Objectives: Ferric carboxymaltose (FCM) is increasingly used in the management of cancer-related anemia, yet it may cause hypophosphatemia. This retrospective study describes the incidence, evolution and risk factors of hypophosphatemia in a cohort of patients with solid tumors receiving FCM.
Methods: Serum phosphorus concentration was assessed longitudinally using a random intercepts model. The probability of developing hypophosphatemia, as graded by CTCAE version 4.0, was investigated using a multi-state model. Transition hazards were modeled non-parametrically and semi-parametrically by a Cox model. Causal marginal risk differences between baseline interventions on serum phosphorus and/or FCM dose were obtained via G-computation.
Results: In 174 ambulatory patients with solid tumors receiving FCM at two university hospitals between October 2020 and September 2021, the risk of developing moderate-to-severe hypophosphatemia was 36.0% (95% confidence interval (CI) 28.2-43.9%) and peaked within 16 days after first FCM administration. The average duration of moderate-to-severe hypophosphatemia was 12.4 days. After adjustment for confounders, lower baseline serum phosphorus (adjusted hazard ratio (aHR) 0.88 per 0.1 mmol/L increase, 95% CI 0.79-0.98) and higher FCM dose (first dose: aHR 1.12 per 1 mg/kg increase, 95% CI 1.01-1.25; second dose: aHR 1.06 per 1 mg/kg increase, 95% CI 1.00-1.13) significantly increased the hazard of moderate-to-severe hypophosphatemia.
Conclusion: Approximately one out of three ambulatory patients with solid tumors may develop moderate-to-severe hypophosphatemia after FCM administration. Baseline serum phosphorus and FCM dose may be modifiable risk factors that should be considered for intervention in order to mitigate the risk of hypophosphatemia.
目的:羧麦芽糖铁(FCM)越来越多地用于癌症相关性贫血的治疗，但它可能导致低磷血症。本回顾性研究描述了接受FCM治疗的实体肿瘤患者低磷血症的发生率、演变和危险因素。方法:采用随机截距法纵向测定血清磷浓度。采用多状态模型研究CTCAE 4.0分级的低磷血症发生概率。采用Cox模型对过渡风险进行非参数化和半参数化建模。基线干预对血清磷和/或FCM剂量的因果边际风险差异通过g计算得到。结果:在2020年10月至2021年9月期间，在两所大学医院接受FCM治疗的174名实体瘤门诊患者中，发生中至重度低磷血症的风险为36.0%(95%置信区间(CI) 28.2-43.9%)，并在首次FCM治疗后16天内达到高峰。中度至重度低磷血症的平均持续时间为12.4天。校正混杂因素后，较低的基线血清磷(校正危险比(aHR)每增加0.1 mmol/L 0.88, 95% CI 0.79-0.98)和较高的FCM剂量(首次剂量:aHR 1.12每增加1 mg/kg, 95% CI 1.01-1.25;第二次剂量:每增加1 mg/kg aHR 1.06, 95% CI 1.00-1.13)显著增加中至重度低磷血症的危险。结论:大约三分之一的实体瘤患者在给予FCM后可能发生中重度低磷血症。基线血清磷和FCM剂量可能是可改变的危险因素，应考虑干预，以减轻低磷血症的风险。
{"title":"Incidence, evolution and risk factors of hypophosphatemia in patients with solid tumors receiving ferric carboxymaltose: a retrospective cohort study.","authors":"Alexander Decruyenaere, Koen Kortbeek, Sigurd Delanghe, Sylvie Rottey, Hannelore Denys, Lore Lapeire","doi":"10.1080/17843286.2022.2153465","DOIUrl":"https://doi.org/10.1080/17843286.2022.2153465","url":null,"abstract":"<p><strong>Objectives: </strong>Ferric carboxymaltose (FCM) is increasingly used in the management of cancer-related anemia, yet it may cause hypophosphatemia. This retrospective study describes the incidence, evolution and risk factors of hypophosphatemia in a cohort of patients with solid tumors receiving FCM.</p><p><strong>Methods: </strong>Serum phosphorus concentration was assessed longitudinally using a random intercepts model. The probability of developing hypophosphatemia, as graded by CTCAE version 4.0, was investigated using a multi-state model. Transition hazards were modeled non-parametrically and semi-parametrically by a Cox model. Causal marginal risk differences between baseline interventions on serum phosphorus and/or FCM dose were obtained via G-computation.</p><p><strong>Results: </strong>In 174 ambulatory patients with solid tumors receiving FCM at two university hospitals between October 2020 and September 2021, the risk of developing moderate-to-severe hypophosphatemia was 36.0% (95% confidence interval (CI) 28.2-43.9%) and peaked within 16 days after first FCM administration. The average duration of moderate-to-severe hypophosphatemia was 12.4 days. After adjustment for confounders, lower baseline serum phosphorus (adjusted hazard ratio (aHR) 0.88 per 0.1 mmol/L increase, 95% CI 0.79-0.98) and higher FCM dose (first dose: aHR 1.12 per 1 mg/kg increase, 95% CI 1.01-1.25; second dose: aHR 1.06 per 1 mg/kg increase, 95% CI 1.00-1.13) significantly increased the hazard of moderate-to-severe hypophosphatemia.</p><p><strong>Conclusion: </strong>Approximately one out of three ambulatory patients with solid tumors may develop moderate-to-severe hypophosphatemia after FCM administration. Baseline serum phosphorus and FCM dose may be modifiable risk factors that should be considered for intervention in order to mitigate the risk of hypophosphatemia.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"78 4","pages":"298-307"},"PeriodicalIF":1.6,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9656884","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}
Pub Date : 2023-08-01DOI: 10.1080/17843286.2022.2146924
Wim L C Van Hooste
I like to make some comments and remarks on the publication by Fraeyman et al. (2022) entitled ‘Body mass index and occupational accidents among health care workers in a large university hospital’ [1]. Besides the well-known obesity-related health risks, the obesity-related safety risks are worth to be investigated because of the significant proportion of the workforce is struggling with overweight and obesity. I will focus on the use of the body mass index (BMI), the multifactorial origin of obesity and accidents, the causality and the biological plausibility of the correlation. BMI is a measure of weight adjusted for height. It is a ‘surrogate’ measure of body fat because it measures excess weight rather than excess fat. It is a simple, inexpensive, and non-invasive measure of body fat. BMI can be routinely measured and calculated with reasonable accuracy, e.g. in an occupational health care setting. The clinical limitations should be considered. Age, sex, ethnicity, and muscle mass can influence the relationship between BMI and body fat. Also, BMI does not distinguish between excess fat, muscle or bone mass, nor does it provide any indication of the distribution of fat among individuals. BMI should serve as the ‘initial’ screening of overweight and obesity. Other factors, such as fat distribution, genetics, and fitness level, should be considered. Measuring waist circumference could be seen as a good alternative or a supplementary measurement to BMI calculation, especially among the working, muscular, adult population [2–5]. That all being said, exact values of BMI are still disputed (e.g. for Afro-Americans and Asian persons). But, if one uses the BMI classification as proposed by the World Health Organization for epidemiological purposes, one must stick to the WHO definitions. Although, Fraeyman and colleagues used on page 3 the classification ‘underweight’ for BMI < 20 kg/m, but that must be < 18.5 kg/m. Also using the classification ‘morbid obesity’ is obsolete, must be ‘Class III Obesity” with a BMI of ≥40 kg/m, or ≥35 kg/m with experiencing obesityrelated health problems [6]. One must further conclude that a (little) overweight is not the same as obesity. One must keep in mind that an occupational accident is mostly caused by a myriad of risk factors, as the origin of obesity is multifactorial. It is too bluntly to try to link these two items without taking account of plenty possible confounders of both. The wellestablished shiftwork and obesity relationship could be a confounding factor . . . Or the many comorbidities of obesity (chronic health conditions as sleep apnea syndrome) could be confounding. Many indirect risk factors could be associated among HCWs, e.g. psychosocial factors like work stress, work relationships, work satisfaction, to mention a few. THE health care worker does not exist because it is obviously a container concept. The functional classes A-D of HCWs by Fraeyman et al. could be a too great simplification of the d
{"title":"Body mass index and occupational accidents among health care workers: in BMI we must trust?","authors":"Wim L C Van Hooste","doi":"10.1080/17843286.2022.2146924","DOIUrl":"https://doi.org/10.1080/17843286.2022.2146924","url":null,"abstract":"I like to make some comments and remarks on the publication by Fraeyman et al. (2022) entitled ‘Body mass index and occupational accidents among health care workers in a large university hospital’ [1]. Besides the well-known obesity-related health risks, the obesity-related safety risks are worth to be investigated because of the significant proportion of the workforce is struggling with overweight and obesity. I will focus on the use of the body mass index (BMI), the multifactorial origin of obesity and accidents, the causality and the biological plausibility of the correlation. BMI is a measure of weight adjusted for height. It is a ‘surrogate’ measure of body fat because it measures excess weight rather than excess fat. It is a simple, inexpensive, and non-invasive measure of body fat. BMI can be routinely measured and calculated with reasonable accuracy, e.g. in an occupational health care setting. The clinical limitations should be considered. Age, sex, ethnicity, and muscle mass can influence the relationship between BMI and body fat. Also, BMI does not distinguish between excess fat, muscle or bone mass, nor does it provide any indication of the distribution of fat among individuals. BMI should serve as the ‘initial’ screening of overweight and obesity. Other factors, such as fat distribution, genetics, and fitness level, should be considered. Measuring waist circumference could be seen as a good alternative or a supplementary measurement to BMI calculation, especially among the working, muscular, adult population [2–5]. That all being said, exact values of BMI are still disputed (e.g. for Afro-Americans and Asian persons). But, if one uses the BMI classification as proposed by the World Health Organization for epidemiological purposes, one must stick to the WHO definitions. Although, Fraeyman and colleagues used on page 3 the classification ‘underweight’ for BMI < 20 kg/m, but that must be < 18.5 kg/m. Also using the classification ‘morbid obesity’ is obsolete, must be ‘Class III Obesity” with a BMI of ≥40 kg/m, or ≥35 kg/m with experiencing obesityrelated health problems [6]. One must further conclude that a (little) overweight is not the same as obesity. One must keep in mind that an occupational accident is mostly caused by a myriad of risk factors, as the origin of obesity is multifactorial. It is too bluntly to try to link these two items without taking account of plenty possible confounders of both. The wellestablished shiftwork and obesity relationship could be a confounding factor . . . Or the many comorbidities of obesity (chronic health conditions as sleep apnea syndrome) could be confounding. Many indirect risk factors could be associated among HCWs, e.g. psychosocial factors like work stress, work relationships, work satisfaction, to mention a few. THE health care worker does not exist because it is obviously a container concept. The functional classes A-D of HCWs by Fraeyman et al. could be a too great simplification of the d","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"78 4","pages":"365-366"},"PeriodicalIF":1.6,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9594634","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}
Pub Date : 2023-08-01DOI: 10.1080/17843286.2023.2165652
Joris Rötgens, Bruno Lapauw, Guy T'Sjoen
A 63-year-old man with spells of reduced consciousness in the morning and a giant abdominal mass presented to our institution for a second opinion. Investigation revealed non-diabetic hypoinsulinemic hypoglycemic events. Removal of the abdominal mass solved the hypoglycemia. Anatomopathological examination confirmed a solitary fibrous tumor (SFT). Doege-Potter syndrome was diagnosed. Doege-Potter syndrome is a potentially life-threatening rare paraneoplastic syndrome characterized by recurrent hypoinsulinemic hypoglycemia due to the overproduction of a prohormone form of insulin-like growth factor-II (pro-IGF-II) from a solitary fibrous tumor. First, we describe the clinical, laboratory and radiologic findings of the case. Second, a brief literature review on Doege-Potter syndrome is provided.
{"title":"Doege-Potter syndrome in a patient with a giant abdominal solitary fibrous tumor: a case report and review of the literature.","authors":"Joris Rötgens, Bruno Lapauw, Guy T'Sjoen","doi":"10.1080/17843286.2023.2165652","DOIUrl":"https://doi.org/10.1080/17843286.2023.2165652","url":null,"abstract":"<p><p>A 63-year-old man with spells of reduced consciousness in the morning and a giant abdominal mass presented to our institution for a second opinion. Investigation revealed non-diabetic hypoinsulinemic hypoglycemic events. Removal of the abdominal mass solved the hypoglycemia. Anatomopathological examination confirmed a solitary fibrous tumor (SFT). Doege-Potter syndrome was diagnosed. Doege-Potter syndrome is a potentially life-threatening rare paraneoplastic syndrome characterized by recurrent hypoinsulinemic hypoglycemia due to the overproduction of a prohormone form of insulin-like growth factor-II (pro-IGF-II) from a solitary fibrous tumor. First, we describe the clinical, laboratory and radiologic findings of the case. Second, a brief literature review on Doege-Potter syndrome is provided.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"78 4","pages":"358-364"},"PeriodicalIF":1.6,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9601492","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}
Pub Date : 2023-08-01DOI: 10.1080/17843286.2022.2140246
Kenan Toprak, Mustafa Kaplangoray, Ali Palice, Mustafa Begenç Taşcanov, İbrahim Halil Altıparmak, Asuman Biçer, Recep Demirbağ
ABSTRACT Objectives Hepatokines are proteins secreted by hepatocytes and many hepatokines such as fetuin A/B, selenoprotein P have been shown to play a role in the pathogenesis of many metabolic dysfunctions such as diabetes, insulin resistance, hypertension, and metabolic syndrome by showing autocrine, paracrine and systemic effects. Ectodysplasin A (EDA) is a recently discovered hepatokine that plays a role in the development of ectodermal structures. In recent studies, it has been revealed that EDA may be associated with the pathogenesis of non-alcoholic liver disease, insulin resistance, Type 2 diabetes mellitus. The close relationship between these metabolic diseases and coronary artery disease (CAD), which may be associated with insulin resistance, has been well documented in previous studies. However, until now, there is no study examining the relationship of EDA with CAD and its effect on long-term outcomes. In this study, we aim to reveal this relationship on patients presenting with ST elevation myocardial infarction (STEMI). Methods EDA levels of 544 patients who applied to the study with STEMI and 544 people without coronary artery disease were included in the control group, and the patients with STEMI were followed for median of 33.7 ± 6.8 months. Results We found that EDA levels were significantly higher in patients with STEMI and that EDA levels were proportional to the severity of CAD (p < 0.001) also EDA levels may be an independent predictor of poor clinical outcome in patients with STEMI. Conclusion These results suggest that EDA is closely related to the presence and severity of CAD.
目的:肝因子是由肝细胞分泌的蛋白质，许多肝因子如胎儿素A/B、硒蛋白P等已被证明在许多代谢功能障碍如糖尿病、胰岛素抵抗、高血压和代谢综合征的发病机制中发挥作用，表现出自分泌、旁分泌和全身作用。外泌素A (Ectodysplasin A, EDA)是最近发现的一种肝因子，在外胚层结构的发育中起作用。近年来的研究表明，EDA可能与非酒精性肝病、胰岛素抵抗、2型糖尿病的发病机制有关。这些代谢性疾病与冠状动脉疾病(CAD)之间的密切关系，可能与胰岛素抵抗有关，在以往的研究中得到了充分的证明。然而，到目前为止，还没有研究考察EDA与CAD的关系及其对长期预后的影响。在这项研究中，我们旨在揭示ST段抬高型心肌梗死(STEMI)患者的这种关系。方法:将544例STEMI患者和544例无冠状动脉疾病患者的EDA水平作为对照组，对STEMI患者进行中位随访(33.7±6.8个月)。结果:我们发现STEMI患者EDA水平明显升高，且EDA水平与CAD的严重程度成正比(p)。结论:这些结果表明EDA与CAD的存在和严重程度密切相关。
{"title":"Ectodysplasin A is associated with the presence and severity of coronary artery disease and poor long-term clinical outcome in patients presenting with ST-elevation myocardial infarction.","authors":"Kenan Toprak, Mustafa Kaplangoray, Ali Palice, Mustafa Begenç Taşcanov, İbrahim Halil Altıparmak, Asuman Biçer, Recep Demirbağ","doi":"10.1080/17843286.2022.2140246","DOIUrl":"https://doi.org/10.1080/17843286.2022.2140246","url":null,"abstract":"ABSTRACT Objectives Hepatokines are proteins secreted by hepatocytes and many hepatokines such as fetuin A/B, selenoprotein P have been shown to play a role in the pathogenesis of many metabolic dysfunctions such as diabetes, insulin resistance, hypertension, and metabolic syndrome by showing autocrine, paracrine and systemic effects. Ectodysplasin A (EDA) is a recently discovered hepatokine that plays a role in the development of ectodermal structures. In recent studies, it has been revealed that EDA may be associated with the pathogenesis of non-alcoholic liver disease, insulin resistance, Type 2 diabetes mellitus. The close relationship between these metabolic diseases and coronary artery disease (CAD), which may be associated with insulin resistance, has been well documented in previous studies. However, until now, there is no study examining the relationship of EDA with CAD and its effect on long-term outcomes. In this study, we aim to reveal this relationship on patients presenting with ST elevation myocardial infarction (STEMI). Methods EDA levels of 544 patients who applied to the study with STEMI and 544 people without coronary artery disease were included in the control group, and the patients with STEMI were followed for median of 33.7 ± 6.8 months. Results We found that EDA levels were significantly higher in patients with STEMI and that EDA levels were proportional to the severity of CAD (p < 0.001) also EDA levels may be an independent predictor of poor clinical outcome in patients with STEMI. Conclusion These results suggest that EDA is closely related to the presence and severity of CAD.","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"78 4","pages":"270-279"},"PeriodicalIF":1.6,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9594624","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}
Pub Date : 2023-08-01DOI: 10.1080/17843286.2022.2137631
Michiel Beyens, Jessy Elst, Marie-Line van der Poorten, Athina Van Gasse, Alessandro Toscano, Anke Verlinden, Katrien Vermeulen, Marie-Berthe Maes, J N G Hanneke Oude Elberink, Didier Ebo, Vito Sabato
Mastocytosis is a complex heterogenous multisystem disorder that is characterized by pathologic activation or accumulation of neoplastic mast cells (MCs) in one or more organs. This clonal MC expansion is often associated with a somatic gain-of-function mutation (D816V in most of the cases) in the KIT gene, encoding for the MC surface receptor KIT (CD117), a stem cell growth factor receptor. Based on clinical and biochemical criteria, the World Health Organization (WHO) divided mastocytosis into different subclasses. The exact prevalence of mastocytosis remains elusive, but it is estimated that the disease affects approximately 1 in 10,000 persons. The clinical presentation of mastocytosis varies significantly, ranging from asymptomatic patients to a life-threatening disease with multiple organ involvement, potentially leading to cytopenia, malabsorption, hepatosplenomegaly, lymphadenopathy, ascites or osteolytic bone lesions with pathological fractures. Patients with mastocytosis may experience symptoms related to release of MC mediators, such as flushing or diarrhea or even more severe symptoms such as anaphylaxis. Recently, a new genetic trait, hereditary alpha tryptasemia (HaT), was described which involves a copy number variation in the TPSAB1-gene. Its role as standalone multisystem syndrome is heavily debated. There is emerging evidence suggesting there might be a link between HaT and due to the increased prevalence of HaT in patients with SM. The aim of this review is to provide a practical roadmap for diagnosis and management of mastocytosis and its associated entities, since there are still many misconceptions about these topics.Abbreviations: AdvSM: Advanced systemic mastocytosis; ASM: Aggressive systemic mastocytosis; aST: acute serum tryptase; BM: Bone marrow; BMM: Bone marrow mastocytosis; bST: baseline serum tryptase; CM: Cutaneous mastocytosis; DCM: Diffuse cutaneous mastocytosis; HVA: Hymenoptera venom allergy; HaT: Hereditary alpha tryptasemia; ISM: Indolent systemic mastocytosis; MC: Mast cell; MCA: Mast cell activation; MCAS: Mast cell activation syndrome; MCL: Mast cell leukemia; MIS: Mastocytosis in the skin; MMAS: Monoclonal mast cell activation syndrome; MPCM: Maculopapular cutaneous mastocytosis; SM: Systemic mastocytosis; SM-AHN: Systemic mastocytosis with associated hematological neoplasm; SSM: Smouldering systemic mastocytosis; VIT: Venom immunotherapy.
{"title":"Mastocytosis and related entities: a practical roadmap.","authors":"Michiel Beyens, Jessy Elst, Marie-Line van der Poorten, Athina Van Gasse, Alessandro Toscano, Anke Verlinden, Katrien Vermeulen, Marie-Berthe Maes, J N G Hanneke Oude Elberink, Didier Ebo, Vito Sabato","doi":"10.1080/17843286.2022.2137631","DOIUrl":"https://doi.org/10.1080/17843286.2022.2137631","url":null,"abstract":"<p><p>Mastocytosis is a complex heterogenous multisystem disorder that is characterized by pathologic activation or accumulation of neoplastic mast cells (MCs) in one or more organs. This clonal MC expansion is often associated with a somatic gain-of-function mutation (D816V in most of the cases) in the KIT gene, encoding for the MC surface receptor KIT (CD117), a stem cell growth factor receptor. Based on clinical and biochemical criteria, the World Health Organization (WHO) divided mastocytosis into different subclasses. The exact prevalence of mastocytosis remains elusive, but it is estimated that the disease affects approximately 1 in 10,000 persons. The clinical presentation of mastocytosis varies significantly, ranging from asymptomatic patients to a life-threatening disease with multiple organ involvement, potentially leading to cytopenia, malabsorption, hepatosplenomegaly, lymphadenopathy, ascites or osteolytic bone lesions with pathological fractures. Patients with mastocytosis may experience symptoms related to release of MC mediators, such as flushing or diarrhea or even more severe symptoms such as anaphylaxis. Recently, a new genetic trait, hereditary alpha tryptasemia (HaT), was described which involves a copy number variation in the TPSAB1-gene. Its role as standalone multisystem syndrome is heavily debated. There is emerging evidence suggesting there might be a link between HaT and due to the increased prevalence of HaT in patients with SM. The aim of this review is to provide a practical roadmap for diagnosis and management of mastocytosis and its associated entities, since there are still many misconceptions about these topics.<b>Abbreviations:</b> AdvSM: Advanced systemic mastocytosis; ASM: Aggressive systemic mastocytosis; aST: acute serum tryptase; BM: Bone marrow; BMM: Bone marrow mastocytosis; bST: baseline serum tryptase; CM: Cutaneous mastocytosis; DCM: Diffuse cutaneous mastocytosis; HVA: Hymenoptera venom allergy; HaT: Hereditary alpha tryptasemia; ISM: Indolent systemic mastocytosis; MC: Mast cell; MCA: Mast cell activation; MCAS: Mast cell activation syndrome; MCL: Mast cell leukemia; MIS: Mastocytosis in the skin; MMAS: Monoclonal mast cell activation syndrome; MPCM: Maculopapular cutaneous mastocytosis; SM: Systemic mastocytosis; SM-AHN: Systemic mastocytosis with associated hematological neoplasm; SSM: Smouldering systemic mastocytosis; VIT: Venom immunotherapy.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"78 4","pages":"325-335"},"PeriodicalIF":1.6,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9603786","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}
Background: To examine safety and efficacy of tezacaftor-ivacaftor (TEZ/IVA) in a real-life setting in adults living with cystic fibrosis.
Methods: A multicentre retrospective observational study, including adults living with cystic fibrosis (pwCF) eligible for TEZ/IVA, with assessments at baseline, 3 months (visit3mo) and 6 months (visit6mo) after start of treatment. Outcomes included change in FEV1, LCI, FeNO, CFQ-R, estimated number of annual acute exacerbations, BMI, dosage of pancreatic enzyme replacement therapy (PERT) and airway microbiology. We also assessed safety.
Results: Forty-eight adult pwCF (mean (±SD) age 33 (±12) years; mean FEV1 65 (±19) %P) were included. Three subgroups were identified: pwCF F/F CFTR modulator-naive (n = 28; 58%), pwCF F/F previously treated with lumacaftor-ivacaftor (n = 11; 23%) and pwCF F/RF (n = 9; 19%). Adverse events were described in 3 pwCF (6%) during the 6-month observation period (in one leading to treatment interruption). At visit3mo, FEV1 had improved in all subgroups. In the entire group, mean FEV1 had increased from 66 (±2.9) %P to 72 (±2.9) %P (p < 0.0001). Similarly, LCI improved by approximately one unit at visit3mo (p = 0.02). At visit6mo mean annual acute exacerbation rate decreased significantly (p = 0.02). Only in the CFQ-R social functioning domain score, a significant improvement was observed at visit6mo (p < 0.01).
Conclusions: We showed that TEZ/IVA is safe, well tolerated and effective in terms of improvement of lung function, ventilation inhomogeneity, health-related social functioning, and reduction of estimated annual acute exacerbation rate, in adult pwCF F/F and F/RF. Results in this real-life study reflect those observed in RCTs.
{"title":"Real-world data on the efficacy and safety of tezacaftor-ivacaftor in adults living with cystic fibrosis homozygous for F508del and heterozygous for F508del and a residual function mutation.","authors":"Stefanie Vincken, Sylvia Verbanck, Sue Braun, Nathalie Buyck, Christiane Knoop, Eef Vanderhelst","doi":"10.1080/17843286.2022.2145684","DOIUrl":"https://doi.org/10.1080/17843286.2022.2145684","url":null,"abstract":"<p><strong>Background: </strong>To examine safety and efficacy of tezacaftor-ivacaftor (TEZ/IVA) in a real-life setting in adults living with cystic fibrosis.</p><p><strong>Methods: </strong>A multicentre retrospective observational study, including adults living with cystic fibrosis (pwCF) eligible for TEZ/IVA, with assessments at baseline, 3 months (visit<sub>3mo</sub>) and 6 months (visit<sub>6mo</sub>) after start of treatment. Outcomes included change in FEV<sub>1</sub>, LCI, FeNO, CFQ-R, estimated number of annual acute exacerbations, BMI, dosage of pancreatic enzyme replacement therapy (PERT) and airway microbiology. We also assessed safety.</p><p><strong>Results: </strong>Forty-eight adult pwCF (mean (±SD) age 33 (±12) years; mean FEV<sub>1</sub> 65 (±19) %P) were included. Three subgroups were identified: pwCF F/F CFTR modulator-naive (n = 28; 58%), pwCF F/F previously treated with lumacaftor-ivacaftor (n = 11; 23%) and pwCF F/RF (n = 9; 19%). Adverse events were described in 3 pwCF (6%) during the 6-month observation period (in one leading to treatment interruption). At visit<sub>3mo,</sub> FEV<sub>1</sub> had improved in all subgroups. In the entire group, mean FEV<sub>1</sub> had increased from 66 (±2.9) %P to 72 (±2.9) %P (p < 0.0001). Similarly, LCI improved by approximately one unit at visit<sub>3mo</sub> (p = 0.02). At visit<sub>6mo</sub> mean annual acute exacerbation rate decreased significantly (p = 0.02). Only in the CFQ-R social functioning domain score, a significant improvement was observed at visit<sub>6mo</sub> (p < 0.01).</p><p><strong>Conclusions: </strong>We showed that TEZ/IVA is safe, well tolerated and effective in terms of improvement of lung function, ventilation inhomogeneity, health-related social functioning, and reduction of estimated annual acute exacerbation rate, in adult pwCF F/F and F/RF. Results in this real-life study reflect those observed in RCTs.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"78 4","pages":"280-284"},"PeriodicalIF":1.6,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9601452","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}
Pub Date : 2023-08-01DOI: 10.1080/17843286.2022.2152566
Yana Davidov, Fadi Abu Baker, Ariel Israel, Ziv Ben Ari
Background: Epidemiologic data regarding chronic hepatitis B virus infections in Israel is limited as extensive population-based studies have not been performed.
Objective: This work aimed to evaluate the current characteristics of hepatitis B infection among Israeli adults and evaluate adherence to the European Association for the Study of the Liver practice guidelines for antiviral treatment.
Methods: Clinical and demographic data of HBsAg-positive patients registered in the Leumit-Health-Service database (one of the four major health maintenance organizations in Israel) between 2000 and 2019 were retrieved. Patients were compared according to eligibility to antiviral treatment and type of nucleos(t)ide analogue (NA) treatment.
Results: In total, 1216 patients had documented HBsAg positivity (males 58.6%, mean age 40.2 ± 14.2 years), 90.6% of whom were HBeAg negative. Antiviral therapy eligibility was met by 37% of patients, among whom 89% received antiviral therapy. Antiviral therapies include NA with a high barrier to resistance (HBR) (64.5%) and NA with a low barrier to resistance (LBR) (35.5%). Compared to patients who received LBR NA, patients receiving HBR NA had shorter treatment (68.7 ± 50 vs. 161.5 ± 42.6 months, p < .001) and follow-up duration (125 ± 68 vs. 188 ± 48 months, p < .001); at the end of follow-up, ALT levels and APRI score were higher among patients on LBR NA compared to patients on HBR NA.
Conclusion: Most patients received antiviral treatment according to the international practice guidelines. However, one-third of them were treated with a less potent NA, probably due to their lower cost. These findings should encourage the optimization of HBV care and full compliance with the professional practice guideline recommendations.
{"title":"Real-world hepatitis B antiviral treatment trends and adherence to practice guidelines: a large cohort study.","authors":"Yana Davidov, Fadi Abu Baker, Ariel Israel, Ziv Ben Ari","doi":"10.1080/17843286.2022.2152566","DOIUrl":"https://doi.org/10.1080/17843286.2022.2152566","url":null,"abstract":"<p><strong>Background: </strong>Epidemiologic data regarding chronic hepatitis B virus infections in Israel is limited as extensive population-based studies have not been performed.</p><p><strong>Objective: </strong>This work aimed to evaluate the current characteristics of hepatitis B infection among Israeli adults and evaluate adherence to the European Association for the Study of the Liver practice guidelines for antiviral treatment.</p><p><strong>Methods: </strong>Clinical and demographic data of HBsAg-positive patients registered in the Leumit-Health-Service database (one of the four major health maintenance organizations in Israel) between 2000 and 2019 were retrieved. Patients were compared according to eligibility to antiviral treatment and type of nucleos(t)ide analogue (NA) treatment.</p><p><strong>Results: </strong>In total, 1216 patients had documented HBsAg positivity (males 58.6%, mean age 40.2 ± 14.2 years), 90.6% of whom were HBeAg negative. Antiviral therapy eligibility was met by 37% of patients, among whom 89% received antiviral therapy. Antiviral therapies include NA with a high barrier to resistance (HBR) (64.5%) and NA with a low barrier to resistance (LBR) (35.5%). Compared to patients who received LBR NA, patients receiving HBR NA had shorter treatment (68.7 ± 50 vs. 161.5 ± 42.6 months, p < .001) and follow-up duration (125 ± 68 vs. 188 ± 48 months, p < .001); at the end of follow-up, ALT levels and APRI score were higher among patients on LBR NA compared to patients on HBR NA.</p><p><strong>Conclusion: </strong>Most patients received antiviral treatment according to the international practice guidelines. However, one-third of them were treated with a less potent NA, probably due to their lower cost. These findings should encourage the optimization of HBV care and full compliance with the professional practice guideline recommendations.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"78 4","pages":"291-297"},"PeriodicalIF":1.6,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9604237","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}
Pub Date : 2023-08-01DOI: 10.1080/17843286.2022.2123483
Elvire Nakhoul, Bert Vaes, Pavlos Mamouris, Jean-Marie Degryse
Context: Since the introduction of direct oral anticoagulants (DOACs) for the treatment of nonvalvular atrial fibrillation (Afib), oral anticoagulants (OACs) prescription has evolved.
Aim: We aim first to explore the OACs prescription behaviour in Flanders from 2002 to 2019 before exploring the impact of switching patients from vitamin K antagonists (VKAs) to DOACs in terms of the burden caused by stroke as a complication of non-valvular Afib.
Methods: Data were obtained from INTEGO, a Flemish, general practice-based morbidity registration network. Comprised patients had at least one visit to their GP per year between 2002 and 2019 and a follow-up of at least 1 year after the diagnosis of Afib. Public prices were retrieved from the Belgian Centre for Pharmacotherapeutic Information (BCFI) and the National Institute for Health and Disability Insurance (RIZIV/ INAMI) sites. The number of Disability-Adjusted Life Years (DALYs) was based on the Global Burden of Disease (GBD) literature. The calculation of the Number Needed to Switch (NNSw) was the basis for conducting cost-utility analyses accounting for the global benefit in terms of the cost of prevented stroke/DALY and the cost of switching Flemish ≥ 65 years patients from VKAs to DOACs in two scenarios.
Results: Increased DOAC use has been observed since 2012. The incremental cost effectiveness ratios (ICERs) yielded 553 to 824 €/DALY of prevented stroke.
Conclusion: In this registry-based study, we found a significant positive trend in OAC use in Flanders between 2002 and 2019. Switching to DOACs seems cost-effective compared to a threshold of 20000€/DALY.
{"title":"Trends in oral anticoagulant prescription in patients with nonvalvular atrial fibrillation in Flanders and the impact of switching patients from vitamin K antagonists to DOACS in terms of the burden caused by complications of the disease: a registry-based study.","authors":"Elvire Nakhoul, Bert Vaes, Pavlos Mamouris, Jean-Marie Degryse","doi":"10.1080/17843286.2022.2123483","DOIUrl":"https://doi.org/10.1080/17843286.2022.2123483","url":null,"abstract":"<p><strong>Context: </strong>Since the introduction of direct oral anticoagulants (DOACs) for the treatment of nonvalvular atrial fibrillation (Afib), oral anticoagulants (OACs) prescription has evolved.</p><p><strong>Aim: </strong>We aim first to explore the OACs prescription behaviour in Flanders from 2002 to 2019 before exploring the impact of switching patients from vitamin K antagonists (VKAs) to DOACs in terms of the burden caused by stroke as a complication of non-valvular Afib.</p><p><strong>Methods: </strong>Data were obtained from INTEGO, a Flemish, general practice-based morbidity registration network. Comprised patients had at least one visit to their GP per year between 2002 and 2019 and a follow-up of at least 1 year after the diagnosis of Afib. Public prices were retrieved from the Belgian Centre for Pharmacotherapeutic Information (BCFI) and the National Institute for Health and Disability Insurance (RIZIV/ INAMI) sites. The number of Disability-Adjusted Life Years (DALYs) was based on the Global Burden of Disease (GBD) literature. The calculation of the Number Needed to Switch (NNSw) was the basis for conducting cost-utility analyses accounting for the global benefit in terms of the cost of prevented stroke/DALY and the cost of switching Flemish ≥ 65 years patients from VKAs to DOACs in two scenarios.</p><p><strong>Results: </strong>Increased DOAC use has been observed since 2012. The incremental cost effectiveness ratios (ICERs) yielded 553 to 824 €/DALY of prevented stroke.</p><p><strong>Conclusion: </strong>In this registry-based study, we found a significant positive trend in OAC use in Flanders between 2002 and 2019. Switching to DOACs seems cost-effective compared to a threshold of 20000€/DALY.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"78 4","pages":"261-269"},"PeriodicalIF":1.6,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9656844","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}
Pub Date : 2023-08-01DOI: 10.1080/17843286.2022.2121364
Stijn Arnaert, Thomas Malfait, Astrid Deruyck, Farah Desoete, Maria Nersisjan, Inge Matthijs, Bart Maes
Objective: Rhabdomyolysis induced by an RSV infection is a clinical entity.
Case presentation: A few years ago, one case of severe rhabdomyolysis associated with RSV was described. We present the case of an 18-year-old patient without relevant medical history with a nearly asymptomatic presentation of severe rhabdomyolysis induced by an RSV infection. He consulted the primary physician because of a cough with purulent sputa. A routine blood sample showed elevated creatine kinase levels and the patient was hospitalized. An extensive work-up could exclude other causes of rhabdomyolysis. Creatine kinase levels showed a spontaneous decrease.
Conclusion: This case report highlights an important but very rare presentation of rhabdomyolysis associated with RSV in an adult patient. RSV should be considered as a possible etiological agent for rhabdomyolysis.
{"title":"RSV induced rhabdomyolysis: a case report.","authors":"Stijn Arnaert, Thomas Malfait, Astrid Deruyck, Farah Desoete, Maria Nersisjan, Inge Matthijs, Bart Maes","doi":"10.1080/17843286.2022.2121364","DOIUrl":"https://doi.org/10.1080/17843286.2022.2121364","url":null,"abstract":"<p><strong>Objective: </strong>Rhabdomyolysis induced by an RSV infection is a clinical entity.</p><p><strong>Case presentation: </strong>A few years ago, one case of severe rhabdomyolysis associated with RSV was described. We present the case of an 18-year-old patient without relevant medical history with a nearly asymptomatic presentation of severe rhabdomyolysis induced by an RSV infection. He consulted the primary physician because of a cough with purulent sputa. A routine blood sample showed elevated creatine kinase levels and the patient was hospitalized. An extensive work-up could exclude other causes of rhabdomyolysis. Creatine kinase levels showed a spontaneous decrease.</p><p><strong>Conclusion: </strong>This case report highlights an important but very rare presentation of rhabdomyolysis associated with RSV in an adult patient. RSV should be considered as a possible etiological agent for rhabdomyolysis.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"78 4","pages":"313-315"},"PeriodicalIF":1.6,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9606722","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}
Pub Date : 2023-08-01DOI: 10.1080/17843286.2022.2146929
Lucas Flamant, Guillaume Giordano Orsini, Laurent Ramont, Marion Gornet, Sebastien De Ruffi, Pierre Leroux, Lukshe Kanagaratnam, Stéphane Gennai
Introduction: In older adults, prognostic performances of admission biomarkers have been poorly investigated. This study aims to compare the prognostic abilities of usual admission biomarkers, especially PCT and CRP, for major clinical outcomes, comparing older to younger adults diagnosed with an infection in the ED, and to investigate the prognostic abilities of PCT and CRP depending on the glomerular filtration rate (GFR).
Methods: It was an observational, single-center, retrospective study, conducted in the Reims University Hospital, France. Endpoints were bacteremia, septic shock, and in-hospital mortality, related to the same ED visit.
Results: Over 1 year, 852 patients were included with 291 (34.2%) ≥75 years, and 127 (15.3%) patients had a GFR <30 mL.min-1.1.73 m2. Overall, 74 bacteremia, 56 septic shock and 82 in-hospital deaths have been observed. Prognostic abilities of admission biomarkers tended to be systematically lower in older compared to younger adults (PCT and CRP AUROC for bacteremia were, respectively, 0.71 and 0.62 in older adults vs 0.75 and 0.70 in younger adults; PCT and CRP AUROC for septic shock were, respectively, 0.71 and 0.66 in older adults vs 0.82 and 0.68 in younger adults). PCT showed a significant discriminating power for septic shock and in-hospital mortality only for GFR ≥ 30, and CRP showed a significant discriminating power for bacteremia and septic shock only for GFR ≥60.
Conclusion: Caution must be taken when interpreting admission biomarkers, as their prognostic abilities are lower in older adults or in patients with renal insufficiency diagnosed with an infection.
{"title":"Association between admission biomarkers and clinical outcome in older adults diagnosed with an infection in the emergency department.","authors":"Lucas Flamant, Guillaume Giordano Orsini, Laurent Ramont, Marion Gornet, Sebastien De Ruffi, Pierre Leroux, Lukshe Kanagaratnam, Stéphane Gennai","doi":"10.1080/17843286.2022.2146929","DOIUrl":"https://doi.org/10.1080/17843286.2022.2146929","url":null,"abstract":"<p><strong>Introduction: </strong>In older adults, prognostic performances of admission biomarkers have been poorly investigated. This study aims to compare the prognostic abilities of usual admission biomarkers, especially PCT and CRP, for major clinical outcomes, comparing older to younger adults diagnosed with an infection in the ED, and to investigate the prognostic abilities of PCT and CRP depending on the glomerular filtration rate (GFR).</p><p><strong>Methods: </strong>It was an observational, single-center, retrospective study, conducted in the Reims University Hospital, France. Endpoints were bacteremia, septic shock, and in-hospital mortality, related to the same ED visit.</p><p><strong>Results: </strong>Over 1 year, 852 patients were included with 291 (34.2%) ≥75 years, and 127 (15.3%) patients had a GFR <30 mL.min<sup>-1</sup>.1.73 m<sup>2</sup>. Overall, 74 bacteremia, 56 septic shock and 82 in-hospital deaths have been observed. Prognostic abilities of admission biomarkers tended to be systematically lower in older compared to younger adults (PCT and CRP AUROC for bacteremia were, respectively, 0.71 and 0.62 in older adults <i>vs</i> 0.75 and 0.70 in younger adults; PCT and CRP AUROC for septic shock were, respectively, 0.71 and 0.66 in older adults <i>vs</i> 0.82 and 0.68 in younger adults). PCT showed a significant discriminating power for septic shock and in-hospital mortality only for GFR ≥ 30, and CRP showed a significant discriminating power for bacteremia and septic shock only for GFR ≥60.</p><p><strong>Conclusion: </strong>Caution must be taken when interpreting admission biomarkers, as their prognostic abilities are lower in older adults or in patients with renal insufficiency diagnosed with an infection.</p>","PeriodicalId":7086,"journal":{"name":"Acta Clinica Belgica","volume":"78 4","pages":"285-290"},"PeriodicalIF":1.6,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9958710","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}
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