Pembrolizumab Plus Carboplatin and Paclitaxel as First-Line Therapy for Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma (KEYNOTE-B10): A Single-Arm Phase IV Trial.

Purpose: Standard-of-care first-line treatment for recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC) is pembrolizumab plus platinum and fluorouracil (FU). However, FU is associated with potential challenges (continuous 4-day infusion, high administration costs, and cardiovascular and gastrointestinal toxicities), creating a clinical need for alternative chemotherapy combinations. We evaluated the efficacy and safety of first-line pembrolizumab plus carboplatin and paclitaxel for R/M HNSCC in the open-label, single-arm, phase IV KEYNOTE-B10 study (ClinicalTrials.

A critical evaluation of the EFS endpoint in AML: Does induction treatment failure timing have a profound impact on study design and results?

Despite emerging novel therapies, treating acute myeloid leukemia (AML) remains challenging. Complexities persist in designing pivotal clinical trials and establishing acceptable endpoints for AML. Recent FDA guidance for drug and biological products development for AML outlines considerations for trial design.

Comprehensive Benefit-Risk Assessment of Noninferior Treatments Using Multicriteria Decision Analysis.

Objectives: To develop a simple approach for evaluating the overall benefit-risk of a new noninferiority treatment compared with a standard of care.

Methods: We propose using multicriteria decision analysis that accounts for uncertainty associated with both clinical outcomes and patient preference data. Because patients' preferences are likely to be influenced by their baseline characteristics, we suggest carrying out a preference study at the beginning of a trial.

Cost analysis of alpha blocker treatments for benign prostatic hyperplasia in Medicare beneficiaries.

Objectives: Tamsulosin is the most widely used alpha-1 blocker medication for managing benign prostatic hyperplasia (BPH) as indicated in the current practice guideline. The aim of this study was to compare all-cause medical costs and BPH-specific medical costs in older male adults with BPH treated with tamsulosin vs other alpha-1 blockers (i.e.

Incomplete data analysis of non-inferiority clinical trials: Difference between binomial proportions case.

Background: Incomplete data analysis continues to be a major issue for non-inferiority clinical trials. Due to the steadily increasing use of non-inferiority study design, we believe this topic deserves an immediate attention.

Methods: We evaluated the performance of various strategies, including complete case analysis and various imputations techniques for handling incomplete non-inferiority clinical trials when outcome of interest is difference between binomial proportions.

The treatment of incomplete data: Reporting, analysis, reproducibility, and replicability.

Proper analysis and reporting of incomplete data continues to be a challenging task for practitioners from various research areas. Recently Nguyen, Strazdins, Nicholson and Cooklin (NSNC; 2018) evaluated the impact of complete case analysis and multiple imputation in studies of parental employment and health. Their work joins interdisciplinary efforts to educate and motivate scientists across the research community to use principled statistical methods when analyzing incomplete data.

Long-term effects of rasagiline and the natural history of treated Parkinson's disease.

Background: The Attenuation of Disease progression with Azilect GIven Once-daily (ADAGIO) delayed-start study demonstrated a benefit of early-start treatment with rasagiline 1 mg/day versus delayed-start treatment in PD. This follow-up study aimed to assess whether these benefits persist and the clinical progression rate during long-term naturalistic treatment.

Methods: The ADAGIO Follow-Up study was initiated approximately 26 months after completion of the ADAGIO study.

The Relationship Between Brain MR Spectroscopy and Disability in Multiple Sclerosis: 20-Year Data from the U.S. Glatiramer Acetate Extension Study.

Background And Purpose: Conventional MRI techniques do not necessarily provide information about multiple sclerosis (MS) disease pathology or progression. Nonconventional MRI techniques, including proton magnetic resonance spectroscopy ( H-MRS), are increasingly used to improve the qualitative and quantitative specificity of MR images. This study explores potential correlations between MRI measures of disease and disability progression as measured by the Expanded Disability Status Scale (EDSS), Functional Systems (FS), and ambulation index scores in a unique cohort of MS patients treated with glatiramer acetate that has been closely monitored for over 20 years.

GLACIER: An open-label, randomized, multicenter study to assess the safety and tolerability of glatiramer acetate 40 mg three-times weekly versus 20 mg daily in patients with relapsing-remitting multiple sclerosis.

Background: The efficacy and safety of glatiramer acetate (GA) 20 mg/mL once-daily subcutaneous injections (GA20) in relapsing-remitting multiple sclerosis (RRMS) is well-established. However, injection-related adverse events (IRAEs) may impede treatment adherence and tolerability. GA 40 mg/mL three-times weekly (GA40) also has a favorable efficacy and safety profile.