Blood advances最新文献

{"title":"Symptoms, unmet needs, and quality of life in Erdheim-Chester disease: a longitudinal registry-based analysis.","authors":"Priya H Marathe, Anne S Reiner, Dana Bossert, Allison M Sigler, Deanna Fournier, Kathleen Brewer, Gaurav Goyal, Thomas M Atkinson, Jun J Mao, Katherine S Panageas, Eli L Diamond","doi":"10.1182/bloodadvances.2024015659","DOIUrl":"10.1182/bloodadvances.2024015659","url":null,"abstract":"<p><strong>Abstract: </strong>Measurement of patient-reported outcomes (PROs) and health-related quality of life (HrQOL) are crucial for comprehensive, patient-centered cancer care. Both PROs and HrQOL have been understudied in patients with Erdheim-Chester disease (ECD), a rare cancer with protean manifestations, dense symptomatology, and frequent diagnostic delay. We sought to evaluate the longitudinal evolution of symptom burden and unmet supportive care needs in patients with ECD, and to identify associations between these PROs and HrQOL. A registry-based cohort of patients with ECD completed a PRO battery including the Functional Assessment of Cancer Therapy-General (FACT-G) and other validated PRO measures. Descriptive statistics were used to characterize the distribution of PROs and FACT-G scores; PROs were modeled by univariable linear regression with FACT-G total score as the dependent variable at (1) registry enrollment and (2) 12-month time points. Changes in FACT-G total score (the difference between the 12-month and enrollment scores) were correlated with changes in PROs using univariable linear regression analysis. In 158 patients, mean total FACT-G was 70.8, lower than observed across multiple cancer cohorts. Higher levels of pain and fatigue, presence of neurologic symptoms, and greater number of unmet needs were all associated with worse HrQOL. Improvement in pain, fatigue, and unmet needs over 12 months was significantly associated with improvement in HrQOL. In patients with ECD, HrQOL is substantially diminished, even when considering other patients with cancer. Mitigation of symptoms and addressing unmet supportive care needs represent opportunities for intervention to improve HrQOL in ECD.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"4415-4424"},"PeriodicalIF":7.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12405683/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144552348","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":"Targeted GVL through HLA mismatch in double cord blood transplant.","authors":"Ann Dahlberg, Filippo Milano","doi":"10.1182/bloodadvances.2025016876","DOIUrl":"10.1182/bloodadvances.2025016876","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":"9 17","pages":"4470-4471"},"PeriodicalIF":7.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12410552/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144943627","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":"\"Optimizing\" HLH diagnoses in patients with lymphoma.","authors":"Joseph M Rocco, William T Johnson","doi":"10.1182/bloodadvances.2025017104","DOIUrl":"10.1182/bloodadvances.2025017104","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":"9 17","pages":"4513-4514"},"PeriodicalIF":7.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12447414/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145022913","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":"Allogeneic hematopoietic stem cell transplantation in germline DDX41 mutated patients with myeloid malignancies.","authors":"Michael Loschi, Marie-Charlotte Villy, Jacques-Emmanuel Galimard, Marie-Mathilde Auboiroux, Nathalie Gachard, Alexandre Perani, Matthieu Duchmann, Laetitia Largeaud, Stephanie Nguyen, Flore Sicre de Fontbrune, Marie Robin, Ibrahim Yakoub-Agha, Etienne Daguindau, Sylvain P Chantepie, Amandine Charbonnier, Delphine Lebon, Sebastien Maury, Helene Labussiere-Wallet, Anne Huynh, Edouard Forcade, Cristina Castilla-Llorente, Thomas Cluzeau, Lionel Adès, Maud D'Aveni, Raynier Devillier, Natacha Maillard, Sami Benachour, Hervé Dombret, Christian Récher, Arnaud Pigneux, Emmanuelle Clappier, Eric Delabesse, Nicolas Duployez, Pascal Turlure, Régis Peffault De Latour, Marie Sebert","doi":"10.1182/bloodadvances.2025017084","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025017084","url":null,"abstract":"<p><p>Germline DDX41 mutations (DDX41mut) are identified in approximately 5% of myeloid malignancies with excess of blasts, representing a distinct MDS/AML entity. The disease is associated with better outcomes compared to DDX41 wild-type (DDX41WT), but patients who do not undergo allogeneic hematopoietic stem cell transplantation (HSCT) may experience late relapse. Due to the recent identification of DDX41mut, data on post-HSCT outcomes remain limited. Here, we first report the HSCT outcomes of 83 DDX41mut MDS/AML patients. With a median follow-up (FU) of 4.4 years, the 2-year leukemia-free survival (LFS) was 68.6% (95% CI, 57.1 - 77.6), with a 2-year non-relapse mortality (NRM) of 21.1% (95% CI, 12.9 - 30.6). We then assessed the impact of DDX41mut using a pair match analysis performed on patients who underwent transplantation in AML clinical trials. No significant differences were observed between DDX41mut and DDX41WT patients in terms of LFS at 2 years (HR: 1.06, 95% CI, 0.59-1.90, p=0.84), overall survival (OS), NRM, relapse, or graft-versus-host disease (GVHD) incidence. The CIR for DDX41mut showed a trend toward a lower relapse rate during the first year and a higher relapse rate after 1 year. Given the familial nature of the disease, we specifically examined patients who relapsed after HSCT with a related donor, identifying 7 cases of DDX41mut donor cell leukemia (DCL). In our study, HSCT in DDX41mut AML patients was not associated with an increased risk of toxicity. However, we observed a potential for later relapse, which could potentially be mitigated by selecting related donors based on DDX41 status.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145028954","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":"Tumor microenvironment differences between diagnostic and relapsed classic Hodgkin lymphoma revealed by scRNA-seq.","authors":"Yifan Yin, Shinya Rai, Aixiang Jiang, Alexander M Xu, Luke O'Brien, Adèle Telenius, Jan Delabie, Lauren C Chong, Stacy S Hung, Akil A Merchant, David W Scott, Kerry J Savage, Tomohiro Aoki, Christian Steidl","doi":"10.1182/bloodadvances.2025017107","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025017107","url":null,"abstract":"<p><p>Classical Hodgkin Lymphoma (CHL) is characterized by a complex tumor microenvironment (TME) that supports disease progression. While immune cell recruitment by Hodgkin and Reed-Sternberg (HRS) cells is well-documented, the role of non-malignant B cells in relapse remains unclear. Using single-cell RNA sequencing (scRNA-seq) on paired diagnostic and relapsed CHL samples, we identified distinct shifts in B-cell populations, particularly an enrichment of naïve B cells and a reduction of memory B cells in early-relapse compared to late-relapse and newly diagnosed CHL. The enrichment of naïve B cells in early relapse biopsies was confirmed in independent validation cohorts using scRNA-seq and immunohistochemistry. Notably, naïve B cells in early-relapse samples exhibited high expression of LGALS9, an immunosuppressive gene encoding Galectin-9, which binds to HAVCR2 (TIM-3, T-cell immunoglobulin and mucin domain-containing protein 3) on regulatory T cells (Tregs). Cell-cell interaction analysis revealed the importance of interactions between LGALS9+ naïve B cells and HAVCR2+ Tregs in the early-relapse setting. Spatial analysis by imaging mass cytometry confirmed close proximity of Galectin-9+ naïve B cells with TIM-3⁺ CD4⁺ T cells and HRS cells, pointing to their role in shaping an immunosuppressive niche. Our findings highlight a previously unrecognized population of Galectin-9+ naïve B cells with immunoregulatory potential in early-relapse CHL and provide new insights into the spatial and transcriptional architecture of the relapsed TME in CHL.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145028992","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":"Efficacy of MRD-triggered Anti-CD20 Retreatment in Patients with High-Risk Chronic Lymphocytic Leukemia: A Case Series.","authors":"Ellinor Goergen, Sandra Robrecht, Rudy Ligtvoet, Kirsten Fischer, Barbara F Eichhorst, Petra Langerbeins, Michael J Hallek","doi":"10.1182/bloodadvances.2025017109","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025017109","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145022837","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":"The efficacy of rosuvastatin to reduce circulating tissue factor extracellular vesicles after ovarian cancer surgery.","authors":"Soravis Osataphan, Rushad Patell, Mrigya Babuta, Rachel P Rosovsky, Yohei Hisada, Charly Edmiston, Pavania Elavalakanar, Michael A Pfeffer, Amy Bregar, Amanda Ramos, Leslie Garrett, Siyang Ren, Donna Neuberg, Gyongyi Szabo, Robert Flaumenhaft, Nigel Mackman, Meghan Shea, Jeffrey I Zwicker","doi":"10.1182/bloodadvances.2025016107","DOIUrl":"10.1182/bloodadvances.2025016107","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"4472-4476"},"PeriodicalIF":7.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12410534/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144233243","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":"HCAR2 is a novel receptor for heme.","authors":"Anne Grunenwald, Julie Peliconi, Alessandra Zarantonello, Jordan D Dimitrov, Lubka T Roumenina, Nicolas S Merle","doi":"10.1182/bloodadvances.2025016197","DOIUrl":"10.1182/bloodadvances.2025016197","url":null,"abstract":"<p><strong>Abstract: </strong>Extracellular heme, released during intravascular hemolysis in sickle cell disease (SCD) and hemolytic anemia, acts as a proinflammatory danger signal, requiring robust defense mechanisms. Previous studies identified G protein-coupled receptor (GPCR) signaling triggered by heme, but the specific receptor remained unknown. Transcriptomic analysis of bulk RNA sequencing of liver tissues from SCD and hemolytic mice (injection of phenylhydrazine) revealed GPCR signaling as a commonly enriched pathway. Unbiased screening of 241 GPCRs identified Hydroxycarboxylic Acid Receptor 2 (HCAR2/GPR109A), an anti-inflammatory receptor for niacin, as a novel heme sensor. Heme binding to human HCAR2 was validated using a functional reporter cell assay and direct interaction analyses via surface plasmon resonance and absorbance spectroscopy. In vivo, HCAR2 was upregulated in the liver of SCD and hemolytic mice, paralleling the expression of the heme-degrading enzyme heme oxygenase-1 (HO-1). HO-1 inhibition or heme injection further increased HCAR2 expression, indicating that heme acts as both a ligand and an inducer of HCAR2. These findings identify HCAR2 as a novel heme receptor and reveal a heme-HCAR2-HO-1 negative feedback loop involved in tissue protection in hemolytic diseases.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"4458-4469"},"PeriodicalIF":7.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12410535/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143981086","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":"Up-front alternative donor HCT in severe aplastic anemia: gaps and opportunities to translate evidence into practice.","authors":"Neel S Bhatt, Azra Borogovac, Yvonne A Efebera, Anna DeSalvo, Steven M Devine, Amy Foley, Valerie Greco-Stewart, Betty K Hamilton, Mykala Heuer, Todd Molfenter, John P Plastaras, Brittany K Ragon, Sarah A Wall, Larisa Broglie, Mark B Juckett, Nandita Khera, Mary M Horowitz, Amy E DeZern","doi":"10.1182/bloodadvances.2024015405","DOIUrl":"10.1182/bloodadvances.2024015405","url":null,"abstract":"<p><strong>Abstract: </strong>Severe aplastic anemia (SAA) is a rare and life-threatening bone marrow failure disorder. Immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporine has long been a frontline treatment option in SAA; however, its limited durability and risk of long-term complications such as secondary malignancies remain a drawback in this treatment modality. Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative option with significantly improved outcomes over the long term, particularly with HLA-matched related donors. However, the use of alternative donors, such as haploidentical, mismatched, or matched unrelated donors, has previously been limited due to increased transplant-related morbidity, particularly graft-versus-host disease (GVHD). HCTs have therefore been limited to young recipients and those with HLA-matched related donors, creating significant disparity for older adults and those who lack matched donor options. Nevertheless, more recent advances in HCT, such as posttransplant cyclophosphamide for GVHD prophylaxis, have led to improved outcomes of HCT with alternative donors; however, alternative donor HCT remains underused as up-front therapy, in part because of limited multicenter trial data. This review discusses current SAA treatment approaches, including both IST and HCT, and highlights remaining gaps. It also discusses how ongoing clinical trials such as CureAA and TransIT could help address these gaps. Furthermore, we discuss the importance of stakeholder engagement and implementation science in the integration of research-based evidence into clinical practice. Bridging these gaps is necessary for achieving equitable access for patients historically excluded from frontline HCT, including older adults and racially or ethnically diverse populations.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"4448-4457"},"PeriodicalIF":7.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12410533/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144483083","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":"A mixed methods study to understand the role of knowledge in transition among youth with sickle cell disease.","authors":"Ashima Singh, Mahua Dasgupta, M Muska Nataliansyah, Nicole Steiner, Jeffrey Karst, Meghan Miller, Joshua J Field, Melissa Azul, Liliana E Pezzin, Amanda M Brandow","doi":"10.1182/bloodadvances.2025015984","DOIUrl":"10.1182/bloodadvances.2025015984","url":null,"abstract":"<p><strong>Abstract: </strong>Disease and management knowledge is crucial for individuals with sickle cell disease (SCD) transitioning from a pediatric to adult health care facility. However, there is a lack of understanding regarding the specific education needed and its association with an individual's ability to successfully transition. This study aims to explore the association between SCD-specific transition readiness assessment scores, patient characteristics, and the perceived importance and confidence in the individual's ability to manage their health and transition to an adult doctor. Semistructured interviews provided insights on education gaps and preferences. Eighty-four individuals completed the transition readiness assessment. Younger patients (aged ≤18 years) had significantly lower scores on the appointment (1.1 [standard deviation (SD), 0.7] vs 1.6 [SD, 0.6]; P = .007) and insurance (0.5 [SD, 0.7] vs 1.5 [SD, 1.1]; P = .0006) domains as compared with older (aged >18 years) individuals. Those who were very confident in their ability to manage their health care had significantly higher scores in disease knowledge (2.4 [SD, 0.5] vs 2.0 [SD, 0.7]; P = .01), medication management (2.5 [SD, 0.6] vs 2.1 [SD, 0.4]; P = .0001), appointments (1.4 [SD, 0.7] vs 0.9 [SD, 0.5]; P = .0006), and insurance (0.9 [SD, 0.9] vs 0.5 [SD, 0.8]; P = .02), compared with those who were not. Semistructured interviews with 9 young adults revealed a preference for transition education to start in their midteens, at least twice a year, and using a combination of approaches. Additional themes identified included the desire for ongoing education, familiarity with the workflow and environment, access to care, and the importance of support systems during their transition.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"4477-4485"},"PeriodicalIF":7.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12410538/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144483172","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}