Real-World Treatment Patterns and Outcomes Among Patients with Early Non-Small Cell Lung Cancer.

Unlabelled: Worldwide, about two million people are diagnosed with lung cancer each year, 85% of whom have non-small cell lung cancer (NSCLC). Recent progress in treating advanced/metastatic NSCLC with targeted therapies has shifted attention to early NSCLC (Stages I-IIIA) and perioperative (neoadjuvant and adjuvant) systemic therapies. However, our comprehension of how targeted therapeutics are incorporated into care and their impact on patient outcomes is just starting to unfold.

Real-world survival and economic burden among patients with locally advanced or metastatic urothelial carcinoma in the United States.

Background: Given the changing treatment landscape for locally advanced or metastatic urothelial carcinoma (la/mUC), this study aimed to describe real-world treatments, overall survival (OS), health care resource utilization (HCRU), and costs among US patients with la/mUC receiving first-line therapy.

Methods: This retrospective study was conducted using 100% Medicare claims data (2015-2020). Patients with la/mUC were selected; initiation of first-line therapy was the index date.

Real-World Treatment Patterns and Clinical Outcomes in Patients With Locally Advanced or Metastatic Urothelial Carcinoma by Eligibility for Maintenance Avelumab.

Introduction: 1L PBC has historically been recommended for patients with la/mUC. Maintenance avelumab is recommended for patients without disease progression following 1L PBC. Real-world data on the proportion of patients eligible for maintenance avelumab are limited, and outcomes among patients ineligible for maintenance avelumab are uncertain.

Patient Preferences for First-Line Treatment of Locally Advanced or Metastatic Urothelial Carcinoma: An Application of Multidimensional Thresholding.

Objectives: Patient preferences have the potential to influence the development of new treatments for locally advanced/metastatic urothelial carcinoma (la/mUC), and therefore we explored how patients with la/mUC value different attributes of first-line treatments.

Methods: An online preference survey and multidimensional thresholding (MDT) exercise were developed following a targeted literature review and qualitative interviews with physicians, patients with la/mUC, and their caregivers. Treatment attributes included two benefits (overall response rate [ORR], pain related to bladder cancer [scored 0-100; 100 being the worst pain possible]) and four treatment-related risks (peripheral neuropathy, severe side effects, mild to moderate nausea, mild to moderate skin reactions).

Health-related Quality of Life in Patients with Previously Treated Advanced Urothelial Carcinoma from EV-301: A Phase 3 Trial of Enfortumab Vedotin Versus Chemotherapy.

Background And Objective: In comparison to chemotherapy, enfortumab vedotin (EV) prolonged overall survival in patients with previously treated advanced urothelial carcinoma in EV-301. The objective of the present study was to assess patient experiences of EV versus chemotherapy using patient-reported outcome (PRO) analysis of health-related quality of life (HRQoL).

Methods: For patients in the phase 3 EV-301 trial randomized to EV or chemotherapy we assessed responses to the validated European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (QLQ-C30) at baseline, weekly for the first 12 wk, and then every 12 wk until discontinuation.

Patient-Reported Outcomes in Patients With Advanced Urothelial Cancer Who Are Ineligible for Cisplatin and Treated With First-Line Enfortumab Vedotin Alone or With Pembrolizumab.

Purpose: Locally advanced/metastatic urothelial cancer (la/mUC) affects patients' quality of life (QOL) and functioning. We describe the impact of first-line (1L) enfortumab vedotin (EV) alone or with pembrolizumab (P) on QOL/functioning/symptoms in patients with la/mUC who were cisplatin-ineligible from EV-103 Cohort K.

Methods: In this phase Ib/II trial, patients were randomly assigned 1:1 to EV + P or EV monotherapy (mono).

Benchmarking Maintenance Therapy Survival in First-Line Platinum-Based Chemotherapy-Treated Patients with Advanced Urothelial Carcinoma Using Simulated Disease Modeling.

Objective: First-line (1L) maintenance avelumab prolonged overall survival (OS) in patients with advanced urothelial carcinoma (aUC) in JAVELIN Bladder 100. OS was measured from maintenance initiation in patients with disease control following 1L platinum-based therapy (PBT). The OS impact of maintenance for the 1L PBT-treated population is unknown since it was not measured from 1L initiation, nor can it be benchmarked with other 1L therapies.

Health care resource utilization, quality metrics, and costs of bladder cancer within the Oncology Care Model.

Objectives: New and emerging therapies have significantly changed the bladder cancer (BC) treatment landscape and can potentially affect spending and patient care in CMS' Oncology Care Model (OCM), a service delivery and payment model for voluntarily participating practices. The objectives of this analysis were to estimate health care resource utilization (HCRU) and benchmark spending per OCM episode of BC, and to model spending drivers and quality metrics.

Study Design: Retrospective cohort study.

Real-world treatment patterns and clinical outcomes with first-line therapy in patients with locally advanced/metastatic urothelial carcinoma by cisplatin-eligibility.

Introduction: Patients with locally advanced/metastatic urothelial carcinoma (la/mUC) have a poor prognosis. With recent therapeutic advances, data on real-world treatment patterns and overall survival (OS) in patients with la/mUC treated with first-line therapy are limited, particularly when comparing patients who are cisplatin-ineligible versus cisplatin-eligible.

Methods: This was a retrospective observational study of real-world first-line treatment patterns and OS in patients with la/mUC stratified by cisplatin-eligibility and treatment.

Systematic Literature Review (SLR) and Network Meta-Analysis (NMA) of First-Line Therapies (1L) for Locally Advanced/Metastatic Urothelial Carcinoma (la/mUC).

To compare efficacy outcomes for all approved and investigational first-line (1L) treatment regimens for locally advanced or metastatic urothelial carcinoma (la/mUC) with standard of care (SOC), a network meta-analysis (NMA) was conducted. A systematic literature review (SLR) identified phase 2 and 3 randomized trials investigating 1L treatment regimens in la/mUC published January 2001-September 2021. Three networks were formed based on cisplatin (cis) eligibility: cis-eligible/mixed (cis-eligible patients and mixed populations of cis-eligible/ineligible patients), cis-ineligible (strict; exclusively cis-ineligible patients), and cis-ineligible (wide; including studies with investigator's choice of carbo).

Oncology Simulation Model: A Comprehensive and Innovative Approach to Estimate and Project Prevalence and Survival in Oncology.

Objective: We demonstrate a new model framework as an innovative approach to more accurately estimate and project prevalence and survival outcomes in oncology.

Methods: We developed an oncology simulation model (OSM) framework that offers a customizable, dynamic simulation model to generate population-level, country-specific estimates of prevalence, incidence of patients progressing from earlier stages (progression-based incidence), and survival in oncology. The framework, a continuous dynamic Markov cohort model, was implemented in Microsoft Excel.

Understanding Treatment Patterns and Outcomes among Patients with De Novo Unresectable Locally Advanced or Metastatic Urothelial Cancer: A Population-Level Retrospective Analysis from Alberta, Canada.

Despite a high disease burden, real-world data on treatment patterns in patients with unresectable locally advanced or metastatic urothelial carcinoma (la/mUC) in Canada are limited. This retrospective, longitudinal cohort study describes treatment patterns and survival in a population of patients with de novo unresectable la/mUC from Alberta, Canada, diagnosed between 1 January 2015 and 31 December 2019, followed until mid-2020. The outcomes of interest were systemic therapy treatment patterns and overall survival (OS).

Clinical and Patient-Reported Outcomes of Advanced Urothelial Carcinoma Following Discontinuation of PD-1/L1 Inhibitor Therapy.

Introduction: The patterns of care and attrition of locally advanced or metastatic urothelial carcinoma (la/mUC) patients eligible for systemic therapy following PD-1/L1 inhibitors are unclear. The objective of this study was to evaluate the clinical characteristics and treatment patterns among patients with la/mUC following discontinuation of first-line (1L) or second-line (2L) PD-1/L1 inhibitor therapy.

Methods: An ambispective, multisite, chart review study was conducted in the United States, including patients with la/mUC.

Treatment with opioids in patients with locally advanced or metastatic urothelial carcinoma and matched non-cancer controls.

Background: Locally advanced or metastatic urothelial carcinoma (la/mUC) is an aggressive disease with a poor long-term survival. While patients frequently report pain, there are limited data on the patient experience with pain and pain medication use. This study used real-world data to quantify treatment with opioids, as a proxy for pain, in patients with la/mUC compared with matched non-cancer controls.

Qualitative Analysis of Pain in Patients With Locally Advanced or Metastatic Urothelial Carcinoma.

Background: Pain is not well described in patients with locally advanced or metastatic urothelial cancer (la/mUC).

Objective: To characterize pain and assess the content validity of the Brief Pain Inventory Short Form (BPI-SF) worst pain item in patients with la/mUC receiving first-line treatment in the US.

Methods: Qualitative interviews were conducted in patients aged≥45 years with confirmed la/mUC, self-reported la/mUC-attributed pain before enrollment, and no major surgery≤3 months prior to being interviewed.

Health-related Quality of Life of Patients with Locally Advanced or Metastatic Urothelial Cancer Treated with Enfortumab Vedotin after Platinum and PD-1/PD-L1 Inhibitor Therapy: Results from Cohort 1 of the Phase 2 EV-201 Clinical Trial.

Background: The EV-201 trial (NCT03219333) demonstrated a clinically meaningful and durable response rate and a tolerable safety profile with enfortumab vedotin (EV) in patients with locally advanced/metastatic urothelial carcinoma (LA/mUC) treated with prior PD-1/PD-L1 inhibitor therapy and platinum-containing chemotherapy (cohort 1). Patient-reported outcome (PRO) measures were included in EV-201 as exploratory endpoints.

Objective: To evaluate PRO data for cohort 1 of EV-201 to better understand the relationship between EV therapy and health-related quality of life (HRQoL).

Real-world outcomes in locally advanced or metastatic urothelial carcinoma following platinum and PD-1/L1 inhibitor therapy.

To investigate real-world overall survival (rwOS) and real-world progression-free survival (rwPFS) in locally advanced/metastatic urothelial carcinoma postplatinum and postprogrammed death receptor-1/death ligand 1 inhibitors. Adult patients diagnosed with locally advanced/metastatic urothelial carcinoma from 1 January 2011 to 31 December 2018 and treated with taxane monotherapy or any therapy postplatinum and post-PD-1/L1 inhibitors were included from a nationwide electronic health record-derived oncology database. Median rwOS among 72 patients treated with taxane monotherapy was 7.

Real-world burden of illness and unmet need in locally advanced or metastatic urothelial carcinoma following discontinuation of PD-1/L1 inhibitor therapy: A Medicare claims database analysis.

Background: Several programmed death-1 or death-ligand 1 (PD-1/L1) inhibitors are approved first- or second-line therapies for locally advanced or metastatic urothelial carcinoma (la/mUC); however, clinical trials show that only ∼20% of patients respond and all ultimately progress. This study elucidated real-world treatment patterns, healthcare resource utilization (HRU), and economic burden among Medicare beneficiaries with la/mUC who discontinue PD-1/L1 inhibitor therapies.

Methods: We conducted a retrospective claims analysis of patients aged ≥65 years diagnosed with la/mUC (2015-2017) who initiated and subsequently discontinued PD-1/L1 inhibitor therapy (index=date of last administration) using Medicare Fee-for-Service Research Identifiable Files.

The direct and indirect economic burden of hypothyroidism in the United States: a retrospective claims database study.

Objective: To examine direct and indirect economic burden associated with hypothyroidism in the United States.

Methods: Medical costs attributable to hypothyroidism were estimated for patients with hypothyroidism. Non-hypothyroid (euthyroid) controls were matched to patients with hypothyroidism based upon patient characteristics and availability of productivity data.

Epidemiology and treatment patterns for locally advanced or metastatic urothelial carcinoma: a systematic literature review and gap analysis.

Several immuno-oncology (IO) agents targeting programmed death-1 or programmed death-ligand 1 (PD-1/L1) are approved second-line therapy options for patients with locally advanced or metastatic urothelial carcinoma (la/mUC) previously treated with platinum-based chemotherapy or first-line options in patients ineligible for cisplatin whose tumors express PD-L1 or for any platinum-based chemotherapy regardless of PD-L1 expression levels. However, literature on the epidemiology of la/mUC is limited, and real-world treatment patterns are not well established, especially with respect to therapies used following IO. To (a) report the epidemiology of urothelial carcinoma (UC) and la/mUC; (b) identify and summarize the published literature on la/mUC treatment patterns, including IO and post-IO treatment; and (c) identify evidence gaps.

The Impact of Intestinal Complications on Health Care Costs Among Patients With Inflammatory Bowel Disease Treated With Anti-Tumor Necrosis Factor Therapies.

Background: Although there is evidence that anti-tumor necrosis factor (TNF) utilization earlier in the inflammatory bowel disease (IBD) course and before the onset of disease-related complications leads to improved patient outcomes, the health care costs and utilization impact have not been well defined. This study assessed differences in health care utilization and costs among patients with IBD treated with anti-TNFs.

Methods: Patients with a diagnosis of ulcerative colitis (UC) or Crohn disease (CD) between January 1, 2001, and December 31, 2014, were identified from a claims database.

Hypothyroidism Treatment Among Older Adults: Evidence from a Claims Database.

Introduction: Hypothyroidism is a common but often unrecognized condition associated with significant morbidity in the older adult population. This study characterizes a large population of older adults diagnosed with hypothyroidism and examines concordance of their treatment with recommendations from expert bodies, e.g.

Levothyroxine Treatment of Pregnant Women with Hypothyroidism: Retrospective Analysis of a US Claims Database.

Background: Approximately 2-4% of women of reproductive age have hypothyroidism. This study characterizes pregnant women with hypothyroidism and examines adherence to guidelines during pregnancy.

Methods: Women age 18 to 49 who were pregnant in 2014 and identified with hypothyroidism (N = 3448) were included in the retrospective study.

Adherence to thyroid hormone replacement therapy: a retrospective, claims database analysis.

Objective: The objective of this analysis was to compare adherence at 6 months and 12 months across levothyroxine formulations for patients with hypothyroidism.

Methods: This retrospective analysis utilized insurance claims data from a commercially insured population from January 1, 2000 through March 31, 2016. Patients were included if they were diagnosed with hypothyroidism and initiated treatment with generic levothyroxine, Levoxyl, Synthroid, Unithroid, or Tirosint.

The association between adherence to levothyroxine and economic and clinical outcomes in patients with hypothyroidism in the US.

Objective: To evaluate outcomes associated with adherence to levothyroxine (LT4) in the US adult hypothyroidism population.

Methods: We used data from Truven's MarketScan databases from 1 July 2011 through 31 December 2015. Patients aged 18 or older were diagnosed with hypothyroidism (confirmed at least twice) and prescribed LT4.