SAFETY OF SIBLING CORD BLOOD CELL INFUSION FOR CHILDREN WITH CEREBRAL PALSY
Notice bibliographique
Résumé
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.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,003 | 0,011 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,000 |
| Bibliométrie | 0,000 | 0,000 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,001 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,001 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,003 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».