AI Article Synopsis
- The new pediatric immune thrombocytopenia (ITP) guidelines update the disease's name and staging, introducing a modified bleeding assessment score.
- Treatment focuses on improving patients' health-related quality of life (HRQoL) by considering factors like platelet counts, bleeding symptoms, activity, and healthcare access.
- First-line treatments include IVIG and corticosteroids, while second-line options consist of thrombopoietin receptor agonists, rituximab, and splenectomy; new therapeutic targets are also under investigation.
Article Abstract
The new guidelines for pediatric immune thrombocytopenia (ITP) not only include changes to the name and staging of the disease, but also introduce the modified Buchanan's bleeding score for the assessment of bleeding symptoms. Treatments should aim to improve patients' health-related quality of life (HRQoL) based on a multidimensional assessment of not only platelet counts but also bleeding symptoms, as well as activity level, lifestyle, and access to healthcare. First-line therapy includes intravenous immunoglobulin therapy (IVIG) and short-term corticosteroids. Second-line therapy includes thrombopoietin receptor agonists, rituximab, and splenectomy. Many novel agents are also in development, with splenic-derived tyrosine kinase (Syk), Bruton's kinase (BTK), and fetal Fc receptor (FcRn) attracting attention as target molecules. Future developments in the treatment of pediatric ITP are eagerly awaited.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.11406/rinketsu.65.1209 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Kidney Injury Following Cardiac Surgery: A Review of Our Current Understanding.
Am J Cardiovasc Drugs
January 2025
Pediatric Nephrology, State University of Campinas, São Paulo, Brazil.
Around one-quarter of all patients undergoing cardiac procedures, particularly those on cardiopulmonary bypass, develop cardiac surgery-associated acute kidney injury (CSA-AKI). This complication increases the risk of several serious morbidities and of mortality, representing a significant burden for both patients and the healthcare system. Patients with diminished kidney function before surgery, such as those with chronic kidney disease, are at heightened risk of developing CSA-AKI and have poorer outcomes than patients without preexisting kidney injury who develop CSA-AKI.
View Article and Find Full Text PDFAn engineered adeno-associated virus mediates efficient blood-brain barrier penetration with enhanced neurotropism and reduced hepatotropism.
J Control Release
January 2025
Wuhan National Laboratory for Optoelectronics, Huazhong University of Science and Technology, 430074 Wuhan, PR China; Shenzhen Key Laboratory of Viral Vectors for Biomedicine, Shenzhen-Hong Kong Institute of Brain Science, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, 518055 Shenzhen, PR China; Key Laboratory of Magnetic Resonance in Biological Systems, State Key Laboratory of Magnetic Resonance and Atomic and Molecular Physics, National Center for Magnetic Resonance in Wuhan, Innovation Academy for Precision Measurement Science and Technology, Chinese Academy of Sciences, 430071 Wuhan, PR China; Key Laboratory of Quality Control Technology for Virus-Based Therapeutics, Guangdong Provincial Medical Products Administration, NMPA Key Laboratory for Research and Evaluation of Viral Vector Technology in Cell and Gene Therapy Medicinal Products, The Brain Cognition and Brain Disease Institute, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, 518055 Shenzhen, PR China; University of Chinese Academy of Sciences, 100049 Beijing, PR China; Center for Excellence in Brain Science and Intelligence Technology, Chinese Academy of Sciences, 200031 Shanghai, PR China. Electronic address:
The blood-brain barrier (BBB) is a formidable barrier that restricts the entry of substances into the brain, complicating the study of brain function and the treatment of neurological conditions. Traditional methods of delivering genes from the periphery to the central nervous system (CNS) using adeno-associated viruses (AAVs) often require high doses, which can trigger immune responses and hepatotoxicity. Here, we developed a new AAV variant named AAVhu.
View Article and Find Full Text PDFMass doses of fractional pneumococcal vaccine in humanitarian settings.
Lancet Infect Dis
January 2025
Every Breath Counts Coalition, New York, NY, USA.
DNA-binding affinity and specificity determine the phenotypic diversity in BCL11B-related disorders.
Am J Hum Genet
January 2025
Institute of Human Genetics, University Medical Center Hamburg-Eppendorf, 20246 Hamburg, Germany; Institute of Human Genetics, University of Regensburg, 93053 Regensburg, Germany; Institute of Clinical Human Genetics, University Hospital Regensburg, 93053 Regensburg, Germany. Electronic address:
BCL11B is a Cys2-His2 zinc-finger (C2H2-ZnF) domain-containing, DNA-binding, transcription factor with established roles in the development of various organs and tissues, primarily the immune and nervous systems. BCL11B germline variants have been associated with a variety of developmental syndromes. However, genotype-phenotype correlations along with pathophysiologic mechanisms of selected variants mostly remain elusive.
View Article and Find Full Text PDFImmunogenicity of yellow fever vaccine co-administered with 13-valent pneumococcal conjugate vaccine in rural Gambia: A cluster-randomised trial.
Vaccine
January 2025
Medical Research Council Unit The Gambia at London School of Hygiene & Tropical Medicine, Banjul, the Gambia; Department of Disease Control, Faculty of Infectious and Tropical Diseases, London School of Hygiene & Tropical Medicine, London, UK; Murdoch Children's Research Institute, Melbourne, Australia; Department of Paediatrics, University of Melbourne, Australia.
Introduction: Because booster doses of pneumococcal conjugate vaccine (PCV) may be given at a similar time to yellow fever vaccine (YF), it is important to assess the immune response to YF when co-administered with PCV. This has been investigated during a reduced-dose PCV trial in The Gambia.
Methods: In this phase 4, parallel-group, cluster-randomized trial, healthy infants aged 0-10 weeks were randomly allocated to receive either a two-dose schedule of PCV13 with a booster dose co-administered with YF vaccine at age 9 months (1 + 1 co-administration) or YF vaccine administered separately at age 10 months (1 + 1 separate) or the standard three early doses of PCV13 with YF vaccine at age 9 months (3 + 0 separate).
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!