Abstract:
BACKGROUND:One of the main challenges for drug evaluation in rare diseases is the often heterogeneous course of these diseases. Traditional outcome measures may not be applicable for all patients, when they are in different stages of their disease. For instance, in Duchenne Muscular Dystrophy, the Six Minute Walk Test is often used to evaluate potential new treatments, whereas this outcome is irrelevant for patients who are already in a wheelchair. A measurement instrument such as Goal Attainment Scaling (GAS) can evaluate the effect of an intervention on an individual basis, and may be able to include patients even when they are in different stages of their disease. It allows patients to set individual goals, together with their treating professional. However, the validity of GAS as a measurement instrument in drug studies has never been systematically reviewed. Therefore, we have performed a systematic review to answer two questions: 1. Has GAS been used as a measurement instrument in drug studies? 2: What is known of the validity, responsiveness and inter- and intra-rater reliability of GAS, particularly in drug trials? METHODS:We set up a sensitive search that yielded 3818 abstracts. After careful screening, data-extraction was executed for 58 selected articles. RESULTS:Of the 58 selected articles, 38 articles described drug studies where GAS was used as an outcome measure, and 20 articles described measurement properties of GAS in other settings. The results show that validity, responsiveness and reliability of GAS in drug studies have hardly been investigated. The quality of the reporting of validity in studies in which GAS was used to evaluate a non-drug intervention also leaves much room for improvement. CONCLUSIONS:We conclude that there is insufficient information to assess the validity of GAS, due to the poor quality of the validity studies. Therefore, we think that GAS needs further validation in drug studies, especially since GAS can be a potential solution when a small heterogeneous patient group is all there is to test a promising new drug. TRIAL REGISTRATION:The protocol has been registered in the PROSPERO international prospective register for systematic reviews, with registration number CRD42014010619. http://www.crd.york.ac.uk/PROSPERO/display_record.asp?ID=CRD42014010619 .
journal_name
BMC Med Res Methodoljournal_title
BMC medical research methodologyauthors
Gaasterland CM,Jansen-van der Weide MC,Weinreich SS,van der Lee JHdoi
10.1186/s12874-016-0205-4subject
Has Abstractpub_date
2016-08-17 00:00:00pages
99issn
1471-2288pii
10.1186/s12874-016-0205-4journal_volume
16pub_type
杂志文章,评审abstract:BACKGROUND:Small number of clusters and large variation of cluster sizes commonly exist in cluster-randomized trials (CRTs) and are often the critical factors affecting the validity and efficiency of statistical analyses. F tests are commonly used in the generalized linear mixed model (GLMM) to test intervention effect...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-015-0026-x
更新日期:2015-04-23 00:00:00
abstract:BACKGROUND:When conducting a meta-analysis of a continuous outcome, estimated means and standard deviations from the selected studies are required in order to obtain an overall estimate of the mean effect and its confidence interval. If these quantities are not directly reported in the publications, they must be estima...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-015-0055-5
更新日期:2015-08-12 00:00:00
abstract:BACKGROUND:Sleeping under a long-lasting insecticidal net (LLIN) is recommended for all pregnant women in sub-Saharan Africa, due to the high prevalence of malaria infection and its associated complications in the region. Despite this, LLIN use has still remained sub-optimal among pregnant women in Maiduguri, Nigeria. ...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-019-0803-z
更新日期:2019-07-18 00:00:00
abstract:BACKGROUND:Claims databases are generally considered inadequate for obesity research due to suboptimal capture of body mass index (BMI) measurements. This might not be true for bariatric surgery because of reimbursement requirements and changes in coding systems. We assessed the availability and validity of claims-base...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-020-01106-8
更新日期:2020-09-07 00:00:00
abstract:BACKGROUND:Estimating costs is essential to the economic analysis of health care programs. Health care costs are often captured from administrative databases or by patient report. Administrative records only provide a partial representation of health care costs and have additional limitations. Patient-completed questio...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/1471-2288-11-45
更新日期:2011-04-13 00:00:00
abstract:BACKGROUND:Chemotherapy is expected to reduce cancer deaths (CD), while possibly being harmful in terms of non-cancer deaths (NCD) because of toxicity. Peto's log-rank test is popular in the medical literature, but its operating characteristics are barely known. We compared this test to the most common ones in the stat...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/1471-2288-14-72
更新日期:2014-05-29 00:00:00
abstract:BACKGROUND:Diet plays an important role in chronic disease, and the use of dietary pattern analysis has grown rapidly as a way of deconstructing the complexity of nutritional intake and its relation to health. Pattern analysis methods, such as principal component analysis (PCA), have been used to investigate various di...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-018-0585-8
更新日期:2018-10-29 00:00:00
abstract:BACKGROUND:The choice between paper data collection methods and electronic data collection (EDC) methods has become a key question for clinical researchers. There remains a need to examine potential benefits, efficiencies, and innovations associated with an EDC system in a multi-center medical record review study. MET...
journal_title:BMC medical research methodology
pub_type: 杂志文章,多中心研究
doi:10.1186/1471-2288-7-23
更新日期:2007-06-18 00:00:00
abstract:BACKGROUND:Retaining participants in cohort studies with multiple follow-up waves is difficult. Commonly, researchers are faced with the problem of missing data, which may introduce biased results as well as a loss of statistical power and precision. The STROBE guidelines von Elm et al. (Lancet, 370:1453-1457, 2007); V...
journal_title:BMC medical research methodology
pub_type: 杂志文章,评审
doi:10.1186/1471-2288-12-96
更新日期:2012-07-11 00:00:00
abstract:BACKGROUND:Dichotomisation of continuous outcomes has been rightly criticised by statisticians because of the loss of information incurred. However to communicate a comparison of risks, dichotomised outcomes may be necessary. Peacock et al. developed a distributional approach to the dichotomisation of normally distribu...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-015-0028-8
更新日期:2015-04-24 00:00:00
abstract:BACKGROUND:When discussing results medical research articles often tear substantive and statistical (methodical) contributions apart, just as if both were independent. Consequently, reasoning on bias tends to be vague, unclear and superficial. This can lead to over-generalized, too narrow and misleading conclusions, es...
journal_title:BMC medical research methodology
pub_type: 杂志文章,评审
doi:10.1186/s12874-018-0490-1
更新日期:2018-04-17 00:00:00
abstract:BACKGROUND:There is increasing awareness that meta-analyses require a sufficiently large information size to detect or reject an anticipated intervention effect. The required information size in a meta-analysis may be calculated from an anticipated a priori intervention effect or from an intervention effect suggested b...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/1471-2288-9-86
更新日期:2009-12-30 00:00:00
abstract:BACKGROUND:The International Cancer Benchmarking Partnership aims to study international differences in cancer survival and the possible causes. Participating countries are Australia, Canada, Norway, Sweden, Denmark and the UK and a particular focus area is differences in awareness and beliefs about cancer. In this con...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-017-0352-2
更新日期:2017-04-26 00:00:00
abstract:BACKGROUND:This paper focuses on measuring the efficiency and effectiveness of two diagramming methods employed in key informant interviews with clinicians and health care administrators. The two methods are 'participatory diagramming', where the respondent creates a diagram that assists in their communication of answe...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/1471-2288-8-53
更新日期:2008-08-08 00:00:00
abstract:BACKGROUND:Multiple imputation is frequently used to deal with missing data in healthcare research. Although it is known that the outcome should be included in the imputation model when imputing missing covariate values, it is not known whether it should be imputed. Similarly no clear recommendations exist on: the util...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-016-0281-5
更新日期:2017-01-09 00:00:00
abstract:BACKGROUND:Standard random-effects meta-analysis methods perform poorly when applied to few studies only. Such settings however are commonly encountered in practice. It is unclear, whether or to what extent small-sample-size behaviour can be improved by more sophisticated modeling. METHODS:We consider likelihood-based...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-018-0618-3
更新日期:2019-01-11 00:00:00
abstract:BACKGROUND:Posttraumatic growth is the positive change resulting from traumatic experiences and is typically assessed with retrospective measures like the Posttraumatic Growth Inventory (PTGI). The PTGI was designed to include reference to a specific traumatic event, making it difficult to implement, without change, in...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-017-0344-2
更新日期:2017-04-20 00:00:00
abstract:BACKGROUND:Many medicines are dosed to achieve a particular therapeutic range, and monitored using therapeutic drug monitoring (TDM). The evidence base for a therapeutic range can be evaluated using systematic reviews, to ensure it continues to reflect current indications, doses, routes and formulations, as well as upd...
journal_title:BMC medical research methodology
pub_type: 杂志文章,评审
doi:10.1186/s12874-017-0363-z
更新日期:2017-06-02 00:00:00
abstract:BACKGROUND:Emergency Departments (EDs) are a first point-of-contact for many youth with mental health and suicidality concerns and can serve as an effective recruitment source for randomized controlled trials (RCTs) of mental health interventions. However, recruitment in acute care settings is impeded by several challe...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-020-01117-5
更新日期:2020-09-14 00:00:00
abstract:BACKGROUND:In Routine Outcome Monitoring (ROM) there is a high demand for short assessments. Computerized Adaptive Testing (CAT) is a promising method for efficient assessment. In this article, the efficiency of a CAT version of the Mood and Anxiety Symptom Questionnaire, - Anhedonic Depression scale (MASQ-AD) for use ...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/1471-2288-12-4
更新日期:2012-01-10 00:00:00
abstract:BACKGROUND:Defeat and entrapment have been shown to be of central relevance to the development of different disorders. However, it remains unclear whether they represent two distinct constructs or one overall latent variable. One reason for the unclarity is that traditional factor analytic techniques have trouble estim...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-018-0470-5
更新日期:2018-01-25 00:00:00
abstract:BACKGROUND:Systematic reviewers seek to comprehensively search for relevant studies and summarize these to present the most valid estimate of intervention effectiveness. The more resources searched, the higher the yield, and thus time and costs required to conduct a systematic review. While there is an abundance of evi...
journal_title:BMC medical research methodology
pub_type: 杂志文章,评审
doi:10.1186/1471-2288-5-24
更新日期:2005-08-10 00:00:00
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journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-016-0120-8
更新日期:2016-02-04 00:00:00
abstract:BACKGROUND:For practical reasons it is not easy to obtain fasting samples in large population health surveys. Non-fasting triglyceride (Tg) values are difficult to interpret. The authors compared the accuracy of statistically corrected non-fasting Tg values with true fasting values and estimated the misclassification o...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/1471-2288-11-63
更新日期:2011-05-10 00:00:00
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journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-016-0229-9
更新日期:2016-10-19 00:00:00
abstract:BACKGROUND:The Anglia Menorrhagia Education Study (AMES) is a randomized controlled trial testing the effectiveness of an education package applied to general practices. Binary data are available from two sources; general practitioner reported referrals to hospital, and referrals to hospital determined by independent a...
journal_title:BMC medical research methodology
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1186/1471-2288-3-17
更新日期:2003-09-24 00:00:00
abstract:BACKGROUND:The aim of this study was to examine the relationship between mortality and statin adherence using two different approaches to adherence measurement (summary versus repeated-measures). METHODS:A retrospective cohort study was conducted using administrative data from Saskatchewan, Canada between 1994 and 200...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-017-0339-z
更新日期:2017-04-20 00:00:00
abstract:BACKGROUND:Although a variety of instruments are available that capture stress experience, the assessment of chronic stress has been hindered by the lack of economical screening instruments. Recently, an English-language version of the Trier Inventory for Chronic Stress (TICS-EN) consisting of 57 items according to a s...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/s12874-020-01156-y
更新日期:2020-12-16 00:00:00
abstract:BACKGROUND:Diagnostic problems in clinical trials are sometimes ordinal. For example, colon tumor staging was performed according to the TNM classification. However, clinical data are limited by markedly small sample sizes in some stage. METHODS:We propose a distribution-free test for detecting ordered alternatives in...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/1471-2288-13-148
更新日期:2013-12-05 00:00:00
abstract:BACKGROUND:Estimating the incidence of medical conditions using claims data often requires constructing a prevalence period that predates an event of interest, for instance the diagnosis of cancer, to exclude those with pre-existing conditions from the incidence risk set. Those conditions missed during the prevalence p...
journal_title:BMC medical research methodology
pub_type: 杂志文章
doi:10.1186/1471-2288-13-32
更新日期:2013-03-06 00:00:00