scholarly journals Improving interpretability of individual Diabetes Symptom Checklist-Revised (DSC-R) scores: the role of patient characteristics

2020 ◽  
Vol 8 (1) ◽  
pp. e001146 ◽  
Author(s):  
Thomas H Wieringa ◽  
Maartje de Wit ◽  
Jos W R Twisk ◽  
Frank J Snoek
Keyword(s):  
Symptom Burden ◽  
Patient Characteristics ◽  
Well Being ◽  
Patient Reported Outcome ◽  
Symptom Checklist ◽  
Hypoglycemic Episode ◽  
Patient Reported ◽  
Almost All ◽  
Domain Scores ◽  
R Domain

IntroductionThe Diabetes Symptom Checklist-Revised (DSC-R) is a well-validated patient-reported outcome designed to assess symptom burden in persons with type 2 diabetes mellitus (T2DM) across eight domains. The DSC-R has so far primarily been used in research settings. With the aim to make the DSC-R applicable in clinical practice by improving its interpretability, we sought to identify patient characteristics associated with DSC-R (domain) scores as a first initiative toward reference values.Research design and methodsWe used baseline data from two large observational studies to select patient characteristics significantly associated with DSC-R domain and total scores. Multivariable Tobit analyses with the backward procedure per (domain) score were performed.ResultsData from 1531 participants with T2DM were included. On a 0–100 scale, the median DSC-R total score was 15.88 (7.06–29.41), with domain scores ranging from 5.00 (0.00–22.50) (pain) to 35.00 (10.00–60.00) (fatigue). Low well-being status was most profoundly associated with higher scores across all domains. Persons with one or more complication, as well as one or more symptomatic hypoglycemic episode during the past 3 months, scored higher on (almost) all domains and the total scale.ConclusionsComplications, symptomatic hypoglycemia, and low well-being are important characteristics to take into account when using the DSC-R in individual patients. Further validation of our findings is warranted in diverse patient populations.

Preoperative Opioid Use in Patients Undergoing Common Hand Surgeries

Hand ◽  
2021 ◽  
pp. 155894472097412
Author(s):  
Ali Aneizi ◽  
Dominique Gelmann ◽  
Dominic J. Ventimiglia ◽  
Patrick M. J. Sajak ◽  
Vidushan Nadarajah ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Hand Surgery ◽  
Patient Characteristics ◽  
Patient Reported Outcome Measures ◽  
Pain Scale ◽  
Patient Reported Outcome ◽  
Measurement Information ◽  
Opioid Use ◽  
Patient Reported ◽  
Opioid Users

Background: The objectives of this study were to determine the baseline patient characteristics associated with preoperative opioid use and to establish whether preoperative opioid use is associated with baseline patient-reported outcome measures in patients undergoing common hand surgeries. Methods: Patients undergoing common hand surgeries from 2015 to 2018 were retrospectively reviewed from a prospective orthopedic registry at a single academic institution. Medical records were reviewed to determine whether patients were opioid users versus nonusers. On enrollment in the registry, patients completed 6 Patient-Reported Outcomes Measurement Information System (PROMIS) domains (Physical Function, Pain Interference, Fatigue, Social Satisfaction, Anxiety, and Depression), the Brief Michigan Hand Questionnaire (BMHQ), a surgical expectations questionnaire, and Numeric Pain Scale (NPS). Statistical analysis included multivariable regression to determine whether preoperative opioid use was associated with patient characteristics and preoperative scores on patient-reported outcome measures. Results: After controlling for covariates, an analysis of 353 patients (opioid users, n = 122; nonusers, n = 231) showed that preoperative opioid use was associated with higher American Society of Anesthesiologists class (odds ratio [OR], 2.88), current smoking (OR, 1.91), and lower body mass index (OR, 0.95). Preoperative opioid use was also associated with significantly worse baseline PROMIS scores across 6 domains, lower BMHQ scores, and NPS hand scores. Conclusions: Preoperative opioid use is common in hand surgery patients with a rate of 35%. Preoperative opioid use is associated with multiple baseline patient characteristics and is predictive of worse baseline scores on patient-reported outcome measures. Future studies should determine whether such associations persist in the postoperative setting between opioid users and nonusers.

Patient-Reported Outcome Measures in Colorectal Surgery: Construction of Core Measures Using Open-Source Research Method

2021 ◽  
pp. 155335062199887
Author(s):  
Alaa El-Hussuna ◽  
Ines Rubio-Perez ◽  
Monica Millan ◽  
Gianluca Pellino ◽  
Ionut Negoi ◽  
...  
Keyword(s):  
Colorectal Surgery ◽  
Outcome Measures ◽  
Short Form ◽  
Wide Spectrum ◽  
Core Outcome Set ◽  
Well Being ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
Eortc Qlq

Purpose. The primary aim of the study was to review the existing literature about patient-reported outcome measures (PROMs) in colorectal cancer and IBD. The secondary aim was to present a road map to develop a core outcome set via opinion gathering using social media. Method. This study is the first step of a three-step project aimed at constructing simple, applicable PROMs in colorectal surgery. This article was written in a collaborative manner with authors invited both through Twitter via the #OpenSourceResearch hashtag. The 5 most used PROMs were presented and discussed as slides/images on Twitter. Inputs from a wide spectrum of participants including researchers, surgeons, physicians, nurses, patients, and patients’ organizations were collected and analyzed. The final draft was emailed to all contributors and 6 patients’ representatives for proofreading and approval. Results. Five PROM sets were identified and discussed: EORTC QLQ-CR29, IBDQ short health questionnaire, EORTC QLQ-C30, ED-Q5-5L, and Short Form-36. There were 315 tweets posted by 50 tweeters with 1458 retweets. Awareness about PROMs was generally limited. The general psycho-physical well-being score (GPP) was suggested and discussed, and then a survey was conducted in which more than 2/3 of voters agreed that GPP covers the most important aspects in PROMs. Conclusion. Despite the limitations of this exploratory study, it offered a new method to conduct clinical research with opportunity to engage patients. The general psycho-physical well-being score suggested as simple, applicable PROMs to be eventually combined procedure-specific, disease-specific, or symptom-specific PROMs if needed.

Significance of patient-reported outcomes for occupational resumption and quality of life after cardiac rehabilitation

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
A Salzwedel ◽  
I Koran ◽  
E Langheim ◽  
A Schlitt ◽  
J Nothroff ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Physical Performance ◽  
Well Being ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Funding Source ◽  
Patient Reported ◽  
Returning To Work

Abstract Introduction Comprehensive cardiac rehabilitation (CR) programs based on the bio-psycho-social approach of the international classification of functioning and disease are carried out to achieve improved prognosis, superior health-related quality of life (HRQL) and social integration. We aimed to identify predictors of returning to work (RTW) and HRQL among cardiovascular risk factors and physical performance as well as patient-reported outcome measures (PROMs) modifiable during CR. Methods We designed a prospective observational multi-center study and enrolled 1,586 patients (2017/18) in 12 German rehabilitation centers regardless of their primary allocation diagnoses (e.g. acute myocardial infarction (AMI), coronary artery bypass grafting (CABG), coronary artery disease (CAD), valvular disease). Besides general data (e.g. age, gender, diagnoses), parameters of risk factor management (e.g. smoking, lipid profile, hypertension, lifestyle change motivation), physical performance (e.g. maximum exercise capacity, endurance training load, 6-min walking distance), and PROMs (e.g. depression, heart-focused anxiety, HRQL, subjective well-being, somatic and mental health, pain, general self-efficacy, pension desire as well as self-assessment of occupational prognosis using several questionnaires) were documented at CR admission and discharge. 6 months after discharge, status of RTW and HRQL (SF-12) were captured by a follow-up (FU) survey and analyzed in multivariable regression models with multiple imputation of missing values. Results Out of the study participants, 1,262 patients (54±7 years, 77% men) responded to the follow-up survey and could be analyzed regarding the outcome parameters. Most of them were assigned to CR primarily due to AMI (40%) or CAD without myocardial infarction (18%), followed by heart valve diseases in 12% of patients and CABG (8%). 864 patients (69%) returned to work within the follow-up period. Pension desire, negative self-assessed occupational prognosis, heart-focussed anxiety, major life events, smoking and heart failure were negatively associated with RTW, while higher endurance training load, HRQL and work stress were positively associated (Figure 1). HRQL after 6 months was determined more by PROMs (e.g. pension desire, heart-focused anxiety, physical/mental HRQL in SF-12, physical/mental health in indicators of rehab-status questionnaire (IRES-24), stress, well-being in the World Health Organization well-being index and self-efficacy expectations) than by clinical parameters or physical performance. Conclusions Patient-reported outcome measures predominantly influenced RTW and HRQL in heart-disease patients, whereas patients' pension desire and heart-focussed anxiety had a dominant impact on all investigated endpoints. Therefore, the multi-component CR approach focussing on psychosocial support is crucial for subjective health prognosis and occupational resumption. Figure 1. Predictors of returning to work Funding Acknowledgement Type of funding source: Public grant(s) – National budget only. Main funding source(s): German pension insurance

scholarly journals Longitudinal study of symptom burden in outpatients with advanced cancers based on electronic Patient-Reported Outcome (ePRO) platform: a single institution, prospective study protocol

BMJ Open ◽  
2020 ◽  
Vol 10 (11) ◽  
pp. e038223
Author(s):  
Lili Tang ◽  
Ying Pang ◽  
Yi He ◽  
Qiuling Shi ◽  
Xinkun Han ◽  
...  
Keyword(s):  
Study Protocol ◽  
Symptom Management ◽  
Depression Scale ◽  
Symptom Burden ◽  
Patient Reported Outcome ◽  
Critical Parameters ◽  
Advanced Lung Cancer ◽  
Optimal Time ◽  
Patient Reported

IntroductionAn electronic Patient-Reported Outcome (ePRO) platform is needed for implementing evidence-based symptom management in outpatients with advanced cancer. We describe the overall protocol and the methodology for measuring symptom burden, to provide critical parameters needed to implement symptom management on the ePRO platform.Methods and analysisThe study focusses on patients with advanced lung cancer, stomach cancer, oesophagus cancer, liver cancer, colorectal cancer or breast cancer. The primary outcome is the change of symptom burden. MD Anderson Symptom Inventory, and other PRO instruments (Insomnia Severity Index, Hospital Anxiety and Depression Scale, 9-item Patient Health Questionnaire and EuroQol-5 dimensions-5 levels version) were used. The secondary outcomes include feasibility of using ePRO, symptom-related quality of life, reasons for no improvement of symptoms, defining frequency of PRO assessments and cut-points, items for screening and management of comorbidity and satisfaction with ePRO platform in patients and health providers. After initial outpatient visit for baseline assessment, ePRO system will automatically send follow-up notification seven times over 4 weeks to patients. The characteristics and changing trajectory of symptoms of patients will be described. Parameters for using PROs, such as optimal time points for follow-up and cut-off point for alert will be determined. The feasibility of ePRO platform to track the changes of target symptoms in outpatients will be evaluated.Ethics and disseminationThe study protocol and related documents were approved by the Institutional Research Board (IRB) of Peking University Cancer Hospital on 13 February 2019 (2019YJZ07). The results of this study will be disseminated through academic workshops, peer-reviewed publications and conferences.Trial registration numberChiCTR1900023560.

scholarly journals Early outcomes and predictors of patient satisfaction after TKA: a prospective study of 200 cases with a contemporary cemented rotating platform implant design

2021 ◽  
Vol 8 (1) ◽  
Author(s):  
Corné van Loon ◽  
Niels Baas ◽  
Verdonna Huey ◽  
James Lesko ◽  
Geert Meermans ◽  
...  
Keyword(s):  
Patient Satisfaction ◽  
Clinical Improvement ◽  
Patient Characteristics ◽  
Patient Reported Outcome ◽  
Implant Design ◽  
Rotating Platform ◽  
Radiographic Outcomes ◽  
Operative Outcomes ◽  
Patient Reported ◽  
Time Point

Abstract Purpose The purpose of the study was to identify the earliest time point where subjects realized the greatest clinical improvement after TKA, and the time when post-operative scores became superior to pre-operative scores. Post-hoc exploratory analyses were conducted to investigate predictors of early post-operative outcomes and patient satisfaction. Methods Six investigators across 4 sites in the Netherlands prospectively implanted 200 subjects with a contemporary cemented rotating platform device. Patient Reported Outcome Measurements (PROMs) KOOS-PS, PKIP, and EQ-5D were collected pre-operatively and post-operatively through 2-years. PROMs change from pre-operative baseline were summarized, along with radiographic outcomes and adverse events (AEs). Pre-operative patient characteristics were explored for correlation with patient outcomes, and patient satisfaction for correlation with KOOS-PS. Results Follow-up compliance was 99% at 6-months, and 95.5% at 2-years. The percentage with higher KOOS-PS compared to baseline was 81.3% at 6-months. KOOS-PS, PKIP, and PKIP subscore means were all better at 6-weeks versus baseline. Gender, BMI, hypertension, and pre-operative KOOS-PS were weakly correlated with 6-week KOOS-PS (multivariate R-squared = 14.1%), but only pre-operative KOOS-PS demonstrated correlation with post-operative KOOS-PS at 6-months or later (R-squared < 5% at 6-months and 2 years). Satisfaction was moderately correlated with concurrent KOOS-PS at each post-operative time point, with (R-squared = 35.3% at 6-months, and 37.5% at 2 years). Conclusion The greatest mean clinical improvement occurred within the first 6-weeks. Although some pre-operative factors were correlated with higher early post-operative KOOS-PS outcomes, these advantages disappeared by 6-months aside from weak correlation with pre-operative KOOS-PS. Post-operative KOOS-PS was moderately correlated with concurrent post-operative satisfaction. These results may be used for pre-operative counseling and management of patient’s postoperative expectations. Trial registration Clinicaltrials.gov, NCT02339610. Registered 15 January 2015.

Coverage of symptomatic toxicities from the common terminology criteria for adverse events (CTCAE) framework within the european organization for research and treatment of cancer (EORTC) item library.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e24117-e24117
Author(s):  
Claire Piccinin ◽  
Andrew Bottomley ◽  
Galina Velikova ◽  
Mogens Groenvold ◽  
Dagmara Kuliś ◽  
...  
Keyword(s):  
Well Being ◽  
Routine Care ◽  
Patient Reported Outcome ◽  
Satisfaction With Care ◽  
Emotional Impact ◽  
Coding System ◽  
European Organization ◽  
Additional Information ◽  
Care Services ◽  
Patient Reported

e24117 Background: The EORTC Item Library is an online, interactive platform comprised of 950 distinct questions (items) from 67 different EORTC patient-reported outcome (PRO) questionnaires, covering a range of symptomatic toxicities, types of functioning, and impact on quality of life. These PROs provide a patient-centred perspective, complementing clinician adverse event (AE) reporting using classifications like the CTCAE. In order to summarize the coverage of symptomatic toxicities and facilitate the identification of items through use of a standardized framework, a mapping study was carried out, aimed at creating a coding system to classify EORTC items according to the CTCAE and link them to corresponding AEs. Methods: All items were searched for within the CTCAE (v5.0) using a deductive approach. Items were coded as linked if they were described within an AE’s title, description, or grading. Items not suitable for CTCAE coding were inductively assigned a descriptive classification. Descriptive classifications were also applied along with CTCAE codes when they provided additional information. Symptoms described in EORTC items but not located in the CTCAE were coded as missing and additional codes were assigned to highlight EORTC items capturing multiple underlying issues and diagnosis only CTCAE codes. Two raters independently coded 249 items and agreement was calculated. The remaining 701 items were coded by one rater and verified by the second, with a third introduced to discuss any discrepancies until a consensus was reached. Results: Agreement for raters following independent coding was 77.9% for at least one AE per item. In total, 625 (65.8%) items were linked to 208 different AEs. Fatigue was the most commonly linked AE, representing 4.9% of linked AEs. The majority of linked items were associated with one (65.6%) or two (23.5%) AEs, with some linked to three or more (10.9%). Multiple linkage resulted from different symptoms relating to the same issue/diagnosis or one symptom relating to multiple diagnoses. Two symptoms captured by six EORTC items but not found in the CTCAE were identified: bowel urgency and tenesmus. Seven descriptive non-CTCAE classifications emerged, with most of these covering the emotional impact of symptom, diagnosis, or treatment (33.6%) and information/satisfaction with care (31.7%). Nineteen items (2%) were linked to multiple underlying issues, and 43 (4.5%) to diagnosis only CTCAE codes. Conclusions: The EORTC Item Library provides extensive coverage of CTCAE symptomatic toxicities, along with other issues that are important to cancer patients, including emotional well-being and satisfaction with care services. Classifying symptomatic PRO items following the CTCAE clinical framework may facilitate future PRO selection and use in clinical trials and routine care.

scholarly journals Web-Based Patient-Reported Outcome Measures for Personalized Treatment and Care (PROMPT-Care): Multicenter Pragmatic Nonrandomized Trial

10.2196/19685 ◽  
2020 ◽  
Vol 22 (10) ◽  
pp. e19685
Author(s):  
Afaf Girgis ◽  
Ivana Durcinoska ◽  
Anthony Arnold ◽  
Joseph Descallar ◽  
Nasreen Kaadan ◽  
...  
Keyword(s):  
Emergency Department ◽  
Patient Reported Outcomes ◽  
Well Being ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Self Management ◽  
Diverse Population ◽  
Web Based ◽  
Patients With Cancer ◽  
Patient Reported

Background Despite the acceptability and efficacy of e–patient-reported outcome (ePRO) systems, implementation in routine clinical care remains challenging. Objective This pragmatic trial implemented the PROMPT-Care (Patient Reported Outcome Measures for Personalized Treatment and Care) web-based system into existing clinical workflows and evaluated its effectiveness among a diverse population of patients with cancer. Methods Adult patients with solid tumors receiving active treatment or follow-up care in four cancer centers were enrolled. The PROMPT-Care intervention supported patient management through (1) monthly off-site electronic PRO physical symptom and psychosocial well-being assessments, (2) automated electronic clinical alerts notifying the care team of unresolved clinical issues following two consecutive assessments, and (3) tailored online patient self-management resources. Propensity score matching was used to match controls with intervention patients in a 4:1 ratio for patient age, sex, and treatment status. The primary outcome was a reduction in emergency department presentations. Secondary outcomes were time spent on chemotherapy and the number of allied health service referrals. Results From April 2016 to October 2018, 328 patients from four public hospitals received the intervention. Matched controls (n=1312) comprised the general population of patients with cancer, seen at the participating hospitals during the study period. Emergency department visits were significantly reduced by 33% (P=.02) among patients receiving the intervention compared with patients in the matched controls. No significant associations were found in allied health referrals or time to end of chemotherapy. At baseline, the most common patient reported outcomes (above-threshold) were fatigue (39%), tiredness (38.4%), worry (32.9%), general wellbeing (32.9%), and sleep (24.1%), aligning with the most frequently accessed self-management domain pages of physical well-being (36%) and emotional well-being (23%). The majority of clinical feedback reports were reviewed by nursing staff (729/893, 82%), largely in response to the automated clinical alerts (n=877). Conclusions Algorithm-supported web-based systems utilizing patient reported outcomes in clinical practice reduced emergency department presentations among a diverse population of patients with cancer. This study also highlighted the importance of (1) automated triggers for reviewing above-threshold results in patient reports, rather than passive manual review of patient records; (2) the instrumental role nurses play in managing alerts; and (3) providing patients with resources to support guided self-management, where appropriate. Together, these factors will inform the integration of web-based PRO systems into future models of routine cancer care. Trial Registration Australian New Zealand Clinical Trials Registry ACTRN12616000615482; https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=370633 International Registered Report Identifier (IRRID) RR2-10.1186/s12885-018-4729-3

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 P1062EXPANDED DIALYSIS (HDX): IS THERE AN IMPACT ON PATIENT REPORTED SYMPTOM?

2020 ◽  
Vol 35 (Supplement_3) ◽  
Author(s):  
Jarrin Penny ◽  
Fabio R Salerno ◽  
Lisa Hur ◽  
Christopher McIntyre
Keyword(s):  
Quality Of Life ◽  
Clinical Outcomes ◽  
Symptom Burden ◽  
Well Being ◽  
Response To Therapy ◽  
High Flux ◽  
Patient Reported ◽  
Dialysis Membranes ◽  
The Impact

Abstract Background and Aims High flux dialysis membranes sufficiently remove smaller sized uremic toxins however, the accumulation and retention of larger middle molecular weight toxins, which are associated with chronic inflammation, cardiovascular disease and suboptimal outcomes are poorly cleared. The recent advent of medium-cut-off dialysis membranes, labelled “expanded dialysis” (HDx) are permeable to molecules of larger size responsible for poor clinical outcomes. However, it remains unclear if HDx can directly impact the symptoms associated with hemodialysis (HD). Symptom burden plays a significant role in quality of life (QOL) and mortality rates in the HD population. The London Evaluation of Illness (LEVIL), an application-based platform has been developed to measure patient reported outcomes (PROM). In comparison to cross-sectional PROM’s, LEVIL more accurately represents the fluctuations in daily symptoms and the impact of intervention. LEVIL evaluates general well-being, energy, sleep, appetite, pain and breathing, all of which are outcomes of interest on symptom burden in chronic kidney disease. Our aim was to determine if HDx therapy had any effect on symtoms/QOL domains using LEVIL. Method 28 patients from two dialysis centers in London Ontario were consented to participate. Patients were required to be over 18 years of age and on conventional thrice weekly maintenance HD for at least three months. 23 participants completed study and analyzed (five lost for various reasons). Baseline (BL) symptom characteristics were obtained while using high flux membrane for two weeks. Symptoms continued to be measured throughout the 12 weeks of HDx therapy two-three times weekly using LEVIL. Laboratory biomarkers including beta-2 microglobulin and free-light chains were collected at baseline and after 12 weeks of HDx therapy. Results Patients were stratified into tertiles (high/middle/low) using mean values of BL symptoms scores in each domain (wellbeing, energy, sleep, appetite, pain, breathing). Those in the high BL group were labeled as “control”. Low and middle BL measures were further stratified into responders vs. non-responders (responders were considered to have a 50% increase in any symptom domain by ≥50%). Of those domains which responded to HDx, 76% also had low BL scores with 27% having middle BL scores. General wellbeing, energy and sleep were domains with the greatest response reaching statistical significance after eight weeks of therapy. HDx had limited effect on appetite, pain and breathing. Although stratification was per domain, overall, 74% of the population studied did respond in at least one domain, with some responding in as many as five. Conclusion HDx using Theranova (Baxter) shows the most benefit in domains with low BL measures. Additionally, not everyone who had low BL scores responded after 12 weeks of therapy, leaving us to question whether HDx may have a latent effect in some individuals/populations. Those who had no response to therapy in certain domains also had greater baseline quality of life respectively. This information may assist in decision making/rationale for the utilization and implementation of such therapy. Although more work is required to further stratify symptoms in relation to demographic/biochemical finding and clinical outcomes. It is evident that HDx improves patient reported symptoms and QOL.

scholarly journals Patient-reported outcome measures in systemic sclerosis–related interstitial lung disease for clinical practice and clinical trials

2020 ◽  
Vol 5 (2_suppl) ◽  
pp. 48-60
Author(s):  
Lesley Ann Saketkoo ◽  
Mary Beth Scholand ◽  
Matthew R. Lammi ◽  
Anne-Marie Russell
Keyword(s):  
Clinical Practice ◽  
Systemic Sclerosis ◽  
Interstitial Lung Disease ◽  
Lung Disease ◽  
Outcome Measures ◽  
Symptom Burden ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
Science Methodology

Systemic sclerosis (SSc) is a progressive vasculopathic, fibrosing autoimmune condition, portending significant mortality; wherein interstitial lung disease (ILD) is the leading cause of death. Although lacking a definitive cure, therapeutics for (SSc-ILD) that stave progression exist with further promising primary and adjuvant compounds in development, as well as interventions to reduce symptom burden and increase quality of life. To date, there has been a significant but varied history related to systemic sclerosis–related interstitial lung disease trial design and endpoint designation. This is especially true of endpoints measuring patient-reported perceptions of efficacy and tolerability. This article describes the underpinnings and complexity of the science, methodology, and current state of patient-reported outcome measures used in (SSc-ILD) systemic sclerosis–related interstitial lung disease in clinical practice and trials.

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