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Statistical analysis of Goal Attainment Scaling endpoints in randomised trials.

ABSTRACT: Goal Attainment Scaling is an assessment instrument to evaluate interventions on the basis of individual, patient-specific goals. The attainment of these goals is mapped in a pre-specified way to attainment levels on an ordinal scale, which is common to all goals. This approach is patient-centred and allows one to integrate the outcomes of patients with very heterogeneous symptoms. The latter is of particular importance in clinical trials in rare diseases because it enables larger sample sizes by including a broader patient population. In this paper, we focus on the statistical analysis of Goal Attainment Scaling outcomes for the comparison of two treatments in randomised clinical trials. Building on a general statistical model, we investigate the properties of different hypothesis testing approaches. Additionally, we propose a latent variable approach to generate Goal Attainment Scaling data in a simulation study, to assess the impact of model parameters such as the number of goals per patient and their correlation, the choice of discretisation thresholds and the type of design (parallel group or cross-over). Based on our findings, we give recommendations for the design of clinical trials with a Goal Attainment Scaling endpoint. Furthermore, we discuss an application of Goal Attainment Scaling in a clinical trial in mastocytosis.

SUBMITTER: Urach S

PROVIDER: S-EPMC6566461 | biostudies-literature | 2019 Jun

REPOSITORIES: biostudies-literature

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Statistical analysis of Goal Attainment Scaling endpoints in randomised trials.

Urach S S Gaasterland Cmw C Posch M M Jilma B B Roes K K Rosenkranz G G Van der Lee J H JH Ristl R R

Statistical methods in medical research 20180619 6

Goal Attainment Scaling is an assessment instrument to evaluate interventions on the basis of individual, patient-specific goals. The attainment of these goals is mapped in a pre-specified way to attainment levels on an ordinal scale, which is common to all goals. This approach is patient-centred and allows one to integrate the outcomes of patients with very heterogeneous symptoms. The latter is of particular importance in clinical trials in rare diseases because it enables larger sample sizes b ...[more]

PMID: 29921167

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Project description:BACKGROUND: Evaluating collaborative community health promotion initiatives presents unique challenges, including engaging community members and other stakeholders in the evaluation process, and measuring the attainment of goals at the collective community level. Goal Attainment Scaling (GAS) is a versatile, under-utilized evaluation tool adaptable to a wide range of situations. GAS actively involves all partners in the evaluation process and has many benefits when used in community health settings. METHODS: The purpose of this paper is to describe the use of GAS as a potential means of measuring progress and outcomes in community health promotion and community development projects. GAS methodology was used in a local community of seniors (n = 2500; mean age = 76 +/- 8.06 SD; 77% female, 23% male) to a) collaboratively set health promotion and community partnership goals and b) objectively measure the degree of achievement, over- or under-achievement of the established health promotion goals. Goal attainment was measured in a variety of areas including operationalizing a health promotion centre in a local mall, developing a sustainable mechanism for recruiting and training volunteers to operate the health promotion centre, and developing and implementing community health education programs. Goal attainment was evaluated at 3 monthly intervals for one year, then re-evaluated again at year 2. RESULTS: GAS was found to be a feasible and responsive method of measuring community health promotion and community development progress. All project goals were achieved at one year or sooner. The overall GAS score for the total health promotion project increased from 16.02 at baseline (sum of scale scores = -30, average scale score = -2) to 54.53 at one year (sum of scale scores = +4, average scale score = +0.27) showing project goals were achieved above the expected level. With GAS methodology an amalgamated score of 50 represents the achievement of goals at the expected level. CONCLUSION: GAS provides a "participatory", flexible evaluation approach that involves community members, research partners and other stakeholders in the evaluation process. GAS was found to be "user-friendly" and readily understandable by seniors and other community partners not familiar with program evaluation.

Project description:BackgroundExtensive studies have been conducted to evaluate the pain relief effect of traditional Chinese medicine (TCM) interventions on patients with low back pain, including in China. However, there is a dearth in the literature documenting the diverse goals of TCM interventions, let alone the overall effect of such interventions. In this study, the goal attainment scaling (GAS) method was adopted to evaluate individualised TCM interventions on chronic low back pain.MethodsA pre-post intervention study was conducted on patients with chronic low back pain who received individualised TCM interventions in community health services. The study was undertaken in three community health centres in Hangzhou of China. A total of 165 eligible patients were invited, and 150 participated in the study, including 136 who completed both pre- and postintervention surveys. Each participant was asked to identify three to five intended goals from a pool of 26 outcome indicators and their corresponding expectations of these goals prior to the TCM interventions. Their conditions were rated against the selected indicators on a self-report five-point Likert scale before and after the TCM interventions, respectively. Gaps between the actual conditions and the expected goals were summed up for each participant and converted into a standardised GAS score, with a higher score indicating higher achievements, and 50 indicting patient expectations were met. Linear regression models were established to determine the factors associated with the pre-post GAS changes after adjustment for variations in other variables.ResultsOn average, an increase of 14.99 (SD = 9.81) in the GAS scores was achieved. This resulted in a mean GAS score of 48.33 (SD = 9.74) after the TCM interventions, falling slightly short (<2) of patient expectations. The multivariate linear regression models revealed that local residents, the retired, and those who perceived lower professional competency of their attending doctors had a smaller increase in the GAS scores after adjustment for variations in other variables.ConclusionThe individualised TCM interventions can help patients with low back pain to achieve their expected goals as measured by the GAS. Further studies are needed to better understand how patients set up their goals and the professional competency requirements to meet patient expectations.

Project description:BackgroundPatient engagement in decisions regarding their health care may lead to improved outcomes and improved adherence to treatment plans. While there are several options for involving patients in their health care, goal setting is a readily accessible method for physical therapists to increase the involvement of patients in health care decisions. Physical therapy goals are often generated by health care providers based on subjective information or standardized, fixed-item, patient-reported outcome measures. However, these outcome measures may not fully reveal the activity and participation limitations of individual patients. Goal attainment scaling (GAS) is a patient-centered approach that allows patients to set meaningful goals. While GAS has been shown to be reliable, valid, and sensitive to change in various populations, there is limited evidence in the United States on utilizing GAS in physical therapy for patients with chronic low back pain (LBP).ObjectiveThe purpose of this paper is to describe the protocol for a study to (1) develop a way to apply GAS procedures for physical therapists treating patients with chronic LBP in the United States and (2) test the feasibility of applying GAS procedures for chronic LBP in an outpatient physical therapy setting.MethodsThis study used a mixed methods design with 2 phases: qualitative and quantitative. The qualitative phase of the study employed focus groups of patients with chronic LBP to identify an inventory of goals that were important and measurable. A series of prompts was developed from this inventory to assist physical therapists in collaboratively establishing goals with patients in a clinical setting. The quantitative phase of the study pilot-tested the inventory developed in the qualitative phase in patients with chronic LBP to determine feasibility, reliability, validity, and responsiveness. We also plan to compare how well GAS reveals change over time relative to traditional, fixed-item, patient-reported measures.ResultsPhase 1 data collection was completed in June 2020, while data collection for phase 2 was performed between March 2021 and December 2021. We anticipate that this study will demonstrate that GAS can be implemented successfully by outpatient physical therapists, and that it will demonstrate clinically important changes in patients with chronic LBP.ConclusionsGAS represents an opportunity for patient-centered care in the physical therapy management of chronic LBP. While GAS is not new, it has never been studied in real-world physical therapy for chronic LBP in a clinical setting. Due to unique time and productivity constraints, for GAS to be successfully implemented in this environment, we must demonstrate that clinicians can be trained efficiently and reliably, that GAS can be implemented in a clinical setting in under 15 minutes, and that GAS is able to detect clinically meaningful changes in patient outcomes.International registered report identifier (irrid)DERR1-10.2196/32457.

Project description:BackgroundRecent phase 3 trials have shown an overall survival benefit in metastatic melanoma. We aimed to assess whether progression-free survival (PFS) could be regarded as a reliable surrogate for overall survival through a meta-analysis of randomised trials.MethodsWe systematically reviewed randomised trials comparing treatment regimens in metastatic melanoma that included dacarbazine as the control arm, and which reported both PFS and overall survival with a standard hazard ratio (HR). We correlated HRs for overall survival and PFS, weighted by sample size or by precision of the HR estimate, assuming fixed and random effects. We did sensitivity analyses according to presence of crossover, trial size, and dacarbazine dose.FindingsAfter screening 1649 reports and meeting abstracts published before Sept 8, 2013, we identified 12 eligible randomised trials that enrolled 4416 patients with metastatic melanoma. Irrespective of weighting strategy, we noted a strong correlation between the treatment effects for PFS and overall survival, which seemed independent of treatment type. Pearson correlation coefficients were 0·71 (95% CI 0·29-0·90) with a random-effects assumption, 0·85 (0·59-0·95) with a fixed-effects assumption, and 0·89 (0·68-0·97) with sample-size weighting. For nine trials without crossover, the correlation coefficient was 0·96 (0·81-0·99), which decreased to 0·93 (0·74-0·98) when two additional trials with less than 50% crossover were included. Inclusion of mature follow-up data after at least 50% crossover (in vemurafenib and dabrafenib phase 3 trials) weakened the PFS to overall survival correlation (0·55, 0·03-0·84). Inclusion of trials with no or little crossover with the random-effects assumption yielded a conservative statement of the PFS to overall survival correlation of 0·85 (0·51-0·96).InterpretationPFS can be regarded as a robust surrogate for overall survival in dacarbazine-controlled randomised trials of metastatic melanoma; we postulate that this association will hold as treatment standards evolve and are adopted as the control arm in future trials.FundingNone.

Project description:BackgroundRehabilitation in iNPH is suggested to be an important factor to improve patients' functions but there are lack of clinical trials evaluating the effect of rehabilitation interventions after shunt surgery in iNPH. The objective of this study was to evaluate the effect of a physical exercise programme and goal attainment for patients with idiopathic normal pressure hydrocephalus (iNPH) after surgery compared to a control group.MethodsThis was a dual centre randomised controlled trial with assessor blinding, intention-to-treat (ITT) and per protocol (PP) analysis. Individuals diagnosed with iNPH scheduled to undergo shunt surgery at the Linköping University Hospital in Linköping and Sahlgrenska University Hospital in Gothenburg, Sweden were consecutively eligible for inclusion. Inclusion was conducted between January 2016 and June 2018. The patients were randomised 1:1 using sequentially numbered sealed envelopes to receive either written exercise information (control group) or written information and an additional supervised high-intensity, functional exercise programme (HIFE) executed twice weekly over 12 weeks (exercise group). Preoperatively, the patients set individual goals. The primary outcome was change from baseline in the total iNPH scale score at the post-intervention follow-up. Secondary outcomes were goal attainment, and change in the separate scores of gait, balance, neuropsychology and continence and in the total score after 6 months.ResultsIn total, 127 participants were randomised to the exercise group (n = 62) and to the control group (n = 65). In the ITT population (exercise group, n = 50; control group, n = 59), there were no between-group differences in the primary outcome, but the attrition rate in the exercise group was high. The exercise group improved more than the control group in the balance domain scores after 6 months. Post-intervention, the PP exercise population achieved their set goals to a greater extent than the controls.ConclusionsAn additional effect of the 12-week HIFE-programme on the overall improvement according to the iNPH-scale after shunt surgery in iNPH was not shown. This could be due to high attrition rate. However, the long-term effect on balance and higher goal achievement indicate beneficial influences of supervised physical exercise. Trial registration clinicaltrials.gov, NCT02659111. Registered 20 January 2016, https://clinicaltrials.gov/ct2/show/NCT02659111.

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Statistical analysis of Goal Attainment Scaling endpoints in randomised trials.

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Statistical analysis of Goal Attainment Scaling endpoints in randomised trials.

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