Efficacy and Safety Analysis in Chinese Patients with Moderate-to-Severe Psoriasis from a Phase 3 Trial: Impact of Treatment Withdrawal and Retreatment of Ixekizumab
In China, approximately 2.3 million people have psoriasis. Continuous treatment is recommended for moderate-to-severe psoriasis. This study aimed to evaluate the outcomes of continuous versus interrupted ixekizumab (IXE) treatment and retreatment with IXE after disease worsening in Chinese patients.
Integrating Genetic Insights, Technological Advancements, Screening, and Personalized Pharmacological Interventions in Childhood Obesity
Childhood obesity is a significant global health challenge with rising prevalence over the past 50 years, affecting both immediate and long-term health outcomes. The increase in prevalence from 0.7% to 5.6% in girls and 0.9% to 7.8% in boys highlights the urgency of addressing this epidemic. By 2025, it is estimated that 206 million children and adolescents aged 5-19 years will be living with obesity. This review explores the complex interplay of genomics and genetics in pediatric obesity, transitioning from monogenic and polygenic obesity to epigenetics, and incorporating advancements in omics technologies. The evolutionary purpose of adiposity, systemic evaluation of hyperphagia, and the role of various genetic factors are discussed. Technological advancements in genotyping offer new insights and interventions. The integration of genetic screening into clinical practice for early identification and personalized treatment strategies is emphasized.
Correction to: Inside ANEMIA of CKD: Projecting the Future Burden of Anemia of Chronic Kidney Disease and Benefits of Proactive Management: A Microsimulation Model of the Chinese Population
Using Chronic Kidney Disease as a Model Framework to Estimate Healthcare-Related Environmental Impact
While the economic and clinical burden of chronic diseases are well documented, their environmental impact remains poorly understood. We developed a framework to estimate the environmental impact of a disease care pathway using chronic kidney disease (CKD) as an example.
Predictors for Irreversibility of Contrast-Induced Acute Kidney Injury in Patients with Obesity After Contrast-Enhanced Computed Tomography Coronary Angiography
Although contrast-induced (CI) acute kidney injury (AKI) is a common complication in high-risk individuals requiring evaluation with contrast-enhanced angiography, the possible predictors of CI-AKI in patients with obesity are not fully understood. The aim of this study was to elucidate plausible factors associated with the irreversibility of CI-AKI in individuals with obesity undergoing contrast-enhanced computed tomography coronary angiography.
Efanesoctocog Alfa Versus Emicizumab in Adolescent and Adult Patients With Haemophilia A Without Inhibitors
The phase 3 XTEND-1 trial (NCT04161495) demonstrated that efanesoctocog alfa prophylaxis provided superior bleed protection compared with pre-trial factor VIII (FVIII) prophylaxis in patients with severe haemophilia A. The aim of this study was to indirectly compare the efficacy of efanesoctocog alfa with non-factor replacement therapy emicizumab in adolescent and adult patients with severe haemophilia A without inhibitors.
Letter to the Editor Regarding "Comparing Cardiovascular Outcomes and Costs of Perindopril-, Enalapril- or Losartan-Based Antihypertensive Regimens in South Africa: Real-World Medical Claims Database Analysis"
Cost-Effectiveness of Vibrant System vs. Linaclotide in Chronic Idiopathic Constipation
Chronic idiopathic constipation (CIC) is a common disorder that has a large unmet clinical need, affecting 8.0-12.0% of the US population and disproportionately affecting female individuals more than male individuals. Patients and physicians are equally dissatisfied with over-the-counter and prescription treatments. Physician dissatisfaction is at 78%. CIC has a significant negative impact on quality of life (QoL). The objective of this analysis was to compare the total cost and QoL of the Vibrant System vs. linaclotide, over 1-3 years of treatment.
A Response to: Letter to the Editor Regarding "Comparing Cardiovascular Outcomes and Costs of Perindopril-, Enalapril- or Losartan-Based Antihypertensive Regimens in South Africa: Real-World Medical Claims Database Analysis"
Oral Corticosteroid-Related Healthcare Resource Utilization and Associated Costs in Patients with COPD
Oral corticosteroids (OCS) are used to manage chronic obstructive pulmonary disease (COPD) exacerbations but are associated with adverse outcomes that may increase healthcare resource utilization and costs. We compared attendance/costs associated with OCS-related adverse outcomes in patients who ever used OCS versus those who never used OCS and examined associations between cumulative OCS exposure and attendance/costs.
Investigation of Severe Hypoglycemia Risk Among Patients with Diabetes Treated with Ultra-Rapid Lispro in Japan
There is no information on the incidence of severe hypoglycemia in real-world patients with diabetes receiving ultra-rapid lispro (URLi). This post-marketing, observational, safety study assessed the incidence proportion and incidence rate of the first severe hypoglycemia event requiring a hospital visit in URLi-treated patients. It also compared the risk of severe hypoglycemia between patients treated with URLi or other rapid-acting insulin analogs (RAIAs).
Real-World Safety and Effectiveness of Dimethyl Fumarate in Patients with MS: Results from the ESTEEM Phase 4 and PROCLAIM Phase 3 Studies with a Focus on Older Patients
Real-world studies in the USA report that 41-56% of patients with multiple sclerosis (MS) are ≥ 50 years old, yet data on their response to disease-modifying therapies (DMTs) is limited. Dimethyl fumarate (DMF) is an oral DMT approved for treating relapsing MS. This analysis evaluated the safety, efficacy, and immunophenotype changes of DMF in patients ≥ 50 years compared with patients < 50 years.
Utility of Loneliness Status to Risk Stratification and Prediction of Recurrent Atrial Fibrillation After Catheter Ablation
Socioeconomic determinants are increasingly important factors in the integrated management of atrial fibrillation (AF). The impact of loneliness status on AF recurrence remains unclear.
Cost-Effectiveness of Trastuzumab Deruxtecan in Patients with Unresectable or Metastatic HER2-Low Breast Cancer Who Have Received Prior Chemotherapy
In 2021, breast cancer affected 75,619 women in Denmark. Approximately 50% of breast cancers are considered human epidermal growth factor receptor 2 (HER2)-low. The DESTINY-Breast04 (DB-04) trial led to European Medicines Agency (EMA) approval of trastuzumab deruxtecan (T-DXd) as a treatment for patients with unresectable or metastatic HER2-low breast cancer who have received prior chemotherapy in the metastatic setting or developed disease recurrence during or within 6 months of completing adjuvant chemotherapy. Moreover, the Danish Breast Cancer Group guidelines recently included T-DXd as a treatment for HER2-low metastatic breast cancer. This economic evaluation aims to estimate the cost-effectiveness of T-DXd for the approved EMA indication in Denmark.
Predicting Asthma Exacerbations Using Machine Learning Models
Although clinical, functional, and biomarker data predict asthma exacerbations, newer approaches providing high accuracy of prognosis are needed for real-world decision-making in asthma. Machine learning (ML) leverages mathematical and statistical methods to detect patterns for future disease events across large datasets from electronic health records (EHR). This study conducted training and fine-tuning of ML algorithms for the real-world prediction of asthma exacerbations in patients with physician-diagnosed asthma.
Indirect Treatment Comparisons in Healthcare Decision Making: A Targeted Review of Regulatory Approval, Reimbursement, and Pricing Recommendations Globally for Oncology Drugs in 2021-2023
Indirect treatment comparisons (ITCs) evaluate novel treatments compared to appropriate comparators when direct evidence is unavailable or infeasible. The objective of this study was to highlight the prevalence of different ITC methods in oncology drug submissions and to provide insights into how ITCs have been used in recent regulatory approval, reimbursement recommendations, or pricing decisions across various regions and diverse assessment frameworks.
Understanding Influenza Vaccine Clinical Performance: A Podcast
Despite well-established vaccination programs, seasonal influenza is still causing substantial clinical, economic and societal burdens. As part of strategies to continually improve influenza vaccine clinical performance, several new approaches are being examined, including high-dose vaccines, adjuvanted vaccines, egg-free vaccines, nasal spray vaccines and mRNA vaccines. Given this range of influenza vaccines, coupled with various vaccine hesitancy concerns, healthcare professionals' understanding and confidence in the clinical performance of influenza vaccines remain key. In this podcast, we discuss the challenges for healthcare professionals in understanding the clinical performance of influenza vaccines and the importance of education in this area, particularly to address perceptions of influenza vaccine failure. We also explore several elements that should be considered in the assessment of influenza vaccine clinical performance: (1) assessing relevant clinical outcomes, such as hospitalization data, (2) utilizing robust methodology in influenza vaccine trials to ensure high quality evidence and (3) approaches used when considering the full body of evidence.
Assessment of Phosphorodiamidate Morpholino Oligomer Treatment Patterns for Patients with Duchenne Muscular Dystrophy: A MarketScan Claims Analysis
Phosphorodiamidate morpholino oligomers (PMOs), weight-based treatments administered weekly by intravenous infusion, are approved in the US for patients with Duchenne muscular dystrophy (DMD) amenable to certain exon skipping. Evidence regarding PMO treatment patterns in real-world settings is limited. This study used longitudinal administrative claims data to characterize PMO treatment patterns among US patients with DMD.
Drug Survival, Retention, and Persistence of Dupilumab in Adults and Adolescents with Atopic Dermatitis: A Narrative Literature Review
Atopic dermatitis (AD) is a chronic, relapsing inflammatory skin condition that can have a negative impact on a patient's quality of life. Long-term effectiveness is required to manage the symptoms of AD (skin inflammation, eczematous lesions, and itching). Because some of the systemic immunosuppressants used to treat AD have been associated with serious adverse events (AEs), other safer, more effective options, including dupilumab, have been proven effective long-term for treatment of adult and adolescent patients with moderate-to-severe AD. The long-term safety and effectiveness of a drug are usually confirmed in real-world studies by evaluating its performance over time. Measures such as drug survival, drug retention, drug persistence, or retention rates reflect whether treatment may be considered as satisfactory by both patients and physicians, meeting key clinical needs. This review aimed to describe the survival, retention, or persistence of dupilumab therapy in adults and adolescents with moderate-to-severe AD by conducting a PubMed search in March 2023 and screening for relevant publications. Globally, real-world studies with dupilumab have regularly reported high drug survival rates after 1, 2, and 3 years of observation, being consistently at 80-90%, with low rates of treatment discontinuation due to lack of efficacy or AEs. These findings are notably higher than 1- and 2-year drug survival rates of systemic immunosuppressants (including cyclosporine [37% and 20%, respectively] and methotrexate [41% and 33%, respectively]). Overall, real-world data on drug survival have confirmed that dupilumab provides long-term sustained efficacy and acceptable safety in patients with moderate-to-severe AD.
Correction to: Effcacy of Ixekizumab in Chinese Patients with Moderate-to-Severe Psoriasis and Special Body Area Involvement: Sub-analysis of a Randomized, Double-Blind, Multicenter Phase 3 Study
Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in Adolescent and Adult Patients with Haemophilia A without Inhibitors
In the Phase 3 XTEND-1 trial, (NCT04161495) efanesoctocog alfa prophylaxis provided superior bleed protection versus pre-study factor VIII (FVIII) replacement therapy in patients with severe haemophilia A. The aim of this study was to indirectly compare bleed outcomes between efanesoctocog alfa and standard/extended half-life (SHL and EHL) FVIII replacement therapies in adolescent and adult patients with severe haemophilia A without inhibitors.