Clinical Lymphoma, Myeloma & Leukemia最新文献

{"title":"Incidence of Cardiovascular Events and Carotid Artery USG Abnormalities in Chronic Myeloid Leukemia Patients on Nilotinib Therapy: A 20-Year Single-Center Study.","authors":"Youngeun Jang, Joon Ho Moon, Yunji Lee, Jung Min Lee, Dong Won Baek, Soon Hee Chang, Sang Kyun Sohn","doi":"10.1016/j.clml.2025.04.015","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.015","url":null,"abstract":"<p><strong>Background: </strong>The incidence of cardiovascular events (CVEs) among patients receiving nilotinib is a critical concern, particularly for those on long-term use. This study aimed to evaluate the incidence of CVEs among chronic myeloid leukemia (CML) patients and assess the efficacy of carotid artery ultrasonography (USG) in predicting CVEs in nilotinib-treated patients.</p><p><strong>Methods: </strong>We retrospectively reviewed 218 patients diagnosed with CML chronic phase who received nilotinib at the Kyungpook National University Hospital, South Korea, from January 2000 to May 2024. Carotid USG was performed on 51 patients. CVEs were defined as acute coronary syndrome, ischemic stroke, and peripheral arterial occlusive disease.</p><p><strong>Results: </strong>Among 218 patients, 41 (18.81%) experienced CVEs, with incidence increasing throughout nilotinib exposure - 15.0% at 5 years, 32.6% at 10 years, and 39.9% at 15 years. Cardiovascular risk factors, including hypertension, diabetes mellitus, dyslipidemia, influenced the occurrence of CVEs (P = .016). The 10-year cumulative CVE incidence in the patient group with ≥2 risk factors was 64.0% after nilotinib treatment. Carotid artery USG was performed in 51 out of 218 patients taking nilotinib. The correlation between cardiovascular risk factors and USG abnormalities was not statistically significant (P = 1.00).</p><p><strong>Conclusions: </strong>Nilotinib treatment in CML patients is associated with significant CVE incidence, which increases with treatment duration and the number of risk factors. Our findings support routine cardiovascular monitoring, particularly carotid USG, within the first year and regular follow-ups every 1 to 2 years based on risk factors. Proactive monitoring, risk-adjusted follow-up, and treatment-free remission considerations could help mitigate CVE risks.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144119102","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"SOHO State of the Art Updates and Next Questions | Transplant in Adult Acute Lymphoblastic Leukemia.","authors":"Curtis Marcoux, Partow Kebriaei","doi":"10.1016/j.clml.2025.04.016","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.016","url":null,"abstract":"<p><p>The landscape of acute lymphoblastic leukemia (ALL) treatment is rapidly evolving, with new therapies challenging traditional treatment paradigms. While allogeneic hematopoietic cell transplantation (allo-HCT) remains essential for many high-risk patients, advances in measurable residual disease (MRD) monitoring and the increasing use of immunotherapies in earlier treatment lines have reshaped transplant decision-making. Improvements in donor availability, conditioning strategies, and post-transplant care have expanded access and improved survival, yet relapse and toxicity remain major challenges. This review examines the evolving role of allo-HCT in ALL, highlighting key advancements and ongoing challenges.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144093065","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Transfusion Independence Corresponds With Survival in Patients With Lower-Risk Myelodysplastic Syndrome: Real-World Evidence From United States Insurance Claims.","authors":"Rami S Komrokji, Dylan Supina, Shyamala Navada, Ravi Potluri, Rohit Tyagi, Tim Werwath, Zhuoer Xie, Eric Padron, David A Sallman","doi":"10.1016/j.clml.2025.04.014","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.014","url":null,"abstract":"<p><strong>Background: </strong>Most patients with lower-risk myelodysplastic syndromes (LR-MDS) develop red blood cell transfusion dependence (RBC-TD). RBC-TD has been associated with decreased quality of life and overall survival (OS).</p><p><strong>Methods: </strong>This study assessed association between RBC-TD and survival using International Classification of Diseases, Tenth Revision (ICD.10), codes and patterns of MDS medication from a large US health insurance claims database (October 2015-March 2023) in 6531 patients who received ≥1 line of treatment.</p><p><strong>Results: </strong>Erythropoiesis-stimulating agent and hypomethylating agent monotherapy were treatments most commonly used in first-line (1L) and second-line (2L) settings. At baseline, 8% of patients had high transfusion burden (≥8 RBC U/8 weeks). In 1L and 2L, ≥16-week RBC transfusion independence (TI) was achieved by 41% (n = 935/2301) and 32% (n = 239/745) of patients, respectively. Median real-world progression-free survival (rwPFS; time from start of treatment to next treatment or progression/death, whichever occurred first) and median OS (mOS) were significantly longer in ≥16-week RBC-TI responders than nonresponders. Median (95% confidence interval [CI]) rwPFS in 1L responders versus nonresponders was 18.0 months (17.0-19.3) versus 3.3 months (3.0-3.5; P < .0001), respectively, and was 20.5 months (17.6-23.7) versus 4.1 months (3.7-4.6; P < .0001), respectively, in 2L. mOS (95% CI) in 1L responders versus nonresponders was 28.7 months (26.1-31.6) versus 8.0 months (7.0-9.1; P < .0001), respectively, and 45.2 months (35.9-49.8) versus 9.0 months (7.6-10.6; P < .0001), respectively, in 2L. RBC-TI achievement was associated with improved survival.</p><p><strong>Conclusions: </strong>Results suggest RBC-TD may be a modifiable factor corresponding to clinical outcomes in LR-MDS, further supporting RBC-TI as a primary endpoint in clinical studies.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144132071","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Risk Factors for Malignant Progression to Adult-T-Cell Leukemia/Lymphoma in HTLV-1 Carriers: A Systematic Review of Cohort and Case-Control Studies.","authors":"Jose M Estrada-Grossmann, Esteban A Alarcón-Braga, Farley E Salazar-Valdivia, Abigail S Jauregui-Cornejo, Karlos Acurio, Niels Pacheco-Barrios, Ali Al-Kassab-Córdova, Daniel Enriquez-Vera","doi":"10.1016/j.clml.2025.04.013","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.013","url":null,"abstract":"<p><strong>Objective: </strong>To systematically evaluate and synthesize the existing evidence from cohort and case-control studies to identify risk factors associated with malignant progression to ATLL in HTLV-1 carriers.</p><p><strong>Methods: </strong>A systematic search was conducted in September 2023 in the following databases: Pubmed, Scopus, Web of Science, Medline and Embase. Only case-control and cohort studies that assessed the risk factors for the malignant progression to ATLL among HTLV-1 carriers in the adult population were included. The risk of bias was assessed through the Newcastle-Ottawa Scale. Meta-analysis was not performed due to high statistical and methodological heterogeneity.</p><p><strong>Results: </strong>After conducting a systematic search, 8170 results were retrieved, of which 9 (7 case-control and 2 cohort studies) met the selection criteria. Seven of them were conducted in Japan and six had low risk of bias. Serum sIL-2R levels, proviral load (PVL), cigarette consumption, Scd30 and TNF-α-857T allele were associated with an increased risk of ATLL progression.</p><p><strong>Conclusion: </strong>PVL emerges as the foremost recommended biomarker for predicting progression in patients with HTLV-1 associated ATLL. Further studies with larger samples sizes, different populations, more rigorous statistical adjustments, and longer follow-up are warranted.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144109952","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Role of a Multidisciplinary Program (Amyloidosis Program of Calgary) on Recognition and Intervention for AL and ATTR Amyloidosis.","authors":"Aksha Burande, Nowell Fine, Robert Jh Miller, Christopher Hahn, Etienne Mahe, Debra Bosley, Lyndsay Litwin, Victor H Jimenez-Zepeda","doi":"10.1016/j.clml.2025.04.009","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.009","url":null,"abstract":"<p><strong>Background: </strong>The diagnosis and management of amyloidosis requires strong coordination among various teams and a multidisciplinary approach which is essential for providing the diagnostic tools needed for patients with organ amyloid deposition.</p><p><strong>Methods: </strong>In this study, we reviewed the referral patterns to the cardiac and hematology amyloid clinics since the inception of the Amyloidosis Program of Calgary (APC) in 2019. The APC was established to facilitate the diagnosis and treatment of amyloidosis. Additionally, a central referral system was created to expedite the triage of cases with potential amyloid-related symptoms.</p><p><strong>Results: </strong>The cardiac amyloid clinic saw an increase in referrals from 41 in 2018 to 105 in 2022, although there was a temporary decrease from 67 in 2019 to 56 in 2020 during the first year of the pandemic. Most referrals came from nonamyloid cardiology clinics, followed by hematology referrals. Fifty percent of the cases referred were patients with ATTR and 18% represented AL cases. Moreover, amyloid clinic at our cancer center also began receiving referrals via the central triage system in 2019, with AL amyloidosis cases increasing from 16 to 25 between 2018 and 2023. Therefore, we report here the significant impact of the APC on increasing referrals for patients with ATTR, with the program facilitating diagnostic pathways for these rare conditions.</p><p><strong>In summary: </strong>Since 2022, the establishment of an amyloid screening clinic has furthered these efforts, aiming to enhance early recognition and improve clinical outcomes.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144086072","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Unusually Indolent CML: Absence of Complete Cytogenetic Response after 10 Years of Tyrosine Kinase Inhibitor Therapy.","authors":"Qiudan Shen, Fadi G Haddad, Elias J Jabbour, Guilin Tang, Hong Fang, Meng Liu, Aileen Y Hu, Wei Wang, Pei Lin, Ghayas C Issa, Hagop M Kantarjian, L Jeffrey Medeiros, Shimin Hu","doi":"10.1016/j.clml.2025.04.012","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.012","url":null,"abstract":"<p><strong>Background: </strong>With BCR::ABL1 tyrosine kinase inhibitor (TKI) therapy, most patients with chronic-phase chronic myeloid leukemia (CML-CP) can achieve complete cytogenetic response (CCyR) within 6 months of therapy. However, no studies have investigated patients who do not achieve CCyR yet maintains stable disease on long-term TKI treatment. Here we investigated 30 CML-CP patients who did not achieve CCyR but remained free of blastic progression for at least 10-year while on TKI therapy.</p><p><strong>Materials and methods: </strong>Patients diagnosed with CML-CP or accelerated phase who received at least 10 years of TKI treatment, did not achieve CCyR, yet remained free of blastic progression for a minimum of 10 consecutive years during treatment, were included.</p><p><strong>Results: </strong>At TKI initiation, the median blast count in the bone marrow was 2%. During TKI therapy, 16 patients had additional chromosomal abnormalities (ACAs). The most common ACAs were +Ph (n = 9) and +8 (n = 8), mostly transient, while high-risk ACAs were less common (n = 3). BCR::ABL1 kinase domain mutations were detected in 20 patients, 12 patients having a single mutation, mostly transient, and 8 having multiple mutations, which were mostly persistent cross TKI therapies. After a median of 4 TKI lines, the best responses included complete hematologic response (CHR) (n = 15), minor cytogenetic response (n = 2), partial cytogenetic response (n = 10), and major molecular response beyond the 10-year period without CCyR (n = 3). Among 15 patients who achieved a response better than CHR, 14 eventually lost their response. After a median follow-up of 245 months from TKI initiation, 6 patients progressed to blast phase after a median of 165 months.</p><p><strong>Conclusion: </strong>Despite not achieving CCyR, most patients in this cohort maintained long-term stable disease. With careful monitoring and individualized TKI treatment, long-term survival may still be achievable in some non-CCyR responders who do not opt for allogenic stem cell transplantation.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143986926","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"SOHO State of the Art Updates and Next Questions | CD7 CAR-T Therapy for Treating CD7-Positive Hematological Malignancies: Clinical Advances and Future Directions.","authors":"Peihua Lu, Jing Long","doi":"10.1016/j.clml.2025.04.011","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.011","url":null,"abstract":"<p><p>CD7 CAR-T cell therapy has emerged as a promising treatment for relapsed/refractory (R/R) CD7-positive hematological malignancies, offering new hope for patients with limited therapeutic options. This review examines the recent clinical advances, challenges, and future directions of CD7 CAR-T therapy. Clinical trials have demonstrated remarkable efficacy of CD7 chimeric antigen receptor T (CD7 CAR-T) cells in treating T-cell acute lymphoblastic leukemia (T-ALL), T-cell lymphoblastic lymphoma (T-LBL), and other CD7-positive malignancies, with complete remission (CR) rates of 90-95% in bone marrow (BM) and 50% to 60% in extramedullary disease (EMD). Various engineering strategies, including naturally selected CD7-targeted CAR-T cells, gene editing, protein blockers and universal CAR-T cells, have been developed to overcome challenges such as fratricide. While CD7 CAR-T therapy has shown promising initial responses, durable remissions often depend on consolidative allogeneic hematopoietic stem cell transplantation (allo-HSCT). Ongoing research is focused on optimizing CAR designs, improving CAR-T cell persistence, and developing novel combination strategies to enhance long-term outcomes. Safety profiles have been generally manageable, with cytokine release syndrome (CRS) and neurotoxicity being the primary concerns. However, prolonged cytopenias and potential long-term immunodeficiency due to depletion of healthy CD7-positive cells remain areas of active investigation. As CD7 CAR-T therapy continues to evolve, future directions include refining patient selection, exploring dual-targeting approaches, and investigating innovative strategies to integrate CAR-T therapy with allo-HSCT. These advancements aim to improve the efficacy, safety, and accessibility of CD7 CAR-T therapy for patients with CD7-positive hematological malignancies.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144076428","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"SOHO State of the Art Updates and Next Questions | Manuscript Title: Bispecific T-Cell Engagers: Sequencing, B-Cell Maturation Antigen, GPRC5D, and Resistance.","authors":"Sabarish Ayyappan, Jeremy Larsen, James Sanchez, Amrita Krishnan","doi":"10.1016/j.clml.2025.04.006","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.006","url":null,"abstract":"<p><p>Bispecific antibodies (BsAbs) simultaneously engage endogenous T cells and malignant cells via binding to a T-cell epitope and an extracellular tumor antigen, leading to cytotoxic T-cell activity and tumor cell death. The incorporation of BsAbs into the treatment of multiple myeloma (MM) has improved options for patients with advanced relapsed MM. Three BsAbs have been approved by the Food and Drug Administration; 2 target BCMA on the surface of MM cells, while the other targets surface GPRC5D. Many other BsAbs are in development, including those that target antigens other than BCMA and GPRC5D, such as FcRH5, or that feature different binding constructs. As new BsAbs and new anti-MM agents in general become available, the question of sequencing these therapies becomes paramount. Moreover, resistance to therapy leading to disease relapse following initial response remains a major clinical obstacle, driving research into identifying mechanisms of resistance and developing approaches to overcome them. Understanding this topic will also guide how to best sequence therapies. Because BsAbs are dependent on T cell fitness, researchers are studying the concepts of employing combination therapies that improve T cell health, using a fixed duration of treatment to limit T cell exhaustion, and administering BsAbs earlier in the disease course.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144093064","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world Efficacy and Safety of Flumatinib as the First-line Treatment in Patients With de novo Philadelphia-positive Acute Lymphoblastic Leukemia.","authors":"Zhibo Zhang, Jia Yin, Jun Wang, Xuefeng He, Xiaohui Hu, Haiwen Huang, Limin Liu, Miao Miao, Ying Wang, Huiying Qiu, Xiaowen Tang, Depei Wu, Xiao Ma, Weiyang Li","doi":"10.1016/j.clml.2025.04.010","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.010","url":null,"abstract":"<p><strong>Background: </strong>The real-world data of flumatinib, a China-developed second-generation tyrosine kinase inhibitors (TKI), on Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) is limited. This study evaluated the efficacy and safety of flumatinib combined with chemotherapy as first-line therapy for Ph+ ALL.</p><p><strong>Methods: </strong>This multicenter retrospective study included 65 adults with de novo Ph+ ALL treated with flumatinib (600 mg/day) plus intensive chemotherapy. Outcomes included complete remission (CR), CR with minimal residual disease negativity detected by multiparameter flow cytometry (CR _flow-), overall survival (OS), event-free survival (EFS), and adverse events (AEs). Survival and prognostic factors were analyzed using the Kaplan-Meier method.</p><p><strong>Results: </strong>CR and CR _flow- were achieved in 96.9% (63/65) and 58.5% (38/65) of patients postinduction, respectively. At 24 months, OS and EFS were 87.4% and 62.6%, respectively. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) improved EFS (P < .0001) and tended to improve OS (P = .0503). Common hematologic AEs included neutropenia (93.8%) and thrombocytopenia (76.9%); severe nonhematologic AEs were rare. Relapses (17/63) predominantly associated with T315I (10/19) and Y253H (5/19) mutations. Age ≥ 35 years, BCR/ABL1^P210, and ABL1 Y253H mutation were significantly associated with worse OS. Meanwhile, female, ABL1 Y253H mutation, ABL1 T315I mutation, and failure with achievement of MMR after the first course of consolidation therapy were risk factors for EFS.</p><p><strong>Conclusions: </strong>Flumatinib-based regimens demonstrated high efficacy and tolerable toxicity in Ph+ ALL, which was comparable to other second-generation TKIs. T315I and Y253H mutations were key drivers of relapse. Although allo-HSCT enhanced survival, longer follow-up period and prospective trials are warranted to validate these findings.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144076427","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment Patterns, Efficacy, and Tolerability of Belantamab Mafodotin in Patients With Relapsed and/or Refractory Multiple Myeloma: A Real-World Analysis.","authors":"Mihir N Patel, Susan C Locke, Caroline Falvey, Jesse D Troy, Kris W Herring, Amanda Bolgioni-Smith, Cherie Elcock, Laura Iadeluca, Cristiana Costa Chase, Cristina Gasparetto, Mark S Newman, Thomas W LeBlanc","doi":"10.1016/j.clml.2025.04.008","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.008","url":null,"abstract":"<p><strong>Purpose: </strong>Belantamab mafodotin is an anti-B cell maturation antigen (BCMA) immunoconjugate for patients with relapsed/refractory multiple myeloma (RRMM). Additional data on its treatment patterns, efficacy, and tolerability in real-world settings are needed.</p><p><strong>Patients and methods: </strong>This single-site, retrospective study examined 30 adults with multiple myeloma receiving care at Duke Cancer Institute who began belantamab mafodotin monotherapy between 8/5/2020 and 11/22/2022. We described baseline clinical characteristics, disease response (per International Myeloma Working Group [IMWG] criteria, as possible given available bone marrow biopsy data), belantamab mafodotin treatment patterns, and ocular adverse events (per the Keratopathy Visual Acuity [KVA] scale).</p><p><strong>Results: </strong>Across 30 patients, the median number of lines of therapy was 4, 20 patients (87%) were triple-/quad-/penta-refractory, and 7 (23%) had high-risk cytogenetics (per IMWG criteria). Overall response rate was 67%. Progression occurred in 22 patients (73%); median progression-free survival was 9.5 months (95% CI 6.6-15.6). Median overall survival was not reached as of 3-year follow-up. Dose reduction occurred in 19 patients (63%); 65% were due to ocular adverse events, 30% hematologic adverse events. Cycle delay occurred in 28 (93%); 85% were due to ocular adverse events. Keratopathy (any grade) occurred in 28 (93%); 2% of events were grade ≥3. Visual acuity change (any grade) occurred in 27 (90%); 3.3% of events were grade ≥3. Treatment discontinuation from ocular toxicity occurred in 4 (13%).</p><p><strong>Conclusion: </strong>Despite a high incidence of manageable keratopathy, these data demonstrate benefit in belantamab mafodotin in patients with RRMM over an extended time amid dose/cycle modifications.</p><p><strong>Registration: </strong>CLINICALTRIALS.GOV: NCT05986682.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143981233","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}