Patient-reported Outcomes In Adults Research Articles

Patient-reported outcomes in Primary Spinal Intradural Tumours: a systematic review.

Systematic review. Primary Spinal Intradural Tumours (PSITs) are rare pathologies that can significantly impact quality of life. This study aimed to review patient reported outcomes (PROs) in PSITs. A systematic search of Pubmed and Embase was performed to identify studies measuring PROs in adults with PSITs. PRO results were categorised as relating to Global, Physical, Social, or Mental health. Outcomes were summarised descriptively. Following review of 2382 records, 11 studies were eligible for inclusion (737 patients). All studies assessed surgically treated patients. Schwannoma was the commonest pathology (n = 190). 7 studies measured PROs before and after surgery, the remainder assessed only post-operatively. For eight studies, PROs were obtained within 12 months of treatment. 21 PRO measurement tools were used across included studies, of which Euro-Qol-5D (n = 8) and the pain visual/numerical analogue scale (n = 5) were utilised most frequently. Although overall QoL is lower than healthy controls in PSITs, improvements following surgery were found in Extramedullary tumours (EMT) in overall physical, social, and mental health. Similar improvements were not significant across studies of Intramedullary tumours (IMT). Overall QoL and symptom burden was higher in IMT patients than in brain tumour patients. No studies evaluated the effect of chemotherapy or radiotherapy. Patients with PSITs suffer impaired PROs before and after surgery. This is particularly true for IMTs. PRO reporting in PSITs is hindered by a heterogeneity of reporting and varied measurement tools. This calls for the establishment of a standard set of PROs as well as the use of registries.

Dupilumab Improves Disease Control and Patient-Reported Outcomes in Adults with Atopic Dermatitis in Clinical Practice: Subgroup Analysis of Black/African American Population from RELIEVE-AD

Dupilumab Improves Disease Control and Patient-Reported Outcomes in Adults with Atopic Dermatitis in Clinical Practice: Subgroup Analysis of Black/African American Population from RELIEVE-AD

Anxiety and Depression in Adults With Congenital Heart Disease

BackgroundA comprehensive understanding of adult congenital heart disease outcomes must include psychological functioning. Our multisite study offered the opportunity to explore depression and anxiety symptoms within a global sample. ObjectivesIn this substudy of the APPROACH-IS (Assessment of Patterns of Patient-Reported Outcomes in Adults With Congenital Heart Disease–International Study), the authors we investigated the prevalence of elevated depression and anxiety symptoms, explored associated sociodemographic and medical factors, and examined how quality of life (QOL) and health status (HS) differ according to the degree of psychological symptoms. MethodsParticipants completed the Hospital Anxiety and Depression Scale, which includes subscales for symptoms of anxiety (HADS-A) and depression (HADS-D). Subscale scores of 8 or higher indicate clinically elevated symptoms and can be further categorized as mild, moderate, or severe. Participants also completed analogue scales on a scale of 0 to 100 for QOL and HS. Analysis of variance was performed to investigate whether QOL and HS differed by symptom category. ResultsOf 3,815 participants from 15 countries (age 34.8 ± 12.9 years; 52.7% female), 1,148 (30.1%) had elevated symptoms in one or both subscales: elevated HADS-A only (18.3%), elevated HADS-D only (2.9%), or elevations on both subscales (8.9%). Percentages varied among countries. Both QOL and HS decreased in accordance with increasing HADS-A and HADS-D symptom categories (P < 0.001). ConclusionsIn this global sample of adults with congenital heart disease, almost one-third reported elevated symptoms of depression and/or anxiety, which in turn were associated with lower QOL and HS. We strongly advocate for the implementation of strategies to recognize and manage psychological distress in clinical settings. (Patient-Reported Outcomes in Adults With Congenital Heart Disease [APPROACH-IS]; NCT02150603)

Association between glycemic control and patient-reported outcomes in adults with type 1 diabetes in Japan: the SAGE study subanalysis.

Psychosocial aspects and the quality of life (QOL) of individuals with diabetes are important for achieving glycemic control and treatment goals. Here, we describe patient-reported outcomes (PROs) of Japanese adults with type 1 diabetes (T1D) and evaluate the association thereof with glycemic control. This subanalysis of a subgroup of 528 Japanese participants in the SAGE study of adults with T1D used data on glycosylated hemoglobin (HbA1c) and PRO scores [Hypoglycemia Fear Survey-II (HFS-II), Problem Areas In Diabetes (PAID), Insulin Treatment Satisfaction Questionnaire (ITSQ), and Audit of Diabetes-Dependent QOL (ADDQoL)] and summarized the score by the predefined age groups (26-44-years: n = 208, 45-64-years: n = 217, and ≥ 65-years: n = 103). The association between PROs, achieving HbA1c < 7.0%, and individualized targets was explored using multivariate logistic regression analysis. The HFS-II and PAID scores were lower, and the ITSQ score was higher in the ≥ 65-years group than in the younger groups with a linear trend of better scores with increasing age (P for trend < 0.05). ADDQoL scores were similar across the age groups, and present QOL (ADDQoL subscale) tended to improve with age (P for trend < 0.05). Achieving HbA1c < 7.0% and individualized targets were associated with satisfaction with insulin treatment regarding glycemic control. In Japanese adults with T1D, the impact on psychosocial aspects and QOL varied across age groups, with a trend of improving scores with age, potentially in relation to the less stringent glycemic control targets adopted in older individuals. Glycemic control was significantly associated with treatment satisfaction. The online version contains supplementary material available at 10.1007/s13340-023-00668-4.

Burosumab Improves Patient-Reported Outcomes in Adults With Tumor-Induced Osteomalacia: Mixed-Methods Analysis.

Tumor-induced osteomalacia (TIO) is an ultra-rare disease caused by tumors that secrete fibroblast growth factor 23, leading to chronic hypophosphatemia, poor skeletal health, and impaired physical function. In a phase 2 trial (UX023T-CL201; NCT02304367; n = 14), 48 weeks of burosumab treatment restored phosphate homeostasis, with improvements in skeletal health, functional mobility, and patient-reported pain, fatigue, and health-related quality of life (HRQL) (SF-36 v2). Here, we report an exploratory mixed-methods analysis of change from baseline after 144 weeks of burosumab treatment alongside qualitative data from exit interviews with 8 of 14 trial participants to evaluate meaningful treatment effects from a patient perspective. The interview subset (n = 8) reported pain and fatigue and compromised HRQL at baseline. In the interviews, participants reported that compromised HRQL and pain were the most important aspects of the disease to treat; both were considered more bothersome than fatigue and compromised physical function and activities of daily living. Improvements in pain and fatigue after treatment were reported, some of which achieved statistically and/or clinically meaningful thresholds. Furthermore, improvements in SF-36 v2 scores were most pronounced in the Physical Component Score and its Physical Function and Bodily Pain domains. Overall, the interview subset provided descriptions of symptomatic improvement and its clinical meaningfulness, including physical function, participation in activities of daily living, and mental well-being. Thus, this exploratory mixed-methods analysis provides deeper understanding of patients' perception of clinical meaningfulness beyond that articulated in validated patient-reported outcome instruments. © 2023 The Authors. Journal of Bone and Mineral Research published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research (ASBMR).

752-P: Improved BMI Category Is Associated with Better Patient-Reported Outcomes (PROs) in Adults with T2D Treated with Tirzepatide

Treatment with tirzepatide, a first-in-class GIP/GLP-1 receptor agonist, resulted in statistically and meaningfully reduced HbA1c, and substantially reduced weight, in adults with T2D in the Phase 3 SURPASS studies. We explored the association between shift in BMI category and PROs in adults treated with tirzepatide 5, 10, or 15mg in SURPASS-1 to -5. Data from all SURPASS-1 to -5 tirzepatide-treated participants with non-missing BMI were pooled, regardless of tirzepatide dose assigned. Participants with ‘improved’ (≥1 category lower vs. baseline), ‘stable’, or ‘worsened’ (≥1 category higher vs. baseline) BMI category at endpoint were assessed using weight-related PRO instruments at baseline and endpoint - Impact of Weight on Self-Perceptions (IW-SP), Impact of Weight on Quality of Life-Lite-Clinical Trials (IWQOL-Lite-CT), and Ability to Perform Physical Activities of Daily Living (APPADL). All PRO scores were greater at endpoint than at baseline for tirzepatide-treated participants with ‘improved’ or ‘stable’ BMI category (Table). Changes were numerically greater across all PROs for participants with ‘improved’ BMI category compared with those whose BMI category remained ‘stable’ or ‘worsened’. Improvements in PRO scores were greater in tirzepatide-treated adults with T2D with improved BMI category than in those with stable or worsened BMI category. Disclosure C.Lee: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. K.Boye: Employee; Eli Lilly and Company. V.Thieu: None. S.Allen: None. W.Dong: Employee; TechData Service Company, Other Relationship; Eli Lilly and Company, AstraZeneca. H.Sapin: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. Funding Eli Lilly and Company

0871 Outcomes in Overlap Syndrome of COPD and OSA: A Secondary Data Analysis of STAGES

Abstract Introduction Chronic obstructive pulmonary disease (COPD) and obstructive sleep apnea (OSA) are highly prevalent, and termed Overlap Syndrome (OVS) when coexistent. Patient-reported outcomes (PROs) are impaired in both COPD and OSA alone, but their combined impact is unclear. We examined differences in the impact of COPD on PROs in adults with and without OSA. Methods Analyses included adults aged≥8 years with polysomnographic (PSG) data from the Stanford Technology Analytics and Genomics in Sleep (STAGES) study. We created sets of individuals with and without COPD, matched up to 1:5 on age±5 years, BMI±2.5 kg/m2, AHI±5 events/hour, and sex. Among all matched sets, we estimated the impact of COPD on fatigue (Fatigue Severity Scale), sleepiness (Epworth Sleepiness Scale), anxiety (Generalized Anxiety Disorder Questionnaire), depression (Personal Health Questionnaire-9), insomnia (Insomnia Severity Index), and function (Functional Outcomes of Sleep Questionnaire) using linear mixed effects models. We also examined whether the effect of COPD differed between those with (AHI≥10 events/hour) and without (AHI&amp;lt; 10 events/hour) OSA. Results Overall, 56 patients with COPD were matched to a total of 199 non-COPD controls (51.0% males). The sample was middle aged (55.8±11.8 years), overweight/obese (BMI 29.8±6.9 kg/m2), and had mild/moderate OSA (AHI 11.0±11.5 events/hour) on average; 26 (46.4%) of COPD cases had an AHI≥10. Among all patients, COPD was associated with worse PROs on average (p&amp;lt; 0.05 for all but ESS [p=0.070]). Among those with AHI&amp;lt; 10, there were no differences for any PRO between groups with and without COPD. However, COPD was associated with worse values of all PROs among those with OSA, including fatigue (mean [95% CI] difference = 9.8 [2.9, 16.7], p=0.006), sleepiness (2.8 [0.01, 5.7], p=0.050), anxiety (3.6 [1.1, 6.0], p=0.005), depression (4.4 [1.8, 7.0], p=0.001), insomnia (4.8 [2.0, 7.6], p=0.001) and function (-3.3 [-5.2, -1.5], p=0.001). Conclusion The effect of COPD on PROs is modified by OSA, with worse outcomes among those with OVS compared to OSA alone, but no impact of COPD among those without OSA. Identification of adults with OVS through early screening is crucial to prompt treatment of disease and reduce symptom burden. Support (if any) T32HL07713 P01 HL094307

Sleep optimization to improve glycemic control in adults with type 1 diabetes: study protocol for a randomized controlled parallel intervention trial

BackgroundDespite improvements in treatment regimens and technology, less than 20% of adults with type 1 diabetes (T1D) achieve glycemic targets. Sleep is increasingly recognized as a potentially modifiable target for improving glycemic control. Diabetes distress, poor self-management behaviors, and reduced quality of life have also been linked to sleep variability and insufficient sleep duration. A significant gap of knowledge exists regarding interventions to improve sleep and the effects of sleep optimization on glycemic control in T1D. The purpose of this study is to determine the efficacy of a T1D-specific sleep optimization intervention (Sleep-Opt) on the primary outcomes of sleep variability, sleep duration, and glycemic control (A1C); other glycemic parameters (glycemic variability, time-in-range [TIR]); diabetes distress; self-management behaviors; quality of life; and other patient-reported outcomes in adults with T1D and habitual increased sleep variability or short sleep duration.MethodsA randomized controlled parallel-arm study will be employed in 120 adults (aged 18 to 65 years) with T1D. Participants will be screened for habitual sleep variability (> 1 h/week) or insufficient sleep duration (< 6.5 h per night). Eligible subjects will be randomized to the Sleep-Opt intervention group or healthy living attention control group for 12 weeks. A 1-week run-in period is planned, with baseline measures of sleep by actigraphy (sleep variability and duration), glycemia (A1C and related glycemic measures: glycemic variability and TIR using continuous glucose monitoring), and other secondary outcomes: diabetes distress, self-management behaviors, quality of life, and additional patient-reported outcomes. Sleep-Opt is a technology-assisted behavioral sleep intervention that we recently developed that leverages the rapidly increasing public interest in sleep tracking. Our behavioral intervention employs four elements: a wearable sleep tracker, didactic content, an interactive smartphone application, and brief telephone counseling. The attention control group will participate in a healthy living information program. Baseline measures will be repeated at midpoint, program completion, and post-program (weeks 6, 12, and 24, respectively) to determine differences between the two groups and sustainability of the intervention.DiscussionA better understanding of strategies to improve sleep in persons with T1D has the potential to be an important component of diabetes.Trial registrationClinical Trial Registration: NCT04506151.

Rationale, design and methodology of APPROACH-IS II: International study of patient-reported outcomes and frailty phenotyping in adults with congenital heart disease

BackgroundIn recent years, patient-reported outcomes (PROs) have received increasing prominence in cardiovascular research and clinical care. An understanding of the variability and global experience of PROs in adults with congenital heart disease (CHD), however, is still lacking. Moreover, information on epidemiological characteristics and the frailty phenotype of older adults with CHD is minimal. The APPROACH-IS II study was established to address these knowledge gaps. This paper presents the design and methodology of APPROACH-IS II. Methods/designAPPROACH-IS II is a cross-sectional global multicentric study that includes Part 1 (assessing PROs) and Part 2 (investigating the frailty phenotype of older adults). With 53 participating centers, located in 32 countries across six continents, the aim is to enroll 8000 patients with CHD. In Part 1, self-report surveys are used to collect data on PROs (e.g., quality of life, perceived health, depressive symptoms, autonomy support), and explanatory variables (e.g., social support, stigma, illness identity, empowerment). In Part 2, the cognitive functioning and frailty phenotype of older adults are measured using validated assessments. DiscussionAPPROACH-IS II will generate a rich dataset representing the international experience of individuals in adult CHD care. The results of this project will provide a global view of PROs and the frailty phenotype of adults with CHD and will thereby address important knowledge gaps. Undoubtedly, the project will contribute to the overarching aim of improving optimal living and care provision for adults with CHD.

Smartphone-based behavioral monitoring and patient-reported outcomes in adults with rheumatic and musculoskeletal disease

BackgroundRheumatic and musculoskeletal diseases (RMD) are associated with depression, fatigue, and disturbed sleep – symptoms that often impact behavior and activity. Patient reported outcomes (PROs) are a way of collecting information on the patient symptom experience directly from the individual. The purpose of this study was to measure and compare user smartphone sensor and activity data in adults with RMDs and assess associations with PROs.MethodsWe invited adults with RMDs enrolled in the FORWARD Databank to participate by installing a custom app on their smartphone and answering PROs (pain, global, HAQ-II) questions daily and weekly over 3 years. Passive data collected included mobility distance, unique calls and text messages, call durations, and number of missed calls. Confounders included sociodemographic, clinical, passive phone behavior, and seasonal factors. Kappa statistics between PRO and flares were computed to measure agreement. The agreement between daily and weekly VAS pain was estimated using the intraclass (ICC) correlation of a two-way random effect model. The relationship between the weekly PRO outcomes and the passive phone data was analyzed with a linear mixed-effect model (LMM), including a random intercept for participant and slope for time in the study with an unstructured covariate structure.ResultsOf the 446 participants, the mean (SD) age was 54 (12) years, most (65.5%) had rheumatoid arthritis (RA), the vast majority (91%) were female, and the US Northeast has the least representation (12%). Longer reaction times, interaction diversity, and higher mobility were associated with worse PROs while longer text messages were associated with better PROs. Participants in this study showed good levels of adherence which holds promise for future interventions using passive behavior measures in self-management and clinical follow-up.ConclusionThis is the first study to examine passive smartphone behavior with PROs in RMDs and we found significant associations between these behaviors and important health outcomes of pain and function. As smartphone usage continues to change, future studies should validate and expand on our findings with a goal of finding changes in patient symptoms passively through mobile device monitoring.

Long-term Adherence To Exercise In Adults With Preserved Ejection Fraction Heart Failure

<h3>Background</h3> Despite exercise being one of few strategies to improve outcomes for individuals with heart failure with preserved ejection fraction (HFpEF), exercise clinical trials in HFpEF are plagued by poor interventional adherence. Over the last 2 decades, our research team has developed, tested, and refined Heart failure Exercise And Resistance Training (HEART) Camp, a multi-component behavioral intervention to promote adherence to exercise in HF. We evaluated the effects of this intervention designed to promote adherence to exercise in HF focusing on a subgroup of participants with HFpEF. <h3>Methods and Results</h3> The randomized controlled trial included 204 adults with stable, chronic HF. Of those enrolled, 59 had HFpEF and were the target of this sub-group analysis. We tested adherence to exercise [defined as greater than or equal to 120 minutes of moderate-intensity (40-80% of heart rate reserve) exercise per week validated with a heart rate monitor] at 6, 12, and 18 months. We also tested intervention effects on symptoms (PROMIS-29 and Dyspnea-fatigue index), HF-related health status (Kansas City Cardiomyopathy Questionnaire, KCCQ), and physical function (6-minute walk test, 6MWT). Participants with HFpEF (n=59) were a mean of 64.6 ±9.3 years old, 54% male, 46% non-white with a mean ejection fraction of 55±6. Participants with HFpEF in the HEART Camp intervention group had significantly greater adherence compared to enhanced usual care at both 12 (43% vs. 14%, <i>phi</i>=.32, medium effect) and 18 months (56% vs. 0%, <i>phi</i>=.67, large effect). HEART Camp significantly improved walking distance on the 6MWT (η² = 0.13, large effect) and the KCCQ overall (η²=0.09, medium effect), clinical summary (η²=0.16, large effect), and total symptom (η²=0.14, large effect) scores. <h3>Conclusions</h3> A multi-component, behavioral intervention can be used to improve long-term adherence to exercise, physical function, and patient-reported outcomes in adults with HFpEF. Our results provide strong rationale for a large HFpEF clinical trial to validate these findings and examine interventional mechanisms and delivery modes that may further promote adherence, and improve clinical outcomes, in this population.

Effects of physical activity interventions using wearables to improve objectively-measured and patient-reported outcomes in adults following orthopaedic surgical procedures: A systematic review.

ObjectiveTo synthesize evidence on physical activity interventions that used wearables, either alone or in combination with education or rehabilitation, in adults following orthopaedic surgical procedures.MethodsPubMed, CINAHL, PsycINFO and EMBASE were searched for randomized controlled trials of wearable-based interventions from each database’s inception to August 2021 in patients undergoing orthopaedic surgery. Relevant outcomes included physical activity, physical function, pain, psychological distress, or general health. PEDro scale scoring ranges from 0 to 10 and was used to appraise studies as high (≥7), moderate (5–6), or poor (<5) quality.ResultsOf 335 articles identified, 6 articles met eligibility criteria. PEDro scores ranged from 2 to 6, with 3 studies of moderate quality and 3 of poor quality. Studies included patients undergoing total knee (number; n = 4) or total knee or hip (n = 1) arthroplasty and lumbar disc herniation surgery (n = 1). In addition to wearables, intervention components included step diary (n = 2), motivational interviewing (n = 1), goal setting (n = 2), tailored exercise program (n = 2), or financial incentives (n = 1). Interventions were delivered in-person (n = 2), remotely (n = 3) or in a hybrid format (n = 1). Intervention duration ranged from 6 weeks to 6 months. Compared to controls, 3 moderate quality studies reported greater improvement in steps/day; however, 1 moderate and 2 poor quality studies showed no between-group difference in physical function, pain, or quality of life. No serious adverse events related to the use of wearable were reported.ConclusionsThe effects of physical activity interventions using wearables, either delivered in-person or remotely, appear promising for increasing steps per day after joint arthroplasty; however, this finding should be viewed with caution since it is based on 3 moderate quality studies. Further research is needed to determine the therapeutic effects of using wearables as an intervention component in patients undergoing other orthopaedic surgical procedures.Trial registrationPROSPERO Registration Number: CRD42020186103

Mobility and gait in adults with cerebral palsy: Evaluating change from adolescence.

Previous studies have looked at the short-term effectiveness of conservative and surgical treatment of children with cerebral palsy (CP), but few have explored the long-term outcomes into adulthood using gait analysis and patient-reported outcome measures. How do gait, mobility, and patient-reported outcomes in adults with CP who received specialized pediatric orthopedic care change from adolescence? We identified 645 adults with 1) CP, 2) age 25-45 years, and 3) an adolescent instrumented gait analysis (IGA) at our center. Measurement outcomes included physical examination, IGA, and select domains of the Patient-Reported Outcomes Measurement Information System (PROMIS). Participants included 136 adults with CP; Gross Motor Function Classification System levels I (21 %), II (51 %), III (22 %), and IV (7%); 57 % males; and average age 16 ± 3/29 ± 3 years (adolescent/adult visits). There was no significant difference in gait deviation index, stride length, or gross motor function between adolescent and adult visits. There were statistically significant but not clinically meaningful declines in gait velocity. At adulthood, PROMIS results revealed limitations in physical function compared with a normative sample but no differences in depression, participation, or pain interference. In this relatively homogeneous group of adults with CP who received orthopedic care from one center, gait and gross motor function showed no clinically meaningful change from adolescence, which differs from recent reports of declining mobility in adulthood. Expert orthopedic care, guided by IGA, may prevent losses in functional mobility for adults with CP.

The HEART Camp Exercise Intervention Improves Exercise Adherence, Physical Function, and Patient-Reported Outcomes in Adults With Preserved Ejection Fraction Heart Failure

BackgroundDespite exercise being one of few strategies to improve outcomes for individuals with heart failure with preserved ejection fraction (HFpEF), exercise clinical trials in HFpEF are plagued by poor interventional adherence. Over the last 2 decades, our research team has developed, tested, and refined Heart failure Exercise And Resistance Training (HEART) Camp, a multicomponent behavioral intervention to promote adherence to exercise in HF. We evaluated the effects of this intervention designed to promote adherence to exercise in HF focusing on subgroups of participants with HFpEF and heart failure with reduced ejection fraction (HFrEF). Methods and ResultsThis randomized controlled trial included 204 adults with stable, chronic HF. Of those enrolled, 59 had HFpEF and 145 had HFrEF. We tested adherence to exercise (defined as ≥120 minutes of moderate-intensity [40%–80% of heart rate reserve] exercise per week validated with a heart rate monitor) at 6, 12, and 18 months. We also tested intervention effects on symptoms (Patient-Reported Outcomes Measurement Information System-29 and dyspnea-fatigue index), HF-related health status (Kansas City Cardiomyopathy Questionnaire), and physical function (6-minute walk test). Participants with HFpEF (n = 59) were a mean of 64.6 ± 9.3 years old, 54% male, and 46% non-White with a mean ejection fraction of 55 ± 6%. Participants with HFpEF in the HEART Camp intervention group had significantly greater adherence compared with enhanced usual care at both 12 (43% vs 14%, phi = 0.32, medium effect) and 18 months (56% vs 0%, phi = 0.67, large effect). HEART Camp significantly improved walking distance on the 6-minute walk test (η2 = 0.13, large effect) and the Kansas City Cardiomyopathy Questionnaire overall (η2 = 0.09, medium effect), clinical summary (η2 = 0.16, large effect), and total symptom (η2 = 0.14, large effect) scores. In the HFrEF subgroup, only patient-reported anxiety improved significantly in the intervention group. ConclusionsA multicomponent, behavioral intervention is associated with improvements in long-term adherence to exercise, physical function, and patient-reported outcomes in adults with HFpEF and anxiety in HFrEF. Our results provide a strong rationale for a large HFpEF clinical trial to validate these findings and examine interventional mechanisms and delivery modes that may further promote adherence and improve clinical outcomes in this population. Clinical Trial Registration: URL: https://clinicaltrials.gov/. Unique identifier: NCT01658670

Efficacy and safety of omalizumab in chronic rhinosinusitis with nasal polyps: a systematic review and meta-analysis of randomised controlled trials

ObjectivesTo assess the efficacy and safety of omalizumab for chronic rhinosinusitis with nasal polyps (CRSwNP) and to identify evidence gaps that will guide future research on omalizumab for CRSwNP.DesignSystematic review...

Patient-Reported Outcomes in Adults With Congenital Heart Disease Following Hospitalization (from APPROACH-IS)

In this international study, we (1) compared patient-reported outcomes (PROs) in adults with congenital heart disease (CHD) who had versus had not been hospitalized during the previous 12 month, (2) contrasted PROs in patients who had been hospitalized for cardiac surgery versus nonsurgical reasons, (3) assessed the magnitude of differences between the groups (i.e., effect sizes), and (4) explored differential effect sizes between countries. APPROACH-IS was a cross-sectional, observational study that enrolled 4,028 patients from 15 countries (median age 32 years; 53% females). Self-report questionnaires were administered to measure PROs: health status; anxiety and depression; and quality of life. Overall, 668 patients (17%) had been hospitalized in the previous 12 months. These patients reported poorer outcomes on all PROs, with the exception of anxiety. Patients who underwent cardiac surgery demonstrated a better quality of life compared with those who were hospitalized for nonsurgical reasons. For significant differences, the effect sizes were small, whereas they were negligible in nonsignificant comparisons. Substantial intercountry differences were observed. For various PROs, moderate to large effect sizes were found comparing different countries. In conclusion, adults with CHD who had undergone hospitalization in the previous year had poorer PROs than those who were medically stable. Researchers ought to account for the timing of recruitment when conducting PRO research as hospitalization can impact results.

Atrial arrhythmias and patient-reported outcomes in adults with congenital heart disease: An international study

Atrial arrhythmias (ie, intra-atrial reentrant tachycardia and atrial fibrillation) are a leading cause of morbidity and hospitalization in adults with congenital heart disease (CHD). Little is known about their effect on quality of life and other patient-reported outcomes (PROs) in adults with CHD. The purpose of this study was to assess the impact of atrial arrhythmias on PROs in adults with CHD and explore geographic variations. Associations between atrial arrhythmias and PROs were assessed in a cross-sectional study of adults with CHD from 15 countries spanning 5 continents. A propensity-based matching weight analysis was performed to compare quality of life, perceived health status, psychological distress, sense of coherence, and illness perception in patients with and those without atrial arrhythmias. A total of 4028 adults with CHD were enrolled, 707 (17.6%) of whom had atrial arrhythmias. After applying matching weights, patients with and those without atrial arrhythmias were comparable with regard to age (mean 40.1 vs 40.2 years), demographic variables (52.5% vs 52.2% women), and complexity of CHD (15.9% simple, 44.8% moderate, and 39.2% complex in both groups). Patients with atrial arrhythmias had significantly worse PRO scores with respect to quality of life, perceived health status, psychological distress (ie, depression), and illness perception. A summary score that combines all PRO measures was significantly lower in patients with atrial arrhythmias (-3.3%; P = .0006). Differences in PROs were consistent across geographic regions. Atrial arrhythmias in adults with CHD are associated with an adverse impact on a broad range of PROs consistently across various geographic regions.

Measurement properties of the product of investigator's global assessment and body surface area in children and adults with atopic dermatitis.

Multiple clinician-reported outcome measures exist for atopic dermatitis (AD) severity. However, there is no gold standard for use in clinical practice. To determine the measurement properties of the product of validated Investigator's Global Assessment for AD (vIGA) and body surface area (BSA) overall or divided into six categories (cBSA: 0%/0.1, <10%/10, <30%/30, <50%/50, <70%/70 and <90%/90-100%) and compare with other clinician-reported and patient-reported outcomes in adults and children with AD. We performed a prospective dermatology practice-based study using questionnaires and evaluation by a dermatologist (n=653). vIGA*BSA and vIGA*cBSA had good convergent validity with BSA (Spearman's ρ=0.97 and 0.93), eczema area and severity index (ρ=0.94 and 0.92), and objective SCORAD (ρ=0.88 and 0.89); and weak-to-good convergent validity with Numeric Rating Scale average itch (ρ=0.22 and 0.22) and worst itch (ρ=0.27 and 0.28), Patient-Oriented Eczema Measure (ρ=0.44 and 0.43), Dermatology Life Quality Index (ρ=0.48 and 0.49), ItchyQOL (ρ=0.45 and 0.46), PROMIS Sleep Disturbance (ρ=0.46 and 0.37) and sleep-related impairment (ρ=0.31 and 0.31) in adults and/or children; very good discriminant validity for physician-reported global AD severity; good responsiveness to change of severity of AD and itch; and good reliability (intraclass correlation coefficient [95% confidence interval]: 0.72 [0.60-0.81] and 0.74 [0.62-0.82]) with no floor or ceiling effects. Thresholds for interpretability bands and clinically important difference were established. vIGA*BSA and vIGA*cBSA scores showed good convergent and discriminant validity, reliability, responsiveness and interpretability in adults and children with AD, and were feasible for use in clinical practice. vIGA*BSA and vIGA*cBSA had slightly lower convergent validity than EASI or objective SCORAD, but might be more efficient to collect and score.

Improvement in Patient-Reported Outcomes in Adults with Type 1 Diabetes Treated with Sotagliflozin plus Insulin Versus Insulin Alone

Background: Diabetes-related distress is common among persons affected by diabetes and is associated with suboptimal glycemic control and complications, thus constituting a relevant patient-report outcome (PRO). Improving glycemic control may reduce diabetes distress and improve treatment satisfaction. This post hoc analysis evaluated PRO data for a pooled cohort of adults with type 1 diabetes (T1D) receiving sotagliflozin as adjunct to optimized insulin in the inTandem1 and inTandem2 studies. Methods: Clinically meaningful changes in the Diabetes Treatment Satisfaction Questionnaire status version (DTSQs) and the two-item Diabetes Distress Scale (DDS2) total and individual scores were examined in the pooled data from the first 24 weeks of the studies. Results: In the cohort of patients with a baseline DTSQs total score ≤32 (∼76% of entire cohort), nearly twice as many patients treated with sotagliflozin 200 (45.9%) or 400 mg (42.3%) experienced a >3-point improvement from baseline versus those treated with placebo (24%). Treatment with sotagliflozin led to statistically significant (P < 0.05) improvements across all DTSQs items. Approximately 42% of all patients were considered to have a high risk of diabetes distress (total DDS2 score ≥6) at baseline following insulin optimization. More patients shifted from high to low risk with sotagliflozin compared with placebo (∼40% vs. 23%; P ≤ 0.0002). The baseline-adjusted difference in DDS2 from placebo was significantly (P < 0.001) reduced by -0.5 and -0.6 for sotagliflozin 200 and 400 mg, respectively. Conclusions: Patients with T1D treated with sotagliflozin in addition to optimized insulin therapy reported meaningful improvements in treatment satisfaction and diabetes distress. NCT02384941 and NCT02421510.

Patient-reported outcomes in adult females with bladder exstrophy: A study of long-term sexual, reproductive and urinary outcomes using social media

Limited data exist on patient-reported outcomes in adults with bladder exstrophy (BE). We partnered with the Association for the Bladder Exstrophy Community (A-BE-C) using social media to survey adult females with BE. The aim of the study was to assess long-term patient-reported sexual, reproductive and continence outcomes. Between December 3, 2018 and January 18, 2019, A-BE-C promoted an anonymous survey of adult females with BE on social media. The survey included the Female Sexual Function Index (FSFI) and International Consultation on Incontinence Questionnaire - Short Form (ICIQ-SF) in addition to questions on demographics, reproductive and gynecological outcomes. A total of 130 women with a median age of 30 years (IQR 26, 41) completed the survey. The majority of women were born in the United States (N=86, 66.2%). Women reported a median of 10 (IQR 5, 17) surgeries performed for their condition. The mean ICIQ-SF score was 6.2±6.2 (moderate). Only 19.2% (N=25) reported volitionally voiding and the majority reported intermittent catheterization through a catheterizeable channel or the urethra (Summary Table). The mean FSFI score was (20.1±9.0), indicating risk for sexual dysfunction (FSFI<26.55). Of the respondents, 28.5% (N=37) were treated for uterine prolapse, 56.9% (N=74) required surgery in order to use tampons or have penetrative intercourse, and over half (55.4%) reported some degree of dissatisfaction with the appearance of their external genitalia. Forty-seven women (36.2%) reported pregnancies, and of these 32 (68.1%) reported complications with pregnancy. Outcomes of 100 pregnancies included miscarriage/abortion (41.0%), preterm vaginal (3.0%), preterm cesarean section (19.0%), term vaginal (2.0%), and term cesarean section (35.0%). Seven (15.2%) women identified having children with a medical diagnosis, none of which included BE. Infertility was reported in 17.3% (22/127) women. Women identified sexual function, fertility, and body image as areas that need future research. This is the largest study of patient-reported outcomes in females with BE achieved through partnership with an advocacy group and social media. We found that continence and sexual function were both impaired in adult women with BE. Respondents reported poor obstetric and gynecological outcomes including infertility, uterine prolapse, vaginal stenosis, and need for multiple surgeries. BE surgeons, through multi-institutional collaboration, should continue to reassess surgical techniques at initial repair to better address these functional issues. These results should be a part of counseling parents of newborns with BE.