Using the Barthel Index and modified Rankin Scale as Outcome Measures for Stroke Rehabilitation Trials; A Comparison of Minimum Sample Size Requirements

Authors: McGill, K., Pandyan, A. et al.

Journal: Journal of Stroke and Cerebrovascular Diseases

Volume: 31

Issue: 2

eISSN: 1532-8511

ISSN: 1052-3057

DOI: 10.1016/j.jstrokecerebrovasdis.2021.106229

Abstract:

Objectives: Underpowered trials risk inaccurate results. Recruitment to stroke rehabilitation randomised controlled trials (RCTs) is often a challenge. Statistical simulations offer an important opportunity to explore the adequacy of sample sizes in the context of specific outcome measures. We aimed to examine and compare the adequacy of stroke rehabilitation RCT sample sizes using the Barthel Index (BI) or modified Rankin Scale (mRS) as primary outcomes. Methods: We conducted computer simulations using typical experimental event rates (EER) and control event rates (CER) based on individual participant data (IPD) from stroke rehabilitation RCTs. Event rates are the proportion of participants who experienced clinically relevant improvements in the RCT experimental and control groups. We examined minimum sample size requirements and estimated the number of participants required to achieve a number needed to treat within clinically acceptable boundaries for the BI and mRS. Results: We secured 2350 IPD (18 RCTs). For a 90% chance of statistical accuracy on the BI a rehabilitation RCT would require 273 participants per randomised group. Accurate interpretation of effect sizes would require 1000s of participants per group. Simulations for the mRS were not possible as a clinically relevant improvement was not detected when using this outcome measure. Conclusions: Stroke rehabilitation RCTs with large sample sizes are required for accurate interpretation of effect sizes based on the BI. The mRS lacked sensitivity to detect change and thus may be unsuitable as a primary outcome in stroke rehabilitation trials.

https://eprints.bournemouth.ac.uk/36620/

Source: Scopus

Using the Barthel Index and modified Rankin Scale as Outcome Measures for Stroke Rehabilitation Trials; A Comparison of Minimum Sample Size Requirements.

Authors: McGill, K., Sackley, C., Godwin, J., Gavaghan, D., Ali, M., Ballester, B.R., Brady, M.C. and VISTA-Rehabilitation collaborators

Journal: J Stroke Cerebrovasc Dis

Volume: 31

Issue: 2

Pages: 106229

eISSN: 1532-8511

DOI: 10.1016/j.jstrokecerebrovasdis.2021.106229

Abstract:

OBJECTIVES: Underpowered trials risk inaccurate results. Recruitment to stroke rehabilitation randomised controlled trials (RCTs) is often a challenge. Statistical simulations offer an important opportunity to explore the adequacy of sample sizes in the context of specific outcome measures. We aimed to examine and compare the adequacy of stroke rehabilitation RCT sample sizes using the Barthel Index (BI) or modified Rankin Scale (mRS) as primary outcomes. METHODS: We conducted computer simulations using typical experimental event rates (EER) and control event rates (CER) based on individual participant data (IPD) from stroke rehabilitation RCTs. Event rates are the proportion of participants who experienced clinically relevant improvements in the RCT experimental and control groups. We examined minimum sample size requirements and estimated the number of participants required to achieve a number needed to treat within clinically acceptable boundaries for the BI and mRS. RESULTS: We secured 2350 IPD (18 RCTs). For a 90% chance of statistical accuracy on the BI a rehabilitation RCT would require 273 participants per randomised group. Accurate interpretation of effect sizes would require 1000s of participants per group. Simulations for the mRS were not possible as a clinically relevant improvement was not detected when using this outcome measure. CONCLUSIONS: Stroke rehabilitation RCTs with large sample sizes are required for accurate interpretation of effect sizes based on the BI. The mRS lacked sensitivity to detect change and thus may be unsuitable as a primary outcome in stroke rehabilitation trials.

https://eprints.bournemouth.ac.uk/36620/

Source: PubMed

Using the Barthel Index and modified Rankin Scale as Outcome Measures for Stroke Rehabilitation Trials; A Comparison of Minimum Sample Size Requirements.

Authors: McGill, K., Sackley, C., Godwin, J., Gavaghan, D., Ali, M., Ballester, B.R., Brady, M.C. and VISTA-Rehabilitation collaborators

Journal: Journal of stroke and cerebrovascular diseases : the official journal of National Stroke Association

Volume: 31

Issue: 2

Pages: 106229

eISSN: 1532-8511

ISSN: 1052-3057

DOI: 10.1016/j.jstrokecerebrovasdis.2021.106229

Abstract:

Objectives

Underpowered trials risk inaccurate results. Recruitment to stroke rehabilitation randomised controlled trials (RCTs) is often a challenge. Statistical simulations offer an important opportunity to explore the adequacy of sample sizes in the context of specific outcome measures. We aimed to examine and compare the adequacy of stroke rehabilitation RCT sample sizes using the Barthel Index (BI) or modified Rankin Scale (mRS) as primary outcomes.

Methods

We conducted computer simulations using typical experimental event rates (EER) and control event rates (CER) based on individual participant data (IPD) from stroke rehabilitation RCTs. Event rates are the proportion of participants who experienced clinically relevant improvements in the RCT experimental and control groups. We examined minimum sample size requirements and estimated the number of participants required to achieve a number needed to treat within clinically acceptable boundaries for the BI and mRS.

Results

We secured 2350 IPD (18 RCTs). For a 90% chance of statistical accuracy on the BI a rehabilitation RCT would require 273 participants per randomised group. Accurate interpretation of effect sizes would require 1000s of participants per group. Simulations for the mRS were not possible as a clinically relevant improvement was not detected when using this outcome measure.

Conclusions

Stroke rehabilitation RCTs with large sample sizes are required for accurate interpretation of effect sizes based on the BI. The mRS lacked sensitivity to detect change and thus may be unsuitable as a primary outcome in stroke rehabilitation trials.

https://eprints.bournemouth.ac.uk/36620/

Source: Europe PubMed Central

Using the Barthel Index and modified Rankin Scale as Outcome Measures for Stroke Rehabilitation Trials; A Comparison of Minimum Sample Size Requirements.

Authors: McGill, K., Pandyan, A. et al.

Journal: Journal of Stroke and Cerebrovascular Diseases

Volume: 31

Issue: 2

ISSN: 1052-3057

Abstract:

OBJECTIVES: Underpowered trials risk inaccurate results. Recruitment to stroke rehabilitation randomised controlled trials (RCTs) is often a challenge. Statistical simulations offer an important opportunity to explore the adequacy of sample sizes in the context of specific outcome measures. We aimed to examine and compare the adequacy of stroke rehabilitation RCT sample sizes using the Barthel Index (BI) or modified Rankin Scale (mRS) as primary outcomes. METHODS: We conducted computer simulations using typical experimental event rates (EER) and control event rates (CER) based on individual participant data (IPD) from stroke rehabilitation RCTs. Event rates are the proportion of participants who experienced clinically relevant improvements in the RCT experimental and control groups. We examined minimum sample size requirements and estimated the number of participants required to achieve a number needed to treat within clinically acceptable boundaries for the BI and mRS. RESULTS: We secured 2350 IPD (18 RCTs). For a 90% chance of statistical accuracy on the BI a rehabilitation RCT would require 273 participants per randomised group. Accurate interpretation of effect sizes would require 1000s of participants per group. Simulations for the mRS were not possible as a clinically relevant improvement was not detected when using this outcome measure. CONCLUSIONS: Stroke rehabilitation RCTs with large sample sizes are required for accurate interpretation of effect sizes based on the BI. The mRS lacked sensitivity to detect change and thus may be unsuitable as a primary outcome in stroke rehabilitation trials.

https://eprints.bournemouth.ac.uk/36620/

Source: BURO EPrints