Background: Allogeneic hematopoietic stem cell transplantation (HSCT) from an unrelated HLA-mismatched donor (MMUD) is one of the alternatives where an HLA-matched donor is not found. The aim of this study was to compare bone marrow (BM) versus peripheral blood stem cells (PBSC) as hematopoietic rescue following allogeneic unrelated mismatched stem cell transplantation (MMUD).
Methods: The patients were divided into two groups: 43 pediatric patients were treated with BM and 17 pediatric patients with PBSC. The study was registered at ClinicalTrials.gov NCT04598789.
Results: The 3-year Overall Survival (OS) was 74% versus 31% (p = .0011). Transplant related mortality (TRM) was 16% versus 33% (p = .025), and relapse incidence (RI) was 16% versus 35% (p = .005). The day-100 acute Graft-versus-host disease (GvHD) incidence grade II-IV and III-IV was 30% versus 28% (p = NS) and 17% versus 17% (p = NS). The 3-year chronic GvHD incidence was 22% versus 33% (p = NS).
Conclusion: Despite all the limits of this retrospective study we were able to show how the combination of BM and ATG is able to prevent GvHDs and guarantee a high OS. Future studies addressing the issue of a post-transplant cellular therapy approach may potentially reduce relapses when GvHD is absent.
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http://dx.doi.org/10.1111/petr.14233 | DOI Listing |
Pediatr Surg Int
December 2024
Department of Pediatric Critical Care, The Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel-Hashomer, Israel.
Background: Burns in children are often complex injuries, leading to prolonged length of stay (LOS) and significant morbidity. LOS in pediatric intensive care units (PICUs) is a key measure for evaluating illness severity, clinical outcomes, and quality of care. Accurate prediction of LOS is vital for improving care planning and resource allocation.
View Article and Find Full Text PDFChilds Nerv Syst
December 2024
Department of Children's and Adolescence Health, Division of Neuropediatrics, University Hospital Leipzig, Leipzig, Germany.
Introduction: Endoscopic Third Ventriculostomy (ETV) is a well-established treatment for pediatric hydrocephalus, particularly in cases of aqueductal stenosis. The ETV Success Score (ETVSS) is a predictive tool widely used to estimate the likelihood of ETV success based on factors like age. Its accuracy, especially in infants under 3 months, is still debated.
View Article and Find Full Text PDFVirulence
December 2025
Department of Epidemiology, College of Public Health, Zhengzhou University, Zhengzhou, Henan, China.
Amino acid metabolism provides significant insight into the development and prevention of many viral diseases. Therefore, the present study aimed to compare the amino acid profiles of hand, foot, and mouth disease (HFMD) patients with those of healthy individuals and to further reveal the molecular mechanisms of HFMD severity. Using UPLC-MS/MS, we determined the plasma amino acid expression profiles of pediatric patients with HFMD (mild, = 42; severe, = 43) and healthy controls ( = 25).
View Article and Find Full Text PDFGenet Med
December 2024
Movement Disorders Program, Department of Neurology and F.M. Kirby Neurobiology Center, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA. Electronic address:
Objectives: Biallelic HPDL variants have been identified as the cause of a progressive childhood-onset movement disorder, with a broad clinical spectrum from severe neurodevelopmental disorder to juvenile-onset pure hereditary spastic paraplegia type 83. This study aims at delineating the geno- and phenotypic spectra of patients with HPDL-related disease, quantitatively modelling the natural history, and uncovering genotype-phenotype associations.
Methods: A cross-sectional analysis of 90 published and one novel case was performed, employing a Human Phenotype Ontology-based approach.
Clin Pharmacol Ther
December 2024
Department of Pharmacy, Uppsala University, Uppsala, Sweden.
The complexity of the currently registered dosing schedules for bedaquiline and delamanid is a barrier to uptake in drug-resistant tuberculosis treatment across all ages. A simpler once-daily dosing schedule is critical to ensure patient-friendly regimens with good adherence. We assessed expected drug exposures with proposed once-daily doses for adults and compared novel model-informed once-daily dosing strategies for children with current World Health Organization (WHO) recommended dosing.
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