, Christian Molnar, Eric Bindels, Sheila Bohler, Mathijs Arnoud Sanders, Leticia Quintanilla-Martinez, Marcin W Wlodarski, Melanie Boerries, Ivo P Touw, Charlotte Niemeyer, Miriam Erlacher, Emma de Pater","doi":"10.1182/bloodadvances.2024015106","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015106","url":null,"abstract":"<p><p>The GATA2 transcription factor is a pivotal regulator of hematopoiesis. Disruptions in the GATA2 gene drive severe hematologic abnormalities and are associated with an increased risk of myelodysplastic syndromes and acute myeloid leukemia; however, the mechanisms underlying the pathophysiology of GATA2 deficiency remain still unclear. We developed two different mouse models that are based on serial and limiting donor cell transplantation of (aged) GATA2 haploinsufficient cells and mirror the symptoms of GATA2 deficiency. Similar to what has been observed in patients, our models show that GATA2 haploinsufficiency leads to B lymphopenia, monocytopenia, lethal bone marrow failure (BMF), myelodysplasia and lymphoblastic leukemia. Leukemia arises exclusively as a result of BMF, driven by somatic aberrations and accompanied by increased Myc target expression and genomic instability. These findings were confirmed in human GATA2+/- K562 cell lines showing defects in cytokinesis and are in line with the fact that monosomy 7 and trisomy 8 are frequent events in patients with MDS.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143728630","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Double mutant DNMT3A AML: a unique subtype experiencing increased DNA damage and poor prognosis.","authors":"Emma L Boertjes, Sanne Massaar, Annelieke Zeilemaker, Jolinda Konijnenburg, Melissa Rijken, François G Kavelaars, Tim Grob, Jurjen Versluis, Bob Löwenberg, Peter J M Valk, Mathijs A Sanders","doi":"10.1182/bloodadvances.2024014698","DOIUrl":"10.1182/bloodadvances.2024014698","url":null,"abstract":"<p><strong>Abstract: </strong>Mutation of DNMT3A, encoding a de novo methyltransferase essential for cytosine methylation, is a common early event in clonal hematopoiesis (CH) and adult acute myeloid leukemia (AML). Spontaneous deamination of methylated cytosines damages DNA, which is repaired by the base excision repair (BER) enzymes methyl-CpG binding domain 4 (MBD4) and thymine DNA glycosylase (TDG). Congenital MBD4 deficiency has been linked to early-onset CH and AML and is marked by exceedingly high levels of DNA damage and mutation of DNMT3A. Strikingly, wild-type (WT) DNMT3A binds TDG, thereby potentiating its repair activity. Because TDG is the only remaining BER enzyme in MBD4-deficient patients with AML capable of repairing methylation damage, we investigated whether mutant DNMT3A negatively affects the repair function of TDG. We found that, although WT DNMT3A stimulates TDG function, mutant DNMT3A impairs TDG-mediated repair of DNA damage in vitro. In light of this finding and to extrapolate our observations to the broader AML patient population, we investigate here the genetic profiles and survival outcomes of patients with AML with single mutant (SM) vs double mutant (DM) DNMT3A. Patients with DM DNMT3A AML show a characteristic driver mutation landscape and reduced overall survival compared with patients with SM DNMT3A AML. Importantly, whole-genome sequencing showed a trend for increased DNA damage in primary DM DNMT3A AML samples, especially when DNMT3A mutations are located at the DNMT3A-TDG interaction interface.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1344-1355"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11954108/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142852833","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Socioeconomic, racial-ethnic, household, and infrastructural disparities of hematologic cancer outcomes in the United States.","authors":"David J Fei-Zhang, Erik Wu, Alexander V Stanisic, Lifang Hou, Leonidas C Platanias, Stephen M Ansell, Marquita W Lewis-Thames, Sherif M Badawy, Jonas Paludo","doi":"10.1182/bloodadvances.2024013956","DOIUrl":"10.1182/bloodadvances.2024013956","url":null,"abstract":"<p><strong>Abstract: </strong>Although social determinants of health (SDoH) investigations have shown limited analyses of socioeconomic and race-ethnic status on certain hematologic malignancies, the impact of factors beyond those across a fuller scope of hematologic cancers remains unknown. The Social Vulnerability Index (SVI), a tool for assessing varied US census-derived sociodemographic factors, allows for the specific quantification of SDoH in dynamic, regional contexts for their associations with hematologic malignancy inequities. To assess the summative influence of varied SDoH factors on hematologic malignancy outcomes and discern which SDoH factors contributed the largest associations toward disparities, 796 005 adults with hematologic malignancies between 1975 to 2017 were identified for this retrospective cohort study. Vulnerability in 15 SDoH factors was measured using composite and subcategory SVI scores geographically matched to patients. Regressions between SVI factors and follow-up time after diagnosis and survival period were performed. Increasing overall SVI correlated with significantly decreased surveillance period in 11 of 14 hematologic malignancies, with decreases upward of 33.4% (39.0-26.0 months for acute lymphocytic leukemia). Increasing SVI significantly associated with decreased survival period across 11 of 14 hematologic malignancies, with decreases upward of 47.2% (89.5-47.3 months for Hodgkin lymphoma). Socioeconomic status and housing and transportation vulnerabilities showed the largest magnitude of contributions, followed by minority language and household composition. Significant decreases in hematologic malignancy prognosis associate with increasing overall SDoH vulnerability in varied sociodemographic contexts in the United States. Furthermore, there are quantifiable differences in which types of SDoH contribute more to trends per malignancy type. These findings demonstrate specific SDoH targets for further research and policy initiatives to combat hematologic malignancy disparity more effectively.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1463-1471"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142982811","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Characteristics of infections after BCMA-directed CAR T-cell therapy for multiple myeloma: a real-world analysis.","authors":"Tim Richardson, Daniel Schütte, Guido Kobbe, Ben-Niklas Baermann, Tobias A W Holderried, Friederike Schmitz, Martina Crysandt, Michael Hallek, Christoph Scheid, Udo Holtick, Oliver A Cornely, Jannik Stemler, Sibylle C Mellinghoff","doi":"10.1182/bloodadvances.2024015008","DOIUrl":"10.1182/bloodadvances.2024015008","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1370-1375"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11957513/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142920295","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Validation of the revised 2022 European LeukemiaNet risk stratification in adult patients with acute myeloid leukemia.","authors":"Leo Ruhnke, Marius Bill, Sven Zukunft, Jan-Niklas Eckardt, Silvia Schäfer, Sebastian Stasik, Maher Hanoun, Thomas Schroeder, Lars Fransecky, Björn Steffen, Stefan W Krause, Sebastian Scholl, Andreas Hochhaus, Tim Sauer, Sabrina Kraus, Kerstin Schäfer-Eckart, Martin Kaufmann, Edgar Jost, Tim Brümmendorf, Christoph Schliemann, Jan-Henrik Mikesch, Utz Krug, Mathias Hänel, Anke Morgner, Markus Schaich, Andreas Neubauer, Roland Repp, Dirk Niemann, Ruth Seggewiss-Bernhardt, Achim Meinhardt, Johannes Kullmer, Ulrich Kaiser, Wolfgang Blau, Alexander Kiani, Götz Ulrich Grigoleit, Aristoteles Giagounidis, Alexander A Wurm, Heidi Altmann, Jan Moritz Middeke, Johannes Schetelig, Carsten Müller-Tidow, Friedrich Stölzel, Claudia D Baldus, Uwe Platzbecker, Hubert Serve, Martin Bornhäuser, Christian Thiede, Christoph Röllig","doi":"10.1182/bloodadvances.2024013304","DOIUrl":"10.1182/bloodadvances.2024013304","url":null,"abstract":"<p><strong>Abstract: </strong>In 2022, the European LeukemiaNet (ELN) risk stratification for patients with acute myeloid leukemia (AML) has been updated. We aimed to validate the prognostic value of the 2022 ELN classification (ELN22) by evaluating 1570 patients with newly diagnosed AML (median age, 56 years) treated with cytarabine-based intensive chemotherapy regimens. Compared with 2017 ELN classification (ELN17), which allocated 595 (38%), 413 (26%), and 562 patients (36%) to the favorable-, intermediate-, and adverse-risk categories, ELN22 classified 575 (37%), 410 (26%), and 585 patients (37%) as favorable, intermediate, and adverse risk, respectively. Risk group allocation was revised in 340 patients (22%). Most patients were reclassified into the ELN22 intermediate- or ELN22 adverse-risk group. The allocation of patients according to the ELN22 risk categories resulted in a significantly distinct event-free survival (EFS), relapse-free survival, and overall survival (OS). Compared with ELN17, reallocation according to the ELN22 recommendations resulted in a significantly improved prognostic discrimination for OS (3-year area under the curve, 0.71 vs 0.67). In patients with ELN22 favorable-risk AML, co-occurring myelodysplasia-related (MR) gene mutations did not significantly affect outcomes. Within the ELN22 adverse-risk group, we observed marked survival differences across mutational groups (5-year OS rate of 21% and 3% in patients with MR gene mutations and TP53 mutations, respectively). In patients harboring MR gene mutations, EZH2-, STAG2-, and ZRSR2-mutated patients showed an intermediate-like OS. In patients with secondary AML and those who underwent allogeneic hematopoietic cell transplantation, EFS and OS significantly differed between ELN22 risk groups, whereas the prognostic abilities of ELN17 and ELN22 classifications were similar. In conclusion, ELN22 improves prognostic discrimination in a large cohort of intensively treated patients with AML. Given the heterogeneous outcome in patients with MR gene alterations, ranging between those of intermediate and adverse risk patients, we suggest re-evaluation of risk allocation in these patients.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1392-1404"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11960536/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590025","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High pulmonary hypertension risk by echocardiogram shortens survival in polycythemia vera.","authors":"Alexander J Gazda, Di Pan, Katie Erdos, Ghaith Abu-Zeinah, Alexandra Racanelli, Evelyn M Horn, Joseph M Scandura","doi":"10.1182/bloodadvances.2024013970","DOIUrl":"10.1182/bloodadvances.2024013970","url":null,"abstract":"<p><strong>Abstract: </strong>Pulmonary hypertension (PH) is a known complication of myeloproliferative neoplasms (MPNs) with an estimated prevalence as high as 50%. Patients with polycythemia vera (PV) report a wide spectrum of symptoms that significantly overlap with those reported by patients with PH. Yet, it is not known how PH affects outcomes and survival in patients with PV. To address this gap, we investigated the impact of echocardiography (ECHO)-based PH risk on survival of patients with PV from our large single-center cohort. Of 637 patients with PV, 134 had at least 1 ECHO and were included for analysis. Overall survival did not differ between patients who had or did not have ECHO. PH risk was established based on tricuspid regurgitation jet velocity. Kaplan-Meier analysis showed that high PH risk is associated with shortened survival compared with mild PH risk (median survival, 1.7 vs 3.7 years) or normal PH risk (median survival, not yet reached). Cox proportional hazard models found high PH risk was associated with a more than threefold increased risk of death, independent of age and thrombosis history. Logistic regression identified age (odds ratio, 6.9) and duration of PV diagnosis (odds ratio, 5.4) as significant risks for PH. Based upon these results and receiver operator characteristic optimization, we recommend echocardiographic screening for patients with PV aged >70 years or with duration of PV of >8 years. Further studies inclusive of invasive hemodynamics, advanced cardiovascular imaging, and MPN-associated biomarkers are needed to best characterize this group 5 PH population for therapeutic interventions.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1320-1329"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11950968/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142852793","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}