Skip to main content

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.