Blood advances最新文献

{"title":"Molecular profiling of primary renal Marginal Zone Lymphoma reveals overlapping features with extranodal Marginal Zone Lymphoma.","authors":"Axel Künstner, Vera von Kopylow, Philipp Lohneis, Matthias Kümmel, Hanno M Witte, Veronica Bernard, Stephanie Stölting, Kathrin Kusch, Sivahari Prasad Gorantla, Manuela Krokowski, Nikolas von Bubnoff, Hartmut Merz, Hauke Busch, Alfred C Feller, Niklas Gebauer","doi":"10.1182/bloodadvances.2025016263","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025016263","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143965867","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":"Clec12a is required for the pathogenesis of NUP98::NSD1 AML.","authors":"Sagarajit Mohanty, Fiorella Charles Cano, Razif Gabdoulline, Courteney K Lai, Basem Othman, Harish Sudarsanam, Thomas Eder, Florian Grebien, Daniel B Lipka, Reinhard Henschler, Michael Heuser","doi":"10.1182/bloodadvances.2024015739","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015739","url":null,"abstract":"<p><p>NUP98::NSD1 is one of the most recurring nucleoporin 98 (NUP98) fusions in acute myeloid leukemia (AML). NSD1-driven AML is associated with adverse outcomes and poor response to conventional treatments. However, limited studies have been done to identify new potential targets to develop better treatment approaches. The C-type lectin domain family 12, member A (CLEC12A) is a cell surface receptor that is differentially expressed in leukemic stem cells (LSCs) compared to healthy hematopoietic stem cells (HSCs). We demonstrated a strong overexpression of CLEC12A in both NUP98::NSD1 patients and murine AML cells transformed with NUP98::NSD1. To understand the role of Clec12a in NUP98::NSD1 AML, we depleted Clec12a expression in NUP98::NSD1+NRASG12D immortalized cells using the CRISPR/Cas9 approach. NUP98::NSD1+NRASG12D/Clec12a knockout cells showed higher levels of apoptosis and lower colony numbers in vitro compared to NUP98::NSD1+NRASG12D/Clec12a wildtype cells. Importantly, the deletion of Clec12a significantly reduced leukemic engraftment and prolonged survival of the NUP98::NSD1+NRASG12D murine model. Our data suggest to further explore CLEC12A as a potential target for the treatment of NUP98::NSD1 AML.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143965955","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":"Dysregulated immune proteins in plasma in the UK Biobank predict Multiple Myeloma 12 years before clinical diagnosis.","authors":"Joshua Fieggen, Anshul Thakur, Christopher C Butler, Karthik Ramasamy, Anjan Thakurta, David A Clifton, Lei Clifton","doi":"10.1182/bloodadvances.2025016120","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025016120","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143987593","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":"Asciminib remained superior vs bosutinib in late-line CML-CP after nearly 4 years of follow-up in ASCEMBL.","authors":"Michael J Mauro, Yosuke Minami, Andreas Hochhaus, Elza Lomaia, Sergey Voloshin, Anna Turkina, Dong-Wook Kim, Jane F Apperley, Jorge Cortes, Andre Abdo, Laura Maria Fogliato, Dennis Dong Hwan Kim, Philipp le Coutre, Susanne Saussele, Mario Annunziata, Timothy P Hughes, Naeem Chaudhri, Lynette Chee, Valentin Garcia-Gutierrez, Koji Sasaki, Carla Boquimpani, Shruti Kapoor, Noemi Espurz-Abad, Vishal Dhamal, Delphine Rea","doi":"10.1182/bloodadvances.2025016042","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025016042","url":null,"abstract":"<p><p>The efficacy of and disease control afforded by tyrosine kinase inhibitors (TKIs) in chronic myeloid leukemia in chronic phase (CML-CP) has led to increased longevity and thus continued pursuit of alternative therapies that are efficacious and maximize tolerability. The 24- and 96-week analyses from ASCEMBL demonstrated superior efficacy, safety, and tolerability of asciminib compared with bosutinib in later-line therapy, meeting primary and key secondary objectives, respectively. With nearly 4 years of follow-up, data from ASCEMBL continued to demonstrate superior efficacy, better safety, and greater tolerability of asciminib compared with bosutinib. At week 156, the major molecular response (MMR) rates remained higher with asciminib (33.8%) than bosutinib (10.5%); the difference in MMR rates between arms, after adjusting for baseline major cytogenetic response, was 23.2% (95% CI, 13.14%-33.18%; 2-sided P<0.001). Asciminib continued to cause fewer grade ≥3 adverse events (AEs) (59.6% vs 68.4%) and AEs leading to treatment discontinuation (8.3% vs 27.6%) than bosutinib. This updated analysis also includes patients who switched to asciminib due to lack of efficacy with bosutinib. Two of 25 patients who switched achieved MMR by the end of study, suggesting that earlier incorporation of asciminib, before other TKIs, may improve responses, albeit modestly. These long-term results further solidify asciminib as the therapy of choice for patients with CML-CP previously treated with ≥2 prior TKIs. This trial was registered at ClinicalTrials.gov as NCT03106779.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144062315","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":"ADORE: an open platform study of ruxolitinib in combination with other novel therapies in patients with myelofibrosis.","authors":"David M Ross, Florian H Heidel, Andrew C Perkins, Andreas Reiter, Carl Claudius Crodel, Caroline Hasselbalch Riley, María Teresa Gómez Casares, Istvan Takacs, Heiko Becker, Thomas Lehmann, Olga Yu Vinogradova, Kate Burbury, Alessandro M Vannucchi, Vikas Gupta, Marielle J Wondergem, Jean-Jacques Kiladjian, Grace Cleary, Angela Zhang, Jagannath Kota, Anirudh Cadapa Prahallad, Monika Wroclawska, Min Lu, Claire N Harrison","doi":"10.1182/bloodadvances.2025015860","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025015860","url":null,"abstract":"<p><p>Ruxolitinib, a Janus kinase (JAK)1/JAK2 inhibitor, is the standard of care for symptomatic patients with myelofibrosis (MF). However, ~70% of patients discontinue ruxolitinib after ~5 years, a third of whom report suboptimal splenic response. ADORE was a Phase 1b/2 study with an innovative open platform design that assessed the safety, efficacy, and pharmacokinetics of novel compounds in combination with ruxolitinib in patients with MF who had a suboptimal response to ruxolitinib alone. Forty-four patients were enrolled in Part 1 of the study of ruxolitinib in combination with siremadlin, rineterkib, sabatolimab, crizanlizumab, or NIS793. Most patients were allocated to receive ruxolitinib+siremadlin (N=23). The most frequent adverse events with siremadlin were gastrointestinal (nausea and diarrhea) and hematological (thrombocytopenia, anemia, and neutropenia). Siremadlin 30 mg orally once daily on days 1-5 of a 28-day cycle was selected as the recommended phase 2 dose. The most robust spleen volume reduction (SVR) at 24 weeks was observed with ruxolitinib+siremadlin 30 mg. Reductions in percent JAK2V617F allele burden at Week 24 were observed, notably in several patients with SVR. An increase in GDF-15 protein levels in patients receiving siremadlin demonstrated the on-target modulation of downstream p53 targets. Overall, available data from ADORE suggest the feasibility and benefits of combining novel agents with ruxolitinib in patients with suboptimal response to ruxolitinib alone. This trial was registered at www.clinicaltrials.gov as #NCT04097821.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143969977","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":"Outcomes of Brexucabtagene Autoleucel in Relapsed/Refractory Acute Lymphoblastic Leukemia Patients with CNS Involvement.","authors":"Ibrahim N Muhsen, Gregory W Roloff, Rawan G Faramand, Tamer Othman, Yannis K Valtis, Noam E Kopmar, Simone E Dekker, Matthew Connor, Santiago Mercadal, Timothy E O'Connor, Kaitlyn C Dykes, Mohamed Ahmed, Nikeshan Jeyakumar, Amy Zhang, Katharine Miller, Katherine C Sutherland, Caitlin Guzowski, Vishal K Gupta, Navneet S Majhail, Minoo Battiwalla, Melhem M Solh, Shahbaz A Malik, John Mathews, Caspian Oliai, Paul J Shaughnessy, Luke Mountjoy, Catherine J Lee, Aaron C Logan, Stephanie B Tsai, Jessica T Leonard, Marc S Schwartz, Joshua P Sasine, Muthu Kumaran, Noelle V Frey, Jae H Park, Divya Koura, Ryan D Cassaday, Bijal D Shah, Ibrahim Aldoss, Lori S Muffly, LaQuisa C Hill","doi":"10.1182/bloodadvances.2024015779","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015779","url":null,"abstract":"<p><p>Relapsed/Refractory (r/r) B-cell acute lymphoblastic leukemia patients with central nervous system involvement (CNS B-ALL) have poor outcomes and were frequently excluded from CD19-targeting chimeric antigen receptor T-cell (CAR T-cell) clinical trials. The efficacy and safety of brexucabtagene autoleucel (brexu-cel) in adults with r/r B-ALL was established by the ZUMA-3 trial, which excluded patients with advanced or symptomatic CNS involvement. In this retrospective multicenter analysis, we investigated the safety and efficacy of brexu-cel in CNS B-ALL patients utilizing data from the Real-World Outcomes Collaborative for CAR T in ALL (ROCCA) consortium. Of 189 patients infused, 31 had CNS-2 (presence of blasts in CSF with < 5 WBC/uL) or CNS-3 (presence of blasts with >5 WBC/uL and/or clinical signs/symptoms) disease pre-apheresis and are the focus of this report. The median age was 46.5 years (range, 24-76), and 58.1% were males. Most (87.1%) received bridging therapy. Following brexu-cel, 21 of 24 with CNS restaging (87.5%) achieved CNS-1. Additionally, 28 of 30 evaluable patients achieved marrow complete remission (CR); 25 were MRD-negative. No statistically significant differences were seen in progression-free survival (PFS) or overall survival (OS) following brexu-cel among patients with or without CNS involvement. Similarly, grade 3-4 immune effector cell associated neurotoxicity syndrome (ICANS) occurred similarly in patients with (35.5%) and without CNS disease (30%). In conclusion, our data suggest that brexu-cel results in high response rates in CNS B-ALL patients with toxicity comparable to patients without CNS involvement.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143979065","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":"Zanubrutinib is well tolerated and effective in CLL/SLL patients intolerant of ibrutinib/acalabrutinib: Updated results.","authors":"Mazyar Shadman, John M Burke, Jennifer Cultrera, Habte A Yimer, Syed F Zafar, Jamal Misleh, Subramanya S Rao, Charles M Farber, Aileen Cleary Cohen, Hui Yao, Adam Idoine, Qi An, Ian W Flinn, Jeff P Sharman","doi":"10.1182/bloodadvances.2024015493","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015493","url":null,"abstract":"<p><p>Bruton tyrosine kinase (BTK) inhibitors such as ibrutinib revolutionized chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) treatment, although treatment-related toxicities limit the use of some BTK inhibitors. Zanubrutinib, a potent next-generation BTK inhibitor, has higher selectivity than ibrutinib or acalabrutinib. The ongoing phase 2, single-arm BGB-3111-215 study investigates the safety and efficacy of zanubrutinib in patients with B-cell malignancies who are intolerant of ibrutinib and/or acalabrutinib. Here, results in patients with CLL/SLL are presented. Patients received zanubrutinib 160 mg BID or 320 mg QD. With a 34.5-month median follow-up, 71 patients (ibrutinib intolerant only, n=44; acalabrutinib intolerant only, n=17; ibrutinib and acalabrutinib intolerant, n=10) received ≥1 zanubrutinib dose. On zanubrutinib, 54% (28/52) of ibrutinib-intolerant patients and 70% (19/27) of acalabrutinib-intolerant patients experienced no recurrence of intolerance AEs; 60% and 72% of intolerance AEs did not recur, respectively. Of recurrent ibrutinib-intolerance AEs, 64% were lower grade; 44% of acalabrutinib-intolerance AEs were lower grade. No intolerance AEs recurred at a higher grade with zanubrutinib. The most common recurrent ibrutinib-intolerance and acalabrutinib-intolerance AEs were fatigue and diarrhea, respectively. The most common TEAEs with zanubrutinib were fatigue (32%), COVID-19 (28%), and diarrhea and contusion (each 24%). Grade ≥3 TEAEs occurred in 61%, serious TEAEs in 32%, and TEAEs leading to discontinuation in 11%. Of 67 efficacy-evaluable patients, 94% experienced disease control: 30% had a best response of stable disease and 64% had a partial or complete response. These data demonstrate that patients intolerant of ibrutinib/acalabrutinib may benefit from switching to zanubrutinib therapy. ClinicalTrials.gov: NCT04116437.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143965999","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":"Intrathecal chemotherapy for ciltacabtagene autoleucel-associated movement and neurocognitive toxicity.","authors":"Kaitlin Kelly, Jennifer Cooperrider, Michael R Bishop, Satyajit Kosuri, Andrzej Jakubowiak, Benjamin A Derman","doi":"10.1182/bloodadvances.2024015721","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015721","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143976059","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":"Pivotal Results of SELECT-MDS-1 Phase 3 Study of Tamibarotene with Azacitidine in Newly Diagnosed Higher-Risk MDS.","authors":"Amy E DeZern, Sylvain Thepot, Stéphane De Botton, Andrea Patriarca, Dries Deeren, Jose Miguel Torregrose Diaz, Giovanni Marconi, Teresa Bernal Del Castillo, Juan Miguel Bergua Burgues, Blanca Xicoy, Anna Jonasova, Amer M Zeidan, Sophie Dimicoli-Salazar, Célestine Simand, David Valcárcel, María Díez-Campelo, Wanxing Chai-Ho, Lalit Saini, Alice Garnier, Klaus Geissler, Yishai Ofran, Zsolt Nagy, Pramila Krishnamurthy, Michael Lübbert, Grzegorz W Basak, Hetty E Carraway, David A Sallman, Uma Borate, Valeria Santini, Victoria Campbell, Pierre Fenaux, Thorsten Braun, Francesco Lanza, Jan Maciej Zaucha, David A Roth, Sofia Paul, Pourab Roy, Michael Kelly, Angela Volkert, Jaime Chisolm, Tanya Abdul Malak, Virginia Klimek, Thomas Cluzeau","doi":"10.1182/bloodadvances.2025016229","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025016229","url":null,"abstract":"<p><p>Higher-risk MDS (HR-MDS) with RARA gene overexpression is a subset of HR-MDS patients (pts) with an actionable target for tamibarotene, an oral and selective RARα agonist. Tamibarotene in combination with azacitidine (AZA) showed high complete remission (CR) rates in AML. SELECT-MDS-1 (NCT04797780) was a Phase 3 study comparing the activity of tamibarotene/ azacitidine (AZA) to placebo/AZA in newly diagnosed (ND) HR-MDS pts with RARA overexpression. Eligible patients had confirmed RARA overexpression by blood-based assay, untreated MDS with higher-risk features by IPSS-R and a bone marrow blast count >5%. Patients were randomized 2:1 to receive either tamibarotene/AZA or placebo/AZA, respectively. A total of 246 participants with HR-MDS and RARA overexpression were randomized with 164 and 82 in the tamibarotene/azacitidine and placebo/azacitidine groups, respectively. Baseline characteristics included: 69.9% male; median age 75 (38-93); primary MDS 89.8%; WHO 2016 classification MDS-EB-1 48%, MDS-EB-2 52%; median bone marrow blasts 9.0%; IPSS-R risk category intermediate (25.5%), high (35.7%), very high (38.9%). The study did not meet the primary endpoint of CR, with a p-value of 0.2084 for the treatment effect in the tamibarotene/AZA group compared to the placebo/AZA group. The CR rates were 23.81% and 18.75% in the tamibarotene/AZA and placebo/AZA groups, respectively. The use of tamibarotene-based therapy to target RARα as a novel approach in HR-MDS pts with RARA gene overexpression is not a paradigm which can augment response rates beyond HMA monotherapy. Further explorations of alternative approaches, including those with a biomarker, to alter the natural history of this disease are warranted.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143966012","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":"Outcomes in Frail Patients Receiving BCMA-directed Bispecific Antibodies for Relapsed/Refractory Multiple Myeloma.","authors":"Benjamin O Adegbite, Carlyn Rose Tan, Tala Shekarkhand, Ross S Firestone, Eric Matthew Jurgens, Kevin C Miller, Alexander M Lesokhin, Gunjan L Shah, Neha Korde, Sridevi Rajeeve, Heather J Landau, Michael Scordo, Hani Hassoun, Kylee H Maclachlan, Urvi A Shah, Malin L Hultcrantz, Issam S Hamadeh, Andriy Derkach, David Nemirovsky, Sergio A Giralt, Sham Mailankody, Saad Z Usmani, Hamza Hashmi","doi":"10.1182/bloodadvances.2025015973","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025015973","url":null,"abstract":"<p><p>In addition to advanced age, patients with relapsed refractory multiple myeloma (RRMM) are often frail with pre-existing comorbidities and poor performance status and have been excluded from clinical trials evaluating bispecific antibodies (BsAb). To evaluate clinical characteristics and outcomes based on frailty, we conducted a single center retrospective study of patients with RRMM who received BCMA-directed BsAb. Frailty was defined using the simplified frailty index based on age, ECOG PS, and Charlson comorbidity index (CCI); non-frail = score 0-1, frail = score ≥2. Of 102 patients analyzed (age range 40 - 88), 40 (39%) were considered frail. The frail group had more patients with age ≥ 70 years (73% vs 29%, p <0.001), ECOG PS ≥ 2 (36% vs 0%, p <0.001), and worse median CCI (2 vs 1, p <0.001). Patients in the frail group experienced similar rates of all grade CRS (58% vs 60%, p= 0.99), ICANS (15% vs 8%, p= 0.44), and TRM (13% vs 21%, p= 0.27) compared to the non-frail group. Best overall response rate (ORR) was 80% (15% sCR/CR, 48% VGPR) in the frail group vs 73% (23% sCR/CR, 31% VGPR) in the non-frail group (p= 0.40). With a median follow-up of 8.6 months (range 3-14), there was no significant difference in median PFS (Not reached vs 11 months, p= 0.051) or OS (37 vs 25 months, p= 0.37) between the frail and non-frail groups, respectively. Hence, BsAb were deemed safe and effective for elderly and frail patients with RRMM.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143963959","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}