A5038346531- Complement system in diseases
- Renal Diseases and Glomerulopathies
- Blood groups and transfusion
- Adenosine and Purinergic Signaling
- Metabolism and Genetic Disorders
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Muscle Physiology and Disorders
- Genetic Neurodegenerative Diseases
- Mitochondrial Function and Pathology
- Hemoglobinopathies and Related Disorders
- Iron Metabolism and Disorders
- Diet and metabolism studies
- Neonatal Health and Biochemistry
- Neurogenetic and Muscular Disorders Research
- Alkaline Phosphatase Research Studies
- Medical and Biological Ozone Research
- Platelet Disorders and Treatments
- Adrenal Hormones and Disorders
- Endoplasmic Reticulum Stress and Disease
- Cystic Fibrosis Research Advances
- Amino Acid Enzymes and Metabolism
- Electrochemical sensors and biosensors
- Advanced Sensor and Energy Harvesting Materials
- Adolescent and Pediatric Healthcare
- Chronic Lymphocytic Leukemia Research
PTC Therapeutics (United States)
2023-2025
Alexion Pharmaceuticals (United States)
2017-2023
AstraZeneca (United States)
2021-2023
Alexion Pharma (Switzerland)
2023
AstraZeneca (Brazil)
2023
Phio Pharmaceuticals (United States)
2021
Alexion Pharmaceuticals (France)
2019
Biorasis (United States)
2009-2010
University of Connecticut
2010
Abstract Background Fatigue is the most common symptom associated with paroxysmal nocturnal hemoglobinuria (PNH). The objective of this analysis was to estimate values that would suggest a clinically important change (CIC) for functional assessment chronic illness therapy-fatigue scale (FACIT-Fatigue) in patients PNH. Methods Adults PNH who initiated eculizumab within 28 days enrollment International Registry as January 2021 baseline FACIT-Fatigue scores were included analysis....
Background Eculizumab has transformed management of paroxysmal nocturnal hemoglobinuria (PNH) since its approval. However, biweekly dosing regimen remains a high treatment burden. Ravulizumab administered every 8 weeks demonstrated noninferiority to eculizumab in two phase 3 trials. In regions where PNH options are available, it is important consider patient preference. Objective The aim this study was assess preference for ravulizumab or eculizumab. Methods Study 302s (ALXN1210-PNH-302s)...
AADCd is a rare neurometabolic disorder presenting in infancy. Children with have motor dysfunction and development delays that result the need for lifelong care; quality of life greatly impacted. Current characterizations health-related associated health state utilities (HSUs) may be underestimated AADCd. Accurate characterization burden important when evaluating benefits treatment, especially improvements observed recently approved disease-modifying therapy eladocagene exuparvovec....
Abstract Background Aromatic L-amino acid decarboxylase deficiency (AADCd) is a rare genetic disorder characterized by movement disorders, motor and autonomic dysfunction, developmental delays. The gene therapy eladocagene exuparvovec has become available in some regions; pooled clinical trial results demonstrate continuous long-term improvement development cognitive function. We sought to characterize clinically meaningful change function, as measured Total Peabody Developmental Motor...
Ravulizumab, engineered from eculizumab, provides sustained C5 inhibition in atypical hemolytic uremic syndrome (aHUS) while reducing dosing frequency (every 8 vs 2 weeks, respectively). Treatment choice often carries significant financial implications. This study compared the economic consequences of ravulizumab and eculizumab for treating aHUS.A cost-minimization model direct medical costs aHUS, assuming equivalent efficacy safety, took a US payer perspective, lifetime horizon, 3.0% cost...
Ravulizumab and eculizumab are approved terminal complement inhibitor treatments for atypical hemolytic uremic syndrome (aHUS). was engineered from to have an increased half-life allowing reduced dosing frequency (8-weekly vs. 2-weekly). To account differences in respective clinical trials, a validated balancing technique used enable indirect comparison of ravulizumab treatment efficacy aHUS. Patient-level data four trials were available pooling with two trials. In the primary analysis,...
This study compared the aggregate duration of treatment administration approved eculizumab and ravulizumab regimens resultant productivity implications for patients with atypical hemolytic uremic syndrome (aHUS) their caregivers.The (which includes waiting time medication preparation infusion, recovery, travel to from clinic) was determined a hypothetical population aHUS treated (10 mg/mL) or 100 mg/mL), in clinic at home, 1 year, Germany, Italy, UK, US. The data US used extend previously...
Paroxysmal nocturnal hemoglobinuria, characterized by intravascular hemolysis and venous thrombosis, can be managed with eculizumab, an inhibitor of the complement system; however, patients may periodically experience breakthrough hemolysis. Ravulizumab is a newly approved treatment for paroxysmal hemoglobinuria that reduce risk, thus improving health-related quality life reducing costs. The objective this study was to compare costs benefit ravulizumab vs eculizumab in adult from US payer...
IntroductionAtypical hemolytic uremic syndrome (aHUS) is a progressive and potentially life-threatening disease characterized by complement-mediated thrombotic microangiopathy. Patients with aHUS may experience fatigue, which can negatively impact their lives, but there knowledge gap regarding burden in these patients.MethodsIn this longitudinal study, patients from the Global Registry who completed patient-reported outcome assessments (Functional Assessment of Chronic Illness...
Objectives: Although complement inhibition is highly effective, patients with paroxysmal nocturnal hemoglobinuria (PNH) may experience intravascular breakthrough hemolysis (BTH). Underlying causes include elevated free C5, pregnancy, or non-pregnancy complement-activating conditions (e.g. infections). This study compared BTH-related resource utilization and costs in PNH treated eculizumab versus ravulizumab.Methods: A cost model was developed using data from a targeted literature review...
Most patients with paroxysmal nocturnal hemoglobinuria (PNH) treated a complement protein 5 (C5) inhibitor achieve full control of terminal activity and intravascular hemolysis. The minority remains anemic transfusion dependent despite this control. Etiology for ongoing anemia is multifactorial includes bone marrow failure, breakthrough hemolysis, extravascular hemolysis (EVH) nutritional deficiencies.To evaluate the potential etiologies hemoglobin levels <10 g/dL receiving C5 therapy, we...
Aim: Ravulizumab and eculizumab are complement C5 inhibitors approved for the treatment of atypical hemolytic uremic syndrome (aHUS). requires less frequent infusions than eculizumab, which may reduce burden. This study investigated patients' preferences impact both treatments on patient caregiver quality life. Materials & methods: Two surveys were conducted (one adult patients with aHUS one caregivers pediatric aHUS) to quantitatively assess preference patient- caregiver-reported...
Ravulizumab and eculizumab have shown efficacy for the treatment of atypical hemolytic uremic syndrome (aHUS), but real-world evidence ravulizumab is limited owing to its more recent approval. This database study examined outcomes adult patients switching from treated with individual treatments.A retrospective, observational using Clarivate Real World Database.US health-insurance billing data (January 2012 March 2021) aged 18 years or older ≥1 diagnosis relevant aHUS, claim ravulizumab, no...
This investigation evaluated the reliability and validity of 6-Minute Walk Test (6MWT) in patients with pediatric hypophosphatasia (HPP). Children (aged 6 to 12 years; n = 11), adolescents (13 17 4), adults (18 65 9) completed 6MWT at screening baseline two clinical studies asfotase alfa. Test-retest 6MWT, Pearson's correlation coefficients (r) for versus baseline, was high children (r 0.95; p < 0.0001), 0.81; 0.125), 0.94; 0.0001). The most conservative minimal clinically important...
Purpose: To develop a patient preference questionnaire (PPQ) assessing eculizumab and ravulizumab treatment for paroxysmal nocturnal hemoglobinuria (PNH).Patients Methods: The development of the PNH-PPQ © was consistent with Food Drug Administration guidelines patient-reported outcome measure development, included 1) targeted literature review; 2) PNH expert clinician input on preferences; 3) review existing qualitative data disease experience; 4) concept elicitation interviews 8 patients...
Background: Since 2007, eculizumab has transformed the management of paroxysmal nocturnal hemoglobinuria (PNH). However, it a treatment burden associated with q2w dosing. Recently, FDA approved ravulizumab for PNH treatment. Ravulizumab administered q8w demonstrated noninferiority to in two phase 3 trials on all primary and key secondary efficacy measures. In view these options, is important consider patient preference determining plan patients (pts). Aims: To assess or treatment, clinical...