Impact of mepolizumab on exacerbations in severe asthma: Results from a U.S. insurance claims data base

Background: In controlled clinical studies, mepolizumab has been shown to reduce exacerbation rates and the use of oral corticosteroids as well as improve asthma control and health-related quality of life compared with placebo in patients with severe eosinophilic asthma. However, real-world data on the impact of mepolizumab on clinical outcomes are limited. Objective: To evaluate the effect of mepolizumab on asthma exacerbations and asthma exacerbation‐related costs in patients with severe asthma in U.S. clinical practice. Methods: This retrospective cohort study used U.S. administrative claims data from patients ages ≥12 years and with severe asthma at mepolizumab treatment initiation (index date; identification period, January 2015‐June 2017) who had received two or more mepolizumab administrations within 180 days of the index date and had no evidence of treatment with another asthma biologic. The exacerbation rate and exacerbation-related costs were assessed in both the 12 months before mepolizumab initiation (baseline period) and the following 12 months (follow-up period). A clinical trial‐like cohort was identified, defined as patients with two or more baseline exacerbations and ≥10 administrations during follow-up. Results: A total of 201 patients were included in the overall population and 74 patients in the clinical trial‐like cohort. Mepolizumab significantly reduced the exacerbation rate between the baseline and follow-up periods in both the overall population and the clinical trial‐like cohort (p < 0.001), which corresponded to 33.6% and 48.6% reductions, respectively. The rate of exacerbations in patients who required hospitalization between the baseline and follow-up periods was also reduced by 35.3% (p = 0.080) and 68.2% (p = 0.015) in the overall population and in the clinical trial‐like cohort, respectively. Cost data were inconclusive. Conclusion: This study, which used real-world data, demonstrated that mepolizumab is associated with reductions in asthma exacerbations, in line with the findings from controlled clinical studies. These results provided further evidence of the effectiveness of mepolizumab in a real-world setting.

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Treatment patterns and characteristics of acute promyelocytic leukemia (APL) patients receiving arsenic trioxide (ATO) therapy in a real-world setting.

Journal of Clinical Oncology ◽

10.1200/jco.2017.35.15_suppl.e18522 ◽

2017 ◽

Vol 35 (15_suppl) ◽

pp. e18522-e18522

Author(s):

Boxiong Tang ◽

Susan Gabriel ◽

Jifang Zhou ◽

Ashutosh K. Pathak ◽

Debra Irwin ◽

...

Keyword(s):

Real World ◽

Index Date ◽

Claims Data ◽

Treatment Patterns ◽

Chronic Obstructive ◽

Administrative Claims ◽

First Line ◽

Real World Data ◽

World Data

e18522 Background: Clinical trials have shown that low-risk APL patients had significantly better outcomes when receiving first-line all-trans retinoic acid (ATRA) + ATO compared with standard ATRA + chemotherapy. Few published studies have used real-world data to describe patients using ATO and their current treatment patterns. This study used United States (US) administrative claims data to describe treatment patterns and characteristics of patients receiving first-line ATO. Methods: This retrospective, observational cohort study used claims data from the MarketScan databases. As there is no ICD-9-CM diagnosis code for APL, ATO treatment was used as a surrogate for the diagnosis of APL since ATO is typically used only in APL patients. Patients were selected if they had ≥1 claims for ATO between January 1, 2000, and June 30, 2015. Date of first use was designated the index date. To identify first-line ATO initiation, patients with ATRA or other APL-indicated chemotherapy claims any time before the index date were excluded. Variable baseline and follow-up periods consisting of ≥3 months of pre-index and ≥30 days of post-index continuous enrollment in medical and pharmacy benefit were used. Results: In total, 331 patients were identified with a subset (n = 265) having ≥2 claims for ATO. The analysis focused on these 265 patients, 54% of whom were male. Mean age was 60.6 years; 45% were covered by Medicare. The most common comorbid conditions measured were diabetes (6%), chronic obstructive pulmonary disease (5%), and congestive heart failure (4%). The most commonly selected APL treatments administered during follow-up were ATRA (17%) and daunorubicin (9%) with the use of idarubicin, cytarabine, and mitoxantrone at less than 3%. Maintenance therapy with methotrexate or 6-mercaptopurine was observed in 7% and 6% of patients, respectively. Conclusions: This is one of the first studies to examine patient characteristics and treatment patterns for first-line ATO using real-world data. Further research is needed to evaluate outcomes for patients receiving ATO as first-line therapy and to re-evaluate treatment guidelines in light of these outcomes.

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Serious infections and cardiovascular complications among patients with chronic lymphocytic lymphoma treated with first-line ibrutinib or bendamustine/rituximab.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.e19503 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. e19503-e19503

Author(s):

Debra E. Irwin ◽

Stephen Thompson ◽

Rinat Ribalov ◽

Azhar Choudhry

Keyword(s):

Real World ◽

Index Date ◽

Cardiovascular Complications ◽

Administrative Claims ◽

First Line ◽

Real World Data ◽

World Data ◽

Serious Infections ◽

Baseline Characteristics

e19503 Background: Clinical trials have shown positive outcomes associated with ibrutinib monotherapy (IM) and bendamustine / rituximab combination (BR) therapy in patients with chronic lymphocytic lymphoma (CLL) compared to other standard treatments, but limited real-world data exist. This study evaluated serious infections and cardiovascular complications in CLL patients treated with first-line IM or BR therapy using US real-world data. Methods: Administrative claims from the MarketScan® Research Databases were used to identify adult patients enrolled in commercial or Medicare supplemental insurance plans based on a first prescription fill of IM or BR therapy (the index date) from 2/1/14 to 9/1/19. Patients were diagnosed with CLL, treatment naïve, and continuously enrolled for ≥12 months prior to and following the index date. Serious infections and cardiovascular complications requiring hospitalization (diagnosis code in any position) were evaluated during a fixed 12-month follow-up period. Statistical differences in outcome distributions between the treatment groups were tested. Multivariate logistic regression models for lower respiratory tract infection (LRTI) and atrial fibrillation (AF) were also conducted to determine the adjusted odds of hospitalization. Results: Of 2,138 CLL patients, 810 had IM and 512 had BR as index therapy with ≥12 months of follow-up data. Patients receiving IM were older and more likely to have had an LRTI during baseline compared to BR patients, otherwise both groups had similar baseline characteristics. Hospitalization for serious infections was more common during follow-up among IM patients than BR patients (17.7% vs. 13.1%; p = 0.027). Specifically, 10.2% of IM patients had a bacterial infection hospitalization compared to 5.7% of BR patients (p = 0.004) and 10.7% of IM patients had a LRTI hospitalization compared to 6.6% of BR patients (p = 0.012). After adjusting for baseline characteristics, IM patients did not have significantly higher odds of a LRTI hospitalization (OR = 1.51; p = 0.069). Hospitalization for cardiovascular complications was more common during follow-up among IM patients than BR patients (18.3% vs. 11.9%; p = 0.002). Specifically, 8.4% of IM patients had an AF-related hospitalization versus 2.7% of BR patients (p < 0.001). After adjusting for baseline characteristics, IM patients were more likely to have a hospitalization for AF versus BR patients (OR = 3.89; p < 0.001). Conclusions: In a real-world setting, serious infections and cardiovascular complications were more common among CLL patients treated with first-line IM compared to BR during 12 months of follow-up. IM patients were more likely than BR patients to have an inpatient admission due to AF after adjusting for other patient characteristics.

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A methodology for the extraction and analysis of real-world radiographic and electronic medical record data to reconstruct treatment arms of historical prostate cancer clinical trials.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.5033 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. 5033-5033

Author(s):

William David Lindsay ◽

Christopher A. Ahern ◽

Aaron Kamauu ◽

Robert Wilder ◽

Karen Chagin ◽

...

Keyword(s):

Prostate Cancer ◽

Clinical Trial ◽

Clinical Trials ◽

Real World ◽

Castration Resistant Prostate Cancer ◽

Efficacy Evaluation ◽

Real World Data ◽

Trial Treatment ◽

Picture Archiving And Communication

5033 Background: Real-world evidence (RWE), including synthetic comparator arms created from historical real-world data (RWD), has the potential to support the safety and efficacy evaluation of new medical products. However, many available RWD sources lack the details necessary to reliably identify patients comparable to clinical trial cohorts or to assess essential oncologic efficacy endpoints. This project demonstrates the ability to extract and analyze RWD to identify patients matching eligibility criteria to four historical clinical trials in metastatic castration-resistant prostate cancer (mCRPC), and calculate outcome measures. Methods: A total of 5,741 patients treated for prostate cancer at multiple institutions (2010-2020) were analyzed in two cohorts using data extracted from the EMR, Tumor Registry, Oncology Information System, and Picture Archiving and Communication System. Of 3,486 patients with prostate cancer in Cohort 1, 422 mCRPC patients were identified: those treated with ADT who achieved castration-level testosterone ( < 50 ng/dL), had evidence of metastatic disease, and exhibited rising PSA (PCWG2). These patients were further matched to four historical clinical trial treatment arms (COU-AA-301: 49, COU-AA-302: 143, AFFIRM: 30, PREVAIL: 79), based on prior chemotherapy and receipt of Abiraterone or Enzalutamide. Overall survival (OS) and time to skeletal related events (SRE) (pathological fracture, spinal compression, surgery to bone, and radiotherapy to bone) were calculated based on diagnosis and procedure codes using the Kaplan-Meier (KM) Estimator. Of 2,255 patients with prostate cancer in Cohort 2, 101 patients received Abiraterone or Enzalutamide and 59 patients had sufficient baseline and follow-up imaging to be scored. Radiographic progression-free survival (rPFS) was calculated from the start of treatment to the time of progression (RECIST 1.1) or loss to follow-up using the KM estimator. Results: In Cohort 1, median OS was 37.7 months (95% CI: 31.5-NR), and median time to SRE was 17.9 months (13.5-22.6). Median OS per patient cohort matched to historical trial treatment arm was COU-AA-301: 23.7 months (10.7-NR), COU-AA-302: 45.9 months (34.9-NR), AFFIRM: 35.3 months (6.34-NR), PREVAIL: 41.5 months (21.9-NR). In Cohort 2, median rPFS was 37.2 months (13.3-NR). Conclusions: The methodology employed in this analysis not only successfully identified a cohort of RWD patients similar to clinical trial-defined patients, but also curated sufficiently reliable data to calculate essential endpoints (e.g., rPFS). At scale, this methodology can be used to generate RWE, including synthetic comparator arms to support clinical trials with radiographic endpoints.

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Real-world incidence and costs of serious infections and cardiovascular complications among patients with indolent non-Hodgkin lymphoma treated with first-line ibrutinib or bendamustine/rituximab.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.e19528 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. e19528-e19528

Author(s):

Debra E. Irwin ◽

Stephen Thompson ◽

Rinat Ribalov ◽

Azhar Choudhry

Keyword(s):

Hodgkin Lymphoma ◽

Real World ◽

Index Date ◽

Cardiovascular Complications ◽

Hospitalized Patients ◽

First Line ◽

Real World Data ◽

Non Hodgkin Lymphoma ◽

Serious Infections

e19528 Background: Patients with Indolent non-Hodgkin lymphoma (iNHL) have shown positive outcomes with ibrutinib monotherapy (IM) as well as bendamustine/rituximab combination (BR) therapy, but no studies have reported on patient outcomes using real-world data. The current analysis evaluated serious infections and cardiovascular complications in iNHL patients treated with first-line IM or BR therapy using US real-world data. Methods: Administrative claims from the MarketScan® Research Databases were used to identify adult patients enrolled in commercial or Medicare supplemental insurance plans based on a first prescription fill of IM or BR therapy (the index date) from 2/1/14 to 9/1/19. Patients included in the study were diagnosed with iNHL, were treatment naïve, and were continuously enrolled for ≥12 months both prior to and following the index date. Serious infections and cardiovascular complications requiring hospitalization (diagnosis code in any position) and associated inpatient costs were evaluated during a fixed 12-month follow-up period. Statistical differences in outcome distributions and costs (reported per patient per month [PPPM]) between the groups were tested. Results: Of 1,948 iNHL patients, 147 had IM and 1,058 had BR as index therapy with ≥12 months of follow-up data. Patients receiving IM were older, more often male, and more likely to have had a lower respiratory tract infection (LRTI) or atrial fibrillation (AF) during baseline compared to BR patients, otherwise the groups had similar baseline characteristics. Hospitalization for serious infections was similar for IM and BR patients during the follow-up period (17.0% vs. 14.4%; p = 0.397); among hospitalized patients, the IM and BR groups incurred similar PPPM inpatient costs ($2,839 vs. $3,954; p = 0.188). Specifically, 7.5% of IM patients had a bacterial infection hospitalization and incurred $2,561 PPPM vs 7.6% of BR patients who incurred $3,975 PPPM (both p > 0.05); 8.8% of IM patients had a LRTI hospitalization and incurred $3,629 PPPM vs 6.1% of BR patients who incurred $4,980 PPPM (both p > 0.05). Hospitalization for cardiovascular complications was similar during follow-up for IM and BR patients (15.6% vs. 12.2%; p = 0.237); among hospitalized patients, the IM and BR groups incurred similar PPPM costs ($3,777 vs. $3,862; p = 0.948). Specifically, 7.5% of IM patients had a hospitalization for AF and incurred $4,481 PPPM vs 3.6% of BR patients who incurred $4,860 PPPM (p = 0.025 and p = 0.875, respectively). Conclusions: In a real-world setting, serious infections and cardiovascular complications requiring hospitalization, including associated costs, were similar among iNHL patients treated with first-line IM or BR over 12 months, although IM patients were more likely to have an AF-related complication.

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Healthcare Resource Utilization and Costs Associated with Obinutuzumab Plus Bendamustine Versus Rituximab Plus Bendamustine for First-Line Treatment of Follicular Lymphoma

Blood ◽

10.1182/blood-2021-145607 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 3004-3004

Author(s):

Tu My To ◽

Jamie T. Ta ◽

Arpamas Seetasith ◽

Rongrong Wang ◽

Dominic Lai ◽

...

Keyword(s):

Clinical Trial ◽

Follicular Lymphoma ◽

Real World ◽

Index Date ◽

Healthcare Resource ◽

Phase Iii ◽

Publicly Traded ◽

First Line ◽

The Past

Abstract Background: Obinutuzumab (G) is an anti-CD20 monoclonal antibody approved in the US for the first-line (1L) treatment of follicular lymphoma (FL), relapsed or refractory FL, and 1L chronic lymphocytic leukemia. G plus chemotherapy (G-chemo) demonstrated superior progression-free survival versus (vs) rituximab (R) plus chemotherapy (R-chemo) in patients with previously untreated FL in the Phase III, randomized GALLIUM study (NCT01332968; Marcus et al. N Engl J Med 2017). R-bendamustine (R-benda) and G-bendamustine (G-benda) are among the most commonly used chemoimmunotherapy (CIT) regimens for FL (Ta et al. J Clin Oncol 2021), and information on comparative healthcare resource use (HRU) and real-world costs associated with G-chemo vs R-chemo in previously untreated FL patients is limited. The aim of this study was to compare HRU and costs for G-benda and R-benda for the 1L treatment of FL using US claims databases. Methods: This was a retrospective cohort study using administrative claims data from the IQVIA PharMetrics® Plus and IBM® MarketScan Commercial and Medicare Supplemental databases. We identified patients aged ≥18 years, who had ≥1 inpatient claim or ≥2 outpatient claims with a diagnosis of FL from February 1, 2015 to September 30, 2020, and received 1L R-benda or G-benda between February 1, 2016 and March 31, 2020. The first claim for FL treatment was the index date. All patients had ≥12 months of pre- and ≥6 months of post-index continuous enrollment in medical and pharmacy benefits. Patients with other primary cancers, FL treatment, diffuse large B-cell lymphoma (DLBCL), or stem cell transplant during the pre-index period, or clinical trial participation or end-stage renal disease during the study period were excluded. Patients initiating 1L G-benda were propensity score matched 1:2 with patients initiating 1L R-benda based on age, sex, Charlson Comorbidity Index (CCI), region, and insurance type. All-cause and FL-related (i.e. claim with a FL diagnosis in any position) HRU and costs (2020 USD) per patient per month (PPPM) during the follow-up period were reported. Patients were followed until the earliest of initiation of second-line therapy, end of continuous follow-up, or end of data availability. Results: Overall, 270 patients were included; of these, 90 (33.3%) patients receiving G-benda were matched to 180 (66.7%) patients receiving R-benda for 1L treatment of FL. 45.2% were male, and the mean (standard deviation [SD]) age and CCI at index date were 59.1 (9.9) years and 1.7 (1.1), respectively. Median follow-up was 7.4 months. After matching, baseline characteristics were well-balanced between R-benda and G-benda patients (standardized mean difference [SMD] <0.1). Both all-cause and FL-related HRU were similar between R-benda and G-benda treated patients across all service categories (Figure 1). Total all-cause mean [SD] PPPM costs were also similar in G-benda vs R-benda patients ($21,378 [$15,242] vs $21,352 [$14,145]; p=0.77) (Figure 2). The majority of the total costs were FL-related and comparable across both patient groups: $17,353 ($11,370) for G-benda vs $17,724 ($13,530) for R-benda (p=0.71). Specifically, FL-treatment related costs PPPM were $17,070 ($14,064) for G-benda; this is comparable to $16,138 ($11,828) PPPM for R-benda (p=0.39). Conclusions: Our study found similar total costs of care and HRU among patients receiving 1L G-benda versus those receiving R-benda, providing real-world economic evidence that complements clinical trial data supporting the use of G-chemo for 1L treatment of FL. Figure 1 Figure 1. Disclosures To: Genentech, Inc.: Current Employment; F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company, Divested equity in a private or publicly-traded company in the past 24 months. Ta: Genentech, Inc.: Current Employment. Seetasith: Genentech, Inc.: Current Employment; F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company. Wang: The SPHERE Institute: Ended employment in the past 24 months; Aurinia Pharmaceuticals Inc.: Current equity holder in publicly-traded company; Novavax, Inc.: Current equity holder in publicly-traded company; Oragenics, Inc.: Current equity holder in publicly-traded company; F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company; Genentech, Inc.: Current Employment; TG Therapeutics, Inc.: Current equity holder in publicly-traded company. Lai: Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company; AbbVie: Current equity holder in publicly-traded company, Divested equity in a private or publicly-traded company in the past 24 months, Ended employment in the past 24 months; Bristol-Myers Squibb: Current equity holder in publicly-traded company. Shapouri: Genentech, Inc.: Current Employment; F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company.

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Dose Modification In MDS Pts Receiving Lenalidomide As a Means To Improve Patient Persistence

Blood ◽

10.1182/blood.v122.21.2987.2987 ◽

2013 ◽

Vol 122 (21) ◽

pp. 2987-2987

Author(s):

B. Douglas Smith ◽

Dalia Mahmoud ◽

Stephanie Lee ◽

Laura Weber

Keyword(s):

Clinical Trial ◽

Clinical Study ◽

Dose Reduction ◽

Real World ◽

Index Date ◽

Response Rates ◽

Baseline Period ◽

Claims Database ◽

The Real

Abstract Background Lenalidomide (LEN) has been studied in transfusion-dependent MDS patients (pts) with IPSS Low/Int-1 risk del5q disease with 55%-70% of pts becoming transfusion-independent (TI) (List, JCO, 2006). A review of Medicare claims showed MDS pts' response rates were higher when receiving >3 cycles of LEN therapy (Zeidan, Cancer 2013, in print). Interestingly, cytopenias and other cytotoxic effects of LEN can challenge physicians and pts to remain on treatment despite the finding that in del5q pts effects may indicate an expected TI Response under study conditions (Sekeres, JCO, 2008). For example, 80% of pts with del5q MDS on the Phase II MDS-003 study required dose modification (DM) due to an adverse event (AE) in achieving excellent response rates, yet less than a third had an AE leading to discontinuation of drug, suggesting that AEs can be managed through DM without discontinuation (Celgene, data on File). Aim This study examined DM and discontinuation in MDS real world pts treated with LEN in the US; results were compared with those seen in the MDS-004 clinical trial. Methods Commercial and Medicare Advantage MDS pts newly initiating therapy with LEN were identified between 01/07 and 12/12 in a US claims database. Both pharmacy and medical benefits and ≥1 fill for LEN indicating a 10 mg/day dose were required; index date was the first LEN claim date. Pts were 18+ years old and continuously enrolled in the health plan 6 months prior to the index date (baseline period) and ≥6 months after the index date (follow-up). At least 1 claim with MDS diagnosis (ICD-9-CM 238.72-238.75) was required during study period. Pts with evidence of LEN during the baseline period were excluded. Pts with a 60+ day gap in LEN fills were considered to have discontinued LEN. Pts with an initial LEN fill of 10 mg/day dose who were subsequently prescribed 5 mg/day dose during the follow-up (prior to discontinuation) were considered to have had dose reduction. LEN fills prior to discontinuation was captured. The clinical study report summarizing the clinical trial data from MDS-004 was examined to identify the proportion of pts who dose modified and/or discontinued during trial. Real world use of LEN was then modeled using clinical trial results from MDS-004 to identify pts who may have benefitted from DM vs. discontinuation of drug. Results Table 1 summarizes use patterns in the MDS-004 clinical trial and real world claims database and estimates impact on real world use of LEN if DM strategy was applied based on MDS-004. In the real world dataset, 168 MDS pts were identified as initiating LEN therapy at 10mg/day. 26% (44 pts) had evidence of DM from 10 mgs to 5 mgs/day in the real world compared to 55% (38 pts) with DM in the MDS-004 study, where LEN dose reductions were required due to AEs. Similarly, 32% (54 pts) discontinued therapy in the real world, compared to 6(9%) pts in the clinical study. Pts who dose reduced in the real world had a higher average persistence of 10.8 LEN fills prior to discontinuation vs. an average of 4.25 LEN fills for pts without of dose reduction. Conclusions Analysis of real world practices examined shows a small proportion of pts dose reduced LEN compared to rates in the clinical trial. LEN's overall impact may be unrealized as studies of prescribing patterns from US claims data suggest that many pt treatment courses are shortened or discontinued after a single course. Additonally, pts who DM were associated with longer persistence on LEN compared to those who did not. Further studies are warranted to assess the clinical factors association with more early discontinuation of LEN vs compared to DM, but we suspect this was in part due to expected and common side effects that were managed more commonly under study conditions. Modeling dosing patterns used in clinical trials of LEN suggests that there are likely pts that can stay on drug longer through DMs, which can be done safely and chances for response would be increased under such care. Disclosures: No relevant conflicts of interest to declare.

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Real-world survival of men with metastatic hormone-sensitive prostate cancer (mHSPC) treated with abiraterone acetate (Abi) or docetaxel (Doc) and comparison with clinical trial outcomes.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.6_suppl.53 ◽

2021 ◽

Vol 39 (6_suppl) ◽

pp. 53-53

Author(s):

Daniel M. Geynisman ◽

Andres F Correa ◽

Chethan Ramamurthy ◽

J Robert Beck ◽

Elizabeth A. Handorf

Keyword(s):

Clinical Trial ◽

Observational Study ◽

Real World ◽

Trial Data ◽

Phase Iii ◽

Real World Data ◽

Large Variability ◽

World Data ◽

Level Data

53 Background: Multiple phase III trials have proven that Abi and Doc both improve overall survival (OS) in men with mHSPC. No randomized trials have compared the two approaches. Methods: We conducted a retrospective, observational study to compare OS in de novo M1 men, treated with Abi vs. Doc using patient-level data from the Flatiron health EHR-derived de-identified database. We also compared this real-world OS to trial level data using extracted data points along the OS curves from CHAARTED and LATITUDE trials. OS was compared via Kaplan-Meier curves. Analyses were adjusted via propensity score weighting for age, Gleason score, PSA at diagnosis, race, ethnicity, ECOG PS, insurance type and treatment setting. Results: The cohort included 418 Abi pts and 807 Doc pts (Table). Median follow-up was 13.5 mo for Abi and 31.6 mo for Doc. Unadjusted median OS for Abi and Doc were 31.6 mo (95% CI 28.1-NA) and 41.8 mo (95% CI 37.4-46.3) respectively (P=0.09). Twelve mo and 24 mo OS for Abi was 86.3% and 69%; for Doc it was 89.8% and 72.1 %. Median adjusted OS for Abi and Doc were 31.6 mo (95% CI 28.0-undefined) and 38.8 mo (95% CI 33.1-46.3) respectively (P=0.4). Twelve mo and 24 mo adjusted OS for Abi was 86.6% and 69.4%; for Doc it was 87.9% and 69.2%. Based on extracted trial data, in LATITUDE, Abi treated pts had 12 mo and 24 mo OS of 93.5% and 77.0%; in CHAARTED, Doc treated pts had 12 mo and 24 mo OS of 94.3% and 83.6%. Conclusions: Utilizing real-world data, we demonstrate that 12 and 24-months OS are clinically and statistically similar between Abi and Doc in men with mHSPC. Median OS is also similar, although due to limited follow-up, the estimate of median OS for Abi has large variability. In addition, we show that clinical trial pts had superior outcomes to those in a real-world clinic population. Recent meta-analyses of trial data have not found significant differences in OS for Abi vs. Doc; this analysis of real-world data confirms these findings and indicates that they may be generalizable to a broader patient population. Although this observational study is subject to residual confounding and missing data, it provides further evidence to support the use of both Abi and Doc in men with mHSPC. Differentiating costs, side-effects and QOL can thus become more prominent when making decisions about therapy. [Table: see text]

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Mortality Among Veterans with a Diagnosis of Pyruvate Kinase (PK) Deficiency: A Real-World Study Using US Veterans Health Administration Data

Blood ◽

10.1182/blood-2020-136693 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 24-25

Author(s):

Erin Zagadailov ◽

Audra Boscoe ◽

Viviana Garcia-Horton ◽

Sherry Shi ◽

Shuqian Liu ◽

...

Keyword(s):

Pyruvate Kinase ◽

Real World ◽

Index Date ◽

Veterans Health ◽

Health Administration ◽

Real World Data ◽

Analysis Group ◽

Diagnosis Codes ◽

Icd 10

Background: Pyruvate kinase (PK) deficiency is a rare, inherited disorder caused by autosomal recessive mutations in the PKLR gene, whereby a glycolytic defect causes reduced adenosine triphosphate levels and leads to hemolytic anemia. Patients with PK deficiency can experience serious complications associated with the disease and its treatment, including osteoporosis, pulmonary hypertension, sepsis, iron overload, and liver cirrhosis. The current standard of care for PK deficiency is supportive, including blood transfusions, splenectomy, iron chelation therapy and/or interventions for other disease-related morbidity. There is no approved therapy for this condition. Identifying PK deficiency in real-world data is challenging due to a lack of diagnosis codes and treatments that are specific to PK deficiency. As a result, population-based studies of PK deficiency using claims or electronic health record databases are limited. In addition, data on mortality in this patient population are lacking and limited to a few individual case reports. This study aimed to identify patients with a PK deficiency diagnosis as documented by physicians and to compare their mortality to an age- and gender-matched cohort of individuals without PK deficiency. Methods: Patients with ≥ 1 diagnosis code related to PK deficiency (i.e., anemia due to disorders of glycolytic enzymes [International Classification of Diseases, Tenth Revision, Clinical Modification (ICD-10-CM) D55.2], other hemolytic anemias due to enzyme deficiency [ICD-9-CM 282.3], or unspecified hereditary hemolytic anemia [ICD-9-CM 282.9, ICD-10-CM D58.9]) between January 1995 and July 2019 were selected from the US Veterans Health Administration (VHA) database. To be considered for inclusion, physicians' notes were required to contain the words "pyruvate", "kinase", and "deficiency." A manual review of these physicians' notes was performed to identify patients with a physician-documented diagnosis of PK deficiency (PK deficiency cohort). The index date for the PK deficiency cohort was defined as the date of the first medical record with a diagnosis code related to PK deficiency. Each patient in the PK deficiency cohort was matched 1:5 by age at index, sex, and index year (± 1 year) to patients from the general VHA population with no diagnosis codes related to PK deficiency (non-PK deficiency cohort). The index date for the non-PK deficiency cohort was defined as a random visit date during their match's index year. Survival time from the index date was compared between the PK deficiency cohort and their non-PK deficiency cohort matches using a univariate Cox proportional hazards model with robust standard error estimation. Results: A total of 18 patients met inclusion criteria for the PK deficiency cohort and were matched to 90 individuals in the non-PK deficiency cohort. Baseline characteristics for both cohorts are shown in the Table. For both cohorts, the mean age at index was 57 years and 94% of patients were male; 83-85% were white and the mean Charlson Comorbidity Index score was 0.4-0.5 (no significant differences between cohorts). Imbalances remained between the two cohorts with regard to region and body mass index. The median follow-up was 6.0 years for the PK deficiency cohort and 8.0 years for the non-PK deficiency cohort. Over the follow-up period, there were 9 (50%) observed deaths in the PK deficiency cohort and 28 (31%) observed deaths in the non-PK deficiency cohort. The median time until death was 10.9 years for the PK deficiency cohort and 17.1 years for the non-PK deficiency cohort; the Kaplan-Meier curves for both cohorts are shown in the Figure. Patients in the non-PK deficiency cohort had a significantly longer time to death than the PK deficiency cohort (hazard ratio: 2.3; p = 0.0306); 10 years after index, 42% of patients in the PK deficiency cohort had died compared with 28% of those in the non-PK deficiency cohort. Conclusions: The results of this study suggest that patients with PK deficiency may be at an increased risk of mortality. Further research to understand cause of death in this population is warranted, as is the replication of this study using larger sample sizes and other real-world data sources that better represent females and the pediatric and adolescent PK deficiency age groups. Disclosures Zagadailov: Agios Pharmaceuticals: Current Employment, Current equity holder in publicly-traded company. Boscoe:Agios Pharmaceuticals: Current Employment, Current equity holder in private company. Garcia-Horton:Agios Pharmaceuticals: Consultancy; Analysis Group, Inc.: Current Employment. Shi:Agios Pharmaceuticals: Consultancy; Analysis Group, Inc.: Current Employment. Liu:Analysis Group, Inc.: Consultancy. Shi:Analysis Group, Inc.: Consultancy. Macaulay:Agios Pharmaceuticals: Consultancy; Analysis Group, Inc.: Current Employment.

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928-P: Dulaglutide Has Higher Adherence and Persistence than Semaglutide and Exenatide QW: 6-Month Follow-Up from U.S. Real-World Data

Diabetes ◽

10.2337/db20-928-p ◽

2020 ◽

Vol 69 (Supplement 1) ◽

pp. 928-P

Author(s):

REEMA MODY ◽

MARIA YU ◽

BAL K. NEPAL ◽

MANIGE KONIG ◽

MICHAEL GRABNER

Keyword(s):

Real World ◽

Real World Data ◽

World Data ◽

Exenatide Qw

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Long Term Real-World Outcomes of Trifluridine/Tipiracil in Metastatic Colorectal Cancer—A Single UK Centre Experience

Current Oncology ◽

10.3390/curroncol28030208 ◽

2021 ◽

Vol 28 (3) ◽

pp. 2260-2269

Author(s):

Daniel Tong ◽

Lei Wang ◽

Jeewaka Mendis ◽

Sharadah Essapen

Keyword(s):

Colorectal Cancer ◽

Metastatic Colorectal Cancer ◽

Real World ◽

Systemic Treatment ◽

Progression Free Survival ◽

Median Number ◽

Current Data ◽

Real World Data

In the UK, Trifluridine-tipiracil (Lonsurf) is used to treat metastatic colorectal cancer in the third-line setting, after prior exposure to fluoropyrimidine-based regimes. Current data on the real-world use of Lonsurf lack long-term follow-up data. A retrospective evaluation of patients receiving Lonsurf at our Cancer Centre in 2016–2017 was performed, all with a minimum of two-year follow-up. Fifty-six patients were included in the review. The median number of cycles of Lonsurf administered was 3. Median follow-up was 6.0 months, with all patients deceased at the time of analysis. Median progression-free survival (PFS) was 3.2 months, and overall survival (OS) was 5.8 months. The median interval from Lonsurf discontinuation to death was two months, but seven patients received further systemic treatment and median OS gained was 12 months. Lonsurf offered a slightly better PFS but inferior OS to that of the RECOURSE trial, with PFS similar to real-world data previously presented. Interestingly, 12.5% had a PFS > 9 months, and this cohort had primarily left-sided and RAS wild-type disease. A subset received further systemic treatment on Lonsurf discontinuation with good additional OS benefit. Lonsurf may alter the course of disease for a subset of patients, and further treatment on progression can be considered in carefully selected patients.

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