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共有 52302 条符合本次的查询结果, 用时 1.5944075 秒

301. ADAR1 in lenalidomide resistance: still immunomodulation?

作者: Sarah Gooding.
来源: Blood. 2025年145卷11期1104-1106页

302. Low counts count after CAR-T for ALL.

作者: Regina M Myers.;Michael A Pulsipher.
来源: Blood. 2025年145卷11期1100-1102页

303. Azacitidine and venetoclax for HR-MDS: election results pending.

作者: Sangeetha Venugopal.;Mikkael A Sekeres.
来源: Blood. 2025年145卷11期1099-1100页

304. Why is InO's divine veil not protective against resistance?

作者: Philippe Rousselot.
来源: Blood. 2025年145卷11期1106-1107页

305. Acute myeloid leukemia with RUNX1::CBFA2T3 fusion.

作者: Wei J Wang.;Sanam Loghavi.
来源: Blood. 2025年145卷11期1226页

306. Heat-insoluble cryoglobulin in peripheral blood.

作者: Keyliz Peraza Cruces.;Katalin Marianna Kovacs.
来源: Blood. 2025年145卷11期1225页

307. Hu L, Shi Y, Hsu J, Gera J, Van Ness B, Lichtenstein A. Downstream effectors of oncogenic ras in multiple myeloma cells. Blood. 2003;101(8):3126-3135.

来源: Blood. 2025年145卷19期2232页

308. A controlled trial for preventing priapism in sickle cell anemia: hydroxyurea plus placebo vs hydroxyurea plus tadalafil.

作者: Ibrahim M Idris.;Aminu A Yusuf.;Ismail I Ismail.;Awwal M Borodo.;Mustapha S Hikima.;Shehu A Kana.;Tukur Aliyu.;Kabiru Musangedu.;Atiku Umar Jibrilla.;Sani A Aji.;Aisha Kuliya-Gwarzo.;Kabir Mohammad.;Jamil A Galadanci.;Rukayya Alkassim.;Mohammad A Suwaid.;Nafiu Hussaini.;Mark Rodeghier.;Arthur L Burnett.;Michael R DeBaun.
来源: Blood. 2025年145卷26期3101-3112页
Recurrent ischemic priapism is a common complication of sickle cell anemia (SCA) and is associated with devastating physical and psychosocial consequences. All previous trials for priapism prevention have failed to demonstrate clear efficacy. We conducted a randomized, controlled, double-blind phase 2 feasibility trial comparing fixed moderate-dose hydroxyurea plus placebo (usual-care arm) with fixed moderate-dose hydroxyurea plus tadalafil (experimental arm) in 64 males (aged 18-40 years) with at least 3 episodes of SCA-related priapism in the past 12 months. Priapism data were obtained via daily text messages to the participants. The trial's primary outcome measures were 100% recruitment, 98.4% retention, and 93.5% adherence rates. Over a median of 10 months (interquartile range, 3-12), 2.5 and 3.02 priapism events per participant-month were recorded in the usual-care and the experimental arms, with an incidence rate ratio of 0.8 (95% confidence interval [CI], 0.3-1.9; P = .654). The rates of serious adverse events (P = .999) and hospitalization (P = .289) were similar in the 2 arms. Sperm concentration, motility, and normal morphology significantly decreased on hydroxyurea therapy but recovered to prehydroxyurea levels 3 months after therapy cessation. Post hoc, single-arm, pre-post analysis showed a 58.3% priapism incidence rate reduction in the usual-care arm (5.9-2.5 events per month; difference, 3.4; 95% CI, 1.1-5.8; P = .005) and a 66.3% priapism reduction in the experimental arm (8.9-3.02 events per month; difference, 5.9; 95% CI, 3.4-8.5; P < .001) compared with the prerandomization rates. A randomized controlled trial for priapism prevention is feasible in men with SCA. This trial was registered at www.clinicaltrials.gov as #NCT05142254.

309. Efficacy of combined CD38 and PD-1 inhibition with isatuximab and cemiplimab for relapsed/refractory NK/T-cell lymphoma.

作者: Seok Jin Kim.;Jing Quan Lim.;Sang Eun Yoon.;Deok-Hwan Yang.;Ji Hyun Lee.;Sung Yong Oh.;Yoon Seok Choi.;Seong Hyun Jeong.;Min Kyoung Kim.;Sung Nam Lim.;Junhun Cho.;Bon Park.;Kyung Ju Ryu.;Seunghyun Choi.;Yoon Park.;Kerry May Huifen Lim.;Nur Ayuni Binte Muhammad Taib.;Choon Kiat Ong.;Soon Thye Lim.;Won Seog Kim.
来源: Blood. 2025年146卷2期155-166页
This study aimed to assess the efficacy and safety of combining cemiplimab, an anti-programmed cell death protein 1 (PD-1) antibody, with isatuximab, an anti-CD38 antibody, in relapsed or refractory extranodal natural killer/T-cell lymphoma (R/R ENKTL). The hypothesis was that CD38 blockade could enhance the antitumor activity of PD-1 inhibitors. Eligible patients received cemiplimab (250 mg on days 1 and 15) and isatuximab (10 mg/kg on days 2 and 16) IV every 4 weeks for 6 cycles. Responders then received cemiplimab (350 mg) and isatuximab (10 mg/kg) every 3 weeks for up to 24 months. The primary end point was the complete response (CR) rate based on the best response. Of 37 patients enrolled, the CR rate was 51% (19/37), exceeding the primary end point of 40%, and the objective response rate was 65% (24/37). After a median follow-up of 30.2 months (95% confidence interval [CI], 25.6-34.8 months), the median progression-free survival was 9.5 months (95% CI, 1.4-17.6 months), whereas the median overall survival had not yet been reached. Patients achieving CR received a median of 28 cycles (range, 4-33 cycles), and the median duration of response for responders (n = 24) was 29.4 months (95% CI, 15.4-43.4 months). Structural variations disrupting the 3'-untranslated region of PD-L1 and high programmed death ligand 1 (PD-L1) expression were observed in responders. Most adverse events were mild (grade 1-2), with grade ≥3 events (32%) and no treatment-related deaths. The combination of isatuximab and cemiplimab demonstrated sustained antitumor activity and a manageable safety profile in R/R ENKTL. This phase 2 trial is registered at www.clinicaltrials.gov as number NCT04763616.

310. Large-scale dependency and drug screens to characterize the therapeutic vulnerabilities of multiple myeloma with 1q.

作者: Romanos Sklavenitis-Pistofidis.;Elizabeth D Lightbody.;Mairead Reidy.;Junko Tsuji.;Jean-Baptiste Alberge.;Michelle P Aranha.;Daniel Heilpern-Mallory.;Harvey G Roweth.;Daisy Huynh.;Stephen J F Chong.;Anna Y Chung.;Jeremy Zhang.;Liam Hackett.;Nicholas J Haradhvala.;Ting Wu.;Nang K Su.;Brianna Berrios.;Saveliy Belkin.;Ankit K Dutta.;Ryan A Knudson.;Carolyn Brandt.;Patricia T Greipp.;Matthew S Davids.;Maria Papaioannou.;Gad Getz.;Irene M Ghobrial.;Salomon Manier.
来源: Blood. 2025年146卷1期89-103页
The development of targeted therapy for patients with multiple myeloma (MM) is hampered by the low frequency of actionable genetic abnormalities. Gain or amplification of chromosome 1q (1q+) is the most frequent arm-level copy number gain in patients with MM and is associated with higher risk of progression and death despite recent therapeutic advances. Thus, developing targeted therapy for patients with MM with 1q+ stands to benefit a large portion of patients in need of more effective management. Here, we used large-scale dependency screens and drug screens to systematically characterize the therapeutic vulnerabilities of MM with 1q+ and displayed increased sensitivity to myeloid cell leukemia-1 (MCL1) and phosphatidyl inositol 3-kinase (PI3K) inhibitors. Using single-cell RNA sequencing, we compared subclones with and without 1q+ within the same patient tumors and demonstrated that 1q+ is associated with higher levels of MCL1 and the PI3K pathway. Furthermore, by isolating isogenic clones with different copy number profiles for part of the chromosome 1q arm, we observed increased sensitivity to MCL1 and PI3K inhibitors with arm-level gain. Lastly, we demonstrated synergy between MCL1 and PI3K inhibitors and dissected their mechanism of action in MM with 1q+, uncovering a cytostatic effect. In conclusion, this study highlights that MM with 1q+ may present enhanced sensitivity to MCL1 and PI3K inhibitors, enabling their use at lower doses without sacrificing efficacy, and may thus accelerate the development of targeted therapy for patients with MM and 1q+.

311. Social determinants of health and access to allogeneic hematopoietic cell transplantation for acute myeloid leukemia.

作者: Natalie Wuliji.;Salene M W Jones.;Ted Gooley.;Aaron T Gerds.;Bruno C Medeiros.;Paul J Shami.;John Galvin.;Kehinde Adekola.;Selina Luger.;Maria R Baer.;David Rizzieri.;Tanya M Wildes.;Eunice S Wang.;Mikkael A Sekeres.;Sudipto Mukherjee.;Julie Smith.;Mitchell Garrison.;Kiarash Kojouri.;Jacob Appelbaum.;Mary-Elizabeth Percival.;Brenda M Sandmaier.;Stephanie Lee.;Frederick R Appelbaum.;Rayne Rouce.;Mohamed L Sorror.
来源: Blood. 2025年145卷25期3041-3051页
Whether allogeneic hematopoietic cell transplant (allo-HCT) to treat acute myeloid leukemia (AML) is equitably accessible regardless of social determinants of health (SDOH) remains unknown. We examined associations of SDOH with access to allo-HCT and other outcomes. Patients presenting for treatment (n = 692) at 13 AML treatment centers were prospectively recruited to a registered clinical trial (number NCT01929408). Various patient-, AML-, and SDOH-specific variables were collected. Outcomes included mortality without allo-HCT, receipt of allo-HCT, and mortality after allo-HCT. Individual multivariable models (Fine-Gray for the first 2 outcomes, Cox regression for the third) were fit for each SDOH variable, adjusting for relevant patient- and AML-specific variables. Allo-HCT was used to treat 46% of patients. A 10% increase in the proportion with less than a high school education, in households receiving Supplemental Nutrition Assistance Program, receiving Supplemental Security Income, or in poverty led to modeled adjusted hazard ratios (aHRs) of 1.21 (0.99-1.46), 1.13 (0.97-1.31), 1.41 (1.01-1.97), and 1.16 (0.96-1.39) for death without allo-HCT. The aHRs were 0.67 (0.55-0.83), 0.88 (0.76-1.01), 0.71 (0.48-1.05), and 0.91 (0.75-1.09) for lessened receipt of allo-HCT. Among those who received allo-HCT, aHRs for mortality were 1.18 (0.87-1.60), 1.13 (0.92-1.38), 1.21 (0.81-1.82), and 1.04 (0.79-1.36). Results highlight increased mortality without allo-HCT and decreased access to allo-HCT, but lesser magnitude of increased mortality after allo-HCT, among patients from lower resourced areas due to limited education and/or increased poverty. Targeted interventions and policy changes are needed to ensure that marginalized patient populations have equitable chances for AML cure compared with others.

312. Clonal hematopoiesis landscape in frequent blood donors.

作者: Darja Karpova.;Hector Huerga Encabo.;Elisa Donato.;Silvia Calderazzo.;Michael Scherer.;Miriam Llorian-Sopena.;Aino-Maija Leppä.;Roberto Würth.;Patrick Stelmach.;Despoina Papazoglou.;Alessandra Ferrelli.;Steven Ngo.;Iuliia Kotova.;Sabine Harenkamp.;Kai Zimmer.;Dominik Wolf.;Jasper Panten.;John Reed.;Adriana Przybylla.;Torsten Tonn.;Annette Kopp-Schneider.;Lars Velten.;John F DiPersio.;Terrence N Wong.;Dominique Bonnet.;Halvard Bonig.;Andreas Trumpp.
来源: Blood. 2025年145卷21期2411-2423页
Donor blood saves lives, yet the potential impact of recurrent large-volume phlebotomy on donor health and hematopoietic stem cells (HSCs) remains largely unexplored. In our study, we conducted a comprehensive screening of 217 older male volunteer donors with a history of extensive blood donation (>100 lifetime donations) to investigate the phenomenon of clonal hematopoiesis (CH). No significant difference in the overall incidence of CH was found in frequent donors (FDs) compared with sporadic donors (<10 lifetime donations; 212 donors). However, upon deeper analysis of mutations in DNMT3A, the most commonly affected gene in CH, we observed distinct mutational patterns between the FD and age/sex-matched control donor cohorts. Functional analysis of FD-enriched DNMT3A variants examined in CRISPR-edited human HSCs demonstrated their competitive outgrowth potential upon stimulation with erythropoietin (EPO), a hormone that increases in response to blood loss. In contrast, clones harboring leukemogenic DNMT3A R882 mutations increase upon stimulation with interferon gamma. Through concurrent mutational and immunophenotypic profiling of primary samples at single-cell resolution, a myeloid bias of premalignant R882 mutant HSCs was found, whereas no significant lineage bias was observed in HSCs harboring EPO-responsive DNMT3A variants. The latter exhibited preferential erythroid differentiation when persistent erythropoietic stress was applied to CRISPR-edited human HSC xenografts. Our data demonstrate a nuanced, ongoing Darwinian evolution at the somatic stem cell level, with EPO identified as a novel environmental factor that favors HSCs carrying certain DNMT3A mutations.

313. Long-term outcomes with single-agent BRAF inhibitor therapy in Erdheim-Chester disease.

作者: Gaurav Goyal.;Anne S Reiner.;Dana Bossert.;Allison M Sigler.;Mario E Lacouture.;Veronica Rotemberg.;Jasmine H Francis.;Ronald S Go.;Raajit K Rampal.;Eli L Diamond.
来源: Blood. 2025年145卷18期2100-2103页
Among 64 patients with Erdheim-Chester disease treated with a BRAF inhibitor (median follow-up 4 years), we found high response rates (85%) but frequent discontinuations (61%), primarily because of adverse events. Additionally, patients experienced poor health-related quality of life and high symptom burden.

314. Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study.

作者: Guy Young.;Kaan Kavakli.;Robert Klamroth.;Tadashi Matsushita.;Flora Peyvandi.;Steven W Pipe.;Savita Rangarajan.;Ming-Ching Shen.;Alok Srivastava.;Jing Sun.;Huyen Tran.;Chur-Woo You.;Bülent Zülfikar.;Laurel A Menapace.;Chuanwu Zhang.;Yuqian Shen.;Marja Puurunen.;Marek Demissie.;Gili Kenet.
来源: Blood. 2025年145卷25期2966-2977页
Fitusiran, a subcutaneous investigational small interfering RNA therapeutic, lowers antithrombin (AT) to increase thrombin generation and rebalance hemostasis in people with hemophilia. This phase 3 open-label extension study (ATLAS-OLE) evaluated safety and efficacy of an AT-based dose regimen (AT-DR) in males aged ≥12 years with severe hemophilia A/B, with/without inhibitors. The original dose regimen (ODR) of 80 mg monthly was optimized to AT-DR targeting AT activity levels 15% to 35% to mitigate thrombotic risk (starting dose of 50 mg once every 2 months, individually adjusted to 20 mg once every 2 months, or 20/50/80 mg monthly as needed). Primary and secondary end points were safety and efficacy, respectively. Integrated safety analyses assessed safety of AT-DR and ODR across all fitusiran studies and integrated efficacy analyses compared efficacy of AT-DR in ATLAS-OLE with phase 3 parent study control groups. At interim data cutoff, 213 participants were enrolled on AT-DR (78% on regimens of once every 2 months). Integrated safety analyses of participants receiving AT-DR (n = 286) demonstrated that AT-DR was well tolerated. In ATLAS-OLE, median observed annualized bleeding rate (ABR) with AT-DR was 3.7 (interquartile range, 0.0-7.5). Integrated efficacy analyses demonstrated superiority of AT-DR over on-demand clotting factor concentrates (CFCs; 71% mean ABR reduction; P < .0001), and on-demand bypassing agents (BPAs; 73% mean ABR reduction; P = .0006); improvement over BPA prophylaxis (70% mean ABR reduction); and ABR comparable with that observed with CFC prophylaxis. Fitusiran AT-DR was well tolerated and maintained bleed protection with as few as 6 injections per year. This trial was registered at www.ClinicalTrials.gov as #NCT03754790.

315. Therapy for MYD88 L265P DLBCL.

作者: Joost S P Vermaat.;Ruben A L de Groen.
来源: Blood. 2025年145卷10期998-1000页

316. Cullin' the herd: Cul5 limits megakaryocyte-biased HSCs.

作者: Eric M Pietras.
来源: Blood. 2025年145卷10期996-998页

317. Air pollution and venous thromboembolism.

作者: Ingrid Pabinger.;Cihan Ay.
来源: Blood. 2025年145卷10期1003-1004页

318. Over 4 decades, French children teach about LCH.

作者: Barbara A Degar.;Barrett J Rollins.
来源: Blood. 2025年145卷10期1000-1001页

319. CD7 CAR-T: a bridge to transplant in AML.

作者: Maksim Mamonkin.
来源: Blood. 2025年145卷10期995-996页

320. AMBRA1 performs a balancing act in β-thalassemia.

作者: Christophe Lechauve.;Mitchell J Weiss.
来源: Blood. 2025年145卷10期1001-1003页
共有 52302 条符合本次的查询结果, 用时 1.5944075 秒