Blood最新文献

{"title":"IKZF1: born to be the repressor.","authors":"Mariana Emerenciano","doi":"10.1182/blood.2024026967","DOIUrl":"https://doi.org/10.1182/blood.2024026967","url":null,"abstract":"","PeriodicalId":9102,"journal":{"name":"Blood","volume":"145 4","pages":"354-356"},"PeriodicalIF":21.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143022111","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":"Montoro J, Eikema D-J, Tuffnell J, et al. Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT. Blood. 2024;144(3):323-333.","authors":"","doi":"10.1182/blood.2024027933","DOIUrl":"https://doi.org/10.1182/blood.2024027933","url":null,"abstract":"","PeriodicalId":9102,"journal":{"name":"Blood","volume":"145 4","pages":"456"},"PeriodicalIF":21.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143022113","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":"Safety and efficacy of pegcetacoplan treatment for cold agglutinin disease and warm antibody autoimmune hemolytic anemia.","authors":"Eloy Roman, Bruno Fattizzo, Merrill Shum, Wahid Hanna, Steven R Lentz, Sergio Schusterschitz S Araujo, Mohammed Al-Adhami, Federico V Grossi, Morie A Gertz","doi":"10.1182/blood.2023022549","DOIUrl":"10.1182/blood.2023022549","url":null,"abstract":"<p><strong>Abstract: </strong>Cold agglutinin disease (CAD) and warm antibody autoimmune hemolytic anemia (wAIHA) are rare autoimmune hemolytic anemias characterized by red blood cell destruction, largely attributable to complement activation resulting in intravascular and extravascular hemolysis. Pegcetacoplan is a subcutaneously administered C3-targeted therapy, which may be suitable for treating CAD and wAIHA. In this open-label phase 2 study, analyses were conducted in 2 cohorts, 1 for patients with CAD and the other for those with wAIHA. In each cohort, patients were randomly assigned to receive pegcetacoplan 270 mg/d or 360 mg/d for up to 48 weeks. Safety end points included the incidence and severity of treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESI). Efficacy end points included change from baseline in hemoglobin (Hb), lactate dehydrogenase, absolute reticulocyte count, haptoglobin, indirect bilirubin, and functional assessment of chronic illness therapy (FACIT)-fatigue scale. Thirteen of 13 (100%) and 10 of 11 (91%) patients with CAD and wAIHA, respectively, experienced at least 1 TEAE. Ten patients had at least 1 serious AE; none were considered related to pegcetacoplan. The only treatment-related AESIs were injection site reactions. Pegcetacoplan increased Hb levels, reduced hemolysis, and increased FACIT-fatigue scale scores in the first weeks; at week 48 the median (interquartile range) change from baseline Hb for the CAD and wAIHA total groups was 2.4 (0.90-3.00) and 1.7 g/dL (-1.40 to 2.90), respectively, and improvements in hemolysis and FACIT-fatigue scale scores were maintained. This study demonstrated that pegcetacoplan is generally well tolerated and suggests it can be effective for patients with CAD and wAIHA. This trial was registered at www.ClinicalTrials.gov as #NCT03226678.</p>","PeriodicalId":9102,"journal":{"name":"Blood","volume":" ","pages":"397-408"},"PeriodicalIF":21.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142562628","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":"How I treat iron overload in adult MDS.","authors":"Heather A Leitch, Rena Buckstein","doi":"10.1182/blood.2023022501","DOIUrl":"10.1182/blood.2023022501","url":null,"abstract":"<p><strong>Abstract: </strong>Although clinical benefits of iron chelation therapy (ICT) in red blood cell (RBC) transfusion-dependent (TD) hereditary anemias such as α-thalassemia major are incontrovertible, the evidence supporting a similar benefit in patients with TD myelodysplastic neoplasms (MDS) and iron overload (IOL) is sometimes debated. MDS presents later in life, has a limited repertoire of life-extending therapies, and patients may have comorbidities acting as competing causes of death. However, refined prognostication identifies patients with MDS with a reasonable life expectancy, and because 50% of patients will ultimately become RBC TD and develop transfusional IOL, ICT should be considered in some. Using illustrative cases, we summarize mechanisms of iron toxicity, strategies for the identification of IOL, and propose definitions of IOL severity. We provide rationale for, and recommend which patients may benefit from, ICT. We discuss currently available chelators, their administration, monitoring, side effects, and their management. Given challenges with the use of iron chelators, we suggest the nuances to be considered when planning chelation initiation to include the rate of iron accumulation, the presence of organ iron and/or dysfunction, and detectable indicators of oxidative stress. Areas for future investigation are identified.</p>","PeriodicalId":9102,"journal":{"name":"Blood","volume":" ","pages":"383-396"},"PeriodicalIF":21.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141466155","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":"CD5-positive high-grade B-cell lymphoma with MYC, BCL2, and BCL6 rearrangements.","authors":"Wei J Wang, Zhihong Hu","doi":"10.1182/blood.2024027114","DOIUrl":"https://doi.org/10.1182/blood.2024027114","url":null,"abstract":"","PeriodicalId":9102,"journal":{"name":"Blood","volume":"145 4","pages":"455"},"PeriodicalIF":21.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143022109","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":"Masthead Subscription","authors":"","doi":"10.1016/s0006-4971(25)00095-3","DOIUrl":"https://doi.org/10.1016/s0006-4971(25)00095-3","url":null,"abstract":"","PeriodicalId":9102,"journal":{"name":"Blood","volume":"77 6 1","pages":""},"PeriodicalIF":20.3,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143072061","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":"Ex vivo venetoclax sensitivity predicts clinical response in acute myeloid leukemia in the prospective VenEx trial.","authors":"Sari Kytölä, Ida Vänttinen, Tanja Ruokoranta, Anu Partanen, Annasofia Holopainen, Joseph Saad, Milla E L Kuusisto, Sirpa Koskela, Riikka Räty, Maija Itälä-Remes, Imre Västrik, Minna Suvela, Alun Parsons, Kimmo Porkka, Krister Wennerberg, Caroline A Heckman, Tero Jalkanen, Teppo Huttunen, Pia Ettala, Johanna Rimpiläinen, Timo Siitonen, Marja Pyörälä, Heikki Kuusanmäki, Mika Kontro","doi":"10.1182/blood.2024024968","DOIUrl":"10.1182/blood.2024024968","url":null,"abstract":"<p><strong>Abstract: </strong>The B-cell lymphoma 2 inhibitor venetoclax has shown promise for treating acute myeloid leukemia (AML). However, identifying patients likely to respond remains a challenge, especially for those with relapsed/refractory (R/R) disease. We evaluated the utility of ex vivo venetoclax sensitivity testing to predict treatment responses to venetoclax-azacitidine in a prospective, multicenter, phase 2 trial. The trial recruited 104 participants with previously untreated (n = 48), R/R (n = 39), or previously treated secondary AML (sAML) (n = 17). The primary end point was complete remission or complete remission with incomplete hematologic recovery (CR/CRi) rate in ex vivo sensitive trial participants during the first 3 therapy cycles. The key secondary end points included the correlations between ex vivo drug sensitivity, responses, and survival. Venetoclax sensitivity was successfully assessed in 102 of 104 participants, with results available within a median of 3 days from sampling. In previously untreated AML, ex vivo sensitivity corresponded to an 85% (34/40) CR/CRi rate, with a median overall survival (OS) of 28.7 months, compared with 5.5 months for ex vivo resistant patients (P = .002). For R/R/sAML, ex vivo sensitivity resulted in a 62% CR/CRi rate (21/34) and median OS of 9.7 vs 3.3 months for ex vivo resistant patients (P < .001). In univariate and multivariate analysis, ex vivo venetoclax sensitivity was the strongest predictor for a favorable treatment response and survival. This trial demonstrates the feasibility of integrating ex vivo drug testing into clinical practice to identify patients with AML, particularly in the R/R setting, who benefit from venetoclax. This trial was registered at www.clinicaltrials.gov as #NCT04267081.</p>","PeriodicalId":9102,"journal":{"name":"Blood","volume":" ","pages":"409-421"},"PeriodicalIF":21.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142364344","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":"Inflammatory Waldenström macroglobulinemia is associated with clonal hematopoiesis: a multicentric cohort.","authors":"Pierre-Edouard Debureaux, Stéphanie Poulain, Stéphanie Harel, Marie Passet, Marie Templé, Chloé Friedrich, Nathalie Forgeard, Dikelele Elessa, William Plas, Laureen Chat, Grégory Lazarian, Lise Willems, Bruno Royer, Alexis Talbot, Tristan Vaugeois, Floriane Theves, Alexandre Terré, Anne Brignier, Marion Malphettes, Daphné Krzisch, Laurent Frenzel, Frédéric Davi, Clotilde Bravetti, Florence Nguyen-Khac, Jehan Dupuis, Wendy Cuccuini, Didier Bouscary, Olivier Hermine, Damien Roos-Weil, Olivier Kosmider, Emmanuelle Clappier, Marion Espéli, Karl Balabanian, Bertrand Arnulf","doi":"10.1182/blood.2024025738","DOIUrl":"10.1182/blood.2024025738","url":null,"abstract":"<p><strong>Abstract: </strong>Inflammatory form of Waldenström macroglobulinemia (iWM) predicts outcomes after immuno-chemotherapy and Bruton tyrosine kinase inhibitors, but its origin is unknown. Here, we unravel increased clonal hematopoiesis in patients with iWM (61% vs 23% in noninflammatory WM), suggesting a contribution of environmental cells to iWM.</p>","PeriodicalId":9102,"journal":{"name":"Blood","volume":" ","pages":"450-454"},"PeriodicalIF":21.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142685960","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":"\"Complementing\" hemolytic anemias: what's next?","authors":"Eleni Gavriilaki, Gloria F Gerber","doi":"10.1182/blood.2024026626","DOIUrl":"https://doi.org/10.1182/blood.2024026626","url":null,"abstract":"","PeriodicalId":9102,"journal":{"name":"Blood","volume":"145 4","pages":"351-352"},"PeriodicalIF":21.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143022106","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":"VenEx precisely predicts ven-aza response.","authors":"Pamela S Becker","doi":"10.1182/blood.2024027054","DOIUrl":"10.1182/blood.2024027054","url":null,"abstract":"","PeriodicalId":9102,"journal":{"name":"Blood","volume":"145 4","pages":"353-354"},"PeriodicalIF":21.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143022117","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}