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Table 3 Study endpoints

From: Intravenous transplantation of mesenchymal stem cells preconditioned with early phase stroke serum: current evidence and study protocol for a randomized trial

Objective Endpoint
Primary endpoint of efficacy:  
Functional outcome Categorical shift in mRS at 90 days after treatment
Secondary endpoints of efficacy:  
Disability Change in NIHSS between pretreatment and 90 days post-treatment ≥5 points improvement or score of 0 to 2 on NIHSS score at 14 days after treatment
Functional outcome mRS ≤2 at 90 days after treatment
Change of mRS between pretreatment and 90 days post-treatment
mBI ≥60 at 90 days after treatment
Change of mBI between pretreatment and 90 days post-treatment
Further demonstration and characteristics of motor recovery Change of gross motor function between pretreatment and 90 days post-treatment
Motricity index and Fugl-Meyer assessment (upper, lower)
Change of fine motor function between pretreatment and 90 days post-treatment
Purdue pegboard test (simple) and box and block test
Change of mobility between pretreatment and 90 days post-treatment
Functional ambulatory category and 10-m gait speed
Cognition Change of MMSE between pretreatment and 90 days post-treatment
Quality of life Change of EQ-5D between pretreatment and 90 days post-treatment
Secondary endpoints of safety:  
Death All causes of death
Recurrence Recurrent stroke or transient ischemic attack
The immediate reaction Allergic reactions (tachycardia, fever, skin eruption, leukocytosis)
Local complications (hematoma or local infection at the site of bone marrow aspiration)
Vascular obstruction (tachypnea, oliguria, or peripheral vascular insufficiency)
Systemic complications (infections, AST/ALT, or BUN/Cr levels)
Long-term adverse effects possibly related to MSC treatment Tumor formation (physical examination, plain X ray, f/u MRI at 90 days after treatment)
Aberrant connections (newly diagnosed seizure or arrhythmia)
Other parameters related to efficacy:  
Exploration of biomarkers to further demonstrate the mechanism of action and genetic profile SDF-1α (chemokine)
S100β (protection and regeneration)
HIF-1 (preconditioning)
Circulating MSCs and MSC-derived microparticles
BDNF levels and polymorphisms and VEGF levels
Multimodal MRI Resting-state functional MRI and diffusion tensor imaging
Neurophysiologic study Motor evoked potentials
  1. ALT alanine aminotransferase, AST aspartate aminotransferase, BDNF brain-derived neurotrophic factor, BUN blood urea nitrogen, Cr creatinine, DWI diffusion-weighted image, EQ-5D EuroQol Five Dimensions, HBV hepatitis B virus, hCG human chorionic gonadotropin, HCV hepatitis C virus, HIF hypoxia-inducible factor, mBI modified Barthel index, MCA middle cerebral artery, MMSE Mini-Mental State Examination, mRS modified Rankin Score, MSC mesenchymal stem cell, NIHSS National Institutes of Health Stroke Scale, SDF-1α, stromal cell-derived factor 1α, VEGF vascular endothelial growth factor.