Objective: Determine the factors associated with epilepsy-related care costs Background: Drivers ... more Objective: Determine the factors associated with epilepsy-related care costs Background: Drivers of epilepsy-related care costs have not been systematically studied Design/Methods: We reviewed electronic medical records and cost data of epilepsy-treated patients between 2013–2016 from Partners Healthcare System’s Enterprise Data Warehouse. Total cost of care for epilepsy (TCC-E) was defined as sum of costs associated with all epilepsy-specific encounters (inpatient, outpatient, procedure, laboratory and hospital pharmacy) over 365 days. Changes in antiepileptic drug (AED) therapy, including additions/substitutions were assessed. Linear regression was used to model TCC-E; logistic regression modelled the likelihood of being in the top 5% and 1% of TCC-E. All models adjusted for demographic factors, insurance type, and neurologist access Results: Data were available from 979 incident and 4392 prevalent cases. The mean and median TCC-E was $4768 and $602, respectively. TCC-E for incide...
ObjectivesParoxysmal nocturnal haemoglobinuria (PNH) is a rare, non‐malignant haematological diso... more ObjectivesParoxysmal 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.MethodsItem‐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. We evaluated ≥2‐level change against the minimal clinically important difference (MCID) of the FACIT‐fatigue total score (≥5 points) and clinical parameters, haemoglobin (Hb; ≥1 g/dL) and normalised absolute reticulocyte count (ARC; 30–100 pg/cells). Logistic regressions estimated baseline‐to‐Week‐16 FACIT‐fatigue item‐level tr...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, acquired, hematologic, life-threate... more Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, acquired, hematologic, life-threatening disease characterized by thrombosis, impaired bone marrow function, and complement-mediated hemolysis. The PEGASUS phase III clinical trial demonstrated superiority of pegcetacoplan over eculizumab regarding improvements in hemoglobin levels in patients with suboptimal response to prior eculizumab treatment. The objective of this post hoc analysis was to compare the patient-reported outcome (PRO) response rates observed among PEGASUS participants and the relationships between their PRO scores with clinical and hematological parameters. Data from the 16-week randomized, controlled (1:1 to pegcetacoplan or eculizumab) period of the PEGASUS trial included comparisons of weekly PRO measurements taken using the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) scale and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening disease with symptoms of... more Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening disease with symptoms of hemolysis and thrombosis. Current therapies for this complement-mediated disease rely predominantly on inhibition of the C5 complement protein. However, data on treatment responses and quality of life in C5-inhibitor (C5i)-treated PNH patients are scarce. The objective of this study was to determine C5i treatment effects on clinical parameters, PNH symptoms, quality of life, and resource use for PNH patients. This cross-sectional study surveyed 122 individuals in the USA receiving treatment for PNH with C5-targeted monoclonal antibodies, eculizumab (ECU) or ravulizumab (RAV). Despite most patients receiving C5i therapy for ≥ 3 months (ECU 100%, n = 35; RAV 95.4%, n = 83), many patients remained anemic with hemoglobin levels ≤ 12 g/dL in 87.5% (n = 28/32) and 82.9% (n = 68/82) of ECU and RAV recipients, respectively. A majority of patients on ECU (88.6%; n = 31/35) and RAV (74.7%; n = 65...
Context: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease involving comple... more Context: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease involving complement-mediated hemolysis. Pegcetacoplan (PEG; a C3-inhibitor recently approved by the FDA) improved fatigue and was superior to eculizumab (ECU; C5-inhibitor) in improving hemoglobin levels at Week 16 (phase 3 PEGASUS; NCT03500549). Objective: These post hoc analyses compare patient-reported measures of fatigue to clinical PNH markers, namely hemoglobin levels, absolute reticulocyte count (ARC), and indirect bilirubin levels from Week 17 to Week 48 in PEG-treated PNH patients. Design: Eighty PNH patients ≥18 years of age with prior suboptimal ECU response (hemoglobin Results: At Week 48, FACIT-Fatigue scores were significantly correlated with hemoglobin (r=0.32, p Conclusions: PNH patients with improved clinical PNH markers following PEG monotherapy for 28 or 48 weeks display clinically meaningful improvements in fatigue. These results suggest that the FACIT-Fatigue scale is a valid patie...
Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired blood disease charact... more Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired blood disease characterized by hemolytic anemia, bone marrow failure, and thrombosis. Prior to the FDA approval of the C3 inhibitor pegcetacoplan, PNH treatment only included the C5 inhibitors (C5i) ravulizumab (RAV) and eculizumab (ECU) to decrease intravascular hemolysis and improve anemia. A Phase III trial of RAV in a treatment-naïve PNH population reported hemoglobin (Hb) stabilization (avoidance a >2 g/dL decrease in baseline Hb) occurred in 68% of RAV and 64% of ECU patients after 6 months. (Lee et al, 2019). However, real world (RW) evidence of the efficacy of these agents on Hb measures in clinical practice are lacking. The objectives of this study were to describe the demographic, healthcare resource utilization (HCRU), and clinical characteristics of PNH patients prior to C5i therapy, and to examine changes in Hb levels from baseline to 6 months after treatment initiation in a PNH patient popul...
Introduction: Current management of patients diagnosed with paroxysmal nocturnal hemoglobinuria (... more Introduction: Current management of patients diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) includes C5 complement inhibitors such as ravulizumab. The ravulizumab dosage regimen varies by patient body weight and consists of a single loading dose followed by maintenance doses administered every 8 weeks via intravenous infusion (see Table for label-recommended loading and maintenance dosing regimens by patient body weight category). Given its recent 2018 FDA approval, real-world data on the dosing patterns of ravulizumab are limited. This study investigated the real-world dosing patterns of patients with PNH treated with ravulizumab in a large US population. Methods: This retrospective longitudinal cohort study was conducted using provider-based claims data from the Symphony Health Integrated Dataverse ® (IDV). Patients were ≥12 years of age and received ≥2 infusions of ravulizumab between June 21, 2019 and May 6, 2021. The index date was defined as the 1st medical claim for...
Objective: Determine the factors associated with epilepsy-related care costs Background: Drivers ... more Objective: Determine the factors associated with epilepsy-related care costs Background: Drivers of epilepsy-related care costs have not been systematically studied Design/Methods: We reviewed electronic medical records and cost data of epilepsy-treated patients between 2013–2016 from Partners Healthcare System’s Enterprise Data Warehouse. Total cost of care for epilepsy (TCC-E) was defined as sum of costs associated with all epilepsy-specific encounters (inpatient, outpatient, procedure, laboratory and hospital pharmacy) over 365 days. Changes in antiepileptic drug (AED) therapy, including additions/substitutions were assessed. Linear regression was used to model TCC-E; logistic regression modelled the likelihood of being in the top 5% and 1% of TCC-E. All models adjusted for demographic factors, insurance type, and neurologist access Results: Data were available from 979 incident and 4392 prevalent cases. The mean and median TCC-E was $4768 and $602, respectively. TCC-E for incide...
ObjectivesParoxysmal nocturnal haemoglobinuria (PNH) is a rare, non‐malignant haematological diso... more ObjectivesParoxysmal 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.MethodsItem‐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. We evaluated ≥2‐level change against the minimal clinically important difference (MCID) of the FACIT‐fatigue total score (≥5 points) and clinical parameters, haemoglobin (Hb; ≥1 g/dL) and normalised absolute reticulocyte count (ARC; 30–100 pg/cells). Logistic regressions estimated baseline‐to‐Week‐16 FACIT‐fatigue item‐level tr...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, acquired, hematologic, life-threate... more Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, acquired, hematologic, life-threatening disease characterized by thrombosis, impaired bone marrow function, and complement-mediated hemolysis. The PEGASUS phase III clinical trial demonstrated superiority of pegcetacoplan over eculizumab regarding improvements in hemoglobin levels in patients with suboptimal response to prior eculizumab treatment. The objective of this post hoc analysis was to compare the patient-reported outcome (PRO) response rates observed among PEGASUS participants and the relationships between their PRO scores with clinical and hematological parameters. Data from the 16-week randomized, controlled (1:1 to pegcetacoplan or eculizumab) period of the PEGASUS trial included comparisons of weekly PRO measurements taken using the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) scale and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening disease with symptoms of... more Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening disease with symptoms of hemolysis and thrombosis. Current therapies for this complement-mediated disease rely predominantly on inhibition of the C5 complement protein. However, data on treatment responses and quality of life in C5-inhibitor (C5i)-treated PNH patients are scarce. The objective of this study was to determine C5i treatment effects on clinical parameters, PNH symptoms, quality of life, and resource use for PNH patients. This cross-sectional study surveyed 122 individuals in the USA receiving treatment for PNH with C5-targeted monoclonal antibodies, eculizumab (ECU) or ravulizumab (RAV). Despite most patients receiving C5i therapy for ≥ 3 months (ECU 100%, n = 35; RAV 95.4%, n = 83), many patients remained anemic with hemoglobin levels ≤ 12 g/dL in 87.5% (n = 28/32) and 82.9% (n = 68/82) of ECU and RAV recipients, respectively. A majority of patients on ECU (88.6%; n = 31/35) and RAV (74.7%; n = 65...
Context: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease involving comple... more Context: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease involving complement-mediated hemolysis. Pegcetacoplan (PEG; a C3-inhibitor recently approved by the FDA) improved fatigue and was superior to eculizumab (ECU; C5-inhibitor) in improving hemoglobin levels at Week 16 (phase 3 PEGASUS; NCT03500549). Objective: These post hoc analyses compare patient-reported measures of fatigue to clinical PNH markers, namely hemoglobin levels, absolute reticulocyte count (ARC), and indirect bilirubin levels from Week 17 to Week 48 in PEG-treated PNH patients. Design: Eighty PNH patients ≥18 years of age with prior suboptimal ECU response (hemoglobin Results: At Week 48, FACIT-Fatigue scores were significantly correlated with hemoglobin (r=0.32, p Conclusions: PNH patients with improved clinical PNH markers following PEG monotherapy for 28 or 48 weeks display clinically meaningful improvements in fatigue. These results suggest that the FACIT-Fatigue scale is a valid patie...
Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired blood disease charact... more Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired blood disease characterized by hemolytic anemia, bone marrow failure, and thrombosis. Prior to the FDA approval of the C3 inhibitor pegcetacoplan, PNH treatment only included the C5 inhibitors (C5i) ravulizumab (RAV) and eculizumab (ECU) to decrease intravascular hemolysis and improve anemia. A Phase III trial of RAV in a treatment-naïve PNH population reported hemoglobin (Hb) stabilization (avoidance a >2 g/dL decrease in baseline Hb) occurred in 68% of RAV and 64% of ECU patients after 6 months. (Lee et al, 2019). However, real world (RW) evidence of the efficacy of these agents on Hb measures in clinical practice are lacking. The objectives of this study were to describe the demographic, healthcare resource utilization (HCRU), and clinical characteristics of PNH patients prior to C5i therapy, and to examine changes in Hb levels from baseline to 6 months after treatment initiation in a PNH patient popul...
Introduction: Current management of patients diagnosed with paroxysmal nocturnal hemoglobinuria (... more Introduction: Current management of patients diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) includes C5 complement inhibitors such as ravulizumab. The ravulizumab dosage regimen varies by patient body weight and consists of a single loading dose followed by maintenance doses administered every 8 weeks via intravenous infusion (see Table for label-recommended loading and maintenance dosing regimens by patient body weight category). Given its recent 2018 FDA approval, real-world data on the dosing patterns of ravulizumab are limited. This study investigated the real-world dosing patterns of patients with PNH treated with ravulizumab in a large US population. Methods: This retrospective longitudinal cohort study was conducted using provider-based claims data from the Symphony Health Integrated Dataverse ® (IDV). Patients were ≥12 years of age and received ≥2 infusions of ravulizumab between June 21, 2019 and May 6, 2021. The index date was defined as the 1st medical claim for...
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