scholarly journals 4224 Development of a Patient Registry and Biospecimen Repository to Identify Biomarkers in Nontuberculous Mycobacteria Lung Disease

2020 ◽  
Vol 4 (s1) ◽  
pp. 128-128
Author(s):  
Bryan Garcia ◽  
Abigail Grady ◽  
Lilian Christon ◽  
Patrick Flume ◽  
Susan Dorman
Keyword(s):  
Disease Activity ◽  
Treatment Response ◽  
Sputum Sample ◽  
The United States ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Registry ◽  
Cross Sectional ◽  
Patient Reported ◽  
The Mean

OBJECTIVES/GOALS: Non-tuberculous mycobacteria (NTM) is an emerging infection in the United States. Therapeutic development is impaired in NTM due to lack of biomarkers associated with disease activity and treatment response. To address this, we established a cross-sectional patient registry and biorepository from NTM patients. METHODS/STUDY POPULATION: Beginning August 2019 patients were recruited in a cross-sectional format from the bronchiectasis and NTM clinics at our institution. All patients provided at least one sputum sample in the six months prior to inclusion. Clinical and epidemiologically relevant data was obtained, patient reported outcome measures including the SGRQ were provided, and blood specimens were processed and preserved. Patients were grouped based on clinical phenotype and descriptive statistics were reported as means and standard deviations. Serum inflammatory profiles will be analyzed using standard Luminex assays. RESULTS/ANTICIPATED RESULTS: 72 patients with prior NTM isolation from sputum have been recruited including 28 patients that do not meet ATS guidelines for NTM treatment (colonized), 29 patients are currently receiving treatment, and 15 patients that have a history of completing therapy. Among all NTM patients, the mean age was 59.5 ±17.6 years and 80.8% were female. The mean FEV1 percent predicted among these patients was 68.0 ±21.5% and mean BMI was 22.35 ±4.1. The most common mycobacterial species isolated was Mycobacterium Avium Complex (47%). The mean SGRQ total score was 40.2 ±20.3 among all NTM patients. We plan to perform standard Luminex assays to identify inflammatory profiles associated with disease phenotype. DISCUSSION/SIGNIFICANCE OF IMPACT: We developed a cross sectional cohort of NTM patients including a registry of clinically relevant data for phenotyping and an accompanying biospecimen repository. Our long-term goal is to identify biomarkers indicative of disease activity and treatment response using these components.

scholarly journals Evaluation of the LFA-REAL clinician-reported outcome (ClinRO) and patient-reported outcome (PRO): data from the Peruvian Almenara Lupus Cohort

2020 ◽  
Vol 7 (1) ◽  
pp. e000419
Author(s):  
Manuel Francisco Ugarte-Gil ◽  
Rocio Violeta Gamboa-Cardenas ◽  
Cristina Reátegui-Sokolova ◽  
Victor Román Pimentel-Quiroz ◽  
Paola Zeña-Huancas ◽  
...  
Keyword(s):  
Disease Activity ◽  
Lupus Erythematosus ◽  
Activity Index ◽  
Patient Reported Outcome ◽  
Physician Global Assessment ◽  
Cross Sectional ◽  
Patient Reported ◽  
Activity Measures ◽  
The Mean ◽  
Disease Activity Measures

ObjectiveThe Lupus Foundation of America Rapid Evaluation of Activity in Lupus (LFA-REAL) clinician-reported outcome (ClinRO) and the LFA-REAL patient-reported outcome (PRO) were developed in order to capture manifestations of SLE from the perspective of both the clinician and the patient. The aim of this study is to compare the LFA-REAL ClinRO and PRO with other lupus disease activity measures.MethodsA cross-sectional analysis of patients from a single-centre cohort was performed using Spearman’s correlation. Disease activity measures included were LFA-REAL ClinRO (range 0–1400), LFA-REAL PRO (range 0–1200), Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K), clinical SLEDAI-2K and Physician Global Assessment (PGA, range 0–100).ResultsTwo hundred and twenty-seven patients with SLE were studied. The mean age was 46.3 (SD: 13.8); 212 (93.4%) were female. The mean (SD) LFA-REAL ClinRO was 25.4 (34.7), LFA-REAL PRO was 241.1 (187.6), PGA was 11.9 (15.4), SLEDAI-2K was 2.3 (3.3) and clinical SLEDAI-2K was 1.6 (2.9). The LFA-REAL ClinRO correlated with PGA (r=0.758, p<0.001), SLEDAI-2K (r=0.608, p<0.001) and clinical SLEDAI-2K (r=0.697, p<0.001); the LFA-REAL PRO correlated modestly with PGA (r=0.160, p=0.016), SLEDAI-2K (r=0.121, p=0.069), clinical SLEDAI-2K (r=0.143, p=0.031) and LFA-REAL ClinRO (r=0.161, p=0.015).ConclusionsThe LFA-REAL ClinRO and the LFA-REAL PRO had good and weak correlations, respectively, with several physician-based disease activity measures in a cross-sectional study, suggesting their potential usefulness in establishing disease severity. Longitudinal studies will be required to determine their value in monitoring patients with SLE.

scholarly journals Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Theresa M. Coles ◽  
Adrian F. Hernandez ◽  
Bryce B. Reeve ◽  
Karon Cook ◽  
Michael C. Edwards ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Contextual Factors ◽  
The United States ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Think Tank ◽  
Level Of Evidence ◽  
Patient Reported ◽  
Evidentiary Standards

Abstract Objectives There has been limited success in achieving integration of patient-reported outcomes (PROs) in clinical trials. We describe how stakeholders envision a solution to this challenge. Methods Stakeholders from academia, industry, non-profits, insurers, clinicians, and the Food and Drug Administration convened at a Think Tank meeting funded by the Duke Clinical Research Institute to discuss the challenges of incorporating PROs into clinical trials and how to address those challenges. Using examples from cardiovascular trials, this article describes a potential path forward with a focus on applications in the United States. Results Think Tank members identified one key challenge: a common understanding of the level of evidence that is necessary to support patient-reported outcome measures (PROMs) in trials. Think Tank participants discussed the possibility of creating general evidentiary standards depending upon contextual factors, but such guidelines could not be feasibly developed because many contextual factors are at play. The attendees posited that a more informative approach to PROM evidentiary standards would be to develop validity arguments akin to courtroom briefs, which would emphasize a compelling rationale (interpretation/use argument) to support a PROM within a specific context. Participants envisioned a future in which validity arguments would be publicly available via a repository, which would be indexed by contextual factors, clinical populations, and types of claims. Conclusions A publicly available repository would help stakeholders better understand what a community believes constitutes compelling support for a specific PROM in a trial. Our proposed strategy is expected to facilitate the incorporation of PROMs into cardiovascular clinical trials and trials in general.

scholarly journals The Correlation of Communication Effectiveness and Patient Satisfaction

2021 ◽  
Vol 8 ◽  
pp. 237437352199883
Author(s):  
Yvonne Versluijs ◽  
Maartje Lemmers ◽  
Laura E. Brown ◽  
Amanda I. Gonzalez ◽  
Joost T. P. Kortlever ◽  
...  
Keyword(s):  
Pain Intensity ◽  
Patient Reported Outcome Measures ◽  
Cross Sectional Study ◽  
Patient Reported Outcome ◽  
Communication Effectiveness ◽  
Cross Sectional ◽  
Patient Reported ◽  
Ceiling Effects ◽  
As Relationship ◽  
Current Experience

This study assessed the correlation of 9 questions addressing communication effectiveness (the Communication Effectiveness Questionnaire [CEQ]) with other patient-reported experience measures (PREMs; satisfaction, perceived empathy) as well as patient-reported outcome measures (PROMs; pain intensity, activity tolerance) in patients with musculoskeletal illness or injury. In a cross-sectional study, 210 patients visiting an orthopedic surgeon completed the CEQ and measures of satisfaction with the visit, perceived empathy, pain intensity, and activity tolerance. We evaluated correlations between CEQ and other PREMs and CEQ and PROMs. We measured ceiling effects of the PREMs. Communication effectiveness correlated moderately with other PREMs such as satisfaction (ρ = 0.54; P < .001) and perceived empathy (ρ = 0.54; P < .001). Communication effectiveness did not correlate with PROMs: pain intensity (ρ = −0.01; P = .93) and activity tolerance (ρ = −0.05; P = .44). All of the experience measures have high ceiling effects: perceived empathy 37%, satisfaction 80%, and CEQ 46%. The observation of notable correlations of various PREMs, combined with their high ceiling effects, direct us to identify a likely common statistical construct (which we hypothesize as “relationship”) accounting for variation in PREMs, and then develop a PREM which measures that construct in a manner that results in a Gaussian distribution of scores. At least within the limitations of current experience measures, there seems to be no association between illness (PROMs) and experience (PREMs).

scholarly journals Patient-reported outcome measures in hemodialysis patients: results of the first multicenter cross-sectional ePROMs study in France

2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Abdallah Guerraoui ◽  
Mathilde Prezelin-Reydit ◽  
Anne Kolko ◽  
Marie Lino-Daniel ◽  
Charlotte Dumas de Roque ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Recovery Time ◽  
Patient Characteristics ◽  
Patient Reported Outcome Measures ◽  
Cross Sectional Study ◽  
Patient Reported Outcome ◽  
Dialysis Session ◽  
Cross Sectional ◽  
Cardiovascular Comorbidities ◽  
Patient Reported

Abstract Background Kidney failure with replacement therapy and hemodialysis are associated with a decrease in quality of life (QOL). Self-reported QOL symptoms are not always prioritized by the medical team, potentially leading to conflicting priorities with patients. Electronic patient-reported outcome measures (ePROMs) allow physicians to better identify these symptoms. The objective was to describe the prevalence of symptoms self-reported by hemodialysis (HD) patients. Methods A multicenter cross-sectional study was conducted in three HD centers. Patients were included if they were 18 years old or over treated with HD for at least 3 months in a center. Data were collected by the patient via a self-administered ePROMs questionnaire. Data included patient characteristics, post-dialysis fatigue and intensity, recovery time after a session, perceived stress, impaired sleep the day before the dialysis session, current state of health and the change from the past year. A multivariate analysis was conducted to identify relations between symptoms. Results In total, we included 173 patients with a mean age of 66.2 years, a mean ± SD hemodialysis duration of 48.9 ± 58.02 months. The prevalence of fatigue was 72%. 66% had a high level of stress (level B or C). Recovery time was more than 6 h after a HD session for 25% of patients and 78% declared they had a better or unchanged health status than the previous year. Sleep disturbance was associated with cardiovascular comorbidities (OR 5.08 [95% CI, 1.56 to 16.59], p = 0.007). Conclusions Fatigue and stress were the main symptoms reported by HD patients. The patient’s care teams should better consider these symptoms.

scholarly journals Digital patient-reported outcome measures (PROMs) for remote monitoring of patients on cancer treatment: a cross-sectional questionnaire feasibility study (Preprint)

2020 ◽  
Author(s):  
Mayuran Ananth Sivanandan ◽  
Catherine Sharma ◽  
Pippa Bullard ◽  
Judith Christian
Keyword(s):  
Cancer Treatment ◽  
Outcome Measures ◽  
Remote Monitoring ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Severe Toxicity ◽  
Cross Sectional ◽  
Treatment Pathways ◽  
Face To Face ◽  
Patient Reported

BACKGROUND Oncology has increasing outpatient activity related to increased cancer incidence, better survival rates and more treatments. Innovative technological solutions could help deal with this increasing demand and digital patient-reported outcome measures (PROMs) to identify those patients that need a face-to-face (FTF) appointment is one potential approach. OBJECTIVE Our study aimed to assess the feasibility of digital PROM questionnaires to enable remote symptom monitoring for patients on cancer treatment and their ability to determine the requirement for a FTF appointment. METHODS This study was performed at a tertiary oncology centre between December 2018 and February 2019. Target clinics covered both systemic therapy and radiotherapy cohorts. The Common Terminology Criteria for Adverse Events (CTCAE) helped form the basis for acute toxicity questionnaires which were adapted into patient-friendly language. Treatment-specific digital PROM questionnaires were answered by patients and their clinicians alongside face-to-face appointments. Patients and clinicians did not see each other’s results, which were not used for clinical decisions. Agreement between patients and clinicians was assessed through descriptive statistics. Patient and staff feedback was also obtained. RESULTS 90 patients took part in the study across 10 different treatment pathways. By comparing paired patient and clinician responses, the sensitivity of the patient-completed questionnaires in correctly determining the need for FTF review was 93.6% and no patients with severe toxicity would have been missed with the questionnaires. Digital PROMs revealed 28.9% of participating patients did not need FTF review based on their symptoms. Certain oncological treatment pathways, such as immunotherapy, were found to have a larger proportion of patients with minimal symptoms compared to others, such as conventional chemotherapy. Patient and staff feedback showed high approval with digital PROMs and their potential for use in remote monitoring. CONCLUSIONS Digital PROM questionnaires can feasibly determine the need for FTF review in ‘on treatment’ oncology clinics. Their use with specific treatments could safely reduce the requirement for FTF care and future work should evaluate their application in the remote monitoring of patients.

The long-term outcome of simple trapeziectomy

2018 ◽  
Vol 44 (2) ◽  
pp. 146-150 ◽  
Author(s):  
Thomas F. M. Yeoman ◽  
Oliver Stone ◽  
Paul J. Jenkins ◽  
Jane E. McEachan
Keyword(s):  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Level Of Evidence ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Sustained Improvement ◽  
Patient Reported ◽  
The Mean ◽  
Joint Arthritis

The aim of this study was to investigate the long-term outcome of simple trapeziectomy by a single surgeon and to compare this with pre-operative function. Two hundred and five patients completed the shortened disabilities of the arm, shoulder and hand questionnaire (QuickDASH) and the EuroQoL five-dimensional questionnaire (EQ-5D) scores at a mean of 8.2 (range 3.5–17) years after simple trapeziectomy. There were no pre-operative scores available, so case controls were selected from our prospective database to compare pre- and post-operative patient-reported outcome measures. The mean QuickDASH score of the post-operative matched group was 37 (SD 17.0) and the mean EQ-5D was 0.56 (SD 0.31). The mean QuickDASH score of the pre-operative group was 54 (SD 17.0). The mean difference in QuickDASH between the pre- and post-operative groups was 17 (95% CI: 8 to 26, p = 0.0003). This study demonstrated a significant and sustained improvement in patient-reported function after simple trapeziectomy. It supports that simple trapeziectomy is a simple, safe and effective treatment for advanced trapeziometacarpal joint arthritis. Level of evidence: IV

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