HaematologicaPub Date : 2025-06-01Epub Date: 2025-02-27DOI: 10.3324/haematol.2025.287535
Bernhard Lämmle
{"title":"The third form of thrombotic thrombocytopenic purpura shows up again: what is it, does it even exist, how to find out? Comment on \"Acquired thrombotic thrombocytopenic purpura without detectable anti-ADAMTS13 antibodies: a possible underlying autoimmune mechanism\" and on \"The different faces of thrombotic thrombocytopenic purpura\".","authors":"Bernhard Lämmle","doi":"10.3324/haematol.2025.287535","DOIUrl":"10.3324/haematol.2025.287535","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1445-1448"},"PeriodicalIF":8.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12130787/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143515445","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}
HaematologicaPub Date : 2025-06-01Epub Date: 2024-12-12DOI: 10.3324/haematol.2024.286465
Jayastu Senapati, Sanam Loghavi, Guillermo Garcia-Manero, Guillin Tang, Tapan Kadia, Nicholas J Short, Hussein A Abbas, Naszrin Arani, Courtney D DiNardo, Gautam Borthakur, Naveen Pemmaraju, Betul Oran, Elizabeth Shpall, Uday Popat, Richard Champlin, Sherry Pierce, Sankalp Arora, Ghayas Issa, Musa Yilmaz, Keyur Patel, Koichi Takahashi, Guillermo Montalban-Bravo, Danielle Hammond, Fadi G Haddad, Farhad Ravandi, Hagop M Kantarjian, Naval G Daver
{"title":"Clinical interrogation of <i>TP53</i> aberrations and its impact on survival in patients with myeloid neoplasms.","authors":"Jayastu Senapati, Sanam Loghavi, Guillermo Garcia-Manero, Guillin Tang, Tapan Kadia, Nicholas J Short, Hussein A Abbas, Naszrin Arani, Courtney D DiNardo, Gautam Borthakur, Naveen Pemmaraju, Betul Oran, Elizabeth Shpall, Uday Popat, Richard Champlin, Sherry Pierce, Sankalp Arora, Ghayas Issa, Musa Yilmaz, Keyur Patel, Koichi Takahashi, Guillermo Montalban-Bravo, Danielle Hammond, Fadi G Haddad, Farhad Ravandi, Hagop M Kantarjian, Naval G Daver","doi":"10.3324/haematol.2024.286465","DOIUrl":"10.3324/haematol.2024.286465","url":null,"abstract":"<p><p>In myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) with TP53 aberrations, dissecting the interaction amongst patient, disease and treatment factors are important for therapeutic decisions and prognostication. This retrospective analysis included patients with newly diagnosed MDS (>5% blasts) and AML with TP53 mutation(s) treated at MD Anderson Cancer Center. We factored patient age, TP53 aberration burden, therapy intensity and use of venetoclax in the AML subgroup, and allogeneic hematopoietic stem cell transplantation (HSCT) to interrogate outcomes. TP53 was annotated as high-risk (TP53HR) if >1 mutation, one mutation plus allelic deletion or a single mutation with variant allele frequency (VAF) ≥40%; TP53 low-risk (TP53LR) included a single TP53 mutation VAF <40%. Four-hundred and thirteen patients (291 AML, 122 MDS) at a median age of 69.4 years were included, 350 (85%) with TP53HR (253 AML [87%], 97 [79%] MDS). Overall response (OR) rate was 53% in AML and 62% in MDS. OR and composite complete response (CRc) rates was similar in patients with AML irrespective of treatment intensity, but higher when treated with venetoclax. At a median follow-up of 77 months, median OS was superior in patients with MDS than AML (10.8 vs. 5.9 months). On multivariate analysis (MVA) MDS had lower hazards of death compared to AML, as was TP53LR and HSCT. In the AML cohort, TP53LR and HSCT were favorable on MVA, though venetoclax did not improve survival. Both the diagnosis of MDS or AML and burden of TP53 aberrations dictated outcomes in our analysis and HSCT consistently led to improved survival outcomes.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1304-1315"},"PeriodicalIF":8.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12130771/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142812942","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}
HaematologicaPub Date : 2025-06-01Epub Date: 2025-02-06DOI: 10.3324/haematol.2022.282557
Yi June Kim, Koichi Takahashi
{"title":"Emerging technologies of single-cell multi-omics.","authors":"Yi June Kim, Koichi Takahashi","doi":"10.3324/haematol.2022.282557","DOIUrl":"10.3324/haematol.2022.282557","url":null,"abstract":"<p><p>The heterogeneity of the hematopoietic system was largely veiled by traditional bulk sequencing methods, which measure the averaged signals from mixed cellular populations. In contrast, single-cell sequencing has enabled the direct measurement of individual signals from each cell, significantly enhancing our ability to unveil such heterogeneity. Building on these advances, numerous single-cell multi-omics techniques have been developed into high-throughput, routinely accessible platforms, delineating the precise relationships among different layers of the central dogma in molecular biology. These technologies have uncovered the intricate landscape of genetic clonality and transcriptional heterogeneity in both normal and malignant hematopoietic systems, highlighting their roles in differentiation, disease progression, and therapy resistance. This review aims to provide a brief overview of the principles of single-cell technologies, their historical development, and a subset of ever-expanding multi-omics tools, emphasizing the specific research questions that inspired their creation. Amidst the evolving landscape of single-cell multi-omics technologies, our main objective is to guide investigators in selecting the most suitable platforms for their research needs.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1269-1277"},"PeriodicalIF":8.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12130758/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143255617","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}
HaematologicaPub Date : 2025-06-01DOI: 10.3324/haematol.2025.287581
Barbara Gianesin, Frédéric B Piel, Khaled M Musallam, Susanna Barella, Maddalena Casale, Elena Cassinerio, Rosario Di Maggio, Antonia Gigante, Maria Rita Gamberini, Giovanna Graziadei, Roberto Lisi, Filomena Longo, Aurelio Maggio, Raffaella Origa, Annamaria Pasanisi, Silverio Perrotta, Antonio Giulio Piga, Valeria Maria Pinto, Rosamaria Rosso, Giacomo Robello, Giovanna Russo, Marco Zecca, Lucia De Franceschi, Gian Luca Forni
{"title":"<i>Erratum</i> to: Prevalence and mortality trends of hemoglobinopathies in Italy: a nationwide study.","authors":"Barbara Gianesin, Frédéric B Piel, Khaled M Musallam, Susanna Barella, Maddalena Casale, Elena Cassinerio, Rosario Di Maggio, Antonia Gigante, Maria Rita Gamberini, Giovanna Graziadei, Roberto Lisi, Filomena Longo, Aurelio Maggio, Raffaella Origa, Annamaria Pasanisi, Silverio Perrotta, Antonio Giulio Piga, Valeria Maria Pinto, Rosamaria Rosso, Giacomo Robello, Giovanna Russo, Marco Zecca, Lucia De Franceschi, Gian Luca Forni","doi":"10.3324/haematol.2025.287581","DOIUrl":"10.3324/haematol.2025.287581","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"110 6","pages":"1449-1450"},"PeriodicalIF":8.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12130751/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144208339","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}
HaematologicaPub Date : 2025-06-01Epub Date: 2025-02-27DOI: 10.3324/haematol.2025.287430
Bengt Zöller
{"title":"Trends in mortality rate after cancer-related thrombosis give hope for the future.","authors":"Bengt Zöller","doi":"10.3324/haematol.2025.287430","DOIUrl":"10.3324/haematol.2025.287430","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1252-1253"},"PeriodicalIF":8.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12130754/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143515446","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}
HaematologicaPub Date : 2025-06-01Epub Date: 2025-01-30DOI: 10.3324/haematol.2024.286171
Margot S F Roeten, Johan Van Meerloo, Suzanne N Van Dijk, Zinia Kwidama, Gaye Jenkins, David T Teachey, Meenakshi Devidas, Mignon L Loh, Gertjan J L Kaspers, Sonja Zweegman, Gerrit Jansen, Terzah M Horton, Jacqueline Cloos
{"title":"High immunoproteasome and low constitutive proteasome subunit levels correlate with sensitivity to bortezomib-containing chemotherapy: update from the Children's Oncology Group AALL1231 trial.","authors":"Margot S F Roeten, Johan Van Meerloo, Suzanne N Van Dijk, Zinia Kwidama, Gaye Jenkins, David T Teachey, Meenakshi Devidas, Mignon L Loh, Gertjan J L Kaspers, Sonja Zweegman, Gerrit Jansen, Terzah M Horton, Jacqueline Cloos","doi":"10.3324/haematol.2024.286171","DOIUrl":"10.3324/haematol.2024.286171","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1379-1383"},"PeriodicalIF":8.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12130773/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143065293","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}
HaematologicaPub Date : 2025-06-01Epub Date: 2024-12-05DOI: 10.3324/haematol.2024.286385
Wolfgang Bethge, Sarah Flossdorf, Franziska Hanke, Christoph Schmid, Mark Ringhoffer, Stefan Klein, Bernd Hertenstein, Johannes Schetelig, Matthias Stelljes, Thomas Schroeder, Igor Wolfgang Blau, Francis Ayuk, Matthias Eder, Robert Zeiser, Katharina Fleischhauer, Nicolaus Kröger, Peter Dreger
{"title":"Does size matter? Center-specific characteristics and survival after allogeneic hematopoietic cell transplantation for acute myeloid leukemia: an analysis of the German Registry for Stem Cell Transplantation and Cell Therapy.","authors":"Wolfgang Bethge, Sarah Flossdorf, Franziska Hanke, Christoph Schmid, Mark Ringhoffer, Stefan Klein, Bernd Hertenstein, Johannes Schetelig, Matthias Stelljes, Thomas Schroeder, Igor Wolfgang Blau, Francis Ayuk, Matthias Eder, Robert Zeiser, Katharina Fleischhauer, Nicolaus Kröger, Peter Dreger","doi":"10.3324/haematol.2024.286385","DOIUrl":"10.3324/haematol.2024.286385","url":null,"abstract":"<p><p>We investigated the effect of center-specific variables on overall survival (OS) after allogeneic hematopoietic cell transplantation (alloHCT) in acute myeloid leukemia (AML). Eligible for the study were adult patients reported to the German Registry for Hematopoietic Stem Cell Transplantation and Cell Therapy (DRST) receiving first alloHCT for AML from a related or matched (>9/10 HLA-match) unrelated donor in the period 2015-2021. Primary endpoint was OS at 12 months from alloHCT. Univariable and multivariable analyses after best subset selection were performed. Of 5,328 patients, 83% received alloHCT in a high-volume center (≥40 alloHCT/year), 90% in a university hospital, 90% in a center performing alloHCT for ≥10 years, and 73% in a Joint Accreditation Committee ISCT-Europe and European Group for Blood and Marrow Transplantation (EBMT) (JACIE) accredited center. 52% of the patients were in first CR, and European LeukemiaNet risk was adverse in 37% and intermediate in 42%. On multivariable analysis, center-specific factors predicting adverse 12-month OS were program duration <5-10 years (Hazard Ratio [HR] 1.23, [95% Confidence Interval: [1.02; 1.49]), center volume <40 alloHCT/year (HR 1.21, [1.02; 1.45]), and treatment at a non-university hospital (HR 1.21, [0.98; 1.49]), whereas JACIE accreditation did not. Spline modeling suggested a negative effect of a center volume up to 45 alloHCT per year. Center volume, center experience, university hospital, but not JACIE accreditation, have an impact on alloHCT outcomes in adult patients with AML in Germany.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1292-1303"},"PeriodicalIF":8.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12130772/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142780188","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}
HaematologicaPub Date : 2025-06-01Epub Date: 2025-01-23DOI: 10.3324/haematol.2024.286468
Somadri Ghosh, Maria Rodriguez-Zabala, Gladys Telliam Dushime, Katrin Reinbach, Ramprasad Ramakrishnan, Ewa Sitnicka, Marcus Järås, Lymphoid Development And Regulation
{"title":"H2-K1 protects murine <i>MLL-AF9</i> leukemia stem cells from natural killer cell-mediated immune surveillance.","authors":"Somadri Ghosh, Maria Rodriguez-Zabala, Gladys Telliam Dushime, Katrin Reinbach, Ramprasad Ramakrishnan, Ewa Sitnicka, Marcus Järås, Lymphoid Development And Regulation","doi":"10.3324/haematol.2024.286468","DOIUrl":"10.3324/haematol.2024.286468","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1389-1394"},"PeriodicalIF":8.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12130766/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143023208","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}
HaematologicaPub Date : 2025-06-01Epub Date: 2024-11-14DOI: 10.3324/haematol.2024.286504
Paul Knöbl
{"title":"The different faces of thrombotic thrombocytopenic purpura.","authors":"Paul Knöbl","doi":"10.3324/haematol.2024.286504","DOIUrl":"10.3324/haematol.2024.286504","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1245-1247"},"PeriodicalIF":8.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12130762/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142619100","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}