Remote Symptom Alerts and Patient-Reported Outcomes (PROS) in Real-World Breast Cancer Practice: Innovative Data to Derive Symptom Burden and Quality of Life.

Treatment for breast cancer (BC) can lead to debilitating symptoms that can reduce outcomes and quality of life (QoL). Symptom surveillance using a remote symptom monitoring (RSM) platform enables the capture and reporting of patient-reported outcomes (PROs) from home. Women with BC used an RSM platform to complete weekly surveys and report any symptoms experienced during treatment.

The Next Horizon of Drug Development: External Control Arms and Innovative Tools to Enrich Clinical Trial Data.

Conducting clinical trials (CTs) has become increasingly costly and complex in terms of designing and operationalizing. These challenges exist in running CTs on novel therapies, particularly in oncology and rare diseases, where CTs increasingly target narrower patient groups. In this study, we describe external control arms (ECA) and other relevant tools, such as virtualization and decentralized clinical trials (DCTs), and the ability to follow the clinical trial subjects in the real world using tokenization.

Treatment approaches for older and oldest patients with diffuse large B-cell lymphoma - Use of non-R-CHOP alternative therapies and impact of comorbidities on treatment choices and outcome: A Humedica database retrospective cohort analysis, 2007-2015.

Introduction: We characterized real-world treatment patterns in older (65-74 years) and oldest (75-85 years) patients with diffuse large B-cell lymphoma (DLBCL) receiving initial therapy (R-CHOP, non-R-CHOP regimens). Impact of comorbidities on treatment choice, and overall and progression-free survival (OS, PFS) were assessed by age.

Patients And Methods: Using the Humedica database, we identified 1436 newly diagnosed patients with DLBCL who received frontline therapy from 1/07-9/15.

A pragmatic patient-reported outcome strategy for rare disease clinical trials: application of the EORTC item library to myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia.

Background: Novel, pragmatic, patient-centered strategies are needed to ensure fit-for-purpose patient-reported outcomes (PRO) instruments in clinical trial research for rare diseases such as myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML). The objective of the current study was to select supplemental items to add to the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life-Core 30 (QLQ-C30) to ensure content coverage of all important clinical concepts in patients with higher-risk (HR) MDS, low-blast count (LB) AML, and CMML, thus, improving the instrument's ability to detect clinically meaningful treatment benefit for this context of use.

Methods: Our mixed methods approach comprised literature review, clinician consultation (n = 3), and qualitative and quantitative analysis of two stages of patient interview data (n = 14, n = 18) to select library bank items to supplement a generic cancer PRO, the EORTC QLQ-C30.

Meta-analytical methods for estimating outcomes from overall response rate in patients with relapsed/refractory diffuse large B-cell lymphoma.

Relapsed/refractory diffuse large B-cell lymphoma (DLBCL) is highly heterogeneous and current trials are investigating new approaches to improve outcomes. Limited data on response endpoints can confound estimation of a treatment effect when designing studies of novel agents in this setting, which can hinder study sample size calculations, especially if a net estimate is required for a 'physician's choice' comparator arm. Here we estimate complete response rate (CRR), overall response rate (ORR), and extrapolate durable response rates (DRR; CR/partial response lasting ≥16 weeks) for such a comparator arm from published ORRs in DLBCL.

Predicting Outcomes in Patients With Diffuse Large B-Cell Lymphoma Treated With Standard of Care.

In diffuse large B-cell lymphoma (DLBCL), predictive modeling may contribute to targeted drug development by enrichment of the study populations enrolled in clinical trials of DLBCL investigational drugs to include patients with lower likelihood of responding to standard of care. In clinical practice, predictive modeling has the potential to optimize therapy choices in DLBCL. The objectives of this study were to create a model for predicting health outcomes in patients with DLBCL treated with standard of care and determine informative predictors of health outcomes for patients with DLBCL.

Economic burden and treatment patterns for patients with diffuse large B-cell lymphoma and follicular lymphoma in the USA.

Aim: Diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL) are common types of non-Hodgkin's lymphoma, and real-world evidence continues to be lacking for healthcare costs and utilization among DLBCL and FL patients. Our study aims to describe medical and pharmacy costs and health resource utilization and to characterize longitudinal treatment patterns among these patients.

Methods: A retrospective observational study was performed among adult patients with DLBCL or FL using the US MarketScan (Truven) administrative claims data from 1 January 2007 to 31 December 2015.

Evaluation of treatment patterns and survival among patients with diffuse large B-cell lymphoma in the USA.

Aim: To evaluate treatment patterns of diffuse large B-cell lymphoma (DLBCL).

Patients & Methods: First-line and relapsed/refractory treatment patterns and survival outcomes following first-line therapy in adult patients newly diagnosed with DLBCL were evaluated.

Results: A total of 1436 DLBCL patients initiated treatment and mainly received a combination regimen versus monotherapy (92.

A retrospective study evaluating treatment patterns and survival outcomes in elderly patients with acute myeloid leukemia treated in the United States with either 7+3 or a hypomethylating agent.

Intensive treatment for newly diagnosed acute myelogenous leukemia (ND-AML) patients are reserved for "fit" patients. While guidelines recommend evaluation of age, performance status and comorbidities, there is no consensus on the definition of "fitness" or optimal therapy for elderly AML patients. This retrospective study evaluated characteristics and survival outcomes of 274 patients (age ≥60 years) with ND-AML treated with 7 + 3 (cytarabine + an anthracycline) vs.

Treatment Patterns and Survival Outcomes in Patients With Follicular Lymphoma: A 2007 to 2015 Humedica Database Study.

Background: Few studies have evaluated real-world treatment patterns and survival in follicular lymphoma (FL). This study evaluated these outcomes among newly diagnosed patients with FL in routine clinical care.

Patients And Methods: A retrospective study was conducted in newly diagnosed patients with FL from Humedica, a large United States electronic medical record database, from January 1, 2008 to July 31, 2015.

Gastrointestinal complications in patients treated with ipilimumab and nivolumab combination therapy or monotherapy.

Aim & methods: A retrospective study using the IBM Explorys Universe Database assessed the risk of gastrointestinal events (enterocolitis or diarrhea) among melanoma and lung cancer patients treated with ipilimumab and nivolumab combination or monotherapy. Results & conclusion: There were 904 melanoma patients (607 ipilimumab, 140 nivolumab and 157 combo) and 1641 lung cancer patients (68 ipilimumab, 1542 nivolumab and 31 combo). Approximately, 37% of lung patients and 46% of melanoma patients experienced at least one adverse event.

Economic Burden of Patients Treated for Higher-Risk Myelodysplastic Syndromes (HR-MDS) in Routine Clinical Care in the United States.

Background And Objective: Significant clinical burden is associated with higher-risk myelodysplastic syndromes (HR-MDS); however, the economic burden has not been fully examined. We examined cost of care and healthcare utilization (HCU) in HR-MDS patients engaged in routine care in the United States (US).

Methods: Adult US patients diagnosed with HR-MDS from 1/1/2008 to 10/31/2015 were identified from the Optum database.

Gastrointestinal adverse events with combination of checkpoint inhibitors in advanced melanoma: a systematic review.

Introduction: Immunotherapies, including checkpoint inhibitors (CIs) such as cytotoxic T-lymphocyte antigen-4 (CTLA-4) and programmed death-1 (PD-1) inhibitors, are revolutionizing the treatment of advanced melanoma. Combining CTLA-4 and PD-1 inhibitors provides additional clinical benefit compared with single agents alone. However, combination therapy can increase the incidence of gastrointestinal adverse events (GI AEs).

Systematic review of therapy used in relapsed or refractory diffuse large B-cell lymphoma and follicular lymphoma.

To identify real-world evidence on outcomes from therapies for relapsed/refractory diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL), we systematically reviewed literature in Medline/Embase for DLBCL/FL-related articles on real-world results published during January 2012-May 2016. Among 33 included articles, therapies included stem cell transplant (SCT) and chemotherapy, including experimental regimens. The highest overall survival rates were observed for SCT, long considered an optimal strategy following initial relapse.

Economic burden of elderly patients with acute myeloid leukemia treated in routine clinical care in the United States.

This retrospective claims database study examined healthcare utilization (HCU) and costs associated with acute myeloid leukemia (AML) in 237 elderly patients who received chemotherapy or a stem cell transplant (SCT) following AML diagnosis. Patients with secondary AML were excluded. Over the entire follow-up period, 92.

Transfusion-free interval is associated with improved survival in patients with higher-risk myelodysplastic syndromes engaged in routine care.

Most higher-risk myelodysplastic syndrome (HR-MDS) patients will become transfusion-dependent, leading to potential complications, including infections or end-organ dysfunction. Data correlating achievement of transfusion-free intervals (TFIs) during first-line therapy (1LT) with survival are sparse. We evaluated HR-MDS patients receiving 1LT diagnosed from 1/1/2008 to 7/31/2015 and the impact of a TFI (≥60-day interval without transfusions) on progression-free and overall survival (PFS, OS) using Cox proportional-hazard models.

Economic burden of patients with diffuse large B-cell and follicular lymphoma treated in the USA.

Aim: Evaluate healthcare costs and utilization of treated diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL) patients.

Materials & Methods: Adults with newly diagnosed DLBCL and FL between 1 January 2008 and 31 October 2015 were identified in the Optum™ claims database. Healthcare costs and utilization were assessed from diagnosis date until end of follow-up.

Effectiveness and Safety of Therapeutic Regimens for Elderly Patients With Acute Myeloid Leukemia: A Systematic Literature Review.

Acute myeloid leukemia (AML) is the second most common leukemia among adults. Although the median age at diagnosis is 67 years, with approximately one third of patients aged 75 years or older, limited treatment options exist for the elderly, who have 5-year survival rates of only 5%. A systematic review was conducted to examine effectiveness and safety outcomes of treatment regimens in elderly (≥60 years old) patients with AML.

Systematic Literature Review of Treatment Options and Clinical Outcomes for Patients With Higher-Risk Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia.

High-dose chemotherapy with allogeneic hematopoietic stem cell transplantation (allo-HSCT) can produce long-term remission in patients with higher-risk myelodysplastic syndromes (HR-MDS) and chronic myelomonocytic leukemia (CMML). However, this treatment regimen is not appropriate for elderly and/or comorbid patients; in these cases, azacitidine is a standard treatment. This systematic review was conducted to evaluate real-world evidence of treatment options for patients with HR-MDS/CMML.

Relationship between age and erectile dysfunction diagnosis or treatment using real-world observational data in the USA.

Aims: With self-reporting of erectile dysfunction (ED) in population-based surveys, men with ED may not represent men who are bothered sufficiently to seek an ED diagnosis and treatment. We used real-world observational data to assess: 1) the prevalence of ED diagnosis or treatment by age subgroups; and 2) the relationship of age with ED diagnosis or treatment after controlling for ED-related comorbidities in the USA.

Methods: This cross-sectional study used de-identified claims data (MarketScan databases; primary analysis).

Utilization of Positive and Negative Controls to Examine Comorbid Associations in Observational Database Studies.

Background: Opportunities to leverage observational data for precision medicine research are hampered by underlying sources of bias and paucity of methods to handle resulting uncertainty. We outline an approach to account for bias in identifying comorbid associations between 2 rare genetic disorders and type 2 diabetes (T2D) by applying a positive and negative control disease paradigm.

Research Design: Association between 10 common and 2 rare genetic disorders [Hereditary Fructose Intolerance (HFI) and α-1 antitrypsin deficiency] and T2D was compared with the association between T2D and 7 negative control diseases with no established relationship with T2D in 4 observational databases.

Impact of Out-of-pocket Costs on Varenicline Utilization and Persistence.

Varenicline is a smoking cessation medication. We analyzed patients' out-of-pocket costs and utilization of and persistence with varenicline. De-identified claims data in the MarketScan® Commercial Claims and Encounters Database were analyzed retrospectively.

Association of out-of-pocket pharmacy costs with adherence to varenicline.

Background: Varenicline, a nicotinic acetylcholine receptor partial agonist, is a pharmacotherapy indicated for smoking cessation treatment. To date, no research has examined the relationship between out-of-pocket (OOP) expense and varenicline adherence among Medicare beneficiaries.

Objectives: To (a) characterize medication utilization patterns of varenicline among Medicare members newly initiated on varenicline and (b) examine the relationship between member OOP expense and varenicline medication adherence.

Burden of smoking on quality of life in patients with chronic obstructive pulmonary disease.

The objective of this study was to assess the impact of smoking on health-related quality of life, Work Productivity and Activity Impairment (WPAI) in chronic obstructive pulmonary disease (COPD) patients. Respondents of the 2009/2010 US National Health and Wellness Survey (NHWS), aged ≥ 40 years, with COPD, chronic bronchitis or emphysema, were included in the study. Current and former (had not smoked for ≥ 11 years) smokers were compared.

Impact of access restrictions on varenicline utilization.

Aim: To assess the impact of access restrictions on varenicline utilization.

Methods: Employer-sponsored health plans contributing to the MarketScan Commercial Claims and Encounters Database were categorized according to 2009 varenicline access restrictions: no coverage; prior authorization; smoking cessation program requirement; no restrictions. The cohort comprised all adults continuously enrolled in plans during 2009.