Blood advances最新文献

{"title":"Mutational profiling links biologically relevant variants to multisystemic Rosai-Dorfman disease.","authors":"Gordon J Ruan, Steven Tessier, Aishwarya Ravindran, Ubenthira Patgunarajah, David Pottinger, Saurabh Zanwar, Aldo A Acosta-Medina, Karen L Rech, Jason R Young, Jay H Ryu, Matthew J Koster, W Oliver Tobin, Mithun V Shah, N Nora Bennani, Robert Vassallo, Muhamad Alhaj Moustafa, Talal Hilal, Jennifer Jiang, Corrie Bach, Lucinda M Gruber, Alana E Gill, Ginny-Tyler Meadows, Adita Saha, Diana Morlote, Gaurav Goyal, Ronald S Go, Jithma P Abeykoon","doi":"10.1182/bloodadvances.2025019245","DOIUrl":"10.1182/bloodadvances.2025019245","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"5847-5851"},"PeriodicalIF":7.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13543939/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147980599","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":"When endurance running leads to functional iron deficiency.","authors":"Franklin Njoku, Victor R Gordeuk","doi":"10.1182/bloodadvances.2026020870","DOIUrl":"10.1182/bloodadvances.2026020870","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":"10 17","pages":"5914-5915"},"PeriodicalIF":7.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13544178/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148849984","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":"Outcomes of adolescents and young adults with AML treated on pediatric vs adult protocols.","authors":"Baher Krayem, Jeetayu Biswas, Andriy Derkach, Michael Rafizadeh, Jenna Ciervo, Christopher Famulare, Omar Abdel-Wahab, Neerav Shukla, Tanya Trippett, Christopher Forlenza, Kavitha Ramaswamy, Martin Tallman, Mark Geyer, Maria Luisa Sulis, Eytan M Stein","doi":"10.1182/bloodadvances.2026020372","DOIUrl":"10.1182/bloodadvances.2026020372","url":null,"abstract":"<p><strong>Abstract: </strong>Adolescents and young adults (AYA) with acute myeloid leukemia (AML) represent a biologically and clinically distinct population managed across pediatric and adult oncology services, with no established optimal treatment paradigm. In this single-center retrospective study, we analyzed 81 AYA patients aged 14 to 29 years with newly diagnosed AML treated at Memorial Sloan Kettering Cancer Center between 2010 and 2025. Patients were stratified by treating service, European LeukemiaNet (ELN) 2022 risk category, and induction regimen. Pediatric patients received 2 cycles of cytarabine, daunorubicin, and etoposide-based induction, whereas adult patients received 1 cycle of cytarabine plus daunorubicin (7+3) induction, 3 of whom required 7+3 reinduction, followed by risk-adapted consolidation including allogeneic hematopoietic stem cell transplantation. Composite complete response rates were similar between adult and pediatric cohorts (79% vs 78%), as were rates of minimal residual disease negativity by multicolor flow cytometry among responders (63% vs 55%). Despite comparable depth of response, 5-year overall survival favored adult-service patients in both the full cohort (77.9% vs 56.7%) and the ELN 2022 intermediate-adverse risk subgroup (62.7% vs 47.9%), whereas relapse-free survival was nearly identical. On multivariable analysis, ELN 2022 risk retained independent prognostic significance, whereas treatment protocol did not. These findings demonstrate that pediatric-style and adult AML regimens achieve comparable remission depth in AYA patients. The survival advantage observed with adult protocols challenges the assumption that further cytotoxic intensification improves outcomes in this population.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"5899-5908"},"PeriodicalIF":7.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13544186/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148276161","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":"EuroNet-PHL-LP1: complete resection or low-dose chemotherapy for pediatric low-risk nodular lymphocyte-predominant Hodgkin lymphoma.","authors":"Ananth Shankar, Judith Landman-Parker, Maurizio Mascarin, Andishe Attarbaschi, Sabah Boudjemaa, Roberta Burnelli, Auke Beishuizen, Michaela Cepelova, Francesco Ceppi, Thomas Georgi, Georgina Hall, Wolfram Klapper, Regine Kluge, Egesta Lopci, Monika L Metzger, Alan Ramsay, Jonas Steglich, Dietrich Stoevesandt, Lars Kurch, Dieter Körholz, Dirk Hasenclever, Christine Mauz-Körholz","doi":"10.1182/bloodadvances.2026019765","DOIUrl":"10.1182/bloodadvances.2026019765","url":null,"abstract":"<p><strong>Abstract: </strong>Children and adolescents with early-stage nodular lymphocyte-predominant Hodgkin lymphoma (nLPHL) have a 5-year event-free survival (EFS) of ∼77% after complete lymph node resection and ∼89% with anthracycline-containing chemotherapy. We investigated whether those patients can be successfully treated with surgical resection alone or with low-intensity, anthracycline-free CVP (cyclophosphamide, vinblastine, and prednisone) chemotherapy. EuroNet-PHL-LP1 was a prospective phase 3 trial enrolling patients aged <18 years with stage IA/IIA nLPHL. Complete resection led to active surveillance. Patients with unresectable disease received 3 cycles CVP. In CVP cohort 1, treatment cessation required negative 2-deoxy-2-[fluorine-18]fluoro-d-glucose positron emission tomography (18F-FDG PET). An amendment in 2014 omitted PET for end-of-treatment response; computed tomography and/or magnetic resonance imaging alone defined response in CVP cohort 2. The primary endpoint was 5-year EFS, with death, relapse, second malignancy, and PET positivity counting as events; for CVP cohort 2, progression-free survival (PFS) was primary. Among 267 registered patients, 247 were evaluable. Seventy-eight underwent resection only; their 5-year EFS/PFS was 79.5%. Six of 18 patients who relapsed after surgical resection reentered the CVP arm. In CVP cohort 1, 51 of 82 (62%) achieved complete metabolic response (CMR). Five-year EFS was 56.4%, whereas 5-year PFS in the CMR group was 91.3%. In contrast, PFS in 84 patients in CVP cohort 2 was 64.7%. During CVP, 68.3% of patients experienced grade 3 to 4 neutropenia. No treatment-related deaths were reported. Excellent outcomes were achieved after complete resection and with low-intensity, anthracycline-free chemotherapy if in CMR after chemotherapy. This strategy now constitutes the EuroNet-PHL standard of care for early-stage nLPHL. The trial was registered at www.clinicaltrialsregister.eu as EudraCT 2007-004092-19.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"5865-5873"},"PeriodicalIF":7.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13544184/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148155268","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":"Increased gene therapy efficacy through the use of extended half-life clotting factors.","authors":"Dries De Wolf, Phuong Anh Nguyen, Nisha Nair, Fangye Gao, Venkata Anudeep Bheemsetty, Ermira Samara-Kuko, Marinee K Chuah, Thierry VandenDriessche","doi":"10.1182/bloodadvances.2025018891","DOIUrl":"10.1182/bloodadvances.2025018891","url":null,"abstract":"<p><strong>Abstract: </strong>The development of more effective gene therapy strategies for hemophilia B remains essential because results of multiple clinical trials show significant interpatient variability in clinical outcomes, with a fraction of patients (up to 13%) even returning to prophylaxis. Another major challenge in current adeno-associated viral (AAV)-based gene therapy for hemophilia B pertains to dose-dependent vector-related immune responses, requiring immune suppression. We investigated whether combining gene therapy with transgenes encoding extended half-life (EHL) coagulation factors could enhance therapeutic efficacy. AAV vectors encoding a hyperactive factor IX variant R338L (ie, FIX-Padua), genetically fused to a mutant human albumin (FIX-R338L-ALB[QMP]), were administered to both wild-type and hemophilia B mice. These chimeric transgenes were codon-optimized and driven by a hepatocyte-specific promoter. The AAV-FIX-R338L-ALB(QMP) vector demonstrated significantly improved efficacy compared with the control AAV-FIX-R338L vector, resulting in a sustained fourfold and threefold increase in FIX antigen levels and activity, respectively. These increases were consistent with durable correction of the bleeding phenotype. Importantly, immunogenicity or liver toxicity was not increased because no anti-FIX antibodies, alanine transaminase elevations, or liver immune infiltrations were detected after treatment. Furthermore, immune tolerance was achieved in most hemophilia B mice treated with AAV-FIX-R338L-ALB(QMP), even after active immunization with FIX protein and adjuvant, which was found to be mediated by regulatory T cells. In addition, no increase in thrombogenic risk was observed, indicated by stable D-dimer levels. These findings support the potential of FIX-R338L-ALB(QMP) fusion constructs encoding EHL FIX as a promising next-generation gene therapy for hemophilia B.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"5852-5864"},"PeriodicalIF":7.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13544183/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148374621","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":"Shuffling the JAKs: playing a new card in chronic GVHD.","authors":"Julie R Boiko, Paul A Carpenter","doi":"10.1182/bloodadvances.2026021099","DOIUrl":"10.1182/bloodadvances.2026021099","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":"10 17","pages":"5834-5835"},"PeriodicalIF":7.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13543950/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148839294","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":"CAR T-cell therapy as a definitive consolidation for older adults with B-ALL in first complete remission.","authors":"Ibrahim Aldoss, Lior Goldberg, Jianying Zhang, Andrew Artz, Mary C Clark, Min Guan, Ruby Espinosa, Thiruvaimozhi Abimannan, Jiaqi Wu, Bryan Garcia, Eric R Haas, Khyatiben V Pathak, Brooke Lovell, Patrick Pirrotte, Vaibhav Agrawal, Neguine Sanani, Stephanie Kasten, Dileshni Tilakawardane, Jamie R Wagner, Jinny Paul, Haoyue Shan, Samer Khaled, Lihua E Budde, Paul Koller, Anthony S Stein, Vinod Pullarkat, Amandeep Salhotra, Ahmed Aribi, Guido Marcucci, Xiuli Wang, Stephen J Forman","doi":"10.1182/bloodadvances.2026020963","DOIUrl":"10.1182/bloodadvances.2026020963","url":null,"abstract":"<p><strong>Abstract: </strong>We report a phase 1 study assessing the safety and efficacy of CD19 chimeric antigen receptor (CAR) T cells as definitive consolidation in older adults (aged ≥55 years) with B-cell acute lymphoblastic leukemia (B-ALL) in first complete remission (CR1). Eighteen patients received lymphodepletion followed by infusion of memory-enriched CD19 CAR T cells. The median age was 64 years, and all patients were measurable residual disease (MRD)-negative before lymphodepletion. There were no dose-limiting toxicities, grade ≥2 cytokine release syndrome, or any grade immune effector cell-associated neurotoxicity syndrome. Estimated 18-month event-free and overall survival were 84% and 100%, respectively. CAR T cells expanded in the blood and cerebrospinal fluid despite patients' MRD-negative status. Comparing clinical samples from patients with relapsed/refractory (R/R) B-ALL from our historical trial (ClinicalTrials.gov identifier: NCT02146924) and patients in CR1, we found that the blood and CAR T-cell products from patients with R/R B-ALL were hyperinflammatory and hyperimmunometabolic, respectively. First-line CAR T-cell therapy was safe and well tolerated and potentially extended remission in patients in MRD-negative CR1. These findings support further investigation of the early use of CAR T-cell therapy for B-ALL. This trial was registered at www.clinicaltrials.gov as NCT05707273.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"5874-5885"},"PeriodicalIF":7.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13544187/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148315812","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":"Clinical course, risk factors, and therapeutic response in multiple myeloma with central nervous system involvement.","authors":"Alexander Xiao, Saurabh Zanwar, Yi Lin, Prashant Kapoor, Nadine Abdallah, Francis K Buadi, Moritz Binder, Angela Dispenzieri, David Dingli, Morie A Gertz, Wilson Gonsalves, Suzanne R Hayman, Taxiarchis Kourelis, Nelson Leung, Eli Muchtar, Mustaqeem Siddiqui, Melinda Tan, Rahma Warsame, S Vincent Rajkumar, Shaji Kumar","doi":"10.1182/bloodadvances.2026020668","DOIUrl":"10.1182/bloodadvances.2026020668","url":null,"abstract":"<p><strong>Abstract: </strong>Extramedullary disease (EMD) is increasingly recognized in relapsed/refractory multiple myeloma (MM), with central nervous system involvement (CNS-MM) representing a rare but highly aggressive manifestation associated with dismal outcomes. Data on its incidence and optimal management, particularly in the era of cellular and bispecific therapies, remain limited. We conducted a retrospective analysis of patients with MM with pathology-confirmed EMD treated at our institution between January 2000 and December 2023. CNS-MM was defined by parenchymal or leptomeningeal involvement. Clinical features, cytogenetics, treatments, and outcomes were analyzed, with predictors of CNS involvement evaluated among patients with EMD. Among 304 patients with EMD, 20 (6.5%) had CNS-MM. CNS involvement occurred a median of 19.2 months after MM diagnosis, though 15% presented at diagnosis. CNS-MM was strongly associated with high-risk cytogenetics (odds ratio [OR], 3.7) and additional visceral EMD (OR, 4.6). Median overall survival (OS) from CNS-MM diagnosis was 4.2 months. Outcomes were significantly improved in patients receiving chimeric antigen receptor T-cell therapy or bispecific antibodies, with a median OS of 19.2 months vs 1.2 months in those who did not receive immune effector therapies. Intrathecal (IT) chemotherapy demonstrated limited and transient benefit. In conclusion, CNS-MM is an aggressive complication enriched in high-risk, multifocal EMD with extremely poor prognosis. Cellular and bispecific therapies show promising activity and may meaningfully improve survival, whereas IT therapy appears to have limited efficacy. Prospective studies are needed to define optimal treatment strategies for CNS-MM.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"5828-5833"},"PeriodicalIF":7.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13543945/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148326554","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":"Stretching a doublet into a triplet for FLT3 AML.","authors":"Keith W Pratz","doi":"10.1182/bloodadvances.2026021100","DOIUrl":"10.1182/bloodadvances.2026021100","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":"10 17","pages":"5819-5820"},"PeriodicalIF":7.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13543938/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148839303","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":"Heestermans R, Olsen C, Ameli S, et al. Blood-derived cell-free DNA is a superior biomarker for noninvasive DNA methylation profiling in multiple myeloma. Blood Adv. 2025;9(16):4306-4310.","authors":"","doi":"10.1182/bloodadvances.2026021127","DOIUrl":"10.1182/bloodadvances.2026021127","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":"10 17","pages":"5909"},"PeriodicalIF":7.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13544166/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148850032","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}