scholarly journals P126 Systematic review of outcome measures used in narcolepsy clinical trials

2021 ◽  
Vol 2 (Supplement_1) ◽  
pp. A62-A62
Author(s):  
A Schokman ◽  
Y Bin ◽  
D Naehrig ◽  
J Cheung ◽  
K Kairaitis ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Primary Outcome ◽  
Treatment Effectiveness ◽  
Treatment Success ◽  
Population Data ◽  
Primary Outcome Measure ◽  
Measurement Instruments ◽  
Health Measurement ◽  
Preliminary Results ◽  
Randomised Controlled

Abstract Introduction Treatment of the sleep disorder narcolepsy is primarily symptomatic. With limited population data and insufficient systems to capture patient data, a greater emphasis has been placed on the patient perspective in determining satisfaction and treatment success in clinical settings. While Randomised Controlled Trials (RCTs) are considered the gold standard when determining treatment effectiveness, how effectiveness is measured in a narcolepsy population remains unknown. Therefore, we aimed to explore the outcome measures used in narcolepsy RCTs, and further evaluate the adequacy of any self-reported outcome measures used. Methods Electronic databases (MEDLINE, EMBASE, CINAHL, SCOPUS, PSYCHINFO) and clinicaltrials.gov were searched in April 2020, with primary and secondary outcomes extracted from eligible studies. Self-reported outcome measures used at least once as a primary outcome measure were further explored, with validation studies of these systematically searched for (using the above databases) and analysed using the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) Risk of Bias tool. Results We identified 82 published RCTs involving patients with narcolepsy, of which 35 objective and 46 subjective measures were used. Of these, the Maintenance of Wakefulness Test (n=29), Epworth Sleepiness Scale (ESS) (n=14) and Multiple Sleep Latency Test (n=9) were the most frequently selected primary outcome measures. While our analysis of self-reported outcome measures is ongoing, preliminary results suggest that few have been adequately validated in a narcolepsy-specific population. Discussion Our preliminary results identify a high level of heterogeneity between outcome measures used in RCTs, thus difficult to draw conclusions and compare interventions.

scholarly journals Patient-reported outcome measures in community-acquired pneumonia: a systematic review of application and content validity

2019 ◽  
Vol 6 (1) ◽  
pp. e000398 ◽  
Author(s):  
Melanie Lloyd ◽  
Emily Callander ◽  
Amalia Karahalios ◽  
Lucy Desmond ◽  
Harin Karunajeewa
Keyword(s):  
Outcome Measures ◽  
Content Validity ◽  
Short Form ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Community Acquired Pneumonia ◽  
Current Evidence ◽  
Measurement Instruments ◽  
Health Measurement ◽  
Patient Reported

IntroductionPatient-reported outcome measures (PROMs) are a vital component of patient-centred care. Community-acquired pneumonia (CAP) is a significant contributor to morbidity, mortality and health service costs globally, but there is a lack of consensus regarding PROMs for this condition.MethodsWe searched MEDLINE, EMBASE and Cochrane Collaboration for studies, both interventional and observational, of adult recovery from CAP that applied at least one validated PROM instrument and were published before 31 December 2017. The full text of included studies was examined and data collected on study design, PROM instruments applied, constructs examined and the demographic characteristics of the populations measured. For all CAP-specific PROM instruments identified, content validity was assessed using the COnsensus based Standards for selection of health Measurement INstruments guidelines (COSMIN).ResultsForty-two articles met the inclusion criteria and applied a total of 17 different PROM instruments including five (30%) classified as CAP specific, six (35%) as generic and six (35%) that measured functional performance or were specific to another disease. The 36-Item Short Form Survey (SF-36) was the most commonly used instrument (15 articles). Only one of 11 (9%) patient cohorts assessed using a CAP-specific instrument had a mean age ≥70 years. The CAP-Sym and CAP-BIQ questionnaires had sufficient content validity, though the quality of evidence for all CAP-specific instruments was rated as very low to low.DiscussionPROM instruments used to measure recovery from CAP are inconsistent in constructs measured and have frequently been developed and validated in highly selective patient samples that are not fully representative of the hospitalised CAP population. The overall content validity of all available CAP-specific instruments is unclear, particularly in the context of elderly hospitalised populations. Based on current evidence, generic health instruments are likely to be of greater value for measuring recovery from CAP in this group.

scholarly journals Computerised cognitive–behavioural therapy for adults with intellectual disability: randomised controlled trial

2017 ◽  
Vol 211 (2) ◽  
pp. 95-102 ◽  
Author(s):  
Patricia Cooney ◽  
Catherine Jackman ◽  
David Coyle ◽  
Gary O'Reilly
Keyword(s):  
Intellectual Disability ◽  
Randomised Controlled Trial ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Controlled Trial ◽  
Behavioural Therapy ◽  
Primary Outcome Measure ◽  
Cognitive Behavioural ◽  
Randomised Controlled

BackgroundDespite the evidence base for computer-assisted cognitive–behavioural therapy (CBT) in the general population, it has not yet been adapted for use with adults who have an intellectual disability.AimsTo evaluate the utility of a CBT computer game for adults who have an intellectual disability.MethodA 2 × 3 (group × time) randomised controlled trial design was used. Fifty-two adults with mild to moderate intellectual disability and anxiety or depression were randomly allocated to two groups: computerised CBT (cCBT) or psychiatric treatment as usual (TAU), and assessed at pre-treatment, post-treatment and 3-month follow-up. Forty-nine participants were included in the final analysis.ResultsA significant group x time interaction was observed on the primary outcome measure of anxiety (Glasgow Anxiety Scale for people with an Intellectual Disability), favouring cCBT over TAU, but not on the primary outcome measure of depression (Glasgow Depression Scale for people with a Learning Disability). A medium effect size for anxiety symptoms was observed at post-treatment and a large effect size was observed after follow-up. Reliability of Change Indices indicated that the intervention produced clinically significant change in the cCBT group in comparison with TAU.ConclusionsAs the first application of cCBT for adults with intellectual disability, this intervention appears to be a useful treatment option to reduce anxiety symptoms in this population.

scholarly journals Graduated compression stockings as adjuvant to pharmaco-thromboprophylaxis in elective surgical patients (GAPS study): randomised controlled trial

BMJ ◽  
2020 ◽  
pp. m1309 ◽  
Author(s):  
Joseph Shalhoub ◽  
Rebecca Lawton ◽  
Jemma Hudson ◽  
Christopher Baker ◽  
Andrew Bradbury ◽  
...  
Keyword(s):  
Venous Thromboembolism ◽  
High Risk ◽  
Confidence Interval ◽  
Outcome Measures ◽  
Lower Limb ◽  
Primary Outcome ◽  
Elective Surgery ◽  
Compression Stockings ◽  
Graduated Compression Stockings ◽  
Randomised Controlled

AbstractObjectivesTo investigate whether the use of graduated compression stockings (GCS) offers any adjuvant benefit when pharmaco-thromboprophylaxis is used for venous thromboembolism prophylaxis in patients undergoing elective surgery.DesignOpen, multicentre, randomised, controlled, non-inferiority trial.SettingSeven National Health Service tertiary hospitals in the United Kingdom.Participants1905 elective surgical inpatients (≥18 years) assessed as being at moderate or high risk of venous thromboembolism were eligible and consented to participate.InterventionParticipants were randomly assigned (1:1) to receive low molecular weight heparin (LMWH) pharmaco-thromboprophylaxis alone or LMWH pharmaco-thromboprophylaxis and GCS.Outcome measuresThe primary outcome was imaging confirmed lower limb deep vein thrombosis with or without symptoms, or pulmonary embolism with symptoms within 90 days of surgery. Secondary outcome measures were quality of life, compliance with stockings and LMWH, lower limb complications related to GCS, bleeding complications, adverse reactions to LMWH, and all cause mortality.ResultsBetween May 2016 and January 2019, 1905 participants were randomised. 1858 were included in the intention to treat analysis (17 were identified as ineligible after randomisation and 30 did not undergo surgery). A primary outcome event occurred in 16 of 937 (1.7%) patients in the LMWH alone group compared with 13 of 921 (1.4%) in the LMWH and GCS group. The risk difference between the two groups was 0.30% (95% confidence interval −0.65% to 1.26%). Because the 95% confidence interval did not cross the non-inferiority margin of 3.5% (P<0.001 for non-inferiority), LMWH alone was confirmed to be non-inferior.ConclusionsFor patients who have elective surgery and are at moderate or high risk of venous thromboembolism, administration of pharmaco-thromboprophylaxis alone is non-inferior to a combination of pharmaco-thromboprophylaxis and GCS. These findings indicate that GCS might be unnecessary in most patients undergoing elective surgery.Trial registrationISRCTN13911492.

scholarly journals Day hospital versus intensive out-patient mentalisation-based treatment for borderline personality disorder: multicentre randomised clinical trial

2019 ◽  
Vol 216 (2) ◽  
pp. 79-84 ◽  
Author(s):  
Maaike L. Smits ◽  
Dine J. Feenstra ◽  
Hester V. Eeren ◽  
Dawn L. Bales ◽  
Elisabeth M. P. Laurenssen ◽  
...  
Keyword(s):  
Borderline Personality Disorder ◽  
Personality Disorder ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Borderline Personality ◽  
Treatment Intensity ◽  
Day Hospital ◽  
Secondary Outcomes

BackgroundTwo types of mentalisation-based treatment (MBT) have been developed and empirically evaluated for borderline personality disorder (BPD): day hospital MBT (MBT-DH) and intensive out-patient MBT (MBT-IOP). No trial has yet compared their efficacy.AimsTo compare the efficacy of MBT-DH and MBT-IOP 18 months after start of treatment. MBT-DH was hypothesised to be superior to MBT-IOP because of its higher treatment intensity.MethodIn a multicentre randomised controlled trial (Nederlands Trial Register: NTR2292) conducted at three sites in the Netherlands, patients with BPD were randomly assigned to MBT-DH (n = 70) or MBT-IOP (n = 44). The primary outcome was symptom severity (Brief Symptom Inventory). Secondary outcome measures included borderline symptomatology, personality functioning, interpersonal functioning, quality of life and self-harm. Patients were assessed every 6 months from baseline to 18 months after start of treatment. Data were analysed using multilevel modelling based on intention-to-treat principles.ResultsSignificant improvements were found on all outcome measures, with moderate to very large effect sizes for both groups. MBT-DH was not superior to MBT-IOP on the primary outcome measure, but MBT-DH showed a clear tendency towards superiority on secondary outcomes.ConclusionsAlthough MBT-DH was not superior to MBT-IOP on the primary outcome measure despite its greater treatment intensity, MBT-DH showed a tendency to be more effective on secondary outcomes, particularly in terms of relational functioning. Patients receiving MBT-DH and MBT-IOP, thus, seem to follow different trajectories of change, which may have important implications for clinical decision-making. Longer-term follow-up and cost-effectiveness considerations may ultimately determine the optimal intensity of specialised treatments such as MBT for patients with BPD.

scholarly journals Diagnosis and outcome measures in trials for neoplastic meningitis: a review of the literature and clinical experience

1998 ◽  
Vol 4 (6) ◽  
pp. E6 ◽  
Author(s):  
Michael J. Glantz ◽  
Marc C. Chamberlin ◽  
Beverly C. Walters
Keyword(s):  
Cerebrospinal Fluid ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Randomized Clinical Trials ◽  
Outcome Measure ◽  
Clinical Care ◽  
Primary Outcome Measure ◽  
Neoplastic Meningitis ◽  
Review Of The Literature ◽  
Cytological Examination

Innovative approaches to the treatment of neoplastic meningitis are being widely tested. Unfortunately, research on diagnostic strategies and outcome measures on which any advances in treatment ultimately depend, has not been avidly pursued. A critical review of the literature on neoplastic meningitis published since 1978 was undertaken by using MEDLINE and other English language databases. All articles addressing the issues of diagnostic or response criteria were included. Randomized clinical trials (RCTs) were emphasized. Prospectively collected data from the authors' institution correlating the results of cerebrospinal fluid (CSF) cytological examinations with Karnofsky Performance Scale (KPS) score are also discussed. Twenty-six studies (representing 1208 patients) fulfilled search criteria. Only three were RCTs. Cerebrospinal fluid cytology was the sole diagnostic criterion in two-thirds of studies. The results of CSF cytological examination alone or in combination with other clinical or laboratory endpoints constituted the primary outcome measure in 85%. Few studies attempted to address known deficiencies in the reliability and validity of these measures, and correlation between measures was poor. Quality of life was never used as a primary outcome measure. All currently available measurements, including CSF cytology, biochemistry, immunological, and molecular markers, neuroimaging studies, clinical examination, and survival, suffer from poor sensitivity and/or specificity, and often correlate poorly with each other. Although CSF cytological examination, performed according to a rigorous, research-supported protocol, may be the optimum diagnostic and outcome measure at this time, additional research is a prerequisite for any further advances in the clinical care of patients with neoplastic meningitis.

scholarly journals Efficiency and safety of ondansetron in preventing postanaesthesia shivering

2016 ◽  
Vol 98 (6) ◽  
pp. 358-366 ◽  
Author(s):  
k He ◽  
H Zhao ◽  
HC Zhou
Keyword(s):  
Lower Risk ◽  
Randomised Controlled Trials ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Meta Analysis ◽  
Primary Outcome Measure ◽  
Cochrane Library ◽  
Significant Difference ◽  
Secondary Outcomes ◽  
Randomised Controlled

Introduction Shivering is one of the most frequent complications of operation during the postanaesthesia period. Ondansetron has been proved to be valid in preventing postanaesthesia shivering (PAS) in several studies. However, its efficiency and safety are still disputable. We therefore performed an updated meta-analysis of randomised controlled trials (RCTs) for evaluation and to clarify this issue. Methods A literature search using the PubMed, Embase™ and Cochrane Library databases was performed (from inception to January 2015). RCTs that evaluated the efficiency and safety of ondansetron in the prevention of PAS were included in the meta-analysis. The primary outcome measure was incidence of PAS, and secondary outcomes included subgroup analysis and the side effects of ondansetron. Results A total of 8 RCTs containing 905 subjects were identified as suitable for this review. Compared with placebo, ondansetron was associated with a significant reduction of PAS (relative risk [RR]: 0.33, 95% confidence interval [CI]: 0.19–0.58, p=0.0001) while no difference was detected between ondansetron and pethidine (RR: 0.89, 95% CI: 0.41–1.94, p=0.78). There was no significant difference between ondansetron and placebo or pethidine in terms of risk of bradycardia but ondansetron was associated with a lower risk of hypotension (RR: 0.26, 95% CI: 0.08–0.79, p=0.020) than placebo. There was no difference in hypotension when ondansetron was compared with pethidine. Conclusions Ondansetron can prevent PAS effectively and reduce the risk of hypotension.

Statistical analysis, trial design and duration in Alzheimer's disease clinical trials: a review

2011 ◽  
Vol 24 (5) ◽  
pp. 689-697 ◽  
Author(s):  
P. A. Thompson ◽  
D. E. Wright ◽  
C. E. Counsell ◽  
J. Zajicek
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trials ◽  
Statistical Analysis ◽  
Statistical Methods ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Primary Analysis

ABSTRACTBackground: The social and economic burden of Alzheimer's disease (AD) and its increasing prevalence has led to much work on new treatment strategies and clinical trials. The search for surrogate markers of disease progression continues but traditional parallel group trial designs that use well-established, but often insensitive, clinical outcome measures predominate.Methods: We performed a systematic search across the Cochrane Library and PubMed abstracts published between January 2004 and August 2009. Information regarding the clinical trial methodology, outcome measures, intervention type and primary statistical analysis techniques was extracted and categorized, according to a standard protocol.Results: We identified 149 papers describing results from clinical trials in AD containing sufficient detail for our purposes. The largest proportion (38%) presented results of trials based on tests of cognition as the primary outcome measure. The primary analysis in most papers (85%) was a univariate significance test of a single primary outcome measure.Conclusions: The majority of trials reported a comparison of baseline and end-point assessment over relatively short patient follow-up periods, using univariate statistical methods to compare differences between intervention and control groups in the primary analysis. There is considerable scope to introduce newer statistical methods and trial designs in treatment evaluations in AD.

scholarly journals Home-based rehabilitation programme compared with traditional physiotherapy for patients at risk of poor outcome after knee arthroplasty: the CORKA randomised controlled trial

BMJ Open ◽  
2021 ◽  
Vol 11 (8) ◽  
pp. e052598
Author(s):  
Karen L Barker ◽  
Jonathan Room ◽  
Ruth Knight ◽  
Susan Dutton ◽  
Francine Toye ◽  
...  
Keyword(s):  
At Risk ◽  
Usual Care ◽  
Outcome Measures ◽  
Knee Arthroplasty ◽  
Primary Outcome ◽  
Rehabilitation Programme ◽  
Poor Outcome ◽  
Home Based ◽  
Secondary Outcomes ◽  
Randomised Controlled

ObjectivesTo evaluate whether a home-based rehabilitation programme for people assessed as being at risk of a poor outcome after knee arthroplasty offers superior outcomes to traditional outpatient physiotherapy.DesignA prospective, single-blind, two-arm randomised controlled superiority trial.Setting14 National Health Service physiotherapy departments in the UK.Participants621 participants identified at high risk of a poor outcome after knee arthroplasty using a bespoke screening tool.InterventionsA multicomponent home-based rehabilitation programme delivered by rehabilitation assistants with supervision from qualified therapists versus usual care outpatient physiotherapy.Main outcome measuresThe primary outcome was the Late-Life Function and Disability Instrument (LLFDI) at 12 months. Secondary outcomes were the Oxford Knee Score (a disease-specific measure of function), Knee injury and Osteoarthritis Outcome Score Quality of Life subscale, Physical Activity Scale for the Elderly, 5 dimension, 5 level version of Euroqol (EQ-5D-5L) and physical function assessed using the Figure of 8 Walk test, 30 s Chair Stand Test and Single Leg Stance.Results621 participants were randomised between March 2015 and January 2018. 309 were assigned to CORKA (Community Rehabilitation after Knee Arthroplasty) home-based rehabilitation, receiving a median five treatment sessions (IQR 4–7). 312 were assigned to usual care, receiving a median 4 sessions (IQR 2–6). The primary outcome, LLFDI function total score at 12 months, was collected for 279 participants (89%) in the home-based CORKA group and 287 participants (92%) in the usual care group. No clinically or statistically significant difference was found between the groups (intention-to-treat adjusted difference=0.49 points; 95% CI −0.89 to 1.88; p=0.48). There were no statistically significant differences between the groups on any of the patient-reported or physical secondary outcome measures at 6 or 12 months.There were 18 participants in the intervention group reporting a serious adverse event (5.8%), only one directly related to the intervention, all other adverse events recorded throughout the trial related to underlying chronic medical conditions.ConclusionsThe CORKA intervention was not superior to usual care. The trial detected no significant differences, clinical or statistical, between the two groups on either primary or secondary outcomes. CORKA offers an evaluation of an intervention utilising a different service delivery model for this patient group.Trial registration numberISRCTN13517704.

scholarly journals Gastric Residual Volumes in the Adult Intensive Care Patient: a Systematic Review

2021 ◽  
Author(s):  
◽  
Rebecca Jane Jarden
Keyword(s):  
Systematic Review ◽  
At Risk ◽  
Intensive Care ◽  
Enteral Nutrition ◽  
Outcome Measures ◽  
Full Text ◽  
Primary Outcome ◽  
Risk Of Bias ◽  
Randomised Controlled ◽  
Patient At Risk

<p>Background: Enteral nutrition is one method of delivering nutrition to intubated patients. There are several issues that prevent optimal delivery of the prescribed enteral nutrition goal rates. The measurement of the patient's gastric residual volume (GRV) may demonstrate tolerability, or intolerability, of enteral nutrition. Identifying a safe GRV, at which to accept and continue enteral nutrition delivery, is essential to ensure the delivery of enteral nutrition adequately achieves the nutritional requirements of patients, and to mitigate the risks associated with the delivery of enteral nutrition. Objectives: This systematic review sought to answer the research question: what is the maximum GRV to accept in order to continue the delivery of enteral nutrition in the Intensive Care Unit (ICU) adult patient? This is specifically related to the primary outcome measures indicative of accepting a specified GRV that is too high or too low. Accepting a GRV that is too high would put the patient at risk of vomiting, regurgitation, aspiration of gastric contents and potentially aspiration pneumonia. Conversely, accepting a GRV that is too low would put the patient at risk of not achieving caloric needs, potentially placing the patient at risk of malnutrition and increased morbidity. Search methods: Databases searched included: CCTR, CLCMR, CLTA, CLEED, OVID MEDLINE (R) (Ovid SP), EMBASE, CINAHL Plus with Full Text (EBSCO host via helicon), AMED, Ovid Nursing Full Text plus, CDSR, ACP Journal Club, DARE, Proquest via helicon (advanced search), Pubmed via helicon (limits "all adult", "humans", "abstract", "title"), all EBM reviews, and the reference lists of articles. Selection criteria: The types of studies eligible for inclusion were published randomised controlled trials, case controlled studies, cohort studies and observational studies. Interventions considered were a comparison of two or more GRV measures. The participants eligible were adult ICU or critical care patients receiving enteral nutrition. The primary outcome measures for study inclusion were caloric requirement met, and specified potential adverse events including vomiting, regurgitation, or aspiration. Data collection and analysis: Data was extracted using a data extraction tool created by the researcher. Risk of bias was assessed by the author using two risk of bias assessment tools. Main results: Three studies met the inclusion criteria for the systematic review (McClave et al., 2005; Metheny, Schallom, Oliver, & Clouse, 2008; Pinilla, Samphire, Arnold, Liu, & Thiessen, 2001). Each of these studies contained methodological risks of bias and limitations related to their study designs. McClave et al.'s study was a prospective study (n = 40), Metheny et al.'s study was a prospective descriptive study (n = 206), and Pinilla et al.'s study was a randomised controlled trial (n = 80). No one study, or a combination of studies, provided conclusive evidence to support the use of one particular GRV over another. Author's conclusion: No recommendation for a definitive GRV was made in this systematic review due to the lack of strong evidentiary support for one GRV over another. There remain opportunities for enhancing practice through developing a consistent, multidisciplinary approach to managing GRVs. There are future research opportunities related to improving the management of GRVs in the enterally fed ICU patient, and achieving optimal volumes of nutrition delivered.</p>

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