Acta Clinica Belgica最新文献

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MASH to cirrhosis: bridging the gaps in MASLD management.
IF 1.6 4区 医学
Acta Clinica Belgica Pub Date : 2025-02-24 DOI: 10.1080/17843286.2025.2466011
Zouhir Gadi, Wilhelmus J Kwanten, Luisa Vonghia, Sven M Francque
{"title":"MASH to cirrhosis: bridging the gaps in MASLD management.","authors":"Zouhir Gadi, Wilhelmus J Kwanten, Luisa Vonghia, Sven M Francque","doi":"10.1080/17843286.2025.2466011","DOIUrl":"https://doi.org/10.1080/17843286.2025.2466011","url":null,"abstract":"<p><p>Metabolic dysfunction-associated steatohepatitis (MASH) represents a critical stage in the progression of metabolic dysfunction-associated steatotic liver disease (MASLD), significantly increasing the risk of cirrhosis, hepatocellular carcinoma (HCC), and liver-related mortality. Despite the rising global prevalence of MASLD, gaps in understanding the pathophysiological mechanisms driving MASH to cirrhosis persist, leading to challenges in early diagnosis, prevention, and treatment. This review explores the current knowledge on MASH, focusing on its pathophysiology, clinical management, and treatment strategies in the advanced stages. The role of metabolic dysfunction, portal hypertension, decompensation, and HCC occurrence is highlighted, alongside an evaluation of therapeutic options including lifestyle intervention, bariatric surgery, pharmacological therapies and liver transplantation. Furthermore, we emphasize the need for a multidisciplinary care approach to improve patient outcomes and address the complex metabolic and hepatic interplay in MASLD. Bridging these gaps will require an integrated effort combining advanced diagnostic tools, novel treatments, and comprehensive care strategies.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":" ","pages":"1-10"},"PeriodicalIF":1.6,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143490360","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}
引用次数: 0
Phenotype variability and therapeutic response to Patisiran in patients with hereditary transthyretin amyloidosis: a Belgian real-world experience.
IF 1.6 4区 医学
Acta Clinica Belgica Pub Date : 2025-02-19 DOI: 10.1080/17843286.2025.2464971
Stephanie Delstanche, Kristl G Claeys, De Bleecker, Gauthier Remiche, Pierre Troisfontaines, Vinciane Van Parys, Antoine Bondue
{"title":"Phenotype variability and therapeutic response to Patisiran in patients with hereditary transthyretin amyloidosis: a Belgian real-world experience.","authors":"Stephanie Delstanche, Kristl G Claeys, De Bleecker, Gauthier Remiche, Pierre Troisfontaines, Vinciane Van Parys, Antoine Bondue","doi":"10.1080/17843286.2025.2464971","DOIUrl":"https://doi.org/10.1080/17843286.2025.2464971","url":null,"abstract":"<p><strong>Introduction: </strong>Hereditary transthyretin amyloidosis (hATTRv) is a rare, genetic, adult-onset, multisystemic disorder which can affect diverse organs, including peripheral nerves, heart, kidneys, gastrointestinal tract, liver, skin and eyes. Currently, several disease-modifying treatments for hATTRv are available in Belgium including the TTR stabilizer tafamidis and TTR mRNA silencers patisiran and vutrisiran. Patisiran contains a small interfering RNA encapsulated into a lipid nanoparticle to deliver to hepatocytes, the main source of TTR protein production, thereby reducing TTR production.</p><p><strong>Methods: </strong>We report and discuss five cases of hATTRv in different clinical scenarios that were successfully managed with patisiran, highlighting our real-world clinical practice.</p><p><strong>Results: </strong>These cases illustrate that patisiran is effective to improve mild symptoms and stabilize the moderate ones. The cases also highlight the importance of red flags recognition to allow early diagnosis and treatment to prevent further disease progression.</p><p><strong>Conclusion: </strong>Due to the multisystemic nature of the disease and its heterogeneous clinical presentation, close collaboration between neurologists and cardiologists is highly recommended, ideally within a multidisciplinary amyloidosis team, to provide holistic care in hATTRv patients.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":" ","pages":"1-10"},"PeriodicalIF":1.6,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143456587","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}
引用次数: 0
Suspect the unexpected: proliferative glomerulonephritis with monoclonal immunoglobulin deposits and a negative hematological work-up. A case of monoclonal gammopathy of renal significance.
IF 1.6 4区 医学
Acta Clinica Belgica Pub Date : 2025-02-09 DOI: 10.1080/17843286.2025.2463354
Benoît Delforge, Elien Mahieu, Amélie Dendooven, Michel Delforge, Alexander Salembier, Laurens Claeys, Celine Vanfraechem
{"title":"Suspect the unexpected: proliferative glomerulonephritis with monoclonal immunoglobulin deposits and a negative hematological work-up. A case of monoclonal gammopathy of renal significance.","authors":"Benoît Delforge, Elien Mahieu, Amélie Dendooven, Michel Delforge, Alexander Salembier, Laurens Claeys, Celine Vanfraechem","doi":"10.1080/17843286.2025.2463354","DOIUrl":"https://doi.org/10.1080/17843286.2025.2463354","url":null,"abstract":"<p><strong>Objectives: </strong>Proliferative glomerulonephritis with monoclonal immunoglobulin deposits (PGNMID) is a subtype of monoclonal gammopathy of renal significance (MGRS). PGNMID can present with insidious, slowly progressing kidney damage to overt nephrotic syndrome or rapidly progressive glomerulonephritis. It is a renal-limited disease that often lacks a detectable plasma or B-cell clone and requires kidney biopsy for diagnosis.</p><p><strong>Methods: </strong>We present the case of a 77-year-old woman who developed nephrotic range proteinuria and progressive chronic kidney disease, despite a normal hematological work-up that showed no evidence of monoclonality.</p><p><strong>Results: </strong>This case highlights the potential risk for severe renal damage caused by monoclonal proteins, even in the absence of a detectable pathological hematologic clone.</p><p><strong>Conclusion: </strong>PGNMID requires further research to gain knowledge regarding pathophysiology and potential serum biomarkers for diagnosis as well as therapy response.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":" ","pages":"1-4"},"PeriodicalIF":1.6,"publicationDate":"2025-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143381499","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}
引用次数: 0
Pulmonary vascular complications of cirrhosis: hepatopulmonary syndrome and portopulmonary hypertension.
IF 1.6 4区 医学
Acta Clinica Belgica Pub Date : 2025-01-28 DOI: 10.1080/17843286.2025.2456697
Maïté Verstraeten, Sander Lefere, Sarah Raevens
{"title":"Pulmonary vascular complications of cirrhosis: hepatopulmonary syndrome and portopulmonary hypertension.","authors":"Maïté Verstraeten, Sander Lefere, Sarah Raevens","doi":"10.1080/17843286.2025.2456697","DOIUrl":"https://doi.org/10.1080/17843286.2025.2456697","url":null,"abstract":"<p><p>Hepatopulmonary syndrome (HPS) and portopulmonary hypertension (POPH) are two distinct pulmonary vascular complications seen in patients with liver disease and/or portal hypertension. HPS is characterized by disturbed gas exchange and hypoxemia because of intrapulmonary vascular dilatations. POPH is defined by pulmonary arterial hypertension, which might lead to right heart failure. HPS affects up to 30% of patients with end-stage liver disease requiring liver transplantation. POPH is rarer and affects 1-5% of this patient population. If not recognized and left untreated, these disorders result in significant mortality. This review provides an update on HPS and POPH and discusses their clinical characteristics, screening and diagnostic modalities, and management, including the place of liver transplantation.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":" ","pages":"1-8"},"PeriodicalIF":1.6,"publicationDate":"2025-01-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143051269","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}
引用次数: 0
Organisational quality and hospital pharmacists' roles of outpatient parenteral antimicrobial therapy (OPAT) in Belgian hospitals: a national survey. 组织质量和医院药剂师在比利时医院门诊非肠外抗菌药物治疗(OPAT)中的作用:一项全国性调查。
IF 1.6 4区 医学
Acta Clinica Belgica Pub Date : 2025-01-11 DOI: 10.1080/17843286.2024.2448357
Anneleen Kaes, Franky Buyle, Charlotte Quintens, Astrid Van Eijgen, Larissa Zubryckyj, Koen Boussery, Anna Vanoverschelde
{"title":"Organisational quality and hospital pharmacists' roles of outpatient parenteral antimicrobial therapy (OPAT) in Belgian hospitals: a national survey.","authors":"Anneleen Kaes, Franky Buyle, Charlotte Quintens, Astrid Van Eijgen, Larissa Zubryckyj, Koen Boussery, Anna Vanoverschelde","doi":"10.1080/17843286.2024.2448357","DOIUrl":"https://doi.org/10.1080/17843286.2024.2448357","url":null,"abstract":"<p><strong>Objectives: </strong>Implementation of outpatient parenteral antimicrobial therapy (OPAT), also known as intravenous (IV) antimicrobial treatment at home, has increased in recent years. Ensuring OPAT quality is crucial to achieve positive patient outcomes. However, data on the Belgian quality of OPAT organisation is lacking. We aimed to monitor the organisational quality of OPAT in Belgian hospitals and identify roles of hospital pharmacists involved in OPAT.</p><p><strong>Methods: </strong>A cross-sectional study applying a web-based survey on OPAT quality was conducted from 2 to 29 April 2024. The survey assessed the presence of six core and five non-core structure indicators, and OPAT-related tasks of hospital pharmacists.</p><p><strong>Results: </strong>Almost two-thirds (64%; 65/101) of Belgian hospitals answered the survey, with 77% of these hospitals providing OPAT, with an increase since 2023. All 11 structure indicators were present in 6% of hospitals, while 18% had all six core structure indicators.Three of the six core structure indicators were formally present in the majority of the hospitals: a policy on patient selection criteria (76%), a structured OPAT programme (70%), and a dedicated team (64%). In contrast, a system for fast communication between the patient and OPAT team members (50%), a mechanism for urgent clinical discussions (42%), and monitoring of quality indicators (28%) were not formally present in the majority of the hospitals. The primary tasks for hospital pharmacists included overseeing OPAT prescriptions and supplying antimicrobials and related materials.</p><p><strong>Conclusion: </strong>While the adoption of OPAT is increasing among Belgian hospitals, significant opportunities remain for improving the quality of the OPAT organisation and expanding the OPAT-related tasks of Belgian hospital pharmacists.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":" ","pages":"1-9"},"PeriodicalIF":1.6,"publicationDate":"2025-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142968950","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}
引用次数: 0
A multimodal treatment of basilar artery re-occlusion - case report. 基底动脉再闭塞的多模式治疗--病例报告。
IF 1.6 4区 医学
Acta Clinica Belgica Pub Date : 2024-09-30 DOI: 10.1080/17843286.2024.2409478
Budišić M, Kalousek V, Bosnar Puretić M, Pilepić L, Dežmalj Grbelja L, Lovrenčić Huzjan A, Gavrančić M, Roje Bedeković M
{"title":"A multimodal treatment of basilar artery re-occlusion - case report.","authors":"Budišić M, Kalousek V, Bosnar Puretić M, Pilepić L, Dežmalj Grbelja L, Lovrenčić Huzjan A, Gavrančić M, Roje Bedeković M","doi":"10.1080/17843286.2024.2409478","DOIUrl":"https://doi.org/10.1080/17843286.2024.2409478","url":null,"abstract":"<p><strong>Background: </strong>Stroke due to basilar artery occlusion (BAO) is a severe neurovascular condition with only recently proven effectiveness of mechanical thrombectomy as treatment. Early re-occlusion of the basilar artery (RE-BAO) is an even more challenging form of stroke to treat, associated with poor outcomes and still no optimal treatment guidelines. There are only a few reported cases covering this topic thus far.</p><p><strong>Case presentation: </strong>We present a 52-year-old male patient with RE-BAO treated with a combination of bridging intravenous (IV) tissue plasminogen activator (tPA), mechanical thrombectomy (MT), rescue intraarterial (IA) tPA, and after re-occlusion, repeated bridging IV tPA and repeated MT in a 75-hour time span.</p><p><strong>Discussion: </strong>In previous trials applying IA tPA after MT showed promising results in patients with anterior circulation stroke. However, our case report implies that using a combined treatment of IV tPA before and IA tPA after MT in posterior circulation shows similar results.</p><p><strong>Conclusion: </strong>To our knowledge, this is the first case of RE-BAO managed with the aforementioned multimodal treatment. Such an approach recently showed promising results in the anterior circulation, and our report supports the effectiveness of multimodal recanalization treatment in the posterior circulation as well.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":" ","pages":"1-5"},"PeriodicalIF":1.6,"publicationDate":"2024-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142339028","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}
引用次数: 0
'Iatrogenic septic pit': empyema, a rare complication after endobronchial valve placement. 先天性化脓性凹陷":支气管内瓣膜置入术后的罕见并发症--肺水肿。
IF 1.6 4区 医学
Acta Clinica Belgica Pub Date : 2024-09-22 DOI: 10.1080/17843286.2024.2405754
Charlotte Verhaeghe, Reinier Wener
{"title":"'Iatrogenic septic pit': empyema, a rare complication after endobronchial valve placement.","authors":"Charlotte Verhaeghe, Reinier Wener","doi":"10.1080/17843286.2024.2405754","DOIUrl":"https://doi.org/10.1080/17843286.2024.2405754","url":null,"abstract":"<p><p><b>Objective:</b> Patients with severe emphysema who do not experience relief with non-invasive therapies such as medication and physical activity may need advanced treatments. Bronchoscopic lung volume reduction using endobronchial valves (EBV) is an alternative therapy that may improve exercise capacity and quality of life in carefully selected cases. This treatment is less invasive compared to lung reduction surgery or transplants.<b>Clinical presentation:</b> In this case report, a rarely described complication after EBV insertion is presented: empyema. <b>Conclusion:</b> However EBV has advantages in selected cases, it can be associated with different complications such as pneumothorax, valve migration, and pneumonia.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":" ","pages":"1-3"},"PeriodicalIF":1.6,"publicationDate":"2024-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142278701","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}
引用次数: 0
A case of VEXAS with microcytic anemia: don't be mislead by an associated condition! 一例伴有小细胞性贫血的 VEXAS:不要被相关疾病误导!
IF 1.6 4区 医学
Acta Clinica Belgica Pub Date : 2024-09-16 DOI: 10.1080/17843286.2024.2402131
Kenza Squalli, Louis Wolff, Frédéric Vandergheynst
{"title":"A case of VEXAS with microcytic anemia: don't be mislead by an associated condition!","authors":"Kenza Squalli, Louis Wolff, Frédéric Vandergheynst","doi":"10.1080/17843286.2024.2402131","DOIUrl":"https://doi.org/10.1080/17843286.2024.2402131","url":null,"abstract":"<p><strong>Background: </strong>VEXAS syndrome encompasses a wide range of rheumatological and hematological manifestations, which often features myelodysplastic syndrome accompanied by either macrocytic anemia or macrocytosis.</p><p><strong>Case report: </strong>A 61-year-old Sicilian male was referred for a microcytic anemia associated with skin lesions, recurrent fever, involuntary weight loss, recurrent superficial venous thrombosis, migratory polyarthritis and a lung nodule. A hemoglobin electrophoresis uncovered a minor beta-thalassemia contributing to the anemia in addition to the chronic inflammation and vitamin B9/B12 deficiencies. A bone marrow aspiration demonstrated the presence of vacuoles in erythroid and myeloid precursors, as well as dysplasia in all three lineages. This led us to consider VEXAS syndrome which was confirmed by the presence of UBA1 mutation type p.M41T. Low-dose steroids and sarilumab (200 mg every 3 weeks) therapy led to a transient partial remission.</p><p><strong>Conclusion: </strong>The pivotal insight from this observation centers around the microcytic characteristic of the anemia, with the confounding factor being minor thalassemia, whereas the type of anemia typically associated with VEXAS is macrocytic. This finding may be of particular relevance to patients from regions with endemic thalassemia. Consequently, the presence of microcytic anemia should not hinder clinicians from considering VEXAS syndrome in the appropriate clinical context.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":" ","pages":"1-3"},"PeriodicalIF":1.6,"publicationDate":"2024-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142278700","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}
引用次数: 0
Outcomes of adjuvant immune checkpoint inhibitor therapy in melanoma: a retrospective study. 黑色素瘤辅助免疫检查点抑制剂疗法的疗效:一项回顾性研究。
IF 1.6 4区 医学
Acta Clinica Belgica Pub Date : 2024-09-13 DOI: 10.1080/17843286.2024.2402622
Fieuws Sarah,De Ridder Margot,Baert Valerie,Decruyenaere Alexander,Brochez Lieve,Rottey Sylvie,Jacobs Celine,Saerens Michael
{"title":"Outcomes of adjuvant immune checkpoint inhibitor therapy in melanoma: a retrospective study.","authors":"Fieuws Sarah,De Ridder Margot,Baert Valerie,Decruyenaere Alexander,Brochez Lieve,Rottey Sylvie,Jacobs Celine,Saerens Michael","doi":"10.1080/17843286.2024.2402622","DOIUrl":"https://doi.org/10.1080/17843286.2024.2402622","url":null,"abstract":"BACKGROUNDAdjuvant treatment of malignant melanoma has improved the outcomes for patients. However, real-world data on efficacy and safety are limited. We investigated outcomes of melanoma patients treated with adjuvant immune checkpoint inhibitors (ICI) in the Ghent University Hospital.METHODSPatients with melanoma (stage III-IV), who received at least one cycle of ICI as adjuvant treatment between 2018 and 2021 were included in this retrospective cohort study. Primary outcomes were recurrence-free (RFS) and overall survival (OS). Other outcomes of interest were relapse patterns and safety.RESULTS59 patients were included, with a median follow-up of 36 months. Disease recurrence or death of any cause was observed in 25/59 (42.4%) of the patients. The median RFS was 56.0 months (95%CI 36.1-75.9 months). At 48 months, RFS and OS were 55.9% and 84%, respectively. 9/23 (39%) recurrences were locoregional and 14/23 (60.9%) patients developed distant metastasis as first recurrence, including 2 (3.4%) with brain metastasis. Median time to recurrence was 9 months (range 2-56 months). 35/59 (59.3%) completed one year of adjuvant treatment, 12/59 (20.3%) stopped because of recurrence and 10/59 (16.9% because of toxicity. Immune-related adverse events wereseen in 29/59 (49.4%) patients, 10/59 (16.9%) developed grade 3-4 toxicity.CONCLUSIONThis study confirms the real-world efficacy and safety of adjuvant ICI for melanoma, achieving RFS and OS comparableto the pivotal clinical trials. About 40% of patients develop arelapse, mainly during the adjuvant treatment. The outcomes ofpatients progressing during adjuvant ICI are poor, emphasizing the need of prospective and real-world studies on optimal management after progression on (neo)adjuvant treatment.","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"11 1","pages":"1-9"},"PeriodicalIF":1.6,"publicationDate":"2024-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142253903","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}
引用次数: 0
Single center, real-world retrospective study of CAR-T cell therapy for relapsed/refractory large B-cell lymphoma beyond second line: five-year results at the University Hospitals Leuven. CAR-T细胞疗法治疗复发/难治性大B细胞淋巴瘤二线治疗的单中心真实世界回顾性研究:鲁汶大学医院的五年研究成果。
IF 1.6 4区 医学
Acta Clinica Belgica Pub Date : 2024-09-01
Jan Brijs, Jonas Van Ham, Benedicte Dubois, Franky Sinap, Vibeke Vergote, Daan Dierickx, Peter Vandenberghe
{"title":"Single center, real-world retrospective study of CAR-T cell therapy for relapsed/refractory large B-cell lymphoma beyond second line: five-year results at the University Hospitals Leuven.","authors":"Jan Brijs, Jonas Van Ham, Benedicte Dubois, Franky Sinap, Vibeke Vergote, Daan Dierickx, Peter Vandenberghe","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Introduction: </strong>Large B-cell lymphomas (LBCL) are the most frequently aggressive B-cell non-Hodgkin lymphomas. Anti-CD19 chimeric antigen receptor (CAR)-T cell therapy has emerged as a new, powerful treatment for relapsed or refractory (R/R) disease. Two CAR-T cell products, tisagenlecleucel (tisa-cel,) and axicabtagene ciloleucel (axi-cel), are reimbursed in Belgium for R/R LBCL beyond second line.</p><p><strong>Objectives and methods: </strong>We conducted a retrospective cohort study to report the outcome with tisa-cel and axi-cel for R/R LBCL beyond second line in the years 2019-2023 at the University Hospitals Leuven for 79 patients selected for apheresis and CAR-T infusion.</p><p><strong>Results: </strong>Eleven patients (14%) did not proceed to CAR-T cell infusion. For infused patients (<i>n</i> = 68), the best overall response rate (ORR)/complete response (CR) rate was 64%/49% for tisa-cel and 88%/66% for axi-cel (<i>p</i> = 0.04 for ORR). After a median follow-up of 13.8 months, progression-free survival (PFS) and overall survival (OS) at 1 year were 30% and 43% for tisa-cel and 48% and 62% for axi-cel. Cytokine release syndrome (CRS) (all grades/grade ≥3) occurred in 82%/9% after tisa-cel and in 97%/0% after axi-cel. Immune effector cell-associated neurotoxicity syndrome (ICANS) (all grades/grade ≥3) occurred in 24%/18% after tisa-cel and in 54%/40% after axi-cel. The non-relapse mortality in the infusion cohort was 13%.</p><p><strong>Conclusion: </strong>Our real-world data show high and durable response rates, with a non-significant trend towards a higher efficacy and higher toxicity for axi-cel compared to tisa-cel. Our results are in line with other real-world registries except for a shorter median OS and more high-grade ICANS.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":" ","pages":"1-9"},"PeriodicalIF":1.6,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142278702","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}
引用次数: 0
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