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BACKGROUND: Whether emergency medical services (EMS) transport improves disability outcomes compared with other transport among acute ischemic stroke (AIS) patients is unknown. OBJECTIVE: To study severity-adjusted associations of hospital arrival mode (EMS vs. other transport) with in-hospital and discharge disability outcomes. DESIGN: Prospective observational study. PARTICIPANTS: AIS patients discharged April 2016 to October 2017 from a safety-net hospital in South Carolina. MAIN MEASURES: National Institutes of Health Stroke Scale (NIHSS) change at discharge (admission NIHSS score minus discharge NIHSS, continuous variable), 24-h NIHSS change (attaining high improvement, admission NIHSS minus 24-h NIHSS being 75th percentile or higher), door to neuroimaging (DTI) time, and IV alteplase receipt. NIHSS change was assessed within stroke severity groups, mild, moderate, and severe (admission NIHSS 0–5, 6–14, and ≥ 15, respectively). KEY RESULTS: Of 1168 patients, 838 were study-eligible (52% male, 52.4% Black, 72.2% EMS arrivals, 56.6% mild strokes). Severe and moderate stroke patients were more likely than mild stroke patients to use EMS (adjusted odds ratios, AOR [95% CI] 11.7 [5.0, 27.4] and 4.0 [2.6, 6.3], respectively). EMS arrival was associated with shorter DTI time (adjusted difference − 88.4 min) and higher likelihood of alteplase administration (AOR 5.3 [2.5, 11.4]), both key mediating variables in disability outcomes. High 24-h NIHSS improvement was more likely for EMS arrivals vs. other arrivals among moderate strokes (AOR 3.4 [1.1, 10.9]) and severe strokes (AOR > 999). EMS arrivals had substantially higher NIHSS improvement at discharge within the severe stroke group (adjusted NIHSS change at discharge, 5.9 points higher, p = 0.01). Alteplase recipients showed higher discharge NIHSS improvement than non-recipients (by 2.8 and 1.9 points among severe and moderate strokes, respectively; p = 0.01, 0.02). CONCLUSIONS: The findings offer evidence for including stroke education as a standard of care in the primary care management of patients with stroke-risk comorbidities/lifestyle in order to minimize post-stroke disability.

Background and Purpose— The 2015 updated US Food and Drug Administration alteplase package insert altered several contraindications. We thus explored clinical factors influencing alteplase treatment decisions for patients with minor stroke. Methods— An expert panel selected 7 factors to build a series of survey vignettes: National Institutes of Health Stroke Scale (NIHSS), NIHSS area of primary deficit, baseline functional status, previous ischemic stroke, previous intracerebral hemorrhage, recent anticoagulation, and temporal pattern of symptoms in first hour of care. We used a fractional factorial design (150 vignettes) to provide unconfounded estimates of the effect of all 7 main factors, plus first-order interactions for NIHSS. Surveys were emailed to national organizations of neurologists, emergency physicians, and colleagues. Physicians were randomized to 1 of 10 sets of 15 vignettes, presented randomly. Physicians reported the subjective likelihood of giving alteplase on a 0 to 5 scale; scale categories were anchored to 6 probabilities from 0% to 100%. A conjoint statistical analysis was applied. Results— Responses from 194 US physicians yielded 156 with complete vignette data: 74% male, mean age 46, 80% neurologists. Treatment mean probabilities for individual vignettes ranged from 6% to 95%. Treatment probability increased from 24% for NIHSS score =1 to 41% for NIHSS score =5. The conjoint model accounted for 25% of total observed response variance. In contrast, a model accounting for all possible interactions accounted for 30% variance. Four of the 7 factors accounted jointly for 58% of total relative importance within the conjoint model: previous intracerebral hemorrhage (18%), recent anticoagulation (17%), NIHSS (13%), and previous ischemic stroke (10%). Conclusions— Four main variables jointly account for only a small fraction (

Background: Despite available treatment for early-stage breast cancer (BC), 15%-25% of patients with early-stage human epidermal growth factor receptor 2–positive (HER2+) BC eventually experience recurrence after initial treatment. The prognosis for women with HER2+ disease recurrence is poor. Most recurrences involve incurable metastatic disease. In the US, the total cost to society attributable to metastatic BC of any subtype was $12.2 billion accrued over 5 years, or $2.4 billion per year ($98,571 per patient-year). Treatment-related cost, 57% of total costs, was the largest contribution, with over $1.0 billion per year. The purpose of this study was to assess the clinical and economic burden of recurrence in patients with early-stage HER2+ BC. Methods: We conducted two systematic literature reviews (SLRs) and one targeted literature review (TLR) in PubMed, Embase, and Cochrane databases. The SLRs (no publication date limit; clinical SLR conducted on November 8, 2016; economic SLR conducted on October 25, 2016) searched for randomized clinical trials of neratinib and other treatments and economic data (models, utility, resource use, and cost), and the TLR (publications published from January 2006 to September 2016) searched for burden-of-illness studies in early-stage HER2+ BC. Results: A total of 4,708 abstracts (2,649 clinical SLR; 969 economic SLR; 1,090 TLR) were identified from all searches, and full-text review was conducted for 796 articles (507 clinical SLR; 151 economic SLR; 138 TLR). Of these, 159 (72 clinical SLR; 33 economic SLR; 54 TLR) followed protocol-specified criteria for inclusion. Based on clinical trials in the neoadjuvant and/or adjuvant setting, disease-free survival rates at 4 years ranged from 78% to 90%. HER2-targeting adjuvant regimens such as lapatinib added to trastuzumab and extending trastuzumab to 2 years have been unsuccessful in reducing the risk of recurrence. Women who had a recurrence, regardless of HER2 status, reported significantly poorer functioning on various quality of life (QoL) domains compared with women who remained disease free. All patients with early-stage BC, regardless of HER2 status, diagnosed with their first recurrence experienced cancer-related distress and no improvement in QoL (physical health and functioning) after 1 year. In the US, the total expected per-patient costs for all BC, regardless of HER2 status, over 10 years was $53,454 with metastatic recurrence, $61,601 with locoregional recurrence, and $61,188 with contralateral recurrence as compared with $42,005 (background costs) with no recurrence (2004 US $). The overall cost of recurrence in women with HER2+ BC in the US was estimated to be $240 million to $1.7 billion over the lifetimes of each 1-year cohort of 7,298 patients (2008 US $). Conclusions: These results identified few studies on patients with early-stage HER2+ BC and suggest that future studies are warranted. Recurrence in women with HER2+ BC is associated with decreased QoL and high costs. After adjuvant therapy, there is still risk of recurrence, thus the clinical and economic burden remains. There is an unmet medical need in early-stage HER2+ BC, and new therapies are needed to reduce the risk of recurrence. Citation Format: Masaquel C, Hurley D, Barnett B, Krieger T, Pearson I, Copley-Merriman C, Kaye JA, Moy B. Clinical and economic burden of HER2-positive breast cancer recurrence in the US: A literature review [abstract]. In: Proceedings of the 2017 San Antonio Breast Cancer Symposium; 2017 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2018;78(4 Suppl):Abstract nr P3-10-14.

In clinical trials, intravenous (IV) recombinant tissue-type plasminogen activator (rt-PA) reduces the likelihood of disability if given within 3 hours of acute ischemic stroke. This study compared real-world outcomes between patients treated and patients not treated with IV rt-PA.In this retrospective study, United States-based neurologists randomly selected eligible acute ischemic stroke patients from their charts who were and were not treated with IV rt-PA. Mortality, hospital readmission, and independence were compared between patients treated and patients not treated with IV rt-PA using Kaplan-Meier curves, log-rank tests, and Cox proportional hazards models.A total of 1026 charts were reviewed with a median follow-up time of 15.5 months. Pretreatment stroke severity, as measured by the National Institutes of Health Stroke Scale, was comparable between cohorts (IV rt-PA =11.7; non-rt-PA = 11.3; P = .165). IV rt-PA patients experienced significantly longer survival (P = .013), delayed hospital readmission (P = .012), and shorter time to independence (P .001) compared with patients not treated with rt-PA. After adjusting for baseline characteristics, IV rt-PA patients had significantly lower mortality (hazard ratio [95% confidence interval] = .52 [.30, .90]) and greater rates of independence (hazard ratio [95% confidence interval] = 1.42 [1.17, 1.71]) than patients not treated with rt-PA.This real-world study indicated that acute ischemic stroke patients treated with IV rt-PA experience long-term clinical benefits in survival and functional status.

Individuals with Down syndrome (DS) experience various comorbidities in excess of the prevalence seen among the non-DS population. However, the extent of the excess burden of comorbidities specifically within commercially and publicly insured DS populations aged21 years is not currently known.To (a) describe the most common diagnoses among individuals with DS who have either commercial or Medicaid insurance and (b) compare the prevalence of those diagnoses between DS cases and non-DS controls.This was a longitudinal, retrospective study using health care claims of commercially insured and Medicaid-insured individuals in the Truven Health MarketScan Databases from 2008 to 2015. Individuals aged2, 2-5, 6-11, and 12-20 years with a DS diagnosis (cases; commercial: n = 15,948; Medicaid: n = 11,958) were matched to individuals without DS (controls; commercial: n = 47,844; Medicaid: n = 35,874) using a 1:3 ratio. The annual number of diagnoses was compared between cases and controls within age groups using t-tests, and the prevalence of the most common diagnoses was compared using chi-square tests.Cases in all age groups in both databases had more diagnoses annually than controls (mean =9-17 per year vs. 4-10 per year, P0.001), and the number of diagnoses decreased with age for cases and controls. Among the most common case diagnoses were upper respiratory infections (28.9%-59.1% vs. 19.5%-52.9%); suppurative otitis media (25.1%-56.8% vs. 8.7%-51.2%); nutrition/metabolic/developmental symptoms (37.9%-50.4% vs. 7.7%-10.6%); delays in development (22.8%-52.8% vs. 4.1%-10.9%); and general symptoms (35.1%-47.2% vs. 22.1%-37.2%), and the prevalence of each was greater among cases versus controls in all age groups in both databases (P0.001). The most common diagnoses among controls included some of the same as among cases, as well as acute pharyngitis (18.7%-31.8% vs. 19.2%-30.5%); allergic rhinitis (19.9%-24.3% vs. 15.3%-20.7%); viral/chlamydial infections (24.2%-26.6% vs. 17.7%-23.5%); and joint disorders (11.6% vs. 16.6%), and most were significantly more prevalent among cases (P0.05).Commercially insured and Medicaid-insured individuals aged21 years with DS experience a greater number and prevalence of concomitant diagnoses compared with non-DS individuals. Awareness of these common diagnoses could help facilitate the optimal care of these individuals by the pediatric health care community.This study was sponsored and funded by Genentech. Truven Health Analytics, an IBM Company, receives payment from Genentech to conduct research, including the research for this study. Truven Health Analytics also receives payment from other pharmaceutical companies to conduct research. Kong and Evans are employed by Truven Health Analytics. Csoboth is employed by Genentech. Brixner reports fees paid to the University of Utah by Truven Health Analytics on her behalf for work related to this study. Hurley reports fees from Genentech for work on this study and for work outside of this study. At the time of this study, Visootsak was employed by F. Hoffman-LaRoche Pharmaceuticals, parent company of Genentech. All authors, including those affiliated with the study sponsor, were involved in the design of the study, interpretation of the data, writing of the manuscript, and the decision to submit the manuscript for publication. Study concept and design were contributed by Kong, Hurley, and Brixner, along with Evans. Kong and Evans collected the data, and data interpretation was performed by Csoboth, Visootsak, Brixner, and Hurley, with assistance from Kong. The manuscript was written by Evans, Kong, Hurley, and Brixner and revised by Kong, Hurley, Evans, and Brixner, with assistance from Csoboth and Visootsak.

Background Patients with acute ischemic stroke (AIS) who use emergency medical services (EMS) receive quicker reperfusion treatment which, in turn, mitigates post-stroke disability. However, nationally only 59% use EMS. We examined why AIS patients use or do not use EMS. Methods During 2016–2018, a convenience sample of AIS patients admitted to a primary stroke center in South Carolina were surveyed during hospitalization if they were medically fit, available for survey when contacted, and consented to participate. The survey was programed into EpiInfo with skip patterns to minimize survey burden and self-administered on a touchscreen computer. Survey questions covered symptom characteristics, knowledge of stroke and EMS importance, subjective reactions, role of bystanders and financial factors. Descriptive and multiple regression analyses were performed. Results Of 108 inpatients surveyed (out of 1179 AIS admissions), 49% were male, 44% African American, mean age 63.5 years, 59% mild strokes, 75 (69%) arrived by EMS, 33% were unaware of any stroke symptom prior to stroke, and 75% were unaware of the importance of EMS use for good outcome. Significant factors that influenced EMS use decisions (identified by regression analysis adjusting for stroke severity) were: prior familiarity with stroke (self or family/friend with stroke) adjusted odds ratio, 5.0 (95% confidence interval, 1.6, 15.1), perceiving symptoms as relevant for self and indicating possible stroke, 26.3 (7.6, 91.1), and bystander discouragement to call 911, 0.1 (0.01,0.7). Further, all 27 patients who knew the importance of EMS had used EMS. All patients whose physician office advised actions other than calling EMS at symptom onset, did not use EMS. Conclusion Systematic stroke education of patients with stroke-relevant comorbidities and life-style risk factors, and public health educational programs may increase EMS use and mitigate post-stroke disability.

Background and Purpose- The 2015 updated US Food and Drug Administration alteplase package insert altered several contraindications. We thus explored clinical factors influencing alteplase treatment decisions for patients with minor stroke. Methods- An expert panel selected 7 factors to build a series of survey vignettes: National Institutes of Health Stroke Scale (NIHSS), NIHSS area of primary deficit, baseline functional status, previous ischemic stroke, previous intracerebral hemorrhage, recent anticoagulation, and temporal pattern of symptoms in first hour of care. We used a fractional factorial design (150 vignettes) to provide unconfounded estimates of the effect of all 7 main factors, plus first-order interactions for NIHSS. Surveys were emailed to national organizations of neurologists, emergency physicians, and colleagues. Physicians were randomized to 1 of 10 sets of 15 vignettes, presented randomly. Physicians reported the subjective likelihood of giving alteplase on a 0 to 5 scale; scale categories were anchored to 6 probabilities from 0% to 100%. A conjoint statistical analysis was applied. Results- Responses from 194 US physicians yielded 156 with complete vignette data: 74% male, mean age 46, 80% neurologists. Treatment mean probabilities for individual vignettes ranged from 6% to 95%. Treatment probability increased from 24% for NIHSS score =1 to 41% for NIHSS score =5. The conjoint model accounted for 25% of total observed response variance. In contrast, a model accounting for all possible interactions accounted for 30% variance. Four of the 7 factors accounted jointly for 58% of total relative importance within the conjoint model: previous intracerebral hemorrhage (18%), recent anticoagulation (17%), NIHSS (13%), and previous ischemic stroke (10%). Conclusions- Four main variables jointly account for only a small fraction (15%) of the total variance related to deciding to treat with intravenous alteplase, reflecting high variability and complexity. Future studies should consider other variables, including physician characteristics.

Introduction: The 2015 updated US FDA package insert for rt-PA allows for a greater degree of clinical judgment regarding contraindications; thus there is flexibility in the decision to treat or not to treat. Hypothesis: Identifiable clinical factors influence rt-PA treatment decisions for patients with minor stroke. Methods: An Expert Panel (N=10) selected 7 factors from which to build a series of survey vignettes: NIHSS (1-5), NIHSS area of primary deficit (visual, language, weakness), baseline functional status (fully independent, mild-moderate disability), previous ischemic stroke (IS) (Yes < 6 wks, Yes ≥ 6 wks or No), previous ICH (Yes < 6 mos, Yes ≥ 6 mos, No), recent use of anticoagulation (AC) (Yes < 48 hrs, Yes ≥ 48 hrs, No), and temporal pattern of symptoms in 1 st hr of care (stable, improving). We used a fractional factorial design (150 vignettes) to provide unconfounded estimates of the effect of all 7 main factors, plus first-order interactions for NIHSS. Surveys were emailed to 5 national organizations of neurologists and/or emergency physicians and investigator colleagues. Physicians were randomized to 1 of 10 sets of 15 vignettes, presented in random order. Physicians reported the subjective likelihood of giving rt-PA on a 0-5 scale; scale categories were anchored to probabilities 0, 20, 40, 60, 80, and 100%. A conjoint statistical analysis was applied. Results: Responses from 194 US physicians yielded 156 with complete vignette data: 74% male, mean age 46 (range 27-76), 80% neurologists. Treatment mean probabilities for individual vignettes ranged from 5-95%. Treatment probability increased from 24% for NIHSS=1 to 41% for NIHSS=5. The conjoint model accounted for 25% of total observed response variance. In contrast, a model accounting for all possible interactions accounted for 30% variance. Four of the 7 factors accounted jointly for 58% of total relative importance within the conjoint model: previous ICH (18%), recent AC (17%), NIHSS (13%), and previous IS (10%). Conclusions: Four main variables jointly account for only a small fraction (< 15%) of the total variance related to deciding to treat with IV rtPA, reflecting high variability and complexity. Future studies should consider other variables, including physician characteristics.

Granulocyte colony stimulating factors (G-CSFs) decrease the incidence of febrile neutropenia (FN) in cancer patients receiving myelosuppressive chemotherapy. There are two G-CSFs (pegfilgrastim and filgrastim) that differ in dosing schedules from which oncologists may prescribe.This study aimed to compare the effectiveness of prophylactic pegfilgrastim and filgrastim on the risk of hospitalizations. The secondary objective was to compare the effectiveness of the timing of initiation (prophylactic versus delayed).A retrospective study of administrative claims from US commercial payers included adult patients with Non-Hodgkin's lymphoma, breast, or lung cancer, treated with chemotherapy between July 2004 and January 2008. For these patients, the first course of chemotherapy and each unique cycle with use of G-CSF was identified and designated 'prophylaxis' if used within the first 5 days of each cycle, or 'delayed', if after day 5. The risk of neutropenia-related and all-cause hospitalization was evaluated for the pegfilgrastim and filgrastim prophylaxis cohorts and for the prophylaxis and delayed G-CSF initiation cohorts.Among 5,571 patient-cycles identified, 88.9% and 11.1% used pegfilgrastim and filgrastim respectively. The rate of neutropenic hospitalization was 1.1% for pegfilgrastim prophylaxis and 3.5% for filgrastim prophylaxis (P = 0.001). Compared to chemotherapy cycles with filgrastim prophylaxis, those with pegfilgrastim prophylaxis had decreased risk of neutropenia-related (adjusted odds ratio (OR) = 0.38, 95% confidence interval (CI) 0.17-0.83) and all-cause hospitalization (adjusted OR = 0.51, 95% CI 0.31-0.84). The neutropenic hospitalization rate was 1.2% for G-CSF prophylactic initiation and 3.7% for delayed G-CSF initiation (P 0.001). Chemotherapy cycles with prophylactic initiation of either G-CSF had decreased risk of neutropenia-related (adjusted OR = 0.34, 95% CI 0.21-0.56) and all-cause hospitalization (adjusted OR = 0.67, 95% CI 0.49-0.91) compared with delayed initiation of G-CSF.Pegfilgrastim prescribed as prophylaxis resulted in lower risk of neutropenia-related and all-cause hospitalizations compared to filgrastim prophylaxis. This reduction was similar for prophylactic G-CSF initiation when compared to delayed G-CSF initiation.

To compare the baseline characteristics, episodes of care, and cost of erythropoiesis-stimulating agents among cancer patients in a US managed-care population.Retrospective analysis of administrative claims data. Episodes of care for patients with cancer receiving erythropoiesis-stimulating agents between January 1, 2004 and January 17, 2006 included all claims for erythropoiesis-stimulating agents withor = 42 days' gap between claims, plus the duration of therapeutic benefit based on median days between consecutive doses.Main outcome measures were average weekly dose of erythropoiesis-stimulating agents and costs of therapy.A total of 15,007 eligible episodes of care (darbepoetin alfa, 7769 episodes [5587 patients]; epoetin alfa 7238 episodes [5157 patients]) were identified. Fewer claims were observed per episode of care for darbepoetin alfa than for epoetin alfa (mean [SD] 3.7 [4.1] vs. 5.3 [6.4]). The median time between consecutive claims was 15 days (darbepoetin alfa) and 8 days (epoetin alfa). The mean (SD) weekly doses were 105 (56) microg (darbepoetin alfa) and 34,242 (28173) U (epoetin alfa), a dose-comparison ratio of 326 : 1. Dose-comparison ratios were sensitive to assumptions about duration of clinical benefit. The mean (95% CI) weekly costs were significantly lower for darbepoetin alfa ($560 [553-567]) than for epoetin alfa ($645 [630-659], p0.0001) when duration of clinical benefit was considered.Significant differences characterize patterns of use of erythropoiesis-stimulating agents. Duration of therapeutic benefit is an important variable in comparing darbepoetin alfa with epoetin alfa; incorporation of this variable in analyses of costs of therapy may have notable effects on calculated treatment costs. Limitations of the study include the potential for database errors or omissions, lack of detailed disease data, and lack of adjustment for differences in the ages and comorbidities of patients.

To determine if granulocyte-colony-stimulating factor (G-CSF) primary prophylaxis is associated with a lower risk of febrile neutropenia (FN) than non-primary prophylaxis.This was a retrospective, cohort study of medical records from a random sample of patients with solid tumours and lymphomas treated in 99 community oncology practices in 2003 (n=5319). Consecutively-sampled patients treated with chemotherapy and either filgrastim (Neupogen), pegfilgrastim (Neulasta) or no G-CSF were included (n=3123). Multivariate logistic regression estimated the odds of FN in patients receiving G-CSF primary prophylaxis (within 3 days of first chemotherapy cycle) compared with non-primary prophylaxis (delayed or no G-CSF).Patients receiving primary prophylaxis were less likely to develop FN than patients receiving non-primary prophylaxis (OR=0.49, 95% CI 0.34-0.71, p0.001). Chemotherapy characteristics were associated with development of FN including, receipt of at least three chemotherapy drugs versus one (OR=2.13, 95% CI 1.17-3.89, p=0.014) and regimens with at least one myelosuppressive drug (OR=2.37, 95% CI 1.19-4.73, p=0.014).Patients receiving G-CSF primary prophylaxis had significantly lower odds of developing FN than those receiving non-primary prophylaxis. Incidence of FN may be underestimated, as care not recorded in the medical oncologist's chart was not captured.

The study examined the impact of chemotherapy-induced neutropenic complications (CINC), defined as neutropenia with fever or infection, on short-term disability (STD) among cancer patients receiving chemotherapy.The key outcome metrics were average monthly STD days and associated indirect costs. Patients with and without CINC were propensity score (PS) matched. Multivariate regressions were conducted on PS-matched cohorts to estimate the marginal impact of CINC.A total of 280 patients with CINC were PS-matched to 280 patients without CINC. Compared with matched patients, patients with CINC on average experienced 0.9 more STD day (3.2 vs. 2.3, p=0.046) and $155 more in indirect costs ($549 vs. 394, p=0.050) per month. After multivariate adjustment, patients with CINC experienced 1.0 more STD day (p=0.029), and incurred $200 more in indirect cost (p=0.016) per month.Patients with CINC experience significantly greater STD leave than patients with no neutropenic complications from cancer chemotherapy. The overall study sample only included patients from large self-insured employers in the US and may not reflect the work loss experience of all employed patients in the US or other countries. Indirect costs associated with absenteeism and presenteeism were not measured.

Disease-modifying therapy (DMT) for multiple sclerosis (MS) can reduce relapses and delay progression; however, poor adherence and persistence with DMT can result in sub-optimal outcomes. The associations between DMT adherence and persistence and inpatient admissions and emergency room (ER) visits were investigated.Patients with MS who initiated a DMT in a US administrative claims database were followed for 1 year. Persistence to initiated DMT was measured as the time from DMT initiation to discontinuation (a gap of60 days without drug 'on hand') or end of 1-year follow-up. Adherence to initiated DMT was measured during the persistent period and was operationalized as the medication possession ratio (MPR). Patients with an MPR0.80 were considered non-adherent. Claims during the 1-year follow-up period were evaluated for the presence of an all-cause inpatient admission or an ER visit. Adjusted odds ratios (AORs) for inpatient admission or ER visit comparing persistent vs non-persistent and adherent vs non-adherent patients were estimated using logistic regression models adjusted for patient characteristics.The final sample included 16,218 patients. During the 1-year follow-up period, 35.3% of patients discontinued their initiated DMT and 13.9% were not adherent while on therapy. During that same period, 10.0% of patients had an inpatient admission and 24.9% had an ER visit. The likelihoods of inpatient admission and ER visit were significantly decreased in persistent patients (AOR [95% CI] = 0.50 [0.45, 0.56] and 0.65 [0.60, 0.69], respectively) and in adherent patients (AOR [95% CI] = 0.83 [0.71, 0.97] and 0.86 [0.77, 0.95], respectively).Persistence and adherence with initiated DMT are associated with decreased likelihoods of inpatient admission or ER visit, which may translate to improved clinical outcomes.

Background: CMV disease remains a major complication of lung transplantation and attempts to prevent it have met with marginal success. In a previous study we documented that universal prophylaxis did not prevent CMV disease but merely delayed it, and was very costly. Methods We compared the efficacy and cost of pre-emptive therapy with ganciclovir, guided by CMV antigenemia, to that of historic controls that received universal prophylaxis with ganciclovir. CMV antigenemia assay was done routinely and pre-emptive therapy was initiated if greater than 25 CMV positive cells per 100,000 polymorphonuclear cells were found. Results Nineteen patients were enrolled; 6 of whom received 12 courses of pre-emptive therapy. The incidence of CMV disease was 26% compared to 38% for the historical controls ( p = 0.51). None of the patients that received pre-emptive therapy developed CMV disease following that therapy. Antigenemia failed to predict disease in 5 patients that developed it, and thus it is unknown if pre-emptive therapy could have prevented it. There was no mortality in either the study patients or historic controls directly related to CMV. The net savings with pre-emptive therapy was $2569 per patient. Conclusion We conclude that pre-emptive therapy with ganciclovir is as safe and effective as universal prophylaxis in preventing CMV disease in lung transplant recipients, and is less expensive. The appropriate surveillance technique and timing remain to be determine to optimize the efficacy of pre-emptive therapy.

Background For patients with multiple myeloma (MM) who are refractory to or relapsed after initial therapy, current US clinical guidelines recommend treatment with proteasome inhibitors (PIs) (e.g., bortezomib [V] or carfilzomib [K]) and/or immunomodulatory drugs (IMiDs) (e.g., lenalidomide [R], thalidomide [T] or pomalidomide [P]), combined with corticosteroids. Data on the real world use of these medications, such as rate of initiation of a new line of therapy (LOT), treatment regimens within LOTs, and duration of treatment (DOT), among US patients with relapsed and/or refractory MM are limited. Methods This was a retrospective cohort study of medication use patterns among US patients with MM who were refractory to or relapsed after initial therapy. Data were from the MarketScan commercial and Medicare research databases between Jan 2006 and Dec 2013 (study period). Study patients included adults who were diagnosed with MM (ICD-9-CM code 203.0x) and initiated second line (2L) therapy during the study period, and were continuously enrolled from ≥180 days prior to MM diagnosis to ≥30 days after initiation of 2L. LOT was identified using claims for PIs, IMiDs, and other MM treatments; corticosteroids were not used in identification of LOTs. Treatment regimens were defined based on claims for MM therapies during the first 90 days after start of a LOT. The start date of a new LOT (and the end date of the current LOT) was identified based on the date of the first claim for a new MM therapy (i.e., not in the current regimen), a claim for any MM therapy after a gap of >90 days without a claim for any MM therapy, or end of follow-up (EOF), whichever occurred first. Time within LOT was separated into on- and off-therapy periods; on-therapy period ended on the date of the last claim during the LOT for any therapy in the regimen (or for the last LOT, EOF if the last claim during the LOT was Results A total of 4,693 patients were included in the study, representing 7,683 LOTs (2L=4,693, 3L=1,868, and 4L+=1,122); 56% were male, 59% were in commercial plans, and 20% had transplant before 2L. Mean (SD) age at 2L initiation was 64 (12) years. Use of R-based regimens was greatest in 3L. Based on the regimen classification described above, use of V-based regimens was similar across LOTs (Figure 1A); P- and K-based regimens were used infrequently ( Conclusions Among relapsed or refractory MM patients in the US, use of R- and V-based regimens increased since 2008, while the use of T-based regimens declined. The portion of patients receiving K- and P-based regimens was small but increasing. Median duration of therapy of R-based regimens was about 100 days longer than that of other regimens. These data could be useful for economic evaluations and research and budget planning. Table 1. Rate of New Starts on MM Therapy per 100 Person Years (PSY) Sex, Age PSY 1L 2L 3L 4L+ Female, Disclosures Hagiwara: Onyx Pharmaceuticals: Research Funding. Moynahan:Onyx Pharmaceuticals: Research Funding. Cong:Onyx Pharmaceuticals: Employment, Equity Ownership. Chen:Onyx Pharmaceuticals: Employment. Duchesneau:Onyx Pharmaceuticals: Research Funding. Hurley:Onyx Pharmaceuticals: Consultancy. Delea:Onyx Pharmaceuticals: Research Funding.