scholarly journals Clinical Burden of Paroxysmal Nocturnal Hemoglobinuria Among Patients Receiving C5 Inhibitors in the United States

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 2-2
Author(s):  
David Dingli ◽  
Joana E. Matos ◽  
Kerri Lehrhaupt ◽  
Sangeeta Krishnan ◽  
Sujata P. Sarda ◽  
...  
Keyword(s):  
United States ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Burden Of Illness ◽  
Thrombotic Event ◽  
The United States ◽  
Adult Patients ◽  
Red Blood Cell Transfusion ◽  
Publicly Traded ◽  
Clinical Measures ◽  
The Impact

INTRODUCTION Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, hematologic disease characterized by chronic complement-mediated hemolysis. Treatment with the C5 inhibitor eculizumab has resulted in a reduction in intravascular hemolysis (IVH) and improvements in morbidity and mortality. However, in a single-center cohort of patients with PNH receiving treatment with eculizumab, 72% remained anemic and 36% continued to require transfusions due to ongoing IVH and extravascular hemolysis (McKinley CE, et al.Blood. 2017;130(Suppl 1):3471; Risitano AM, et al.Front Immunol. 2019;10:1157). This study aims to describe the burden of illness in patients with PNH currently being treated with C5 inhibitors (eculizumab and ravulizumab). Overall, the study aims to understand the clinical and hematological outcomes associated with burden of illness in about 150 patients with PNH globally. In these preliminary analyses, the impact of PNH on hematologic and clinical measures is assessed from patients in the United States. METHODS A cross-sectional survey was administered to adult patients ≥18 years of age in the United States with a self-reported diagnosis of PNH, recruited through a patient advocacy group. Inclusion criteria to complete the secure online survey included current treatment with either eculizumab or ravulizumab, informed consent, and agreement to adverse event reporting. This study was initiated in July 2020 and is ongoing. Results presented herein are preliminary. Impact of PNH on hematologic and clinical measures will be assessed using the following variables: diagnosis levels; and any patient history of blood transfusions, thrombotic events, renal impairment, fatigue, and other PNH-associated symptoms as well as dosing frequency and treatment patterns. For these preliminary analyses, descriptive statistics will be reported for patients who have completed the survey. RESULTS As of August 6, 2020, 58 adult patients with a median age of 52 years (range, 21-88) completed the survey, among which 78% were female. Current medications included eculizumab (n = 20 [34.5%]) or ravulizumab (n = 38 [65.5%]), as well as concurrent anticoagulants (n = 9 [15.5%]) and/or anti-thrombotics (n = 2 [3%]). Most patients initiated treatment with eculizumab (n = 20 [100%]) or with ravulizumab (n = 34 [90%]) ≥3 months before. Median (interquartile range) last known hemoglobin level for patients on eculizumab and ravulizumab was 9.3 g/dL (8.0-11.1) and 10.1 g/dL (8.9-11.5), respectively. Overall, 45 (82%) patients reported hemoglobin values <12 g/dL (eculizumab: 90%; ravulizumab: 78%). Forty (69%) patients reported having ≥1 red blood cell transfusion at any point during their disease. Within the previous 12 months, 53% and 26% of eculizumab- and ravulizumab-treated patients, respectively, had ≥1 transfusion, and 12% and 17% were unsure. Among those patients who had ever received ≥1 transfusion, 6% and 13% had >4 transfusions in the previous 12 months for eculizumab and ravulizumab, respectively. Seventeen patients (29%) reported ≥1 thrombotic event at any point during their disease. Seven patients reported thrombotic events over the previous 12 months; six were receiving ravulizumab. The majority (77%) of patients reported fatigue. Fatigue was reported by nearly 95% of eculizumab-treated patients and 68% of ravulizumab-treated patients. CONCLUSIONS Preliminary results from this burden of illness survey demonstrate that a majority of patients with PNH report remaining anemic, despite treatment with C5 inhibitors eculizumab and ravulizumab for a period of ≥3 months. Disclosures Dingli: Sanofi-Genzyme:Consultancy;Karyopharm Therapeutics:Research Funding;Bristol Myers Squibb:Research Funding;Millenium:Consultancy;Alexion:Consultancy;Apellis:Consultancy;Rigel:Consultancy;Janssen:Consultancy.Matos:Kantar:Current Employment.Lehrhaupt:Kantar:Current Employment.Krishnan:Apellis:Current Employment, Current equity holder in publicly-traded company.Sarda:Apellis:Current Employment, Current equity holder in publicly-traded company.Baver:Apellis:Current Employment, Current equity holder in publicly-traded company.

scholarly journals Work Productivity Loss and Quality of Life in Paroxysmal Nocturnal Hemoglobinuria Among Patients Receiving C5 Inhibitors in the United States

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 3-3
Author(s):  
David Dingli ◽  
Joana E. Matos ◽  
Kerri Lehrhaupt ◽  
Sangeeta Krishnan ◽  
Scott B. Baver ◽  
...  
Keyword(s):  
Quality Of Life ◽  
United States ◽  
Burden Of Illness ◽  
Productivity Loss ◽  
Work Productivity ◽  
The United States ◽  
Publicly Traded ◽  
Eortc Qlq ◽  
The Impact

INTRODUCTION Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, hematologic disease characterized by chronic complement-mediated hemolysis. Treatment with the C5 inhibitor eculizumab has resulted in a reduction in intravascular hemolysis and improvements in morbidity and mortality. Even with the clinical benefit in PNH, eculizumab entails twice-monthly intravenous infusions in a hospital setting in most countries, adversely impacting patients' work productivity (Mastellos DC, et al.Semin Hematol. 2018;55(3):167-175). Lost productivity associated with eculizumab ranged from $344,000 in Russia to $4.3 million in the United States, without caregivers (Levy AR, et al.Blood. 2019;134(Supplement_1):4803). Furthermore, patients in a real-world study treated with eculizumab for 1 year experienced continued impairment in overall quality-of-life relative-to-normative reference scores for the general adult population (Ueda Y, et al.Int J Hematol. 2018;107(6):656-665). This study aims to understand the clinical, humanistic, and economic outcomes associated with burden of illness in about 150 patients with PNH globally. In these preliminary analyses, productivity loss and quality of life (QoL) in patients with PNH currently being treated with C5 inhibitors (eculizumab and ravulizumab) are assessed in patients in the United States. METHODS This cross-sectional survey administered to adult patients in the United States, ≥18 years of age, with self-reported diagnosis of PNH, was initiated in July 2020 and is ongoing. Patients were recruited through a patient advocacy group. Inclusion criteria to complete the secure online survey include current treatment with either eculizumab or ravulizumab, and agreement to provide informed consent and adverse event reporting. To investigate the impact of PNH on employment and activity, the Work Productivity and Activity Impairment-General Health (WPAI-GH) questionnaire was used. QoL was assessed using Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30). For the preliminary WPAI-GH analysis presented here, descriptive statistics are reported for patients who have completed the survey thus far. Analyses examining the impact of PNH on FACIT-Fatigue, EORTC QLQ-C30, and other clinical outcomes assessments among patients on anti-C5 therapy are ongoing. RESULTS A total of 58 adult patients completed the survey as of August 6, 2020. Patients' median age was 52 years (range, 21-88) and 78% of patients were female. Twenty patients (34%) were on eculizumab and 38 (66%) were on ravulizumab. Most patients (93%) had initiated treatment ≥3 months prior to enrollment. In total, 23 (40%) patients reported that they were gainfully employed. Overall, 52% of employed patients reported missing hours of work in the prior 7 days due to their health problems (67% eculizumab and 43% ravulizumab). About 77% of working patients reported that their illness affected their productivity at work (89% eculizumab and 69% ravulizumab) due to the same reason. Employed patients reported an average of 13% (standard deviation, 21%) absenteeism (ie, work time lost due to being absent for illness in the previous week; eculizumab, 22% ± 29%, ravulizumab, 7% ± 12%). Patients reported 26 ± 27% impairment while working over the past 7 days (ie, presenteeism; eculizumab, 39 ± 31%, ravulizumab, 18 ± 22%). Total work productivity impairment was on average 32 ± 31% (eculizumab, 46 ± 35%; ravulizumab, 23 ± 24%). Nearly all patients (n = 54 [93%]) reported at least some impairment in their usual activities regardless of employment (eculizumab, 100%; ravulizumab, 90%). On average, patients reported 38 ± 23% of impaired activity in the previous week (eculizumab, 43 ± 20%; ravulizumab, 36 ± 25%). CONCLUSIONS Preliminary results from this burden of illness survey evaluating humanistic and economic outcomes in patients with PNH demonstrated substantial loss of work-related productivity, greatly diminished ability to work, and limitations in patients' usual activities while being treated with the C5 inhibitors eculizumab and ravulizumab. Disclosures Dingli: Karyopharm Therapeutics:Research Funding;Alexion:Consultancy;Bristol Myers Squibb:Research Funding;Janssen:Consultancy;Rigel:Consultancy;Apellis:Consultancy;Sanofi-Genzyme:Consultancy;Millenium:Consultancy.Matos:Kantar:Current Employment.Lehrhaupt:Kantar:Current Employment.Krishnan:Apellis:Current Employment, Current equity holder in publicly-traded company.Baver:Apellis:Current Employment, Current equity holder in publicly-traded company.Sarda:Apellis:Current Employment, Current equity holder in publicly-traded company.

scholarly journals Comparison of Lost Productivity Due to Eculizumab and Ravulizumab Treatments for Paroxysmal Nocturnal Hemoglobinuria in France, Germany, Italy, Russia, Spain, the United Kingdom, and the United States

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4803-4803
Author(s):  
Adrian R Levy ◽  
Laura Dysart ◽  
Yogesh Patel ◽  
Andrew Briggs ◽  
John Schneider ◽  
...  
Keyword(s):  
United States ◽  
Population Size ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Burden Of Illness ◽  
Productivity Loss ◽  
The United States ◽  
Annual Income ◽  
Productivity Costs ◽  
The United Kingdom ◽  
Lost Productivity

INTRODUCTION Little is known about the productivity loss experienced by patients and their caregivers due to treatment of paroxysmal nocturnal hemoglobinuria (PNH) in countries of different economic status. Some insurers in the United States and other countries require pharmaceutical manufacturers to submit economic evaluations on direct medical costs alone, which excludes productivity costs such as absenteeism, presenteeism, time lost from normal activities, and burden of illness to society. Productivity costs are particularly consequential for PNH because substantial time commitments are required from patients and their caregivers for the intravenous administration of treatment (eculizumab or ravulizumab) at infusion clinics. When patients rely on unpaid caregivers, a complete accounting of costs needs to include caregivers' time loss. However, lost productivity for PNH patients and their caregivers has not been reported. The objective of this study was to assess productivity loss for PNH patients and caregivers due to PNH treatments administered at infusion clinics in France, Germany, Italy, Russia, Spain, the United Kingdom, and the United States. METHODS With a cost-consequence approach and published inputs, productivity costs were estimated for PNH patients who were treated with eculizumab once every 2 weeks (q2w) or ravulizumab once every 8 weeks (q8w) only in infusion clinics in 7 countries for 2 years. Inputs included the estimated PNH patient population size (prevalence: 1 in 500,000 of country population size; Schuller Y, et al. Orphanet J Rare Dis. 2015), the published mean annual income (World Bank, 2019), and assumption that all were employed. Total duration of therapy required for treatment was estimated by considering travel time to the infusion center, wait time in the clinic for medication preparation, infusion time for loading and maintenance doses, and recovery time (240 minutes for eculizumab and 330 minutes for ravulizumab) and was assumed to be the same across all countries. Lost wages were estimated using the human capital approach based on the hourly wage rate, which was derived from each country's 2018 gross national income per capita and assumed a 40-hour work week. Costs for each country were converted to 2018 USD. In the baseline analysis for each country, we did not consider caregivers. We then undertook 11 simulations (each composed of 1000 iterations) in which patient population size and lost wages were treated as normally distributed random variables and the proportion of patients with caregivers increased by 10% intervals. RESULTS The mean annual income varied 6-fold between the included countries. The table shows the total productivity loss due to eculizumab and ravulizumab treatment for a population of PNH patients and a 10% increase in caregivers in each country. For eculizumab, lost productivity ranged from $344,000 in Russia to $4.3 million in the United States, without caregivers. Within each country, the lost productivity due to ravulizumab treatment was reduced by approximately three-quarters relative to eculizumab treatment due to the less frequent dosing and ranged from $123,000 in Russia to $1.5 million in the United States, without caregivers. When the individual effects of population size and lost wages were isolated, the latter had larger proportionate effects in lower-income countries (data not shown). CONCLUSIONS Omitting productivity costs related to unpaid caregiving in PNH underestimates the total burden of illness on society. Switching from a q2w to a q8w treatment results in substantial savings to the patient's productivity. This study showed that this effect was numerically greater in higher-income countries (United States and Germany), in which the opportunity cost of a patient's time is greater. In chronic diseases such as PNH that require lifelong therapies, when the value of treatment is assessed, the burden of illness on society and the relative treatment effect should be considered. Disclosures Levy: Alexion: Consultancy. Dysart:Alexion: Consultancy. Patel:Alexion: Employment. Briggs:ALK: Consultancy; Merck: Consultancy; CVRx: Consultancy; Bayer Steering Committee: Consultancy; Sword Health: Consultancy; Janssen: Consultancy; Novartis: Consultancy; Eisai: Consultancy. Myren:Alexion Pharmaceuticals: Employment. Tomazos:Alexion: Employment.

scholarly journals Corporate Financial Distress: The Impact of Profitability, Liquidity, Asset Productivity, Activity and Solvency

2021 ◽  
Vol 28 (2) ◽  
pp. 104
Author(s):  
Karikari Amoa-Gyarteng
Keyword(s):  
United States ◽  
Financial Distress ◽  
Activity Ratio ◽  
The United States ◽  
Securities And Exchange Commission ◽  
Financial Health ◽  
Publicly Traded ◽  
Distressed Firms ◽  
The Impact ◽  
Global Financial System

This study aims to determine the importance of liquidity, profitability, asset productivity, activity, and solvency in cases of corporate financial distress. One hundred and five firms in the extractive industry in the United States were analyzed. Firms must be publicly traded and have filed form 10-K reports with the securities and exchange commission of the United States to be considered for the study’s population. The measure of corporate financial distress is the Altman Z-score. By using the Altman discriminant function, this study identifies the precipitants of corporate financial distress. This is especially important because widespread corporate financial distress could cause global financial system volatility. The indicators were measured in the last two years before the distressed firms declared bankruptcy. The results indicate that liquidity, profitability, asset productivity and solvency have an impact on the financial health of firms and therefore, on financial distress. The study further determines that activity ratio does not have a statistically significant relationship with financial distress.

scholarly journals ESTIMATING COVID-19 INFECTIONS IN HOSPITAL WORKERS IN THE UNITED STATES

2020 ◽  
Author(s):  
Junaid A. Razzak ◽  
Junaid A. Bhatti ◽  
Ramzan Tahir ◽  
Omrana Pasha-Razzak
Keyword(s):  
United States ◽  
Patient Care ◽  
Burden Of Illness ◽  
The United States ◽  
Direct Care ◽  
Direct Patient Care ◽  
Hospital Workers ◽  
Hospital Worker ◽  
The Us ◽  
The Impact

ABSTRACTObjectiveWe estimated that how many hospital workers in the United States (US) might get infected or die in the COVID-19 pandemic. We also estimated the impact of personal protective equipment (PPE) and age restrictions on these estimates.MethodsOur secondary analyses estimated hospital worker infections in the US based on health worker infection and death rates per 100 deaths from COVID-19 in Hubei and Italy. We used Monte Carlo simulations to compute point estimates with 95% confidence intervals for hospital worker infections in the US based on the two scenarios. We computed potential decrease in infections if the PPE were available only to those involved in direct care of COVID-19 patients (∼ 30%) and if workers aged ≥ 60 years are restricted from patient care. Estimates were adjusted for hospital workers per bed in the US compared to China and Italy.ResultsThe hospital worker infections per 100 deaths were 108.2 in Hubei and 94.1 in Italy. Based on Hubei scenario, we estimated that about 53,640 US hospital workers (95% CI: 43,160 to 62,251) might get infected from COVID-19. The Italian scenario suggested 53,097 US hospital worker (95% CI: 37,133 to 69,003) might get infected during the pandemic. Availability of PPE to high-risk workers could reduce counts to 28,100 (95% CI: 23,048 to 33,242) considering Hubei and to 28,354 (95% CI: 19,829 to 36,848) considering Italy. Restricting hospital workers aged ≥ 60 years from direct patient care reduced counts to 1,985 (95% CI: 1,627 to 2,347) considering Hubei and to 2,002 (95% CI: 1,400 to 2,602) considering the Italian scenario.ConclusionWe estimated significant burden of illness due to COVID-19 if no strategies are adopted. Making PPE available to all hospital workers and reducing exposure of hospital workers above the age of 60 could have significant reductions in hospital worker infections.VISUAL ABSTRACTFigure 1.Estimated number of COVID-19 related infections among healthcare workers in the United States based on Hubei and Italian scenarios

scholarly journals Initial estimates of COVID-19 infections in hospital workers in the United States during the first wave of pandemic

PLoS ONE ◽  
2020 ◽  
Vol 15 (12) ◽  
pp. e0242589
Author(s):  
Junaid A. Razzak ◽  
Junaid A. Bhatti ◽  
Muhammad Ramzan Tahir ◽  
Omrana Pasha-Razzak
Keyword(s):  
United States ◽  
High Risk ◽  
Patient Care ◽  
Burden Of Illness ◽  
The United States ◽  
Population Based ◽  
Direct Patient Care ◽  
Hospital Workers ◽  
The Us ◽  
The Impact

Objective We estimated the number of hospital workers in the United States (US) that might be infected or die during the COVID-19 pandemic based on the data in the early phases of the pandemic. Methods We calculated infection and death rates amongst US hospital workers per 100 COVID-19-related deaths in the general population based on observed numbers in Hubei, China, and Italy. We used Monte Carlo simulations to compute point estimates with 95% confidence intervals for hospital worker (HW) infections in the US based on each of these two scenarios. We also assessed the impact of restricting hospital workers aged ≥ 60 years from performing patient care activities on these estimates. Results We estimated that about 53,000 hospital workers in the US could get infected, and 1579 could die due to COVID19. The availability of PPE for high-risk workers alone could reduce this number to about 28,000 infections and 850 deaths. Restricting high-risk hospital workers such as those aged ≥ 60 years from direct patient care could reduce counts to 2,000 healthcare worker infections and 60 deaths. Conclusion We estimate that US hospital workers will bear a significant burden of illness due to COVID-19. Making PPE available to all hospital workers and reducing the exposure of hospital workers above the age of 60 could mitigate these risks.

scholarly journals Trial in Progress: Quantifying the Humanistic and Socioeconomic Burden of Paroxysmal Nocturnal Hemoglobinuria in the COMMODORE Burden of Illness Study

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 26-27
Author(s):  
Roger J Hampton ◽  
Pablo Katz ◽  
Aijing Shang ◽  
Harpal Dhillon ◽  
Hayley Hubberstey
Keyword(s):  
Data Collection ◽  
Resource Utilization ◽  
Economic Burden ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Burden Of Illness ◽  
Third Party ◽  
Publicly Traded ◽  
Illness Study ◽  
Socioeconomic Burden ◽  
The Impact

Background Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease associated with major complications such as thrombotic events and impaired renal function. Prior to the introduction of C5 inhibitors in 2007, PNH had been fatal in about 35% of patients within 5 years of diagnosis; yet, this fatality rate continues in countries without access to these medications. Worldwide access to the C5 inhibitor eculizumab is hindered by the unavailability of treatment, and in places where treatment is approved, cost of treatment, reimbursement issues, infrastructure, or patient restrictions may further impede access (Risitano et al, Am J Hematol. 2018; Risitano et al, Front Immunol. 2019). PNH treatment places a significant economic burden on healthcare systems. In a number of countries, this has resulted in negative health technology appraisals (HTAs), indicative that optimal care and resource utilization are not being achieved (Coyle et al, Med Decis Making. 2014). New therapies with more convenient modes of administration may have the potential to improve how PNH is clinically treated and may have a positive effect on the economic burden and access to C5 inhibitors globally. Robust data on the real-life burden and cost of PNH are therefore needed to assess the impact of current therapies and establish a baseline for new therapeutic approaches. The data derived from this study will be used to support HTA processes and inform the value of new therapies for PNH. Objectives The COMMODORE Burden of Illness (BOI) study will quantify the direct medical costs (eg, treatment and hospitalization), direct nonmedical costs (eg, travel), and indirect costs (eg, impact on work productivity and family burden) associated with PNH for patients and care providers and determine the impact of PNH on health-related quality of life (HRQoL). Study Design and Methods This is an international, prevalence-based, bottom-up, burden of illness study containing both retrospective and prospective data collection. The study will be overseen by an expert reference group consisting of multidisciplinary stakeholders. The study protocol and materials will be submitted for ethical approval to the University of Chester in the United Kingdom. Physicians will provide information on sociodemographic, clinical, and medical resource utilization using an electronic case record form (eCRF). Through patient and public involvement and engagement, patients, after giving informed consent, will provide further information on the economic and HRQoL impact of PNH by completing patient-reported outcome surveys. Patients from France, Germany, United Kingdom, and China will be included. The study aims to recruit 94 physicians reporting 350 patient eCRFs with an expected return of 140 patient surveys and longitudinal data collection after 6 to 12 months for each patient. Mean per-patient costs, including direct medical and nonmedical costs, and indirect resource utilization will be calculated by multiplying the individual resource utilization with country-specific unit costs. National economic burden will be extrapolated by applying national prevalence estimates of PNH. Additionally, the impact of PNH on HRQoL in patients will be assessed within this study. Summary The COMMODORE BOI study aims to characterize current PNH treatment via quantification of the humanistic and socioeconomic burden at the patient, healthcare system, and societal level to enhance the evidence base for treatment and decision-making in this community. Final results are expected be available by the second quarter of 2022. Disclosures Hampton: F. Hoffmann-La Roche Ltd: Current Employment, Current equity holder in publicly-traded company, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Katz:F. Hoffmann-La Roche Ltd: Current Employment, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Shang:F. Hoffmann-La Roche Ltd: Current Employment, Current equity holder in publicly-traded company, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Dhillon:F. Hoffmann-La Roche Ltd: Other: All authors received editorial support for this abstract, furnished by Scott Battle, funded by F. Hoffmann-La Roche Ltd, Basel, Switzerland. ; HCD Economics: Current Employment. Hubberstey:HCD Economics: Current Employment; Huntingdon's Disease Youth Organization: Membership on an entity's Board of Directors or advisory committees; F. Hoffmann-La Roche Ltd: Other: All authors received editorial support for this abstract, furnished by Scott Battle, funded by F. Hoffmann-La Roche Ltd, Basel, Switzerland. .

Sign in / Sign up

Export Citation Format

Share Document