Epidemiology of Myeloproliferative Disorders in US - a Real World Analysis.

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 2834-2834
Author(s):  
Ruben A. Mesa ◽  
Jyotsna Mehta ◽  
Hongwei Wang ◽  
Yanxin Wang ◽  
Usman Iqbal ◽  
...  
Keyword(s):  
Real World ◽  
Research Funding ◽  
Myeloproliferative Neoplasms ◽  
Age Distribution ◽  
Employment Equity ◽  
Prevalence Estimates ◽  
The Us ◽  
Equity Ownership ◽  
Estimate Prevalence ◽  
Adjusted Incidence

Abstract Abstract 2834 Background: Myelofibrosis (MF), Polycythemia Vera (PV) and Essential Thrombocythemia (ET) are 3 classic BCR ABL negative myeloproliferative neoplasms. MF is comprised of both primary myelofibrosis (PMF) and those with an antecedent PV or ET (Post PV and Post ET MF, respectively). The term secondary MF (SMF) when used sometimes includes those with histologic myelofibrosis without an MPN (i.e. secondary to another myeloid neoplasm or autoimmune conditions) as well as MPN associated MF (PMF and Post PV/ET MF). Limited published epidemiology data is available on these disorders. Literature reports MF incidence in the range of 0.4–1.4 per 100000. The objective of this study was to estimate prevalence and incidence using 2 real world US health insurance claims databases. Method: The US IMPACT® (∼30M enrollees/year) and US MARKETSCAN (∼40M enrollees/year) were used to retrospectively identify unique patients with PMF, SMF, PV and ET between 1/1/08 and 12/31/10. These databases can track patients longitudinally over multiple years, are linked at the patient level by a unique identifier that is consistent across services, health plans, and time and shown to be representative of the US population. ICD9 CM codes were used to identify PMF, SMF, PV and ET. Any MF was defined as having a diagnosis of either PMF or SMF. Incident cohort was constructed by selecting patients who had their first MF (or PV or ET) in 2010 confirmed by absence of diagnosis 3 years prior. Prevalent cohort was constructed by identifying patients who had MF (or PV or ET) in a given year. From the MF cohort, post ET and post PV patients were identified as those who had any ET or PV diagnosis in the calendar year prior. Primary MF was defined as PMF diagnosis alone without prior PV or ET. Annual prevalence rates were calculated for each calendar year from 2008–10. Continuous enrollment was required during the time period being evaluated. Estimates were adjusted for age distribution from census data. Result: About 8.5M enrollees from IMPACT and 17M from MARKETSCAN each year were included in prevalence estimates. Mean age of MF patients was 61 years in IMPACT and 67 in MARKETSCAN. Age adjusted incidence estimates of MF were about 2.4 per 100000 per year using IMPACT database, 1.7 per 100000 per year from MARKETSCAN. There is a slight increase in MF prevalence over time: from 4.9 to 5.6 in IMPACT, 3.6 to 4.2 in MARKETSCAN. Among MF patients, about 35–40% had PMF, 10% were post-PV patients, 15% were post-ET patients and the remaining had MF arising from myelodysplastic syndrome (∼30%), lupus (∼2%) and other heme and solid tumors (∼3%). Mean age of PV patients was 55 and 36% were female in IMPACT database. In MARKETSCAN, mean age was 58 with 35% being female. Using IMPACT, annual age adjusted prevalence for PV was 57 cases per 100000. Estimates using MARKETSCAN varied from 45 to 48 cases per 100000. Age adjusted incidence of PV ranges from 22 to 27 cases per 100000 patients per year. Mean age of ET patients was 53 and 57 years in each database. In both, 67% of patients were female. Using US IMPACT, annual age adjusted prevalence for ET was 54 to 56 cases per 100000. Estimates using MARKETSCAN varied from 39 to 44 cases per 100000. Age adjusted incidence of ET ranges from 22 to 31 cases per 100000 patients per year. Conclusion: This is the first study utilizing two large national US claims databases to estimate prevalence of MPN disorders. In the US, MF prevalence ranges from 3.6–5.7 per 100,000 patients. Incidence estimates of MF range from 1.7–2.4 per 100,000 patients. PV prevalence estimates range from 45–57 cases and ET prevalence ranges from 39–57 cases per 100,000 patients. PV tends to affect more males while ET affects more female. These data provide compelling evidence to suggest that prevalence and burden of MPN associated MF is higher than has been reported in the past. Additional research using other national databases and/or study designs is needed to substantiate these findings. Disclosures: Mesa: Incyte: Research Funding; Lilly: Research Funding; Sanofi: Research Funding; NS Pharma: Research Funding; YM Bioscience: Research Funding. Mehta:Sanofi: Employment. Wang:Sanofi: Employment, Equity Ownership. Iqbal:Sanofi: Employment, Equity Ownership. Neumann:Sanofi-aventis: Employment. Zhang:Sanofi-aventis: Employment.

Analysis of the Impact and Burden of Illness of Polycythemia Vera (PV) and Essential Thrombocythemia (ET) in the US

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 2071-2071
Author(s):  
Jyotsna Mehta ◽  
Hongwei Wang ◽  
Usman Iqbal ◽  
Ruben A. Mesa
Keyword(s):  
Annual Cost ◽  
Research Funding ◽  
Burden Of Illness ◽  
Control Group ◽  
Employment Equity ◽  
Patient Demographics ◽  
The Us ◽  
Average Annual Cost ◽  
Equity Ownership ◽  
Hospital Days

Abstract Abstract 2071 Background: PV and ET are clonal stem cell diseases belonging to the 3 BCR ABL negative myelopoliferative neoplasms. Patients with PV and ET suffer from splenomegaly and disease associated symptoms such as pruritus, night sweats, fatigue, and bone pain. Both diseases, if progressive despite standard therapies, are associated with an increased risk of thrombosis, bleeding, and progression to MF or even acute myeloid leukemia. Literature estimates looking at burden of illness are lacking for these disorders. Hence, real world analyses evaluating economic burden are imperative. The objective of this study was to evaluate the clinical and economic burden of these disorders by describing the patient demographics, prevalence, comorbidities, utilization and costs using large scale databases. Method: The US IMPACT® claims database was used to retrospectively identify unique patients with PV and ET between 1/1/08 and 12/31/10. This database is a fully de-identified, HIPAA compliant national database that captures the complete medical history for over 100M managed care individuals, including patient demographics, disease description, laboratory results, and details of medical, pharmacy, outpatient, and inpatient claims. ICD9 CM codes were used to identify PV and ET. Charlson Comorbidity Index (CCI) was used to assess overall comorbid disease status. Enrollment was restricted to those with a full year of medical and pharmacy benefit. Control group was age and gender matched but without any diagnosis of PV or ET. Medical costs include inpatient, outpatient and ER cost. Result: In 2010, we identified 5752 PV patients from ∼12M enrollees. This corresponds to an age adjusted prevalence of 56.5 cases/100000 patients. Compared with age gender matched control patients, PV patients had higher overall comorbidities (mean CCI 1.2 vs 0.7), were hospitalized more often (16% vs 8%), had higher average number of hospital days spent (1.7 vs 0.8), and had more outpatient visits (31 vs 18). PV patients incurred much higher average annual cost ($14,903 vs $7,913) than age gender matched controls driven by both medical ($12,006 vs $6,188) and pharmacy ($2,897 vs $1,724) cost. In 2010, we identified 5483 ET patients from ∼12M enrollees. This corresponds to an age adjusted prevalence of 56.1 cases/100000 patients. Compared with age gender matched control patients, ET patients had higher overall comorbidities (mean CCI 1.4 vs 0.7), were hospitalized more often (30% vs 9%), had higher average number of hospital days spent (5 vs 0.9 days), and had more outpatient visits (37 vs 19). ET patients incurred much higher average annual cost ($29,553 vs $8,026) than age gender matched controls driven by both medical ($26,287 vs $6,394) and pharmacy ($3,267 vs $1,631) cost. Similar trend was observed in 2008 and 2009. Over a period of 3 years, annual cost of PV ranged from $14,000-$16,000 and those of ET ranged from $29,000-$31,000. The utilization rate and total cost in each of conditions were significantly higher than those of their matched patients in each of the 3 years. Conclusion: PV and ET are associated with significant burden of illness. Healthcare expenditure was twice as higher in PV patients compared with the control group. ET patients incurred more than 3 times the healthcare expenditure than the control group. Our study indicates that PV and ET associated medical resource utilization and the corresponding expenditures for those services are substantive. In order to reduce the burden of illness associated with these diseases, continued efforts in the development of more efficacious treatments for these disorders are needed. Disclosures: Mehta: Sanofi: Employment. Wang:Sanofi: Employment, Equity Ownership. Iqbal:Sanofi: Employment, Equity Ownership. Mesa:Incyte: Research Funding; Lilly: Research Funding; Sanofi: Research Funding; NS Pharma: Research Funding; YM Bioscience: Research Funding.

scholarly journals Treatment Patterns Among Adults with Newly Diagnosed Primary Immune Thrombocytopenia in the United States

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 497-497
Author(s):  
Karynsa Cetin ◽  
Leah J McGrath ◽  
Robert Overman ◽  
Diane Reams ◽  
Anjali Sharma ◽  
...  
Keyword(s):  
Cumulative Incidence ◽  
Research Funding ◽  
Rescue Therapy ◽  
The United States ◽  
Advisory Board ◽  
Oral Steroid ◽  
Newly Diagnosed ◽  
Employment Equity ◽  
The Us ◽  
Equity Ownership

Abstract Introduction: Immune thrombocytopenia (ITP) is a rare platelet disorder that can lead to an increased tendency to bleed. Recommended first-line therapies include corticosteroids, intravenous immunoglobulin (IVIg) and intravenous (IV) anti-D. An estimated two-thirds of adult patients with ITP will develop persistent or chronic disease (ITP lasting 3-12 months or >12 months, respectively). Several evidence-based options for second-line treatment exist, but no randomized trials have directly compared one therapy to another. Patterns of treatment in routine clinical practice therefore vary. There is a paucity of data on current real-world treatment dynamics in ITP, and such data could help identify gaps in care and inform future studies of real-world comparative effectiveness and safety. We described the types of treatments administered following an ITP diagnosis, as well as the subsequent occurrence of bleeding and requirement for rescue therapy among adults being managed in routine practice in the United States (US). Methods: We used electronic health record data from hematology clinics across the US (Flatiron Health, Inc.) linked to MarketScan® employer-based and Medicare Supplemental administrative health insurance claims databases (Truven Health Analytics, Inc.). We included patients aged 18 years or older with a new ITP diagnosis from January 1, 2011 through June 30, 2016, continuous enrollment in MarketScan prior to diagnosis, and no previous diagnosis of a secondary cause of thrombocytopenia. The cumulative incidence of each ITP treatment after diagnosis was estimated using competing risk models to account for deaths occurring before initiation. Estimates were provided specifically for 90 days and 1 year following diagnosis to describe treatment uptake in the newly diagnosed and persistent phases, respectively. The incidence of bleeding events and rescue therapy was quantified after the start of the more prevalent second-line therapies: rituximab, splenectomy, and thrombopoietin receptor agonists (TPO RAs) - eltrombopag and romiplostim. Rescue therapies (those that rapidly increase platelet counts in the setting of severe thrombocytopenia or active bleeding) included IV anti-D, IVIg, IV steroids, and platelet transfusions. Results: Among the cohort of 447 adults diagnosed with primary ITP, 47% were male, 61% were white, 32% were 65 years or older, and the median lowest platelet count in the 60 days prior to diagnosis was 85x109/L (IQR: 39, 125). Use of each ITP therapy by 90 days and 12 months post-diagnosis are provided in the Table. Oral corticosteroids were the most commonly used; the cumulative incidence of initiation was 41% by 90 days and 50% by 1 year following ITP diagnosis. IV steroids and rituximab were the next most frequently used medications (16% and 11% at 90 days; and 26% and 16% by 1 year, respectively). The cumulative incidence of the TPO RAs, eltrombopag and romiplostim, by 90 days was 3% and 7%, respectively, and by 1 year was 5% and 9%, respectively. Splenectomy was relatively rare (<4% by 1 year) as was use of all other non-rescue ITP medications (≤1% by 1 year). At 180 days post-ITP treatment initiation, rituximab initiators (N = 84) had a slightly lower incidence of bleeding overall (12% [6, 20]) than the other treatment groups (17% [6, 33] among 31 eltrombopag initiators; 19% [9, 31] among 49 romiplostim initiators; and 19% [6, 38] among 21 splenectomized patients). However, rituximab initiators had the highest cumulative incidence of rescue therapy use (48% [36, 58] compared with 29% [14, 46] for eltrombopag, 26% [14, 39] for romiplostim, and 19% [6, 39] for splenectomized patients). Subsequent oral steroid use was less frequent among TPO RA initiators than rituximab initiators or patients who underwent splenectomy. Conclusions: In this descriptive study of patients with primary ITP receiving care in the US, oral steroids were the most commonly used medication after diagnosis, reflecting their continued role as a frontline therapy. By 1 year after diagnosis, approximately 15% received rituximab, nearly 10% received romiplostim, and 5% received eltrombopag. Splenectomy was less common. Among the medical treatments, although bleeding risk overall appeared lowest in rituximab patients, oral steroid and rescue therapy use were lowest among the patients who initiated TPO RAs. Table. Table. Disclosures Cetin: Amgen Inc: Employment, Equity Ownership. Sharma:Amgen: Employment, Equity Ownership. Brookhart:Amgen Inc: Consultancy, Research Funding; NoviSci: Equity Ownership; Union Chimique Belge: Consultancy; GlaxoSmithKline: Consultancy; Merck: Consultancy; Genentech: Consultancy; TargetPharma: Consultancy; RxAnte: Consultancy; AstraZeneca: Research Funding. Altomare:Genentech: Consultancy; Ipsen: Other: Advisory Board Member; Amgen: Consultancy; Celgene: Other: Advisory Board Member; Novartis: Consultancy; Bayer: Consultancy; Incyte: Consultancy. Wasser:Amgen Inc: Consultancy; Novartis: Consultancy; Becton Dickinson: Equity Ownership; Abbott Labs: Equity Ownership; Biogen: Equity Ownership; Allergan: Equity Ownership; Eli Lilly: Equity Ownership; Incyte: Research Funding; Merck: Equity Ownership, Research Funding; Pfizer: Equity Ownership, Research Funding; Guardant: Research Funding.

scholarly journals Health Care Utilization and Costs Among Patients with Chronic Myeloid Leukemia Using Tyrosine Kinase Inhibitors

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 3537-3537
Author(s):  
Joanna C Huang ◽  
Sudeep Karve ◽  
Sanchita Porwal ◽  
Kushan Thakkar ◽  
Thomas Marshall ◽  
...  
Keyword(s):  
Tyrosine Kinase ◽  
Emergency Room ◽  
Tyrosine Kinase Inhibitors ◽  
Health Plan ◽  
Real World ◽  
Index Date ◽  
Kinase Inhibitors ◽  
Employment Equity ◽  
The Us ◽  
Equity Ownership

Abstract INTRODUCTION: Tyrosine kinase inhibitors (TKIs) remain mainstay in the management of patients with CML. Several TKIs have been approved over the last two decades. Even though efficacy and safety remain as the primary drivers in treatment selection, in recent years importance has been placed on treatment affordability and economic burden associated with the long-term cancer treatments such as TKIs. However, current literature lacks real-world data on healthcare utilization (HCU) and costs among patients with CML using TKIs, which this study aims to address. METHODS: Retrospective cohort study was conducted using MarketScan Commercial, and Supplemental Medicare databases (2012-2016). Data includes medical and pharmacy utilization and costs for over 90 million individuals enrolled in employer-sponsored health-plans in the US. Data includes information on but not limited to medical diagnosis, procedures, drugs dispensed, date of service, health plan enrollment. Study involves adult patients (≥18 years) with ≥2 medical claims with a diagnosis of CML (ICD-9-CM: 205.10 - 205.12; ICD-10: C92.10-C92.12) and with a prescription claim for TKI. The date of first-TKI claim defined the index date. Patients were required to have continuous health plan enrollment ≥6 months before (defined as baseline period) and ≥6 months after index date. Selected patients were further classified into 5 sub-groups based on the index TKI observed (prescribed) post CML diagnosis (imatinib, dasatinib, nilotinib, bosutinib, ponatinib). Among the selected patients, all-cause HCU and costs were assessed from index date until end of database or end of enrollment, whichever occurred earlier. HCU and associated costs were assessed overall and by care settings including inpatient, emergency room, physician office, outpatient hospital, outpatient pharmacy, nursing facility and ancillary care. In addition, baseline (6 month) utilization and costs were assessed. Monthly and annual resource utilization and costs were reported for the overall CML cohort (across all TKI users) and by index TKI sub-groups. All costs were reported from a payer perspective (i.e., costs reimbursed by health plan) and adjusted to 2017 US dollars using the US consumer price index (medical component). All analyses were descriptive in nature. RESULTS: The study cohort included 2,213 CML patients. Distribution for the index TKI treatment was as follows: 41% imatinib, 36% dasatinib, 21% and 1% each for bosutinib and ponatinib. Mean age (standard deviation [SD]) of the cohort was 55 (15) years which was similar for individual TKI sub-groups. Majority of patients were males (55%) and 56% were enrolled in a preferred provider organization plan. The mean follow-up duration post-TKI initiation was 607 (442) days. The average baseline monthly all-cause costs were $4,365 with inpatient and pharmacy costs accounting for over 3/4th of the total costs (Figure 1). Post-TKI initiation the average monthly costs were twice ($9,288) compared with the baseline costs ($4,365) and the increase was primarily attributable to higher outpatient pharmacy costs ($6,619, accounted for 71% of total costs) (Figure 2). Monthly costs across other care settings (inpatient, outpatient, emergency room) were similar for the baseline and post-TKI initiation. On average patients had 1.4 office visits, 2.5 prescriptions and 0.7 hospital outpatient visits per month at baseline, which increased by 23%, 38% and 49%, respectively post TKI-initiation. During the 1st year post TKI-initiation, 17% patients in the overall CML cohort had at least 1 inpatient admission and this was consistent across individual TKI-sub-groups (except ponatinib, 50%). CONCLUSIONS: Findings on TKI utilization and costs in employer-sponsored health-plan database indicate that average costs and utilization were similar across the TKI sub-groups with pharmacy costs accounting for 71% of the total post TKI initiation costs. Overall, this study helps address the literature gap by providing recent real-world treatment care-setting specific utilization and costs among TKI uses and these data can be of value to several healthcare stakeholders including physicians, managed care plans and researchers in supporting clinical and formulary decisions and also serve as inputs for economic models. Finally, the sample sizes for ponatinib and bosutinb were small and results for these TKIs should be interpreted with caution. Disclosures Huang: ZS Associates: Employment; Novo Nordisk Inc: Equity Ownership; AstraZeneca: Research Funding. Karve:AbbVie: Employment, Equity Ownership. Porwal:ZS Associates: Employment. Thakkar:ZS Associates: Employment. Marshall:AbbVie: Employment, Equity Ownership. Rosenberg:AbbVie: Employment, Equity Ownership.

scholarly journals Prognostic Molecular Stratification in Relapsed/Refractory Multiple Myeloma - Results of the Pomalidomide Mukseven (NCT02406222) Biomarker Trial

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4327-4327
Author(s):  
James Croft ◽  
Andrew Hall ◽  
Amy L Sherborne ◽  
Katrina Walker ◽  
Sidra Ellis ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Flow Cytometry ◽  
T Cell ◽  
High Risk ◽  
Real World ◽  
Research Funding ◽  
Unmet Need ◽  
Employment Equity ◽  
Equity Ownership

Background Treatment of relapsed/refractory multiple myeloma (RRMM) remains challenging as durable remissions are achieved in patient sub-groups only. Identifying patients that are likely to benefit prior to or early after starting relapse treatments remains an unmet need. MUKseven is a trial specifically designed to investigate and validate biomarkers for treatment optimization in a 'real-world' RRMM population. Design In the randomized multi-center phase 2 MUKseven trial, RRMM patients (≥2 prior lines of therapy, exposed to proteasome inhibitor and lenalidomide) were randomized 1:1 to cyclophosphamide (500 mg po d1, 8, 15), pomalidomide (4 mg days 1-21) and dexamethasone (40 mg; if ≥75 years 20 mg; d1, 8, 15, 21) (CPomD) or PomD and treated until progression. All patients were asked to undergo bone marrow (BM) and peripheral blood (PB) bio-sampling at baseline, cycle 1 day 14 (C1D14, on-treatment) and relapse. For biomarker discovery and validation, IGH translocations were profiled by qRT-PCR, copy number aberrations by digital MLPA (probemix D006; MRC Holland), GEP by U133plus2.0 array (Affymetrix), PD protein markers by IHC and PB T-cell subsets by flow cytometry for all patients with sufficient material. Primary endpoint was PFS, secondary endpoints included response, OS, safety/toxicity and biomarker validation. Original planned sample size was 250 patients but due to a change in UK standard of care during recruitment with pomalidomide becoming available, a decision was made to stop recruitment early. Results In total, 102 RRMM patients were randomized 1:1 between March 2016 and February 2018. Trial entry criteria were designed to include a real-world RRMM population, permitting transfusions and growth factor support. Median age at randomization was 69 years (range 42-88), 28% of patients had received ≥5 prior lines of therapy (median: 3). Median follow-up for this analysis was 13.4 months (95% CI: 12.0-17.5). 16 patients remained on trial at time of analysis (median number of cycles: 19.5; range 8-28). More patients achieved ≥PR with CPomD compared to PomD: 70.6% (95% CI: 56.2-82.5%) vs. 47.1% (CI: 32.9-61.5%) (P=0.006). Median PFS was 6.9 months (CI: 5.7-10.4) for CPomD vs. 4.6 months (CI: 3.5-7.4) for PomD, which was not significantly different as per pre-defined criteria. Follow-up for OS is ongoing and will be presented at the conference. High-risk genetic aberrations were found at following frequencies: t(4;14): 6%, t(14;16)/t(14;20): 2%, gain(1q): 45%, del(17p): 13%. Non-high risk lesions were present as follows: t(11;14): 22%, hyperdiploidy: 44%. Complete information on all high-risk genetic markers was available for 71/102 patients, of whom 12.7% had double-hit high-risk (≥2 adverse lesions), 46.5% single-hit high-risk (1 adverse lesion) and 40.8% no risk markers, as per our recent meta-analysis in NDMM (Shah V, et al., Leukemia 2018). Median PFS was significantly shorter for double-hit: 3.4 months (CI: 1.0-4.9) vs. single-hit: 5.8 months (CI: 3.7-9.0) or no hit: 14.1 months (CI: 6.9-17.3) (P=0.005) (Figure 1A). GEP was available for 48 patients and the EMC92 high-risk signature, present in 19% of tumors, was associated with significantly shorter PFS: 3.4 months (CI: 2.0-5.7) vs. 7.4 (CI: 3.9-15.1) for EMC92 standard risk (P=0.037). Pharmacodynamic (PD) profiling of cereblon and CRL4CRBN ubiquitination targets (including Aiolos, ZFP91) in BM clots collected at baseline and C1D14 is currently ongoing. Preliminary results for the first 10 patients demonstrate differential change of nuclear Aiolos (Figure 1C), with a major decrease in Aiolos H-scores in 7/10 patients from baseline to C1D14 and reconstitution at relapse. T-cell PB sub-sets were profiled at baseline and C1D14 by flow cytometry. Specific sub-sets increased with therapy from baseline to C1D14, e.g. activated (HLA-DR+) CD4+ T-cells, as reported at last ASH. CD4+ T-cell % at baseline was associated with shorter PFS in these analyses in a multi-variable Cox regression model (P=0.005). PD and T-cell biomarker results will be updated and integrated with molecular tumor characteristics and outcome. Discussion Our results demonstrate that molecular markers validated for NDMM predict treatment outcomes in RRMM, opening the potential for stratified delivery of novel treatment approaches for patients with a particularly high unmet need. Additional immunologic and PD biomarkers are currently being explored. Disclosures Croft: Celgene: Other: Travel expenses. Hall:Celgene, Amgen, Janssen, Karyopharm: Other: Research funding to Institution. Walker:Janssen, Celgene: Other: Research funding to Institution. Pawlyn:Amgen, Janssen, Celgene, Takeda: Other: Travel expenses; Amgen, Celgene, Janssen, Oncopeptides: Honoraria; Amgen, Celgene, Takeda: Consultancy. Flanagan:Amgen, Celgene, Janssen, Karyopharm: Other: Research funding to Institution. Garg:Janssen, Takeda, Novartis: Other: Travel expenses; Novartis, Janssen: Research Funding; Janssen: Honoraria. Couto:Celgene Corporation: Employment, Equity Ownership, Patents & Royalties. Wang:Celgene Corporation: Employment, Equity Ownership. Boyd:Novartis: Consultancy, Honoraria; Janssen: Consultancy, Honoraria; Takeda: Consultancy, Honoraria; Amgen: Consultancy, Honoraria; Celgene: Consultancy, Honoraria. Pierceall:Celgene: Employment. Thakurta:Celgene: Employment, Equity Ownership. Cook:Celgene, Janssen-Cilag, Takeda: Honoraria, Research Funding; Janssen, Takeda, Sanofi, Karyopharm, Celgene: Consultancy, Honoraria, Speakers Bureau; Amgen, Bristol-Myers Squib, GlycoMimetics, Seattle Genetics, Sanofi: Honoraria. Brown:Amgen, Celgene, Janssen, Karyopharm: Other: Research funding to Institution. Kaiser:Takeda, Janssen, Celgene, Amgen: Honoraria, Other: Travel Expenses; Celgene, Janssen: Research Funding; Abbvie, Celgene, Takeda, Janssen, Amgen, Abbvie, Karyopharm: Consultancy.

scholarly journals Epidemiology of PNH and Real-World Treatment Patterns Following an Incident PNH Diagnosis in the US

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3407-3407 ◽  
Author(s):  
Jessica J Jalbert ◽  
Umesh Chaudhari ◽  
Haixin Zhang ◽  
Jonathan Weyne ◽  
Jamile M. Shammo
Keyword(s):  
Incidence Rate ◽  
Real World ◽  
Treatment Duration ◽  
Research Funding ◽  
The United States ◽  
Treatment Patterns ◽  
Employment Equity ◽  
Equity Ownership ◽  
Procedure Codes ◽  
Rbc Transfusion

Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired, life-threatening blood disease. While PNH is known to be a rare disease, the incidence and prevalence of the condition has been described only in a few small studies. In addition, while the International PNH registry is a rich source of data on real-world PNH patients globally, it is not possible to estimate the incidence and prevalence of PNH directly from the registry. As complement inhibitors are becoming the standard of care for PNH treatment, we also sought to explore how patients are managed following an incident PNH diagnosis. The objective of this study was to estimate the incidence and prevalence of PNH and to describe real-world treatment patterns among patients newly diagnosed with PNH in the United States (US). Methods: We conducted a retrospective cohort study using Truven US MarketScan Commercial/Medicare data (1 Jan 2015 to 30 June 2018), an employer-sponsored insurance claims database including annually approximately 30 million insured patients and their dependents' complete longitudinal records of inpatient services, outpatient services, and prescription drug claims covered under a variety of fee-for-service and capitated health plans. While these data are considered nationally representative of Americans with employer-provided health insurance, data come mainly from large employers. To estimate prevalence, we identified patients with ≥ 1 PNH diagnosis (ICD10: D59.5) among persons continually enrolled in the databases in 2017. To estimate incidence, we required ≥1-year of baseline enrolment and no PNH diagnosis or eculizumab exposure, identified using national drug codes [NDC] or procedure codes for drug administration, during the baseline period. Person-time accrued post-baseline until PNH diagnosis, end of study period, or disenrollment. We stratified incidence and prevalence estimates by age and sex and described patients with incident PNH in terms of demographics, comorbidities, and past-year healthcare resource utilization. Using Kaplan-Meier estimators, we estimated incidence of eculizumab initiation, timing of initiation, treatment duration, and risk of discontinuation/treatment holiday (&gt;42 days between eculizumab exposures [i.e. 14-day exposure period + 28-day grace period between infusions], the equivalent of missing 2 infusions assuming a bi-weekly infusion schedule for eculizumab) following the incident PNH diagnosis. While accounting for censoring, we also investigated patterns of red blood cell (RBC) transfusions, identified using procedure codes, in terms of incidence and timing of first transfusion following an incident PNH diagnosis. Results: The prevalence of PNH varied little between 2016 and 2017, from 12 to 13 per 1,000,000. The incidence rate over the study period was 5.7 per 1,000,000 person-years, representing 257 incident PNH cases. The incidence rate of PNH increased with age and was similar across sex. At diagnosis, mean age was 50.0 years (standard deviation [SD]: 18.6), 3.1% (8/257) were less than 18 years, 52.1% were women, 19.5% had a past-year diagnosis of aplastic anemia, 8.2% had a past-year diagnosis of myelodysplastic syndrome, 14.0% had a past-year RBC transfusion, and 31.5% had been hospitalized in the past-year. Over a mean follow-up time of 385.6 days (SD: 253.2), 10.3% (95% confidence interval [CI]: 6.3-14.1%) of patients initiated eculizumab on average 60.5 days (SD: 55.9) from PNH diagnosis. At 1 year, about one third of patients discontinued eculizumab or had taken a treatment holiday; average treatment duration was 328.2 days (SD:245.4). Cumulative incidence of RBC transfusions at 6 months and 1 year was 14.6% (10.1-18.9%) and 17.4% (12.2-22.3%), respectively. On average, the first RBC transfusion occurred within 63.6 days (SD: 114.4) of an incident PNH diagnosis. Conclusions: In routine clinical practice, only a minority of patients recently diagnosed with PNH are initiated on eculizumab. Among PNH patients treated with eculizumab, less than 70% remain on treatment after 1 year. Findings must be interpreted in the context of limitations including lack of information on clone size, symptom burden, measures of disease activity, or bone marrow failure state which may affect treatment course. Future studies should explore factors affecting eculizumab initiation and persistence on treatment. Disclosures Jalbert: Regeneron Pharmaceuticals, Inc.: Employment, Equity Ownership. Chaudhari:Regeneron Pharmaceuticals, Inc.: Employment, Equity Ownership. Zhang:Regeneron Pharmaceuticals, Inc.: Employment, Equity Ownership. Weyne:Regeneron Pharmaceuticals, Inc.: Employment, Equity Ownership. Shammo:Incyte: Consultancy, Honoraria, Research Funding, Speakers Bureau; Celgene: Consultancy, Honoraria, Research Funding, Speakers Bureau; Alexion: Consultancy, Honoraria, Research Funding, Speakers Bureau; Onconova: Research Funding; Apellis: Membership on an entity's Board of Directors or advisory committees; Sanofi: Consultancy, Honoraria, Speakers Bureau; Astex Pharma: Research Funding; Novartis: Consultancy, Honoraria; Otsuka: Consultancy, Honoraria; CTI Pharma: Research Funding.

scholarly journals Cardiac Events in Real-World Multiple Myeloma Patients Treated with Carfilzomib: A Retrospective Claims Database Analysis

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3319-3319 ◽  
Author(s):  
Ajai Chari ◽  
Sanjay K. Aggarwal ◽  
Khalid Mezzi ◽  
Kenneth Wang ◽  
Christopher Kim ◽  
...  
Keyword(s):  
Real World ◽  
Research Funding ◽  
Cardiac Event ◽  
Index Date ◽  
Hospitalized Patients ◽  
Cardiac Events ◽  
Employment Equity ◽  
Claims Database ◽  
History Of ◽  
Equity Ownership

Abstract Introduction: Multiple myeloma (MM) is a hematologic cancer that mostly affects elderly patients who are at increased risk for development of cardiac-related comorbidities due to age-, disease-, and treatment-related factors. In real world data, approximately 25-30% of MM patients are hospitalized for a cardiac event after MM diagnosis (Kistler et al. American Society Hematology 2012 Annual Meeting, poster 2916). The proteasome inhibitor, carfilzomib, is approved for treatment of patients with relapsed or refractory MM. In carfilzomib clinical trials, grade 3 or higher cardiac failure was reported for ~4% of patients (Stewart et al. NEJM 2015;372:142-52, Dimopoulos et al. Lancet Oncol 2016;17:27-38). In this retrospective analysis, we sought to evaluate the incidence rate (IR) of cardiac events in MM patients treated with carfilzomib in a real-world setting in the US and to describe differences in baseline characteristics between carfilzomib-treated MM patients who do and do not have cardiac events. Methods: Newly-diagnosed MM patients with symptomatic disease were identified in the Truven MarketScan claims database from 1/1/05 to 6/30/15 using an algorithm validated for ascertainment of MM patients in claims data (Princic et al. Blood 2015;126:4521). All carfilzomib-treated patients were included in the analysis. The first dose of carfilzomib was the index date. The baseline period was 12 months prior to the MM diagnosis date to the index date. Cardiac events (hypertension, heart failure, ischemic heart disease, arrhythmias and conduction disorders, and cardiomyopathy) were identified during baseline by inpatient (IP) or outpatient (OP) claims and after index date by primary diagnosis on IP claims (hospitalized events). Logistic regression was used to estimate baseline covariates associated with any cardiac event. Due to sample size constraints, multivariate regression was not performed. The incidence rate (IR) per 1000 patient-years (PYRs) and associated 95% confidence interval (CI) of hospitalized cardiac events during carfilzomib treatment which included 30 days after the last carfilzomib dose were calculated among patients without a history of the corresponding hospitalized event during the baseline period. Results: The cohort included 498 MM patients treated with carfilzomib; 108 (22%) patients had ≥1 cardiac event identified by both IP and OP claims and 24 (5%) had ≥1 hospitalized cardiac event. Of the 24 hospitalized patients, 14 were hospitalized during carfilzomib treatment (median days to onset = 104) and 8 were hospitalized after carfilzomib treatment (>30 days post dosing; median days to onset = 284). The median age (range) of the 24 patients with hospitalized cardiac events and those with no hospitalized cardiac claims (n=474) was 62 (37, 79) and 61 (36, 96) years, respectively (Table). Baseline medical history of any cardiac event was evident for 92% of hospitalized patients and 84% of non-hospitalized patients. Among hospitalized patients, 25% were over 75 years as compared with 13% of non-hospitalized patients. In univariate analysis, patients with baseline amyloidosis, hypertensive chronic kidney disease, and chronic kidney disease had higher odds of having a hospitalized cardiac event. No significant differences were seen in the proportion of patients with or without a hospitalized cardiac event when evaluated by line of therapy or by carfilzomib treatment regimen (monotherapy or combination). Among patients aged 75+ years, 8.7% were hospitalized, while 2.5% and 4.9% of 65-74 and 18-64 year old patients were hospitalized, respectively (Table). IR per 1000 PYRs [95% CI] among the 14 carfilzomib-treated MM patients hospitalized for any cardiac event during the carfilzomib treatment period was 89.7 (49.0; 150.5) - see Figure for IRs of specific cardiac events. Conclusions: Among carfilzomib-treated patients in a US claims database, hospitalization for a cardiac event was uncommon (~5%) and does not exceed what has been observed in MM patients. Patients at highest risk for cardiac hospitalizations had a history of amyloidosis or chronic renal failure. Additional analyses in much larger populations are needed to better understand risk factors for these events. Disclosures Chari: Array Biopharma: Consultancy, Research Funding; Celgene: Consultancy, Research Funding; Amgen Inc.: Honoraria, Research Funding; Novartis: Consultancy, Research Funding; Takeda: Consultancy, Research Funding; Pharmacyclics: Research Funding; Janssen: Consultancy, Research Funding. Aggarwal:Amgen: Employment, Equity Ownership. Mezzi:Amgen Inc.: Employment, Equity Ownership. Wang:Amgen Inc.: Employment, Equity Ownership. Kim:Amgen Inc.: Employment, Equity Ownership. Zhu:Amgen Inc.: Consultancy. Braunlin:Amgen Inc.: Employment, Equity Ownership. Werther:Amgen Inc.: Employment, Equity Ownership. Mikhael:Abbvie: Research Funding; Celgene: Research Funding; Sanofi: Research Funding; Onyx: Research Funding.

Does Faster Rituximab Infusion for Patients with NHL Lower Cancer Care Costs?

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 2063-2063
Author(s):  
John Hornberger ◽  
Minghan Dai ◽  
Hans-Peter Goertz ◽  
Keith L Dawson ◽  
Carolina Reyes
Keyword(s):  
Adverse Events ◽  
Research Funding ◽  
B Cell Lymphoma ◽  
Target Population ◽  
Employment Equity ◽  
Medical Resources ◽  
Off Label ◽  
Group Research ◽  
The Us ◽  
Equity Ownership

Abstract Abstract 2063 Introduction Rituximab in combination with chemotherapy is a recommended regimen for non-Hodgkin's lymphoma (NHL), which has been shown in randomized trials and real-world studies to provide a survival benefit compared with chemotherapy alone. Per the US Prescribing Information (USPI), rituximab is typically administered over 4–6 hours for the first infusion and 3–4 hours for subsequent infusions. A 90-minute infusion schedule for rituximab has been found to be safe and feasible in NHL patients who tolerate their first infusion administered at the standard rate and who do not have significant cardiovascular disease or high circulating lymphocytes. This study's primary objective was to assess the cost from a US societal perspective of faster 90-minute rituximab infusion vs the conventional 4–6 hour rituximab infusion. Methods Analyses were performed in accordance with best research guidelines of the International Society of Pharmacoeconomic Outcomes and Research. The target population was patients with previously untreated diffuse large B-cell lymphoma (DLBCL) or follicular lymphoma (FL) who were scheduled to receive rituximab 375 mg/m2plus CHOP or CVP chemotherapy. Medical resources assessed were: chemotherapy administration, chemotherapy and pre-treatment drugs, and Grade 3/4 adverse events. Indirect costs include societal resources such as income foregone by patients and informal caregivers/spouses as a result of visit duration for infusion. Trial data were used to extract event rates of adverse events and duration of infusions, and patient drop-out rate by cycle. Prices for medical resources were obtained from publicly available Medicare Fee Schedules. Per capita hourly wage rates were extracted from statistics of the US Labor Department. Outcomes were costs per year for patients treated in the US and cost per infusion and per course of therapy in 2012 US dollars. Results A projected 23,519 newly diagnosed DLBCL and FL patients comprise the target population. Estimated total direct medical cost for conventional infusion is $925,318,162 and $9,855,416 in foregone income for patients and caregivers. The 90-minute infusion reduced direct medical costs by $22,362,397 (2%) and foregone income for patients and caregivers by $5,115,629 (52%). The average cost savings were $359 per infusion and $2,119 per course. The most influential parameters were administration cost and patient foregone income due to time for infusions. The savings per course may be greater if infusion centers are able to spread fixed costs, e.g., overhead, among a greater number of patients resulting from increased capacity to schedule more patients. Conclusion The 90-minute rituximab infusion has been found to be a safe and feasible alternative to conventional rituximab infusion for patients with untreated DBLCL or FL. In an era of landmark legislation (The Patient Protection and Affordable Care Act of 2010) designed to address concerns about rising costs of healthcare and place more emphasis on patient-centered research, the faster rituximab infusion regimen offers direct medical savings and reduces foregone income to patients and caregivers. Disclosures: Hornberger: Cedar Associates LLC: Employment; Genentech, A Member of the Roche Group: Research Funding. Off Label Use: Rituxan (Rituximab) - faster infusion is off-label. Dai:Cedar Associates: Employment; Genentech, Inc, A Member of the Roche Group: Research Funding. Goertz:Genentech, Inc, A Member of the Roche Group: Employment, Equity Ownership. Dawson:Genentech, A Member of the Roche Group: Employment, Equity Ownership. Reyes:Genentech, Inc, A Member of the Roche Group: Employment, Equity Ownership.

Epidemiology of Myelofibrosis (MF), Polycythemia Vera (PV) and Essential Thrombocythemia (ET) in the European Union (EU)

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 1744-1744 ◽  
Author(s):  
Odile Moulard ◽  
Jyotsna Mehta ◽  
Robert Olivares ◽  
Usman Iqbal ◽  
Ruben A. Mesa
Keyword(s):  
Bone Marrow ◽  
Real World ◽  
Research Funding ◽  
Hematological Malignancies ◽  
Research Network ◽  
Published Data ◽  
Employment Equity ◽  
Myeloid Metaplasia ◽  
Epidemiology Data ◽  
Equity Ownership

Abstract Abstract 1744 Background: MF, ET and PV are BCR ABL negative myeloproliferative neoplasms (MPN). These are a subset of bone marrow disorders characterized by an overproduction of one or more types of blood or fiber cells in the bone marrow. There is disparate published data on the epidemiological estimates for these diseases in the EU. An evidence based synthesis of MPN epidemiology data can effectively inform population health care planning and economic decision support for these disorders. The aim of our study is to synthesize and provide a comprehensive review of epidemiology estimates from available sources including literature, publicly available registries as well as real world data. Methods: We conducted a search of literature published between 1995–2012 using terms related to MF, PV and ET. Elsevier and Medline databases were searched using Embase platform. Orphan disease registries, specific hematological malignancies registries, country specific databases and registries included RARECARE, Orphanet, Registry of hematological malignancies in France, UK Hematologic Malignancy research network. Finnish cancer registry and Sweden National registry were also evaluated. The RARECARE project is responsible for surveillance of rare cancers in Europe based on data from 88 cancer registries in 22 European countries and includes cancer cases diagnosed in 1995–2002. Orphanet provides an estimate of the prevalence of rare diseases in Europe based on a systematic survey of the literature. Calculation of prevalence in Orphanet is done by multiplying incidence with the mean duration of disease. UK THIN (∼5 million enrollees each year) general practitioner database was used to retrospectively identify patients with primary and secondary MF between 1/1/08 and 12/31/10. READ codes were used to identify MF. This database can track patients longitudinally over multiple years, are linked at the patient level by a unique identifier that is consistent across health related services and time and have been shown to be representative of the UK population. Results: Only six articles looking at the European population were retrieved through the literature. These articles as well as international/national reports were used to summarize epidemiological estimates for Myelofibrosis using several different terms. MF terminology varied among registries. Terms included “Primary myelofibrosis”, “myelosclerosis with myeloid metaplasia” and “myelofibrosis”. Annual prevalence of MF ranged from 0.5–9 cases per 100,000 individuals. For PV, even if terminology and coding is consistent, estimates still varied depending on registry used. RARECARE reported prevalence of 5.5 per 100,000 and Orphanet reported a range of 10–50 cases per 100,000. For ET, prevalence was reported only in RARECARE with 4.4 cases per 100,000 individuals. Prevalence estimates in UK were slightly higher compared to RARECARE; 0.9 cases per 100,000 for MF, 6 cases per 100,000 for PV and ET. Incidence for MF, PV and ET ranged from 0.3–1.9, 0.6–2.8, 0.5–2.2 per 100,000 patients respectively. Conclusions: Few publications and registries report epidemiology data for MF, PV or ET. Especially, prevalence data is very rare. There is wide variation in both prevalence and incidence estimates for MPN across EU data sources that can limit informed application of epidemiological research. The differences stem from lack of a unifying term for MF and coding differences coupled with limitations inherent in registry data including potential sources of bias, missing data, and issues with assessment of statistical significance. Additional research using standardized definitions/coding across appropriate real world databases and enhanced tumor registries are needed to add precision to or substantiate these results. Disclosures: Moulard: Sanofi: Employment, Equity Ownership. Mehta:Sanofi: Employment. Olivares:Sanofi: Employment, Equity Ownership. Iqbal:Sanofi: Employment, Equity Ownership. Mesa:Incyte: Research Funding; Lilly: Research Funding; Sanofi: Research Funding; NS Pharma: Research Funding; YM Bioscience: Research Funding.

Real-World Ponatinib Prescribing Patterns and Outcomes in US Patients

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 1591-1591
Author(s):  
Michael J. Mauro ◽  
Lisa J. McGarry ◽  
Mo Yang ◽  
Stephanie Lustgarten ◽  
Hui Huang
Keyword(s):  
Dose Reduction ◽  
Real World ◽  
Dose Adjustment ◽  
Prescribing Patterns ◽  
Employment Equity ◽  
Line Therapy ◽  
Starting Dose ◽  
The Us ◽  
Equity Ownership ◽  
Over Time

Abstract Background: In Jan 2014, ponatinib was reintroduced to the US market after an 11 week withdrawal to review data on arterial thrombotic events, revise US prescribing information (USPI) and implement a risk evaluation and mitigation strategy (REMS). The USPI was revised to narrow the indicated population and recommend a starting dose of 45 mg, with consideration of 1) lower doses in patients with selected comorbidities or to manage adverse events, 2) dose reduction in chronic phase (CP) and accelerated phase (AP) chronic myeloid leukemia (CML) patients achieving major cytogenetic response, and 3) discontinuation if response has not occurred at 3 months. In the US, ponatinib is available exclusively through a specialty pharmacy that maintains prescribing data for all US ponatinib-treated patients since reintroduction. Examining these data provides insight into practitioners' patient selection and prescribing patterns, and real-world ponatinib outcomes. Methods: We performed a retrospective analysis of patients starting treatment with ponatinib between 01 January 2014 and 25 March 2015 using data from referring physicians, patient intake forms and pharmacy dispensing records. Patient and prescriber characteristics, and dosing and dose modifications were documented. Clinical, demographic and physician characteristics were examined as predictors of initial dose and dose modification using logistic regression; therapy duration was assessed using Kaplan-Meier techniques and proportional hazard regression. Results: 758 US patients initiated treatment with ponatinib over this 15-month period, (58% male; median age 55 years [range: 11-98]). Among 730 patients with a specified diagnosis, 80% had CML and 4% Philadelphia chromosome positive (Ph+) acute lymphocytic leukemia (ALL); the remainder had unspecified ALL (10%), other hematologic malignancies (3%), and solid tumors (3%). Of 411 CML patients reporting disease phase, 61% were in CP, 18% AP and 21% blast phase (BP). 12% of CML and 8% of Ph+ ALL patients had a reported T315I mutation. 21% of CP, 34% of AP, 12% of BP and 31% of Ph+ ALL patients were receiving ponatinib as 2nd-line therapy, with the remainder in 3rd line or later. Most recent prior TKI was dasatinib for 48%, nilotinib for 23%, bosutinib for 17%, and imatinib for 12% of patients in all therapy lines. 50% received 45 mg as their initial dose, 33% 30 mg and 17% 15 mg. Prescribers' practice setting was 49% community and 51% academic. Most prescribers (82%) had only 1 ponatinib patient; only 7% had 3 or more. Prescribers with >1 ponatinib patient were less likely to prescribe 45 mg starting dose (OR=0.53 for those with 2 patients; OR=0.25 for 3+ patients.) 23% of patients had at least one dose adjustment, including 17% with dose reduction. Among CP patients initially on 45 mg, with at least 6 months of therapy, 42% reduced dose (29% to 30 mg; 13% to 15 mg). Dose reduction decreased significantly for later therapy lines in CP, but did not differ by disease phase. Median time on therapy was >15 months for CP, 10.6 months for AP, 7.0 months for BP, and >14 months for Ph+ ALL. CP patients' time on therapy was longer for those started on 15 mg, although this difference was not significant (p=0.14) (Figure.) Reasons for dose adjustment and discontinuation were not well documented, but they appeared to occur at a relatively constant rate over time rather than at time points recommended for response monitoring. Conclusions: Real-world US data shows ponatinib is prescribed across disease phase, therapy line, and mutation status. While a majority of patients were in their 3rd line of therapy or later, a substantial proportion of patients, especially in AP CML and Ph+ ALL, received ponatinib as 2nd line therapy. Physicians appear to be selecting patients who are younger than those enrolled in registrational trial for ponatinib (55 years vs. PACE trial median age, 64 years), and mitigating against potential risk using lower starting doses and dose reduction. Most prescribers have only 1 ponatinib patient, but physicians with >1 ponatinib patient favor lower starting doses. Dose reduction and discontinuation occurred steadily over time rather than clustered at routine response milestone time points. CP CML Patients starting at 15 mg appear to have similar or better treatment duration compared with those started at higher doses. Disclosures Mauro: Ariad: Consultancy; Pfizer: Consultancy; Novartis Pharmaceutical Corporation: Consultancy, Research Funding; Bristol-Myers Squibb: Consultancy. McGarry:ARIAD: Employment, Equity Ownership. Yang:ARIAD Pharmaceuticals, Inc: Employment. Lustgarten:ARIAD Pharmaceuticals Inc.: Employment, Equity Ownership, Other: Stock. Huang:ARIAD: Employment, Equity Ownership.

Disease and Clinical Characteristics of Patients with Polycythemia Vera: An Early View of the Reveal Study

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 2813-2813
Author(s):  
Brady Stein ◽  
Alison Moliterno ◽  
Ralph V. Boccia ◽  
Ahmad B. Naim ◽  
Joseph A. Cordaro ◽  
...  
Keyword(s):  
Research Funding ◽  
Clinical Characteristics ◽  
Thromboembolic Event ◽  
Employment Equity ◽  
Advisory Committees ◽  
Reveal Study ◽  
Patient Reported ◽  
The Us ◽  
History Of ◽  
Equity Ownership

Abstract Background: Patients with polycythemia vera (PV) have an increased symptom burden (eg, fatigue, pruritus, and splenomegaly-related symptoms) and an increased risk of mortality compared with the general population, often resulting from thrombotic events, disease transformation to myelofibrosis, or secondary malignancies. There are limited data regarding PV burden and treatment patterns in a contemporary, real-world US setting. REVEAL is an ongoing observational study being conducted to describe contemporary demographics, burden of disease, clinical management, patient-reported outcomes, and healthcare resource utilization among patients with PV in the US. The objective of this assessment is to describe the clinical characteristics at enrollment among patients currently enrolled in REVEAL (enrollment from July 22, 2014 to June 9, 2015). Methods: REVEAL is a multicenter, noninterventional, nonrandomized, prospective, observational study. Eligible patients are ≥18 years of age with a PV diagnosis and currently under active management by a physician in the US. Data regarding disease and clinical characteristics, patient-reported outcomes, and health resource utilization are being collected at usual-care visits over a planned 36-month observation period. Analyses of these preliminary data were descriptive. Results: At data cutoff, 865 patients were enrolled (total planned enrollment, n=2000) from 174 sites (academic, n=27; community, n=147). Median (range) age at enrollment was 67 (22-95) years, 55.5% were male, 89.5% were white, and 45.4% had a history of smoking. Most patients (99.4%) had health insurance, including Medicare (56.0%), PPO (24.3%), or HMO (11.6%). Fifty-two percent were retired, 29.6% were employed, and 4.5% were unable to work or disabled. A total of 40.2% of patients completed a college/graduate degree, 19.9% had some college, and 32.5% completed high school or the equivalent. Documented major World Health Organization diagnostic criteria included JAK2V617F mutation in 50.9% of patients, elevated hemoglobin in 52.3%, and red cell mass in 5.5%. A bone marrow biopsy was conducted in 24.0% of patients, approximately half of whom (52.4% of the 24.0%) had trilineage myeloproliferation. Erythropoietin levels were abnormal in 19.7% of patients. Approximately half (46.6%) of patients had physician-reported PV-related symptoms at diagnosis, most frequently fatigue (25.2%) and pruritus (14.0%). At enrollment, median disease duration was 52.6 months; 42.3% of patients were diagnosed ≥5 years before enrollment. The most common comorbidities were hypertension (51.3%), obesity (14.2%), dyspnea (13.4%), myalgia (11.2%), and diabetes mellitus (11.1%). Mean hematocrit was 44.9%, hemoglobin was 14.5 g/dL, white blood cell count was 11.4×109/L, platelet count was 363.7×109/L, and absolute neutrophil count was 363.7×109/L. Twenty-one percent of patients had a history of thrombotic events, with 13.1% and 8.6% of patients experiencing ≥1 venous or arterial thromboembolic event, respectively. Of the 261 reported thrombotic events, 43.7% occurred after diagnosis of PV. The most common PV symptoms recorded at enrollment (using the Myeloproliferative Neoplasm Symptom Assessment Form) were fatigue (37.1%), pruritus (24.0%), headache (18.6%), insomnia (17.0%), dizziness (13.2%), bone pain (10.5%), and blurred vision (7.9%); 19.7% of 518 evaluable patients had palpable splenomegaly. Fewer than half of patients were receiving aspirin at enrollment (47.7%). The most common PV treatments ± aspirin were phlebotomy (PBT; 63.5%), hydroxyurea (HU; 42.2%), watchful waiting (5.3%), and ruxolitinib (3.1%); 23.0% of patients received PBT + HU. Conclusion: The REVEAL study is unique in that it captures data from both academic and community centers, and therefore includes a broader segment of the PV population typically not described in the literature. REVEAL can provide novel insights into questions related to the contemporary demographics of PV, practice patterns regarding diagnosis and therapy, and the real-world burden of PV and its impact on patients' quality of life and work productivity. Preliminary descriptive data from this early subset of patients suggest that a high proportion of patients report having PV-related symptoms at diagnosis, fewer patients are treated with aspirin than expected, and that thromboembolic event rates remain high after diagnosis. Disclosures Stein: Incyte Corporation: Consultancy, Membership on an entity's Board of Directors or advisory committees. Moliterno:Incyte Corporation: Membership on an entity's Board of Directors or advisory committees. Boccia:Incyte Corporation: Honoraria. Naim:Incyte Corporation: Employment, Equity Ownership. Cordaro:Incyte Corporation: Employment, Equity Ownership. Peng:Incyte Corporation: Employment, Equity Ownership. Sun:Incyte Corporation: Employment, Equity Ownership. Parasuraman:Incyte Corporation: Employment, Equity Ownership. Mesa:Promedior: Research Funding; Novartis Pharmaceuticals Corporation: Consultancy; NS Pharma: Research Funding; CTI Biopharma: Research Funding; Gilead: Research Funding; Pfizer: Research Funding; Incyte Corporation: Research Funding; Genentech: Research Funding.

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