Blood advances最新文献

{"title":"MRD-guided ibrutinib plus venetoclax improves quality of life in relapsed/refractory CLL: HOVON 141/VISION trial results.","authors":"Laura Elisabeth Maria Eurelings, Emelie Curovic Rotbain, Sabina Kersting, Lina Van Der Straten, Lisbeth Enggaard, Gerrit-Jan Veldhuis, Rogier Mous, Christian Bjørn Poulsen, Henrik Frederiksen, Ann Mh Janssens, Ida Schjødt, Laurens Laterveer, Juha Ranti, Christian Brieghel, Mattias Mattsson, Mar Bellido, Hoa Thi Tuyet T T Tran, Claudia A M Stege, Kazem Nasserinejad, Arnon P Kater, Carsten Utoft Niemann, Mark-David Levin","doi":"10.1182/bloodadvances.2025019500","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025019500","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.7,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149223235","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":"Response to \" Bone Mineral Density in Hemophilia & VWD\".","authors":"Divyaswathi Citla-Sridhar, Simon Chung, Andrew W Brown, Shelley E Crary, Sanjay Ahuja, Steven Bruzek, Vera Ignjatovic, Neil A Goldenberg, Jin-Ran Chen","doi":"10.1182/bloodadvances.2026021513","DOIUrl":"https://doi.org/10.1182/bloodadvances.2026021513","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.7,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149223199","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":"Advancing Sickle Cell Disease Care Through Collaboration: the HHS and ASH Registry Leaders' Roundtable Initiative.","authors":"William A Wood, Ted Wun, Alexis A Thompson","doi":"10.1182/bloodadvances.2025017965","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025017965","url":null,"abstract":"<p><p>Access to robust data is essential to addressing complex problems for people living with SCD; however, current data sources have significant limitations (1). Manual data capture programs are highly curated but are challenging to scale, while claims or EHR-based databases scale rapidly but face challenges with reliability and validity. There have been limited coordinated efforts to address programmatic redundancies and minimize burden for clinicians, researchers, and patients. In 2022 and 2023, three Sickle Cell Disease (SCD) Registry Leaders Roundtable sessions were convened by the U.S. Department of Health and Human Services (HHS) in collaboration with the American Society of Hematology (ASH). HHS subsequently initiated development of an SCD-specific U.S. Core Data for Interoperability Plus program (USCDI+ SCD) (2). In this viewpoint, we argue that a strong framework for interoperability, linkages between existing data efforts, and targeted curation informed by clinical expertise are necessary components of a national SCD data infrastructure.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.7,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149223176","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":"Long-Term Outcomes in Patients with Relapsed or Refractory Severe Aplastic Anemia after Immunosuppression.","authors":"Fieke W Hoff, Luiz Fernando Bazzo Catto, Ruba N Shalhoub, Olga Julia Rios, Colin O Wu, Neal S Young, Bhavisha A Patel, Emma M Groarke","doi":"10.1182/bloodadvances.2026021514","DOIUrl":"https://doi.org/10.1182/bloodadvances.2026021514","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.7,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149223207","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":"National Alliance of Sickle Cell Disease Consensus Recommendations for Gene Therapy Implementation.","authors":"Alexis Leonard, Sharl Azar, Christopher M McKinney, Roy L Kao, John P Manis, Sophie Lanzkron, Julie Kanter","doi":"10.1182/bloodadvances.2026021027","DOIUrl":"https://doi.org/10.1182/bloodadvances.2026021027","url":null,"abstract":"<p><p>Gene therapy for sickle cell disease (SCD) is rapidly advancing, with approved products and others in development, creating an urgent need to define how treatment centers can safely and effectively deliver this care. The National Alliance of Sickle Cell Centers (NASCC) conducted a modified Delphi consensus process involving 64 SCD centers across the United States with expertise in SCD care to identify essential elements required for gene therapy delivery and longitudinal patient management. Participants evaluated proposed elements of care delivery and center infrastructure, with consensus defined a priori as ³70% agreement on categorization. Twenty-four elements were identified as essential, and four as recommended for optimal implementation. These elements span clinical infrastructure, multidisciplinary care, treatment delivery, and longitudinal follow-up. The resulting framework provides a practical foundation to guide centers in developing and standardizing gene therapy programs for SCD. These consensus recommendations support safe, consistent, and high-quality care as access to gene therapy expands and may inform future clinical practice, program development, and research in this evolving field.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.7,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149223217","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":"Thromboprophylaxis and HA-VTE Prediction.","authors":"Mengyao Li, Zhu Zhang, Zhenguo Zhai, Bingzhang Zou","doi":"10.1182/bloodadvances.2026022436","DOIUrl":"https://doi.org/10.1182/bloodadvances.2026022436","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.7,"publicationDate":"2026-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149206525","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":"Achieved hemoglobin is not a dosing target in MDS.","authors":"Na Liao, Ting Ye","doi":"10.1182/bloodadvances.2026022545","DOIUrl":"https://doi.org/10.1182/bloodadvances.2026022545","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.7,"publicationDate":"2026-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149206488","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":"Clinical characteristics and outcome of a cohort of CLL patients with BTK T474 gatekeeper mutation.","authors":"Zulfa Omer, Kerry A Rogers, Max Yano, Jayesh Rai, Joyce Chen, Jennifer A Woyach, Seema A Bhat, Daniel Jones, Michael Rhodes Grever, Erin Hertlein, Sara Elgamal, John C Byrd","doi":"10.1182/bloodadvances.2025019251","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025019251","url":null,"abstract":"<p><p>BTK T474 gatekeeper mutations confer resistance to both commercially approved covalent and non-covalent BTK inhibitors (BTKis) in chronic lymphocytic leukemia (CLL), yet their clinical implications remain poorly characterized. While pirtobrutinib was developed to overcome resistance mediated by BTK C481 mutations, its use has been increasingly associated with the emergence of BTK T474, which is associated with resistance to this and commercially available BTKis. We retrospectively analyzed 17 relapsed/refractory CLL patients from two academic centers who acquired BTK T474 mutations on BTKi therapy, integrating paired next-generation sequencing (NGS), cytogenetics, and clinical outcomes. Results showed that this was a heavily pretreated, high-risk cohort; 42% had del(17p) and/or TP53 mutations. T474I was the most common substitution (82%). At first T474 detection, patients were receiving ibrutinib (6/17), acalabrutinib (5/17), pirtobrutinib (5/17), or nemtabrutinib (1/17); median time on the current BTKi at time of mutation detection was 28 months (range, 4-69). Concurrent BTK C481 mutations were present in 14/17 (82%). Median T474 VAF at progression was 13%, and all patients progressed after T474 detection with a median time to progression of 4 months. Non-BTK mutations were frequent (82%), most commonly TP53 and SF3B1. After progression, venetoclax-based therapy produced the longest median progression-free survival (18 months). Median overall survival was 44 months from first documentation of T474 mutation (95% CI, 20-62) and PFS after T474 detection was 6 months. This analysis showed that BTK T474 emergence identifies a high-risk CLL population with rapid progression after detection, underscoring the need for effective post-BTKi strategies.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.7,"publicationDate":"2026-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149206442","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":"Persistent Thromboxane Biosynthesis Despite Aspirin Therapy Predicts Preeclampsia.","authors":"Paola G Simeone, Rossella Liani, Margherita Alfonsetti, Alessa Ricci, Valerio Cianci, Zeeba Saeed, Marina Molino, Stefano Palma, Raniero Malizia, Myriam Luongo, Augusto Di Castelnuovo, Sabina Gallina, Francesco Cipollone, Fabrizio Ricci, Giulia Renda, Francesco D'Antonio, Francesca Santilli","doi":"10.1182/bloodadvances.2026021230","DOIUrl":"https://doi.org/10.1182/bloodadvances.2026021230","url":null,"abstract":"<p><p>Preeclampsia (PE) is associated with enhanced platelet activation, with thromboxane A₂ playing a key pathogenic role. Urinary 11-dehydro-thromboxane B₂ (TXM) reflects in vivo platelet activation. Although low-dose aspirin (ASA) is recommended for PE prevention in high-risk pregnancies, variable thromboxane suppression may lead to persistent platelet activation. This study aimed at identifying platelet-derived predictors of PE in high-risk women receiving ASA and assessing biomarkers improving risk stratification. This observational study included 25 high-risk pregnant women on ASA and 12 with physiological pregnancies. Clinical evaluation and blood/urine sampling were performed at baseline (T0), and at 21 (T4) and 27 (T5) weeks. Urinary TXM and percentage inhibition (<75% = reduced inhibition) were assessed. Platelet biomarkers included thrombopoietin (TPO), glycocalicin (GC), P-selectin, and CD40L. Late-onset PE occurred in 6/25 (24%) high-risk women and in none of the controls. TXM levels were higher in high-risk versus physiological pregnancies (p=0.009), especially at T4-T5 (p<0.0001). Women developing PE had higher TXM at T4 (adjusted p=0.014) and lower TXM inhibition (51% vs 81%; p=0.020). Reduced inhibition (<75%) was associated with increased PE risk (44.4% vs 14.3%; OR 4.8). TPO showed a different longitudinal pattern according to PE occurrence (time-by-outcome interaction p=0.036). Glycocalicin showed higher estimated values at baseline in women who developed PE (p=0.048), but the time-specific contrast was attenuated after Holm adjustment (adjusted p=0.19). Incomplete thromboxane suppression during ASA therapy is associated with PE. TXM, together with opposite trends of TPO and GC, identifies a profile consistent with altered platelet homeostasis and possible changes in platelet turnover, potentially enhancing risk prediction in high-risk pregnancies.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.7,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149191193","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":"Perspectives on FDA Guidance for Myelodysplastic Syndromes.","authors":"Asha Kodan, Amy E DeZern, Mikkael A Sekeres, Joseph Kannarkat","doi":"10.1182/bloodadvances.2026022255","DOIUrl":"https://doi.org/10.1182/bloodadvances.2026022255","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.7,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149191234","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}