Objective: For the diagnosis of pediatric osteomyelitis, the sensitivity, specificity, and predictive value of erythrocyte sedimentation rate (ESR) were evaluated in this study. Methods: A systematic computer-based search was performed for relevant articles focusing on the ESR diagnosis of pediatric osteomyelitis in PubMed, Embase, and the Cochrane Library with an inclusion criteria: 1) the diagnostic utility of ESR for diagnosing osteomyelitis patients under the age of 18;2) two-by-two contingency tables can be obtained. Case reports, review papers, and animal experiments were excluded. Results: The diagnostic meta-analysis included 8 studies involving 348 children with osteomyelitis, all of whom were tested for ESR. Diagnostic meta-analysis revealed a sensitivity and specificity of 0.90, 95% confidence interval (CI) (0.86– 0.93), and 0.50 (95% CI,0.47– 0.54) for ESR in pediatric osteomyelitis diagnosis, respectively. The positive likelihood ratio (LR), negative LR, and diagnostic odds ratio were 1.38,(95% CI,1.08– 1.78), 0.46, (95% CI,0.26– 0.73), and 3.20, (95% CI,1.33– 7.69), respectively. The area under the curve (AUC) was determined to be 0.80 based on the summary receiver operating characteristic curve (SROC). Conclusion: The literature on the use of ESR in pediatric osteomyelitis diagnosis was thoroughly reviewed in this study. It was also found that ESR may be useful as a biomarker for pediatric osteomyelitis diagnosis. Due to its low specificity, it should be used in combination with other markers such as C-reactive protein, neutrophil percentage, and white blood cell count.
{"title":"Erythrocyte Sedimentation Rate for Assisted Diagnosis of Pediatric Osteomyelitis: A Meta-Analysis","authors":"Han Qi, Zhitao Zhu, Dongsheng Zhu","doi":"10.2147/tcrm.s440996","DOIUrl":"https://doi.org/10.2147/tcrm.s440996","url":null,"abstract":"<strong>Objective:</strong> For the diagnosis of pediatric osteomyelitis, the sensitivity, specificity, and predictive value of erythrocyte sedimentation rate (ESR) were evaluated in this study.<br/><strong>Methods:</strong> A systematic computer-based search was performed for relevant articles focusing on the ESR diagnosis of pediatric osteomyelitis in PubMed, Embase, and the Cochrane Library with an inclusion criteria: 1) the diagnostic utility of ESR for diagnosing osteomyelitis patients under the age of 18;2) two-by-two contingency tables can be obtained. Case reports, review papers, and animal experiments were excluded.<br/><strong>Results:</strong> The diagnostic meta-analysis included 8 studies involving 348 children with osteomyelitis, all of whom were tested for ESR. Diagnostic meta-analysis revealed a sensitivity and specificity of 0.90, 95% confidence interval (CI) (0.86– 0.93), and 0.50 (95% CI,0.47– 0.54) for ESR in pediatric osteomyelitis diagnosis, respectively. The positive likelihood ratio (LR), negative LR, and diagnostic odds ratio were 1.38,(95% CI,1.08– 1.78), 0.46, (95% CI,0.26– 0.73), and 3.20, (95% CI,1.33– 7.69), respectively. The area under the curve (AUC) was determined to be 0.80 based on the summary receiver operating characteristic curve (SROC).<br/><strong>Conclusion:</strong> The literature on the use of ESR in pediatric osteomyelitis diagnosis was thoroughly reviewed in this study. It was also found that ESR may be useful as a biomarker for pediatric osteomyelitis diagnosis. Due to its low specificity, it should be used in combination with other markers such as C-reactive protein, neutrophil percentage, and white blood cell count.<br/><br/><strong>Keywords:</strong> osteomyelitis, pediatric, ESR, diagnosis, meta-analysis<br/>","PeriodicalId":22977,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2023-12-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138548266","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Purpose: The Ramathibodi Rapid Response System (RRRS), implemented in March 2017, aims to identify and respond to patients with deteriorating conditions outside the ICU. It employs the Ramathibodi early warning score and clinical signs to monitor all admitted patients, providing expert physician monitoring and early treatment for stabilization and appropriate care triage. This study assesses the RRRS’s effectiveness in reducing in-hospital mortality and CPR events outside the ICU. Patients and Methods: We conducted a retrospective observational study from March 2014 to February 2020 in a tertiary care hospital’s general wards. We included adult patients experiencing unplanned ICU admission, sudden cardiac arrest, or unexpected death. The study compared in-hospital mortality and CPR incidence outside the ICU between pre- and post-RRRS implementation groups. The associations between RRRS implementation and in-hospital mortality and the incidence of CPR outside the ICU were assessed using multiple logistic regression analyses. Results: We evaluated 17,741 admissions, with 9168 before RRRS implementation (1 March 2014 to 28 February 2017) and 8573 after RRRS implementation (1 March 2017 to 29 February 2020). The implementation of RRRS was associated with a significant reduction in in-hospital mortality, which decreased from 30.0% to 20.8% (odds ratio, 0.62; 95% confidence interval [CI], 0.57 to 0.66; P< 0.0001). Even after adjusting for age, sex, and comorbidities, the reduction in in-hospital mortality remained significant (adjusted odds ratio, 0.58; 95% CI, 0.54 to 0.63; P< 0.0001). The incidence of CPR outside the ICU also decreased from 1.8% to 1.1% (adjusted odds ratio, 0.6; 95% CI, 0.46 to 0.77; P< 0.0001). Additionally, the rate of ICU transfer increased from 85.4% to 92.1% (risk difference, 6.7; 95% CI, 7.6 to 5.8; P< 0.0001) after implementing the RRRS. Conclusion: Implementing the RRRS is associated with a reduction in in-hospital mortality and the incidence of CPR outside the ICU.
Keywords: rapid response system, rapid response team, deteriorating patient, in-hospital mortality, cardiopulmonary resuscitation, intensive care unit, implementation protocol
{"title":"Effect of the Ramathibodi Rapid Response System Triggered by the Ramathibodi Early Warning Score and Clinical Warning Signs on in-Hospital Mortality and the Incidence of Cardiopulmonary Resuscitation in Adult Hospitalized Patients","authors":"Chutipong Kwantong, Yuda Sutherasan, Detajin Junhasavasdikul, Tananchai Petnak, Pongdhep Theerawit","doi":"10.2147/tcrm.s426061","DOIUrl":"https://doi.org/10.2147/tcrm.s426061","url":null,"abstract":"<strong>Purpose:</strong> The Ramathibodi Rapid Response System (RRRS), implemented in March 2017, aims to identify and respond to patients with deteriorating conditions outside the ICU. It employs the Ramathibodi early warning score and clinical signs to monitor all admitted patients, providing expert physician monitoring and early treatment for stabilization and appropriate care triage. This study assesses the RRRS’s effectiveness in reducing in-hospital mortality and CPR events outside the ICU.<br/><strong>Patients and Methods:</strong> We conducted a retrospective observational study from March 2014 to February 2020 in a tertiary care hospital’s general wards. We included adult patients experiencing unplanned ICU admission, sudden cardiac arrest, or unexpected death. The study compared in-hospital mortality and CPR incidence outside the ICU between pre- and post-RRRS implementation groups. The associations between RRRS implementation and in-hospital mortality and the incidence of CPR outside the ICU were assessed using multiple logistic regression analyses.<br/><strong>Results:</strong> We evaluated 17,741 admissions, with 9168 before RRRS implementation (1 March 2014 to 28 February 2017) and 8573 after RRRS implementation (1 March 2017 to 29 February 2020). The implementation of RRRS was associated with a significant reduction in in-hospital mortality, which decreased from 30.0% to 20.8% (odds ratio, 0.62; 95% confidence interval [CI], 0.57 to 0.66; P< 0.0001). Even after adjusting for age, sex, and comorbidities, the reduction in in-hospital mortality remained significant (adjusted odds ratio, 0.58; 95% CI, 0.54 to 0.63; P< 0.0001). The incidence of CPR outside the ICU also decreased from 1.8% to 1.1% (adjusted odds ratio, 0.6; 95% CI, 0.46 to 0.77; P< 0.0001). Additionally, the rate of ICU transfer increased from 85.4% to 92.1% (risk difference, 6.7; 95% CI, 7.6 to 5.8; P< 0.0001) after implementing the RRRS.<br/><strong>Conclusion:</strong> Implementing the RRRS is associated with a reduction in in-hospital mortality and the incidence of CPR outside the ICU.<br/><br/><strong>Keywords:</strong> rapid response system, rapid response team, deteriorating patient, in-hospital mortality, cardiopulmonary resuscitation, intensive care unit, implementation protocol<br/>","PeriodicalId":22977,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2023-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138512489","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Abstract: Deutetrabenazine (DTBZ) is used for the treatment of tardive dyskinesia (TD) and chorea in Huntington’s Disease (HD). Four pivotal clinical trials showed the efficacy of DTBZ in these conditions. Long term follow-up studies confirmed evidence of overall safety and continued efficacy of this drug. Indirect comparisons revealed relative superiority of DTBZ over TBZ in terms of safety, but direct comparisons of safety and efficacy between the VMAT2 and dopamine blocking agents is lacking. Deutetrabenazine is safe and effective in the treatment of TD and chorea in HD in doses up to 72 mg daily and for up to three years in duration.
Keywords: chorea, tardive dyskinesia, hyperkinetic movement disorders, VMAT2 inhibitors
{"title":"Clinical Utility of Deutetrabenazine as a Treatment Option for Chorea Associated with Huntington’s Disease and Tardive Dyskinesia","authors":"Samuel Frank, Aljoharah Alakkas","doi":"10.2147/tcrm.s279332","DOIUrl":"https://doi.org/10.2147/tcrm.s279332","url":null,"abstract":"<strong>Abstract:</strong> Deutetrabenazine (DTBZ) is used for the treatment of tardive dyskinesia (TD) and chorea in Huntington’s Disease (HD). Four pivotal clinical trials showed the efficacy of DTBZ in these conditions. Long term follow-up studies confirmed evidence of overall safety and continued efficacy of this drug. Indirect comparisons revealed relative superiority of DTBZ over TBZ in terms of safety, but direct comparisons of safety and efficacy between the VMAT2 and dopamine blocking agents is lacking. Deutetrabenazine is safe and effective in the treatment of TD and chorea in HD in doses up to 72 mg daily and for up to three years in duration.<br/><br/><strong>Keywords:</strong> chorea, tardive dyskinesia, hyperkinetic movement disorders, VMAT2 inhibitors<br/>","PeriodicalId":22977,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138512477","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-29eCollection Date: 2023-01-01DOI: 10.2147/TCRM.S432856
Jennifer Gallardo-Cóndor, Pablo Naranjo, Sebastián Atarihuana, Dayana Coello, Patricia Guevara-Ramírez, Rodrigo Flores-Espinoza, Germán Burgos, Andrés López-Cortés, Alejandro Cabrera-Andrade
Purpose: Thiopurine S-methyltransferase (TPMT) is an enzyme that metabolizes purine analogs, agents used in the treatment of acute lymphoblastic leukemia. Improper drug metabolism leads to toxicity in chemotherapy patients and reduces treatment effectiveness. TPMT variants associated with reduced enzymatic activity vary across populations. Therefore, studying these variants in heterogeneous populations, such as Ecuadorians, can help identify molecular causes of deficiency for this enzyme.
Methods: We sequenced the entire TPMT coding region in 550 Ecuadorian individuals from Afro-Ecuadorian, Indigenous, Mestizo, and Montubio ethnicities. Moreover, we conducted an ancestry analysis using 46 informative ancestry markers.
Results: We identified 8 single nucleotide variants in the coding region of TPMT. The most prevalent alleles were TPMT*3A, TPMT*3B, and TPMT*3C, with frequencies of 0.055, 0.012, and 0.015, respectively. Additionally, we found rare alleles TPMT*4 and TPMT*8 with frequencies of 0.005 and 0.003. Correlating the ancestry proportions with TPMT-deficient genotypes, we observed that the Native American ancestry proportion influenced the distribution of the TPMT*1/TPMT*3A genotype (OR = 5.977, p = 0.002), while the contribution of African ancestral populations was associated with the TPMT*1/TPMT*3C genotype (OR = 9.769, p = 0.003). The rates of TPMT-deficient genotypes observed in Mestizo (f = 0.121) and Indigenous (f = 0.273) groups provide evidence for the influence of Native American ancestry and the prevalence of the TPMT*3A allele. In contrast, although Afro-Ecuadorian groups demonstrate similar deficiency rates (f = 0.160), the genetic factors involved are associated with contributions from African ancestral populations, specifically the prevalent TPMT*3C allele.
Conclusion: The distribution of TPMT-deficient variants offers valuable insights into the populations under study, underscoring the necessity for genetic screening strategies to prevent thiopurine toxicity events among Latin American minority groups.
目的:硫嘌呤s -甲基转移酶(TPMT)是一种代谢嘌呤类似物的酶，嘌呤类似物用于治疗急性淋巴细胞白血病。药物代谢不良导致化疗患者出现毒性，降低治疗效果。与酶活性降低相关的TPMT变异因人群而异。因此，在异质人群中研究这些变异，如厄瓜多尔人，可以帮助确定这种酶缺乏的分子原因。方法:我们对来自非裔厄瓜多尔人、土著、梅斯蒂索人和蒙特比奥种族的550名厄瓜多尔人的整个TPMT编码区进行了测序。此外，我们使用46个信息性祖先标记进行了祖先分析。结果:我们在TPMT编码区鉴定出8个单核苷酸变异。最常见的等位基因为TPMT*3A、TPMT*3B和TPMT*3C，频率分别为0.055、0.012和0.015。此外，我们发现罕见的等位基因TPMT*4和TPMT*8的频率分别为0.005和0.003。将祖先比例与TPMT缺陷基因型相关联，我们发现美洲原住民祖先比例影响TPMT*1/TPMT*3A基因型的分布(OR = 5.977, p = 0.002)，而非洲祖先群体的贡献与TPMT*1/TPMT*3C基因型相关(OR = 9.769, p = 0.003)。在混血儿(f = 0.121)和土著(f = 0.273)群体中观察到的TPMT基因型缺陷率为美洲土著血统的影响和TPMT*3A等位基因的流行提供了证据。相比之下，尽管非裔厄瓜多尔人群体显示出相似的缺乏率(f = 0.160)，但所涉及的遗传因素与非洲祖先人群的贡献有关，特别是普遍存在的TPMT*3C等位基因。结论:tpmt缺陷变异的分布为研究人群提供了有价值的见解，强调了在拉丁美洲少数群体中进行遗传筛查以预防硫嘌呤毒性事件的必要性。
{"title":"Population-Specific Distribution of <i>TPMT</i> Deficiency Variants and Ancestry Proportions in Ecuadorian Ethnic Groups: Towards Personalized Medicine.","authors":"Jennifer Gallardo-Cóndor, Pablo Naranjo, Sebastián Atarihuana, Dayana Coello, Patricia Guevara-Ramírez, Rodrigo Flores-Espinoza, Germán Burgos, Andrés López-Cortés, Alejandro Cabrera-Andrade","doi":"10.2147/TCRM.S432856","DOIUrl":"10.2147/TCRM.S432856","url":null,"abstract":"<p><strong>Purpose: </strong>Thiopurine S-methyltransferase (TPMT) is an enzyme that metabolizes purine analogs, agents used in the treatment of acute lymphoblastic leukemia. Improper drug metabolism leads to toxicity in chemotherapy patients and reduces treatment effectiveness. <i>TPMT</i> variants associated with reduced enzymatic activity vary across populations. Therefore, studying these variants in heterogeneous populations, such as Ecuadorians, can help identify molecular causes of deficiency for this enzyme.</p><p><strong>Methods: </strong>We sequenced the entire <i>TPMT</i> coding region in 550 Ecuadorian individuals from Afro-Ecuadorian, Indigenous, Mestizo, and Montubio ethnicities. Moreover, we conducted an ancestry analysis using 46 informative ancestry markers.</p><p><strong>Results: </strong>We identified 8 single nucleotide variants in the coding region of <i>TPMT</i>. The most prevalent alleles were <i>TPMT*3A, TPMT*3B</i>, and <i>TPMT*3C</i>, with frequencies of 0.055, 0.012, and 0.015, respectively. Additionally, we found rare alleles <i>TPMT*4</i> and <i>TPMT*8</i> with frequencies of 0.005 and 0.003. Correlating the ancestry proportions with <i>TPMT</i>-deficient genotypes, we observed that the Native American ancestry proportion influenced the distribution of the <i>TPMT*1/TPMT*3A</i> genotype (OR = 5.977, p = 0.002), while the contribution of African ancestral populations was associated with the <i>TPMT*1/TPMT*3C</i> genotype (OR = 9.769, p = 0.003). The rates of TPMT-deficient genotypes observed in Mestizo (<i>f</i> = 0.121) and Indigenous (<i>f</i> = 0.273) groups provide evidence for the influence of Native American ancestry and the prevalence of the <i>TPMT*3A</i> allele. In contrast, although Afro-Ecuadorian groups demonstrate similar deficiency rates (<i>f</i> = 0.160), the genetic factors involved are associated with contributions from African ancestral populations, specifically the prevalent <i>TPMT*3C</i> allele.</p><p><strong>Conclusion: </strong>The distribution of TPMT-deficient variants offers valuable insights into the populations under study, underscoring the necessity for genetic screening strategies to prevent thiopurine toxicity events among Latin American minority groups.</p>","PeriodicalId":22977,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2023-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10693761/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138483004","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Objective: This study aimed to create and validate a novel nomogram to predict the risk of symptomatic intracranial hemorrhage (sICH) in patients with acute ischemic stroke (AIS) who underwent intravenous thrombolysis (IVT).
Methods: In this retrospective study, 784 patients with AIS who received IVT were enrolled. The patients were randomly divided into two groups: a training set (n=550, 70%) and a testing set (n=234, 30%). Utilizing multivariable logistic regression analysis, relevant factors for the predictive nomogram were selected. The performance of the nomogram was evaluated using various metrics, including the area under the receiver operating characteristic curve (AUC-ROC), the Hosmer-Lemeshow goodness-of-fit test, calibration plots, and decision curve analysis (DCA).
Results: Multivariable logistic regression analysis showed that specific factors, including National Institutes of Health Stroke Scale (NIHSS) scores, Early infarct signs (EIS), and serum sodium, were identified as independent predictors of sICH. Subsequently, a nomogram was constructed using these predictors. The AUC-ROC values of the nomogram were 0.864 (95% CI: 0.810-0.919) and 0.831 (95% CI: 0.770-0.891) in the training and the validation sets, respectively. Both the calibration plots and the Hosmer-Lemeshow goodness-of-fit test showed favorable agreement in both the training and the validation sets. Additionally, the DCA indicated the practical clinical utility of the nomogram.
Conclusion: The novel nomogram, which included NIHSS, EIS and serum sodium as variables, had the potential for predicting the risk of sICH in patients with AIS after IVT.
{"title":"A Novel Nomogram to Predict Symptomatic Intracranial Hemorrhage in Ischemic Stroke Patients After Intravenous Thrombolysis.","authors":"Zhuangzhuang Jiang, Dongjuan Xu, Hongfei Li, Xiaolan Wu","doi":"10.2147/TCRM.S436145","DOIUrl":"10.2147/TCRM.S436145","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to create and validate a novel nomogram to predict the risk of symptomatic intracranial hemorrhage (sICH) in patients with acute ischemic stroke (AIS) who underwent intravenous thrombolysis (IVT).</p><p><strong>Methods: </strong>In this retrospective study, 784 patients with AIS who received IVT were enrolled. The patients were randomly divided into two groups: a training set (n=550, 70%) and a testing set (n=234, 30%). Utilizing multivariable logistic regression analysis, relevant factors for the predictive nomogram were selected. The performance of the nomogram was evaluated using various metrics, including the area under the receiver operating characteristic curve (AUC-ROC), the Hosmer-Lemeshow goodness-of-fit test, calibration plots, and decision curve analysis (DCA).</p><p><strong>Results: </strong>Multivariable logistic regression analysis showed that specific factors, including National Institutes of Health Stroke Scale (NIHSS) scores, Early infarct signs (EIS), and serum sodium, were identified as independent predictors of sICH. Subsequently, a nomogram was constructed using these predictors. The AUC-ROC values of the nomogram were 0.864 (95% CI: 0.810-0.919) and 0.831 (95% CI: 0.770-0.891) in the training and the validation sets, respectively. Both the calibration plots and the Hosmer-Lemeshow goodness-of-fit test showed favorable agreement in both the training and the validation sets. Additionally, the DCA indicated the practical clinical utility of the nomogram.</p><p><strong>Conclusion: </strong>The novel nomogram, which included NIHSS, EIS and serum sodium as variables, had the potential for predicting the risk of sICH in patients with AIS after IVT.</p>","PeriodicalId":22977,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2023-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10693780/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138483003","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-27eCollection Date: 2023-01-01DOI: 10.2147/TCRM.S361706
Stacy Dixon, Xuan Kang, Dianna Quan
Variant transthyretin amyloidosis (ATTRv) is an autosomal dominant inherited genetic disorder that affects 5000-10,000 people worldwide. It is caused by mutations in the transthyretin (TTR) gene and results in amyloid deposition in a variety of organs due to abnormal accumulation of TTR protein fibrils. Although this is a multisystem disorder, the heart and peripheral nerves are the preferentially affected organs. Over 150 TTR gene mutations have been associated with this disease and the clinical phenotype can vary significantly. Severe forms of the disorder can be fatal. Fortunately, the oligonucleotide-based therapy era has resulted in the development of several novel treatment options. Patisiran is a small interfering RNA (siRNA) encapsulated in a lipid nanoparticle that targets both mutant and wild-type TTR and results in significant reductions of the TTR protein in the serum and in tissue deposits. Patisiran has been approved for treatment of adults with polyneuropathy due to hereditary TTR-mediated amyloidosis in both the United States (US) and European Union (EU). In this review, we will discuss the development of patisiran, the clinical trials that lead to treatment approval, and provide guideline parameters for use in clinical practice. .
{"title":"Practical Guidance for the Use of Patisiran in the Management of Polyneuropathy in Hereditary Transthyretin-Mediated Amyloidosis.","authors":"Stacy Dixon, Xuan Kang, Dianna Quan","doi":"10.2147/TCRM.S361706","DOIUrl":"10.2147/TCRM.S361706","url":null,"abstract":"<p><p>Variant transthyretin amyloidosis (ATTRv) is an autosomal dominant inherited genetic disorder that affects 5000-10,000 people worldwide. It is caused by mutations in the transthyretin (TTR) gene and results in amyloid deposition in a variety of organs due to abnormal accumulation of TTR protein fibrils. Although this is a multisystem disorder, the heart and peripheral nerves are the preferentially affected organs. Over 150 TTR gene mutations have been associated with this disease and the clinical phenotype can vary significantly. Severe forms of the disorder can be fatal. Fortunately, the oligonucleotide-based therapy era has resulted in the development of several novel treatment options. Patisiran is a small interfering RNA (siRNA) encapsulated in a lipid nanoparticle that targets both mutant and wild-type TTR and results in significant reductions of the TTR protein in the serum and in tissue deposits. Patisiran has been approved for treatment of adults with polyneuropathy due to hereditary TTR-mediated amyloidosis in both the United States (US) and European Union (EU). In this review, we will discuss the development of patisiran, the clinical trials that lead to treatment approval, and provide guideline parameters for use in clinical practice. .</p>","PeriodicalId":22977,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2023-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10691373/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138478581","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Background: The efficacy of chronic heart failure (CHF) checklist management in reducing adverse outcomes of heart failure patients is still uncertain. This study explores whether CHF checklist management is more useful than usual care in reducing adverse health outcomes in the medium- and long-term among CHF patients.
Methods: In our prospective study, 132 patients with CHF were randomly assigned to CHF management group and usual care group by random number method. Patients in CHF management group were conducted through CHF checklist by cardiologists and general practitioner. Patients assigned to usual care were treated by non-stationary medical group without checklist. All groups were followed up for 18 months.
Results: There was no significant difference in overall mortality rate between management group and control group during 18 months (12.3% [8/65] vs. 11.7% [7/60], P = 0. 912]). The re-hospitalization rate of heart failure in management group (18.5% [12/65]) was significantly lower than that in usual care group (38.3% [23/60]) after 18 months of follow-up (P = 0.013). Median NT-proBNP level (632.3 ng/l vs. 1678 ng/l, p = 0.004) was lower in management group than that in usual care group. Cardiac ultrasonography was performed at 18 months between the management and usual care group. LVEDD (55.88±7.11 mm vs. 60.92±8.06 mm) and LVESD (43.25±8.42mm vs. 48.41± 9.02mm) were decreased (P<0.01). LVEF was increased (45.36±10.64% vs. 39.96 ±10.15%, P<0.01). The utilization rate of ACEI/ARB/ARNI, β-blocker were high in management group.
Conclusion: CHF checklist management by cardiologists and general practitioners can significantly reduce the re-hospitalization and improve cardiac function. CHF management through heart failure checklist may improve prognosis in patients with CHF in the medium- and long-term.
背景:慢性心力衰竭(CHF)检查表管理在减少心力衰竭患者不良结局方面的效果尚不确定。本研究探讨CHF检查表管理在减少CHF患者中长期不良健康结局方面是否比常规护理更有用。方法:采用前瞻性研究方法，采用随机数字法将132例CHF患者随机分为CHF管理组和常规护理组。CHF管理组患者由心内科医生和全科医生通过CHF检查表进行检查。常规护理组采用不设检查表的非固定治疗组。各组随访18个月。结果:治疗组与对照组18个月总死亡率比较，差异无统计学意义(12.3% [8/65]vs. 11.7% [7/60]， P = 0。912])。随访18个月后，管理组心衰再住院率(18.5%[12/65])显著低于常规护理组(38.3%[23/60])，差异有统计学意义(P = 0.013)。治疗组NT-proBNP水平中位数(632.3 ng/l vs 1678 ng/l, p = 0.004)低于常规护理组。治疗组和常规护理组在18个月时进行心脏超声检查。LVEDD(55.88±7.11 mm vs. 60.92±8.06 mm)和LVESD(43.25±8.42mm vs. 48.41±9.02mm)降低(p结论:心内科医生和全科医生对CHF检查表进行管理可显著减少再次住院，改善心功能。通过心力衰竭检查表进行CHF管理可以改善CHF患者的中长期预后。
{"title":"Effectiveness of Heart Failure Checklist Management in Patients with Chronic Heart Failure: An Open-Label, Single-Center Controlled Study During 18 Months of Follow-Up.","authors":"Xiaorong Xu, Jing Cheng, Yeping Zhang, Xin Wang, Mulei Chen, Lin Xu, Wenshu Zhao","doi":"10.2147/TCRM.S417426","DOIUrl":"10.2147/TCRM.S417426","url":null,"abstract":"<p><strong>Background: </strong>The efficacy of chronic heart failure (CHF) checklist management in reducing adverse outcomes of heart failure patients is still uncertain. This study explores whether CHF checklist management is more useful than usual care in reducing adverse health outcomes in the medium- and long-term among CHF patients.</p><p><strong>Methods: </strong>In our prospective study, 132 patients with CHF were randomly assigned to CHF management group and usual care group by random number method. Patients in CHF management group were conducted through CHF checklist by cardiologists and general practitioner. Patients assigned to usual care were treated by non-stationary medical group without checklist. All groups were followed up for 18 months.</p><p><strong>Results: </strong>There was no significant difference in overall mortality rate between management group and control group during 18 months (12.3% [8/65] vs. 11.7% [7/60], P = 0. 912]). The re-hospitalization rate of heart failure in management group (18.5% [12/65]) was significantly lower than that in usual care group (38.3% [23/60]) after 18 months of follow-up (P = 0.013). Median NT-proBNP level (632.3 ng/l vs. 1678 ng/l, p = 0.004) was lower in management group than that in usual care group. Cardiac ultrasonography was performed at 18 months between the management and usual care group. LVEDD (55.88±7.11 mm vs. 60.92±8.06 mm) and LVESD (43.25±8.42mm vs. 48.41± 9.02mm) were decreased (P<0.01). LVEF was increased (45.36±10.64% vs. 39.96 ±10.15%, P<0.01). The utilization rate of ACEI/ARB/ARNI, β-blocker were high in management group.</p><p><strong>Conclusion: </strong>CHF checklist management by cardiologists and general practitioners can significantly reduce the re-hospitalization and improve cardiac function. CHF management through heart failure checklist may improve prognosis in patients with CHF in the medium- and long-term.</p>","PeriodicalId":22977,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2023-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10691431/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138478580","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-23eCollection Date: 2023-01-01DOI: 10.2147/TCRM.S432565
Ahmed Khalil, Asmaa Mohamed, Manasik Hassan, Samar Magboul, Hossamaldein Ali, Ahmed Salah Elmasoudi, Khaled Ellithy, Mohammad Qusad, Abdulla Alhothi, Eman Al Maslamani, Mohammed Al Amri, Ashraf Soliman
Introduction: While most children experience mild coronavirus disease 2019 (COVID-19) infections, a minority of cases progress to severe or critical illness. This study aimed to assess the efficacy and safety of Remdesivir (RDV) therapy in children with moderate to severe COVID-19, enhancing clinical decision-making and expanding our understanding of antiviral treatments for pediatric patients.
Methods: The study included 60 patients, 38 receiving RDV treatment and 22 serving as the control group. Data was collected retrospectively from January 2021 to January 2022 through electronic hospital records.
Results: Regarding the main clinical symptoms reported, most patients experienced Upper Respiratory Tract Infections (93.3%), indicating respiratory involvement. Additional symptoms included Central Nervous System (11.7%) and Gastrointestinal (10.0%). Among the 38 cases in the RDV group included in the study, the adverse effects associated with using RDV: Hypoalbuminemia in 19 cases (50.0%) and anemia in 18 cases (47.4%), making them the most common adverse effects. Only one case in the RDV group experienced non-RDV-related death with a different clinical diagnosis. The results showed that RDV treatment was well-tolerated in pediatric patients, with no significant differences in hospital stay and oxygen treatment compared to the control group with P values (0.2, 0.18), respectively.
Conclusion: The outcomes indicate that Remdesivir may represent a safe and therapeutic choice for children with coronavirus disease 2019 (COVID-19).
{"title":"Efficacy and Safety of Remdesivir in Hospitalized Pediatric COVID-19: A Retrospective Case-Controlled Study.","authors":"Ahmed Khalil, Asmaa Mohamed, Manasik Hassan, Samar Magboul, Hossamaldein Ali, Ahmed Salah Elmasoudi, Khaled Ellithy, Mohammad Qusad, Abdulla Alhothi, Eman Al Maslamani, Mohammed Al Amri, Ashraf Soliman","doi":"10.2147/TCRM.S432565","DOIUrl":"https://doi.org/10.2147/TCRM.S432565","url":null,"abstract":"<p><strong>Introduction: </strong>While most children experience mild coronavirus disease 2019 (COVID-19) infections, a minority of cases progress to severe or critical illness. This study aimed to assess the efficacy and safety of Remdesivir (RDV) therapy in children with moderate to severe COVID-19, enhancing clinical decision-making and expanding our understanding of antiviral treatments for pediatric patients.</p><p><strong>Methods: </strong>The study included 60 patients, 38 receiving RDV treatment and 22 serving as the control group. Data was collected retrospectively from January 2021 to January 2022 through electronic hospital records.</p><p><strong>Results: </strong>Regarding the main clinical symptoms reported, most patients experienced Upper Respiratory Tract Infections (93.3%), indicating respiratory involvement. Additional symptoms included Central Nervous System (11.7%) and Gastrointestinal (10.0%). Among the 38 cases in the RDV group included in the study, the adverse effects associated with using RDV: Hypoalbuminemia in 19 cases (50.0%) and anemia in 18 cases (47.4%), making them the most common adverse effects. Only one case in the RDV group experienced non-RDV-related death with a different clinical diagnosis. The results showed that RDV treatment was well-tolerated in pediatric patients, with no significant differences in hospital stay and oxygen treatment compared to the control group with P values (0.2, 0.18), respectively.</p><p><strong>Conclusion: </strong>The outcomes indicate that Remdesivir may represent a safe and therapeutic choice for children with coronavirus disease 2019 (COVID-19).</p>","PeriodicalId":22977,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2023-11-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10680468/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138462771","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
The new Calcitonin Gene-Related Peptide (CGRP)-targeted therapies have proven high efficacy and tolerability in episodic and chronic migraine. Eptinezumab is a humanized monoclonal antibody that selectively binds CGRP with high affinity. Eptinezumab was approved by the Food and Drug Administration on February 21st, 2020, for the preventive treatment of migraine in adults. It is administered intravenously over 30 minutes with a standard dose of 100 mg and has a T-max of 30 minutes-1 hour and a half-life of 27 days. These pharmacological properties allow for a very rapid onset of effect and a quarterly administration. It is the first time that a preventive treatment for migraine can be offered as an intravenous administration. As the range of therapeutic possibilities in migraine is expanding, the treatment process must include common decision-making, where physicians should explain in detail to patients the different characteristics of treatment options beyond efficacy and side effects. Patients can now express a preference on a range of opportunities: pharmacological versus non-pharmacological approaches, route of administration, frequency of administration, efficacy, rapidity, side effects, costs, the possibility of titration or dosing, and durability of effectiveness at suspension. Also, patient preferences can be influenced by age, country, migraine severity, and earlier experience with CGRP-targeted therapies. Besides, adherence may be influenced by several factors, including route and the schedule of administration. This narrative review describes a new perspective from the patient's point of view. Clinicians should ally with patients to select treatments that meet each patient's needs and thus apply a tailored approach, addressing not only headaches. In this way, physicians would care for the patients globally and stand out their preferences on different aspects of treatment. Besides, healthcare professionals shall be aware that patients' beliefs about therapies are subject to change with increasing experience with new therapeutic approaches.
{"title":"Eptinezumab for the Prevention of Migraine: Clinical Utility, Patient Preferences and Selection - A Narrative Review.","authors":"Claudia Altamura, Nicoletta Brunelli, Marilena Marcosano, Alessandro Alesina, Luisa Fofi, Fabrizio Vernieri","doi":"10.2147/TCRM.S263824","DOIUrl":"https://doi.org/10.2147/TCRM.S263824","url":null,"abstract":"<p><p>The new Calcitonin Gene-Related Peptide (CGRP)-targeted therapies have proven high efficacy and tolerability in episodic and chronic migraine. Eptinezumab is a humanized monoclonal antibody that selectively binds CGRP with high affinity. Eptinezumab was approved by the Food and Drug Administration on February 21st, 2020, for the preventive treatment of migraine in adults. It is administered intravenously over 30 minutes with a standard dose of 100 mg and has a T-max of 30 minutes-1 hour and a half-life of 27 days. These pharmacological properties allow for a very rapid onset of effect and a quarterly administration. It is the first time that a preventive treatment for migraine can be offered as an intravenous administration. As the range of therapeutic possibilities in migraine is expanding, the treatment process must include common decision-making, where physicians should explain in detail to patients the different characteristics of treatment options beyond efficacy and side effects. Patients can now express a preference on a range of opportunities: pharmacological versus non-pharmacological approaches, route of administration, frequency of administration, efficacy, rapidity, side effects, costs, the possibility of titration or dosing, and durability of effectiveness at suspension. Also, patient preferences can be influenced by age, country, migraine severity, and earlier experience with CGRP-targeted therapies. Besides, adherence may be influenced by several factors, including route and the schedule of administration. This narrative review describes a new perspective from the patient's point of view. Clinicians should ally with patients to select treatments that meet each patient's needs and thus apply a tailored approach, addressing not only headaches. In this way, physicians would care for the patients globally and stand out their preferences on different aspects of treatment. Besides, healthcare professionals shall be aware that patients' beliefs about therapies are subject to change with increasing experience with new therapeutic approaches.</p>","PeriodicalId":22977,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2023-11-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10680459/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138462772","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-21eCollection Date: 2023-01-01DOI: 10.2147/TCRM.S435628
Nicholas Mielke, Yuying Xing, Steven Matthew Gibson, Emily DiLoreto, Amit Bahl
Objective: Education and training is core to improving peripheral intravenous access outcomes. This study aimed to show that a vascular access training program (Operation STICK) in the emergency department (ED) improves the outcomes of traditionally placed peripheral intravenous catheters (PIVC).
Methods: This was a pre-post quasi-experimental study of traditionally placed PIVCs at a large ED in southeastern Michigan, United States. A control group (non-OSTICK) was compared to an experimental group (OSTICK) using a 3:1 propensity score matched analysis. Groups were comprised of ED patients with traditional PIVC placements in two separate six-month periods: non-OSTICK PIVCs from April to September 2021 and OSTICK PIVCs (placed by an OSTICK graduate) from October 2022 to March 2023. The primary outcome was PIVC functionality. The secondary outcome was adherence to best practices.
Results: A total of 6512 PIVCs were included in the study; 4884 (75.0%) were in the non-OSTICK group, while 1628 (25.0%) were in the OSTICK group. 68.1% of OSTICK PIVCs and 59.7% of non-OSTICK PIVCs were placed by ED technicians (p < 0.001). 91.3% of OSTICK PIVCs were placed on the first attempt, and 98.5% were placed within two attempts. A subgroup analysis of admitted patients (2540 PIVCs; 553 (21.8%) OSTICK-trained and 1987 (78.2%) non-OSTICK-trained) revealed 87.6% of OSTICK PIVCs and 80.3% of non-OSTICK PIVCs were 20 gauge (p < 0.001). The median proportion of dwell time to hospital length of stay was 94% for OSTICK PIVCs, compared to 88% for non-OSTICK PIVCs (p < 0.001).
Conclusion: This study underscores the value of education and training in enhancing vascular access outcomes. Implementing Operation STICK, a comprehensive vascular access training program, at a large ED has led to high first-stick success, adherence to best practice recommendations for site and device selection, and improved PIVC functionality for traditionally placed catheters.
{"title":"Bridging the Gap in Traditional PIVC Placement: An Evaluation of Operation STICK Vascular Access Outcomes.","authors":"Nicholas Mielke, Yuying Xing, Steven Matthew Gibson, Emily DiLoreto, Amit Bahl","doi":"10.2147/TCRM.S435628","DOIUrl":"https://doi.org/10.2147/TCRM.S435628","url":null,"abstract":"<p><strong>Objective: </strong>Education and training is core to improving peripheral intravenous access outcomes. This study aimed to show that a vascular access training program (Operation STICK) in the emergency department (ED) improves the outcomes of traditionally placed peripheral intravenous catheters (PIVC).</p><p><strong>Methods: </strong>This was a pre-post quasi-experimental study of traditionally placed PIVCs at a large ED in southeastern Michigan, United States. A control group (non-OSTICK) was compared to an experimental group (OSTICK) using a 3:1 propensity score matched analysis. Groups were comprised of ED patients with traditional PIVC placements in two separate six-month periods: non-OSTICK PIVCs from April to September 2021 and OSTICK PIVCs (placed by an OSTICK graduate) from October 2022 to March 2023. The primary outcome was PIVC functionality. The secondary outcome was adherence to best practices.</p><p><strong>Results: </strong>A total of 6512 PIVCs were included in the study; 4884 (75.0%) were in the non-OSTICK group, while 1628 (25.0%) were in the OSTICK group. 68.1% of OSTICK PIVCs and 59.7% of non-OSTICK PIVCs were placed by ED technicians (p < 0.001). 91.3% of OSTICK PIVCs were placed on the first attempt, and 98.5% were placed within two attempts. A subgroup analysis of admitted patients (2540 PIVCs; 553 (21.8%) OSTICK-trained and 1987 (78.2%) non-OSTICK-trained) revealed 87.6% of OSTICK PIVCs and 80.3% of non-OSTICK PIVCs were 20 gauge (p < 0.001). The median proportion of dwell time to hospital length of stay was 94% for OSTICK PIVCs, compared to 88% for non-OSTICK PIVCs (p < 0.001).</p><p><strong>Conclusion: </strong>This study underscores the value of education and training in enhancing vascular access outcomes. Implementing Operation STICK, a comprehensive vascular access training program, at a large ED has led to high first-stick success, adherence to best practice recommendations for site and device selection, and improved PIVC functionality for traditionally placed catheters.</p>","PeriodicalId":22977,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2023-11-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10676087/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138462770","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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