European Journal of Haematology最新文献

TP53 Mutation Is the Only Robust Mutational Biomarker for Outcome Found in a Consecutive Clinical Cohort of Real-Word Patients With Primary Large B-Cell Lymphoma.

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2024-12-18 DOI: 10.1111/ejh.14364

Marie Fredslund Breinholt, Lone Schejbel, Anne Ortved Gang, Ib Jarle Christensen, Torsten Holm Nielsen, Lars Møller Pedersen, Estrid Høgdall, Peter Nørgaard

{"title":"TP53 Mutation Is the Only Robust Mutational Biomarker for Outcome Found in a Consecutive Clinical Cohort of Real-Word Patients With Primary Large B-Cell Lymphoma.","authors":"Marie Fredslund Breinholt, Lone Schejbel, Anne Ortved Gang, Ib Jarle Christensen, Torsten Holm Nielsen, Lars Møller Pedersen, Estrid Høgdall, Peter Nørgaard","doi":"10.1111/ejh.14364","DOIUrl":"https://doi.org/10.1111/ejh.14364","url":null,"abstract":"Introduction: Large B-cell lymphoma (LBCL) taxonomy has moved in the direction of a molecular classification, but further clinical experience is needed. We present high-risk gene mutations, which predict outcome in an exploratory study of a consecutive real-world cohort of patients with primary LBCL treated with R-CHOP or R-CHOP-like therapy.Methods: The study was a Registry Study Research Project. Sixty-one patients with LBCL, who had a diagnostic tumor sample successfully examined with a 59-gene next-generation sequencing (NGS) panel as a part of routine clinical work, were included in an otherwise unselected cohort. Data were extracted from patient files and pathology reports.Results: Mutations in NOTCH2 (HR 9.69; 95% CI [2.46-38.11]; p = 0.0012), PRDM1 (HR 3.54; 95% CI [1.03-12.22]; p = 0.045), and TP53 (HR 5.89; 95% CI [1.71-20.32]; p = 0.005) were significantly associated with inferior survival in patients with primary LBCL treated with intention to cure with at least three series of R-CHOP or R-CHOP-like therapy. Neither MYD88 (HR 0.66; 95% CI (0.17-2.49), p = 0.54) nor CD79B (HR 0.84;95% CI (0.18-3.88), p = 0.82) mutations were associated with inferior survival.Conclusion: With a targeted gene panel and NGS methodology feasible in daily diagnostic routine, we identified high-risk gene mutations with a significant prognostic impact of which TP53 mutations were reproducible across validation cohorts.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142846101","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}

引用次数: 0

Direct Oral Anticoagulants in Budd-Chiari Syndrome.

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2024-12-17 DOI: 10.1111/ejh.14363

Shrinjaya B Thapa, Gabriel Roman Souza, Mahati Paravathaneni, Sean Cohen, Turab Mohammed, Damian A Laber

{"title":"Direct Oral Anticoagulants in Budd-Chiari Syndrome.","authors":"Shrinjaya B Thapa, Gabriel Roman Souza, Mahati Paravathaneni, Sean Cohen, Turab Mohammed, Damian A Laber","doi":"10.1111/ejh.14363","DOIUrl":"https://doi.org/10.1111/ejh.14363","url":null,"abstract":"Aims: Budd-Chiari syndrome (BCS) is managed by interventions aimed at relieving hepatic venous obstruction and anticoagulation. Despite robust data supporting the tolerability and efficacy of direct oral anticoagulants (DOACs) in patients with other venous thromboembolism, its utility in BCS is not well documented. This study aims to evaluate the efficacy and tolerability of DOACs in Primary BCS from the available literature.Methods: Published studies that reported data on patients with BCS treated with DOACs were included.Results: Two retrospective studies and nine case reports met the criteria for inclusion. The combined data from these two retrospective studies include 58 patients administered DOAC and 101 patients treated with VKA/LMWH. The combined re-stenosis or failure rates after percutaneous endovascular intervention, angioplasty, TIPS, or OLT were 17.2% for the DOAC group and 15.8% for the LMWH/VKA group. The incidence of major bleeding was 8.62% in the DOAC group and 5.94% in the LMWH/VKA group, while minor bleeding rates were 20.7% and 4.95%, respectively. Procedure-related bleeding was 4.5% in DOAC group and 12.8% in VKA/LMWH group. Nine case reports using apixaban in 3, rivaroxaban in 5, and one with dabigatran- described patients tolerating the treatment well and experiencing no major adverse events.Conclusions: DOACs appear to be at least equally effective to LMWH/VKA for the anticoagulation of patients with BCS. We believe DOACs to be preferred over LMWH/VKA for the anticoagulation of patients with BCS due to the known advantages in administration, but randomized trials might be needed to answer this question.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-12-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142834550","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}

引用次数: 0

BAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A: Results From the LEOPOLD Kids Extension Study.

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2024-12-12 DOI: 10.1111/ejh.14362

Rolf Ljung, Anthony K C Chan, Sanjay P Ahuja, Maria Elisa Mancuso, Jose Francisco Cabre Marquez, Florian Volk, Victor Blanchette, Bryce A Kerlin, Sonata Saulyte Trakymiene, Heidi Glosli, Gili Kenet

{"title":"BAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A: Results From the LEOPOLD Kids Extension Study.","authors":"Rolf Ljung, Anthony K C Chan, Sanjay P Ahuja, Maria Elisa Mancuso, Jose Francisco Cabre Marquez, Florian Volk, Victor Blanchette, Bryce A Kerlin, Sonata Saulyte Trakymiene, Heidi Glosli, Gili Kenet","doi":"10.1111/ejh.14362","DOIUrl":"https://doi.org/10.1111/ejh.14362","url":null,"abstract":"Objectives: To report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase.Methods: Patients received BAY 81-8973 (25-50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82). Efficacy endpoints were assessed in patients without high-titre inhibitors/immune tolerance induction (n = 67).Results: Children (n = 82) received BAY 81-8973 for a median of 3.1 years per patient and a median of 405 exposure days per patient. Long-term BAY 81-8973 treatment was well tolerated, with no cases of de novo inhibitor development in the extension phase. Annualised bleeding rates (ABRs) within 48 h of prophylaxis were low for all bleeds (median [IQR], 0.7 [0-1.9]; mean, 1.4 [SD, 2.1]) and for joint bleeds (median [IQR], 0 [0-0.7]; mean, 0.5 [SD, 1.1]) (n = 67). Twenty-one of 67 patients (31.3%) had zero bleeds within 48 h of prophylaxis; the treatment response was 'good'/'excellent' in 87.9% of bleeds, and most bleeds resolved with ≤ 2 BAY 81-8973 infusions (83.5%).Conclusion: Long-term BAY 81-8973 treatment is well tolerated and maintains low ABRs for all bleeds and joint bleeds in children with severe haemophilia A.Trial registration: ClinicalTrials.gov identifier: NCT01311648.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142817319","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}

引用次数: 0

The Effect of the Pre-Transplant Disease Status on the Outcome for Recipients of T-Cell Depleted Allogeneic Haematopoietic Stem Cell Transplants for Large B Cell Lymphomas.

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2024-12-11 DOI: 10.1111/ejh.14361

Maria A V Marzolini, Irfan Kayani, Ben Carpenter, Arian Laurence, Donal McLornan, Kavita Raj, Maeve O'Reilly, Claire Roddie, Kate Stringaris, Panagiotis Kottaridis, Emma C Morris, Kirsty J Thomson, Karl S Peggs

{"title":"The Effect of the Pre-Transplant Disease Status on the Outcome for Recipients of T-Cell Depleted Allogeneic Haematopoietic Stem Cell Transplants for Large B Cell Lymphomas.","authors":"Maria A V Marzolini, Irfan Kayani, Ben Carpenter, Arian Laurence, Donal McLornan, Kavita Raj, Maeve O'Reilly, Claire Roddie, Kate Stringaris, Panagiotis Kottaridis, Emma C Morris, Kirsty J Thomson, Karl S Peggs","doi":"10.1111/ejh.14361","DOIUrl":"https://doi.org/10.1111/ejh.14361","url":null,"abstract":"Objectives: Deauville scores (DS) from PET/CT imaging are increasingly being used to direct response-adjusted treatment strategies in lymphoma, including large B cell lymphomas (LBCL). We aimed to investigate the outcome of allogeneic haematopoietic stem cell transplantation (alloHSCT) in LBCL and the role played by pre-transplant disease status, as determined by DS.Methods: We performed a retrospective, observational study of adults treated with a T-cell depleted alloHSCT for de novo DLBCL or high-grade transformation.Results: Sixty-four patients received an alloHSCT. Forty-four had acute GvHD (38 had Grade 1-2). Overall non-relapse mortality (NRM) at 1 year was 20.31%. Patients ≥ 55 years had a higher cumulative incidence of NRM (66.67%) than those who were < 55 years (25.08%) (p = 0.00660). A 4-year relapse risk was 22.5%. Fourteen patients had disease relapse. The 4-year overall survival (OS) was 49.80%; median OS was 3.7 years (1.4-7.1). Patients with a pre-alloHSCT DS of 1-2 had a higher OS than a DS of 3-5 (61.97% vs. 34.23%; p = 0.0167); this was confirmed on multivariate analysis. Younger patients (< 55 years) had a higher OS than those ≥ 55 years (60.91% vs. 18.75%; p = 0.0246).Conclusions: The pre-transplant Deauville score was predictive of the clinical outcome and patients with an absence of metabolically active disease pre-transplant had superior outcomes.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142812313","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}

引用次数: 0

Secondary Malignancies After Autologous Stem Cell Transplantations in Patients With Malignant Lymphoma and Multiple Myeloma.

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2024-12-10 DOI: 10.1111/ejh.14355

Bernd Metzner, Thomas H Müller, Jochen Casper, Christoph Kimmich, Eduard K Petershofen, Ruth Thole, Andreas Voß, Claus Henning Köhne

{"title":"Secondary Malignancies After Autologous Stem Cell Transplantations in Patients With Malignant Lymphoma and Multiple Myeloma.","authors":"Bernd Metzner, Thomas H Müller, Jochen Casper, Christoph Kimmich, Eduard K Petershofen, Ruth Thole, Andreas Voß, Claus Henning Köhne","doi":"10.1111/ejh.14355","DOIUrl":"https://doi.org/10.1111/ejh.14355","url":null,"abstract":"Background: Secondary malignancies are potential complications after high-dose therapy and autologous stem cell transplantation (ASCT). Information on the detailed course of such events is scarce, yet may be essential to minimize the impact of these sequelae.Patients and methods: We regularly monitored 877 patients for up to 31 years after ASCT in our outpatient department.Results: Four-hundred and five patients with malignant lymphoma and 472 patients with multiple myeloma were analysed with a median follow-up from the initial malignant diagnosis of 7.0 years (0.5-35.5), from ASCT of 4.8 years (range 0.2-31.3). Eighty-nine of these patients suffered from 94 invasive secondary malignancies (30 haematologic malignancies, 64 solid tumours). In 29 patients a non-melanoma skin cancer was observed. 31/64 (48%) patients with solid tumours were diagnosed in an early stage of the disease (UICC 0-2) and had a chance of cure. 5% of the patients developed an invasive malignancy within 5 years, 12% within 10 years, 21% within 20 years. Risk factors were advanced age, the diagnosis lymphoma and in lymphoma patients additionally male sex.Conclusions: Secondary malignancies after ASCT are relatively frequent complications. Our findings suggest that systematic life-long cancer screening after ASCT is essential to improve the prognosis of these adverse events.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142799600","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}

引用次数: 0

ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B.

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2024-12-05 DOI: 10.1111/ejh.14357

Fabienne Volot, Sabine Castet, Alexandra Fournel, Birgit Frotscher, Benjamin Gillet, Dominique Desprez, Brigitte Tardy, Antoine Rauch, Pierre Chamouni, Christine Biron-Andreani, Jean-Baptiste Valentin, Annie Harroche, Yesim Dargaud, Brigitte Pan Petesch, Roseline d'Oiron, Claire Berger, Claire Reynes, Thomas Lauvray, Emmanuelle de Raucourt, Abel Hassoun, Aurélien Lebreton, Vincent Cussac, Hasan Catovic, Cédric Martin, Benoit Guillet

{"title":"ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B.","authors":"Fabienne Volot, Sabine Castet, Alexandra Fournel, Birgit Frotscher, Benjamin Gillet, Dominique Desprez, Brigitte Tardy, Antoine Rauch, Pierre Chamouni, Christine Biron-Andreani, Jean-Baptiste Valentin, Annie Harroche, Yesim Dargaud, Brigitte Pan Petesch, Roseline d'Oiron, Claire Berger, Claire Reynes, Thomas Lauvray, Emmanuelle de Raucourt, Abel Hassoun, Aurélien Lebreton, Vincent Cussac, Hasan Catovic, Cédric Martin, Benoit Guillet","doi":"10.1111/ejh.14357","DOIUrl":"https://doi.org/10.1111/ejh.14357","url":null,"abstract":"Objectives: 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.Methods: 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.Results: 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) (n = 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.Conclusion: This analysis confirmed that switching to rIX-FP allows for reducing injection frequency and FIX consumption while maintaining good bleed protection.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Venetoclax in the Treatment of Multiple Myeloma: A Retrospective Analysis of 79 Patients.

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2024-12-05 DOI: 10.1111/ejh.14343

Eli Zolotov, Vanisha Patel, Yedidyah Weiss, Noa Biran, Pooja Phull, David H Vesole, David S Siegel, Harsh Parmar

{"title":"Venetoclax in the Treatment of Multiple Myeloma: A Retrospective Analysis of 79 Patients.","authors":"Eli Zolotov, Vanisha Patel, Yedidyah Weiss, Noa Biran, Pooja Phull, David H Vesole, David S Siegel, Harsh Parmar","doi":"10.1111/ejh.14343","DOIUrl":"https://doi.org/10.1111/ejh.14343","url":null,"abstract":"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, p < 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.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"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}

引用次数: 0

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2024-12-05 DOI: 10.1111/ejh.14358

Tine Rosenberg, Sören Möller, Niels Abildgaard, Jakob Nordberg Nørgaard, Anna Lysén, Galina Tsykonova, Cristina Joao, Annette Vangsted, Fredrik Schjesvold, Lene Kongsgaard Nielsen

{"title":"Health-Related Quality of Life During Carfilzomib-Lenalidomide-Dexamethasone Consolidation: Findings From the Multiple Myeloma CONPET Study.","authors":"Tine Rosenberg, Sören Möller, Niels Abildgaard, Jakob Nordberg Nørgaard, Anna Lysén, Galina Tsykonova, Cristina Joao, Annette Vangsted, Fredrik Schjesvold, Lene Kongsgaard Nielsen","doi":"10.1111/ejh.14358","DOIUrl":"https://doi.org/10.1111/ejh.14358","url":null,"abstract":"Background: 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.Methods: 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 or 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.Results: 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 (p-value 0.048). Compared with baseline, patients reported significant, and clinically relevant worsening in dyspnea during consolidation.Conclusion: Our study confirmed earlier findings of carfilzomib causing shortness of breath during KRd administration and revealed dyspnea during consolidation compared to baseline.Trial registration: Clinicaltrials.gov: NCT03314636, EudraCT: 2017-000586-72.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"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}

引用次数: 0

The Prevalence of Diffuse Large B-Cell Lymphoma Varies Substantially by Methods Applied: Results From a Population-Based Study.

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2024-12-05 DOI: 10.1111/ejh.14359

Mikkel Runason Simonsen, Jonas Faartoft Jensen, Thomas Stauffer Larsen, Inger-Lise Gade, Peter de Nully Brown, Tarec Christoffer El-Galaly

{"title":"The Prevalence of Diffuse Large B-Cell Lymphoma Varies Substantially by Methods Applied: Results From a Population-Based Study.","authors":"Mikkel Runason Simonsen, Jonas Faartoft Jensen, Thomas Stauffer Larsen, Inger-Lise Gade, Peter de Nully Brown, Tarec Christoffer El-Galaly","doi":"10.1111/ejh.14359","DOIUrl":"https://doi.org/10.1111/ejh.14359","url":null,"abstract":"Objectives: 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.Methods: In the present nationwide Danish cohort study, the prevalence of DLBCL was assessed using different methodologies, including register-based and formula-based approaches.Results: 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.Conclusions: 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.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"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}

引用次数: 0

Chromosome 1 Alterations in Multiple Myeloma: Considerations for Precision Therapy.

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2024-12-04 DOI: 10.1111/ejh.14352

Niamh McAuley, Izabela Cymer, Roisin McAvera, Ann M Hopkins, Siobhan V Glavey

{"title":"Chromosome 1 Alterations in Multiple Myeloma: Considerations for Precision Therapy.","authors":"Niamh McAuley, Izabela Cymer, Roisin McAvera, Ann M Hopkins, Siobhan V Glavey","doi":"10.1111/ejh.14352","DOIUrl":"https://doi.org/10.1111/ejh.14352","url":null,"abstract":"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 MCL-1, BCL9, F11R, and CKS1B, 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.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0