scholarly journals Impact of Myeloproliferative Neoplasms on Patients' Employment and Income: Findings from the Living with MPN Survey

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2250-2250
Author(s):  
Simon Condliffe ◽  
Jingbo Yu ◽  
Dilan Chamikara Paranagama ◽  
Shreekant Parasuraman
Keyword(s):  
United States ◽  
Employment Status ◽  
Early Retirement ◽  
Myeloproliferative Neoplasms ◽  
Good Health ◽  
The United States ◽  
Employment Equity ◽  
Disability Leave ◽  
Part Time ◽  
Equity Ownership

Abstract Background: Patients with myeloproliferative neoplasms (MPNs), including myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET), experience a high symptom burden that may compromise daily functioning and quality of life. The objective of this analysis was to evaluate income loss associated with disease-related employment changes among patients with MPNs in the United States. Methods: The Living with MPN survey was completed online by adult patients (aged 18-70 years) with MF, PV, or ET in the United States between April and November of 2016. Respondents employed at the time of MPN diagnosis were asked questions about disease-related changes in employment status and salaries occurring between diagnosis and the year of survey (2016). In addition, details related to patient demographics, MPN diagnosis, and MPN-related symptoms were collected. Cumulative income losses as a result of disease-related employment changes up to the time of the survey were calculated based on the timing of employment changes and salaries, which were reported in nominal dollars. Results: Of the 904 survey respondents, 592 (65%) were employed at the time of MPN diagnosis. Among those employed, mean age was 54.0 years, 70.6% were female, and 72.3% were married or had domestic partners at the time of survey. The average duration of disease was 6.1 years (MF, 4.6; PV, 6.9; ET, 6.3). Approximately half (50.5%) of the employed respondents experienced at least one change in employment status because of their diagnosis. Employment status changes and associated impact on income in patients with MPNs was greatest for those who took early retirement, medical disability leave, or left a job due to their disease ($419,610, $169,048, $168,245, respectively). Respondents who changed from full- to part-time employment, reduced hours, or were reassigned to a lower-paying job because of their disease also reported income losses ($79,492, $47,104, $51,872, respectively; Table). Among respondents who were 45-64 years old at the time of the survey (n=383), 18.8% reported retiring early as a result of their disease. In comparison, according to nationally representative data from the Medical Expenditures Panel Survey (MEPS), only 7.8% of individuals aged 45-64 years in excellent or very good health and 9.2% of individuals in poor health reported being retired (longitudinal data set 2014-2015). Moreover, 30.5% (117/383) of respondents aged 45-64 years in the Living with MPN survey reported leaving a job as a result of their disease. In comparison, 5.5% of MEPS individuals aged 45-64 years in excellent or very good health and 16.4% of individuals in poor health were working at the start of 2014 but not by the end of 2015. Conclusions: About half of employed patients living with MPNs experienced a variety of employment changes as a result of their disease, which in turn had a considerable impact on income. The most frequently reported disease-related employment change was leaving a job, followed by medical disability leave, reduced hours, early retirement, switching from full-time to part-time, and being reassigned to a lower-paying job. Patients 45-64 years old with MPNs were more than twice as likely to have left a job or retired early compared with an age-matched US general population cohort. On average, the foregone income due to disease-related employment changes was greatest for early retirees ($419,610), followed by those who went on medical disability leave ($169,048), and left a job ($168,245). Early, effective management of MPNs and associated symptoms may help patients avoid these disease-related changes to their employment status and the subsequent economic and financial impact. Disclosures Condliffe: Incyte Corporation: Consultancy. Yu:Incyte Corporation: Employment, Equity Ownership. Paranagama:Incyte: Employment, Equity Ownership. Parasuraman:Incyte: Employment, Equity Ownership.

scholarly journals Impact of Myeloproliferative Neoplasms on Patients' Employment Status and Work Productivity in the United States: Results from the Living with MPN Patient Survey

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4256-4256 ◽  
Author(s):  
Jingbo Yu ◽  
Shreekant Parasuraman ◽  
Dilan Paranagama ◽  
Ahmad Naim ◽  
David Dubinski ◽  
...  
Keyword(s):  
Employment Status ◽  
Research Funding ◽  
Myeloproliferative Neoplasms ◽  
Work Productivity ◽  
The United States ◽  
Work Time ◽  
Employment Equity ◽  
Activity Impairment ◽  
Equity Ownership ◽  
The Mean

Abstract Background: Patients with myeloproliferative neoplasms (MPNs), including myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET), report high symptom burden that may compromise activities of daily living and quality of life. Results from the MPN Landmark survey suggest that for gainfully employed patients, disease symptoms may impact work productivity or career opportunities. The objective of this study was to assess the effects of MPNs on employment status, work productivity, and daily activities of patients in the United States. Methods: Adult patients (18-70 years) diagnosed with MF, PV, or ET participated in an online survey conducted between April and July 2016. The survey asked about diagnosis, disease-related history, MPN-related symptoms, functional status, changes in employment status since diagnosis due to MPNs, and work productivity (assessed using the Work Productivity and Activity Impairment Specific Health Problem Questionnaire, WPAI-SHP V2). Descriptive statistics were used to report MPN-related symptoms, employment changes, and WPAI scores for patients with MPNs who were employed at diagnosis and currently. Results: A total of 595 patients (MF, n=148; PV, n=284; ET, n=163) completed the survey, with 387 (65.0%) employed full- or part-time at diagnosis (MF, n=90; PV, n=178; ET, n=119). Among patients who were employed at diagnosis, mean (SD) age was 54.3 (10.5) years, 70.0% were female, and 47.8% had a bachelor's degree or higher. Mean (SD) duration of disease was 6.4 (6.6) years. After diagnosis, almost half of the patients (49.9%) experienced ≥1 change in employment resulting from their MPN, one-third (34.1%) had ≥2 different types of changes, and almost one-quarter (22.7%) had ≥3 different types of changes (Table). Almost one-third of patients (31.3%) reported 'leaving a job' due to their condition, which was also the most common employment change. The mean (SD) time from diagnosis to first job change due to MPN disease was 2.4 (5.2) years. Among those currently employed, the mean percentage of overall effects on work productivity due to MPNs were 29.3% for work impairment (MF, 28.4%; PV, 30.0%; ET, 28.6%), 25.7% for impairment while at work (presenteeism; MF, 22.1%; PV, 26.5%; ET, 26.4%), and 6.4% for work time missed (absenteeism; MF, 9.4%; PV, 6.6%; ET, 4.9%). The mean (SD) number of hours of work missed per week was 2.0 (4.9). The mean percentage of daily activity impairment was 31.6% (MF, 32.1%; PV, 32.0%; ET, 30.8%). The effects of MPNs on work productivity and daily activities are similar to those reported by patients with other significant chronic conditions such as rheumatoid arthritis (work time missed, 19.1%; impairment while working, 24.0%; activity impairment; 33.3%; Bansback N, et al. Rheumatology. 2012;51:375-84). Conclusion: MPNs have a substantial negative impact on patients' employment status and work productivity. Half of the employed patients with MPNs surveyed had a change in employment status (eg, leaving job, medical disability leave, early retirement) due to their disease. Moreover, currently employed patients reported a meaningful loss in work productivity due to their MPNs. Disclosures Yu: Incyte Corporation: Employment, Equity Ownership. Parasuraman:Incyte Corporation: Employment, Equity Ownership. Paranagama:Incyte Corporation: Employment, Equity Ownership. Naim:Incyte Corporation: Employment, Equity Ownership. Dubinski:Incyte Corporation: Employment, Equity Ownership. Bai:Incyte Corporation: Employment, Equity Ownership. Mesa:Novartis: Consultancy; Galena: Consultancy; Ariad: Consultancy; Incyte Corporation: Research Funding; Promedior: Research Funding; Gilead: Research Funding; Celgene: Research Funding; CTI: Research Funding.

Registry Of Patients Treated With Protein C Concentrate (Human) In The United States and Europe: Interim Results

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 1146-1146
Author(s):  
Marilyn J. Manco-Johnson ◽  
Paul Knoebl ◽  
Amy D. Shapiro ◽  
Maureen Finnerty ◽  
Leman Yel ◽  
...  
Keyword(s):  
United States ◽  
Board Of Directors ◽  
Research Funding ◽  
Protein C ◽  
The United States ◽  
Employment Equity ◽  
Advisory Committees ◽  
Protein C Concentrate ◽  
Concentrate Treatment ◽  
Equity Ownership

Abstract Background Protein C (PC) is a naturally occurring, vitamin-K dependent anticoagulant produced by the liver. Deficiency of PC, which can be congenital or acquired, results in a hypercoagulable state. Patients with severe PC deficiency may manifest severe, often life-threatening, disseminated intravascular coagulation (DIC), purpura fulminans (PF), and/or thromboembolism. Highly purified protein C concentrate prepared from human donor plasma (Ceprotin; Baxter Healthcare Corporation, Westlake Village, CA) is an approved therapy for prevention and treatment of venous thrombosis and PF in patients with severe congenital PC deficiency (SCPCD) in the United States. It is indicated in patients with SCPCD for PF and coumarin-induced skin necrosis, and short-term prophylaxis in the European Union and other countries. Use of protein C concentrate (human) has been reported in patients with acquired PC deficiency. The Ceprotin Treatment Registry is a prospective, international, multi-center, open-label, non-interventional, observational study designed to examine the long-term safety and effectiveness of protein C concentrate (human) in the clinical setting. This is the first large, real-world assessment of the treatment of a rare disease with protein C concentrate (human). Here we report data from the first interim analysis in June 2013 covering a 3-year enrollment period. Methods Patients of any age who received, or were initiating/receiving protein C concentrate are included. The study duration is 5 years (3-year enrollment period plus 2 years of follow-up). All study visits/assessments are in accordance with standard of care, with protein C concentrate dose, dose frequency, duration and route of administration determined by the investigator. The study objectives are to determine the most common medical diagnoses associated with protein C concentrate treatment, protein C concentrate treatment regimens, and safety information based on all serious adverse events (SAEs) and rate of treatment-related AE. Other objectives include: an examination of the relationship between protein C concentrate treatment and outcomes in all registry participants, as well as various subgroups. Descriptive statistical analyses are used. Results At the time of data extraction, 34 patients were enrolled from 26 centers; 10 centers in the United States and 16 centers in Europe. Half of the patients were males. The primary diagnosis of PC deficiency was congenital in 25 patients (73.5%) and acquired in 9 patients (26.5%). The median age at diagnosis was 0.03 years (range 0–19.9). Mean plasma PC activity level was 9% (range 1–40.0). The most common (in ≥3 patients) thrombotic disease-associated conditions were PF (50.0%); blindness (44.1%); thromboembolic disease (41.2%), which included deep vein thrombosis, arterial thrombosis and DIC; stroke (32.4%), and renal dysfunction (8.8%). Of the 23 patients being treated with protein C concentrate, 15 were administered protein C concentrate intravenously, and 8 patients received protein C concentrate subcutaneously. The body weights of patients receiving subcutaneous treatment ranged from 10.0 kg to 57.9 kg. A total of 15 patients received an anticoagulant treatment in addition to protein C concentrate. Eight patients reported 23 SAEs, all of which were considered not related to treatment. Eighteen (52.9%) patients reported 72 AEs; only 1 of them, an episode of upper respiratory infection, was considered possibly treatment-related. In 18 patients in whom PC activity recovery was determined after protein C concentrate treatment, there were no patients documented with poor recovery. Protein C concentrate was used during 18 surgeries/invasive procedures and considered effective in all interventions for which data were available. Conclusions Data from the first interim analysis of the Ceprotin Treatment Registry demonstrate that patients with both congenital and acquired severe PC deficiency who are treated with protein C concentrate (human) have a low incidence of treatment related SAEs and AEs, and treatment with protein C concentrate appears to be effective when used during surgery/invasive procedures. Further patient follow up will shed light onto clinical treatment outcomes. Disclosures: Manco-Johnson: CSL Behring: Membership on an entity’s Board of Directors or advisory committees, Research Funding; Novo Nordisk: Membership on an entity’s Board of Directors or advisory committees; Biogen Idec: Membership on an entity’s Board of Directors or advisory committees; Baxter BioScience: Membership on an entity’s Board of Directors or advisory committees; Bayer HealthCare: Membership on an entity’s Board of Directors or advisory committees, Research Funding; Eisai: Research Funding. Knoebl:Novo Nordisk: Consultancy, Honoraria; Baxter: Consultancy, Honoraria. Shapiro:Cangene: Research Funding; Biogen Idec: Research Funding; Baxter BioScience: Research Funding; Bayer HealthCare: Research Funding. Finnerty:Baxter: Employment, Equity Ownership. Yel:Baxter: Employment, Equity Ownership. Gelmont:Baxter: Employment, Equity Ownership.

Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Following Hematopoietic Stem Cell Transplantation or Chemotherapy--Results from the Italian Therapeutic Use Protocol

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3107-3107 ◽  
Author(s):  
Franco Locatelli ◽  
Maura Faraci ◽  
Simone Cesaro ◽  
Daria Pagliara ◽  
Franca Fagioli ◽  
...  
Keyword(s):  
United States ◽  
Distress Syndrome ◽  
Treatment Protocol ◽  
The United States ◽  
Therapeutic Use ◽  
Occlusive Disease ◽  
Sinusoidal Obstruction Syndrome ◽  
Employment Equity ◽  
Hematopoietic Stem ◽  
Equity Ownership

Introduction Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is an unpredictable, potentially life-threatening complication of chemotherapy (CT) given as conditioning for hematopoietic stem cell transplantation (HSCT), or as primary treatment. Severe hepatic VOD/SOS, traditionally defined by occurrence of multi-organ dysfunction/failure (MOD/MOF), may be associated with mortality rates >80%. In the European Union, defibrotide is approved for treatment of severe VOD/SOS post-HSCT. It is not approved in the United States, but is available through an expanded-access program. Here, we report the results of an Italian therapeutic use program, defibrotide treatment protocol (TUT), in which defibrotide was provided upon physician request for treatment of patients with VOD/SOS. Methods This was a multicenter, single-arm, open-label program of defibrotide treatment in patients with hepatic VOD/SOS, with or without MOD/MOF, from 2010-2014. Patients were eligible if they were diagnosed with VOD/SOS having met ≥2 of the following criteria: bilirubin (>2 mg/dL), ascites, unexplained weight gain >5% above baseline (weight on day 1 of HSCT conditioning or CT), or hepatomegaly. Patients with biopsy-proven VOD/SOS were also eligible. Exclusion criteria were use of anticoagulants (beyond those for IV line maintenance), significant uncontrolled acute bleeding (transfusions after dialysis were allowed), pregnancy, and hemodynamic instability requiring >2 vasopressor drugs. MOD/MOF was defined as renal (creatinine >3x baseline, creatinine clearance or glomerular filtration rate <40% of baseline, or dialysis dependence) or pulmonary (oxygen saturation <90% on room air, requiring oxygen supplementation, or ventilator dependence) dysfunction. Patients were treated with defibrotide 25 mg/kg/d in 4 divided doses given intravenously over 2 hours each. Outcomes included survival at day +100 post-HSCT or post-CT and adverse events (AEs). VOD/SOS and MOD/MOF were not reported as AEs unless the event was considered serious. Demographics and AEs were analyzed using descriptive statistics; efficacy was estimated by the SAS/STAT LifeTest Procedure. Results A total of 98 patients had hepatic VOD/SOS (92% had VOD/SOS post-HSCT [81% allogeneic, 10% autologous, 1% unknown], 8% post-CT); of these, 21% had MOD/MOF and 79% did not. Median age was 13.4 years (range 0-68; 57 [58%] aged <18 years and 41 [42%] ≥18 years), 55% were male, and 65% were white. The most common primary diseases were acute myelogenous leukemia (25%) and acute lymphoblastic leukemia (19%). The most common prophylaxis regimens for graft-vs-host disease (GvHD) were reported as cyclosporine + methotrexate (38%), cyclosporine (12%), and cyclosporine + methotrexate + other (12%); tacrolimus- and sirolimus-containing regimens, which have been associated with development of VOD/SOS, were received by 5% and 4% of patients, respectively. The median defibrotide dose was 25 mg/kg/d (range, 6.15-40.0) and median duration of treatment was 14 days (range: 1-84). The estimated day +100 survival for all patients was 68.4% (95% CI, 58.0%-76.7%). Day +100 survival in post-HSCT patients was 67.0% (95% CI, 56.2%-75.8%) and for post-CT patients was 85.7% (95% CI, 33.4%-97.9%). Across the entire study, 38 deaths were reported; primary causes were progression of VOD/SOS and MOD/MOF (44.7%), infection (21.1%), GvHD (15.8%), and disease progression (13.2%). Treatment-emergent AEs were reported in 21% of patients in this therapeutic use program; the most common (reported in ≥2 patients) were MOD/MOF (9%), VOD/SOS (6%), and cytomegalovirus infection and acute respiratory distress syndrome (2% each). Treatment-related AEs were reported by 5% of patients, and included hemorrhagic cystitis, urinary tract hemorrhage, hemorrhages (unspecified), hemorrhagic diathesis, and pulmonary hemorrhage (1 patient each). Serious AEs were reported in 15% of patients, most commonly MOD/MOF (7%), VOD/SOS (4%), and acute respiratory distress syndrome (2%). Conclusion In the Italian therapeutic use program, defibrotide treatment protocol (TUT), approximately one-fifth of the patients with hepatic VOD/SOS had MOD/MOF. The estimated survival rate of 68.4% at day +100, and the AE profile, was consistent with efficacy and safety data reported in previous studies in similar VOD/SOS patient populations. Support: Jazz Pharmaceuticals. Disclosures Off Label Use: Defibrotide is an investigational treatment for hepatic veno-occlusive disease/sinusoidal obstruction syndrome in the United States.. Amber:Jazz Pharmaceuticals: Employment, Equity Ownership. Banerjee:Jazz Pharmaceuticals: Employment, Equity Ownership. Finetto:Gentium S.p.A.: Employment; Jazz Pharmaceuticals: Equity Ownership.

scholarly journals Disease Characteristics of Minority Patient Populations with Polycythemia Vera: An Analysis from the Reveal Study

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4735-4735 ◽  
Author(s):  
Ivy Altomare ◽  
Philomena Colucci ◽  
Shreekant Parasuraman ◽  
Dilan Chamikara Paranagama ◽  
Anas Al-Janadi
Keyword(s):  
United States ◽  
High Risk ◽  
The United States ◽  
Low Risk ◽  
Employment Equity ◽  
Disease Characteristics ◽  
Reveal Study ◽  
High Risk Disease ◽  
Caucasian Patients ◽  
Equity Ownership

Abstract Introduction: Polycythemia vera (PV) is associated with increased blood cell counts, risk of thrombosis, and symptoms including fatigue and pruritus. Few studies have examined the presence or absence of racial/ethnic disparities among patients with PV. The objective of this analysis is to describe differences in disease characteristics, diagnosis, treatment, and quality of life (QOL) among Caucasian and non-Caucasian patients with PV in the United States enrolled in the prospective, observational REVEAL study. Methods: The ongoing REVEAL study (ClinicalTrials.gov ID, NCT02252159) is a prospective, multicenter, observational study of adult patients with PV in the United States. Patients were observed during a 36-month period, during which clinical data were collected from usual care visits. This analysis compared demographics, disease and clinical characteristics, disease management, comorbidities, and QOL between Caucasian and non-Caucasian patients with PV at enrollment. QOL was measured by the European Organisation for Research and Treatment of Cancer Questionnaire C30 (EORTC QLQ-C30) and Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS). Results are summarized with descriptive statistics. Results: Of the 2,510 patients enrolled in REVEAL, 2,237 were Caucasian (89.1%); 199 (7.9%) were non-Caucasian, comprised of African American (5.7%), Asian (1.5%), Native American Indian (0.2%), Pacific Islander (0.1%), and other patients (0.4%); no information was provided regarding race or ethnicity for 74 patients (2.9%). Baseline disease characteristics were similar for Caucasian and non-Caucasian groups with respect to gender and disease duration. There were no differences in method of diagnosis, laboratory values, or overall history of thrombosis between groups (Figure 1A). Mean age was higher among Caucasian patients compared to non-Caucasian patients (66.6 vs 63.8 years, respectively). The proportion of patients from rural areas was higher among Caucasian vs non-Caucasian patients (28.8% vs 12.6%); similarly, the proportion of patients from urban areas was lower among Caucasian vs non-Caucasian patients (23.1% vs 46.7%). The proportion of patients with some college or higher level of education was higher among Caucasian vs non-Caucasian patients (64.1% vs 50.3%). A higher proportion of Caucasian vs non-Caucasian patients were retired (52.0% vs 43.2%); a higher proportion of non-Caucasian patients reported being unable to work or were disabled (3.8% vs 10.1%). More Caucasian patients had high-risk disease (78.0%) compared with non-Caucasian patients (71.4%), and patients with high-risk disease were managed similarly between groups. However, Caucasian patients with low-risk disease received more phlebotomies (56.6%) than non-Caucasian patients with low-risk disease (40.4%), and over twice as many non-Caucasian patients received hydroxyurea (38.6%) than Caucasian patients (15.6%) (Figure 1B). MPN-SAF TSSs were higher for non-Caucasian patients compared with Caucasian patients, suggesting a worse symptom burden. Similarly, non-Caucasian patients reported lower functional and symptom outcomes on the EORTC QLQ-C30, including a disparity in financial difficulties, compared to Caucasian patients (Figure 1C). Conclusions: This analysis evaluated a cohort of racial/ethnic minority patients with PV treated in the United States. As in other cancer-related trials, there is a risk that racial and ethnic minorities may be underrepresented in REVEAL. With this limitation in mind, in this analysis, differences were not observed among Caucasian and non-Caucasian patients with respect to method of diagnosis, duration of disease, thrombosis rates, or management of high-risk disease. Non-Caucasian patients demonstrated higher rates of low-risk disease and cytoreductive therapy for low-risk disease yet had worse symptom burden, lower functional scores, and greater disability. This study underscores the importance of symptom assessment and ancillary resource availability for patients with PV Disclosures Altomare: Bayer: Consultancy; Genentech: Consultancy; Ipsen: Other: Advisory Board Member; Celgene: Other: Advisory Board Member; Incyte: Consultancy; Novartis: Consultancy; Amgen: Consultancy. Colucci:Incyte: Employment, Equity Ownership. Parasuraman:Incyte: Employment, Equity Ownership. Paranagama:Incyte: Employment, Equity Ownership.

scholarly journals Persistence of Ruxolitinib Treatment in Patients with Myelofibrosis at Community Oncology Practices in the United States-Contemporary Data

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5385-5385
Author(s):  
Jingbo Yu ◽  
Jonathan K. Kish ◽  
Dilan Paranagama ◽  
Philomena Colucci
Keyword(s):  
United States ◽  
High Risk ◽  
Chart Review ◽  
The United States ◽  
Current Analysis ◽  
First Line ◽  
Employment Equity ◽  
Persistence Rates ◽  
Equity Ownership

Introduction Myelofibrosis (MF) is a chronic myeloproliferative neoplasm frequently associated with debilitating disease-related symptoms and shortened overall survival (OS). Ruxolitinib, a Janus kinase (JAK)1/JAK2 inhibitor, was approved on November 16, 2011 for patients with intermediate- and high-risk MF. The phase 3 COMFORT trials demonstrated that ruxolitinib reduced spleen volume, improved MF-related symptoms, and prolonged OS. In the COMFORT trials, the median duration of ruxolitinib treatment was 2.8 years (5-year pooled analysis). The objective of this real-world chart review study is to describe persistence of ruxolitinib treatment early on in the treatment course in patients with MF at community hematology/oncology practices in the United States. Methods Medical charts from community hematology/oncology practices in Cardinal Health Oncology Provider Extended Network (OPEN) were reviewed and abstracted into electronic case report forms by physicians. Physicans were instructed to abstract data from the earliest eligible patients and then chronologically, thereafter. This analysis included adult patients diagnosed with primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF between 01/2012 and 12/2016. Patients were required to have intermediate- or high-risk MF (based on a data-derived International Prognostic Scoring System [IPSS] risk categorization), have platelet count >50 × 109/L at the time of diagnosis, have received ruxolitinib as first-line treatment, and have ≥6 months of follow up after the initiation of ruxolitinib; patients who died within 6 months of initiation of ruxolitinib were considered as discontinued patients and were included in the analysis. Three- and 6-month persistence rates are reported. Results A total of 141 patients from 22 community hematology/oncology practices were included in the analysis; 3 patients who were still on ruxolitinib at the end of the study period, but with less than 6 months of follow up, were excluded from the analysis. Mean (SD) age at diagnosis was 69.8 (10.2) years, 61.7% were male, 71.6% had primary MF, and 56.7% were high-risk at diagnosis. Median time from diagnosis to ruxolitinib initiation was 36 (interquartile range [IQR], 20-91) days. The majority of patients initiated ruxolitinib in 2016 or later (Figure). The starting dose of ruxolitinib was 5 mg twice daily [BID] in 7.8%, 10 mg BID in 9.9%, 15 mg BID in 39.7%, 20 mg BID in 34.8%, and 25 mg BID in 7.8%. Three- and 6-month persistence rates were 97.2% and 91.5%, respectively (Table). Discussion A previous retrospective chart review of prescription claims and patient level data evaluated a cohort of patients with MF prescribed ruxolitinib just prior to and shortly after its approval (November 1, 2011 to August 31, 2012); in that study, 3- and 6-month persistence rates of 39.1% and 27.0%, respectively, were reported (Blood. 2013[122]:2833). In the current study, the 3- and 6-month persistence rates were notably higher at 97.2% and 91.5%, respectively. These higher rates are likely attributable to a number of factors. The current analysis was limited to patients treated first-line with ruxolitinib. Furthermore, the index period in the current analysis extended almost 5 years beyond ruxolitinib approval; in this 5-year period, there were a number of publications in support of ruxolitinib dose optimization with particular focus on the first 8-12 weeks of treatment (J Hematol Oncol. 2013[6]:79; Int J Hematol. 2016[104]4:420-9). The 3- and 6-month persistence rates reported here demonstrate that only a small proportion of patients with myelofibrosis treated with first-line ruxolitinib discontinued the drug early on in their treatment course. Conclusion This contemporary analysis demonstrates high rates of 3- and 6-month persistence of first-line ruxolitinib in patients with MF treated in community practices. Proper monitoring and management of the expected mechanism-based toxicity of a JAK inhibitor and proper dose titration for insufficient response, early in the treatment course, may help patients derive maximum, long-term benefits from ruxolitinib. Disclosures Yu: Incyte: Employment, Equity Ownership. Kish:Cardinal Health: Employment. Paranagama:Incyte: Employment, Equity Ownership. Colucci:Incyte: Employment, Equity Ownership.

An Adjusted Treatment Comparison of Bortezomib/Cyclophosphamide /Dexamethasone and Bortezomib/Thalidomide/Dexamethasone from Real-World Data in Patients with Newly Diagnosed Multiple Myeloma Who Are Transplant-Eligible

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2142-2142
Author(s):  
Maneesha Mehra ◽  
Sarah Cote ◽  
Tobias Kampfenkel ◽  
Sandhya Nair
Keyword(s):  
United States ◽  
Multiple Myeloma ◽  
Real World ◽  
The United States ◽  
Induction Treatment ◽  
Newly Diagnosed ◽  
Real World Data ◽  
Employment Equity ◽  
Equity Ownership ◽  
Baseline Characteristics

Introduction: For patients with newly diagnosed multiple myeloma (NDMM) who are eligible for autologous stem-cell transplantation (ASCT), two standard of care (SoC) induction regimens are bortezomib/cyclophosphamide/dexamethasone (VCd) and bortezomib/thalidomide/dexamethasone (VTd), each followed by ASCT. While VCd and VTd are both treatment options according to international guidelines, treatment selection varies by country. Additionally, while some clinical studies have evaluated the efficacy and safety of these therapies, direct comparisons have been limited to response endpoints post-induction and post-transplant (Moreau P, et al. Blood. 2016;127[21]2569-2574; Cavo M, et al. Blood. 2014;124[21]197; Cavo M, et al. Leukemia. 2015;29[12]2429-31). Herein we describe real-world treatment patterns in the United States for patients with NDMM who are transplant-eligible, and report results from a matched adjusted comparison to evaluate real-world long-term efficacy (overall survival, OS) for VCd +ASCT versus VTd +ASCT. Methods: Data for the VCd and VTd real-world evidence (RWE) cohorts were identified from 3 US data sources collectively covering the period January 2000 to March 2017: the OPTUM™ Commercial Claims database, the OPTUM™ Integrated (CLAIMS+EMR) database, and the Surveillance, Epidemiology, and End Results (SEER)-Medicare Linked database. RWE data were from patients with an index MM diagnosis on or after 1 January 2007, medical prescription coverage in place at diagnosis, no prior malignancies in the 1-year period prior to index diagnosis, a 1-year look-back period prior to index diagnosis, received ≥1 line of therapy, and received stem cell transplantation with induction as frontline treatment. The Kaplan-Meier method and Cox proportional hazard model compared outcomes with and without adjustments for baseline characteristics (age, sex, renal impairment, and anemia) and induction treatment duration; comparisons were also conducted with inverse probability of treatment weighting (IPTW). Results: Analysis of RWE from the United States demonstrated that bortezomib (V)-based regimens were the most common induction treatment (together accounting for approximately 75% of therapies), with bortezomib/lenalidomide/dexamethasone (VRd) being the most common (31%). Use of VCd (13%) and VTd (5%) was limited. Comparisons were conducted for VCd (n = 135) and VTd (n = 51). Baseline characteristics were generally similar between groups, except for fewer male patients in the VCd group than the VTd group (57% vs 65%), and lower rates of renal impairment in the VCd group than the VTd group (29% vs 43%; Table 1). The naïve and adjusted comparisons of OS for VCd versus VTd therapy showed these treatments were not statistically different (adjusted hazard ratio, 1.180 [95%: 0.468-2.972]; P = 0.7260; Figure 1). The IPTW method generated similar results. Conclusions: Real-world data from the United States show that V-based induction regimens are the most commonly used for treatment of patients with NDMM who are transplant-eligible. Results from the naïve, adjusted, and IPTW comparisons all showed that OS was not significantly different for VCd + ASCT versus VTd + ASCT. Survival data for VTd from RWE are generally consistent with VTd data reported in the recent phase 3 CASSIOPEIA study, although OS data from CASSIOPEIA remain immature (Moreau P, et al. Lancet. 2019;394[10192]:29-38). Disclosures Cote: Janssen: Employment, Equity Ownership. Kampfenkel:Janssen: Employment, Equity Ownership. Nair:Janssen: Employment, Equity Ownership.

scholarly journals Impact of Post-Hematopoietic Cell Transplant (HCT) Survival on Cost-Effectiveness of CPX-351 Versus 7+3 in the Treatment of Therapy-Related AML or AML-MRC in the United States

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 5837-5837
Author(s):  
Anuraag Kansal ◽  
Odette Reifsnider ◽  
Lora Todorova ◽  
Anna Coughlan ◽  
Kathleen F. Villa
Keyword(s):  
United States ◽  
Cost Effectiveness ◽  
Life Expectancy ◽  
The United States ◽  
Cell Transplant ◽  
Employment Equity ◽  
Treatment Pathways ◽  
Life Years ◽  
Equity Ownership ◽  
The Impact

Abstract Background: A central goal of treatment for high-risk AML is enabling patients to receive HCT. CPX-351 is a dual-drug liposomal encapsulation of cytarabine and daunorubicin at a synergistic ratio. A large randomized, open-label, multicenter, phase 3 trial demonstrated superior outcomes, including significantly longer overall survival and a larger fraction of patients reaching HCT, for CPX-351 versus conventional cytarabine/daunorubicin (7+3 regimen) in patients aged 60 to 75 years with newly diagnosed therapy-related AML (tAML) or AML with myelodysplasia-related changes (AML-MRC; Lancet JE, et al. J Clin Oncol. 2018). In order to estimate the cost-effectiveness of treatments for AML, and for CPX-351 in particular, it is necessary to extrapolate survival of patients undergoing HCT to the long-term. The objective of this analysis was to assess the impact of different estimates of post-HCT mortality on cost-effectiveness of CPX-351. Methods: An existing economic model of CPX-351 versus 7+3 for the treatment of tAML or AML-MRC was used to project lifetime health and cost outcomes. The modeled population included 30% patients <60 years of age and 70% between 60 and 75 years of age. The model uses a survival-partition approach, with distinct survival curves for cohorts defined by treatment pathways (response and transplantation). Projected life expectancy for each cohort was estimated based on trial data (for those 60-75 years of age), US population life-tables, and published data. For patients younger than age 60, response rates were adjusted based on published literature. Standardized mortality ratio (SMR) values following HCT were selected individually for the younger and older cohorts. SMR values were calibrated in the model to match the literature finding that post-HCT mortality results in a loss of approximately 30% of life expectancy. This yielded an SMR of 2.25 for older patients and 4.0 for younger patients. Values ranged from 1.0 to 4.0 in sensitivity analyses based on recent appraisals by the United Kingdom's National Institute for Health and Care Excellence (NICE). Model outputs included survival, quality-adjusted life-years (QALYs), costs, and incremental cost-effectiveness ratio (ICER). Results: In the base case with different SMR values by age, the model yielded a predicted ICER value of $111,841/QALY in the overall cohort, which compares favorably to the typical $150,000/QALY threshold (Bae 2014) for good value care in the United States. Using the same estimates of SMR in all patients yielded ICER values of $125,280/QALY and $151,793/QALY for SMR values of 2.25 and 4.0, respectively. Assuming no excess mortality post-HCT relative to the general population, using an SMR of 1.0, resulted in modest improvements in cost-effectiveness (ICER = $102,298/QALY). Conclusions: CPX-351 is a cost-effective option for the treatment of patients with tAML or AML-MRC when accounting for potentially increased mortality following HCT. Disclosures Kansal: Jazz Pharmaceuticals: Consultancy. Reifsnider:Jazz Pharmaceuticals: Consultancy. Todorova:Jazz Pharmaceuticals: Employment, Equity Ownership. Coughlan:Jazz Pharmaceuticals: Consultancy. Villa:Jazz Pharmaceuticals: Employment, Equity Ownership, Other: Stock and stock options.

scholarly journals Impact of Myeloproliferative Neoplasms (MPNs) on Patient Employment and Income: Findings From the Living with MPNs Survey

2019 ◽  
Author(s):  
Simon Condliffe ◽  
Jingbo Yu ◽  
Dilan Paranagama ◽  
Shreekant Parasuraman
Keyword(s):  
Disease Management ◽  
Chronic Conditions ◽  
Employment Status ◽  
Early Retirement ◽  
Myeloproliferative Neoplasms ◽  
Employment Change ◽  
Income Loss ◽  
Disability Leave ◽  
Before And After

Abstract Background The objective of this analysis was to evaluate income loss resulting from disease-related employment changes among patients with myeloproliferative neoplasms (MPNs). Methods Patients aged 18 to 70 years diagnosed with an MPN were eligible to participate in the online Living with MPNs survey (April–November, 2016). Respondents employed at the time of diagnosis were asked questions about changes in employment status related to their MPN and their salaries before and after those changes. Cumulative income loss resulting from disease-related employment changes up to the time of the survey was calculated based on the timing of changes in employment and salary, which was reported in inflation-adjusted 2018 US dollars. Results Among 904 patients with an MPN who responded to the survey, 299 were employed at the time of MPN diagnosis and reported ≥1 disease-related employment change. For nearly half (144/299 [48.2%]) of employed patients who reported a change in employment status, the first change occurred within 1 year of diagnosis, and most (229/299 [76.6%]) had ≥1 change within 2 years. Employment status changes and the associated impact on income were greatest among patients who took early retirement ($517,866 lost income), followed by those who took medical disability leave ($289,910) or left a job because of their disease ($257,178). Conclusions Disease-related employment changes contributed to considerable income loss among patients with MPNs. Similar to other chronic conditions, potential exists for disease-related employment changes and income loss among patients with MPNs that should be considered during disease management.

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