Motor function and safety after allogeneic cord blood and cord tissue-derived mesenchymal stromal cells in cerebral palsy: An open-label, randomized trial.
Jessica M Sun, Laura E Case, Colleen McLaughlin and 7 others
PMID 35811372WHAT IT FOUND
Allogeneic cord blood infusion showed greater gross motor improvement than natural history at 12 months on the GMFM-66.
This was a post hoc finding in an underpowered safety trial. No significant difference was found for cord tissue-derived cells.
Key findings
01The primary endpoint, actual-minus-expected change in GMFM-66, showed significant improvement in all three groups, but the study was not powered to detect differences between them.
02In post hoc analysis, the cord blood group had a mean 12-month GMFM-66 score 3.3 points higher than the natural history group, a difference that overlaps with the minimal clinically important difference.
03Cord tissue-derived mesenchymal stromal cells did not show a significant difference in motor function compared to natural history at 12 months.
STILL TO COME
How it was doneWhat they foundWhat it means for PTsWhat it means for OTs
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What it does not show
The study was designed as a safety and feasibility trial and was not powered to detect differences between treatment groups. Families were not blinded to treatment assignment, which could introduce bias in daily care or reporting, although the assessing therapist was blinded. The trial was disrupted by the COVID-19 pandemic, preventing approximately a quarter of participants from completing the 12-month in-person evaluations. The positive finding for cord blood was a post hoc analysis of an exploratory outcome, not the prespecified primary endpoint. The study included children with different causes of CP (stroke, hypoxic-ischemic encephalopathy, and periventricular leukomalacia), which may affect generalizability to specific subgroups.
Declared interests
The work was supported by The Marcus Foundation. The authors declared no other conflicts of interest.
The easy way to misread this
Do not interpret the 3.3-point difference in GMFM-66 scores as proof that cord blood is effective. This was a post hoc finding in a small trial that was not powered for comparisons, and the primary endpoint showed no significant difference between the groups.