Blood advances最新文献_第7页

Therapeutic innovations in hemophilia: the essential role of a positive reinvestment cycle. 血友病治疗创新：积极再投资周期的重要作用。

IF 7.1 1区医学

Blood advances Pub Date : 2025-09-23 DOI: 10.1182/bloodadvances.2025016497

Cedric Hermans, Glenn F Pierce

{"title":"Therapeutic innovations in hemophilia: the essential role of a positive reinvestment cycle.","authors":"Cedric Hermans, Glenn F Pierce","doi":"10.1182/bloodadvances.2025016497","DOIUrl":"10.1182/bloodadvances.2025016497","url":null,"abstract":"Abstract: Hemophilia stands out among rare genetic diseases for its significant therapeutic advancements, closely tied to substantial financial investments. Key factors driving this progress include severe hemorrhagic consequences from an early age, its impact on royal families, the HIV and hepatitis C contamination tragedies, the identification of factor VIII (FVIII) and FIX genes, and advancements in biotechnology. Maintaining low, measurable concentrations of FVIII or FIX in the blood has proven pivotal in improving patient outcomes. The mobilization of the global hemophilia community, led by the World Federation of Hemophilia, the European Association for Haemophilia and Allied Disorders, and the National Bleeding Disorder Foundation, has continuously advocated for access to safe, effective treatments. With reinvestments from biopharmaceutical partners, revolutionary options, including gene therapy, have emerged. However, this cycle of innovation and investment, essential for curing all patients worldwide, faces potential threats. This article aims to highlight the critical importance of investing in hemophilia treatment and research, a topic of concern for all stakeholders within the hemophilia community.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"4631-4639"},"PeriodicalIF":7.1,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12496250/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144538961","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}

引用次数: 0

Gene-edited CAR T cells: going the distance in B-ALL. 基因编辑的CAR - T细胞：在B-ALL中走完全程。

IF 7.1 1区医学

Blood advances Pub Date : 2025-09-23 DOI: 10.1182/bloodadvances.2025017508

John A Ligon, Nirali N Shah

引用次数: 0

Efficacy and safety of BCMA nanobody CAR T-cell therapy in relapsed or refractory plasma cell myeloma. BCMA纳米体CAR-T细胞治疗复发或难治性浆细胞骨髓瘤的疗效和安全性。

IF 7.1 1区医学

Blood advances Pub Date : 2025-09-23 DOI: 10.1182/bloodadvances.2025016322

Xian G Zhang, Lin Wang, Junfang Yang, Xiaona N Hu, Hui Wang, Lina N Zhang, Xiaoge Zhou, Ying Liu, Qinglong Wang, Peihua H Lu

{"title":"Efficacy and safety of BCMA nanobody CAR T-cell therapy in relapsed or refractory plasma cell myeloma.","authors":"Xian G Zhang, Lin Wang, Junfang Yang, Xiaona N Hu, Hui Wang, Lina N Zhang, Xiaoge Zhou, Ying Liu, Qinglong Wang, Peihua H Lu","doi":"10.1182/bloodadvances.2025016322","DOIUrl":"10.1182/bloodadvances.2025016322","url":null,"abstract":"Abstract: B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell therapy has demonstrated promising therapeutic efficacy in relapsed or refractory (R/R) multiple myeloma. However, distinct CAR T-cell constructs exhibit varying therapeutic outcomes. As the antigen-recognition domain, nanobodies offer a small, stable, single-domain structure with enhanced affinity and specificity compared with conventional single-chain variable fragments. We explored the use of nanobody-based BCMA(S103) CAR T-cell therapy for R/R plasma cell myeloma. The CAR construct incorporates dual-nanobody variable domain of the heavy chain of heavy chain antibody (VHHs) targeting BCMA. A cohort of 27 patients was treated with S103 CAR T-cell therapy, which included 4 patients of plasma cell leukemia, and 1 patient of anaplastic plasma cell myeloma. Eleven patients had multiple extramedullary lesions, and 11 patients exhibited high-risk genetic abnormalities, including 4 with TP53 mutations. One month after CAR T-cell infusion, the overall response rate (ORR) was 96.3% (26/27), with a complete response (CR) + very good partial response (VGPR) rate of 59.2% (16/27). At the 3-month follow-up, the ORR increased to 100% (27/27), with a CR + VGPR rate of 81.5% (22/27). The median duration of remission was 11 months (range, 2-36 months). The 1-year overall survival rate was 61.1%, and progression-free survival was 57.2%. In conclusion, BCMA CAR T-cell therapy, utilizing dual-nanobody VHHs targeting BCMA, demonstrates a high ORR and manageable safety profile in treating patients with R/R plasmacytic myeloma, including those with high-risk features such as extramedullary lesions, high-risk cytogenetic abnormalities, plasma cell leukemia, or anaplastic plasmacytoma. This trial was registered at www.ClinicalTrials.gov as #NCT04447573.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"4543-4552"},"PeriodicalIF":7.1,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12452673/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144504859","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}

引用次数: 0

Bing-Neel syndrome: a case series of 46 patients from the United Kingdom. Bing-Neel综合征——来自英国的46例病例系列。

IF 7.1 1区医学

Blood advances Pub Date : 2025-09-23 DOI: 10.1182/bloodadvances.2025016360

Oliver Tomkins, Jahanzaib Khwaja, Shiwen Koay, Nicole Japzon, Chandrashekar Hoskote, Rajeev Gupta, Robert Baker, Jindriska Lindsay, Charalampia Kyriakou, Michael P Lunn, Shirley D'Sa

引用次数: 0

Platelet glycosylation in myelodysplastic syndromes correlates with disease severity. 骨髓增生异常综合征的血小板糖基化与疾病严重程度相关。

IF 7.1 1区医学

Blood advances Pub Date : 2025-09-23 DOI: 10.1182/bloodadvances.2025016349

Katelyn E Rosenbalm, Tin Long Wong, Donny Hoang, Valerie L Andersen, George Steinhardt, Shikan Zheng, Elizabeth A Griffiths, Tongjun Gu, Karin M Hoffmeister, Joseph T Y Lau, Michael J Nemeth

引用次数: 0

Weighing the evidence: apixaban for pediatric leukemia. 权衡证据：阿哌沙班治疗儿童白血病。

IF 7.1 1区医学

Blood advances Pub Date : 2025-09-23 DOI: 10.1182/bloodadvances.2025016948

Leslie Raffini, Hilary Whitworth

引用次数: 0

Luspatercept for Patients with Lower-Risk Myelodysplastic Syndromes/Neoplasms: A Systematic Review and Meta-Analysis. Luspatercept用于低风险骨髓增生异常综合征/肿瘤患者：系统回顾和荟萃分析。

IF 7.1 1区医学

Blood advances Pub Date : 2025-09-18 DOI: 10.1182/bloodadvances.2025017611

Abdulrahman Alhajahjeh, Naira Link Woite, Benjamin Rolles, Jessica M Stempel, Alain Mina, Lourdes M Mendez, Tariq Kewan, Nikolai A Podoltsev, Maximilian F Stahl, Alyssa A Grimshaw, Amer M Zeidan, Jan P Bewersdorf

{"title":"Luspatercept for Patients with Lower-Risk Myelodysplastic Syndromes/Neoplasms: A Systematic Review and Meta-Analysis.","authors":"Abdulrahman Alhajahjeh, Naira Link Woite, Benjamin Rolles, Jessica M Stempel, Alain Mina, Lourdes M Mendez, Tariq Kewan, Nikolai A Podoltsev, Maximilian F Stahl, Alyssa A Grimshaw, Amer M Zeidan, Jan P Bewersdorf","doi":"10.1182/bloodadvances.2025017611","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025017611","url":null,"abstract":"Luspatercept has emerged as a novel therapy for anemia in transfusion-dependent (TD) lower-risk myelodysplastic syndromes (LR-MDS). This systematic review and meta-analysis aims to evaluate the efficacy and safety of luspatercept in LR-MDS. We conducted a systematic review and meta-analysis to evaluate the efficacy and safety of luspatercept in TD LR-MDS. Six databases were searched through March 2025 to find relevant material. Studies were screened and extracted by two independent authors. A total of 20 studies encompassing 3,455 patients were included in the analysis. The pooled 8-week transfusion independence (TI) rate was 51.2% (95% CI: 39.9%-60.4%; I² = 94.9%), with higher rates observed among RS⁺ patients (57.8%; 95% CI: 47.4%-67.7%; I² = 86%) and those with low transfusion burden (LTB) (72.9%; 95% CI: 60.4%-82.6%; I² = 0%). The 12-week and 24-week TI rates were 57.0% (95% CI: 48.1%-65.5%; I² = 90%) and 35.8% (95% CI: 28.7%-43.6%; I² = 82.1%), respectively. Hematologic improvement-erythroid was achieved in 51.3% of patients (95% CI: 41.3%-61.2%; I² = 93%). The most frequent adverse events were peripheral edema (17.8%; 95% CI: 11.4%-26.8%), diarrhea (15.6%; 95% CI: 8.2%-27.7%), and fatigue (11.4%; 95% CI: 5.4%-22.6%). Serious adverse events occurred in 28.0% of patients (95% CI: 12.8%-50.7%; I² = 97.2%). Luspatercept is an effective and well-tolerated treatment for anemia in TD LR-MDS, especially in RS⁺ and LTB patients. Its favorable safety profile and higher TI rates, particularly in ESA-naïve populations supports its use in the frontline setting.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.1,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145085150","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}

引用次数: 0

Do not forget vaccinations in patients on Ig replacement. 不要忘记给Ig替代患者接种疫苗。

IF 7.1 1区医学

Blood advances Pub Date : 2025-09-18 DOI: 10.1182/bloodadvances.2025018127

Sigrun Einarsdottir, Silvia Escribano Serrat, Marina Gomez-Llobell, Malin L Hultcrantz, Mini Kamboj, Miguel-Angel Perales, Roni Shouval, Zainab Shahid, Per Ljungman

引用次数: 0

Statins Enhance the Efficacy of Pegylated Interferon-α2 in Philadelphia-Negative Chronic Myeloproliferative Neoplasms. 他汀类药物增强聚乙二醇化干扰素-α2治疗费城阴性慢性骨髓增生性肿瘤的疗效。

IF 7.1 1区医学

Blood advances Pub Date : 2025-09-18 DOI: 10.1182/bloodadvances.2025017016

Isabella Diana Davidsen, Morten Kranker Larsen, Lea Löffler, Vibe Skov, Lasse Kjær, Trine Alma Knudsen, Anders Lindholm Sørensen, Sarah Friis Christensen, Sabrina Cordua, Christina Schjellerup Eickhardt-Dalbøge, Marie Hvelplund Kristiansen, Christina Ellervik, Troels Wienecke, Hans Carl Hasselbalch

{"title":"Statins Enhance the Efficacy of Pegylated Interferon-α2 in Philadelphia-Negative Chronic Myeloproliferative Neoplasms.","authors":"Isabella Diana Davidsen, Morten Kranker Larsen, Lea Löffler, Vibe Skov, Lasse Kjær, Trine Alma Knudsen, Anders Lindholm Sørensen, Sarah Friis Christensen, Sabrina Cordua, Christina Schjellerup Eickhardt-Dalbøge, Marie Hvelplund Kristiansen, Christina Ellervik, Troels Wienecke, Hans Carl Hasselbalch","doi":"10.1182/bloodadvances.2025017016","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025017016","url":null,"abstract":"Chronic inflammation may be a key driving force in the development and progression of Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs). Statins, commonly used to lower cholesterol, also possess anti-proliferative, proapoptotic and anti-inflammatory properties, that may be beneficial in the treatment of MPN patients. This retrospective cohort study investigated whether statin use, in addition to standard cytoreductive therapy, shortens the time required to achieve hematological and molecular responses while allowing for lower cytoreductive drug dosages. A total of 129 patients were included, with 53 receiving statins from diagnosis. The study found that statin users achieved complete hematologic response (CHR) significantly faster than non-users (median time: 8 versus 18 months; HR 2.1, 95%CI 1.4-3.1, P = 0.0003). Among patients treated with pegylated interferon-alpha2 (IFN), the CHR rate was 97% in statin users versus 83% in non-users (HR 2.5, 95%CI 1.5-3.9, P = 0.0004), and a higher proportion of statin users sustained CHR throughout follow-up. Additionally, IFN-treated statin users received a significantly lower mean dose of IFN. A dose-response relationship was observed, with higher statin intensity associated with an increase of CHR. Furthermore, statin use was significantly associated with achieving a partial molecular response among IFN-treated patients (HR 2.6, 95%CI: 1.1-6.0, P = 0.029). No significant association was observed in hydroxyurea-treated patients. These findings suggest that statins may enhance the efficacy of IFN in MPN patients, while their benefit in hydroxyurea-treated patients remains unclear. Prospective studies are warranted to further explore the therapeutic potential of statins in MPNs.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.1,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145084577","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}

引用次数: 0

Development and Validation of a Gene Expression Signature to Predict Early Events in Patients with Follicular Lymphoma. 预测滤泡性淋巴瘤患者早期事件的基因表达标记的开发和验证。

IF 7.1 1区医学

Blood advances Pub Date : 2025-09-18 DOI: 10.1182/bloodadvances.2025016827

Colleen A Ramsower, George Wright, Hongli Li, James R Cerhan, Matthew J Maurer, Raphael Mwangi, Allison C Rosenthal, Anne J Novak, Brian K Link, Thomas E Witzig, Thomas M Habermann, Robert Kridel, Michael L LeBlanc, Mazyar Shadman, Sonali M Smith, Jonathan W Friedberg, David W Scott, Christian Steidl, Louis M Staudt, Lisa M Rimsza

{"title":"Development and Validation of a Gene Expression Signature to Predict Early Events in Patients with Follicular Lymphoma.","authors":"Colleen A Ramsower, George Wright, Hongli Li, James R Cerhan, Matthew J Maurer, Raphael Mwangi, Allison C Rosenthal, Anne J Novak, Brian K Link, Thomas E Witzig, Thomas M Habermann, Robert Kridel, Michael L LeBlanc, Mazyar Shadman, Sonali M Smith, Jonathan W Friedberg, David W Scott, Christian Steidl, Louis M Staudt, Lisa M Rimsza","doi":"10.1182/bloodadvances.2025016827","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025016827","url":null,"abstract":"Although follicular lymphoma (FL) typically follows an indolent course, patients with FL who experience early events such as transformation or progression have increased risk of death related to lymphoma. The FL24Cx is an algorithm based on a 45-target gene expression profiling (GEP) assay, which was developed and trained using 265 formalin-fixed, paraffin-embedded tissue samples on a reliable platform to predict, at the time of diagnosis, if a patient will experience an event within 24 months. The modeling also confirmed and relied upon previously reported synergy between immune response (IR) gene expression signatures IR1 and IR2. Once locked, the 5-factor logistic regression FL24Cx model was independently validated in a retrospectively assessed cohort of 232 patients from two immunochemotherapy-treated arms of SWOG Cancer Research Network S0016 phase III clinical trial, where it assigned 169 to the low-risk group with 29 events before 24 months (17.2%) and 63 to the high-risk group with 24 events before 24 months (38.1%). The relative risk of an event within 24 months after registration among patients who were classified into the high risk group relative to patients who were classified into the low risk group was 2.2 (95% CI: 1.41 to 3.51). An upfront GEP biomarker such as the FL24Cx, rigorously validated in a clinical laboratory, with a clinically-relevant turn-around time, could identify and steer enrollment of patients at high risk for early events in clinical trials, thus enabling timely interpretation of such trials and increasing the pace of innovation.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.1,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145085078","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}

引用次数: 0