Outcomes Data Collection Research Articles

The design, implementation, and impact of an automated patient-reported outcome data collection and adverse event surveillance tool: a randomized trial

BackgroundIncorporating patient-reported outcome measures into routine clinical care can improve the patient experience, increase engagement, and establish a structured method for gathering adverse event (AE) data. Systematically collecting this information on a large scale can also inform new solutions for removing treatment barriers like medication nonadherence. This study evaluated whether implementing a patient-reported outcome data collection and adverse event surveillance tool would result in greater treatment continuation for patients receiving care on a telehealth platform.MethodsWe used iterative plan-study-do-act cycles to evaluate how this data collection and surveillance tool—a short prompt for patients to provide information on treatment satisfaction and side effects—impacted treatment continuation, the outcome of interest. We tested two cycles in n = 2,000 patients receiving care for erectile dysfunction on a telehealth platform as a randomized controlled trial, and accounted for incidents where true randomization was not possible during implementation. The first cycle tested the tool alone, while the second cycle tested the tool in conjunction with a messaging template system that provided standardized side effect counseling.ResultsCompared to patients in the control group, patients in the intervention group were more likely to refill their prescription over the duration of the study period (75% vs. 71%, Kaplan Meier log-rank test, p = 0.04). Receiving standardized counseling as part of the AE response system was positively associated with treatment continuation (p = 0.0005).ConclusionsPrompting patients to report side effects and outcomes outside of routine clinical visits has the potential to improve quality of care in virtual treatment.Trial registrationThis trial has been retrospectively registered as a clinical trial (ClinicalTrials.gov Identifier: NCT05895539, registered June 8, 2023).

AHP P01 A Survey To Explore The Use Of Nutritional Outcome Measures In Clinical Practice By Dietitians In Oesophago-Gastric Cancer Care In The British Isles

Abstract Background Dietitians in clinical practice face the challenge of providing high quality care whilst demonstrating clinical effectiveness and continuous improvement. Assessing the effectiveness of dietetic intervention is vital in ensuring the provision of clinically effective dietetic services. Nutritional outcome measures are key in identifying the impact of dietetic interventions; enabling dietitians to objectively measure the effectiveness of their care as well as providing patients, other health care professionals and commissioners with data around the value of care provision. Currently there are no specific nutritional outcomes associated with oesophago-gastric cancer care in the UK. Methods A survey was disseminated through direct personal emails to 93 Dietitians known to be working in UGI, a list derived from NOGCA audit sites of 2021, with the aim of assessing the current use of nutritional outcome measures by Dietitians working in oesophago-gastric cancer acute care in the British Isles. Further promotion was directed through a UGI Dietitians Forum and through social media posts. A YouTube video was produced and distributed alongside the survey, with the aim of providing education around identifying nutritional outcomes. The video also aimed to connect personally with people, with a visual presentation from the researcher. Results 54 responses represented the entire patient journey. Whilst 72% of respondents reported identifying and documenting dietetic outcomes, the quality of these assessments was low, mainly focusing on weight, weight changes and hand grip strength. Other common themes related to nutritional intake, quality of life and symptom impact. Less common outcomes measured included standardised nutritional assessment (PG-SGA), gastro-intestinal symptom evaluation, optimised prehabilitation, biochemical parameters and patient experience. There was little standardisation in how outcomes are used and when they were taken. There is no national database available to collate OG nutritional outcome data, representing a missed opportunity to support national benchmarking. Conclusions This study has presented not only the inconsistencies in nutritional outcome data collection (definition, timing and tools) and the need for standardisation of outcomes use in this speciality, it has also demonstrated the challenges around the promotion of outcomes measures and application/roll out into clinical practice. Novel ways of obtaining and evaluating nutritional outcomes will need to be considered. This will form the basis of a further research study, exploring barriers to completion of nutritional outcomes.

Outcomes of late-stages infantile Blount's disease managed by acute single stage: medial hemi-plateau elevation and metaphyseal osteotomy. Eight case series.

This study aimed to evaluate the clinical and radiological outcomes of surgical intervention involving acute medial hemi-plateau elevation and metaphyseal osteotomy with internal fixation and growth modulation for late-stages Blount's disease. A retrospective analysis was conducted on consecutive patients with late-stages infantile tibia vara between 2014 and 2020. Inclusion criteria consisted of children aged 8 years and older with Blount's disease with Langenskiold stage IV, V or VI, tibia vara more than 30*, medial plateau depression angle (MPDA) exceeding 25* and knee instability with a minimum follow-up period of 3 years. Patients with Limb length discrepancy greater than 5 cm were excluded. Data collection included assessments of age, weight, disease stage, clinical tibiofemoral angle (TFA), mechanical medial proximal tibial angle (mMPTA), MPDA, tibial torsion and knee instability. Functional outcomes were evaluated using modified Pediatric Outcome Data Collection Instruments (PODCI) scores. The study included 5 children with 8 affected limbs. Among them, three children with five limbs had recurrent deformities after previous surgeries. All cases showed significant improvements in TFAs, mMPTA, tibial torsion, knee instability and modified PODCI scores. Complications included 4 cases of superficial infections, 1 intra-articular fracture, 2 minor screw breakage and 2 on-table under correction. Surgical intervention involving acute Single-stage medial hemi-plateau elevation and metaphyseal osteotomy leads to satisfactory outcomes in late-stages Blount's disease among older children. It is crucial to achieve proper correction of all deformity components during surgery, with epiphysiodesis on the lateral side to prevent further recurrence. Continuous follow-up until skeletal maturity is essential for optimal results.

NCOG-29. THE NEURO-ONCOLOGY MAGNITUDE OF CLINICAL BENEFIT SCALE (NEURO-MCBS) AS A COMPREHENSIVE AND CLINICALLY RELEVANT ASSESSMENT TOOL TO DETERMINE CLINICAL BENEFIT FROM TARGETED THERAPIES IN CNS TUMORS

Abstract BACKGROUND Once first-line therapy options fail, there are no effective therapy options for most neuro-oncological conditions, including glioblastoma, gliomas, and brain metastases, thereby representing a high unmet clinical need. Novel targeted therapy options are often explored in small phase-I studies or are given as off-label therapies. Assessment of treatment efficacy in this setting is challenging and should incorporate outcome measures such as progression-free survival (PFS) or objective response rate (ORR) as well as clinical parameters. To address this, we developed the Neuro-Oncology Magnitude of Clinical Benefit Scale (Neuro-MCBS) as a comprehensive clinical tool to assess treatment efficacy in patients with CNS tumors who receive off-label targeted therapies outside of clinical trials. We prospectively evaluated the feasibility and clinical utility of the Neuro MCBS in a neuro-oncology patient cohort enrolled in MTB@ZPM. METHODS MTB@ZPM (NCT03503149) is a longitudinal observational study that provides comprehensive molecular profiling, consensus-guided and ranked targeted therapy recommendations and collection of prospective clinical outcome data. The Neuro-MCBS (grade 1 + 2=clinical benefit, grade 3=minimal benefit, grade 0=no benefit) was applied and correlated with PFS, the PFS2/PFS1 ratio, performance status (KPS, ECOG), and imaging parameters. RESULTS From February 1st, 2022 until January 31st, 2023, 70 patients were enrolled. While only a minority of n=17/36 (47%) glioblastoma and n=2/8 (25%) IDH-mutant astrocytoma patients benefited from targeted therapies, n=7/8 (88%) meningioma, n=9/10 (90%) brain metastases, and n=5/8 (63%) other tumor patients achieved a Neuro-MCBS of 1 or 2, i.e. derived clinical benefit from the molecularly based therapy. CONCLUSIONS The Neuro-MCBS is a comprehensive and clinically relevant assessment tool that incorporates clinical, imaging, and survival outcome data to determine the degree of clinical benefit from targeted therapies in patients with primary and secondary CNS tumors. Its incorporation into clinical practice provides an objective outcome measure of clinical utility vs. futility of targeted therapies.

Video-Delivered Family Therapy for Perinatal Women With Depressive Symptoms and Family Conflict: Feasibility, Acceptability, Safety, and Tolerability Results From a Pilot Randomized Trial.

Although individual-level treatments exist for pregnant and postpartum women with depression, family conflict is a significant factor that can contribute to the development and severity of perinatal depressive symptoms. Yet, there is a lack of research on family therapy for perinatal women with moderate to severe depressive symptoms and family conflict. Further, research is needed on the feasibility, acceptability, safety, and tolerability of family therapies for perinatal depression that are delivered using Health Insurance Portability and Accountability Act-compliant videoconferencing technology (VCT). This paper describes the feasibility, acceptability, safety, and tolerability of a VCT-based family therapeutic intervention, Resilience Enhancement Skills Training (REST), for perinatal women with moderate to severe depressive symptoms and moderate to high conflict with their family members. This paper includes data from an ongoing randomized trial that compares an experimental family therapeutic intervention (REST) to standard of care (VCT-based problem-solving individual therapy) for the treatment of moderate to severe depressive symptoms in perinatal women with moderate to high family conflict. Both interventions were delivered by masters-level therapists using VCT. A total of 83 perinatal women and their adult family members (N=166 individuals) were recruited for participation in the study. Feasibility, defined as therapist adherence to ≥80% of REST session content, was assessed in audio-recorded sessions by 2 expert raters. Acceptability was defined as ≥80% of families completing REST, including completion of ≥80% homework assignments and family report of satisfaction with REST. Completion of REST was assessed by review of therapist session notes, and satisfaction was assessed by participant completion of a web-based questionnaire. The Beck Depression Inventory-Second Edition was administered to perinatal women by research assistants (blind to study group assignment) to assess safety, defined as a reduction in depressive symptoms during the treatment phase. The Family Environment Scale-Family Conflict subscale was administered by therapists to participants during the treatment phase to assess tolerability, defined as a reduction in family conflict during the treatment phase. On average, the therapists achieved 90% adherence to REST session content. Of the families who started REST, 84% (32/38) of them completed REST, and on average, they completed 89% (8/9) of the homework assignments. Families reported satisfaction with REST. The results showed that REST is safe for perinatal women with moderate to severe depressive symptoms, and none discontinued due to worsened depressive symptoms. The results showed that REST is well tolerated by families, and no families discontinued due to sustained family conflict. The results show that REST is feasible, acceptable, safe, and tolerable for families. These findings will guide our interpretation of REST's preliminary effectiveness upon completion of outcome data collection. ClinicalTrials.gov NCT04741776; https://clinicaltrials.gov/ct2/show/NCT04741776.

Effects of Idecabtagene Vicleucel (Ide-Cel) Versus Standard Regimens on Health-Related Quality of Life (HRQoL) in Patients with Relapsed/Refractory Multiple Myeloma (RRMM) Who Had Received 2-4 Prior Regimens: Updated Results from the Phase 3 KarMMa-3 Trial

Background: An interim analysis (data cutoff date April 18, 2022) of the international, open-label, phase 3 KarMMa-3 trial (NCT03651128) showed the chimeric antigen receptor T cell therapy idecabtagene vicleucel (ide-cel) significantly improved progression-free survival and treatment response rates, as well as HRQoL, as compared with standard regimens, in patients with triple-class exposed (TCE) RRMM who had received 2-4 prior regimens (Rodriguez-Otero P, et al. N Engl J Med 2023;388:1002-1014; Delforge M, et al., HemaSphere, 2023;7(S3):P905). The aim of the present study was to analyze the effect of ide-cel versus standard regimens (daratumumab [DARA], pomalidomide, and dexamethasone [DEX]; DARA, bortezomib, and DEX; ixazomib, lenalidomide, and DEX; carfilzomib and DEX; or elotuzumab, pomalidomide, and DEX) on HRQoL in KarMMa-3 trial participants with an extended follow-up of patient-reported outcomes (PRO) data collection (data cutoff date April 28, 2023). Methods: In KarMMa-3, HRQoL was assessed using the European Organisation for Research and Treatment of Cancer (EORTC) Quality-of-Life Questionnaire-Core 30 (QLQ-C30), EORTC Quality of Life Questionnaire Multiple Myeloma Module (QLQ-MY20), and EQ-5D-5L. HRQoL assessments were completed at screening (baseline); the day of ide-cel infusion or the first dose of standard treatment; and monthly from 2 to 28 months; and every 3 months thereafter. The prespecified primary outcomes of interest were EORTC QLQ-C30 global health status/QoL, physical functioning, cognitive functioning, fatigue, and pain; EORTC QLQ-MY20 disease symptoms and side effects of treatment; and the EQ-5D visual analog scale (EQ-VAS). Least squares (LS) mean changes from baseline through month 25 for ide-cel and standard regimens were compared by constrained longitudinal data analysis (cLDA). Effect sizes were estimated by Hedges' g. The effect of ide-cel versus standard regimens on time to confirmed improvement and deterioration in HRQoL (defined by prespecified change thresholds and sustained duration) were compared by stratified Cox proportional hazards regression. There was no adjustment for multiple testing, and all P values reported are nominal. Results: Overall, 254 patients were randomized to the ide-cel arm and 132 to the standard regimens arm. Baseline characteristics were well balanced across treatment arms; both arms had a median age of 63 years, a median of 3 prior anti-myeloma regimens, and median time from MM diagnosis to screening was 4 years. The baseline HRQoL scores were generally comparable between treatment arms. Overall LS mean changes from baseline to month 25 showed significant differences (nominal P value < 0.05) in favor of ide-cel for 18 out of 21 HRQoL domains, with effect sizes (Hedges' g) of 0.27 to 0.82 (Table). For 13 of these domains (including 6 primary domains of interest), the difference in overall LS mean change from baseline exceeded the prespecified minimum important difference (MID) threshold. Time to confirmed improvement was significantly shorter in the ide-cel arm than the standard regimens arm for 19 domains, including all primary domains of interest (Table). Time to confirmed deterioration showed trends of being longer in the ide-cel arm than the standard regimens arm for most domains and was significantly longer for emotional functioning, cognitive functioning, social functioning, dyspnea, and constipation as measured by the EORTC QLQ-C30. The time to confirmed deterioration in side effects of treatment (based on the EORTC QLQ-MY20) was significantly longer for patients in the ide-cel arm and time to confirmed improvement was significantly faster, as compared with the standard regimens arm (Table). Conclusions: In the KarMMa-3 trial, patients with TCE RRMM who had received 2-4 prior regimens, ide-cel significantly and meaningfully improved MM-relevant symptoms, functioning, and overall health status/HRQoL compared with standard regimens. Confirmed HRQoL improvements in the ide-cel arm occurred sooner than standard regimens. PRO improvements were sustained over more than 2 years, which is notable after a single infusion treatment with ide-cel. These patient-reported findings further support the benefit of ide-cel in this patient population.

The use of outcome data in patient consultations from the healthcare professionals’ and patients’ perspectives: A mixed methods study

ObjectivesTo gain insight into healthcare professionals’ (HCPs’) perspectives on the use of outcome data in consultations and to understand which aggregated outcomes patients find important. MethodsThis study had a mixed-methods design and consisted of two steps:1.HCPs (n = 11) were interviewed about the use of outcome data in consultations. Thematic analysis was used for data analysis.2.Patients with prostate cancer, lung cancer, and inflammatory bowel disease (IBD) completed questionnaires (n = 283) to identify important outcomes. Descriptive statistics were used for data analysis. ResultsHCPs indicated that aggregated outcome data are not routinely used in consultations. They pointed out various barriers to using outcome data, e.g., low response rates of PROMs, and suggested actions to address these barriers, including training of HCPs in outcome data usage. Patients rated the majority of aggregated outcomes as important, although preferences differed between the studied health conditions. ConclusionBoth HCPs and patients underscored the importance of discussing outcome data in consultations. Nevertheless, HCPs encountered several barriers to using outcome data. Furthermore, patients with different health conditions have somewhat different information needs. Practice implicationsThe study identified several actionable steps to enhance the collection and application of outcome data in consultations.

ASSESSMENT OF THE QUALITY OF LIFE OF CHILDREN WITH NATAL SPINAL INJURY AND OPTIMIZATION OF REHABILITATION MEASURES

The purpose of this study was to compare upper limb function and quality of life in preschool children with complications of birth injuries to the cervical spinal cord and spine. Research methods: the study used a clinical neurological examination and the results of the Pediatric Outcomes Data Collection Instrument (PODCI) scale, neurofunctional (electroneuromyography - ENMG), neuroimaging (ultrasound - ultrasound of the cervical spinal cord and spine) research methods. Results and discussion. Upper extremity dysfunction is an area that primarily reduces quality of life in children with HSDD. This result highlights how difficult it is to manipulate objects with the upper limbs during certain activities, such as lifting heavy books, pouring milk, opening a previously opened can, using cutlery, combing hair, buttoning clothes, putting on a coat, and writing with a pencil. All of these may be related to the limited range of motion in these children, which is impaired by muscle weakness, simultaneous activation of antagonists, differences in the length of the affected limb, and contractures. Subsequently, children with HSDD demonstrate limitations in performing functional tasks that are necessary for independence in daily activities and quality of life, as well as tasks requiring fine motor skills. Conclusion. Thus, the quality of life of preschool children before treatment was reduced on all scales. When the results for each domain were analyzed separately, both groups showed poor results in the domain that reflects hand activity among all domains. Impaired hand function is the main area that reduces the quality of life in children with complications of PTSD. This domain has the lowest score compared to other domains, which has also been confirmed by other studies. As a result of the course of rehabilitation measures, a positive statistically significant dynamics of quality of life indicators was noted KEYWARDS. Children, natal spinal injury, the quality of life, rehabilitation measures.

Impact on quality of life caused by vitamin D deficiency in bariatric patients: a literature review

Introduction: Bariatric surgery is a procedure performed to control obesity and its associated morbidities. Surgeries with malabsorptive and mixed techniques cause a deficit in the absorption of micronutrients, including vitamin D. Objective: The objective of this literature review was to investigate studies that verified the impact of vitamin D deficiency on the preand postoperative quality of life of individuals undergoing bariatric surgery. Methods: The search strategy was carried out in the virtual databases PubMed, Scielo, LILACS, Scopus, Web of Science, Embase, and the Virtual Health Library, in addition to gray literature such as Google Scholar, OpenGrey, Ibict/BDTD (Brazilian digital library of theses and dissertations) and ProQuest using the Medical Subject Headings (MeSH) and Health Sciences Descriptors (DeCS) descriptors with Boolean logical operators (AND, OR and NOT. The studies considered potentially eligible were those that presented in the same article the levels of vitamin D in the pre and postoperative period of the individuals, as well as the effect generated on their quality of life from where the data collection and analysis of the main outcomes found were made. Results: The online search resulted in 197 studies located in the virtual bibliographic databases and 115 in the gray literature. After removing the duplicates, 235 articles remained that were read by title and abstract, and 12 articles were chosen for reading the full text, leaving 7 that met the inclusion criteria. In most of the included studies, vitamin D deficiency was higher preoperatively than postoperatively in bariatric individuals. Better levels of 25(OH)D were correlated with reduced body weight, decreased BMI, and improved glycemic profile. Conclusion: Combating obesity remains an important issue in Public Health, with bariatric surgery being a safe way to reduce overweight since vitamin D levels are lower in the postoperative period of bariatric individuals, however, it is suggested to choose the appropriate technique to maintain ideal vitamin indices.

Outcomes of patients with early calcific aortic valve disease detected by clinically indicated echocardiography.

Previous studies have demonstrated relatively slow rates of progression of early calcific aortic valve disease (CAVD), which encompasses aortic sclerosis (ASc) and mild aortic stenosis (AS). The potential evolution to clinically significant AS is unclear, and we therefore examined the long-term outcomes of patients with ASc and mild AS detected at the time of clinically indicated echocardiography. Data from initial clinically indicated echocardiograms performed between 2010 and 2018 in patients aged ≥18 years were extracted and linked to nationally collected outcome data. Those with impaired right or left systolic ventricular function or other significant left-sided valve disease were excluded. A time to first event analysis was performed with a composite primary outcome of cardiovascular death and aortic valve intervention (AVI). Of the 13 313 patients, 8973 had no CAVD, 3436 had ASc, and 455 had mild AS. The remainder had moderate or worse stenosis. Over a median follow-up period of 4.2 (interquartile range 1.8-6.7) years (and after adjustment for age and sex), those with ASc were at greater risk of the primary outcome [hazard ratio (HR) 2.9, 95% confidence interval (CI) 2.1-4.0] and need for AVI (HR 26.8, 95% CI 9.1-79.1) compared with those with no CAVD. Clinical event rates accelerated after ∼5 years in those with mild AS. Patients with ASc are >25 times more likely to require AVI than those with no CAVD, and follow-up echocardiography should be considered within 3-4 years in those with mild AS.

Predictors of Physical Activity Levels in Ambulant/Semiambulant Children and Adolescents With Cerebral Palsy: A Clinical Cohort Study.

Children and adolescents with cerebral palsy (CP) show a reduced physical activity (PA) level compared with their typically developed peers. This study aimed to identify potential predictors of objectively evaluated habitual PA using data from a national clinical registry-the Cerebral Palsy Follow-Up Program(CPFP)-and proxy-reported questionnaires. Data from the CPFP database showed that female sex was associated with a lower PA level. Additional questionnaire data revealed age, popliteal angle limitation, Pediatric Outcomes Data Collection Instrument-Sports and Physical Functioning Scale score, screen time, Functional Mobility Scale score at 50m, and fatigue as predictors. The proposed models can be used for the prediction and early detection of the PA level and consequently for the potential improvement among ambulant/semiambulant individuals with CP. Further research should investigate the predictive impact of personal, social, and environmental factors on the PA level and the gap in PA levels between girls and boys.

71 Trends, Practice Patterns, and Opportunities for Improvement in Postnatal Steroids Use for Bronchopulmonary Dysplasia – Eleven Years Observational Study

Abstract Background Postnatal steroids (PNS) use in preterm infants improves pulmonary function in a select subset of patients but can potentially increase the risk of spontaneous intestinal perforation and cerebral palsy. Despite national guidelines, reducing intra-centre variation and demonstrating responsible stewardship in PNS use have been challenging. Objectives To describe (i) PNS type, regimen, time of initiation, use over time, and identify opportunities for improving practices in the use of PNS for bronchopulmonary dysplasia (BPD); (ii) outcome at discharge for infants who received PNS. Design/Methods We conducted a retrospective observational study in a quaternary NICU that included infants born at <33 weeks gestational age who received PNS to prevent or treat BPD between 2011 and 2021. We initially identified all infants who received PNS from the pharmacy registry, followed by demographic, PNS use details, and outcome data collection from the Canadian Neonatal Network (CNN) database, discharge summaries and, occasionally, by reviewing individual charts. Results The mean (SD) gestational age and birth weight of 184 eligible infants were 25(24-26) weeks and 720(625-841) grams respectively. Based on timing of first course of PNS, 2(1.1%), 93(50.5%), 85(46.2%), and 4(2.2%) infants received PNS in early (E) (< 7 days), early evolving (EE) (7-28 days), late evolving (LE) (29 days – 36 + 6 weeks PMA), and established (Es) (≥ 37 weeks) phases, respectively. 157 (85.3%) infants received dexamethasone and 22 (12%) received hydrocortisone as the first PNS course. Infants in the EE group, when compared with the LE group, received more days and higher cumulative dose of PNS 13(10-28) vs. 10(10-16) p= 0.04, and 24(24-60) vs. 24(24-25) hydrocortisone equivalents in milligrams/kilograms, p=0.01, respectively. Two common regimens used at our centre were 10-day Dexamethasone: A Randomized Trial (DART) and 22-day tapering doses of hydrocortisone (STOP-BPD). Deviation from published protocols based on duration (±20%), dose (±10%) or timing was 66.7% and 33.3% with hydrocortisone and dexamethasone use. Dexamethasone usage remained stable, but a small increase in hydrocortisone use and the cumulative PNS dose received occurred over time. Survival, survival without major morbidity, moderate to severe BPD, and technology dependence at discharge were 87%, 1.6%, 90.8% and 66.9%, respectively. Identified opportunities for improvement were: adapting national guidelines to local context, creating order sets to reduce deviations, and using this study’s outcomes during PNS decision-making conversations with family. Conclusion Variation in the use of hydrocortisone was higher than for dexamethasone, and cumulative dose received per infant increased over time. Potential interventions to reduce variation in PNS use and optimize decision- making were identified.

Self-reported Health-related Quality of Life in Adolescents With Cerebral Palsy.

Happiness, comfort, and motor function contribute to satisfaction with life for individuals with cerebral palsy (CP). Evidence-based medical care can improve motor function and physical health of youth with CP. Less is known about medical care and its relationship to health-related quality of life (HRQOL) in adolescents and young adults with CP. This study aimed to describe HRQOL among adolescents with CP to examine differences between adolescent (self) and parent (proxy) reports of HRQOL and to explore associations of pain, age, and gross motor function with HRQOL. This is a retrospective study including adolescents with CP classified as Gross Motor Function Classification System levels I to V, ages 11 to 20 years, reading ≥ a fourth-grade level, and who completed the self-reported Pediatric Outcomes Data Collection Instrument (PODCI). Parents completed the PODCI concurrently or within 12 months and scores were compared. In addition, self-reported scores were compared between age bands, across Gross Motor Function Classification System levels, with typically developing youth (TDY), and between youth with/without pain. PODCI scores from 102 adolescents [59 males; 15.0 (SD: 2.6) years old] were examined. Scores from 50 adolescents and parents were matched. Mean self-reported scores were significantly higher than mean parent-reported scores in 4 domains: upper extremity and physical function (P=0.018), sports and physical function (P=0.005), happiness (P=0.023), and global functioning (P=0.018). All domains, except Happiness, were significantly < TDY (P<0.01). The presence of pain was associated with lower scores in all domains (P<0.05). Examining HRQOL with the PODCI revealed significant limitations in physical function and higher pain in adolescents with CP compared with TDY. Self- and parent-reported PODCI results should be considered separately. Adolescents report higher HRQOL compared with parent proxy. Recognizing and validating the perspectives of youth and their parents presents an opportunity for providers to discuss different points of view with families. Such engagement can help promote self-efficacy in youth with CP as they transition to the responsibility of guiding their own care in adulthood. III, Retrospective comparative study.

S12-4 Feasibility evaluation of the Move Well, Feel Good movement behaviours intervention

Abstract Purpose Improving children’s motor competence through school-based physical activity interventions may be a mechanism for promoting positive mental health through psychosocial factors. Move Well, Feel Good (MWFG) is a such an intervention that was co-designed with children, class teachers, physical education (PE) teachers, and school leaders. This presentation describes the feasibility evaluation of MWFG. Methods Five primary schools were recruited from a low socioeconomic status community in northwest England. A 10-week intervention was co-designed which aimed to enhance children’s mental health through motor competence and psychosocial development. The core delivery of the programme involved one PE lesson per week devoted to development of locomotor, object control, and stability skills. This was supplemented by optional recess and home activities, and by non-PE curricular activities. Teachers were asked to record details of each MWFG session taught, and feasibility was evaluated against pre-defined criteria using surveys, interviews (teachers), and participatory focus groups (children). Pre- and post-intervention assessments of motor competence, mental health, wellbeing, psychosocial factors, and 24-hour movement behaviours were also completed. Results The 5 schools represented 83% of the target number of schools and 108 children consented (43% of target). PE and class teachers were recruited in all schools (100% of target). Intervention dose was reflected by 76% of scheduled PE lessons being delivered. Adherence was strong with &amp;gt;85% of children attending ≥75% of lessons. Positive indicators of acceptability were provided by 86% of children, 83% of PE teachers, and 90% of class teachers. Teachers commented on the children’s positive engagement irrespective of ability, the quality of resources provided, and the challenges of competing demands for curriculum time and spaces to deliver MWFG. The data collection methods were deemed acceptable by 91% of children and 80% of class teachers, and children spoke positively about participating in the data collection. Secondary outcome data collection was completed by 87%-100% of children, with a 0%-6% attrition rate at post-intervention. Descriptive analysis revealed favourable changes in motor competence and mental health. Conclusions MWFG was deemed to be feasible to develop further into a pilot trial, with improved school and child recruitment strategies warranted.

Electronic Patient-Reported Outcome Data Collection Systems in Oncology Clinical Trials: A Survey of Clinical Research Professionals (an Alliance Study).

To describe clinical research professionals (CRPs)' experiences with electronic patient-reported outcome (ePRO) data collection systems in oncology clinical trials and identify correlates of CRPs' attitude toward technology. An online survey was conducted among 210 CRPs from 125 National Cancer Institute-funded research sites. Measures included CRPs' demographic characteristics, working years, employment locations, and previous experiences with various types of ePROs. Their attitude toward technology was measured by the Technology Attitude Scale-Adapted. The Wilcoxon signed-rank test was used to compare two subdomains of attitude (perceived usefulness [PU] and perceived ease of use [PEU]). Multiple linear regression was used to explore correlates of (1) overall attitude, (2) PU, and (3) PEU. The significance level was 5%. Participants' median age was 41 years (range, 21-67). Most were female (90%) and White (82%). More than half of the participants had previous experiences with web-based ePROs using patients' own devices (72%) or site-/sponsor-provided on-site devices (eg, kiosks or tablets; 64%). CRPs who were 60 years or older (β = -0.32, P < .05) or worked for 10-20 years (β = -0.11, P < .05) had relatively negative attitudes, controlling for other factors. Previous experiences with more ePRO types were associated with more positive attitudes (β = 0.08, P = .02). Similar correlates were found with PU but not with PEU. This study revealed that CRPs had various experiences with ePRO systems and attitudes toward technology. Age, working years, and previous experiences with ePROs were correlates of overall attitude toward technology and PU. These findings suggest necessary targeted training to facilitate ePRO use in oncology clinical trials by improving CRPs' awareness and attitude toward technology.

Rationale and design of the DAPA-MI trial: Dapagliflozin in patients without diabetes mellitus with acute myocardial infarction

BackgroundTherapies that could further prevent the development of heart failure (HF) and other cardiovascular and metabolic events in patients with recent myocardial infarction (MI) represent a large and unmet medical need. MethodsDAPA-MI is a multicenter, parallel-group, registry-based, randomized, double-blind, placebo-controlled phase 3 trial in patients without known diabetes or established HF, presenting with MI and impaired left ventricular systolic function or Q-wave MI. The trial evaluated the effect of dapagliflozin 10 mg vs placebo, given once daily in addition to standard of care therapy, on death, hospitalization for HF (HHF), and other cardiometabolic outcomes. The primary objective of the trial was to determine, using the win-ratio method, if dapagliflozin is superior to placebo by comparing the hierarchical composite outcome of death, HHF, nonfatal MI, atrial fibrillation/flutter, new onset of type 2 diabetes mellitus, HF symptoms as measured by New York Heart Association Functional Classification at last visit, and body weight decrease ≥5% at last visit. Assuming a true win-ratio of 1.20 between dapagliflozin and placebo, 4,000 patients provide a statistical power of 80% for the test of the primary composite outcome. A registry-based randomized controlled trial framework allowed for recruitment, randomization, blinding, and pragmatic data collection of baseline demographics, medications, and clinical outcomes using existing national clinical registries (in Sweden and the UK) integrated with the trial database. ConclusionsThe trial explores opportunities to improve further the outcome of patients with impaired LV function after MI. The innovative trial design of DAPA-MI, incorporating national clinical registry data, has facilitated efficient patient recruitment as well as outcome ascertainment. Trial registrationClinicalTrials.gov Identifier NCT04564742.

Validity and reliability of the Turkish version of the hand-use-at-home questionnaire for children with unilateral cerebral palsy and neonatal brachial plexus palsy

Purpose To translate the Hand-Use-at-Home questionnaire (HUH), assesses the amount of spontaneous use of the affected hand in children with 18 bimanual activities, into Turkish and examine its validity and reliability on children with neonatal brachial plexus palsy (NBPP) or unilateral cerebral palsy (UCP). Materials and methods The HUH was translated and cross-culturally adapted to Turkish and administered to children with NBPP (n = 25) and UCP (n = 42) between 3 and 10 years. The psychometric analyses included reliability by internal consistency (Cronbach’s alpha) and test/retest reliability (intraclass correlation coefficient, ICC) structural validity was evaluated with exploratory factor analysis, and construct validity was investigated by matching the HUH with the Pediatric Outcome Data Collection Instrument Upper Extremity Scale (PODCI) (NBPP only), and Children’s Hand-Use Experience Questionnaire (CHEQ) (UCP only). Results HUH showed excellent test–retest reliability (ICC2,1 = 0.988 Cl (0.977–0.992)), excellent internal consistency (Cronbach’s-α = 0.989), and moderate correlation with CHEQ (rs = 0.558) in UCP and high correlation with PODCI Scale (rs = 0.789) in NBPP group. The HUH had low and moderate correlation respectively lesion-extent levels (r=-0.457) in NBPP and 5 Manual Ability Classification System levels (r=-0.688) in the UCP group. Conclusion The HUH is a valid and reliable tool to assess the amount of spontaneous use of the affected hand in Turkish children with NBPP and UCP.

Feasibility of conducting a cohort randomized controlled trial assessing the effectiveness of a nurse-led package of care for knee pain.

To evaluate the feasibility of conducting a cohort randomized controlled trial (RCT) of a nurse-led package of care for knee pain and determining a treatment sequence for use in a future trial. This study was an open-label, three-arm, single-centre, mixed-methods, feasibility cohort RCT. Adults aged ≥40 years with moderate-to-severe knee pain for ≥3 months were eligible. Participants were randomized into group A (non-pharmacological treatment first), group B (pharmacological treatment first), or group C (usual care). The intervention was delivered over 26 weeks. Outcomes were dropout rate, recruitment rate, intervention fidelity, ability to collect outcome data, and treatment acceptability. Seventeen participants were randomized and enrolled into each of groups A and B (5.2% recruitment rate), and 174 participants were randomized to group C. The participant characteristics at randomization were comparable across the three arms. Coronavirus disease (COVID-19) paused the study from March-November 2020. Participants enrolled in groups A and B before March 2020 were withdrawn at the restart. Of the 20 participants enrolled after the restart, 18 completed the study (10% dropout). The nurse reported delivering most aspects of the intervention with high fidelity. The participants viewed the package of care as structured, supportive and holistic, they learnt about self-managing knee pain, and they could engage with and follow the non-pharmacological treatment. Most found the non-pharmacological treatment more useful than the pharmacological treatment, preferring to receive it before or alongside analgesia. Many self-report questionnaires were not fully completed. The nurse-led package of care for knee pain was acceptable, with low dropout, although the cohort RCT design may not be feasible for a definitive trial. ClinicalTrials.gov, https://clinicaltrials.gov, NCT03670706.

Assessment of proxy-reported responses as predictors of motor and sensory peripheral neuropathy in children with B-lymphoblastic leukemia.

Chemotherapy-induced peripheral neuropathy (CIPN), a common condition in children with acute lymphoblastic leukemia, can be challenging to diagnose. Using data from Children's Oncology Group AALL0932 physical function study, we sought to determine if parent/guardian proxy-reported responses from the Pediatric Outcomes Data Collection Instrument could identify children with motor or sensory CIPN diagnosed by physical/occupational therapists (PT/OT). Four variables moderately discriminated between children with and without motor CIPN (c-index 0.76, 95% confidence interval [CI]: 0.64-0.84), but sensory and optimism-corrected models had weak discrimination (c-index sensory models 0.65, 95% CI: 0.54-0.74). New proxy-report measures are needed to identify children with PT/OT diagnosed CIPN.

Prediction of in-hospital bleeding in acutely ill medical patients: External validation of the IMPROVE bleeding risk score

IntroductionPharmacological thromboprophylaxis slightly increases bleeding risk. The only risk assessment model to predict bleeding in medical inpatients, the IMPROVE bleeding risk score, has never been validated using prospectively collected outcome data. MethodsWe validated the IMPROVE bleeding risk score in a prospective multicenter cohort of medical inpatients. Primary outcome was in-hospital clinically relevant bleeding (CRB) within 14 days of admission, a secondary outcome was major bleeding (MB). We classified patients according to the score in high or low bleeding risk. We assessed the score's predictive performance by calculating subhazard ratios (sHRs) adjusted for thromboprophylaxis use, positive and negative predictive values (PPV, NPV), and the area under the receiver operating characteristic curves (AUC). ResultsOf 1155 patients, 8 % were classified as high bleeding risk. CRB and MB within 14 days occurred in 0.94 % and 0.47 % of low-risk and in 5.6 % and 3.4 % of high-risk patients, respectively. Adjusted for thromboprophylaxis, classification in the high-risk group was associated with an increased risk of 14-day CRB (sHR 4.7, 95 % confidence interval [CI] 1.5–14.5) and MB (sHR 4.9, 95%CI 1.0–23.4). PPV was 5.6 % and 3.4 %, while NPV was 99.1 % and 99.5 % for CRB and MB, respectively. The AUC was 0.68 (95%CI 0.66–0.71) for CRB and 0.73 (95%CI 0.71–0.76) for MB. ConclusionThe IMPROVE bleeding risk score showed moderate to good discriminatory power to predict bleeding in medical inpatients. The score may help identify patients at high risk of in-hospital bleeding, in whom careful assessment of the risk-benefit ratio of pharmacological thromboprophylaxis is warranted.