European Journal of Haematology最新文献

{"title":"ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B","authors":"Fabienne Volot,&nbsp;Sabine Castet,&nbsp;Alexandra Fournel,&nbsp;Birgit Frotscher,&nbsp;Benjamin Gillet,&nbsp;Dominique Desprez,&nbsp;Brigitte Tardy,&nbsp;Antoine Rauch,&nbsp;Pierre Chamouni,&nbsp;Christine Biron-Andreani,&nbsp;Jean-Baptiste Valentin,&nbsp;Annie Harroche,&nbsp;Yesim Dargaud,&nbsp;Brigitte Pan Petesch,&nbsp;Roseline d'Oiron,&nbsp;Claire Berger,&nbsp;Claire Reynes,&nbsp;Thomas Lauvray,&nbsp;Emmanuelle de Raucourt,&nbsp;Abel Hassoun,&nbsp;Aurélien Lebreton,&nbsp;Vincent Cussac,&nbsp;Hasan Catovic,&nbsp;Cédric Martin,&nbsp;Benoit Guillet","doi":"10.1111/ejh.14357","DOIUrl":"10.1111/ejh.14357","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>To assess the real-world efficacy and safety of recombinant factor IX albumin fusion protein (rIX-FP) in patients with hemophilia B (HB) in France.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Data on dosing frequency, weekly consumption, and bleeds before-and-after switching to rIX-FP, were collected from December 2021 to February 2024. Annualized (spontaneous) bleeding rates [A(s)BRs] were calculated only in patients on prophylaxis with a follow-up ≥ 6 months.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>This interim analysis focused on 77 patients ≥ 12 years; 62 (81%) had severe HB. After switching to rIX-FP, the infusion interval was 14 (7–14) days. Weekly consumption was 43 (35.5–53) IU/kg. ABRs and AsBRs were 0.5 (0–1.9) and 0 (0–0.7) (<i>n</i> = 63) at 18.2 (12.3–21.9) months of follow-up. Prophylactic efficacy of rIX-FP was considered ‘Excellent’/‘Good’ in 65/68 (95%) patients. Among the 43 patients previously treated with rFIXFc, 21 increased the infusion interval from 7 (7–11) days with rFIXFc to 14 (7–14) days with rIX-FP; 33/43 (77%) reduced weekly factor IX (FIX) consumption from 59.95 (46.35–77.93) to 42.5 (35.88–50.25) IU/kg. Patients maintained good protection against bleeds.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>This analysis confirmed that switching to rIX-FP allows for reducing injection frequency and FIX consumption while maintaining good bleed protection.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"508-516"},"PeriodicalIF":2.3,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14357","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142784585","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Venetoclax in the Treatment of Multiple Myeloma: A Retrospective Analysis of 79 Patients","authors":"Eli Zolotov,&nbsp;Vanisha Patel,&nbsp;Yedidyah Weiss,&nbsp;Noa Biran,&nbsp;Pooja Phull,&nbsp;David H. Vesole,&nbsp;David S. Siegel,&nbsp;Harsh Parmar","doi":"10.1111/ejh.14343","DOIUrl":"10.1111/ejh.14343","url":null,"abstract":"<div>\u0000 \u0000 <p>Venetoclax, the first-in-class BCL-2 inhibitor, has shown efficacy in multiple myeloma (MM), particularly in patients with the t(11;14) translocation. This single-center retrospective analysis included MM patients treated with venetoclax from November 2017 to March 2023. Data encompassed demographics, disease characteristics, treatment regimens, and outcomes using median progression-free-survival (mPFS). Eligibility required patients to be aged 21 and older and to have received at least one venetoclax cycle. Seventy-nine patients (median age 61, 58.2% female) were included, with 31.6% having t(11;14). Patients had received a median of 5 lines of therapy (LOT) before venetoclax. The mPFS was 3.4 months, with a significant difference between t(11;14) positive (7.8 months) and negative patients (2.4 months, <i>p</i> &lt; 0.01). Patients achieving a very good partial response or better had a mPFS of 7.1 months. Common adverse events included fatigue (31.6%), diarrhea (26.5%), and respiratory infections (30.3%). Univariable and multivariable analyses identified sex, disease response, and t(11;14) presence as significant survival predictors. Our real-world study suggests that venetoclax demonstrates moderate efficacy in heavily pretreated patients with relapsed/refractory MM (RRMM), compared to previous studies where patients received 1–3 prior LOT, such as the BELLINI study (mPFS of 22.4 months). Notably, the efficacy was higher in patients with t(11;14). Despite the heavily pretreated nature of our cohort, venetoclax was well tolerated, with a manageable safety profile and low incidence of severe infections, largely due to effective prophylactic measures. Venetoclax should be carefully considered in heavily pretreated populations, and further multicenter research is essential to better define its role in RRMM.</p>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"500-507"},"PeriodicalIF":2.3,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142784607","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Health-Related Quality of Life During Carfilzomib–Lenalidomide–Dexamethasone Consolidation: Findings From the Multiple Myeloma CONPET Study","authors":"Tine Rosenberg,&nbsp;Sören Möller,&nbsp;Niels Abildgaard,&nbsp;Jakob Nordberg Nørgaard,&nbsp;Anna Lysén,&nbsp;Galina Tsykonova,&nbsp;Cristina Joao,&nbsp;Annette Vangsted,&nbsp;Fredrik Schjesvold,&nbsp;Lene Kongsgaard Nielsen","doi":"10.1111/ejh.14358","DOIUrl":"10.1111/ejh.14358","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>In the CONPET study, multiple myeloma patients with abnormal 18FDG positron emission/computed tomography scan after upfront autologous stem cell transplantation were treated with four cycles of carfilzomib–lenalidomide–dexamethasone (KRd). Side effect registrations show that carfilzomib might cause dyspnea, cough, respiratory tract infections, and heart failure. The aims were to investigate patient-reported shortness of breath and dyspnea during KRd consolidation.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>To assess shortness of breath, patients completed the Functional Assessment of Cancer Therapy—Pulmonary Symptom Index (FACT-PSI) and the EORTC QLQ-C30 to assess dyspnea. Shortness of breath was defined as decrease in FACT-PSI score <i>or</i> starting/increasing diuretic drugs. Mixed effect logistic regression was used for the effect analysis. Linear mixed model and clinical relevance were used to investigate dyspnea.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total of 50 patients were included, median age 62 years (interquartile range 54–67). 17% reported shortness of breath at Day 15 Cycles 1–4 versus 11% at Day 1 Cycles 2–4, Cycle 4 Day 29, and 1 month posttreatment (<i>p</i>-value 0.048). Compared with baseline, patients reported significant, and clinically relevant worsening in dyspnea during consolidation.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Our study confirmed earlier findings of carfilzomib causing shortness of breath during KRd administration and revealed dyspnea during consolidation compared to baseline.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Trial Registration</h3>\u0000 \u0000 <p>\u0000 Clinicaltrials.gov: NCT03314636, EudraCT: 2017–000586-72</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"517-527"},"PeriodicalIF":2.3,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142784581","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Prevalence of Diffuse Large B-Cell Lymphoma Varies Substantially by Methods Applied: Results From a Population-Based Study","authors":"Mikkel Runason Simonsen,&nbsp;Jonas Faartoft Jensen,&nbsp;Thomas Stauffer Larsen,&nbsp;Inger-Lise Gade,&nbsp;Peter de Nully Brown,&nbsp;Tarec Christoffer El-Galaly","doi":"10.1111/ejh.14359","DOIUrl":"10.1111/ejh.14359","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>Accurate prevalence estimates of diffuse large B-cell lymphoma (DLBCL) are important for numerous purposes including orphan drug designation. A key criterion for orphan drug designation is a disease prevalence of less than 5/10,000 persons. The objective is to apply and compare different methods of prevalence assessment.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>In the present nationwide Danish cohort study, the prevalence of DLBCL was assessed using different methodologies, including register-based and formula-based approaches.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The prevalence calculations were based on 9,492 patients diagnosed with DLBCL since year 2000. Incidence increased and survival improved in the period, resulting in higher prevalence of DLBCL. In year 2023, the 2-,3-,5-,10-, and 20-year prevalences were 1.53, 2.19, 3.45, 6.08, and 8.80 per 10,000 adults using the register-based approach. The formula-based approach was generally accurate when using restricted mean survival. However, when using median survival, the total prevalence was estimated at 8.1 per 10,000 adults. Furthermore, when extrapolating the median survival from the 5-year survival under constant hazard assumption as done in some orphan drug designation reports, the prevalence was estimated at 6.6 per 10,000 adults.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>In conclusion, the estimated DLBCL prevalences are sensitive to the applied method. DLBCL would disqualify from orphan drug designation in some of the mentioned scenarios.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"528-535"},"PeriodicalIF":2.3,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142784587","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Chromosome 1 Alterations in Multiple Myeloma: Considerations for Precision Therapy","authors":"Niamh McAuley,&nbsp;Izabela Cymer,&nbsp;Roisin McAvera,&nbsp;Ann M. Hopkins,&nbsp;Siobhan V. Glavey","doi":"10.1111/ejh.14352","DOIUrl":"10.1111/ejh.14352","url":null,"abstract":"<p>Multiple myeloma (MM) is an incurable blood malignancy characterized by the clonal expansion of plasma cells and the secretion of monoclonal immunoglobulins. High-risk MM, defined by specific cytogenetic abnormalities, poses significant therapeutic challenges and is associated with inferior survival outcomes compared to standard-risk disease. Although molecularly targeted therapies have shown efficacy in other hematologic malignancies, currently venetoclax is the only targeted therapy approved for MM (t(11;14)). However, chromosome 1q gains, amplifications, and 1p deletions are frequently observed in MM, and have been linked to drug resistance and poor patient prognosis. Accordingly, this review focuses on emerging MM precision therapies capable of targeting dysregulated genes within these regions. It addresses gene therapies, small molecule inhibitors and monoclonal antibodies currently under investigation to antagonize oncogenic drivers including <i>MCL-1</i>, <i>BCL9</i>, <i>F11R</i>, and <i>CKS1B</i>, all of which are implicated in cell survival, proliferation or drug resistance. In conclusion, the link between chromosome 1 abnormalities and high-risk disease in MM patients offers a compelling rationale to identify and explore therapeutic targeting of chromosome 1 gene products as a novel precision medicine approach for a poorly served patient population.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"400-410"},"PeriodicalIF":2.3,"publicationDate":"2024-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14352","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142779710","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Pilot Study Assessing the Accuracy of AI ChatGPT Responses for AL Amyloidosis","authors":"Ludovic Saba,&nbsp;Raymond Comenzo,&nbsp;Jack Khouri,&nbsp;Faiz Anwer,&nbsp;Heather Landau,&nbsp;Chakra Chaulagain","doi":"10.1111/ejh.14347","DOIUrl":"https://doi.org/10.1111/ejh.14347","url":null,"abstract":"<div>\u0000 \u0000 <p>AL amyloidosis is a rare, complex and often challenging disease for both patients and healthcare providers. The availability of accurate medical information is crucial for effective diagnosis and management. In recent years, artificial intelligence (AI) has emerged as a potential tool for providing medical information. This study aims to assess the accuracy of AI ChatGPT responses for AL amyloidosis related common questions and compare them to expert opinions. A scoring system was developed to evaluate responses provided by five participating expert physicians. AI ChatGPT demonstrated an overall accuracy rate of 82% in answering AL amyloidosis-related questions. Responses on prognosis and patient support received the highest scores (100%), while questions related to treatment options showed lower accuracy (30%–60%). The results indicate that while the AI ChatGPT demonstrates overall accuracy, there are areas for improvement and potential discrepancies compared to expert opinions. These findings highlight the importance of ongoing refinement and validation of AI-powered medical tools and cannot yet replace the advice of experts in the disease.</p>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"495-499"},"PeriodicalIF":2.3,"publicationDate":"2024-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143253751","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluation of Gemtuzumab Ozogamicin and Anthracycline Dosing for Favorable Risk Acute Myeloid Leukemia","authors":"Joseph F. Mort,&nbsp;David Brighton,&nbsp;Samantha DiBenedetto,&nbsp;Leah Wells,&nbsp;Stephen M. Clark,&nbsp;Justin Reid,&nbsp;Imari Patel,&nbsp;Clayton Jackson,&nbsp;Bradley Yelvington,&nbsp;Ryan Miller,&nbsp;Matthew Perciavalle,&nbsp;Katherine Walsh,&nbsp;Heather Wolfe,&nbsp;Susan C. Locke,&nbsp;Joshua F. Zeidner,&nbsp;Vu H. Duong,&nbsp;Daniel R. Reed,&nbsp;Bhagirathbhai Dholaria,&nbsp;Thomas W. LeBlanc,&nbsp;Michael Keng,&nbsp;Bethany Horton,&nbsp;Firas El Chaer","doi":"10.1111/ejh.14354","DOIUrl":"10.1111/ejh.14354","url":null,"abstract":"<div>\u0000 \u0000 <p>Gemtuzumab ozogamicin (GO) is a CD33-targeting antibody-drug conjugate approved for the treatment of CD33-positive de novo and relapsed and refractory acute myeloid leukemia (AML). Subset analyses have demonstrated improved clinical outcomes in patients with favorable-risk disease. It is unclear whether the addition of GO to cytarabine and anthracycline chemotherapy (7+3) improves clinical outcomes compared with other conventional regimens for AML. We evaluated the real-world experience with GO added to 7+3 chemotherapy for patients with favorable risk AML. This retrospective analysis included 174 patients with de novo favorable risk AML undergoing induction chemotherapy between 2010 and 2020. The primary outcome was overall survival (OS) and secondary outcomes included rates of remission, measurable residual disease (MRD), and toxicity. Eighteen patients received GO, 37 received a high-dose (HD) anthracycline, and 119 received an intermediate-dose anthracycline. Composite complete remission was achieved in 162 patients (93.1%). Among the 54 patients who were assessed for MRD at remission, 66.7% were undetectable. An improvement in OS was seen for patients who received GO and those treated with HD anthracycline, which was better explained by differences in patient performance status and comorbidities. Patients who received GO did not show increased toxicity.</p>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"481-494"},"PeriodicalIF":2.3,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142727412","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Talquetamab in Multiple Myeloma: Efficacy, Safety, and Future Directions","authors":"Caterina Labanca,&nbsp;Enrica Antonia Martino,&nbsp;Ernesto Vigna,&nbsp;Antonella Bruzzese,&nbsp;Francesco Mendicino,&nbsp;Eugenio Lucia,&nbsp;Virginia Olivito,&nbsp;Noemi Puccio,&nbsp;Antonino Neri,&nbsp;Fortunato Morabito,&nbsp;Massimo Gentile","doi":"10.1111/ejh.14353","DOIUrl":"10.1111/ejh.14353","url":null,"abstract":"<p>Relapsed and refractory multiple myeloma (RRMM) remains a challenging condition despite advances in immunotherapies. Novel bispecific antibodies (BsAbs), including talquetamab, have shown promising efficacy in heavily pretreated patients, even those with triple- and penta-refractory disease. Talquetamab, recently approved by the FDA and EMA, is indicated for patients who have progressed after at least three or four prior lines of therapy (LOTs). Administered following a step-up dosing phase to manage cytokine release syndrome (CRS), talquetamab demonstrated a high overall response rate (ORR) of approximately 70%, including in patients previously treated with T-cell redirecting therapies. Its safety profile is consistent with other BsAbs, with hematologic adverse events such as anemia and neutropenia commonly reported, alongside unique on-target off-tumor toxicities like dysgeusia and skin-related events. Infections were less frequent compared to other BsAbs. The optimal sequencing of talquetamab and other therapies, including CAR-T cell treatments, remains an area of active research, as resistance to anti-BCMA therapies presents ongoing clinical challenges. Current trials are exploring the use of talquetamab in combination therapies, as well as therapeutic strategies post-treating progression. The real-world data further support talquetamab's efficacy, making it a valuable addition to the RRMM treatment landscape.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"386-399"},"PeriodicalIF":2.3,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14353","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142738977","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multilevel Analysis of MYC and BCL2 Aberrations in Diffuse Large B-Cell Lymphoma: Identifying a High-Risk Patient Subgroup Across Cell-of-Origin Using Targeted Sequencing","authors":"Gayaththri Vimalathas,&nbsp;Cecilie Steensboe Lang,&nbsp;Tina Marie Green,&nbsp;Michael Boe Møller,&nbsp;Charlotte Guldborg Nyvold,&nbsp;Marcus Høy Hansen,&nbsp;Thomas Stauffer Larsen","doi":"10.1111/ejh.14345","DOIUrl":"10.1111/ejh.14345","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Diffuse large B-cell lymphoma (DLBCL) exhibits striking clinical and biological heterogeneity. Recent studies have identified new subgroups within germinal center B-cell like (GCB) DLBCL, associated with inferior prognosis, irrespective of <i>MYC</i> and <i>BCL2</i> translocations. We explored the existence of such a DLBCL high-risk subgroup, based on multilevel aberrations, especially focusing on <i>MYC</i> and <i>BCL2</i>.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Tissue samples from 111 DLBCL patients were sequenced with a 90-gene lymphoma panel, followed by integrative analyses combining sequencing data, immunohistochemistry, fluorescent in situ hybridization, and clinical data.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>We identified a high-risk subgroup in DLBCL defined by: dual immunohistochemical MYC and BCL2 expression (DEL), concurrent <i>MYC</i> and <i>BCL2</i> translocations (DHL-<i>BCL2</i>), mutations in <i>MYC, CXCR4</i>, or both, and<i>/</i>or <i>BCL2</i> amplification. The high-risk subgroup constituted 41% of the cohort and included DHL-<i>BCL2</i>, DEL, a GCB subgroup likely representing the recently described GCB subgroups, and a subset of non-GCB patients. In multivariate analysis, high-risk features provided independent predictive value from age and IPI. The 5-year overall survival was 36% in high-risk patients, compared to 76% in non-high-risk patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>We identified a distinct high-risk DLBCL subgroup, characterized by <i>MYC</i> and <i>BCL2</i> aberrations, beyond conventional DHL-<i>BCL2</i> and DEL, and irrespective of cell-of-origin, thereby expanding the poor-prognosis group.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"469-480"},"PeriodicalIF":2.3,"publicationDate":"2024-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142675540","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effectiveness of Damoctocog Alfa Pegol to Treat Patients With Hemophilia A Enrolled in the ATHNdataset","authors":"Martin Chandler,&nbsp;Thomas Moulton,&nbsp;Lena Charafi,&nbsp;Jessica Charlet,&nbsp;Michael Recht","doi":"10.1111/ejh.14337","DOIUrl":"10.1111/ejh.14337","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>Health information for 17 109 people living with hemophilia A (PLwHA) is contained within the ATHNdataset. We aimed to evaluate the real-world effectiveness of damoctocog alfa pegol (BAY 94-9027, Jivi®) for hemophilia A.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The ATHNdataset was queried for PLwHA receiving damoctocog alfa pegol between January 1, 2010 and April 30, 2022. Data captured via patient charts were analyzed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>At data cutoff, 205 PLwHA were treated with damoctocog alfa pegol: 150 (73.2%) severe (1 female [0.5%]) and 55 (26.8%) mild/moderate (3 [1.5%] female). In total, 32/205 (25.9%) PLwHA received on-demand treatment; 172 (83.9%) received prophylaxis—161 (93.6%) continuous prophylaxis. Documented bleed rates were available for 187 (91.2%) PLwHA, including those on prophylaxis and on-demand regimens, with 150 (80.2%) treated for &gt; 12 months. Overall annualized bleeding rates and proportion of PLwHA with zero bleeds, receiving prophylaxis during the observation period, were mean (SD) 0.26 (1.03) and 138/157 (87.9%), respectively. No new or recurring inhibitors were reported.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>A low number of bleeds were observed with damoctocog alfa pegol in the real world in both male and female PLwHA. Data should be interpreted with caution owing to limitations of real-world studies and insubstantial data for female PLwHA.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"448-457"},"PeriodicalIF":2.3,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14337","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142667677","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}