Blood Cancer Journal最新文献

{"title":"Effect of Intravenous Immunoglobulin (IVIG) Supplementation on infection-free survival in recipients of BCMA-directed bispecific antibody therapy for multiple myeloma","authors":"Meera Mohan, Aniko Szabo, Heloise Cheruvalath, Anna Clennon, Vineel Bhatlapenumarthi, Anannya Patwari, Metodi Balev, Divaya Bhutani, Asis Shrestha, Sharmilan Thanendrarajan, Binod Dhakal, Maurizio Zangari, Anup Trikannad, Sruthi Vellanki, Samer Al-Hadidi, Suzanne Lentzsch, Frits van Rhee, Aishee Bag, Anita D’Souza, Nishi Shah, Rajshekhar Chakraborty, Mansi R. Shah, Carolina Schinke","doi":"10.1038/s41408-025-01282-0","DOIUrl":"https://doi.org/10.1038/s41408-025-01282-0","url":null,"abstract":"<p>The main objective of this multi-institutional study is to understand the effect of primary intravenous immunoglobulin (IVIG) replacement on clinical outcomes in recipients of BCMA-directed bispecific antibody (bsAb), where infection remains an important cause of morbidity and mortality. This is a retrospective study of patients treated with either standard of care teclistamab or BCMA-directed investigational bsAb between Nov 2017 and Dec 2023. Primary IVIG prophylaxis was defined as starting IVIG prior to first documented infection. All analyses were adjusted for immortal-time bias inherent in this grouping. A total of 225 patients were included in this analysis. Primary IVIG prophylaxis was defined as starting IVIG prior to first documented infection. The median follow-up of patients treated with and without primary IVIG prophylaxis was, 9 and 11 months, respectively. The cumulative incidence of all grade infections at 12 months with and without primary IVIG prophylaxis were 56% (95%CI 40%,78%) and 60% (95% CI 48%, 76%); <i>p</i> = 0.72, respectively. The 12-month cumulative incidence of ≥ grade 3 infections was 35% (95% CI 21%, 57%) with primary IVIG prophylaxis and 45% (95% CI 34%, 60%) without; <i>p</i> = 0.37. The median infection free survival (IFS) for all-grade infections was 7.7 (95% CI 3.3, 14) months with primary IVIG prophylaxis and 3 (95% CI 2.6, 4.5) months without (<i>p</i> = 0.021). The median ≥ grade 3 IFS was 14 (95% CI 8.8, NR) and 7.5 (95% CI 6.1, 14) months, with and without primary IVIG respectively; <i>p</i> = 0.022. Patients on primary IVIG prophylaxis had a superior progression-free-survival (PFS) [median PFS 15 vs 8 months; <i>p</i> = 0.026] and overall-survival (OS) [median OS 16 vs 44 months; <i>p</i> = 0.007]. On multivariate analysis, primary IVIG prophylaxis was independently associated with improved OS (HR = 0.37; <i>p</i> = 0.021), while the presence of extra-medullary (HR = 2.71; <i>p</i> = &lt;0.001) and high-risk disease (HR = 1.88; <i>p</i> = 0.031) conferred poor outcomes. In recipients of BCMA-directed bsAb, IVIG supplementation was associated with an improved clinical outcome, including favorable IFS and OS.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"19 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143866326","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":"Establishing measurable residual disease trajectories for patients on treatment for newly diagnosed multiple myeloma as benchmark for deployment of T-cell redirection therapy","authors":"Susan Bal, Tylan Magnusson, Gayathri Ravi, Smith Giri, Kelly Godby, Binod Dhakal, Natalie S. Callander, Rebecca W. Silbermann, Bhagirathbhai Dholaria, Vishnu B. Reddy, Luciano J. Costa","doi":"10.1038/s41408-025-01252-6","DOIUrl":"https://doi.org/10.1038/s41408-025-01252-6","url":null,"abstract":"<p>Autologous stem cell transplantation (ASCT) has been the prime consolidative strategy to increase the depth and duration of response in newly diagnosed multiple myeloma (NDMM), albeit with short- and long-term toxicities. Minimal residual disease (MRD) is an important early response endpoint correlating with clinically meaningful outcomes and may be used to isolate the effect of ASCT. We report the impact of ASCT on MRD burden and generate a benchmark for evaluation of novel treatments as consolidation. We collected MRD by next generation sequencing (NGS; clonoSEQ®) post induction and post-ASCT in consecutive patients (<i>N</i> = 330, quadruplet, <i>N</i> = 279; triplet, <i>N</i> = 51). For patients receiving quadruplets, MRD &lt; 10⁻⁵ post-induction was 29% (MRD &lt; 10⁻⁶ 15%) increasing to 59% post-ASCT (MRD &lt; 10⁻⁶ 45%). Among patients with MRD &gt; 10⁻⁵ post-induction, ASCT lowered the MRD burden&gt;1 log₁₀ for 69% patients. The use of quadruplet induction (vs. triplet) did not reduce the effect of ASCT on MRD burden. Reduction in MRD burden with ASCT was most pronounced in patients with high-risk chromosome abnormalities.</p><p>This dataset provides granular data to delineate the impact of ASCT on MRD as legacy consolidative strategy in NDMM and provides an important benchmark for evaluation of efficacy of TCRT as experimental consolidative strategy.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"1 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143862954","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":"Basophilia and eosinophilia in primary myelofibrosis: phenotype, genotype, and prognostic correlates","authors":"Sarah Dingli, Saubia Fathima, Priyansh Faldu, Naseema Gangat, David Dingli, Ayalew Tefferi","doi":"10.1038/s41408-025-01285-x","DOIUrl":"https://doi.org/10.1038/s41408-025-01285-x","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"49 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143853254","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":"Comment on: \"Switching TKIs during CML therapy is frequent, mostly driven by intolerance, and does not affect survival: a prospective Quebec registry study\" by Busque et al.","authors":"Ahmet Emre Eşkazan","doi":"10.1038/s41408-025-01288-8","DOIUrl":"https://doi.org/10.1038/s41408-025-01288-8","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"32 1","pages":"70"},"PeriodicalIF":12.8,"publicationDate":"2025-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143851052","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":"Risk of lymphoid malignancy associated with cancer predisposition genes","authors":"Nicholas J. Boddicker, Raphael Mwangi, Dennis P. Robinson, Cristine Allmer, Allison C. Rosenthal, Thomas M. Habermann, Andrew L. Feldman, Lisa M. Rimsza, Rebecca L. King, Melissa C. Larson, Bri J. Negaard, Aaron D. Norman, Nikhil Rajkumar, Stephen M. Ansell, Angela Dispenzieri, David L. Murray, Vincent Rajkumar, Shaji Kumar, Jithma P. Abeykoon, Grzegorz S. Nowakowski, Thomas E. Witzig, Anne J. Novak, Susan L. Slager, Celine M. Vachon, James R. Cerhan","doi":"10.1038/s41408-025-01283-z","DOIUrl":"https://doi.org/10.1038/s41408-025-01283-z","url":null,"abstract":"<p>We investigated the prevalence of rare inherited pathogenic variants (PV) in 19 cancer predisposition genes regularly included on multi-gene panel testing based on NCCN guidelines and their association with the risk of lymphoid malignancies (LM) overall and by common lymphoma subtypes and multiple myeloma. The study population included newly diagnosed LM cases (<i>N</i> = 6990) and unrelated controls (<i>N</i> = 42,632), excluding individuals with a history of hematologic malignancy. Whole exome sequencing was performed on DNA from whole blood. PV were defined as loss-of-function (i.e., nonsense, frameshift, consensus splice sites) or identified as “pathogenic” or “likely pathogenic” in the ClinVar database. A total of 1816 (3.7%) individuals had a PV across the 19 genes, higher in cases (4.7%) than controls (3.5%). In controls, <i>CHEK2</i> (1.0%), <i>ATM</i> (0.4%), <i>BRCA2</i> (0.4%), and <i>BRCA1</i> (0.3%) had the highest prevalence. <i>ATM</i> (odds ratio [OR] = 1.86, 95% confidence interval [CI]: 1.36–2.49), <i>CHEK2</i> (OR = 1.74, 95% CI: 1.42–2.13) and <i>TP53</i> (OR = 9.07, 95% CI: 4.51–18.87) were associated with increased risk of LM overall and were further validated in the UK Biobank. We observed heterogeneity in associations by LM subtype. These results demonstrate that several commonly tested cancer predisposition genes are associated with an increased risk of LM.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"17 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143849702","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":"Real world outcomes of momelotinib in myelofibrosis patients with anemia: results from the MOMGEMFIN study","authors":"Lucía Pérez-Lamas, Adrián Segura Diaz, Regina García Delgado, Alberto Álvarez-Larrán, María Alicia Senin, Elvira Mora, María Laura Fox, Irene Pastor Galan, Gemma Azaceta, Sara Garrido Paniagua, Raúl Pérez Lopez, Diana Margarita Trejos Carvajal, Anna Angona, Carmen Albo López, Pablo Lorente Alegre, Miriam Vara, Juan Antonio Vera Goñi, Dunia De Miguel Llorente, Ángeles Fernández Rodríguez, Alberto Marín Sanchez, Adriana Hernando Megido, María Teresa Gómez Casares, Ruth Stuckey, Gonzalo Carreño-Tarragona, Natalia De Las Heras Rodriguez, Blanca Xicoy, Manuel Pérez Encinas, Raquel Mata Serna, Lucia Núñez Martin-Buitrago, Francisca Ferrer Marín, Neus Amer Salas, Carolina Guillén Rienda, Patricia Velez, Laura Lamarca Eraso, Sandra Martín, Iryna Luts Khoroz, Erik De Cabo López, Angela Gil, Sofía Martín-Consuegra Ramos, Fernando Marco De Lucas, María José́ Otero Martinez-Fornes, María Luisa Martín Mateos, Teresa Arquero, Elena Cabezudo Pérez, Luis Antonio López Gómez, Ángela Martínez Hellin, Aurelia Tejedor, Esther Herrera de Pablo, María Isabel Mata Vazquez, Inmaculada Castillo Valero, María José Fernández, Carlos Aguilar, Marta Santaliestra, Antonio García Menchon, Begoña Navas Elorza, María Antonia Duran, María Pérez Sala, Teresa Hernández Santamaría, Ma Ángeles Muñoz Jarreño, Julio Dávila-Valls, Williana Torres Jiménez, Joan Alfons Gonzálvez Fernández, Hugo Alexander Torres Mantilla, Teresa Cobo Rodríguez, Aitor Abuin Blanco, Francisco Pérez, Santiago Osorio Prendes, Paola Beneit Villena, Raisa Peralta, Federico Herrera, Eloi Cañamero Giro, Reyes Jiménez Bárcenas, Mercedes Gasior Kabat, Sonia González De Villambrosia, Mariana Teresa Tercero-Mora Rodriguez, Marina Menéndez Cuevas, Beatriz Cuevas Ruiz, Marta Fonseca-Santos, Sonia Garcés Piquer, Rosalía De La Puerta, Álvaro Lorenzo Vizcaya, Juan Carlos Hernández Boluda, Valentín García Gutiérrez","doi":"10.1038/s41408-025-01275-z","DOIUrl":"https://doi.org/10.1038/s41408-025-01275-z","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"108 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143841348","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":"Reduced venetoclax exposure to 7 days vs standard exposure with hypomethylating agents in newly diagnosed AML patients","authors":"Christophe Willekens, Alexandre Bazinet, Samy Chraibi, Alex Bataller, Justine Decroocq, Naszrin Arani, Benjamin Carpentier, Caitlin Rausch, Delphine Lebon, Abhishek Maiti, Nicolas Gauthier, Nicholas Short, Sarah Bonnet, Koji Sasaki, Sabine Khalife-Hachem, Mahesh Swaminathan, Jean-Baptiste Micol, Florence Pasquier, Christophe Marzac, Damien Roos-Weil, Laurent Pascal, Naval Daver, Tapan Kadia, Didier Bouscary, Farhad Ravandi, Arnaud Pages, Hagop Kantarjian, Stéphane De Botton, Courtney DiNardo","doi":"10.1038/s41408-025-01269-x","DOIUrl":"https://doi.org/10.1038/s41408-025-01269-x","url":null,"abstract":"<p>Hypomethylating agent (HMA) plus venetoclax (VEN) regimens are standard of care in patients with acute myeloid leukemia (AML) ineligible for intensive chemotherapy. While the VEN label recommends continuous 28-day cycles, shortened VEN durations may induce similar response rates and improve tolerability. It is unknown how a VEN exposure reduced to 7 days during cycles compares to standard HMA + VEN. We retrospectively compared newly diagnosed AML patients treated with azacitidine (AZA) x 7 days plus VEN x 7 days (“7 + 7” regimen) from the first cycle (<i>n</i> = 82) vs patients treated with standard dose HMA + VEN (std-HMA/VEN) (<i>n</i> = 166). Composite complete remission rate was similar between cohorts (72% vs 72%; <i>p</i> = 0.95) and a median number of cycles to best response was 2 with “7 + 7” vs 1 with std-HMA/VEN (<i>p</i> = 0.03). Concerning toxicity, platelet transfusion rates during cycle 1 as well as early mortality at 8-weeks (6% vs 16%; <i>p</i> = 0.03) were lower in “7 + 7” cohort. Finally, the median OS was 11.2 months (2-year 28%) with “7 + 7” vs 10.3 months (2-year 34%) with “std-HMA/VEN” (<i>p</i> = 0.75). In summary, acknowledging limitations of a retrospective comparison, a shortened course of VEN used for 7 days every 28 days resulted in similar response rates and survival when compared to standard VEN exposure.</p><figure></figure>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"8 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143841345","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":"Long-term outcomes and clinical phenotypes associated with best response to low dose alemtuzumab in cutaneous T-cell lymphoma","authors":"Cecilia Larocca, Ai-Tram N. Bui, John T. O’Malley, Anita Giobbie-Hurder, Marianne Tawa, Jessica E. Teague, Rachael A. Clark, Corey Cutler, Eric Jacobsen, David C. Fisher, Thomas S. Kupper, Nicole R. LeBoeuf","doi":"10.1038/s41408-025-01237-5","DOIUrl":"https://doi.org/10.1038/s41408-025-01237-5","url":null,"abstract":"<p>Cutaneous T-cell lymphomas (CTCL) have in common malignant T-lymphocyte infiltration in the skin. Low dose alemtuzumab (LDA) appears to be an effective treatment for leukemic disease, but in the absence of clinical trials, there is need for improved characterization of patients with CTCL most likely to benefit. A retrospective cohort study of 38 patients with CTCL treated with LDA with at least 5 years’ follow-up data was conducted. As a surrogate for a central memory T-cell (T_CM) clinical phenotype, we evaluated whether the absence of a history of papules, plaques, and tumors (PPT) predicts better global and skin response. Twenty-five (65.8%) patients responded to LDA (95% CI: 49–80%). Patients with a T_CM phenotype were more than eight times as likely to achieve a CR [OR: 8.2, 95% CI: 1.2–57.6]. CR rate in the skin was 81.8% in T_CM phenotype patients compared to 37.0% in patients with a history of PPT (resident memory T-cell phenotype, T_RM) [OR: 7.7, 95% CI: 1.4–42.7]. Three patients experienced any infection requiring systemic intervention. LDA monotherapy can safely produce exceptional response rates in those presenting with diffuse erythema but without a history of PPT.</p><figure></figure>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"90 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143847090","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":"PDL1-expressing macrophages infiltrate diffuse large B-cell lymphoma and promote lymphoma growth in a MYC-driven experimental model","authors":"Ningxuan Cui, Peter Leary, Vanesa-Sindi Ivanova, Kristin Stirm, Lydia Kirsche, Nicola Aceto, Frank Stenner, Lothar C. Dieterich, Michael Detmar, Ekaterina Petrova, Sarah Mundt, Melanie Greter, Alexandar Tzankov, Anne Müller","doi":"10.1038/s41408-025-01281-1","DOIUrl":"https://doi.org/10.1038/s41408-025-01281-1","url":null,"abstract":"<p>The infiltration of diffuse large- and other mature B-cell lymphomas with T- and myeloid cells is a key tumor microenvironmental feature but is not currently factored into treatment decisions. Here, we have used multiplex immunofluorescence microscopy to quantify the immune infiltrates of &gt;260 diffuse large B-cell- (DLBCL), follicular- (FL) and mantle cell lymphomas (MCL), and chronic lymphocytic leukemias (CLL) relative to clinical outcomes, mutational landscape and phenotype. MCL were found to be the “coldest” and DLBCL the “hottest” entities. The lymphoma microenvironment of DLBCL featured numerically dominant populations of CD8⁺ and T-follicular helper (Tfh) T-cells that were indicative of superior prognosis. Mutations in <i>EZH2</i>, <i>PTEN</i> and <i>KMT2D</i> were overrepresented in DLBCL with low CD8⁺ T-cell infiltration. A unique feature of DLBCL was its infiltration by large numbers of PDL1⁺ macrophages that constituted up to 70% of total cellularity. PDL1⁺ macrophage infiltration was mutually exclusive with regulatory T-cell infiltration. The inducible ablation of PDL1 on macrophages was sufficient to improve immune control of MYC-expressing lymphoma in a syngeneic immunocompetent model. These results implicate the macrophage/CD8⁺ T-cell axis as a key pathogenetic determinant and immunotherapeutic target in a subset of DLBCL patients with poor prognosis.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"5 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143841347","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 patients with relapsed/refractory lymphoplasmacytic lymphoma/waldenström macroglobulinemia treated with venetoclax: a multicenter retrospective analysis","authors":"Y. Sawalha, S. Sarosiek, R. L. Welkie, S. Seif, S. Thapa, S. Zanwar, K. Cahill, R. Treitman, H. Shah, S. Arora, G. Pongas, A. Winter, A. Major, P. A. Riedell, M. L. Palomba, P. Kapoor, A. Grajales-Cruz, K. H. Shain, S. K. Thomas, J. J. Castillo","doi":"10.1038/s41408-025-01271-3","DOIUrl":"https://doi.org/10.1038/s41408-025-01271-3","url":null,"abstract":"<p>Venetoclax showed promising activity in a small phase II trial in relapsed/refractory Waldenström macroglobulinemia (WM). To report the clinical activity of venetoclax and prognostic factors associated with outcomes in a larger cohort, we retrospectively identified 76 patients with relapsed/refractory lymphoplasmacytic lymphoma (LPL)/WM treated with venetoclax monotherapy at nine US medical centers. The median age at venetoclax treatment initiation was 66 years. <i>MYD88, CXCR4</i>, and <i>TP53</i> mutations were detected in 65 (94%), 23 (40%), and 10 (22%) patients, respectively. The median number of prior lines of treatment was 3, including covalent BTK inhibitor in 82% and alkylating agent in 71% of patients. The overall and major response rates to venetoclax were 70% and 63%, respectively. The median and 2-year progression-free survival (PFS) were 28.5 months and 57%, respectively. The median and 2-year overall survival were not reached and 82%, respectively. Prior treatment with BTK inhibitor was the only factor associated with PFS in multivariate analysis (hazard ratio 2.97, p = 0.012). Venetoclax dose interruptions and/or reductions occurred in 27 patients (41%). Five patients (7%) developed laboratory tumor lysis syndrome (TLS), including 3 (4%) with clinical TLS. Venetoclax resulted in a high response rate and a prolonged PFS in patients with heavily pretreated LPL/WM.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"13 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143831795","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}