Exposure –Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome
ConclusionE –R efficacy modeling demonstrated significant relationships between trofinetide exposure and RSBQ, CSBS-DP-IT Social Composite, and RTT-COMC scores. Trofinetide is efficacious within the target exposure range, supporting the approved dosing regimen for trofinetide.Trial RegistrationNCT01703533, NCT02715115, NCT04181723. (Source: Advances in Therapy)
Source: Advances in Therapy - February 16, 2024 Category: Drugs & Pharmacology Source Type: research

Risk Factors for Thromboembolic Events in Patients With Dialysis-Dependent CKD: Pooled Analysis of Phase  3 Roxadustat Trials in Japan
ConclusionIn addition to established risk factors, iron deficiency may be related to thromboembolic events.Graphical Abstract available for this article.Trial RegistrationNCT02780726, NCT02952092, NCT02780141, NCT02779764. (Source: Advances in Therapy)
Source: Advances in Therapy - February 16, 2024 Category: Drugs & Pharmacology Source Type: research

Respiratory Syncytial Virus (RSV) Burden in Infants in the Kingdom of Saudi Arabia and the Impact of All-Infant RSV Protection: A Modeling Study
ConclusionCompared with current SoP, an nirsevimab immunoprophylaxis strategy in the KSA for all infants during their first RSV season was estimated to dramatically decrease healthcare resource use, and economic burden associated with RSV. (Source: Advances in Therapy)
Source: Advances in Therapy - February 15, 2024 Category: Drugs & Pharmacology Source Type: research

Efficacy and Tolerability of Adjunctive Brivaracetam in Patients with Focal-Onset Seizures on Specific Concomitant Antiseizure Medications: Pooled Analysis of Double-Blind, Placebo-Controlled Trials
ConclusionBRV was efficacious and well tolerated regardless of the specific concomitant ASMs used as part of their treatment regimen. These data show that in patients with focal-onset seizures, BRV provides additional efficacy to a broad range of ASMs. (Source: Advances in Therapy)
Source: Advances in Therapy - February 15, 2024 Category: Drugs & Pharmacology Source Type: research

Application of Northern Goshawk Back-Propagation Artificial Neural Network in the Prediction of Monohydroxycarbazepine Concentration in Patients with Epilepsy
ConclusionThe NGO-BPANN model exhibits exceptional predictive capability and can be a practical instrument for forecasting MHD concentration in patients with epilepsy.Clinical Trial Registrationwww.chiCTR-OOC-17012141. (Source: Advances in Therapy)
Source: Advances in Therapy - February 15, 2024 Category: Drugs & Pharmacology Source Type: research

Cost-Effectiveness Analysis of Sintilimab Plus Chemotherapy in Advanced Non-Squamous Non-Small Cell Lung Cancer: A Societal Perspective
ConclusionSintilimab plus chemotherapy is a cost-effective first-line treatment therapy for advanced nsNSCLC in China when compared to standard chemotherapy. These findings, along with the improved progression-free survival and overall survival (OS)  observed in ORIENT-11, support the use of this regimen in eligible candidates for advanced nsNSCLC. (Source: Advances in Therapy)
Source: Advances in Therapy - February 15, 2024 Category: Drugs & Pharmacology Source Type: research

Exploring Perceptions of Biologic Therapies: A Qualitative Study Among Canadians Living with Severe Asthma
ConclusionsFindings suggest that the clinical benefits of biologics align with patient perceptions of achieving good asthma control. However, treatment gaps persist among individuals who do not experience a meaningful improvement in their asthma symptoms and those who face barriers accessing biologics. People with severe asthma attributed importance to greater availability of at-home treatment options, improved access to financial support to cover treatment costs and support to address safety concerns. This research provides insight into patient-based treatment priorities and preferences for biologics, which may help infor...
Source: Advances in Therapy - February 13, 2024 Category: Drugs & Pharmacology Source Type: research

Risk of Stroke in Real-World US Individuals with Type  2 Diabetes Receiving Semaglutide or a Dipeptidyl Peptidase 4 Inhibitor
ConclusionThis proof-of-concept analysis indicates that semaglutide has the potential to reduce the risk of stroke in people with T2D when prescribed in clinical practice.Graphical Abstract (Source: Advances in Therapy)
Source: Advances in Therapy - February 10, 2024 Category: Drugs & Pharmacology Source Type: research

Letter to the Editor Regarding “Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China”
(Source: Advances in Therapy)
Source: Advances in Therapy - February 10, 2024 Category: Drugs & Pharmacology Source Type: research

A Response to: Letter to the Editor Regarding “Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China”
(Source: Advances in Therapy)
Source: Advances in Therapy - February 10, 2024 Category: Drugs & Pharmacology Source Type: research

Cost-Effectiveness Analysis of HRD Testing for Previously Treated Patients with Advanced Ovarian Cancer in Italy
ConclusionThe identification of genetic anomalies in patients with advanced OC is a costly process. Regardless, HRD upfront testing compared toBRCA testing had a cost-effective profile, allowing the efficient use of healthcare resources and better life expectancy and quality of life for patients. (Source: Advances in Therapy)
Source: Advances in Therapy - February 8, 2024 Category: Drugs & Pharmacology Source Type: research

Contraceptive Recommendations for Women with Immune-Mediated Inflammatory Diseases: A Delphi Consensus
ConclusionRaising awareness and promoting a multidisciplinary relationship among the physicians involved in the therapeutic decisions by considering all the risk factors when prescribing a contraceptive method is important to prevent VTE in women with IMID. (Source: Advances in Therapy)
Source: Advances in Therapy - February 7, 2024 Category: Drugs & Pharmacology Source Type: research

Outcomes of Patients with COPD Treated with ICS/LABA Before and After Initiation of Single-Inhaler Triple Therapy with Fluticasone Furoate/Umeclidinium/Vilanterol (FF/UMEC/VI)
ConclusionPatients with COPD treated with ICS/LABA during baseline, including patients specifically treated with BUD/FORM and those with a history of ≥ 1 exacerbation, had fewer COPD exacerbations and lower COPD-related HCRU after initiating FF/UMEC/VI. (Source: Advances in Therapy)
Source: Advances in Therapy - February 4, 2024 Category: Drugs & Pharmacology Source Type: research

Efficacy and Safety of Esaxerenone in Hypertensive Patients with Left Ventricular Hypertrophy (ESES-LVH) Study: A Multicenter, Open-Label, Prospective, Interventional Study
ConclusionEsaxerenone showed favorable antihypertensive and cardioprotective effects and safety in hypertensive patients with cardiac hypertrophy.Trial RegistrationJapan Registry of Clinical Trials (jRCTs071190043). (Source: Advances in Therapy)
Source: Advances in Therapy - February 4, 2024 Category: Drugs & Pharmacology Source Type: research

The Association Between Short-Acting β2-Agonist Over-Prescription, and Patient-Reported Acquisition and Use on Asthma Control and Exacerbations: Data from Australia
ConclusionBoth self-reported SABA overuse and zero SABA prescriptions were associated with poor asthma outcomes. The disconnect between prescribing authorisation, OTC availability and actual use, make it difficult for clinicians to quantify SABA use. (Source: Advances in Therapy)
Source: Advances in Therapy - February 4, 2024 Category: Drugs & Pharmacology Source Type: research