Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one allele: 144-week interim results from a 192-week open-label extension study.

Background: In two pivotal phase 3 trials, up to 24 weeks of treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was efficacious and safe in patients with cystic fibrosis (CF) ≥12 years of age who have at least one allele. The aim of this study is to assess long-term safety and efficacy of ELX/TEZ/IVA in these patients.

Methods: In this phase 3, open-label, single-arm extension study, participants with -minimal function (from a 24-week parent study; n=399) or - (from a 4-week parent study; n=107) genotypes receive ELX/TEZ/IVA at the same dose (ELX 200 mg once daily, TEZ 100 mg once daily and IVA 150 mg every 12 h).

A Hybrid Effectiveness/Implementation Clinical Trial of Adherence to Long-Term Oxygen Therapy for Chronic Obstructive Pulmonary Disease.

Interventions to promote adherence to long-term oxygen therapy (LTOT) in chronic obstructive pulmonary disease (COPD) are needed. To examine the real-world effectiveness of phone-based peer coaching on LTOT adherence and other outcomes in a pragmatic trial of patients with COPD. In a hybrid effectiveness/implementation pragmatic trial, patients were randomized to receive phone-based proactive coaching (educational materials, five phone-based peer coaching sessions over 60 d), reactive coaching (educational materials, peer coaching when requested), or usual care.

Non-respiratory health-related quality of life in people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor.

Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with cystic fibrosis (CF) heterozygous for F508del and a minimal function mutation (F/MF) or homozygous for F508del (F/F) in two pivotal Phase 3 trials, significantly improving percentage predicted forced expiratory volume in 1 second, Cystic Fibrosis Questionnaire-Revised, Respiratory Domain (CFQ-R RD) scores, and sweat chloride concentration. Here, we analyzed the 11 non-respiratory domains (non-RDs) of the CFQ-R, which assess general health-related quality of life (i.e.

Pragmatic Clinical Trial to Improve Patient Experience Among Adults During Transitions from Hospital to Home: the PArTNER study.

Introduction: Minority-serving hospitals (MSHs) need evidence-based strategies tailored to the populations they serve to improve patient-centered outcomes after hospitalization.

Methods: We conducted a pragmatic randomized clinical trial (RCT) from October 2014 to January 2017 at a MSH comparing the effectiveness of a stakeholder-supported Navigator intervention vs. Usual care on post-hospital patient experience, outcomes, and healthcare utilization.

Triple Therapy for Cystic Fibrosis -Gating and -Residual Function Genotypes.

Background: Elexacaftor-tezacaftor-ivacaftor is a small-molecule cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen shown to be efficacious in patients with at least one allele, which indicates that this combination can modulate a single allele. In patients whose other allele contains a gating or residual function mutation that is already effectively treated with previous CFTR modulators (ivacaftor or tezacaftor-ivacaftor), the potential for additional benefit from restoring Phe508del CFTR protein function is unclear.

Methods: We conducted a phase 3, double-blind, randomized, active-controlled trial involving patients 12 years of age or older with cystic fibrosis and -gating or -residual function genotypes.

A Systematic Review of the Effect of Physical Activity on Asthma Outcomes.

Background: Asthma is a chronic respiratory disease that affects millions worldwide. Medication management is the current mainstay of treatment; however, there is evidence to suggest additional benefit with lifestyle changes, particularly with increased physical activity.

Objective: To discover and evaluate the effects of physical activity on asthma outcomes.

Home Oxygen Therapy for Adults with Chronic Lung Disease. An Official American Thoracic Society Clinical Practice Guideline.

Evidence-based guidelines are needed for effective delivery of home oxygen therapy to appropriate patients with chronic obstructive pulmonary disease (COPD) and interstitial lung disease (ILD). The multidisciplinary panel created six research questions using a modified Delphi approach. A systematic review of the literature was completed, and the Grading of Recommendations Assessment, Development and Evaluation approach was used to formulate clinical recommendations.

Home Oxygen Evaluation by Respiratory Therapists in Patients Hospitalized for COPD Exacerbations: The RIsOTTO Study.

Background: The majority of prescriptions for supplemental oxygen are written when patients are discharged to home from the hospital and the evaluation of these patients is inconsistent. Respiratory Therapists receive training in the evaluation and management of patients needing oxygen. The primary goal of the study was to estimate the frequency with which respiratory therapists (RTs) evaluate the need for home oxygen in patients hospitalized for COPD exacerbations before discharge.

The Burden of Cough and Phlegm in People With COPD: A COPD Patient-Powered Research Network Study.

Rationale: Cough and phlegm are common symptoms of chronic obstructive pulmonary disease (COPD) and may significantly affect quality of life. This study assessed the burden of cough and phlegm on clinical outcomes and quality of life among people with a self-reported physician diagnosis of COPD.

Methods: Patient-reported data from the COPD Foundation's Patient-Powered Research Network (COPD PPRN) were utilized.

Home Oxygen Therapy for Patients With COPD: Time for a Reboot.

Just over 100 years ago, John Scott Haldane published a seminal report about the therapeutic potential of supplemental oxygen to treat hypoxemia. In the 1980s, a pair of clinical trials confirmed the benefit of long-term oxygen therapy in improving survival in patients with COPD associated with severe resting hypoxemia. This review provides a summary of evidence supporting long-term and short-term oxygen therapy, as well as the various types of oxygen equipment commonly used in homes to deliver supplemental oxygen.

Daily Physical Activity in Patients With COPD After Hospital Discharge in a Minority Population.

Background: Low physical activity in patients with chronic obstructive pulmonary disease (COPD) is associated with increased morbidity and mortality. To inform the design of a home-based physical activity promotion program for patients with COPD recently discharged from a minority-serving hospital, we conducted a cohort study to evaluate objectively measured daily physical activity and patient-reported outcomes.

Methods: This was a 12-week prospective cohort study of patients with a physician diagnosis of COPD recently hospitalized (≤ 12 weeks) for respiratory symptoms.

Design of the patient navigator to Reduce Readmissions (PArTNER) study: A pragmatic clinical effectiveness trial.

Previous work indicates the potential for community health workers and peer coaches serving as patient navigators to improve processes of care and health outcomes during care transitions, but have not been sufficiently tested to determine if such programs improve measures of patient experience in minority serving institutions. The objectives of the Patient Navigator to Reduce Readmissions (PArTNER) study was to: 1) conduct a pragmatic clinical effectiveness trial comparing a multi-faceted, stakeholder-supported Navigator intervention (in-person CHW visits in the hospital and after hospital discharge, plus telephone-based peer coaching) versus usual care on the experience of hospital-to-home care transitions in patients hospitalized with heart failure, pneumonia, chronic obstructive pulmonary disease, myocardial infarction, or sickle cell disease; 2) examine the effectiveness of the Navigator intervention in patient subgroups; and 3) understand the barriers and facilitators of successfully implementing the Navigator intervention across patient populations. The co-primary outcomes are the 30-day changes in: 1) Patient Reported Outcomes Measurement Information System (PROMIS) emotional distress-anxiety, and 2) PROMIS informational support.

Optimizing Home Oxygen Therapy. An Official American Thoracic Society Workshop Report.

More than 1.5 million adults in the United States use supplemental oxygen for a variety of respiratory disorders to improve their quality of life and prolong survival. This document describes recommendations from a multidisciplinary workshop convened at the ATS International Conference in 2017 with the goal of optimizing home oxygen therapy for adults.

Evaluation and Documentation of Supplemental Oxygen Requirements is Rarely Performed in Patients Hospitalized With COPD.

Patients hospitalized with chronic obstructive pulmonary disease (COPD) who require supplemental oxygen (O) are at increased risk of hospital readmissions. There is a paucity of information regarding quality of evaluation and documentation regarding the need for supplemental O in this population.  To determine the extent to which evaluation and documentation regarding the need for supplemental O occurs prior to hospital discharge in patients with COPD.

Thoracic splenosis: History is the key.

Splenosis is an acquired ectopic autotransplantation of splenic tissue; that occurs after traumatic splenic rupture and splenectomy [1]. Splenosis is a rare but benign disease, and the diagnosis can be challenging as the multiple incidentally found nodules could mimic malignancy [2]. Abdominopelvic Splenosis is thought to occur in as many as 65% of cases of splenic rupture [1].

Engaging Patients and Caregivers to Design Transitional Care Management Services at a Minority Serving Institution.

Limited socioeconomic resources contribute to high readmission rates at minority serving institutions (MSIs). A better understanding of patient-level factors and need for patient navigators could inform approaches to enhance care transitions tailored to these vulnerable patient populations. We sought to understand the perspectives of patients and their caregivers about hospital to home transitions from an MSI, as well as their attitudes about patient navigators to facilitate care transitions.

Can Commercially Available Pedometers Be Used For Physical Activity Monitoring In Patients With COPD Following Exacerbations?

Background: Commercially available pedometers have been used as tools to measure endpoints in studies evaluating physical activity promotion programs. However, their accuracy in patients recovering from COPD exacerbations is unknown. The objectives of this study were to 1) assess the relative accuracy of different commercially available pedometers in healthy volunteers and 2) evaluate the accuracy of the top-performing commercially available pedometer in patients recovering from COPD exacerbations following hospital discharge.

Patient Involvement in the Design of a Patient-Centered Clinical Trial to Promote Adherence to Supplemental Oxygen Therapy in COPD.

Background: Patients are increasingly viewed as key stakeholders who can contribute in meaningful ways to clinical research and are emphasized in research funded by the Patient-Centered Outcomes Research Institute (PCORI). We are not aware of other peer-reviewed publications that report methods and outcomes of patient engagement to refine study design for a PCORI-sponsored clinical effectiveness trial.

Objective: The aim of this report was to describe the process and outcomes of involving patients in the design of a clinical trial to promote adherence to supplemental oxygen therapy among patients with chronic obstructive pulmonary disease.

Integrating COPD into Patient-Centered Hospital Readmissions Reduction Programs.

About 1 in 5 patients hospitalized for exacerbations of chronic obstructive pulmonary disease (COPD) in the United States are readmitted within 30 days. The U.S.

Multicenter study comparing case definitions used to identify patients with chronic obstructive pulmonary disease.

Rationale: Clinical trials in chronic obstructive pulmonary disease (COPD) usually require evidence of airflow obstruction and clinical risk factors. International Classification of Diseases, Ninth Revision (ICD-9) diagnosis codes or patient-reported physician diagnoses are often used for epidemiologic studies and performance improvement programs.

Objectives: To evaluate agreement between these case definitions for COPD and to assess the comparability of study populations identified as having COPD not using the clinical trial reference standard.