Blood advances最新文献_第3页

Too short or unnecessarily long: walking the fine line. 过短或过长：把握分寸。

IF 7.4 1区医学

Blood advances Pub Date : 2024-10-22 DOI: 10.1182/bloodadvances.2024013812

Chiranjeevi Sainatham, Tania Jain

引用次数: 0

Inhibition of MICA and MICB shedding enhances memory-like NK-cell-mediated cytotoxicity against multiple myeloma. 抑制 MICA 和 MICB 的脱落可增强记忆型 NK 细胞介导的细胞毒性，从而抗击多发性骨髓瘤。

IF 7.4 1区医学

Blood advances Pub Date : 2024-10-22 DOI: 10.1182/bloodadvances.2023010869

Sabrin Tahri, Sara Piccinelli, Nang Kham Su, Luisa Lampe, Han Dong, Juliana Vergara Cadavid, Rebecca Boiarsky, Natalie Papazian, Elizabeth D Lightbody, Amanda Cao, Jean-Baptiste Alberge, Lucas Ferrari de Andrade, Mahshid Rahmat, Yujia Shen, Laura Blanco Fernández, Aintzane Zabaleta, Andreas Günther, Gad Getz, Pieter Sonneveld, Tom Cupedo, Kai W Wucherpfennig, Irene M Ghobrial, Rizwan Romee

引用次数: 0

Haploidentical vs HLA-matched sibling donor HCT with PTCy prophylaxis: HLA factors and donor age considerations. 单倍体与 HLA 匹配的同胞捐献者造血干细胞与 PTCy 预防：HLA因素和供体年龄考虑因素。

IF 7.4 1区医学

Blood advances Pub Date : 2024-10-22 DOI: 10.1182/bloodadvances.2024013853

Rohtesh S Mehta, Jeremy Ramdial, Partow Kebriaei, Richard E Champlin, Uday Popat, Katayoun Rezvani, Elizabeth J Shpall

{"title":"Haploidentical vs HLA-matched sibling donor HCT with PTCy prophylaxis: HLA factors and donor age considerations.","authors":"Rohtesh S Mehta, Jeremy Ramdial, Partow Kebriaei, Richard E Champlin, Uday Popat, Katayoun Rezvani, Elizabeth J Shpall","doi":"10.1182/bloodadvances.2024013853","DOIUrl":"10.1182/bloodadvances.2024013853","url":null,"abstract":"Abstract: HLA-matched sibling donors (MSDs) are preferred for hematopoietic cell transplantation (HCT). However, the use of alternative donors, especially haploidentical, is increasing, as is our understanding of the impact of HLA factors such as B-leader and DRB1-matching on its outcomes. Yet, data comparing these donor types, particularly considering these HLA factors, is lacking. Herein, we compared haploidentical-HCT (n = 1052) with MSD-HCT (n = 400), both with posttransplant cyclophosphamide (PTCy)-based graft-versus-host disease prophylaxis. In multivariate analysis, haploidentical group had similar overall survival (OS; hazard ratio (HR), 0.94; 95% confidence interval [CI], 0.78-1.14; P = .54), nonrelapse mortality (HR, 0.98; 95% CI, 0.72-1.32; P = .87), and relapse (HR, 0.87; 95% CI, 0.70-1.08; P = .20) as the MSD group. Younger donor age was a significant predictor of improved OS. Next, we directly compared the outcomes of \"younger\" haploidentical (donor age <35 years, n = 347) vs an \"older\" MSD (donor age ≥50 years, n = 143) in older recipients (patient age ≥50 years). Patients with younger haploidentical B-leader-matched donors had significantly superior OS (HR, 0.65; 95% CI, 0.48-0.90; P = .009) than the older MSD group. Additionally, patients with younger DRB1-mismatched haploidentical donors (HR, 0.63; 95% CI, 0.46-0.87; P = .004) had significantly lower risk of relapse than older MSDs. Our study suggests that haploidentical-HCT may offer comparable outcomes to MSD-PTCy HCT. Moreover, among older patients, a younger haploidentical B-leader-matched donor might be preferable to an older MSD. These findings need validation in larger data sets.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11497453/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142016423","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

Elritercept, a Modified Activin Receptor IIA Ligand Trap, increased Erythropoiesis and Thrombopoiesis in a Phase 1 Trial. Elritercept是一种改良的Activin受体IIA配体捕获剂，在一期试验中能增加红细胞生成和血栓形成。

IF 7.4 1区医学

Blood advances Pub Date : 2024-10-22 DOI: 10.1182/bloodadvances.2024014172

Jennifer L Lachey, Christopher Rovaldi, Suresh Bobba, Jared Tur, Harveen Dhillon Natarajan, Ben Snyder, Jasbir Seehra

{"title":"Elritercept, a Modified Activin Receptor IIA Ligand Trap, increased Erythropoiesis and Thrombopoiesis in a Phase 1 Trial.","authors":"Jennifer L Lachey, Christopher Rovaldi, Suresh Bobba, Jared Tur, Harveen Dhillon Natarajan, Ben Snyder, Jasbir Seehra","doi":"10.1182/bloodadvances.2024014172","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024014172","url":null,"abstract":"The TGF-β superfamily plays a crucial role in regulating biological processes of virtually every tissue and system in the body, including hemostasis and hematopoiesis. Elritercept (KER-050) is an investigational, modified activin receptor type IIA ligand trap designed to bind and inhibit activin A and other select TGF-β superfamily ligands including activin B and growth differentiation factor (GDF)-8 and GDF-11. The objectives of this Phase 1 randomized, placebo-controlled study of elritercept were to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamic markers of activin inhibition and hematopoiesis in healthy, post-menopausal women (N=48). This study was comprised of 2 parts: single ascending doses ranging from 0.05 to 4.5 mg/kg, and multiple (up to 2 doses) ascending doses of 0.75 mg/kg administered subcutaneously (SC) every 4 weeks. Elritercept was generally well-tolerated at all dose levels, with no dose limiting toxicities observed. There were no severe or serious adverse events nor clinically significant changes in safety laboratory measures. Serum concentrations increased in dose-proportional manner following single SC doses, with peak concentrations achieved in 4.5 to 6 days and a mean elimination half-life of 12 days. These parameters were comparable following multiple doses. Elritercept elicited rapid, sustained, and dose-dependent increases in reticulocytes, red blood cells, hemoglobin, and platelets without eliciting detrimental changes in white blood cells such as neutrophils and lymphocytes. The time course and duration of changes in these cell populations supported a differentiated pharmacologic profile that is consistent with stimulation of both early- and late-stage hematologic pathways. Australian New Zealand Clinical Trial Registry (ACTRN12619000318189).","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142495374","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

Siltuximab for Chimeric Antigen Receptor T-Cell Therapy Related CRS and ICANS - A Multicenter Retrospective Analysis. 用于嵌合抗原受体 T 细胞疗法相关 CRS 和 ICANS 的 Siltuximab - 一项多中心回顾性分析。

IF 7.4 1区医学

Blood advances Pub Date : 2024-10-22 DOI: 10.1182/bloodadvances.2024013688

Amneet Bajwa, Qiuhong Zhao, Marcus J Geer, Chenyu Lin, James Westholder, Joeseph Maakaron, Monalisa Ghosh, David G Frame, Ahmed Galal, Justin Tossey, Nausheen Ahmed, Evandro D Bezerra, Nathan Denlinger, Marcos J de Lima, Narendranath Epperla, Paolo F Caimi, Timothy J Voorhees

{"title":"Siltuximab for Chimeric Antigen Receptor T-Cell Therapy Related CRS and ICANS - A Multicenter Retrospective Analysis.","authors":"Amneet Bajwa, Qiuhong Zhao, Marcus J Geer, Chenyu Lin, James Westholder, Joeseph Maakaron, Monalisa Ghosh, David G Frame, Ahmed Galal, Justin Tossey, Nausheen Ahmed, Evandro D Bezerra, Nathan Denlinger, Marcos J de Lima, Narendranath Epperla, Paolo F Caimi, Timothy J Voorhees","doi":"10.1182/bloodadvances.2024013688","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024013688","url":null,"abstract":"Chimeric Antigen Receptor T-Cell therapies (CAR-T) are effective in many hematologic malignancies; however, adverse events including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) can affect a significant number of patients. Those who develop refractory CRS or ICANS have few treatment options. Siltuximab, a monoclonal antibody binding circulating IL-6, has been proposed to have clinical activity in both CRS and ICANS. We conducted a multi-center retrospective analysis of siltuximab treatment for CRS and ICANS following CAR-T cell therapy in a real-world cohort from six academic centers. Fifty-four patients were evaluated. Sixteen patients had CRS previously treated with tocilizumab and 17 patients had ICANS previously treated with steroids. Of the all the patients with CRS at the time of siltuximab, 75% had improvement in CRS grade. Of the all the patients with ICANS at the time of siltuximab, 60% had improvement in ICANS grade. To our knowledge, this is the largest cohort of patients treated with siltuximab for CRS and/or ICANS following CAR-T. Siltuximab appeared to be effective for both CRS and ICANS, including previously treated toxicities. These data support the use of siltuximab in CRS and ICANS as well as provide rationale for future prospective studies.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142495381","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

CARs to the rescue: rare aggressive lymphoma gets a power up. CARs 救援：罕见侵袭性淋巴瘤重获新生

IF 7.4 1区医学

Blood advances Pub Date : 2024-10-22 DOI: 10.1182/bloodadvances.2024014118

Manali Kamdar

引用次数: 0

Tyrosine kinase inhibitor for CML: all the same? 治疗 CML 的酪氨酸激酶抑制剂：都一样吗？

IF 7.4 1区医学

Blood advances Pub Date : 2024-10-22 DOI: 10.1182/bloodadvances.2024014060

Lukas Veltmaat, Jorge Cortes

引用次数: 0

What makes a good Samaritan: age, HLA matching, or both? 怎样才能成为一个好撒玛利亚人：年龄、HLA 匹配度，还是两者兼而有之？

IF 7.4 1区医学

Blood advances Pub Date : 2024-10-22 DOI: 10.1182/bloodadvances.2024014188

Pietro Crivello

引用次数: 0

Impact of an individualized pain plan to treat sickle cell disease vaso-occlusive episodes in the emergency department. 在急诊科治疗镰状细胞病血管闭塞性发作的个性化疼痛计划的影响。

IF 7.4 1区医学

Blood advances Pub Date : 2024-10-22 DOI: 10.1182/bloodadvances.2023012439

Lauren Siewny, Allison King, Cathy L Melvin, Christopher R Carpenter, Jane S Hankins, Joseph S Colla, Liliana Preiss, Lingzi Luo, Lisa Cox, Marsha Treadwell, Natalia Davila, Rita V Masese, Sarah McCuskee, S Siera Gollan, Paula Tanabe

{"title":"Impact of an individualized pain plan to treat sickle cell disease vaso-occlusive episodes in the emergency department.","authors":"Lauren Siewny, Allison King, Cathy L Melvin, Christopher R Carpenter, Jane S Hankins, Joseph S Colla, Liliana Preiss, Lingzi Luo, Lisa Cox, Marsha Treadwell, Natalia Davila, Rita V Masese, Sarah McCuskee, S Siera Gollan, Paula Tanabe","doi":"10.1182/bloodadvances.2023012439","DOIUrl":"10.1182/bloodadvances.2023012439","url":null,"abstract":"Abstract: To address acute vaso-occlusive episodes (VOEs), the leading cause of emergency department (ED) visits among individuals with sickle cell disease (SCD), we conducted the clinical study, ALIGN (An Individualized Pain Plan with Patient and Provider Access for Emergency Department care of SCD), across 8 sites. We hypothesized an improvement of 0.5 standard deviations in perceived quality of ED pain treatment of a VOE after implementing individualized pain plans (IPPs) accessible to both patients and providers. Patients with SCD were aged 18 to 45 years, owned a cell phone, and had an ED VOE visit within 90 days prior. Patients completed the perceived quality of care surveys at baseline and within 96 hours after an ED VOE visit. Providers completed surveys regarding comfort managing VOEs at baseline and after managing an enrolled patient. Most of the 153 patients were African American (95.4%), female (64.7%), and had Hb SS/Sβ0 genotype (71.9%). The perceived quality of ED pain treatment was high at both baseline and after implementation of IPPs; our primary outcome hypothesis was not met, because no statistically significant change in the patient-perceived quality of ED treatment occurred. A total of 135 providers completed baseline and follow-up surveys. On a scale of 1 to 7, with 7 being extremely comfortable managing VOEs, 60.5% reported a score ≥6 after IPP implementation vs 57.8% at baseline. Almost all (97.6%) ordered the recommended medication, and 94.7% intended to use IPPs. In this implementation protocol, all sites successfully implemented IPPs. Patients and ED providers both endorsed the use of IPPs. This trial was registered at www.ClinicalTrials.gov as # NCT04584528.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141178956","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

Nilotinib vs dasatinib in achieving MR4.5 for de novo chronic myeloid leukemia: the randomized JALSG CML212 study. 尼罗替尼与达沙替尼治疗新发慢性粒细胞白血病达到 MR4.5：JALSG CML212 随机研究。

IF 7.4 1区医学

Blood advances Pub Date : 2024-10-22 DOI: 10.1182/bloodadvances.2024012655

Itaru Matsumura, Shigeki Ohtake, Yoshiko Atsuta, Mio Kurata, Yosuke Minami, Naoto Takahashi, Chiaki Nakaseko, Noriyoshi Iriyama, Katsumichi Fujimaki, Kazuhiko Kakihana, Yoji Ogasawara, Takaaki Ono, Masaya Okada, Tetsuzo Tauchi, Toshihiro Miyamoto, Kazunori Ohnishi, Emiko Sakaida, Shin Fujisawa, Yukio Kobayashi, Norio Asou, Tomoki Naoe, Hitoshi Kiyoi, Yasushi Miyazaki

{"title":"Nilotinib vs dasatinib in achieving MR4.5 for de novo chronic myeloid leukemia: the randomized JALSG CML212 study.","authors":"Itaru Matsumura, Shigeki Ohtake, Yoshiko Atsuta, Mio Kurata, Yosuke Minami, Naoto Takahashi, Chiaki Nakaseko, Noriyoshi Iriyama, Katsumichi Fujimaki, Kazuhiko Kakihana, Yoji Ogasawara, Takaaki Ono, Masaya Okada, Tetsuzo Tauchi, Toshihiro Miyamoto, Kazunori Ohnishi, Emiko Sakaida, Shin Fujisawa, Yukio Kobayashi, Norio Asou, Tomoki Naoe, Hitoshi Kiyoi, Yasushi Miyazaki","doi":"10.1182/bloodadvances.2024012655","DOIUrl":"10.1182/bloodadvances.2024012655","url":null,"abstract":"Abstract: Deep molecular response (DMR) is a prerequite for treatment-free remission (TFR) in chronic myeloid leukemia in chronic phase (CML-CP). The JALSG (Japan Adult Leukemia Study Group) conducted a prospective randomized phase 3 CML212 study for de novo CML-CP to compare the cumulative achievement of molecular response 4.5 (MR4.5; international scale BCR::ABL1 ≤0.0032%) by 18 months between nilotinib and dasatinib treatment as a primary end point. A total of 454 patients were randomly assigned to the 300 mg nilotinib twice daily arm or to the 100 mg dasatinib daily arm (both n = 227). BCR::ABL1 messenger RNA levels were monitored every 3 months. Study treatment was stopped if the patients were judged as failure according to the European LekemiaNet 2009 criteria or showed intolerance. The cumulative achievement rates of MR4.5 by 18 months were 32.6% (95% confidence interval [CI], 26.5-39.1) in the nilotinib arm and 30.8% (95% CI, 24.9-37.3) in the dasatinib arm with no significant difference (P = .66). The cumulative achievement rates of early molecular response, complete cytogenetic response, and major molecular response by 12, 18, 24, and 36 months were almost the same between the 2 arms. There was no significant difference in progression-free survival (PFS) or overall survival (OS) between the 2 arms by log-rank tests (PFS, P = .58; OS, P = .64). These results suggest that nilotinib and dasatinib would be equally effective for patients with de novo CML-CP. This trial was registered in the University Hospital Medical Information Network Clinical Trials Registry as #UMIN000007909.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11493191/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141537632","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