Utilization and safety of onabotulinumtoxinA for the prophylactic treatment of chronic migraine from an observational study in Europe

Objective To examine treatment utilization patterns and safety of onabotulinumtoxinA for the prophylactic treatment of chronic migraine in routine clinical practice. Background Clinical trials support onabotulinumtoxinA for the prophylaxis of headache in patients with chronic migraine, but real-world data are limited. Design/methods A prospective, observational, post-authorization study in adult patients with chronic migraine treated with onabotulinumtoxinA. Data were collected at the first study injection and approximately every three months for 52 weeks for utilization and 64 weeks for safety data, and summarized using descriptive statistics. Results Eighty-five physicians (81% neurologists) at 58 practices in the United Kingdom, Germany, Spain, and Sweden participated and recruited 1160 patients (84.2% female, median age 46.6 years). At baseline, 85.8% of patients had physician diagnoses of chronic migraine/transformed migraine and reported an average of 11.3 (SD=6.9) severe headache days per 28 days;50.6% had previously used onabotulinumtoxinA for chronic migraine. A total of 4017 study treatments were observed. The median number of injection sites (n=31) and total dose (155 U) were consistent across all treatment sessions, with a median 13.7 weeks observed between sessions. At least one treatment-related adverse event was reported by 291 patients (25.1%);the most frequently reported treatment-related adverse event was neck pain (4.4%). Most patients (74.4%) were satisfied/extremely satisfied with onabotulinumtoxinA treatment. Conclusions Patient demographics/characteristics are consistent with published data on the chronic migraine population. Utilization of onabotulinumtoxinA treatment for chronic migraine appears to be consistent with the Summary of Product Characteristics and published PREEMPT injection paradigm. No new safety signals were identified

Darolutamide and health-related quality of life in patients with non-metastatic castration-resistant prostate cancer : An analysis of the phase III ARAMIS trial

Copyright © 2021 The Authors. Published by Elsevier Ltd.. All rights reserved.BACKGROUND: In the ARAMIS trial, darolutamide plus androgen deprivation therapy (ADT) versus placebo plus ADT significantly improved metastasis-free survival (MFS), overall survival (OS) and time to pain progression in patients with non-metastatic castration-resistant prostate cancer (nmCRPC). Herein, we present analyses of patient-reported health-related quality of life (HRQoL) outcomes. PATIENTS AND METHODS: This double-blind, placebo-controlled, phase III trial randomised patients with nmCRPC and prostate-specific antigen doubling time ≤10 months to darolutamide 600 mg (n = 955) twice daily or matched placebo (n = 554) while continuing ADT. The primary end-point was MFS; the secondary end-points included OS and time to pain progression. In this analysis, HRQoL was assessed by the time to deterioration using the Functional Assessment of Cancer Therapy-Prostate (FACT-P) prostate cancer subscale (PCS) and the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Prostate Cancer Module (EORTC QLQ-PR25) subscales. RESULTS: Darolutamide significantly prolonged time to deterioration of FACT-P PCS versus placebo (hazard ratio [HR] 0.80, 95% confidence interval [CI] 0.70-0.91; P = 0.0005) at the primary analysis (cut-off date: 3rd September 2018). Time to deterioration of EORTC QLQ-PR25 outcomes showed statistically significant delays with darolutamide versus placebo for urinary (HR 0.64, 95% CI 0.54-0.76; P < 0.0001) and bowel (HR 0.78, 95% CI 0.66-0.92; P = 0.0027) symptoms. Time to worsening of hormonal treatment-related symptoms was similar between the two groups. CONCLUSION: In patients with nmCRPC who are generally asymptomatic, darolutamide maintained HRQoL by significantly delaying time to deterioration of prostate cancer-specific quality of life and disease-related symptoms versus placebo.publishersversionPeer reviewe

Functional Substitution by TAT-Utrophin in Dystrophin-Deficient Mice

James Ervasti and colleagues show that injection of a truncated form of utrophin transduced all tissues examined, integrated with members of the dystrophin complex, and reduced serum levels of creatine kinase in a mouse model of muscular dystrophy

An analysis of factors associated with influenza, pneumoccocal, Tdap, and herpes zoster vaccine uptake in the US adult population and corresponding inter-state variability

Despite longstanding recommendations for routine vaccination against influenza; pneumococcal; tetanus, diphtheria, acellular pertussis (Tdap); and herpes zoster (HZ) among the United States general adult population, vaccine uptake remains low. Understanding factors that influence adult vaccination and coverage variability beyond the national level are important steps toward developing targeted strategies for increasing vaccination coverage. A retrospective analysis was conducted using data from the Behavioral Risk Factor Surveillance System (2011–2014). Multivariable logistic regression modeling was employed to identify individual factors associated with vaccination (socio-demographics, health status, healthcare utilization, state of residence) and generate adjusted vaccination coverage and compliance estimates nationally and by state. Results indicated that multiple characteristics were consistently associated with a higher likelihood of vaccination across all four vaccines, including female sex, increased educational attainment, and annual household income. Model-adjusted vaccination coverage estimates varied widely by state, with inter-state variability for the most recent year of data as follows: influenza (aged ≥18 years) 30.2–49.5%; pneumococcal (aged ≥65 years) 64.0–74.7%; Tdap (aged ≥18 years) 18.7–46.6%; and HZ (aged ≥60 years) 21.3–42.9%. Model-adjusted compliance with age-appropriate recommendations across vaccines was low and also varied by state: influenza+Tdap (aged 18–59 years) 7.9–24.7%; influenza+Tdap+HZ (aged 60–64 years) 4.1–14.4%; and influenza+Tdap+HZ+pneumococcal (aged ≥65 years) 3.0–18.3%. In summary, after adjusting for individual characteristics associated with vaccination, substantial heterogeneity across states remained, suggesting that other local factors (e.g. state policies) may be impacting adult vaccines uptake. Further research is needed to understand such factors, focusing on differences between states with high versus low vaccination coverage