SAFETY OF SIBLING CORD BLOOD CELL INFUSION FOR CHILDREN WITH CEREBRAL PALSY
Bibliographic record
Abstract
Background: Cerebral palsy (CP) is the most common physical disability of childhood but has no cure. Stem cells have the potential to improve brain injury and have been proposed as a therapy for CP. However, many questions remain unanswered about the most appropriate cell type, timing of infusions, dose required, and associated risks. Therefore, human safety and efficacy trials are necessary to progress knowledge in the field. Aims: The primary objective of this study was to gain preliminary information on the safety of 12/12 HLA matched sibling umbilical cord blood cell (UCBC) infusions in children aged 1-16 years with CP who do not have immune suppression. The secondary objectives of this study were to gain preliminary information on the effectiveness of 12/12 HLA matched UCBC infusion relative to baseline, the length of time that infused matched sibling UCBCs remain within recipients and the mechanistic activity of UCBCs. Methods: This was a single group open-labelled study of 12 participants. UCBCs were sourced from TGA licenced private cord blood banks. The study first recruited 6 participants with severe CP before recruiting 6 participants with all motor severities with a review by an independent data and safety monitoring committee of the study data after every 3 participants prior to continuing the study. Children were monitored for adverse events and the duration that donor cells were detected using a highly sensitive surrogate chimerism analysis of donor DNA. Assessments at baseline, 3, and 12 months investigated the safety and change in gross motor, fine motor, cognitive, and quality of life outcomes from baseline. Results: UBCB cell infusions appeared to be safe, with only one serious adverse event, an anaphylactic reaction to cryoprotectant, a known potential risk of stem cell infusion. No participant had detectable donor DNA at or beyond six months post-infusion, indicating no evidence of engraftment and low risk of graft versus host disease. There was little change in gross motor function from baseline at 3 months. However, assessment with the Quality of Upper Extremity Skills Test (QUEST) implied improvement greater than could be expected from natural history and maturation at 3 months post-infusion for some of the children (a change from baseline of 8.57 (95% CI -0.93 -18.07) at 3 months, compared with a change of 4.89 which is regarded as clinically important. Conclusion: Preliminary results suggest that there are no serious adverse reactions related to UCBCs and that there is not cell persistence, but there may be some gains in the quality of upper limb function. However, numbers were too small to make any firm conclusions. Further appropriately powered studies are needed to provide information for families and professionals as to whether this is a worthwhile treatment option.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.003 | 0.011 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.001 | 0.001 |
| Insufficient payload (model declined to judge) | 0.003 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".