ABSTRACT
BACKGROUND AND OBJECTIVE: Immune thrombocytopenia (ITP) is a common bleeding disorder in childhood. The management of ITP in children is controversial, requiring personalized assessment of patients and therapeutic choices. Thrombopoietin receptor agonists (TPO-RAs), eltrombopag and romiplostim, have been shown to be safe and effective for the treatment of pediatric ITP. The aim of our research is to define the role of thrombopoietin receptor agonists in the management of pediatric ITP. METHODS: This review focuses on the use of TPO-RAs in pediatric ITP, in randomized trials and in clinical routine, highlighting their key role in the management of the disease. RESULTS: Eltrombopag and romiplostim appear effective treatment options for children with ITP. Several clinical studies have assessed that the use of TPO-RAs increases platelet count, decreases bleeding symptoms and improves health-related quality of life. Moreover, TPO-RAs are well tolerated with minor side effects. CONCLUSION: Although long term efficacy and safety of TPO-RAs still require further investigations, their use is gradually expanding in the clinical practice of children with ITP.
Subject(s)
Benzoates/therapeutic use , Blood Platelets/drug effects , Hydrazines/therapeutic use , Purpura, Thrombocytopenic, Idiopathic/drug therapy , Pyrazoles/therapeutic use , Receptors, Fc/therapeutic use , Receptors, Thrombopoietin/agonists , Recombinant Fusion Proteins/therapeutic use , Thrombopoietin/therapeutic use , Adolescent , Age Factors , Benzoates/adverse effects , Blood Platelets/immunology , Blood Platelets/metabolism , Child , Child, Preschool , Evidence-Based Medicine , Female , Humans , Hydrazines/adverse effects , Infant , Male , Purpura, Thrombocytopenic, Idiopathic/blood , Purpura, Thrombocytopenic, Idiopathic/diagnosis , Purpura, Thrombocytopenic, Idiopathic/immunology , Pyrazoles/adverse effects , Quality of Life , Randomized Controlled Trials as Topic , Receptors, Thrombopoietin/metabolism , Recombinant Fusion Proteins/adverse effects , Signal Transduction , Thrombopoietin/adverse effects , Treatment OutcomeABSTRACT
We measured hormonal levels in blood samples from pulmonary and radial arteries in 117 patients undergoing aorto-coronary by-pass surgery with the aim of investigating the role of the pulmonary vessel endothelium in hormone metabolism. Insulin and glucagon concentrations were significantly higher in pulmonary artery blood with respect to radial artery blood (73 +/- 65 vs. 65 +/- 47 pmol/l, p < 0.005, and 80 +/- 49 vs. 73 +/- 51 ng/l, p < 0.01, respectively), while no difference was found for growth hormone, prolactin, C peptide, insulin-like growth factor I, follicle stimulating hormone, luteinizing hormone, thyroid stimulating hormone, parathyroid hormone, thyroglobulin, triiodothyronine, thyroxine, free triiodothyronine, and free thyroxine. Moreover, prolactin concentrations were more than twice the normal levels, this being an effect of propafol and the opiate fentanyl used for the general anesthesia. Assuming that the arteriovenous differences observed are a marker of peptide hormone degradation, our study has demonstrated that with similar kinetics insulin and glucagon secreted into portal circulation and escaping from hepatic extraction undergo further homeostatic removal of about 9-10 % in the pulmonary circulation before entering the general circulation.
