European Journal of Haematology最新文献

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Impact of TP53 Mutation Status in Elderly AML Patients When Adding All-Trans Retinoic Acid or Valproic Acid to Decitabine. 在地西他滨基础上加用全反式维甲酸或丙戊酸时TP53突变状态对老年急性髓细胞白血病患者的影响
IF 2.3 3区 医学
European Journal of Haematology Pub Date : 2024-10-13 DOI: 10.1111/ejh.14304
Helena Bresser, Claudia Schmoor, Olga Grishina, Dietmar Pfeifer, Johanna Thomas, Usama-Ur Rehman, Martina Crysandt, Edgar Jost, Felicitas Thol, Michael Heuser, Katharina S Götze, Richard F Schlenk, Helmut R Salih, Marcus M Schittenhelm, Gerhard Heil, Carsten Schwaenen, Carsten Müller-Tidow, Wolfram Brugger, Andrea Kündgen, Maike de Wit, Aristoteles Giagounidis, Sebastian Scholl, Andreas Neubauer, Jürgen Krauter, Gesine Bug, Annette M May, Ralph Wäsch, Justus Duyster, Konstanze Döhner, Arnold Ganser, Hartmut Döhner, Björn Hackanson, Heiko Becker, Michael Lübbert
{"title":"Impact of TP53 Mutation Status in Elderly AML Patients When Adding All-Trans Retinoic Acid or Valproic Acid to Decitabine.","authors":"Helena Bresser, Claudia Schmoor, Olga Grishina, Dietmar Pfeifer, Johanna Thomas, Usama-Ur Rehman, Martina Crysandt, Edgar Jost, Felicitas Thol, Michael Heuser, Katharina S Götze, Richard F Schlenk, Helmut R Salih, Marcus M Schittenhelm, Gerhard Heil, Carsten Schwaenen, Carsten Müller-Tidow, Wolfram Brugger, Andrea Kündgen, Maike de Wit, Aristoteles Giagounidis, Sebastian Scholl, Andreas Neubauer, Jürgen Krauter, Gesine Bug, Annette M May, Ralph Wäsch, Justus Duyster, Konstanze Döhner, Arnold Ganser, Hartmut Döhner, Björn Hackanson, Heiko Becker, Michael Lübbert","doi":"10.1111/ejh.14304","DOIUrl":"https://doi.org/10.1111/ejh.14304","url":null,"abstract":"<p><p>In a randomized phase II trial (AMLSG 14-09, NCT00867672) of elderly, newly diagnosed AML patients, ATRA combined with decitabine (DEC) significantly improved the overall response rate (ORR) and survival also in patients with adverse-risk genetics, without adding toxicity. We performed a post hoc analysis to determine the predictive impact of TP53 status. Despite a nominally higher ORR, the clinically meaningful survival benefit when adding ATRA to DEC was diminished, but not completely negated, in TP53-mutated patients. Indeed, 2 out of 14 TP53-mutated patients (14%) randomized to a DEC + ATRA-containing regimen lived for > 36 months. Further studies of ATRA combined with hypomethylating agents appear warranted in non-M3 AML patients ineligible for HMA/venetoclax therapy. Trial Registration: ClinicalTrials.gov identifier: NCT00867672.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461085","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
Therapy-Related Acute Promyelocytic Leukemia: Case Series and Current Insights 与治疗相关的急性早幼粒细胞白血病:病例系列和当前见解。
IF 2.3 3区 医学
European Journal of Haematology Pub Date : 2024-10-13 DOI: 10.1111/ejh.14327
Antonella Bruzzese, Enrica Antonia Martino, Caterina Labanca, Francesco Mendicino, Eugenio Lucia, Virginia Olivito, Rosellina Morelli, Teresa Rossi, Antonino Neri, Fortunato Morabito, Massimo Gentile, Ernesto Vigna
{"title":"Therapy-Related Acute Promyelocytic Leukemia: Case Series and Current Insights","authors":"Antonella Bruzzese,&nbsp;Enrica Antonia Martino,&nbsp;Caterina Labanca,&nbsp;Francesco Mendicino,&nbsp;Eugenio Lucia,&nbsp;Virginia Olivito,&nbsp;Rosellina Morelli,&nbsp;Teresa Rossi,&nbsp;Antonino Neri,&nbsp;Fortunato Morabito,&nbsp;Massimo Gentile,&nbsp;Ernesto Vigna","doi":"10.1111/ejh.14327","DOIUrl":"10.1111/ejh.14327","url":null,"abstract":"<p>Therapy-related acute promyelocytic leukemia (t-APL) is rare and often linked to previous treatment with alkylating agents or topoisomerase II inhibitors. This report describes three cases of t-APL treated at the Haematology Department of Cosenza Hospital between 2022 and 2024, which occurred after alkylating agents and exemestane, alkylating agents and radiation therapy, alkylating agents, taxane, and checkpoint inhibitor, respectively. Each case was managed with a different therapeutic approach. The first case involved a 71-year-old man with colorectal and breast cancer, who developed low-risk t-APL and achieved complete remission (CR) with ATRA alone. A second 71-year-old man case with colorectal cancer developed high-risk t-APL with PML/RARA and FLT3-ITD fusion transcripts; he achieved CR with idarubicin and ATRA despite severe sepsis and acute heart failure. The third case involved a 74-year-old man with lung squamous cell carcinoma who developed intermediate-risk t-APL following chemoimmunotherapy but unfortunately succumbed to pseudotumor cerebri complications during induction therapy.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"195-198"},"PeriodicalIF":2.3,"publicationDate":"2024-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14327","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461101","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}
引用次数: 0
Biological Findings and Clinical Outcomes in Patients Treated With R-CHOP Plus High-Dose Methotrexate as First-Line Therapy in Large B-Cell Lymphoma With Testis Involvement 以R-CHOP加大剂量甲氨蝶呤作为一线疗法治疗睾丸受累大B细胞淋巴瘤患者的生物学发现和临床疗效
IF 2.3 3区 医学
European Journal of Haematology Pub Date : 2024-10-12 DOI: 10.1111/ejh.14325
R. Liévin, B. Burroni, E. Balducci, P. Palmic, J. Decroocq, B. Deau-Fischer, P. Franchi, M. Vignon, J. Zerbit, A. S. Cottereau, A. Touzart, P. Villarese, S. Kaltenbach, L. Lhermitte, V. Asnafi, D. Bouscary, L. Willems
{"title":"Biological Findings and Clinical Outcomes in Patients Treated With R-CHOP Plus High-Dose Methotrexate as First-Line Therapy in Large B-Cell Lymphoma With Testis Involvement","authors":"R. Liévin,&nbsp;B. Burroni,&nbsp;E. Balducci,&nbsp;P. Palmic,&nbsp;J. Decroocq,&nbsp;B. Deau-Fischer,&nbsp;P. Franchi,&nbsp;M. Vignon,&nbsp;J. Zerbit,&nbsp;A. S. Cottereau,&nbsp;A. Touzart,&nbsp;P. Villarese,&nbsp;S. Kaltenbach,&nbsp;L. Lhermitte,&nbsp;V. Asnafi,&nbsp;D. Bouscary,&nbsp;L. Willems","doi":"10.1111/ejh.14325","DOIUrl":"10.1111/ejh.14325","url":null,"abstract":"<p>Primary testicular lymphoma (PTL) is a rare occurrence of diffuse large B-cell lymphoma (DLBCL) that accounts for 1%–2% of all cases. Nodal DLBCL with testis involvement (DLBCL-T) and PTL are associated with poor prognosis, with high incidence of central nervous system relapse. Fifteen patients (median age 60 years) with PTL (<i>n</i> = 5) or DLBCL-T (<i>n</i> = 10) received high-dose methotrexate + R-CHOP. Overall, complete response (CR) rate was 73% and overall response rate 86%. With a 3.9-year median follow-up, 100% of patients with PTL had CR and none relapsed. On the contrary, 55% of DLBCL-T patients achieved CR among which only one was still in remission at the end of follow-up. Molecular parallels between PTL and Primary CNS Lymphoma (PCNSL) suggest shared origins, urging further research for tailored treatments and enhanced understanding of these lymphomas' biology.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"186-194"},"PeriodicalIF":2.3,"publicationDate":"2024-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14325","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461083","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}
引用次数: 0
Proteomic Profiling of Lymph Nodes Differentiates Classic Hodgkin Lymphoma With and Without Skeletal Involvement 淋巴结蛋白质组学分析区分有无骨骼受累的典型霍奇金淋巴瘤
IF 2.3 3区 医学
European Journal of Haematology Pub Date : 2024-10-11 DOI: 10.1111/ejh.14326
Maja Dam Andersen, Katharina Wolter, Marie Beck Hairing Enemark, Mette Abildgaard Pedersen, Lars Christian Gormsen, Kristina Lystlund Lauridsen, Jørn Starklint, Stephen Jacques Hamilton-Dutoit, Francesco d'Amore, Maja Ludvigsen, Bent Honoré, Peter Kamper
{"title":"Proteomic Profiling of Lymph Nodes Differentiates Classic Hodgkin Lymphoma With and Without Skeletal Involvement","authors":"Maja Dam Andersen,&nbsp;Katharina Wolter,&nbsp;Marie Beck Hairing Enemark,&nbsp;Mette Abildgaard Pedersen,&nbsp;Lars Christian Gormsen,&nbsp;Kristina Lystlund Lauridsen,&nbsp;Jørn Starklint,&nbsp;Stephen Jacques Hamilton-Dutoit,&nbsp;Francesco d'Amore,&nbsp;Maja Ludvigsen,&nbsp;Bent Honoré,&nbsp;Peter Kamper","doi":"10.1111/ejh.14326","DOIUrl":"10.1111/ejh.14326","url":null,"abstract":"<p>Classic Hodgkin lymphoma (CHL) is a highly curable disease, even in advanced stages. Controversy remains over whether bone involvement negatively affects overall and progression-free survival in patients treated with intensive chemotherapy regimens. Whether cases that present with bone lesions harbor specific tumor microenvironmental features is unknown. We investigated protein expression in diagnostic lymph node biopsies from CHL patients with and without skeletal involvement at diagnosis to identify potential markers of skeletal disease. Protein expression patterns in diagnostic formalin-fixed paraffin-embedded lymphoma lymph node samples from CHL patients were analyzed by nano-liquid chromatography–tandem mass spectrometry. Patients were grouped according to skeletal involvement, which was defined as the presence of one or more FDG-avid lesions on a diagnostic FDG-PET/CT scan. Protein profiles identified patients with skeletal disease at diagnosis and showed disrupted cellular pathways, including immune system processes, cell adhesion, and cell growth/survival. Immunohistochemical evaluation also demonstrated differential expressions of angiotensin-converting enzyme (ACE), intercellular adhesion molecule 3 (ICAM3), integrin alpha-X (ITGAX), and calreticulin (CALR). In conclusion, proteomics identified altered protein expression profiles in lymph nodes among CHL cases presenting with disease disseminated to the skeletal system, which implies altered disease pathogenesis for these patients.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"173-185"},"PeriodicalIF":2.3,"publicationDate":"2024-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14326","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461099","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}
引用次数: 0
Adherence and Quality of Life in Adult Patients With Haemophilia A, Haemophilia B and Von Willebrand Disease: A National Cross-Sectional Survey 甲型血友病、乙型血友病和冯-威廉布兰德氏病成年患者的依从性和生活质量:全国横断面调查。
IF 2.3 3区 医学
European Journal of Haematology Pub Date : 2024-10-10 DOI: 10.1111/ejh.14324
Cecilie Maria Lüthje Clausen, Eva Funding, Anders Tolver, Mary Jarden
{"title":"Adherence and Quality of Life in Adult Patients With Haemophilia A, Haemophilia B and Von Willebrand Disease: A National Cross-Sectional Survey","authors":"Cecilie Maria Lüthje Clausen,&nbsp;Eva Funding,&nbsp;Anders Tolver,&nbsp;Mary Jarden","doi":"10.1111/ejh.14324","DOIUrl":"10.1111/ejh.14324","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>New treatments for patients with bleeding disorders (PWB) have emerged, including products with extended half-life and subcutaneous administration. These less frequent treatments can potentially enhance quality of life (QoL), but adherence becomes critically important.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To investigate adherence and QoL among PWB and explore the correlation between treatment adherence and QoL in adult patients with haemophilia A (HA), haemophilia B (HB) and Von Willebrand disease (vWD) in Denmark.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Method</h3>\u0000 \u0000 <p>This survey used disease-specific patient-reported questionnaires: Veritas-PRO and Veritas-PRN to measure adherence, and Haemo-A-QoL and VWD-QoL to assess QoL.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Responses were obtained from 149 patients with HA, 32 with HB and 118 with vWD. Adherence was reported by 87.1% of patients on prophylaxis and 71.2% of patients treated on demand, according to Veritas-PRO and Veritas-PRN cut-off scores. High QoL was generally reported, decreasing with age in HA and HB, but not in vWD.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Danish patients with HA, HB and vWD reported high QoL and high adherence to prescribed treatments. There was no correlation between treatment adherence and QoL among the different patient groups. These findings highlight the need for further research to better understand adherence behaviours and identify opportunities to further improve QoL in PWB.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"164-172"},"PeriodicalIF":2.3,"publicationDate":"2024-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14324","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142399771","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}
引用次数: 0
Real-World Evidence on Prognostic Value of MRD in Multiple Myeloma Using Flow Cytometry 使用流式细胞术对多发性骨髓瘤 MRD 的预后价值进行实证分析。
IF 2.3 3区 医学
European Journal of Haematology Pub Date : 2024-10-10 DOI: 10.1111/ejh.14316
Ludmila Muronova, Ondrej Soucek, David Zihala, Tereza Sevcikova, Tereza Popkova, Hana Plonkova, Ondrej Venglar, Ludek Pour, Martin Stork, Lucie Rihova, Renata Bezdekova, Jiri Minarik, Vojtech Látal, Martin Novak, Alexandra Jungova, Tereza Dekojova, Jan Straub, Martin Spacek, Vladimira Rezacova, Vladimir Maisnar, Jakub Radocha, Roman Hajek, Tomas Jelinek
{"title":"Real-World Evidence on Prognostic Value of MRD in Multiple Myeloma Using Flow Cytometry","authors":"Ludmila Muronova,&nbsp;Ondrej Soucek,&nbsp;David Zihala,&nbsp;Tereza Sevcikova,&nbsp;Tereza Popkova,&nbsp;Hana Plonkova,&nbsp;Ondrej Venglar,&nbsp;Ludek Pour,&nbsp;Martin Stork,&nbsp;Lucie Rihova,&nbsp;Renata Bezdekova,&nbsp;Jiri Minarik,&nbsp;Vojtech Látal,&nbsp;Martin Novak,&nbsp;Alexandra Jungova,&nbsp;Tereza Dekojova,&nbsp;Jan Straub,&nbsp;Martin Spacek,&nbsp;Vladimira Rezacova,&nbsp;Vladimir Maisnar,&nbsp;Jakub Radocha,&nbsp;Roman Hajek,&nbsp;Tomas Jelinek","doi":"10.1111/ejh.14316","DOIUrl":"10.1111/ejh.14316","url":null,"abstract":"<p>Minimal residual disease (MRD) is one of the most important prognostic factors in multiple myeloma (MM) and a valid surrogate for progression-free survival (PFS) and overall survival (OS). Recently, MRD negativity was approved as an early clinical endpoint for accelerated drug approval in MM. Nevertheless, there is limited evidence of MRD utility in real-world setting. In this retrospective multicenter study, we report outcomes of 331 newly diagnosed MM patients with MRD evaluation at Day+100 after autologous stem cell transplantation using flow cytometry with a median limit of detection of 0.001%. MRD negativity was reached in 47% of patients and was associated with significantly prolonged median PFS (49.2 months vs. 18.4 months; hazard ratios (HR) = 0.37; <i>p</i> &lt; 0.001) and OS (not reached vs. 74.9 months; HR = 0.50; <i>p</i> = 0.007). Achieving MRD negativity was associated with PFS improvements regardless of age, International Staging System (ISS) stage, lactate dedydrogenase (LDH) level, or cytogenetic risk. Importantly, MRD positive patients benefited from lenalidomide maintenance versus no maintenance (18-months PFS: 81% vs. 46%; HR = 0.24; <i>p</i> = 0.002) while in MRD negative patients such benefit was not observed (<i>p</i> = 0.747). The outcomes of our real-world study recapitulate results from clinical trials including meta-analyses and support the idea that MRD positive patients profit more from lenalidomide maintenance than MRD negative ones.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"155-163"},"PeriodicalIF":2.3,"publicationDate":"2024-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14316","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142399772","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}
引用次数: 0
Reduced Intensity Conditioning Prior Autologous Stem Cell Transplantation in Elderly DLBCL Patients 老年 DLBCL 患者自体干细胞移植前的降低强度调理。
IF 2.3 3区 医学
European Journal of Haematology Pub Date : 2024-10-09 DOI: 10.1111/ejh.14320
Tim Strüßmann, Philipp Hermes, Gabriele Ihorst, Jürgen Finke, Jesús Duque-Afonso, Monika Engelhardt, Justus Duyster, Reinhard Marks
{"title":"Reduced Intensity Conditioning Prior Autologous Stem Cell Transplantation in Elderly DLBCL Patients","authors":"Tim Strüßmann,&nbsp;Philipp Hermes,&nbsp;Gabriele Ihorst,&nbsp;Jürgen Finke,&nbsp;Jesús Duque-Afonso,&nbsp;Monika Engelhardt,&nbsp;Justus Duyster,&nbsp;Reinhard Marks","doi":"10.1111/ejh.14320","DOIUrl":"10.1111/ejh.14320","url":null,"abstract":"<p>High-dose chemotherapy (HDCT) followed by autologous stem cell transplantation (ASCT) is widely used in patients with diffuse large B-cell lymphoma. HDCT/ASCT is associated with increased morbidity in elderly/unfit patients. We retrospectively evaluated the use of reduced intensity conditioning in DLBCL patients. Our study included 146 patients aged 60 years and older treated at our institution between 2005 and 2019; 86 patients received standard intensity conditioning (SI group) with BEAM or TEAM (BCNU or thiotepa, etoposide, cytarabine, melphalan). Sixty patients received reduced intensity high-dose conditioning (RI group) with BM (BCNU, melphalan, 43.3%), TM (thiotepa, melphalan, 16.7%), BCNU or busulfan thiotepa (38.4%), or bendamustine melphalan (1.7%). Median follow-up was 62.4 months. We observed comparable toxicities in the SI and RI groups. The cumulative incidence of relapse at 3 years was higher in the RI group (30.8% vs. 23.4%, <i>p</i> = 0.034). There was no difference in nonrelapse mortality (NRM). In univariate analyses, SI vs. RI conditioning resulted in superior progression-free survival (PFS) (HR 1.80 CI 1.11–2.92, <i>p</i> = 0.017) but not in superior overall survival (OS) (HR 1.48 CI 0.86–2.56, <i>p</i> = 0.152). On multivariate analysis, we observed no difference in PFS (HR 0.74 CI 0.40–1.38, <i>p</i> = 0.345) and a trend toward better OS with RI conditioning (HR 0.45 CI 0.22–0.94, <i>p</i> = 0.032). Age 60–69 versus ≥ 70 years and remission prior to ASCT were the only factors predicting better PFS. Factors associated with better OS were RI conditioning, age 60–69 versus ≥ 70 years, ECOG 0 versus ≥ 1 performance status, bulky disease, and prior lines 1 versus ≥ 2. In conclusion, RI conditioning prior to ASCT may be feasible in elderly patients and led to a comparable outcome when corrected for several significant confounders.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"139-146"},"PeriodicalIF":2.3,"publicationDate":"2024-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14320","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142389072","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}
引用次数: 0
Leveraging Multi-Omics Approaches and Advanced Technologies to Unravel the Molecular Complexities, Modifiers, and Precision Medicine Strategies for Hemoglobin H Disease 利用多指标方法和先进技术揭示血红蛋白 H 疾病的分子复杂性、修饰因子和精准医疗策略。
IF 2.3 3区 医学
European Journal of Haematology Pub Date : 2024-10-09 DOI: 10.1111/ejh.14319
Akshata Pahelkar, Deep Sharma, Payaam Vohra, Sayli Sawant
{"title":"Leveraging Multi-Omics Approaches and Advanced Technologies to Unravel the Molecular Complexities, Modifiers, and Precision Medicine Strategies for Hemoglobin H Disease","authors":"Akshata Pahelkar,&nbsp;Deep Sharma,&nbsp;Payaam Vohra,&nbsp;Sayli Sawant","doi":"10.1111/ejh.14319","DOIUrl":"10.1111/ejh.14319","url":null,"abstract":"<p>Hemoglobin H (HbH) disease, a form of alpha-thalassemia, poses significant clinical challenges due to its complex molecular underpinnings. It is characterized by reduced synthesis of the alpha-globin chain. The integration of multi-omics and precision medicine holds immense potential to comprehensively understand and capture interactions at the molecular and genetic levels. This review integrates current multi-omics approaches and advanced technologies in HbH research. Furthermore, it delves into detailed pathophysiology and possible therapeutics in the upcoming future. We explore the role of genomics, transcriptomics, proteomics, and metabolomics studies, alongside bioinformatics tools and gene-editing technologies like CRISPR/Cas9, to identify genetic modifiers, decipher molecular pathways, and discover therapeutic targets. Recent advancements are unveiling novel genetic and epigenetic modifiers impacting HbH disease severity, paving the way for personalized precision medicine interventions. The significance of multi-omics research in unraveling the complexities of rare diseases like HbH is underscored, highlighting its potential to revolutionize clinical practice through precision medicine approaches. This paradigm shift can pave the way for a deeper understanding of HbH complexities and improved disease management.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 6","pages":"738-744"},"PeriodicalIF":2.3,"publicationDate":"2024-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14319","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142389070","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}
引用次数: 0
Overall Survival and Treatment Patterns Among Patients With Warm Autoimmune Hemolytic Anemia in Sweden: A Nationwide Population-based Study 瑞典温暖型自身免疫性溶血性贫血患者的总生存期和治疗模式:一项基于全国人口的研究
IF 2.3 3区 医学
European Journal of Haematology Pub Date : 2024-10-09 DOI: 10.1111/ejh.14311
Honar Cherif, Qian Cai, Concetta Crivera, Ann Leon, Iffat Rahman, Amy Leval, Wim Noel, Christian Kjellander
{"title":"Overall Survival and Treatment Patterns Among Patients With Warm Autoimmune Hemolytic Anemia in Sweden: A Nationwide Population-based Study","authors":"Honar Cherif,&nbsp;Qian Cai,&nbsp;Concetta Crivera,&nbsp;Ann Leon,&nbsp;Iffat Rahman,&nbsp;Amy Leval,&nbsp;Wim Noel,&nbsp;Christian Kjellander","doi":"10.1111/ejh.14311","DOIUrl":"10.1111/ejh.14311","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>Warm autoimmune hemolytic anemia (wAIHA) is a rare autoantibody-mediated disorder, and first-line treatment primarily relies on corticosteroids. This study assessed overall survival (OS) and treatment patterns of wAIHA in Sweden.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Adults with ≥ 1 primary diagnosis code for wAIHA (or AIHA plus oral corticosteroids (OCS)/immunosuppressants as sensitivity analyses) between 2011 and 2022 were identified from five Swedish national registers and linked through each patient's unique identity number. Kaplan–Meier curves with log-rank tests and Cox regressions were performed to assess OS for patients with primary versus secondary wAIHA and patients with wAIHA and long-term versus short-term (≥ 3 vs. &lt; 3 months) OCS users.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The main analysis included 292 patients; 1791 patients were included in the sensitivity analysis. At a median 3.7-year follow-up, a median OS in primary wAIHA was not reached versus 6.0 years for secondary wAIHA (log-rank test: <i>p</i> = 0.003). Subgroup analyses showed no significant difference in risk of death between long-term and short-term OCS users; however, in the sensitivity analysis, long-term OCS users showed significantly higher risk of death (adjusted hazard ratio: 1.45; 95% confidence interval: 1.180, 1.781; <i>p</i> &lt; 0.001) versus short-term OCS users.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Secondary wAIHA or long-term OCS use was associated with lower OS, underscoring the disease burden and unmet need for efficacious wAIHA treatments.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"129-138"},"PeriodicalIF":2.3,"publicationDate":"2024-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14311","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142389071","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}
引用次数: 0
Infusion Reactions With Alternative Therapies During the National Shortage of Iron Dextran 全国性右旋糖酐铁短缺期间替代疗法的输液反应。
IF 2.3 3区 医学
European Journal of Haematology Pub Date : 2024-10-09 DOI: 10.1111/ejh.14322
Brigid Jacob, Maria Jamil, Shahm Raslan, Kylie Springer, Zeinab Nasser, Philip Kuriakose
{"title":"Infusion Reactions With Alternative Therapies During the National Shortage of Iron Dextran","authors":"Brigid Jacob,&nbsp;Maria Jamil,&nbsp;Shahm Raslan,&nbsp;Kylie Springer,&nbsp;Zeinab Nasser,&nbsp;Philip Kuriakose","doi":"10.1111/ejh.14322","DOIUrl":"10.1111/ejh.14322","url":null,"abstract":"<div>\u0000 \u0000 <p>Prior to the national shortage of iron dextran in early 2023, it was the most commonly administered intravenous iron infusion at our institution. After the shortage impacted the health system, alternatives such as iron sucrose and sodium ferric gluconate/sucrose were required that utilized lower doses given at more frequent patient visits. Coinciding with their more prevalent use, an increase in iron infusion reactions was observed. Our study analyzed 880 patients who received iron infusions in three Henry Ford Hospital clinics in metropolitan Detroit, Michigan, from July 2022–June 2023. The 74 reactions that occurred were most commonly associated with iron sucrose at the 500 mg dose (41/74, 55.41%, <i>p</i> &lt; 0.0001). Most reactions observed across all iron formulations and doses were mild, with 83.7% being Grade 0 or 1 as defined by the United States Drug Allergy Registry (USDAR) grading scale for immediate reactions. Patients who experienced an infusion reaction were less likely to complete their infusion plans (OR 0.004 for iron dextran, OR 0.128 for iron sucrose, <i>p</i> &lt; 0.0001), with infusions most commonly being completely discontinued thereafter, with a minority pursuing alternative options. More patients with lower number of doses scheduled for iron dextran completed their infusion schedules than those with more doses, but the opposite was seen for iron sucrose. We assessed the impact of the national shortage of iron dextran examining infusion reactions with various iron infusions and doses.</p>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"147-154"},"PeriodicalIF":2.3,"publicationDate":"2024-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142389069","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
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