Association between bronchiectasis exacerbations and longitudinal changes in FEV in patients from the US bronchiectasis and NTM research registry.

Background: This study aimed to evaluate the association between the number of non-cystic fibrosis bronchiectasis (bronchiectasis) exacerbations during baseline and follow-up (objective 1) and to identify longitudinal changes in FEV associated with exacerbation frequency (objective 2).

Methods: This was a retrospective cohort study of adult patients enrolled in the US Bronchiectasis and Nontuberculous Mycobacteria Research Registry September 2008 to March 2020. Objective 1 outcome was association between exacerbations during baseline (24 months) and 0-to-24 month and 24-to-48 month follow-up windows.

Pulmonary exacerbations in insured patients with bronchiectasis over 2 years.

Background: Patients with bronchiectasis experience persistent symptoms and frequent pulmonary exacerbations; this study investigated the frequency of exacerbations and all-cause hospitalisation.

Methods: This longitudinal, retrospective, claims database study (IBM® MarketScan®) identified patients aged ≥18 years from 1 July 2015 through 30 September 2018. Exacerbations were identified by bronchiectasis inpatient claim or a healthcare interaction, followed by antibiotic prescription within 7 days.

Impact of initiation of amikacin liposome inhalation suspension on hospitalizations and other healthcare resource utilization measures: a retrospective cohort study in real-world settings.

Background: Mycobacterium avium complex lung disease (MAC-LD) is an infection that is increasing in frequency, associated with substantial disease burden, and often refractory to treatment. Amikacin liposome inhalation suspension (ALIS) is the first therapy approved for refractory MAC-LD. In the CONVERT study of adult patients with refractory MAC-LD, adding ALIS to a multidrug background regimen showed evidence of MAC infection elimination in sputum by month 6, which was maintained in most patients through the end of treatment (≤ 12 months post-conversion).

Clinical outcomes in high-hypoglycaemia-risk patients with type 2 diabetes switching to insulin glargine 300 U/mL versus a first-generation basal insulin analogue in the United States : Results from the DELIVER High Risk real-world study.

Aims: To compare 12-month clinical effectiveness of insulin glargine 300 units/mL (Gla-300) versus first-generation basal insulin analogues (BIAs) (insulin glargine 100 units/mL [Gla-100] or insulin detemir [IDet]) in patients with type 2 diabetes (T2D) who were at high risk of hypoglycaemia and switched from one BIA to a different one (Gla-300 or Gla-100/IDet) in a real-world setting.

Methods: DELIVER High Risk was a retrospective observational cohort study of 2550 patients with T2D who switched BIA to Gla-300 (Gla-300 switchers) and were propensity score-matched (1:1) to patients who switched to Gla-100 or IDet (Gla-100/IDet switchers). Outcomes were change in glycated haemoglobin A1c (HbA1c), attainment of HbA1c goals (<7% and <8%), and incidence and event rates of hypoglycaemia (all-hypoglycaemia and hypoglycaemia associated with an inpatient/emergency department [ED] contact).

How many people with type 2 diabetes fulfil the eligibility criteria for randomized, controlled trials of insulin glargine 300 U/mL in a real-world setting?

Randomized controlled trial (RCT) populations often do not reflect those typically seen in clinical practice. This retrospective, observational cohort study analysed the real-world data of people with type 2 diabetes (T2DM) prescribed basal insulin analogues from electronic medical records (EMRs) in the Explorys database, which includes data from 39 integrated healthcare systems in the United States, to determine how representative selected RCTs investigating insulin glargine 300 U/mL (Gla-300) are of T2DM populations in a real-world setting. Applying eligibility criteria derived from the EDITION 1, 2 and 3 (Gla-300 vs.

Reduced Hypoglycemia Risk in Type 2 Diabetes Patients Switched to/Initiating Insulin Glargine 300 vs 100 U/ml: A European Real-World Study.

Introduction: Randomized controlled trials and real-world data from the USA have shown similar glycemic control with insulin glargine 300 U/ml (Gla-300) and insulin glargine 100 U/ml (Gla-100) and reduced hypoglycemia risk with Gla-300. This real-world study describes the efficacy and safety of Gla-300 and Gla-100 in patients with type 2 diabetes (T2D) in France, Spain, and Germany.

Methods: This retrospective chart review analysis used anonymized data for adults with T2D switching basal insulin analog (BIA) therapy to Gla-300 or Gla-100, or insulin-naïve patients initiating Gla-300 or Gla-100.

Use of real-world evidence in regulatory decisions for rare diseases in the United States-Current status and future directions.

Following the release of the framework for the Real-World Evidence (RWE) Program, the US Food and Drug Administration (FDA) is actively evaluating and exploring ways to optimize the utility of real-world data (RWD) and RWE to support regulatory decision making. For rare conditions, conducting traditional randomized clinical trials may not always be feasible, and RWD and RWE have played and will continue to play an important role. We use three case examples-cerliponase alfa, asfotase alfa, and uridine triacetate-to illustrate how RWD from disease registries, medical records with chart review, and literature, respectively, have been used to generate RWE to support regulatory decisions for selected rare diseases.

Early hypoglycaemia and adherence after basal insulin initiation in a nationally representative sample of Medicare beneficiaries with type 2 diabetes.

Aims: To estimate risk factors associated with early hypoglycaemia and its impact on adherence to and persistence with therapy in Medicare Part D beneficiaries with type 2 diabetes who are initiating basal insulin (BI).

Materials And Methods: This retrospective analysis used a 5% sample of Medicare files from 2007-2013, identifying beneficiaries with type 2 diabetes initiating BI from 1 January 2008 to 31 December 2012. Early hypoglycaemia was defined as ≥1 hypoglycaemic event ≤6 months postindex.

Switching to insulin glargine 300 units/mL in real-world older patients with type 2 diabetes (DELIVER 3).

Aim: To compare the second-generation basal insulin glargine 300 units/mL (Gla-300) and first-generation basal insulins on glycaemic control and hypoglycaemia risk in older adults with type 2 diabetes (T2D).

Materials And Methods: DELIVER 3 was a retrospective observational cohort study of electronic medical records. A total of 1176 older adults (aged ≥ 65 years) with T2D and ≥1 HbA1c value during 6 month baseline and 3 to 6 month follow-up who switched from basal insulin to Gla-300 were propensity score-matched to 1176 older adults who switched to a first-generation basal insulin [insulin detemir (IDet) or insulin glargine 100 units/mL (Gla-100)].

Comparable glycaemic control and hypoglycaemia in adults with type 2 diabetes after initiating insulin glargine 300 units/mL or insulin degludec: The DELIVER Naïve D real-world study.

Aim: To compare glycaemic control, hypoglycaemia and treatment discontinuation of insulin glargine 300 units/mL (Gla-300) and insulin degludec (IDeg) in a real-world study of insulin-naïve adults with type 2 diabetes (T2D).

Materials And Methods: DELIVER Naive D was a retrospective observational study that used electronic medical record data from the IBM Watson Health Explorys database. Insulin-naïve adults with T2D who started Gla-300 or IDeg between March 2015 and September 2017 were identified.

The US Food and Drug Administration's Risk Evaluation and Mitigation Strategy (REMS) Program - Current Status and Future Direction.

The US Food and Drug Administration (FDA) Amendments Act of 2007 granted the FDA new authorities to enhance drug safety by requiring application holders to submit a proposed Risk Evaluation and Mitigation Strategy (REMS). A REMS is a required risk management plan that uses tools beyond the package insert. REMS elements may include a medication guide and patient package insert for patients and a communication plan focused on health care professionals.

Risk of Wernicke's encephalopathy and cardiac disorders in patients with myeloproliferative neoplasm.

The objectives of the study were to estimate the incidence of Wernicke's encephalopathy (WE) and cardiac disorders among patients with myeloproliferative neoplasms (MPN), and compare it with those without MPN. A total of 39,761 MPN patients were identified from the US Marketscan database. Approximately 27% of them were 65+ years of age, and 51% were male.

Assessment of dronedarone utilization using US claims databases.

Background: A dronedarone utilization study using US MarketScan and InVision Data Mart databases was conducted to estimate the prevalence of the following: (1) dronedarone use in contraindicated patients with worsening heart failure (HF) or hospitalization for HF within 1 month before dronedarone prescription; (2) concomitant prescribing of contraindicated drugs; and (3) recommended creatinine testing after dronedarone initiation among dronedarone users.

Methods: In this retrospective cohort study, data in the MarketScan database between July 20, 2009, and December 31, 2011, and in the InVision Data Mart database between July 20, 2009, and March 31, 2012, were analyzed. The study population included patients who received ≥1 dronedarone prescription during the study period.

Exposure to potentially dangerous drug-drug interactions involving antipsychotics.

Objective: Antipsychotic drug therapy is the cornerstone of treatment of persons with schizophrenia. Because most antipsychotics are metabolized by the hepatic cytochrome P450 system, concomitant use of an antipsychotic and medications that are competitively metabolized by the same system may cause a potentially harmful drug-drug interaction. This study used a large state's Medicaid claims database to examine the proportion of patients exposed to such interactions and the risk factors associated with exposure.

Incidence of serious upper and lower gastrointestinal events in older adults with and without Alzheimer's disease.

Objectives: To estimate and compare the incidence of serious upper and lower gastrointestinal (GI) events in individuals aged 65 and older with and without Alzheimer's disease (AD).

Design: Retrospective cohort study.

Setting: PharMetrics, a large population-based health insurance claims database was used for the study.

Patient-rated troubling symptoms of depression instrument results correlate with traditional clinician- and patient-rated measures: a secondary analysis of a randomized, double-blind, placebo-controlled trial.

Background: The Patient-Rated Troubling Symptoms of Depression (PaRTS-D) instrument assesses the presence and troublesomeness of 8 commonly reported depression-related symptoms from the patient's perspective. A post hoc analysis of a double-blind, randomized risperidone augmentation to antidepressant therapy trial in patients with major depressive disorder explored the relationship between the PaRTS-D instrument and other clinician- and patient-rating scales.

Methods: Patients completed the PaRTS-D; the Patient Global Improvement Scale (PGIS), Quality of Life Enjoyment and Satisfaction Questionnaire (Q-LES-Q), and the Sheehan Disability Scale (SDS), while clinicians completed the Hamilton Rating Scale for Depression (HRSD-17), and the Clinical Global Impressions of Severity (CGI-S) at baseline and at pre-determined study weeks.

Potential bias in testing for hyperprolactinemia and pituitary tumors in risperidone-treated patients: a claims-based study.

Background: A reporting association of risperidone with pituitary tumors has been observed. Because such tumors are highly prevalent, there may be other reasons why they were revealed in association with risperidone treatment. We assessed two potential explanations: disproportionately more prolactin assessment and head/brain imaging in risperidone-treated patients vs patients treated with other antipsychotics.

Psychometric evaluation of a patient-rated troubling symptom scale for generalized anxiety disorder clinical trials.

Background: The majority of clinical outcome assessments developed for generalized anxiety disorder (GAD) may not efficiently and sensitively reflect heterogeneous symptom clusters from a patient perspective. The Patient-Rated Troubling Symptoms Scale for Anxiety (PaRTS-A) instrument was developed to provide an individualized assessment of patient relevant GAD symptoms.

Objective: The objective of this analysis was to evaluate the psychometric characteristics of the PaRTS-A.

Hospitalization rates before and after initiation of paliperidone ER in patients with schizophrenia: results from open-label extensions of the US double-blind trials.

Objective: To assess differences in the number of days hospitalized among schizophrenic patients receiving paliperidone extended-release (paliperidone ER) during the open-label extension (OLE) phases, compared to a similar time period prior to the screening for entry into the double-blind (DB) trials conducted in the United States.

Methods: Mental health-related hospital days during the 52 weeks before entering the DB trials and during the OLE phases were compared. The mean number of hospital days per person per year in the pre- and post-periods was calculated and the statistical significance of pre-post differences was assessed using bootstrap resampling methods.

Comparative costs of ertapenem and piperacillin-tazobactam in the treatment of diabetic foot infections.

Purpose: To evaluate potential cost savings, trial data were used to determine the clinical outcomes for i.v. ertapenem given once daily and i.

Adherence to infectious diseases society of America guidelines for empiric therapy for patients with community-acquired pneumonia in a commercially insured cohort.

Background: There is little published research addressing how the 2003 Infectious Diseases Society of America (IDSA) guidelines for empiric therapy of community-acquired pneumonia (CAP) are implemented in clinical practice.

Objective: This study was designed to describe antibiotic treatment patterns among patients with CAP treated in ambulatory settings in light of the IDSA guidelines.

Methods: Health insurance claims data from a large managed care organization with -30 million enrollees located in geographically diverse regions of the United States were analyzed.

Patterns of health care resource utilization after macrolide treatment failure: results from a large, population-based cohort with acute sinusitis, acute bronchitis, and community-acquired pneumonia.

Background: Macrolide antibiotics are used as first-line therapy for the treatment of respiratory tract infections. The recent emergence of macrolide-resistant pathogens is a major concern.

Objective: This study quantifies the frequency of macrolide treatment failure in respiratory infections and examines its impact on health care use.

Cost-effectiveness analysis of interventions to enhance mammography compliance using computer modeling (CAN*TROL).

Objective: Tailored telephone counseling and physician-based and clinic-based interventions have been shown to be cost-effective in enhancing utilization of mammography among nonadherent women. The objective of this study was to evaluate the costs and benefits of a broad implementation of these interventions from a health payer perspective.

Methods: CAN*TROL computer modeling was employed in the cost-effectiveness analysis of interventions in a 2000 Texas female population.

Impact of diabetes on cognitive function among older Latinos: a population-based cohort study.

Background And Objectives: Type 2 diabetes, which is highly prevalent in older Mexican Americans, may influence cognitive functioning. We examined the association of diabetes with decline in global cognitive function and memory function over a 2-year period.

Methods: Study subjects were derived from an existing cohort of Latinos aged 60 and over in the SALSA project (n=1,789).