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Introduction 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 are 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.Methods and analysis This is a single group study with sample size n=12 to investigate safety of single-dose intravenous 12/12 human leucocyte antigen-matched sibling cord blood cell infusion to children with CP aged 1–16 years without immune suppression. The study is similar to a 3+3 design, where the first two groups of participants have severe CP, and the final six participants include children with all motor severities. Children will be monitored for adverse events and the duration that donor cells are detected. Assessments at baseline, 3 and 12 months will investigate safety and preliminary evidence of change in gross motor, fine motor, cognitive and quality of life outcomes.Ethics and dissemination Full approval was obtained from The Royal Children’s Hospital Human Research Ethics Committee, and a clinical trial notification was accepted by Australia’s Therapeutic Goods Administration. Participant guardian informed consent will be obtained before any study procedures. The main results of this study will be submitted for publication in a peer-reviewed journal.Trial registration number ACTRN12616000403437, NCT03087110.

The response to medications in children differs not only in comparison to adults but also between children of the different age groups and according to the disease. This is true for anti-infectives that are widely prescribed in children with malignancy. In the absence of pharmacokinetic/pharmacodynamic paediatric studies, dosage is frequently based on protocols adapted to adults. After a short presentation of the drugs, we reviewed the population pharmacokinetic studies available for glycopeptides (vancomycin and teicoplanin, n = 5) and antifungals (voriconazole, posaconazole, and amphotericin B, n = 9) currently administered in children with onco-hematological malignancies. For each of them, we reported the main study characteristics including identified covariates affecting pharmacokinetics and proposed paediatric dosage recommendations. This review highlighted the very limited amount of data available, the lack of consensus regarding PK/PD targets used for dosing optimization and regarding dosage recommendations when available. Additional PK studies are urgently needed in this specific patient population. In addition to pharmacokinetics, efficacy may be altered in immunocompromised patients and prospective clinical evaluation of new dosage regimen should be provided as they are missing in most cases.