Determining minimally clinically important differences for outcome measures in patients with chronic motor deficits secondary to traumatic brain injury

Michael A McCrea; Steven C Cramer; David O Okonkwo; Soeren Mattke; Susan Paadre; Damien Bates; Bijan Nejadnik; Joseph T Giacino

doi:10.1080/14737175.2021.1968299

Determining minimally clinically important differences for outcome measures in patients with chronic motor deficits secondary to traumatic brain injury

Expert Rev Neurother. 2021 Sep;21(9):1051-1058. doi: 10.1080/14737175.2021.1968299. Epub 2021 Aug 26.

Authors

Michael A McCrea¹, Steven C Cramer², David O Okonkwo³, Soeren Mattke⁴, Susan Paadre⁵, Damien Bates⁶, Bijan Nejadnik⁷, Joseph T Giacino⁸

Affiliations

¹ Co-Director, Center For Neurotrauma Research; And Professor, Department Of Neurosurgery, Medical College Of Wisconsin, Milwaukee, USA.
² Professor, Department Of Neurology, University Of California, Los Angeles; Los Angeles, Ca; And Medical Director Of Research, California Rehabilitation Institute; Los Angeles, CA, USA.
³ Director, Neurotrauma Clinical Trials Center; And Professor, Department Of Neurological Surgery, University Of Pittsburgh Medical Center, Pittsburgh, PA, USA.
⁴ Director, Center For Improving Chronic Illness Care, USC Dornsife, Los Angeles, Ca, USA.
⁵ Associate Director, Biostatistics, Biostatistical Consulting Inc., Lexington, MA, USA.
⁶ Consultant, SanBio, Inc., Mountain View, CA, USA.
⁷ Chief Medical Officer, Global Head Of Regulatory, Medical Affairs, Research and Clinical Development, SanBio Inc., CA, USA.
⁸ Director Of Rehabilitation Neuropsychology; Director, SRN Disorders Of Consciousness Program; Project Director, Spaulding-Harvard TBI Model System, Spaulding Rehabilitation Hospital, Charlestown, MA; And Consulting Neuropsychologist, Department Of Psychiatry, Massachusetts General Hospital, Boston, MA; And Professor, Department Of Physical Medicine And Rehabilitation, Harvard Medical School, Boston, MA, USA.

PMID: 34402352
DOI: 10.1080/14737175.2021.1968299

Abstract

Objective: To determine minimally clinically important differences (MCIDs) for Disability Rating Scale (DRS), Fugl-Meyer Upper Extremity Subscale (FM-UE), Fugl-Meyer Lower Extremity Subscale (FM-LE), and Fugl-Meyer Motor Scale (FMMS) in patients with chronic motor deficits secondary to traumatic brain injury (TBI).

Methods: Retrospective analysis from the 1-year, double-blind, randomized, surgical sham-controlled, Phase 2 STEMTRA trial (NCT02416492), in which patients with chronic motor deficits secondary to TBI (N = 61) underwent intracerebral stereotactic implantation of modified bone marrow-derived mesenchymal stromal (SB623) cells. MCIDs for DRS, FM-UE, FM-LE, and FMMS were triangulated with distribution-based, anchor-based, and Delphi panel estimates.

Results: Triangulated MCIDs were: 1) -1.5 points for the Disability Rating Scale; 2) 6.2 points for the Fugl-Meyer Upper Extremity Subscale; 3) 3.2 points for the Fugl-Meyer Lower Extremity Subscale; and 4) 8.4 points for the Fugl-Meyer Motor Scale.

Conclusions: For the first time in the setting of patients with chronic motor deficits secondary to TBI, this study reports triangulated MCIDs for: 1) DRS, a measure of global outcome; and 2) Fugl-Meyer Scales, measures of motor impairment. These findings guide the use of DRS and Fugl-Meyer Scales in the assessment of global disability outcome and motor impairment in future TBI clinical trials.

Keywords: Brain injuries; Delphi technique; disability evaluation; humans; minimally clinically important difference; traumatic.

Publication types

Clinical Trial, Phase II
Randomized Controlled Trial
Research Support, Non-U.S. Gov't

MeSH terms

Brain Injuries, Traumatic* / complications
Disability Evaluation
Humans
Outcome Assessment, Health Care
Recovery of Function
Retrospective Studies
Stroke Rehabilitation*
Stroke*

Associated data

ClinicalTrials.gov/NCT02416492