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Journal of Plastic, Reconstructive &... Apr 2020Congenital melanocytic naevi (CMN) can have a great impact on patients' lives due to the remarkable appearance and the risk of developing melanoma and neurocutaneous...
BACKGROUND
Congenital melanocytic naevi (CMN) can have a great impact on patients' lives due to the remarkable appearance and the risk of developing melanoma and neurocutaneous melanosis. Comparison of treatment efficacy is currently hindered by the lack of standard and uniform outcome reporting; this impedes guidance on optimal management policy. To address this, we aim to perform the first step in developing a core outcome set. With this systematic review, we identified a list of domains, outcomes (including patient reported outcomes) and outcome measurement instruments used in CMN research.
METHODS
The review was registered in PROSPERO, registration number CRD42018095235. A search was conducted in EMBASE (Ovid), PubMed and the Cochrane Library from 2006 to January 2019. Studies with 10 or more patients, with all sizes of CMN and reporting outcomes on interventional and conservative management were included.
RESULTS
A total of 1,285 individual studies was found; 63 studies were included. We extracted 57 different outcomes and 34 outcome measurement instruments showing large heterogeneity. Patient-reported outcomes were included in 38% of studies. Few outcome measurement instruments were described. Moreover, none of the studies reported that the used instruments were ever validated in a CMN population.
CONCLUSION
Heterogeneity exists in outcomes and instruments used in CMN research. The development of a core outcome set may reduce this heterogeneity in future research, thereby enabling treatment comparison and eventually facilitating guidance on management. Furthermore, this overview demonstrates a need for the use and validation of (patient reported) outcome measurement instruments for CMN.
Topics: Biomedical Research; Humans; Nevus, Pigmented; Outcome Assessment, Health Care; Skin Neoplasms
PubMed: 31917187
DOI: 10.1016/j.bjps.2019.11.023 -
Headache Feb 2021Over the last six decades (earliest included publication from 1959), clinical trials of migraine preventive treatments have led to the regulatory approval of many...
BACKGROUND
Over the last six decades (earliest included publication from 1959), clinical trials of migraine preventive treatments have led to the regulatory approval of many medications and devices. Despite similar clinical goals, the outcomes and endpoints used in these trials are broad and not well standardized.
OBJECTIVE
To describe results from a systematic literature review focused on outcomes and endpoints used in preventive migraine clinical trials.
METHOD
A systematic literature review, following a pre-specified (unregistered) protocol developed to adhere to recommendations of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses, was conducted to characterize the endpoints and outcomes used in preventive migraine clinical trials. Predetermined terms were searched in PubMed on October 28, 2019. Data related to trial design, subject characteristics, outcomes, and endpoints reported in each publication were extracted. Descriptive summaries of these features were tabulated for the recent subset of publications, published during or after 1988, that were randomized, blinded, and focused on pharmacological or device therapies for the preventive treatment of migraine.
RESULTS
The initial literature search identified 1506 publications, of which 757 publications were eligible for data extraction. Of specific clinical interest were the recent subset of 268 articles (268/757, 35.4%) fulfilling the targeted criteria. Results showed that the outcomes used to define endpoints varied substantially across publications. For example, in the recent subset of publications, 68.7% (184/268) of the publications examined ≥1 migraine-specific outcome, 39.6% (106/268) examined ≥1 headache-specific outcome, 50.7% (136/268) examined ≥1 acute/rescue medication use outcome, 40.3% (108/268) examined ≥1 headache-related patient-reported outcome measure (PROM), and 22.0% (59/268) examined ≥1 non-headache-specific PROM. Furthermore, the definition of the endpoints used (e.g., change from baseline, fixed timepoint comparisons, categorization of "responders" to treatment based on wide variety of "responder definitions") also differed across publications.
CONCLUSION
Publications from clinical trials of preventive migraine pharmacologic and device treatments differed in terms of study design, endpoint definitions, and how endpoints and outcomes were measured. Although there were common outcomes and endpoints used across publications, no clear "standardized" set of endpoints and outcomes emerged. The inconsistencies in endpoints and outcomes within this literature suggest that the development of a uniform set of outcomes and endpoints could improve the clinical meaningfulness of clinical trial results, facilitate cross-trial comparisons and better inform patient care. This standard set of outcomes and endpoints should be statistically robust and informed by the priorities of various stakeholders, most importantly, the needs and preferences of people living with migraine.
Topics: Clinical Trials as Topic; Humans; Migraine Disorders; Outcome Assessment, Health Care
PubMed: 33600610
DOI: 10.1111/head.14069 -
Facial Plastic Surgery & Aesthetic... 2022Forehead reduction, or hairline lowering surgery, is becoming more popular as a cosmetic procedure for patients with disproportionately large foreheads. A large... (Meta-Analysis)
Meta-Analysis
Forehead reduction, or hairline lowering surgery, is becoming more popular as a cosmetic procedure for patients with disproportionately large foreheads. A large forehead can make a patient appear older, be masculinizing, and less attractive. To quantify reported outcomes in patients undergoing forehead reduction. We performed a systematic review and meta-analysis of adults undergoing forehead reduction. A review protocol was published in PROSPERO (CRD42020183366). A research librarian created search strategies in multiple databases. Abstracts and full texts were reviewed in duplicate. The Newcastle-Ottawa scale and Cochrane Collaboration Risk of Bias tool were used. Random effects meta-analyses were performed. The primary outcome was amount of reduction. Other extracted data included study type, location, sample size, scalp fixation method, incision, complications, follow-up time, percentage female, and age. Our search strategy found 376 unique citations, and 8 studies were included. All eight were retrospective cohort studies, comprising 882 patients (range 5-525). Study quality was high, and risk of bias ranged from unclear to high. Four studies were included for meta-analysis, totaling 801 patients. Mean amount of reduction was 1.6 cm (95% confidence interval: 1.4-1.8). Complications included temporary and permanent alopecia, unacceptable scarring, persistent paresthesia, and hematoma. The pooled complication rate was 1% or less. Forehead reduction is associated with a low complication rate (<1%), and a mean lowering of 1.6 cm is reported. Future studies should report mean and standard deviation of reduction, and should follow patients for at least 12 months.
Topics: Cosmetic Techniques; Forehead; Humans; Outcome Assessment, Health Care; Postoperative Complications
PubMed: 33601981
DOI: 10.1089/fpsam.2020.0474 -
Maturitas Oct 2022To chart peer-reviewed literature regarding the psychological and social health outcomes of physical activity (PA) around menopause in a systematic manner. (Review)
Review
OBJECTIVE
To chart peer-reviewed literature regarding the psychological and social health outcomes of physical activity (PA) around menopause in a systematic manner.
METHODS
Nine electronic databases and 10 core journals were searched using specific search strings to identify eligible articles. Manual checking of reference lists was also performed. The selection process was guided by the stages in PRISMA-ScR.
RESULTS
Eighty peer-reviewed articles representing 67 studies from 25 countries were included. All articles were published between 1994 and 2021. For all studies, surveys were the primary method of measuring psychological and social health outcomes, in cross-sectional studies (36 papers), intervention studies (33), longitudinal cohort studies (10) and one paper reporting a mixed-method study. The dataset comprised a total of 103,826 women, with an average age of 52.6 and a variety of menopausal states. Most of the studies involved primarily Caucasian, relatively healthy, married and employed participants. Nineteen psychological and social health outcomes were assessed, including psychological menopause symptoms (N = 34), quality of life (N = 33), depression (N = 30), anxiety (N = 11), mental wellbeing (N = 21), perceived stress (N = 9), satisfaction with life (N = 7) and self-esteem (N = 5).
CONCLUSIONS
Collectively, the findings of these studies indicate a relatively evident positive impact of PA on the respective health outcomes, with only a few studies reporting no association. It is also noteworthy that most studies did not report any difference related to menopausal status. Future studies would benefit from, inter alia, a qualitative approach to lived experiences of psychological and social health outcomes of PA during the menopausal transition.
Topics: Cross-Sectional Studies; Exercise; Female; Humans; Longitudinal Studies; Menopause; Outcome Assessment, Health Care; Quality of Life
PubMed: 35964395
DOI: 10.1016/j.maturitas.2022.07.014 -
International Journal of Stroke :... Feb 2023Central adjudication of outcomes is common in randomized clinical trials in stroke. The rationale for adjudication is clear; centrally adjudicated outcomes should have... (Review)
Review
BACKGROUND
Central adjudication of outcomes is common in randomized clinical trials in stroke. The rationale for adjudication is clear; centrally adjudicated outcomes should have less random and systematic errors than outcomes assessed locally by site investigators. However, adjudication brings added complexities to a clinical trial and can be costly.
AIM
To assess the evidence for outcome adjudication in stroke trials.
SUMMARY OF REVIEW
We identified 12 studies evaluating central adjudication in stroke trials. The majority of these were secondary analyses of trials, and the results of all of these would have remained unchanged had central adjudication not taken place, even for trials without sufficient blinding. The largest differences between site-assessed and adjudicator-assessed outcomes were between the most subjective outcomes, such as causality of serious adverse events. We found that the cost of adjudication could be upward of £100,000 for medium to large prevention trials. These findings suggest that the cost of central adjudication may outweigh the advantages it brings in many cases. However, through simulation, we found that only a small amount of bias is required in site investigators' outcome assessments before adjudication becomes important.
CONCLUSION
Central adjudication may not be necessary in stroke trials with blinded outcome assessment. However, for open-label studies, central adjudication may be more important.
Topics: Humans; Stroke; Outcome Assessment, Health Care; Research Design
PubMed: 35373672
DOI: 10.1177/17474930221094682 -
Clinical Psychology & Psychotherapy 2023Treatment of schizotypal personality disorder is complex. Currently, there are no clear evidence-based recommendations for use of psychotherapy for individuals suffering... (Review)
Review
BACKGROUND
Treatment of schizotypal personality disorder is complex. Currently, there are no clear evidence-based recommendations for use of psychotherapy for individuals suffering from this mental illness, and studies are sparse. Our aim in this review is to map and describe the existing research and to answer the research question: What do we know about the use of psychotherapy for people with schizotypal personality disorder?
METHODS
We conducted a scoping review using systematic searches in the Embase, MEDLINE and PsycINFO databases. Two reviewers screened possible studies and extracted data on subject samples, type of psychotherapy, outcomes and suggested mechanisms of change. The review is based on the PRISMA checklist for scoping reviews.
RESULTS
Twenty-three papers were included, and we found a wide variety of study types, psychotherapeutic orientations and outcomes. Few studies emerged that focused solely on schizotypal personality disorder.
CONCLUSION
Psychotherapy as a treatment for schizotypal personality disorder is understudied compared with diagnoses such as schizophrenia and borderline personality disorder. Our results included two randomized controlled studies, as well as mainly smaller studies with different approaches to diagnostic criteria, psychotherapeutic orientation and outcome measures. The findings are too sparse and too diverse to make any evidence-based recommendations. We found some indications that psychotherapy may support and assist individuals with schizotypal personality disorder.
Topics: Humans; Schizotypal Personality Disorder; Psychotherapy; Borderline Personality Disorder; Schizophrenia; Outcome Assessment, Health Care
PubMed: 37675964
DOI: 10.1002/cpp.2901 -
Epilepsia Nov 2022Several instruments and outcomes measures have been reported in pediatric patients undergoing epilepsy surgery. The objective of this systematic review is to summarize,... (Review)
Review
Several instruments and outcomes measures have been reported in pediatric patients undergoing epilepsy surgery. The objective of this systematic review is to summarize, evaluate, and quantify outcome metrics for the surgical treatment of pediatric epilepsy that address seizure frequency, neuropsychological, and health-related quality of life (HRQL). We performed a systematic review according to Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines to identify publications between 2010 and June 2021 from PubMed, Embase, and the Cochrane Database of Systematic Reviews that report clinical outcomes in pediatric epilepsy surgery. Eighty-one articles were included for review. Overall, rates of postoperative seizure frequency were the most common metric reported (n = 78 studies, 96%). Among the seizure frequency metrics, the Engel Epilepsy Surgery Outcome Scale (n = 48 studies, 59%) was most commonly reported. Neuropsychological outcomes, performed in 32 studies (40%) were assessed using 36 different named metrics. HRQL outcomes were performed in 16 studies (20%) using 13 different metrics. Forty-six studies (57%) reported postoperative changes in antiepileptic drug (AED) regimen, and time-to-event analysis was performed in 15 (19%) studies. Only 13 outcomes metrics (1/5 seizure frequency, 6/13 HRQL, 6/36 neuropsychological) have been validated for use in pediatric patients with epilepsy and only 13 have been assessed through reliability studies (4/5 seizure frequency, 6/13 HRQL, and 3/36 neuropsychological). Of the 81 included studies, 17 (21%) used at least one validated metric. Outcome variable metrics in pediatric epilepsy surgery are highly variable. Although nearly all studies report seizure frequency, there is considerable variation in reporting. HRQL and neuropsychological outcomes are less frequently and much more heterogeneously reported. Reliable and validated outcomes metrics should be used to increase standardization and accuracy of reporting outcomes in pediatric patients undergoing epilepsy surgery.
Topics: Humans; Child; Quality of Life; Reproducibility of Results; Treatment Outcome; Epilepsy; Seizures; Outcome Assessment, Health Care
PubMed: 35847999
DOI: 10.1111/epi.17369 -
Clinical Psychology Review Feb 2020Self-criticism involves constant and harsh self-scrutiny, overly critical evaluations of one's own behavior, and negative reactions to perceived failures in terms of... (Meta-Analysis)
Meta-Analysis
Self-criticism involves constant and harsh self-scrutiny, overly critical evaluations of one's own behavior, and negative reactions to perceived failures in terms of active self-bashing. Self-criticism is associated with various mental disorders and psychotherapy outcome. This paper provides (1) a meta-analysis of the association between pre-treatment self-criticism and multiple treatment outcomes, and (2) a systematic review of the association between change in self-criticism and therapy outcome. Based on a systematic literature search, 49 longitudinal studies (56 independent effect sizes; 3277 patients) were included in the meta-analysis and 7 studies were identified for the additional systematic review. A random-effects meta-analysis was performed to assess the magnitude of the association between self-criticism and outcome, also considering potential moderators. The overall association between pre-treatment self-criticism and psychotherapy outcome was r = -.20 (95% CI = -.25 - -.16, p < .0001), suggesting that higher levels of self-criticism are related to poorer outcome. Although effect sizes showed little heterogeneity, the association varied by type of mental health problem and indicated stronger associations with certain disorders (e.g., eating disorders). The review based on change scores yielded inconsistent results. Our findings support the relevance of self-criticism for psychotherapy outcome.
Topics: Feeding and Eating Disorders; Humans; Mental Disorders; Outcome Assessment, Health Care; Perfectionism; Psychotherapy; Self-Assessment
PubMed: 31864153
DOI: 10.1016/j.cpr.2019.101808 -
Headache Feb 2021To review the acute migraine clinical trial literature and provide a summary of the endpoints and outcomes used in such trials.
BACKGROUND/OBJECTIVE
To review the acute migraine clinical trial literature and provide a summary of the endpoints and outcomes used in such trials.
METHOD
A systematic literature review, following a prespecified (but unregistered) protocol developed to adhere to recommendations of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses, was conducted to understand endpoints and outcomes used in acute migraine clinical trials. Predefined terms were searched in PubMed to locate clinical trials assessing acute migraine treatments. Final database search was conducted on October 28, 2019. Identified publications were reviewed against established inclusion and exclusion criteria to determine eligibility. Data related to general trial design characteristics, sample characteristics, and outcomes and endpoints reported in each publication were extracted from eligible publications. Descriptive summaries of design features, sample characteristics, and the endpoints and outcomes employed across publications were constructed. Outcomes are presented within four broad categories: (a) pain-related outcomes (pain relief, pain freedom, etc.), (b) associated symptoms (nausea, photophobia, etc.), (c) disability/impairment/impact, (d) patient-reported outcome measures (PROMs, general health and migraine/headache-specific). Endpoint types were categorized within three broad categories: (a) change from baseline, (b) fixed timepoint, and (c) responder definitions (e.g., 50% reduction). This review focuses on a subset of recent (1998 or later) randomized and blinded publications evaluating drugs or medical devices.
RESULTS
Of 1567 publications found through the initial search and reference section reviews, 705 met criteria and were included for data extraction. Inter-rater agreement kappas for the descriptive variables extracted had an average kappa estimate of 0.86. The more recent, randomized and blinded pharmaceutical and medical device article subset includes 451 publications (451/705, 63.9%). The outcomes and endpoints varied substantially across trials, ranging from pain relief or freedom, freedom from or relief of migraine-associated symptoms, use of acute or rescue medication, and various other PROMs, including measures of satisfaction and quality of life. Within the recent randomized and blinded article subset, most articles examined ≥1 pain-related outcome (430/451, 95.3%). Of the publications that examined pain, outcomes most often used were pain relief (310/430, 72.1%), pain freedom (279/430, 64.9%), and headache recurrence (202/43,051, 47.0%) or rescue medication use (278/430, 64.9%). Associated symptoms such as nausea, photophobia, and phonophobia were more frequently measured (299/451, 66.3%) compared to most bothersome associated symptom (16/451, 3.5%), as it is a new addition to regulatory guidance. Over one-third of eligible publications examined disability/impairment (186/451, 41.2%) or ≥1 PROM (159/451, 35.3%). The definition of the endpoints used (e.g., change from baseline, fixed timepoint comparisons, categorization of "responders" to treatment based on wide variety of "responder definitions") also differed substantially across publications.
CONCLUSION
Acute migraine clinical trials exhibit a large amount of variability in outcomes and endpoints used, in addition to the variability in how outcomes and endpoints were used from trial-to-trial. There were some common elements across trials that align with guidance from the International Headache Society, the Food and Drug Administration and other regulatory agencies (e.g., assessing pain and associated symptoms, 2-hour post-treatment). Other aspects of acute migraine clinical trial design did not follow guidance. For example, multi-item PROMs intended to measure constructs (e.g., scales) are rarely used, the use of pain-related outcomes is inconsistent, some associated symptom assessments are idiosyncratic, and the timing of the assessment of primary endpoints is variable. The development of a core set of outcomes and endpoints for acute migraine clinical trials that are patient-centered and statistically robust could improve the conduct of individual trials, facilitate cross-trial comparisons, and better support informed treatment decisions by healthcare professionals and patients.
Topics: Acute Disease; Clinical Trials as Topic; Humans; Migraine Disorders; Outcome Assessment, Health Care
PubMed: 33611818
DOI: 10.1111/head.14067 -
The Journal of Surgical Research Nov 2023Low levels of health literacy have been shown to increase healthcare utilization and negatively affect health outcomes within medical specialties. However, the... (Review)
Review
INTRODUCTION
Low levels of health literacy have been shown to increase healthcare utilization and negatively affect health outcomes within medical specialties. However, the relationship of health literacy with clinical, patient-centered, and process-oriented surgical outcomes is not as well understood.
MATERIALS AND METHODS
We sought to systematically review the current evidence base regarding the relationship between health literacy and a range of outcomes in patients experiencing surgical care. Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, we searched six databases and then identified and extracted data from 25 cross-sectional or cohort studies deemed eligible for a systematic review.
RESULTS
Among included studies, strong evidence exists to support an association between low health literacy and worse patient-centered outcomes, as well as an association between low health literacy and poorer process-oriented surgical outcomes. However, the relationship between health literacy and clinical outcomes remains unclear.
CONCLUSIONS
Substantial opportunities remain to improve our understanding of the impact of health literacy on surgical outcomes. Future work should expand the range of institutional and specialized surgical settings studied, implement a standardized set of validated health literacy assessment tools, include more diverse patient populations, and investigate a comprehensive range of patient-reported outcomes.
Topics: Humans; Health Literacy; Cross-Sectional Studies; Outcome Assessment, Health Care; Delivery of Health Care; Treatment Outcome
PubMed: 37572516
DOI: 10.1016/j.jss.2023.06.044