Goal attainment scaling as an outcome measure in rare disease trials: a conceptual proposal for validation

Gaasterland, C. M. W.; Weide, M. C. Jansen – van der; Roes, Kit C. B.; Lee, J. H. van der

doi:10.1186/s12874-019-0866-x

Cited by 28 publications

(43 citation statements)

References 36 publications

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“…This was also indicated by the frequent use of self-designed outcome measures in the included studies. Instruments such as patient-reported outcome measures, 44 , 50 Goal Attainment Scaling, 51 or experience-sampling methods 52 may be considered, enabling quantitative expression of meaningful subjective patient experiences while translating these into evidence. 43 As personalized outcome measures may compromise generalizability, inclusion of generalization measures can provide information on transfer effects of the intervention to other behaviors, settings, or disorders that may be either closely or distally related to the target behavior.…”

Section: Discussionmentioning

confidence: 99%

Systematic Review of N-of-1 Studies in Rare Genetic Neurodevelopmental Disorders

et al. 2021

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Objective:To improve the use of N-of-1 studies in rare genetic neurodevelopmental disorders, we systematically reviewed the literature and formulated recommendations for future studies.Methods:The systematic review protocol was registered in the PROSPERO International Prospective Register of Systematic Reviews (CRD42020154720). EMBASE and MEDLINE were searched for relevant studies. Information was recorded on types of interventions, outcome measures, validity, strengths and limitations using standard reporting guidelines and critical appraisal tools. Qualitative and descriptive analyses were performed.Results:Twelve studies met N-of-1 inclusion criteria, including both single trials and series. Interventions were mainly directed to neuropsychiatric manifestations. Main strengths were the use of personalized and clinically relevant outcomes in most studies. Generalizability was compromised due to limited use of validated and generalizable outcome measures.Conclusion:N-of-1 studies are sporadically reported in rare genetic neurodevelopmental disorders. Properly executed N-of-1 studies may provide a powerful alternative to larger randomized controlled trials in rare disorders and a much needed bridge between practice and science. We provide recommendations for future N-of-1 studies in rare genetic neurodevelopmental disorders, ultimately optimizing evidence-based and personalized care.

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Section: Discussionmentioning

confidence: 99%

Systematic Review of N-of-1 Studies in Rare Genetic Neurodevelopmental Disorders

et al. 2021

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“…Further, although GAS is useful, it is not a perfect measure. Further work needs to be done on analysis, 34 and on the approach to validation—especially the need to test in double‐blinded placebo‐controlled trials, 35 and to ensure that goals be set before randomization and unalterable thereafter.…”

Section: Discussionmentioning

confidence: 99%

The Clinician's Interview‐Based Impression of Change (Plus caregiver input) and goal attainment in two dementia drug trials: Clinical meaningfulness and the initial treatment response

Stanley¹,

Howlett

Dunn³

et al. 2021

Alzheimer's & Dementia

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INTRODUCTION The Clinician's Interview‐Based Impression of Change Plus caregiver input (CIBIC‐Plus) has been widely used in dementia drug trials to evaluate cognition, behavior, and function. New trials of symptomatic drugs forecast renewed interest in this measure. METHODS To test its clinical meaningfulness, we examined how CIBIC‐Plus performed in two cholinesterase inhibitor trials compared to goal attainment scaling Scale (GAS) scores, a patient‐reported outcome measure. RESULTS Net goal attainment was seen for all but one GAS domains in subjects who improved on the CIBIC‐Plus. Subjects who improved initially on CIBIC‐Plus scores were likely to remain improved across all other outcomes for each trial's duration, except for Disability Assessment for Dementia scores. DISCUSSION The initial response to treatment, as assessed by CIBIC‐Plus, remained stable for most outcome measures. Even small CIBIC‐Plus improvement changes are associated with clinically meaningful change as assessed by GAS. Other tests detect decline better than improvement.

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“…GAS is a relative measure, as there is no checkpoint for comparison. One of the main problems hindering the use of the method is the lack of validation (8) . A three-stage study conducted at intervals of 0-6-12 weeks with the questionnaire (GAS-Hem) for a patient with hemophilia showed little correlation with measures of the quality of life (QoL SF-36) (9) .…”

Section: Discussionmentioning

confidence: 99%

“…The creation of these goals should comply with the SMART criteria (Specific, Measurable, Agreed upon, Realistic, Time-related) (10) . Currently, GAS is used in rehabilitation, but also in pediatrics, urology, orthopedics, gynecology, and oncology (8,(11)(12)(13)(14) . Goal Attainment Scaling has also been used in a clinical trial of mastocytosis, a rare dermatological disease (15) .…”

Section: Discussionmentioning

confidence: 99%

Nomothetic EORTC IN-PATSAT 32 and idiographic GAS questionnaires in the assessment of surgical treatment in patients with endometrial cancer

Trawińska¹,

Skręt-Magierło²,

Raś³

et al. 2021

Curr Gynecol Oncol

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Introduction: Identification and attainment of the goals of cancer patients is an important aspect of personalized treatment. Aim of the study: The study aimed to assess the following aspects in patients treated surgically for endometrial cancer: 1) level of satisfaction with hospitalization using the EORTC IN-PATSAT32 nomothetic questionnaire; 2) degree of goals attainment using the Goal Attainment Scaling (GAS) idiographic questionnaire; 3) correlation between these evaluation methods. Material and method: The study included 123 patients with endometrial cancer (FIGO I–II) treated surgically at the Department of Obstetrics and Gynecology in Rzeszów in 2012–2014. EORTC IN-PATSAT32 and GAS questionnaires were used. The collected material was analyzed using the Statistica 10.0 software. Results: The overall level of satisfaction measured with the IN-PATSAT32 scale was 72.2 ± 20.5. The technical skills were rated the highest in nurses (74.5 ± 17.6) and doctors (69.3 ± 17.8), while the lowest score was awarded for hospital assess (54.7 ± 23.3). The overall satisfaction with care was 72.2 ± 20.5. In the personalized GAS scale, the patients listed individual expectations before the surgery, assigning ranks to their importance. For most of them, it was a very high (A) or high (B) rank. The patients assigned the highest ranks to quick mobilization, success of the operation, and willingness to be healthy. The average value of the level of goal attainment on the discharge date was 63.7 ± 9.4 points. Statistically significant correlations between the questionnaires were found for the level of goal attainment and the assessment of various aspects of hospital care. Conclusions: The study proved that the EORTC IN-PATSAT32 questionnaire was correlated with GAS questionnaire, and additionally provided knowledge about individual goals of care and the degree of their attainment. The use of nomothetic and idiographic tools gives wider possibilities in the planning and implementation of personalized care.

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Goal attainment scaling as an outcome measure in rare disease trials: a conceptual proposal for validation

Cited by 28 publications

References 36 publications

Systematic Review of N-of-1 Studies in Rare Genetic Neurodevelopmental Disorders

Systematic Review of N-of-1 Studies in Rare Genetic Neurodevelopmental Disorders

The Clinician's Interview‐Based Impression of Change (Plus caregiver input) and goal attainment in two dementia drug trials: Clinical meaningfulness and the initial treatment response

Nomothetic EORTC IN-PATSAT 32 and idiographic GAS questionnaires in the assessment of surgical treatment in patients with endometrial cancer

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