Assessment Of Outcome Measures Research Articles

Quality of Life Assessments Utilized in Vitiligo Clinical Trials

Introduction. Vitiligo is an acquired autoimmune disease associated with high psychosocial burden. As novel treatments are being developed in clinical trials, assessing vitiligo disease burden extends beyond physical manifestations. Including quality of life (QoL) measures in vitiligo clinical trials can better capture disease‐specific psychosocial concerns and facilitate cross comparisons amongst interventions. Objective. To determine the frequency and types of QoL measures utilized in vitiligo clinical trials and comment on how this has changed longitudinally. Methods. A search of vitiligo clinical trials using clinicaltrials.gov was conducted. Phase 2 and phase 3 trials published in English from January 2000 to July 2023 were eligible for this review. Characteristics of clinical trial parameters were compared to those of non‐QoL reporting clinical trials using Pearson’s χ2 tests (or Fisher’s if low n). Results. A total of 60 clinical trials were eligible for this review, of which 40% included a QoL measure in their study design. Phase 3 clinical trials (p = 0.002), larger (100+ participants) trials (p = 0.063), U.S. trials (p = 0.029), and pharmaceutical interventions (p = 0.022) were more likely to include QoL measures in their design. The number of clinical trials has been increasing over time, with 8 trials from 2000 to 2010, 32 total trials from 2011 to 2020, and 20 trials from 2021 to 2023. The most commonly used QoL measures were the Dermatology Life Quality Index (DLQI, 55.2%), Children′s Dermatology Life Quality Index (CDLQI, 13.8%), and Vitiligo‐specific quality of life instrument (VitiQoL, 13.8%). Over time, the VitiQoL and CDLQI have been used more frequently. Conclusion. Although vitiligo is associated with high psychological and emotional burden, less than half of vitiligo trials utilize QoL measures. The general dermatology QoL measures, namely the DLQI and CDLQI, are the most commonly used QoL assessments. As the number of clinical trials is increasing, vitiligo‐specific questionnaires may better capture unique vitiligo‐specific concerns. Standardizing the types of and implementation of QoL questionnaires in clinical trials can aid in assessing outcome measures across clinical trials worldwide and allow for better data interpretation, comparability, and clinical application of results.

The role of liver transplant for intrahepatic cholangiocarcinoma: the UK NHSBT liver advisory group pilot programme

Aim: Liver transplantation (LT) offers a potential curative treatment for non-metastatic intrahepatic cholangiocarcinoma (iCCA) in patients with chronic liver disease who are not amenable to liver resection (LR). Recent evidence suggests that cirrhotic patients with “very early” iCCA (single tumour, ≤ 2 cm) might benefit the most from LT, with a 5-year survival as high as 73%. In view of these developments, NHS Blood and Transplant’s Liver Advisory Group (LAG) established a Fixed Term Working Group (FTWG) to determine whether iCCA in patients with background cirrhosis should be considered for LT in the United Kingdom. Methods: The FTWG included cholangiocarcinoma/LT patient representatives, experts in cholangiocarcinoma surgery/oncology, LT surgery, hepatology, hepatobiliary radiology, hepatobiliary pathology, nuclear medicine, and representation from various national hepatobiliary/oncology and transplant professional bodies. The objective was to make recommendations on appropriate indications, patient selection criteria, referral criteria, radiological assessment, transplant listing pathways, data management, and overall quality assurance. Results: The FTWG recommended LT for very early iCCA in cirrhotics, who are otherwise not suitable for LR. In this paper, we summarise the selection criteria, patient pathways, referral framework, pre-transplant assessment criteria, outcome measures, and dissemination strategy for implementing this new indication for LT in the UK. Conclusion: The introduction and evaluation of this pilot programme is an important breakthrough for iCCA patients in the UK, marking a significant stride in the field of transplant oncology. The results of this service evaluation will describe the role of LT in iCCA and guide future programmes to optimise patient selection, management, and outcomes.

Are Bubbles the Future of Dysphagia Rehabilitation: A Systematic Review Analysing Evidence on the Use of Carbonated Liquids in Dysphagia Rehabilitation.

Dysphagia poses a huge health issue in our ageing population, impacting patients psychologically and through risk of aspiration, malnutrition and airway obstruction. The use of carbonated liquids to provide sensory enhancement as a tool to stimulate neuromuscular activity in dysphagia rehabilitation remains an area with limited research. This article reviews current evidence. A data search of PubMed, CINAHL, EMBASE and Cochrane was undertaken with set search terms. Abstracts were reviewed and selected by two clinicians according to inclusion criteria and papers were assessed using PRISMA methodology. Selected publications (1992-2022) involved a median of 23 participants with predominantly neurogenic dysphagia. Despite the differences in study designs all used videofluroscopy (VF) to assess outcome measures except Morishita et al. who used fiberoptic endoscopic evaluation of swallow (FEES). The studies were small scale but showed encouraging results. However, there was heterogeneity between results of specific outcome measures. One study surveyed taste which was overall positively received. There continues to remain limited evidence to direct the use of carbonated liquids in rehabilitation of dysphagia, however its role shows some promise. The heterogeneity of not just study designs but also study participants seems to be a primary barrier. Whilst evidence is encouraging, further prospective studies standardising patient cohorts, methodologies and quantitative outcome measures must be carried out. Longitudinal studies to look at the role of carbonated liquids in secretion management is another area of potential interest. In conclusion the use of carbonated liquid in dysphagia rehabilitation may have a potential role but without firm evidence-based research, successful use in clinical practice cannot be implemented.

Effectiveness and cost effectiveness of palliative care interventions in people with chronic heart failure and their caregivers: a systematic review

BackgroundChronic heart failure is a common condition, and its prevalence is expected to rise significantly over the next two decades. Research demonstrates the increasing multidimensional needs of patients and caregivers. However, access to palliative care services for this population has remained poor. This systematic review was to provide an evidence synthesis of the effectiveness and cost-effectiveness of palliative care interventions for people with chronic heart failure and their caregivers.MethodsRelevant publications were identified via electronic searches of MEDLINE, Embase, PsychInfo, CINAHL, CENTRAL and HMIC from inception to June 2019. Grey literature databases, reference list, and citations of key review articles were also searched. Quality was assessed using the Revised Cochrane Risk of Bias Tool.ResultsOf the 2083 records, 18 studies were identified including 17 having randomised controlled trial (RCT) designs and one mixed methods study with an RCT component. There was significant heterogeneity in study settings, control groups, interventions delivered, and outcome measures used. The most commonly assessed outcome measures were functional status (n = 9), psychological symptoms (n = 9), disease-specific quality of life (n = 9), and physical symptom control (n = 8). The outcome measures with the greatest evidence for benefit included general and disease-specific quality of life, psychological symptom control, satisfaction with care, physical symptom control, medical utilisation, and caregiver burden. Moreover, the methodological quality of these studies was mixed, with only four having an overall low risk of bias and the remaining studies either demonstrating high risk of bias (n = 10) or showing some concerns (n = 4) due to small sample sizes and poor retention. Only two studies reported on economic costs. Both found statistically significant results showing the intervention group to be more cost effective than the control group, but the quality of both studies was at high risk of bias.ConclusionsThis review supports the role of palliative care interventions in patients with chronic heart failure and their caregivers across various outcomes, particularly quality of life and psychological wellbeing. Due to the highly heterogeneous nature of palliative care interventions, it is not possible to provide definitive recommendations as to what guise palliative care interventions should take to best support the complex care of this population. Considerable future research, particularly focusing on quality of care after death and the caregiver population, is warranted.

Airflow grades, outcome measures and response to pulmonary rehabilitation in individuals after an exacerbation of severe chronic obstructive pulmonary disease

BackgroundIndividuals with COPD may be staged according to symptoms and exacerbation history (GOLD groups: A-D) and on airflow obstruction (GOLD grades: 1–4). Guidelines recommend pulmonary rehabilitation (PR) for these individuals, including those recovering from an exacerbation (ECOPD) ObjectiveTo evaluate whether in individuals with clinically severe COPD, recovering from an ECOPD, the effect size of an in-hospital PR program would be affected by airflow severity grades and assessed outcome measures. MethodsRetrospective, multicentre study. Participants were compared according to different GOLD airflow grades. In addition to the MRC dyspnoea scale, six-minute walking distance test and COPD assessment test (CAT), Barthel dyspnoea index (Bid), and Short Physical Performance Battery (SPPB) were assessed, evaluating the proportion of individuals reaching the minimum clinically important difference (MCID) (responders). ResultsData of 479 individuals, completing the program were evaluated. Most of the participants were allocated in GOLD grades 4, (57.6%) and 3 (22.1%). All outcome measures significantly improved after PR (p < 0.05), without any significant difference in the proportion of responders in any measure. Conclusionsin individuals with severe COPD, recovering from ECOPD the success rate of PR does not depend on airflow severity, or outcome measure assessed. In addition to the most used outcome measures, also Bid and SPPB are sensitive to PR.

Transcatheter Aortic Valve Implantation Versus Surgical Aortic Valve Replacement in Low-risk Patients: A Meta-Analysis Based on a 2-Year Follow-Up.

Previous studies have shown that transcatheter aortic valve implantation is the best alternative therapy to surgical aortic valve replacement in high-risk surgical patients with aortic stenosis. However, it is not clear whether transcatheter aortic valve implantation can be utilized in low-risk surgical patients with aortic stenosis. This studyaimed to evaluate the safety and efficacy of transcatheter aortic valve implantation in low-risk patients. From the outset of our initiative until April 2022, PubMed, EMBASE, and the Cochrane database were thoroughly searched, yielding the selection of 3 randomized controlled trials including 2644 patients with aortic stenosis, to assess outcome measures at distinct follow-up time. The mean Society of Thoracic Surgeons Predicted Risk of Mortality score of patients was 2.2. At the 30-day and 1-year follow-up, transcatheter aortic valve implan- tation was associated with a lower incidence of all-cause mortality, cardiovascular mor- tality, acute kidney injury (stage 2 or 3), life-threatening or significant bleeding, and new atrial fibrillation but an increased risk of permanent pacemaker implantation. At the 2-year follow-up, transcatheter aortic valve implantation only had an advantage in new atrial fibrillation (relative risk, 0.27; 95% CI, 0.14-0.51; P &lt; .0001), with no significant differ- ence in all-cause mortality or cardiovascular mortality. For low-risk surgical patients with aortic stenosis, compared to surgical aortic valve replacement, transcatheter aortic valve implantation was associated with lower all-cause mortality at 30-day follow-up and lower cardiovascular mortality at 1-year follow-up. Except for the advantages in new atrial fibrillation, transcatheter aor- tic valve implantation had no significant impact on mortality at 2-year follow-up.

Patient-Reported Outcome Measures for care quality assessment of a breast cancer Care Pathway

Abstract Background Breast Cancer (BC) is the most common type of cancer among women in Europe, accounting for around 28% of newly diagnosed female cancers. A clinical Pathway (CP) is an effective tool able to deliver high quality care especially if linked to a Monitoring System. Still, it is essential to integrate them with the patient’s perspective according to Value-Based Care. This paper aims to define and test a tool for care quality assessment in patients with breast cancer, in a value-based perspective. Methods A scoping search of the main databases (PubMed, Scopus, Web of Science) and official websites of institutions and organizations (AIOM, CIPOMO, EORTC, ICHOM, Istat) was carried out. A Delphi survey was conducted to assess the tool, according to four criteria (general relevance, evidence-based, measurability, actionability). We only included indicators that achieved strong agreement. Time-points for data collection were defined and validated in relation to the different steps of the CP. Results The final tool consists of 21 questions coming from the following sources: BREAST-Q and EORT QLQ-BR23 questionnaire. BREAST-Q’s questions, have to be administered at T0 (first medical contact) and T12 (follow-up), while the EORT QLQ-BR23’s questions have to be administered at T0 (first medical contact) T Surgery, T6 (follow-up) and T12 (follow-up). The survey will be administered to a sample of 152 BC patients (MOE 5%; CL 95%) undergoing surgery in the period June-September 2021. Conclusions The present tool will give a quick view of Value provided by the CP to these patients. In light of the high volume of patients treated at our center (1008 breast cancer surgery hospitalisations in 2020), this study acquires further public health relevance. Additionally, this approach can be extended for further evaluation of other CPs. Key messages • We assess care quality for Breast Cancer patients in a large hospital in Italy through Patient-Reported Outcome Measures, based on an evidence-based, Value-based, Delphi-validated tool. • Assessing Value brought to patients with Breast Cancer gives a unique perspective on care quality assessment and paves the way for further quality improvements and extension to other health issues.

Parkinsonics: A Randomized, Blinded, Cross-Over Trial of Group Singing for Motor and Nonmotor Symptoms in Idiopathic Parkinson Disease.

Introduction Parkinson's disease (PD) frequently causes communication difficulties due to various voice impairments and there are few treatment options for vocal/communication complaints. We assessed the effects of weekly group singing on PD patients' objective vocal and motoric function, cognition, mood, self-efficacy, and quality of life. Methods Thirty-two participants were randomly assigned to either a singing group or a facilitated discussion group weekly over 12 weeks. After 12 weeks, participants crossed over for an additional 12 weeks. Evaluations were performed at baseline and every six weeks for 30 weeks. Objective voice measures included volume/loudness (decibels), held vowel duration, jitter, shimmer, and harmonic-to-noise ratio. Additional outcome measures included patient-centered quality of life, voice-related quality of life, MDS-UPDRS, Montreal Cognitive Assessment, and questionnaires assessing depression, self-efficacy, and overall well-being. Results Twenty-six participants (16 M/10F; Hoehn & Yahr stage 2.3 (range 2–3); and age 68.6 (55–89)) completed the study. Across participants in both groups (intention-to-treat analyses), there was significant improvement from baseline in average loudness on the Cookie Theft picture description at 24 weeks (end of interventions), corresponding with improved minimal reading volumes at 24 weeks and 30 weeks (end of study). Similarly, there were improvements in minimal loudness on Rainbow passage reading at 24 and 30 weeks. There were improvements observed in the Emotional Well-Being (mean delta −12.7 points, p = 0.037) and Body Discomfort (mean delta −18.6 points, p = 0.001) domains of the PDQ-39 from baseline to week 24 in the overall cohort and greater improvement in the Communication domain for Group S than Group D after 12 weeks of singing (delta −12.9 points, p = 0.016). Baseline differences between the participant groups (age, gender, Hoehn & Yahr stage, and several voice loudness measures) and observed improvements during the weekly discussion group period limited our ability to attribute all of the above results specifically to singing (per-protocol analyses). No significant changes in other assessed outcome measures were found. Conclusions Weekly group singing may improve some aspects of conversational voice volume and quality of life in PD. Some improvements were sustained at least six weeks after interventions ended. Further investigations of the mechanism of benefit and randomized controlled studies (without crossover) to assess the longitudinal effects of singing in PD are necessary.

1621P miRNA signature as early predictor of hyponatremia and survival in lung cancer: Preliminary results from the ISA retrospective study

Syndrome of inappropriate antidiuresis (SIAD) in cancer is the main cause of hyponatremia and is associated with poorer survival, hospitalization and health costs in lung cancer (LC) patients (pts). Micro-RNAs (miRNAs) represent promising biomarkers with therapeutic potential. Our retrospective study aims to assess a miRNA signature to discriminate hyponatremia and predict clinical outcome in LC pts. We performed a miRNA sequencing in 20 FFPE tumor samples of 10 eunatremic and 10 hyponatremia-developing LC pts through Illumina NextSeq500 sequencer. Pts’ records were reviewed to assess outcome measures. The differential miRNA expression profile was analyzed between the two groups. miRNAs with |log2(fold change)|>0.58 and p<0.05 were considered statistically significant and correlated with survival outcomes, relapse-free survival (RFS), progression-free survival (PFS) and overall survival (OS), using three Cox multivariate regression models. 12 miRNAs were differentially expressed at diagnosis between hypo- and eunatremic pts at univariate analysis (p<0.05). At multivariate analysis, miR-147b-3p (median expression 10.8 vs 92.6, p=0.025), miR-147b-5p (2.6 vs 15.9, p=0.002), miR-937-3p (3.4 vs 14.6, p=0.039) and miR-146a-5p (6106.6 vs 16601.4, p=0.035) were significantly downregulated in hypo- vs eunatremic pts. Similarly, miR-539-3p (3.4 vs 14.9, p=0.083) and miR-625-3p (97.8 vs 308.9, p=0.061) showed a trend towards significance for downregulation. Contrarily, miR-190a-5p (304.2 vs 179.5, p=0.006) and miR-32-3p (7 vs 3, p=0.017) were upregulated in the hyponatremic vs control group. Looking at survival outcomes, the upregulation of miR-937-3p (p=0.042), miR-190a-5p (p=0.001) and miR-9-3p (p=0.035) was associated with worse OS, while the upregulation of miR-32-3p was associated with improved OS (p=0.010) and RFS (p=0.064). Our results reveal new insights into the role of vasopressin pathways in LC. These miRNAs appear as valuable early predictors of hyponatremia and their expression is associated with a significant impact on pts’ survival. Further investigations, including our prospective study, are warranted to provide advances in SIAD management.

Current clinical practice in managing somatosensory impairments and the use of technology in stroke rehabilitation

Stroke-induced somatosensory impairments seem to be clinically overlooked, despite their prevalence and influence on motor recovery post-stroke. Interest in technology has been gaining traction over the past few decades as a promising method to facilitate stroke rehabilitation. This questionnaire-based cross-sectional study aimed to identify current clinical practice and perspectives on the management of somatosensory impairments post-stroke and the use of technology in assessing outcome measures and providing intervention. Participants were 132 physiotherapists and occupational therapists currently working with stroke patients in public hospitals and rehabilitation centres in Singapore. It was found that the majority (64.4%) of the therapists spent no more than half of the time per week on somatosensory interventions. Functional or task-specific training was the primary form of intervention applied to retrain somatosensory functions in stroke survivors. Standardised assessments (43.2%) were used less frequently than non-standardised assessments (97.7%) in clinical practice, with the sensory subscale of the Fugl-Meyer Assessment being the most popular outcome measure, followed by the Nottingham Sensory Assessment. While the adoption of technology for assessment was relatively scarce, most therapists (87.1%) reported that they have integrated technology into intervention. There was a common agreement that proprioception is an essential component in stroke rehabilitation, and that robotic technology combined with conventional therapy is effective in enhancing stroke rehabilitation, particularly for retraining proprioception. Most therapists identified price, technology usability, and lack of available space as some of the biggest barriers to integrating robotic technology in stroke rehabilitation. Standardised assessments and interventions targeting somatosensory functions should be more clearly delineated in clinical guidelines. Although therapists were positive about technology-based rehabilitation, obstacles that make technology integration challenging ought to be addressed.

Deriving stair-climbing performance outcome measures using the smartphone barometer: Results of an algorithm development study

As we seek to gain richer insights to understand intervention effects, and increasingly decentralise aspects of clinical trials to simplify participation, there is a growing interest in leveraging wearables and sensors to generate novel and informative clinical outcome measures for at-home assessment. The sensors embedded within smartphone technology provide one approach to capture of this data, and may be particularly useful when patients are already using mobile devices for at-home capture of other clinical trials data, such as patient-reported outcomes.We describe the results of an initial algorithm development study to determine whether the atmospheric pressure data provided by an onboard smartphone sensor is sufficiently informative to enable detection of a small height gain, such as that achieved during a short stair climb performance test. We were able to sufficiently distinguish height changes of 0.6 m in indoor conditions, representing around 4 stairs on an average staircase. This suggests that the smartphone barometer may indeed be suitable for inclusion within future work developing a stair-climbing performance outcome test instrumented using a mobile application.

Outcome measurement instruments in Rett syndrome: A systematic review.

The aim of the study was to identify and characterize outcome measures for objective and subjective assessment in persons with Rett syndrome (RS). A systematic review was conducted consulting the EBSCO, Cochrane, Web of Science, Scielo, MEDLINE and PsycINFO databases for published studies describing the use of patient-reported outcome measures (PROMs) and other outcome measures in persons with RS. Validation studies and observational studies were included. The PROMs were first described, and then the measurement properties were evaluated using predefined criteria according to the COnsensus-based Standards for the selection of health Measurement Instruments (COSMIN). The outcome measures were then grouped according to the International Classification of Functioning, Disability and Health (ICF) to establish a relationship between outcome measures and ICF domains. Twenty out of 2327 articles were appraised, and seventeen different outcome measures were identified and described. Ten outcome measures corresponded to evaluation questionnaires, while the remaining seven assessed functional outcomes: walking distance, physical activity level and ability to interact visually. A relation between these outcome measures that assess RS and the ICF allows understanding that most of the instruments (fifteen) include the assessment of activity limitations. The findings of this study seem to be promising for their use by clinicians and researchers, although they have methodological limitations. The accuracy and quality of these individual outcome measures should continue to be assessed in an attempt to gather a consensus on the best tools used in RS.

Post-Viral Polyarthritis

Background: Viral infection is the infection caused by a virus in the human body. The immune response to a virus infection is attacking the body’s own cells. This causes stress and inflammation of tissues in the body and damage to the cells. Polyarthritis is one of the most common complications caused by viral infection. Post viral polyarthritis mainly affects the large joint such as the knee, but can affect several joints such as ankle, wrist. The full recovery time is still not sure, some studies have shown that it usually takes six weeks or it can even last for months or years. Case Description: A 15-year-old male complains of fever with dry cough for 2 days followed by Multiple joint pain and soft tissue pain since 2 months. Swelling was also noticed in both wrist and knee for a week. For assessment outcome measures like VAS, MMT, ROM were taken prior to the treatment. Patient underwent a 4 weeks physiotherapy rehab program and showed marked improvement in the pain, range of motion, muscle strength and functional activities. Result: When the post-assessment was done, patient showed significant improvement in the pain relief, showed improvement in range of motion of the joints, increase in the muscle strength and noticeable improvement in functional capacity. Conclusion: This concluded that starting physiotherapy management in the sub-acute stage of the condition is effective in reducing pain increasing mobility and increasing the strength and endurance.

HPR9 An Updated Review of FDA Warning and Untitled Letters for Clinical Outcome Assessment Claims between 2013 and 2020

The FDA communicates violations to manufacturers through warning letters (WLs) and untitled letters (ULs). Symonds et.al. (2006), reported that in 2006-2012, 19% (n = 41) of the 213 issued letters contained information about patient-reported outcome (PRO) infringements, with the most common being “PRO measure was not fit for purpose”. This study updates the prior review to 2013-2020 and expands it to clinical outcome assessment (COA) measures.

Is There a Difference in Fear-Avoidance, Beliefs, Anxiety and Depression Between Post-Surgery and Non-Surgical Persistent Spinal Pain Syndrome Patients?

IntroductionPatients with post-surgery persistent spinal pain syndrome (PSPS) or non-surgical PSPS might be affected by sustained fear-avoidance beliefs (FAB), anxiety and depression. In this scenario, this study aimed to describe those aspects in patients with post-surgery PSPS and non-surgical PSPS.MethodsThis study included patients with PSPS, and non-surgical PSPS, over 18 years, with quarterly evaluations at the Chronic Pain Clinic. After evaluation, demographic and clinical characteristics were obtained. The Beck Depression Inventory-II, Beck Anxiety Inventory, Douleur neuropathique 4 questions, Visual Analog Pain Scale, and Fear-Avoidance Beliefs Questionnaire–Brazilian Version (FABQ-Brazil) were used to evaluate psychological aspects.ResultsForty-six patients were included, 23 patients with post-surgery PSPS and 23 with non-surgical PSPS. Both groups had high scores in the physical and work domains of the FABQ, high rates of absenteeism and most patients in these groups had moderate-to-severe neuropathic pain and some degree of anxiety and/or depression. The groups showed no statistically significant difference (p > 0.05) when comparing all questionnaires.DiscussionThis is one of the first studies to evaluate FAB and other associated psychological factors, such as anxiety and depression, in patients with post-surgery PSPS in a follow-up several years after surgery and compare with patients diagnosed with non-surgical PSPS. In this study, most patients in both groups had high scores in the FABQ domains, not having statistically relevant difference between groups.ConclusionEven though there was no statistically relevant difference between the PSPS patient with or without surgical history in terms of the assessed outcome measures, the described scores for fear-avoidance beliefs, pain, anxiety and depression were high, showing an interference in the daily life activities of those patients.

The feasibility and acceptability of a home-based, virtual exercise intervention for older patients with hepatocellular carcinoma: protocol for a non-randomised feasibility study (TELEX-Liver Cancer)

BackgroundThe number of incident cases and deaths from primary liver cancer, predominantly hepatocellular carcinoma (HCC), has increased markedly in the last two decades. HCC is generally diagnosed at an advanced stage, and most new cases are in people aged over 70 years with age-related comorbidities. Treatment options are often limited, with most patients receiving palliative treatment or supportive care only. As a consequence, maintaining quality of life (QoL) through symptom management is critically important and is a core objective of clinical care. Strong evidence supports the efficacy of supervised exercise training for addressing certain cancer-related symptoms, including QoL, physical function, and fatigue. However, there are many barriers to implementing supervised exercise programmes within cancer care pathways, including economic pressures on healthcare systems and personal barriers for patients. Recent advances in technology allow patients to exercise at home under the ‘virtual’ supervision of an exercise professional through videoconferencing software (termed ‘telehealth exercise’). Despite its potential, there are uncertainties relating to the feasibility, acceptability, and safety of telehealth exercise in people living with HCC.MethodsThis is a protocol for a prospective, single-centre, single-arm, pretest-posttest feasibility trial. We aim to recruit 20 patients aged 60 years or older who have received treatment for HCC and are undergoing routine clinical monitoring. Patients will be invited to take part in two online, home-based, group exercise sessions per week for 10 consecutive weeks. The ‘virtual’ exercise sessions will be delivered in real time by an exercise professional through videoconferencing software. Each session will comprise 30 min of aerobic and resistance exercise performed at a moderate intensity, as guided by the 10-point Borg rating of perceived exertion scale. Feasibility outcomes include recruitment, retention, adherence, intervention fidelity, and safety. Acceptability of the intervention will be assessed using a mixed-methods approach via monthly online surveys and an exit telephone interview. Physical function, accelerometry-measured physical activity, mid-upper arm circumference, and patient-reported outcome measures (PROMS) will be assessed before and after the intervention to determine the feasibility of assessing outcome measures. Physical function outcomes include the short physical performance battery and Liver Frailty Index. PROMS include the Functional Assessment of Cancer Therapy-Hepatobiliary questionnaire, Functional Assessment of Chronic Illness Therapy-Fatigue questionnaire, Activities-specific Balance Confidence scale, Hospital Anxiety and Depression Scale, and the Godin Leisure-Time Exercise Questionnaire.DiscussionThis mixed-methods study will address uncertainties relating to the feasibility and acceptability of delivering live, online, home-based, group exercise sessions to patients with HCC. The findings will inform whether any modifications are required to refine and optimise the intervention, and the assessment of outcome measures will provide information on the likely size and variability of intervention effects. Collectively, the data generated will inform the design of a subsequent, adequately powered, randomised controlled trial to evaluate the efficacy of the telehealth exercise intervention.Trial registrationISRCTN14411809

P266 Secukinumab provides clinical improvements in patients with active oligoarticular psoriatic arthritis: results from a pooled analysis of five phase 3 studies

Abstract Background/Aims Evidence for the efficacy of biologics in patients with oligoarticular psoriatic arthritis (PsA) is limited. We evaluated the efficacy of the interleukin 17 inhibitor secukinumab in patients with oligoarticular PsA pooled from 5 phase 3 studies. Methods This post hoc analysis included patients with oligoarticular PsA from the FUTURE 2-5 and MAXIMISE trials (NCT01649375, NCT01989468, NCT02294227, NCT02404350, and NCT02721966). Oligoarticular PsA was defined as the presence of 1-4 tender joints and 1-4 swollen joints at baseline as measured by the tender joint count of 78 joints (TJC78) and the swollen joint count of 76 joints (SJC76), respectively. Patients were pooled by treatment received at week 12 and week 52 in the following groups: week 12 (secukinumab 300 mg, secukinumab 150 mg with or without loading dose [LD], or placebo) and week 52 (any secukinumab 300 mg or any secukinumab 150 mg). Efficacy was assessed by the proportion of patients achieving 50% improvement in Disease Activity in Psoriatic Arthritis (DAPSA50), DAPSA75, DAPSA-based low disease activity (LDA; DAPSA ≤14) and remission (REM; DAPSA ≤4), resolution of TJC78, resolution of SJC76, and a ≥ 0.35-point improvement in the Health Assessment Questionnaire-Disability Index score. Descriptive statistics are provided for each endpoint. Logistic regression analysis was used to identify potential predictors of DAPSA response at weeks 12 and 52; multivariate logistic regression analyses were performed thereafter. Results 84 patients were included: 48 with active PsA from the FUTURE 2-5 trials and 36 with active PsA and axial manifestations from the MAXIMISE trial. Demographics and baseline disease characteristics were comparable across groups. At week 12, notable improvements were observed for the secukinumab 300 and 150 mg versus placebo groups across all the assessed outcome measures and were sustained or increased through week 52. More than 78% of patients who received secukinumab 300 mg or secukinumab 150 mg achieved LDA or REM at week 12. More than 90% of patients who received any secukinumab 300 mg or secukinumab 150 mg achieved LDA or REM at week 52. At week 52, a higher proportion of patients who received any secukinumab 300 mg versus any secukinumab 150 mg achieved higher hurdle endpoints of DAPSA75 (58.1% versus 43.6%, respectively) and DAPSA REM (61.3% versus 37.5%, respectively). At week 12, younger age was a predictor for the achievement of DAPSA LDA or REM and the achievement of DAPSA50, while lower baseline SJC was a predictor of the achievement of DAPSA REM (P &amp;lt; 0.05). No predictors were identified at week 52. Conclusion Secukinumab provides greater improvements versus placebo across a number of disease activity and physical function outcome measures in patients with oligoarticular PsA at week 12, with sustained responses through week 52. Disclosure L.C. Coates: Honoraria; AbbVie, Amgen, Celgene, Galapagos, Gilead, Janssen, Eli Lilly, Novartis, Pfizer, UCB. Grants/research support; AbbVie, Celgene, Eli Lilly, Novartis, Pfizer. A. Ogdie: Consultancies; Amgen, AbbVie, BMS, Celgene, Novartis, Lilly, Janssen, Gilead, Pfizer, UCB. Grants/research support; National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases, Rheumatology Research Foundation, National Psoriasis Foundation, Amgen, Pfizer, Novartis. D.D. Gladman: Consultancies; AbbVie, Amgen, Bristol Myers Squibb, Celgene, Galapagos, Gilead, Janssen, Eli Lilly, Novartis, Pfizer, UCB. Grants/research support; AbbVie, Amgen, Celgene, Eli Lilly, Novartis, Pfizer, UCB. E. Pournara: Shareholder/stock ownership; Novartis. Other; Employee of Novartis. X. Meng: Other; Employee of Novartis. B. Parikh: Other; Employee of Novartis. P.J. Mease: Consultancies; AbbVie, Amgen, Boehringer Ingelheim, Bristol Myers Squibb, Celgene, Galapagos, Gilead, GlaxoSmithKline, Janssen, Eli Lilly, Novartis, Pfizer, Sun Pharma, UCB. Member of speakers’ bureau; AbbVie, Amgen, Janssen, Eli Lilly, Novartis, Pfizer, UCB. Grants/research support; AbbVie, Amgen, Bristol Myers Squibb, Celgene, Gilead, Janssen, Eli Lilly, Novartis, Pfizer, Sun Pharma, UCB.

Psychometric Evaluation of the Disabilities of the Arm, Shoulder and Hand Outcome Questionnaire for Brachial Plexus Injury Using Rasch Analysis.

Background: Disabilities of the Arm, Shoulder and Hand (DASH) questionnaire is the most widely used patient-reported outcome measure (PROM) for assessment of upper extremity disability assessment. However, DASH is a multidimensional measurement with different difficulty levels and ratio apportionment of the items categorised by ordinal scale. This has caused a misinterpretation of the total disability scores. We created a modified DASH adapted to the Rasch model. The aim of this study is to compare the functional recovery and quality of life (QOL) improvement and to assess the validity of the original DASH and modified DASH between C56/C567, C5-8 and total types of brachial plexus injury (BPI) following surgical reconstruction. Methods: A total of 183 BPI patients who underwent reconstructive surgery were evaluated for functional recovery using the range of motion and power of the affected limb, and improvement in QOL with DASH. The collected data were analysed using Rasch measurement theory to detect the misfit items. The original and modified DASH were compared under the three different types of BPI after item reduction by removing the misfit items. Results: There were significant differences in functional recovery between three types of palsy. However, PROM using DASH score with or without misfit items (12 items) did not show any significant differences. Conclusions: DASH is not suitable for comparison of upper extremity disabilities even after being corrected mathematically due to the inclusion of items from many different domains unequally. Therefore, each item of the function (with or without compensation of the uninjured hand), pain and impact to the patients should be evaluated separately. Level of Evidence: Level IV (Prognostic).

Development and validation of the health-related quality of life instrument for Chinese infertile couples: a mixed-methods study protocol

BackgroundInfertility and its treatment have negative impacts on a couple’s marital relationship, sexual life, psychological state and interpersonal relationships, causing personal distress. Health-related quality of life (HRQoL) has become an important component of health outcomes. HRQoL instruments developed in western culture are not always appropriate for use in China due to cultural differences. Probably due to the unique concept of fertility in China, infertility patients can be looked down upon and the family may feel shameful. This study aims to develop a HRQoL instrument for infertile couples based on the Chinese social and cultural setting.MethodsComplementary mixed methods will be used to develop a new HRQoL instrument for Chinese infertile couples. The study consists of four stages: the first stage will involve a systematic review and qualitative interviews to construct draft candidate items. In the second stage, quantitative research [e.g., exploratory factor analysis (EFA), item response theory (IRT)] and cognitive interviews will be used for item selection. The third stage will be instrument validation, in which classical test theory (CTT) and IRT will be applied. In the final stage, the minimal clinically important difference (MCID) will be calculated by using distribution-based methods and anchor-based methods (e.g., logistic regression, receiver operating characteristic curve).DiscussionThe new HRQoL instrument for Chinese infertile couples will be developed, which will provide a standard and effective HRQoL instrument in clinical outcome assessment and health outcome measurement.

Maternal, Perinatal and Neonatal Outcomes of Triplet Pregnancies According to Chorionicity: A Systematic Review of the Literature and Meta-Analysis.

Triplet pregnancies are rare events that affect approximately 93 in 100,000 deliveries in the world, especially due to the increased use of assisted reproductive techniques and older maternal age. Triplet pregnancies are associated with a higher risk of fetal and maternal morbidity and mortality compared to twins and singletons. Chorionicity has been proposed as a major determinant of perinatal and maternal outcomes in triplet pregnancies, although further evidence is needed to clarify the extent and real influence of this factor. Thus, the aim of this study was to conduct a systematic review of the literature and a meta-analysis of the maternal and perinatal outcomes of triplet pregnancies, evaluating how chorionicity may influence these results. A total of 46 studies with 43,653 triplet pregnancies and 128,145 live births were included. Among the main results of our study, we found a broad spectrum of fetal and maternal complications, especially in the group of monochorionic and dichorionic pregnancies. Risk of admission to NICU, respiratory distress, sepsis, necrotizing enterocolitis, perinatal and intrauterine mortality were all found to be higher in non-TCTA pregnancies than in TCTA pregnancies. To date, our meta-analysis includes the largest population sample and number of studies conducted in this field, evaluating a wide variety of outcome measures. The heterogeneity and retrospective design of the studies included in our research represent the main limitations of this review. More evidence is needed to fully assess outcome measures that could not be studied in this review due to scarcity of publications or insufficient sample size.