PEGASUS

Aim: To map patient-level data collected on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC) QLQ-C30 to EQ-5D-5L data for estimating health-state utilities in patients with paroxysmal nocturnal hemoglobinuria (PNH). Materials & methods: European cross-sectional PNH patient survey data populated regression models mapping EORTC QLQ-C30 domains (covariates: sex and baseline age) to utilities calculated with the EQ-5D-5L French value set. A genetic algorithm allowed selection of the best-fitting between a set of models with and without interaction terms...

BACKGROUND: Gallium-68 lung perfusion PET/CT is an emerging imaging modality for the assessment of regional lung function, especially to optimise radiotherapy (RT) planning. A key step of lung functional avoidance RT is the delineation of lung functional volumes (LFVs) to be integrated into radiation plans. However, there is currently no consistent and reproducible delineation method for LFVs. The aim of this study was to develop and evaluate an automated delineation threshold method based on total lung function for LFVs delineation with Gallium-68 MAA lung PET/CT imaging...

OBJECTIVES: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, non-malignant haematological disorder associated with disabling fatigue and reduced health-related quality of life. Post-hoc analysis of PEGASUS phase 3 trial (NCT03500549) characterised improvements in patient-reported fatigue measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-fatigue) instrument item-level ratings for pegcetacoplan and eculizumab for the treatment of PNH. METHODS: Item-level responder analysis was conducted on a ≥2-level change from baseline (CFB) clinically important response (CIR) for the FACIT-fatigue 13 individual items rated on a 5-level Likert scale...

Here we examine how our knowledge of present day Venus can inform terrestrial exoplanetary science and how exoplanetary science can inform our study of Venus. In a superficial way the contrasts in knowledge appear stark. We have been looking at Venus for millennia and studying it via telescopic observations for centuries. Spacecraft observations began with Mariner 2 in 1962 when we confirmed that Venus was a hothouse planet, rather than the tropical paradise science fiction pictured. As long as our level of exploration and understanding of Venus remains far below that of Mars, major questions will endure...

Measurements of thermal diffusivity, heat capacity and thermal expansion of X37CrMoV5-1 (1.2343) hot-work tool steel and Maraging 350 (1.6355) steel in the temperature range from -50 °C to 1000 °C were carried out in this paper. Both X37CrMoV5-1 and Maraging 350 are tested for military use as barrel steels. Thermophysical properties were tested using specialised test stands from NETZSCH. Thermal diffusivity was studied using both the LFA 427 laser flash apparatus in the temperature range of RT-1000 °C and the LFA 467 laser flash apparatus in the temperature range of -50 °C-500 °C...

The study aimed to determine whether the reaction time (RT) to the starting signal has an impact on the points scored by elite male motorcycle speedway riders, or whether it depends on the starting position (gate). Differences among junior and senior riders, and how it changes during a single match (15 heats) and in the subsequent phases of the competitive season (the main and knockout phases) were investigated. The database of reaction times to the starting signal obtained by motorcycle speedway riders was collected from a mobile application called PGE Ekstraliga ver...

Victorian values permeate contemporary scientific culture, maintains a historian.

BACKGROUND: The aim of this protocol is to describe the study protocol changes made and subsequently implemented to the Pediatric Guideline Adherence and Outcomes (PEGASUS) Argentina randomized controlled trial (RCT) for care of children with severe traumatic brain injuries (TBI) imposed by the COVID-19 pandemic. The PEGASUS study group met in spring 2020 to evaluate available literature review guidance and the study design change or pausing options due to the potential interruption of research...

BACKGROUND: We evaluated the effectiveness of extended dual antiplatelet therapy (DAPT) usage after 2nd-generation drug elution stent implantation in acute myocardial infarction (AMI) survivors with high ischemic risk characteristics who had no major bleeding for 24 months under at least 1 year of DAPT maintenance. MATERIALS AND METHODS: The primary ischemic and bleeding endpoints were the risk of mortality and the risk of BARC 3 or 5 (major) bleeding. We investigated the event rates for 2-5 years after the index procedure...

Subcutaneous pegcetacoplan (EMPAVELI® in the USA and ASPAVELI® in the EU) is the first complement component 3 (C3) inhibitor approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the USA, and in adults with PNH who are anaemic after ≥ 3 months of treatment with a C5 inhibitor in the EU. In the phase III PRINCE trial in adults with PNH who were anaemic and naïve to a complement inhibitor therapy, pegcetacoplan was superior to the control group (supportive care, excluding complement inhibitors) in achieving haemoglobin stabilization and reducing lactate dehydrogenase levels...

A writer probes humanity's often fraught and paradoxical relationships with other creatures.

AIM: In patients with prior myocardial infarction (MI) on aspirin, the addition of ticagrelor reduces ischaemic risk but increases bleeding risk. The simultaneous assessment of baseline ischaemic and bleeding risk may assist clinicians in selecting patients who are most likely to have a favourable risk/benefit profile with long-term ticagrelor. METHODS AND RESULTS: PEGASUS-TIMI 54 randomized 21 162 prior MI patients, 13 956 of which to the approved 60 mg dose or placebo and who had all necessary data...

BACKGROUND: Patients with previous acute coronary syndrome (ACS) are at high risk of recurrent adverse cardiovascular events. Recently, prolonged dual antiplatelet therapy (DAPT) and oral anticoagulation therapy (OAT) have been shown to reduce recurrent ischemic events to the expense of an increase in bleeding events. The number of patients potentially eligible for these therapies in real life remains to be determined. METHODS: Among ACS patients from five registries and one randomized controlled trial, we assessed the proportion of patients eligible for the PEGASUS strategy only and the proportion of patients eligible for the COMPASS strategy only, and set out the proportion of patients with an overlap between the strategies...

Background and context This paper outlines the process of moving a continuing education programme for primary healthcare professionals from a fully in-person model to fully online so it could continue during coronavirus disease 2019 (COVID-19) lockdowns. The programme uses a peer-led Small Group model with the leader facilitating interactive discussion based on background content researched by a team from Pegasus Health. Assessment of problem When the COVID-19 restrictions were implemented in March 2020, the usual in-person Small Group meetings could not continue...

BACKGROUND: To understand provider perceptions of the COVID-19 pandemic on priorities of severe pediatric traumatic brain injury (TBI) care across hospitals in South America. METHODS: Site principal investigators (PIs) from 17 hospitals in South America enrolled in the PEGASUS-Argentina randomized controlled trial completed questionnaires regarding order of tasks performed in the care of a typical pediatric patient with severe TBI before (2019) and during (2021) the COVID-19 pandemic...

CONTEXT: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, hematologic disease characterized by complement-mediated hemolysis. PNH symptoms include anemia and debilitating fatigue. In the phase 3 PEGASUS trial (NCT03500549), pegcetacoplan demonstrated superiority over eculizumab in improving hemoglobin levels in adults with PNH. However, improvement in fatigue symptoms, measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), and time to improvement are also valuable to clinicians considering treatment options...

CONTEXT: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease characterized by chronic complement-mediated hemolysis. PNH-associated symptoms include anemia and fatigue, with patients requiring frequent blood transfusions. Pegcetacoplan is an FDA/EMA-approved C3 complement inhibitor for adults with PNH. OBJECTIVE: Evaluate normalization rates of hematologic markers, fatigue, and safety after 16 weeks of pegcetacoplan monotherapy in a subgroup of patients with PNH and baseline hemoglobin levels ≥10...

CONTEXT: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease involving complement-mediated hemolysis. Pegcetacoplan is the first FDA/EMA-approved C3 complement-inhibitor for adults with PNH. OBJECTIVE: We report hemoglobin, lactate dehydrogenase (LDH), and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue normalization rates in the phase 3 PEGASUS (NCT03500549) and PRINCE (NCT04085601) studies. DESIGN: The PEGASUS study (2018-2020) randomized patients with hemoglobin levels <10...

BACKGROUND: In the PEGASUS trial, the complement C3 inhibitor, pegcetacoplan, showed superiority to eculizumab in improving haematological outcomes in adult patients with paroxysmal nocturnal haemoglobinuria and suboptimal response to eculizumab at 16 weeks. The aim of the open-label period was to evaluate the long-term efficacy and safety of pegcetacoplan through to 48 weeks. METHODS: PEGASUS was a phase 3, randomised, open-label, active-comparator controlled trial conducted in 44 centres in Australia, Belgium, Canada, France, Germany, Japan, Russia, South Korea, Spain, the UK, and the USA...

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, hematologic disease characterized by complement-mediated hemolysis, thrombosis, and various degrees of bone marrow dysfunction. Until recently, C5 inhibition with eculizumab or ravulizumab represented the only therapies approved for patients with PNH by the United States Food and Drug Administration (US FDA). Although C5-inhibitors reduce PNH-related signs and symptoms, many patients continue to exhibit persistent anemia and require frequent blood transfusions...