Impact Of Disease In Patients Research Articles

Construct validity of PROMIS pain interference, fatigue, and physical function as patient-reported outcomes in adults with idiopathic inflammatory myopathies: An international study from the OMERACT myositis working group

BackgroundValidated patient-reported outcome measures to assess disease impact in patients with adult idiopathic inflammatory myopathies (IIMs) are needed. The objective of this study was to assess the construct validity of PROMIS Pain Interference, Fatigue, and Physical Function measures in comparison with core disease activity measures. MethodsAdults with IIM, excluding inclusion body myositis, from OMERACT Myositis Working Group (MWG) clinic sites completed PROMIS Short Form v1.0—Pain Interference 6a, PROMIS Short Form v1.0—Fatigue 7a, and PROMIS Short Form v2.0—Physical Function 8b measures. Core disease activity measures including patient and physician global disease activity assessments, manual muscle testing, serum creatine kinase activity, and Health Assessment Questionnaire Disability Index (HAQ-DI) were simultaneously assessed. To evaluate construct validity, a priori hypotheses for the expected correlations between PROMIS measures, age, and core disease measures were determined by >70 % agreement among MWG members and were compared against observed Pearson's correlations. Internal consistency of items and floor or ceiling effects for the PROMIS measures were also assessed. Subgroup analysis according to IIM subtype (dermatomyositis vs. non-dermatomyositis IIM) was performed. Results135 adults with IIM from 5 countries across North America, Europe, Asia, and Australia were included. For construct validity, a priori hypotheses were confirmed for 5 of 6 (83 %) PROMIS Pain Interference, 4 of 5 (80 %) PROMIS Fatigue, and 3 of 4 (75 %) PROMIS Physical Function correlations. Internal consistency was high for each PROMIS measure (Cronbach's alpha >0.9). Ceiling effects were observed only for PROMIS Pain Interference, with low/no pain in 29 % of patients. Subgroup analysis between dermatomyositis (n = 65) and non-dermatomyositis (n = 70) subtypes demonstrated similar correlations between PROMIS measures and disease activity measures. ConclusionsPROMIS Short Form v1.0—Pain Interference 6a, PROMIS Short Form v1.0—Fatigue 7a, and PROMIS Short Form v2.0—Physical Function 8b measures demonstrate strong construct validity when compared to core disease activity measures in IIM, with consistent results across IIM subtypes. These findings support the use of these selected PROMIS measures to assess core domains of interest for measuring life impact in IIMs.

Effect of bimekizumab on patient-reported disease impact in patients with psoriatic arthritis: 1-year results from two phase 3 studies.

To evaluate 1-year bimekizumab efficacy in PsA from the patient perspective using the 12-item PsA Impact of Disease (PsAID-12) questionnaire. BE OPTIMAL (NCT03895203; biologic DMARD [bDMARD]-naïve), BE COMPLETE (NCT03896581; inadequate response/intolerance to TNF inhibitors [TNFi-IR]) and BE VITAL (NCT04009499; open-label extension) assessed bimekizumab 160 mg every 4 weeks in patients with PsA. Post hoc analyses of patient-reported disease impact, assessed by the PsAID-12 questionnaire, are reported to 1 year (collected to Week 40 in BE COMPLETE). Overall, 1,112 total patients were included (698 bimekizumab, 414 placebo). Rapid improvements observed with bimekizumab treatment at Week 4 continued to Week 16 and were sustained to 1 year. At 1 year, mean (SE) change from baseline in PsAID-12 total score was comparable between bimekizumab-randomized patients and patients who switched to bimekizumab at Week 16 (bDMARD-naïve bimekizumab -2.3 [0.1], placebo/bimekizumab -2.2 [0.1]; TNFi-IR bimekizumab -2.5 [0.1], placebo/bimekizumab -2.2 [0.2]). Proportions of bimekizumab-randomized patients achieving clinically meaningful within-patient improvement (≥3-point decrease from baseline) at Week 16 were sustained to 1 year (bDMARD-naïve 49.0%; TNFi-IR 48.5%) and were similar for placebo/bimekizumab patients (bDMARD-naïve 44.4%; TNFi-IR 40.6%). Across studies and arms, 35.3% to 47.8% of patients had minimal or no symptom impact at 1 year. Improvements were observed to 1 year across all single-item domains, including pain, fatigue and skin problems. Bimekizumab treatment resulted in rapid and sustained clinically meaningful improvements in disease impact up to 1 year in bDMARD-naïve and TNFi-IR patients with PsA. BE OPTIMAL: NCT03895203; BE COMPLETE: NCT03896581; BE VITAL: NCT04009499 (ClinicalTrials.gov).

The Long-Term Impact on Patients with MDR-TB Using the Saint George’s Respiratory Questionnaire

Abstract Background: Multidrug-resistant tuberculosis (MDR-TB) continues to be a global health catastrophe. This prospective study examines the subjective impact of the disease as experienced by patients using the Saint George’s Respiratory Questionnaire. Methodology: The study involved 244 patients diagnosed with drug-sensitive and drug-resistant pulmonary tuberculosis over a 10-year years. These patients were administered the Saint George’s Respiratory Questionnaire, which was evaluated in comparison between the two groups to determine the progressive impact of disease in patients with MDR-TB compared to patients with drug-sensitive tuberculosis (DS-TB). Results: The analysis showed that patients with MDR-TB had statistically significantly higher symptomatic activity and impact scores compared to patients with drug-sensitive tuberculosis. Conclusions: The results suggest that patients diagnosed with MDR-TB at different stages of disease progression need urgent attention from healthcare providers.

Effectiveness and feasibility of a mobile health self-management intervention in rheumatoid arthritis: study protocol for a pragmatic multicentre randomised controlled trial (AEGORA)

BackgroundRheumatoid arthritis (RA) considerably impacts patients’ lives. Patients’ confidence in their ability to manage this impact, or self-efficacy, can be supported with self-management interventions. One approach is to use mobile health (mHealth) applications, which can additionally provide insight into disease impact by remotely monitoring patient-reported outcomes. However, user engagement with mHealth-apps is variable, and concerns exist that remote monitoring might make patients overly attentive to symptoms.MethodsApp-based Education and GOal setting in RA (AEGORA) is a multicentre, pragmatic randomised controlled trial investigating an mHealth-based self-management intervention to improve self-efficacy and remotely monitor disease impact in patients with RA. The intervention is provided via an adapted version of the application Sidekick (Sidekick Health, Reykjavik, Iceland) and consists of education, goal setting, lifestyle advice, and remote assessment of the Rheumatoid Arthritis Impact of Disease (RAID) questionnaire.Across two centres, 120 patients will be recruited and randomised (2:1:1) to usual care or intervention group A/B (study app with weekly/monthly prompts to complete the RAID, respectively). Outcomes are assessed at baseline and after 4–6 months. The primary endpoint is a clinically important improvement (≥ 5.5/110) in the Arthritis Self-Efficacy Scale in the combined intervention group compared to usual care. Secondary endpoints are (a) non-inferiority regarding pain catastrophising, as a measure of symptom hypervigilance; (b) superiority regarding the RAID, sleep quality, and physical activity; and (c) participant engagement with the study app. Finally, the relationship between engagement, prompted frequency of RAID questionnaires, and the primary and secondary outcomes will be explored.DiscussionThe AEGORA trial aims to study the effectiveness of mHealth-based, multicomponent self-management support to improve self-efficacy in the context of RA, while providing potentially valuable insights into temporal disease activity dynamics and the feasibility and possible negative effects of remote symptom monitoring in this population.Trial registrationClinicaltrials.gov NCT05888181. Retrospectively registered on March 23, 2023. Study inclusion started on March 3, 2023.

A Novel Approach to Assess the Impact of Disease in Patients with SCN8A-Related Developmental and Epileptic Encephalopathy (P3-9.008)

<h3>Objective:</h3> Clinical data generated using Invitae’s Ciitizen patient-consented, real-world data platform were used to explore disease burden in <i>SCN8A</i>-DEE. <h3>Background:</h3> <i>SCN8A</i>-related DEE is a rare disease associated with pathogenic variants in the <i>SCN8A</i> gene encoding the voltage-gated sodium channel alpha subunit Na_v1.6. Due to significant phenotypic heterogeneity, comprehensive understanding of disease impact and progression is limited. <h3>Design/Methods:</h3> Real-world clinical data were extracted from patients with an <i>SCN8A</i> variant using the Ciitizen platform, which employs a proprietary approach combining machine learning and expert human review to streamline generation of research-grade data from unstructured health records spanning ~10 years. Extracted data including seizure history, comorbidities, and therapeutic interventions were analyzed. <h3>Results:</h3> 79 patients were enrolled and contributed data for analyses. Mean patient age was 8.2 years (1–23), with similar percentages of males and females. Patients were categorized based on age at seizure onset; ≤6 months (75.6%) or &gt;6 months (34.2%). A further group included patients without seizures (5%). Mean age at seizure onset was 70.4 days (1–175) for patients with early (≤6 months) seizures and 749.3 days (224–4409) for those with later (&gt;6 months) seizures. While seizure frequency was variable, most patients continued to experience seizures over time. Procedural interventions and hospitalizations were high across the lifespan; nearly a third occurring in the first year of life. Mean number of medications prescribed over a patient’s lifetime was 17.6 (3–44). Patients experienced multiple comorbidities; global developmental delay was the most common (81%), with sleep disorders, hypotonia, feeding difficulties and GERD reported in &gt;50%. <h3>Conclusions:</h3> This work represents a new generation of natural history study providing the most robust real-world dataset to date on disease burden and progression in <i>SCN8A</i>-DEE. Knowledge from this study is anticipated to accelerate drug development efforts by providing new insights into the broad, longitudinal impact of disease, therefore informing trial endpoints beyond seizure symptomatology. <b>Disclosure:</b> Kelley Dalby has received personal compensation for serving as an employee of Praxis Precision Medicines. Kelley Dalby has stock in Praxis Precision Medicines. Kelley Dalby has stock in RogCon. Mr. Snyder has received personal compensation for serving as an employee of Praxis Precision Medicines. Mr. Snyder has received stock or an ownership interest from Praxis Precision Medicines. Dr. G Matthews has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Praxis Precision Medicines. Dr. G Matthews has stock in Praxis Precision Medicines. Dr. Oldham has received personal compensation for serving as an employee of Praxis Precision Medicines. Steven Petrou has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Praxis Precision Medicines. Steven Petrou has stock in Praxis Precision Medicines. The institution of Steven Petrou has received research support from Praxis Precision Medicines. The institution of Steven Petrou has received research support from Medical Research Future Fund. Steven Petrou has received intellectual property interests from a discovery or technology relating to health care. Steven Petrou has received intellectual property interests from a discovery or technology relating to health care. Dr. Souza has received personal compensation for serving as an employee of Praxis Precision Medicines. Dr. Souza has stock in Praxis Precision Medicines.

Content Validity and Cognitive Debriefing of a Patient-Reported Outcome Instrument Evaluating Symptoms and Disease Impact in Patients with Geographic Atrophy.

Geographic atrophy (GA) occurs in the later stages of dry age-related macular degeneration (AMD) and impairs visual acuity, eventually causing permanent blindness in some patients and impacting patient quality of life. Patient-reported outcome (PRO) measures that assess the experience of patients with visual impairment do not sufficiently capture all concepts salient to patients with GA. In this study the experience of patients with GA secondary to dry AMD was evaluated, and items from the novel 10-item Visual Impairment Symptom Severity Assessment (VISSA-10) PRO instrument were mapped to salient symptoms to assess its content validity, ease of use, and relevance. Concept elicitation interviews were conducted with patients with GA to determine salient symptoms and impacts of GA, and a conceptual model was developed to reflect these. The items in the VISSA-10 instrument were then mapped onto the salient symptoms included in this conceptual model. Cognitive debriefing interviews were also conducted with the same cohort to determine the comprehensiveness and comprehensibility of the instrument, and to qualitatively assess levels of change considered meaningful by patients. In total, 25 symptoms and 36 impacts were reported by 19 patients with GA, with seven symptoms and 11 impacts identified as salient. Of these, 12 symptoms and 15 impacts reported were not included in a previously published conceptual model for patients with dry AMD. Overall, eight of the ten items from the VISSA-10 instrument mapped to salient symptoms reported by patients with GA. All patients reported that the instrument was clear and easy to understand. The VISSA-10 instrument was shown to be content valid, clear, and comprehensible, with sufficient concept coverage to measure the experience of patients with GA. Although further quantitative validation is required, this instrument has demonstrated potential for implementation in future clinical trials to evaluate the efficacy of new treatments for GA.

Long-term reductions in disease impact in patients with chronic migraine following preventive treatment with eptinezumab

BackgroundEptinezumab is an anti-calcitonin gene-related peptide humanized monoclonal antibody approved for the preventive treatment of migraine in adults. The PREVAIL study demonstrated a favorable safety profile with sustained reductions in overall migraine-related burden in patients with chronic migraine (CM). This post hoc analysis aimed to examine item-level changes in the Migraine Disability Assessment (MIDAS) questionnaire over 2 years in participants with CM on eptinezumab treatment.MethodsPREVAIL was an open-label, phase 3 trial that included 96 weeks of treatment where 128 adults received intravenous eptinezumab administered over 30 min every 12 weeks (wks) for up to 8 doses of 300 mg. MIDAS was administered at baseline, Wk12, and every 12wks thereafter. Two supplementary MIDAS items not included in the total score calculation assessed number of headache days in the past 3 months (MIDAS headache) and average headache pain severity (from 0 [none] to 10 [worst]). MIDAS total scores were summed from 5 items, each quantifying the number of days in the past 3 months with migraine-related disability. Items 1, 3, and 5 assessed absenteeism, namely how many days the patient missed work/school (Q1), household work (Q3), or family/social/leisure activities (Q5). Items 2 and 4 were measures of presenteeism, namely how many days the patient had reduced productivity in work/school (Q2) or household work (Q4).ResultsMean MIDAS headache days decreased from 47.4 (baseline) to 17.1 (Wk12) and 16.3 (Wk104). The average headache pain severity score (0‒10) decreased from a mean of 7.3 (baseline) to 5.5 (Wk12) to 4.5 (Wk104). Mean MIDAS scores measuring absenteeism (Q1, 3, 5) changed from 9.7 days at baseline to 3.2 days (Wk12) and to 3.9 days (Wk104). Mean MIDAS scores measuring presenteeism (Q2, 4) at Wk12 decreased from 14.2 days at baseline to 5.2 days (Wk12, 104). Patients categorized with very severe MIDAS disability had a mean total MIDAS score of 84.8, with an average reduction of 56.7 days (Wk12), which was maintained at 32 days at Wk104.ConclusionsLong-term treatment with eptinezumab in patients with CM suggested sustained reductions in MIDAS-quantified disability, consistent with the sustained reductions in headache frequency and pain severity.Trial registrationClinicalTrials.gov identifier: NCT02985398.

Prevalence and Prognostic Impact of Carotid Artery Disease in Patients Undergoing TAVI

Prevalence and Prognostic Impact of Carotid Artery Disease in Patients Undergoing TAVI

Confounders contributing to explain the association between sex and disease impact in patients with recent-onset psoriatic arthritis.

To evaluate the effect of potential confounders on the association between sex and disease impact in recent-onset psoriatic arthritis. We performed a multicentre observational prospective study (2-year follow-up, regular annual visits). The study population comprised patients aged ≥18 years who fulfilled the CASPAR criteria and less than 2 years since the onset of symptoms. The dataset was generated using data for each patient at the 3 visits (baseline, first year, and second year of follow-up) matched with the PsAID values at each of the 3 visits. Once variables associated with both PsAID ≥4 and sex were selected, those that led to a difference of >10% between the adjusted and crude estimations were identified as potential confounders in the association between sex and PsAID. Lastly, the final multivariate logistic regression model estimating the association between sex and PsAID was defined. The dataset contained 418 observations (158 at baseline, 135 at the first follow-up visit, and 125 at the second visit). The confounders identified in the multivariate model were HAQ, global pain, level of physical activity, and joint pattern at diagnosis. After adjustment for these variables, no statistically significant association was observed between female sex and PsAID ≥4. The association between female sex and greater disease impact could be explained by the influence of other variables, specifically higher HAQ score, greater intensity of pain, differences in the level of physical activity and in the joint pattern at diagnosis (lower frequency of the spondylitis pattern in women).

Impact of chronic obstructive pulmonary disease in patients undergoing coronary artery bypass graft surgery

Introduction. Cardiovascular disease is the leading cause of morbidity, disability and mortality in modern society. Coronary artery disease (CAD) and chronic obstructive pulmonary disease (COPD) are the most common comorbid pathology that worsens the quality of life and prospective prognosis of patients.Aim. The literature review is focused on postoperative outcomes of patients with COPD undergoing coronary artery bypass grafting surgery (CABG).Results. The review presents national and international data the prevalence of COPD in patients having CABG. The issues of risk stratification of comorbid patients in cardiac surgery are considered. It is shown that patients with CAD in combination with COPD are more at risk of various complications after CABG. COPD increases the chances of a complicated course of the in-hospital period by 2.1 times and risk of death in the long-term period after CAPG by 1.8 times. Patients with COPD are at a higher risk of developing postoperative pneumonia, respiratory failure, stroke, kidney failure, and wound infection of the sternum. Long-term 5-year and 10-year survival after CABG is lower in patients with COPD. The longterm adverse prognosis depends on the severity of the bronchial obstruction. Achievements in the field of cardiac surgery have had a significant impact on the results of surgical interventions in the comorbid course of CAD and COPD. Intensive pulmonary rehabilitation after surgery and effective drug therapy can improve the outcomes after CABG in patients with COPD. Hybrid coronary revascularization and minimally invasive coronary surgery appear to be viable alternatives to conventional CABG, offering a less invasive approach to coronary revascularization, which may be especially beneficial to high-risk patients with COPD.

Interrelations between mental health, generic and thyroid-related quality of life in patients with Hashimoto’s thyroiditis receiving levothyroxine replacement

The assessment of health-related quality of life (QoL) in chronic conditions is considered as relevant, especially in patients with hypothyroidism where a high comorbidity with depressive and anxiety issues is reported. The present cross-sectional pilot study aims at: i) examining the correlation of thyroid-specific and generic measures of QoL in patients with Hashimoto’s Thyroiditis (HT); ii) investigating the associations of generic and thyroid-specific measures of QoL with depression and anxiety in patients with HT; iii) comparing generic and thyroid-specific measures of QoL in patients with HT versus controls. Twenty-one patients with serologically and/or ultrasonographically verified HT and sixteen controls with non-toxic goiter or post-surgical hypothyroidism were recruited for this study. Generic and disease-specific QoL were assessed by Health Survey Short Form-36 (SF-36) and Thyroid Patient Reported Outcome (ThyPRO) questionnaires, respectively, whereas depression and anxiety were measured through Beck Depression Inventory-second edition (BDI-II) and Hamilton Anxiety Rating Scale (HAM-A) questionnaires, respectively. Findings showed that ThyPRO and SF-36 scores were associated with each other only regarding the mental health domain, and that such QoL measures were consistently associated with depression levels but not with anxiety. Besides, no statistically significant difference was found between patients with HT and controls with regard to generic and thyroid-specific QoL. The present study suggests that generic and disease-specific measures of QoL can sufficiently assess the mental functioning domain and capture depressive symptoms, but only thyroid-specific measures (such as ThyPRO) can provide an accurate examination of physical aspects of QoL and the overall disease impact in patients’ lives. Besides, some methodological limitations should be taken into account in both using generic and disease-specific instruments with regard to their tendency to underestimate possible anxiety problems.

Clinical impact of advanced chronic kidney disease in patients with non-HIV pulmonary cryptococcosis

BackgroundPulmonary cryptococcosis is an uncommon infectious disease that can develop in both immunocompromised and immunocompetent patients. The severity of chronic kidney disease (CKD) was reported to be one of the risk factors for pulmonary cryptococcosis, but its clinical characteristics have not been fully assessed. The purpose of this study was to clarify the clinical characteristics of advanced CKD in patients with pulmonary cryptococcosis.MethodsThe present study retrospectively investigated 56 patients who had non-human immunodeficiency virus (HIV) pulmonary cryptococcosis and were treated at Saga University Hospital between 2005 and 2018. The clinical characteristics were evaluated and compared between patients with estimated glomerular filtration rate (eGFR) > 45 mL/min/1.73 m2 (n = 42, early CKD) and those with eGFR < 45 mL/min/1.73 m2 (n = 14, advanced CKD.ResultsCompared with patients with early CKD, those with advanced CKD had significantly higher rate of disseminated cryptococcosis (21.4% vs. 2.4%, p = 0.03); lower percentage of patients who recovered after treatment (63.6% vs. 92.5%, p = 0.02); and more frequent clinical features of fever (57.1% vs. 19.0%, p < 0.01), pleural effusion (21.4% vs. 2.4%, p = 0.03), high white blood cell count (8550/mL vs. 6150/mL, p = 0.01) and C-reactive protein (CRP) (2.1 mg/dL vs. 0.2 mg/dL, p = 0.02), and low level of serum albumin (3.0 g/dL vs. 3.8 g/dL, p < 0.01). Multivariate analysis adjusted by immunosuppressive drug use indicated the significant factors of fever (odds ratio or β value [95% confidence interval] 6.4 [1.65–20.09], p < 0.01), high white blood cell count (1293.2 [110.2–2476.2], p = 0.03), C-reactive protein (0.89 [0.18–1.59], p = 0.01) and low level of serum albumin (− 0.34 [− 0.54 – − 0.14], p < 0.01) in patients with eGFR < 45 mL/min/1.73m2.ConclusionAdvanced CKD was associated with poor clinical characteristics and outcomes in patients with non-HIV pulmonary cryptococcosis.Trial registrationThe patients in this study were registered retrospectively.

Disease features associated with a low disease impact in patients with psoriatic arthritis: results of a cross-sectional multicenter study

BackgroundPatient-reported outcomes measures, such as those provided by the Psoriatic Arthritis Impact of Disease (PsAID) questionnaire, have been found to be a reliable indicator of change during treatment, predictive of long-term outcomes, and the impact of psoriatic arthritis (PsA) on patients’ lives. The objective of the study was to describe the demographic and clinical characteristics of PsA patients with a low disease impact and to analyze predictive factors for that state.MethodsPost hoc analysis of a cross-sectional multicenter study that included 223 consecutive patients. PsAID questionnaire was used to estimate disease impact. Patients with a PsAID < 4 were considered in low disease impact. Minimal disease activity (MDA) response and the Health Assessment Questionnaire (HAQ) were also assessed. The degree of agreement between the different outcomes was addressed by Cohen’s kappa index.ResultsOne hundred and twenty-two (54.7%) patients reached a PsAID < 4. Among them, 52.0% and 68.0% presented articular or skin remission, respectively. Almost 75% of patients were in MDA state and 85.2% presented a low disability state according to the HAQ. A moderate concordance between HAQ ≤ 0.5 and PsAID < 4 (k = 0.53), fair between MDA and PsAID < 4 (k = 0.36), and moderate between DAPSA remission and PsAID < 4 (k = 0.46) was observed. Multivariate logistic regression analysis showed that patients with distal interphalangeal joint (DIP) disease (OR 0.40, 95%CI, 0.20–0.79, p = 0.009), family history of PsA (OR 0.25, 95%CI, 0.09–0.72, p = 0.010), and higher C-reactive protein (OR 0.92, 95%CI, 0.85–0.99, p = 0.036) were significantly less likely to reach a PsAID < 4.ConclusionsThere is certain discrepancy between disease activity measures and a low impact of disease in PsA. Clinical features (DIP joint involvement), biologic activity, and genetic factors (familial history) seem to be associated with lower odds of reaching a low disease impact.

P759 Incidence and clinical impact of perianal disease in patients with ulcerative colitis: a nationwide population-based study

P759 Incidence and clinical impact of perianal disease in patients with ulcerative colitis: a nationwide population-based study

Experience of Symptoms and Disease Impact in Patients with Adenomyosis.

BackgroundAdenomyosis is a poorly understood, benign disease of the uterus.ObjectiveIn this study, patient interviews were conducted to characterize the symptoms and impact of adenomyosis.MethodsThis was a cross-sectional study in which women with adenomyosis were recruited from five US clinics and a health-related social network forum. Participants (aged 18–55 years) were pre-menopausal with a history of regular menstrual cycles. Participants were interviewed about their experiences with adenomyosis, symptoms and impacts on day-to-day activities (concept elicitation), and subsequently about the occurrence, relative severity, and impact of symptoms (card-sorting exercise).ResultsIn total, 31 women were interviewed. Mean duration since onset of first adenomyosis symptom was 5.7 years; 41.9% reported severe/very severe adenomyosis. Over 50 symptoms and 30 impacts of adenomyosis were reported in the concept elicitation; 87% of symptoms were reported after 7 interviews and 78% of impacts after 5 interviews, indicating a condition with a significant symptom burden and a consistent presentation. The most common symptoms were heavy menstrual bleeding (87%), cramps (84%), and blood clots during menstrual bleeding (84%). The most common impacts were burdensome self-care hygiene (71%), and fatigue/low energy (71%). In the card-sorting exercise, the most commonly endorsed symptoms were pain during menstruation/menstrual cramps and heavy menstrual bleeding (both frequently rated as severe). The symptom with the highest impact was heavy menstrual bleeding.ConclusionInitiatives to understand women’s experiences with adenomyosis may improve management of the condition. This study provides a first step in understanding their experience and new information on the symptom profile of adenomyosis.Electronic supplementary materialThe online version of this article (10.1007/s40271-017-0284-2) contains supplementary material, which is available to authorized users.

TCT-180 Impact of Chronic Obstructive Pulmonary Disease in Patients with Left Main Disease Randomized to PCI vs. CABG: A Propensity Score Matched Analysis From the EXCEL Trial

In the EXCEL trial, PCI with everolimus-eluting stents was non-inferior to CABG at 3 years for treatment of left main coronary artery disease (LMCAD) with low or intermediate SYNTAX scores. We sought to assess whether chronic obstructive pulmonary disease (COPD), an independent risk factor for worse

Effects of pulmonary rehabilitation on exercise capacity and disease impact in patients with chronic obstructive pulmonary disease and obesity

ObjectivesTo explore the influence of obesity on outcomes of exercise capacity and disease impact in patients with chronic obstructive pulmonary disease (COPD) in response to pulmonary rehabilitation (PR) and to compare outcomes to those of normal weight and overweight counterparts. DesignSecondary data analysis of clinical database. SettingSt. James’s Hospital, Dublin, Ireland. Participants155 participants with a primary diagnosis of COPD who completed a PR programme between 2012 and 2014. Main Outcome MeasuresExercise capacity evaluated using the Six Minute Walk Test (6MWT) and the COPD Assessment Test (CAT) evaluated disease impact. ResultsWalking distance in the 6MWT improved significantly [mean difference of 55m (95% CI: 42 to 68; p<0.001)] and similarly [F(2, 92)=1.434, p=0.24] across all BMI categories, although the level of improvement reached clinical significance in the normal/underweight and overweight categories only. Disease impact on the CAT score improved across all body mass index (BMI) classifications by 2.3 points (95% CI: 0.9 to 3.6; p<0.050) which reached clinical significance and did not vary across BMI categories [F(2, 80)=0.534, p=0.58]. ConclusionExercise capacity and self-report disease impact of individuals with COPD improved similarly in response to PR irrespective of BMI.

Cysteamine (Cystagon®) adherence in patients with cystinosis in Spain: successful in children and a challenge in adolescents and adults.

BackgroundCysteamine has improved survival and prognosis in cystinosis. Increasing numbers of patients reach adulthood and face new challenges such as compliance that wanes over time. The aim of this study was to evaluate adherence to cysteamine treatment in a group of cystinotic patients in Spain in an attempt to identify potential therapy pitfalls and improve the overall care of affected individuals. Despite the impact of cysteamine on prognosis, there is a paucity of data regarding adherence.MethodThirty-four cystinotic patients (21 male) 38% ≥18 years were enrolled in a voluntary, anonymous survey. Replies were obtained from patients (15/34), mothers (11/34), fathers (4/34) and both parents (4/34).ResultsPatient age (median and interquartile range) at diagnosis was 1 year (0.57–1), and patient age at Cystagon® initiation was also 1 year (0.8–1.8). Sixteen (47%) were kidney transplant (KTx) recipients; six were retransplanted. Age at first KTx 10 years (8.7–13.7). Patient understanding of multiorgan involvement in cystinosis: 4.1 organs reported; eye 97% and kidney 91%. Cysteamine was given by mother (100%) and father (83%) in <11 year olds, or self-administered (94%) in ≥11 year olds. Four daily doses in 89% versus 56% in <11 year olds or ≥11 year olds, with fixed schedule in 94% versus 50% in <11 or ≥11 year olds and progressive loss of reminders over time. Furthermore, 44% complained of unpleasant smell. Motivation for treatment compliance was 100% versus 40% in <11 versus ≥11 year olds, respectively. Disease impact in patients <18 years is as follows: school (29%), social (14%), ‘feeling different’ (10%); in patients ≥18 years: ‘feeling different’ (62%), professional (39%) and job absenteeism (31%). Referring physician: paediatric nephrologist (94%) and nephrologist (63%) in <11 versus ≥11 year olds. Ophthalmological follow-up: 83% versus 38% in <11 versus ≥11 year olds. Patient opinion of physician expertise: paediatric nephrologist (94%) and nephrologist (44%). New treatment options (65%) and better information (42%) were demanded to improve adherence.ConclusionTreatment with Cystagon is effective in young patients. However, adherence diminishes over time in adolescents and adults despite disease impact. Strategies such as better information on the disease, patient self-care promotion and facilitated transition to adult healthcare services are required to improve compliance and the clinical management of cystinosis.

Detecting clinically-relevant changes in progressive multiple sclerosis

Objective: To investigate which changes in different clinical outcome measures contribute most to increased disease impact, as reported by the patient, in progressive multiple sclerosis (MS). Methods: From a cohort of prospectively-followed MS patients, we selected progressive patients with two visits, 4–6 years apart. We assessed long-term changes on the Expanded Disability Status Scale (EDSS), Timed 25-Foot Walk (T25FW), 9-Hole Peg Test (9HPT) and Guy’s Neurological Disability Scale (GNDS). We defined the presence or absence of clinically meaningful change by using the Multiple Sclerosis Impact Scale (MSIS-29) as an anchor measure. We also studied change on recently identified sub-scales of GNDS. Results: Change on GNDS (especially the spinal-plus subscale) contributed most to increased disease impact. Also change on the T25FW contributed largely. Specific profiles of change in T25FW and MSIS seemed to exist (generally, a lower increase in disease impact in patients with longer disease duration and higher baseline impact/disability). In some patients a dissociation existed between increased impact, according to the MSIS-29, and objective physical worsening of the T25FW. Conclusion: These results support using GNDS (particularly the spinal-plus domain) and T25FW in outcome measurement in progressive MS. We suggest there is a relation between baseline clinical characteristics and an increased impact at follow-up. This may have implications for patient selection in trials for progressive MS.

Usefulness of the CAT, LCOPD, EQ-5D and COPDSS Scales in Understanding the Impact of Lung Disease in Patients with Alpha-1 Antitrypsin Deficiency

Alpha-1-antitrypsin deficiency (AATD) is an inherited disorder responsible for early onset emphysema associated with a significant impairment of health-related quality of life (HRQoL). Our aim was to assess the usefulness of different instruments to evaluate the HRQoL in patients with AATD compared to non-AATD COPD. Observational, cross-sectional study in which all patients filled out a series of questionnaires: the COPD severity score (COPDSS), the EuroQoL 5-Dimensions (EQ-5D), the Living with COPD (LCOPD) and the COPD Assessment Test (CAT).A total of 96 patients were included, 35 with AATD (mean age 56.5 yrs, 57.1% male and mean FEV1(%) 48.7% and 61 non-AATD COPD (70.3 yrs, 80.3% men and FEV1(%) 47%. The questionnaire scores were similar, with a tendency towards worse scores in AATD for the EQ-5D (VAS) (64.8 (20.2) vs. 71.6 (17.1); p = 0.08). The correlations between the different scores and FEV1(%) were significant in both groups for COPDSS and LCOPD, but not for CAT and EQ-5D. In general, the correlations of scores with FEV1(%) were stronger for AATD compared with non-AATD COPD patients: COPDSS r = −0.570, p < 0.01 for AATD and r = −0.260, p < 0.05 for COPD; LCOPD r = −0.502, p < 0.001 for AATD and r = −0.304, p < 0.05 for non-AATD COPD.Patients with AATD have a similar degree of HRQoL impairment as older subjects with non-AATD COPD and showed a stronger correlation between HRQoL measurements and lung function impairment compared with non-AATD COPD. This may be related to the characteristics of the disease in these patients who are usually younger, with less co-morbidity and lower smoking consumption.