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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 Q2 Medicine 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

Objectives: 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.

Methods: 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.

Results: This case highlights the potential risk for severe renal damage caused by monoclonal proteins, even in the absence of a detectable pathological hematologic clone.

Conclusion: PGNMID requires further research to gain knowledge regarding pathophysiology and potential serum biomarkers for diagnosis as well as therapy response.

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引用次数: 0
Pulmonary vascular complications of cirrhosis: hepatopulmonary syndrome and portopulmonary hypertension.
IF 1.6 4区 医学 Q2 Medicine Pub Date : 2025-01-28 DOI: 10.1080/17843286.2025.2456697
Maïté Verstraeten, Sander Lefere, Sarah Raevens

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.

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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 Q2 Medicine 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

Objectives: 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.

Methods: 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.

Results: 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.

Conclusion: 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.

目的:近年来,门诊静脉外抗菌药物治疗(OPAT),也称为家庭静脉(IV)抗菌药物治疗的实施有所增加。确保OPAT的质量对于获得积极的患者结果至关重要。然而,关于比利时OPAT组织质量的数据缺乏。我们的目的是监测比利时医院OPAT的组织质量,并确定参与OPAT的医院药剂师的角色。方法:采用基于网络的OPAT质量调查方法,于2024年4月2日至29日进行横断面研究。调查评估了6项核心结构指标和5项非核心结构指标的存在情况,以及医院药师与opat相关的任务。结果:几乎三分之二(64%;65/101)的比利时医院回答了调查,其中77%的医院提供OPAT,自2023年以来有所增加。6%的医院拥有全部11项结构指标,18%的医院拥有全部6项核心结构指标。六项核心结构指标中的三项在大多数医院正式存在:关于患者选择标准的政策(76%)、结构化的OPAT方案(70%)和专门的团队(64%)。相比之下,在大多数医院中,患者和OPAT团队成员之间的快速沟通系统(50%)、紧急临床讨论机制(42%)和质量指标监测(28%)都没有正式出现。医院药剂师的主要任务包括监督OPAT处方和提供抗菌剂及相关材料。结论:虽然比利时医院中OPAT的采用正在增加,但仍有很大的机会可以提高OPAT组织的质量,并扩大比利时医院药剂师的OPAT相关任务。
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引用次数: 0
Flemish consensus statement on the prevention, diagnosis and treatment of urinary tract infections in older nursing home residents. 弗拉芒关于预防、诊断和治疗老年疗养院居民尿路感染的共识声明。
IF 1.6 4区 医学 Q2 Medicine Pub Date : 2024-11-05 DOI: 10.1080/17843286.2024.2423120
Jodie Langbeen, Veroniek Saegeman, Laura Heireman, Koen Magerman, Hilde Jansens, Dana Van Kerkhoven, Wouter Dhaeze, Jan De Lepeleire, Katrien Latour, Indira Coenen, Erwin Ho, Dieter Vereecke, Door Jouck, Frederik Van Hoecke, Dirk Vogelaers

Background: Urinary tract infections (UTIs) are one of the most commonly reported infections in Belgian nursing home residents. In older adults, UTI diagnosis and management is complex, often leading to over-diagnosis and irrational antimicrobial use, stressing the need for a guideline approach.

Objectives and methods: A consensus statement on the prevention, diagnosis and treatment of UTIs in older adults residing in nursing homes was developed in a collaborative effort between the Flemish Hospital Outbreak Support Teams, the Flemish Agency for Care and Health, the Association of the Flemish Coordinating and Advising General Practitioners, the Belgian Association of Urology, the Belgian Society for Gerontology and Geriatrics and PhD researchers based on a combination of clinical expertise, (inter)national guidelines and peer-reviewed studies.

Results: Optimizing fluid intake, appropriate toilet behaviour and posture, mobilization and local estrogen therapy in women are of proven value in UTI prevention, whereas the use of cranberry and probiotics is not to be advocated. The importance of avoiding bladder catheterization is stressed. In older nursing home residents, the diagnosis of UTIs remains challenging, mostly due to atypical systemic symptoms. A consensus diagnostic algorithm for UTI among residents with and without a urinary catheter was developed, including the presence of suggestive clinical symptoms and a positive urine culture. Urine dipsticks have a high negative but a low positive predictive value. C-reactive protein point-of-care testing is not recommended. Asymptomatic bacteriuria should not be screened for, in order to avoid unnecessary triggers for treatment. In cystitis, nitrofurantoin is the primary choice for treatment, with fosfomycin as an alternative; in prostatitis and uncomplicated pyelonephritis a fluoroquinolone is the advocated empirical antimicrobial.

背景:尿路感染(UTI)是比利时养老院居民最常报告的感染之一。在老年人中,尿路感染的诊断和治疗非常复杂,常常导致过度诊断和不合理使用抗菌药物,这就强调了制定指导方针的必要性:弗拉芒医院疫情支援小组、弗拉芒护理与健康机构、弗拉芒全科医师协调与咨询协会、比利时泌尿学协会、比利时老年学与老年医学学会以及博士研究人员在综合临床专业知识、(国家间)指南和同行评审研究的基础上,共同制定了一份关于预防、诊断和治疗疗养院老年人尿毒症的共识声明:结果:对女性而言,优化液体摄入量、适当的如厕行为和姿势、活动和局部雌激素疗法对预防UTI具有显著价值,而蔓越莓和益生菌的使用则不值得提倡。强调避免膀胱导尿的重要性。在老年疗养院居民中,UTI 的诊断仍然具有挑战性,主要是由于不典型的全身症状。针对有导尿管和没有导尿管的住院患者,我们制定了尿毒症的共识诊断算法,包括出现提示性临床症状和尿培养阳性。尿液浸滴法的阴性预测值较高,但阳性预测值较低。不建议进行 C 反应蛋白床旁检测。不应筛查无症状菌尿,以免引发不必要的治疗。对于膀胱炎,硝基呋喃妥因是治疗的首选药物,磷霉素可作为替代药物;对于前列腺炎和无并发症的肾盂肾炎,氟喹诺酮类药物是首选的经验性抗菌药物。
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引用次数: 0
Validation of EUCAST rapid antimicrobial susceptibility testing directly from positive blood cultures in a non-automated lab setting. 在非自动化实验室环境中直接从阳性血液培养物进行 EUCAST 快速抗菌药物敏感性测试的验证。
IF 1.6 4区 医学 Q2 Medicine Pub Date : 2024-10-25 DOI: 10.1080/17843286.2024.2421075
Gregory Strubbe, Eveline Van Honacker, Stien Vandendriessche, Anne-Sophie Messiaen, Bruno Verhasselt, Jerina Boelens

Introduction: To speed up antimicrobial susceptibility testing (AST), the European Committee on Antimicrobial Susceptibility Testing (EUCAST) proposed rapid AST (RAST), a disk diffusion method to be read after 4, 6 and 8 hours of incubation. We investigated the feasibility of implementation of RAST in a non-automated lab setting.

Materials & methods: To this end, reference strains as well as a variety of clinical and resistant strains were used to spike sterile hemocultures (BioMérieux BACT/ALERT 3D® and Becton Dickinson BACTEC FX® systems), followed by RAST in comparison to classical long-incubation AST.

Results & conclusion: Our results with reference strains show that reading RAST after 4 hours is frequently too soon to obtain clinical results, and that Streptococcus pneumoniae reference strain did yield readable inhibition zones in RAST when harvested from BioMérieux BACT/ALERT 3D® bottle cultures. In a wider panel of strains, Gram positives RAST results were very similar to standard AST, while with Gram negative species errors were more frequently observed, limiting clinical implementation.

简介:为了加快抗菌药物药敏试验(AST)的速度,欧洲抗菌药物药敏试验委员会(EUCAST)提出了快速抗菌药物药敏试验(RAST),这是一种在培养 4、6 和 8 小时后读取结果的磁盘扩散法。我们研究了在非自动化实验室环境中实施 RAST 的可行性:为此,我们使用参考菌株以及各种临床菌株和耐药菌株进行无菌血液培养(生物梅里埃 BACT/ALERT 3D® 和 Becton Dickinson BACTEC FX® 系统),然后使用 RAST 与传统的长孵育 AST 进行比较:我们对参考菌株的研究结果表明,4 小时后读取 RAST 往往太快,无法获得临床结果,而从生物梅里埃 BACT/ALERT 3D® 瓶培养物中提取的肺炎链球菌参考菌株在 RAST 中确实产生了可读取的抑制区。在更广泛的菌株中,革兰氏阳性菌的 RAST 结果与标准 AST 非常相似,而革兰氏阴性菌则经常出现误差,从而限制了临床应用。
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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 Q2 Medicine 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

Background: 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.

Case presentation: 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.

Discussion: 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.

Conclusion: 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.

背景:基底动脉闭塞(BAO)导致的中风是一种严重的神经血管疾病,机械性血栓切除术的治疗效果最近才得到证实。基底动脉早期再闭塞(RE-BAO)是一种治疗难度更大的卒中形式,预后差,至今仍无最佳治疗指南。迄今为止,关于这一主题的病例报道寥寥无几:我们介绍了一名 52 岁男性 RE-BAO 患者,他接受了桥接静脉注射组织纤溶酶原激活剂(tPA)、机械取栓术(MT)、挽救性动脉内 tPA 以及再闭塞后在 75 小时内重复桥接静脉注射 tPA 和重复 MT 的综合治疗:讨论:在之前的试验中,前循环卒中患者在 MT 后应用 IA tPA 取得了良好的效果。然而,我们的病例报告表明,在后循环患者中,在 MT 之前使用 IV tPA 和 MT 之后使用 IA tPA 的联合治疗也能取得类似的效果:据我们所知,这是首例采用上述多模式治疗的 RE-BAO 病例。据我们所知,这是第一例采用上述多模式治疗的 RE-BAO 病例。这种方法最近在前循环中取得了良好的效果,而我们的报告支持了多模式再通化治疗在后循环中的有效性。
{"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 Q2 Medicine Pub Date : 2024-09-22 DOI: 10.1080/17843286.2024.2405754
Charlotte Verhaeghe, Reinier Wener

Objective: 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.Clinical presentation: In this case report, a rarely described complication after EBV insertion is presented: empyema. Conclusion: However EBV has advantages in selected cases, it can be associated with different complications such as pneumothorax, valve migration, and pneumonia.

目的:严重肺气肿患者在接受药物治疗和体育锻炼等非侵入性疗法后病情仍未缓解,可能需要接受先进的治疗。使用支气管内瓣膜(EBV)进行支气管镜肺容积缩小术是一种替代疗法,在经过仔细筛选的病例中,它可以改善运动能力和生活质量。与肺减容手术或移植手术相比,这种治疗方法创伤较小:在本病例报告中,介绍了插入 EBV 后出现的一种罕见并发症:肺水肿。临床表现:本病例报告了插入 EBV 后出现的罕见并发症:肺水肿:尽管 EBV 在经过选择的病例中具有优势,但它也可能伴有不同的并发症,如气胸、瓣膜移位和肺炎。
{"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
The first reported familial case of statin-induced immune-mediated necrotizing myopathy associated with anti-hydroxy-3-methylglutaryl-CoA reductase autoantibodies and HLA DRB1*11:01 首例报道的他汀类药物诱导的免疫介导坏死性肌病家族病例,伴有抗羟基-3-甲基戊二酰-CoA 还原酶自身抗体和 HLA DRB1*11:01
IF 1.6 4区 医学 Q2 Medicine Pub Date : 2024-09-16 DOI: 10.1080/17843286.2024.2403694
M. Bronchain, L. Pothen, L. Taihi, A. Cordenier, V. Van Parys
Immune-mediated necrotizing myopathy (IMNM) is a rare type of auto-immune myositis, characterized by symmetric muscle pain, proximal weakness, elevated serum CK levels and pathologic findings of ne...
免疫介导的坏死性肌病(IMNM)是一种罕见的自身免疫性肌炎,其特征是对称性肌肉疼痛、近端无力、血清 CK 水平升高以及病理发现的新...
{"title":"The first reported familial case of statin-induced immune-mediated necrotizing myopathy associated with anti-hydroxy-3-methylglutaryl-CoA reductase autoantibodies and HLA DRB1*11:01","authors":"M. Bronchain, L. Pothen, L. Taihi, A. Cordenier, V. Van Parys","doi":"10.1080/17843286.2024.2403694","DOIUrl":"https://doi.org/10.1080/17843286.2024.2403694","url":null,"abstract":"Immune-mediated necrotizing myopathy (IMNM) is a rare type of auto-immune myositis, characterized by symmetric muscle pain, proximal weakness, elevated serum CK levels and pathologic findings of ne...","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"41 1","pages":""},"PeriodicalIF":1.6,"publicationDate":"2024-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142253902","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 Q2 Medicine Pub Date : 2024-09-16 DOI: 10.1080/17843286.2024.2402131
Kenza Squalli, Louis Wolff, Frédéric Vandergheynst

Background: VEXAS syndrome encompasses a wide range of rheumatological and hematological manifestations, which often features myelodysplastic syndrome accompanied by either macrocytic anemia or macrocytosis.

Case report: 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.

Conclusion: 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.

背景:VEXAS综合征包括多种风湿病和血液病表现,通常以骨髓增生异常综合征为特征,伴有巨幼红细胞性贫血或巨幼红细胞增多症:病例报告:一名 61 岁的西西里岛男性因微量红细胞性贫血伴有皮肤损伤、反复发热、不自主体重减轻、反复浅静脉血栓形成、移行性多关节炎和肺部结节而转诊。血红蛋白电泳结果显示,除了慢性炎症和维生素 B9/B12 缺乏外,轻微的 beta 型地中海贫血也是导致贫血的原因之一。骨髓穿刺显示,红细胞和髓细胞前体中存在空泡,所有三个系都出现发育不良。这促使我们考虑 VEXAS 综合征,并通过 UBA1 基因突变 p.M41T 证实了这一点。小剂量类固醇和沙利单抗(200 毫克,每 3 周一次)治疗后,病情得到了短暂的部分缓解:这一观察结果的关键之处在于贫血的小红细胞特性,其混杂因素是轻微地中海贫血,而 VEXAS 典型的贫血类型是大红细胞性贫血。这一发现可能与地中海贫血流行地区的患者特别相关。因此,小细胞性贫血的存在不应妨碍临床医生在适当的临床背景下考虑 VEXAS 综合征。
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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 Q2 Medicine 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
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.
背景恶性黑色素瘤的辅助治疗改善了患者的预后。然而,有关疗效和安全性的实际数据却很有限。我们调查了根特大学医院接受免疫检查点抑制剂(ICI)辅助治疗的黑色素瘤患者的预后情况。方法这项回顾性队列研究纳入了2018年至2021年间接受至少一个周期ICI辅助治疗的黑色素瘤患者(III-IV期)。主要结果为无复发(RFS)和总生存率(OS)。其他相关结果为复发模式和安全性。结果共纳入59例患者,中位随访时间为36个月。25/59(42.4%)名患者因任何原因导致疾病复发或死亡。中位 RFS 为 56.0 个月(95%CI 36.1-75.9 个月)。48 个月时,RFS 和 OS 分别为 55.9% 和 84%。9/23(39%)例复发为局部复发,14/23(60.9%)例患者首次复发为远处转移,其中2例(3.4%)为脑转移。中位复发时间为9个月(2-56个月)。35/59(59.3%)名患者完成了一年的辅助治疗,12/59(20.3%)名患者因复发而停止治疗,10/59(16.9%)名患者因毒性而停止治疗。29/59(49.4%)名患者出现了免疫相关不良事件,10/59(16.9%)名患者出现了 3-4 级毒性。结论这项研究证实了 ICI 辅助治疗黑色素瘤的实际疗效和安全性,其 RFS 和 OS 达到了关键临床试验的水平。约40%的患者会出现病情进展,主要是在辅助治疗期间。在 ICI 辅助治疗期间病情进展的患者疗效不佳,因此需要对(新)辅助治疗进展后的最佳治疗方法进行前瞻性和真实世界研究。
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引用次数: 0
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