Immune thrombocytopenia (ITP) is a common acquired hemorrhagic disease. Conventional view considered its pathogenesis as the destruction of platelets induced by platelet associated antibodies, the target of treatment are inhibiting the production of antibodies and blocking the destruction of platelets in reticuloendothelial system, but they are ineffective in part of ITP patients, who transform to chronic/refractory ITP (C/RITP). As to children's C/RITP, the effect of first-line therapy is low, while the second-line therapy isn't effective definitely and has obvious side effects. The safe and effective second-line drugs to prevent disease progressing are urgently required. Recently, a pathogenesis that decrease the platelet production has been confirmed, thrombopoietic drugs, including thrombopoietin (TPO) and its receptor agonist (TRA), are under research and clinical application gradually. Recombinate human TPO (rhTPO) has accomplished Phase III clinical trails in adult C/RITP and tumor children. The Phase III clinical trails of romiplostim and eltrombopag, as the representative of TRA, in adult C/RITP have been performed. There are also two clinical trails of TRA for children's C/RITP, the efficacy and safety have been approved, with the convenience for using. In pediatric population, they have a good clinical application. In this article the research and development of thrombopoietic drugs and their perspective in pediatric clinical use are reviewed.
Child ; Clinical Trials, Phase III as Topic ; Humans ; Thrombocytopenia ; drug therapy ; etiology ; Thrombocytopenia, Neonatal Alloimmune ; drug therapy ; Thrombopoietin ; therapeutic use

OBJECTIVE: To investigate the pathogenesis and clinical diagnosis of fetal/neonatal alloimmune thrombocytopenia (FNAIT) and analyze the laboratory test results and clinical data related to the disease, so as to provide reference for clinical treatment and improvement of prognosis. METHODS: The clinical data of six neonatal patients with FNAIT in the Neonatology Department of our hospital from March 2017 to September 2020 were retrospectively analyzed, which included laboratory diagnosis, clinical symptoms, treatment, and prognosis. RESULTS: Among six patients, two cases occurred in the first pregnancy and four cases in the second pregnancy. The platelet count of six cases were decreased at admission or during hospitalization and maternal and neonatal serum autoimmune platelet antibody tests were positive. Five cases were accompanied by different degrees of skin and facial bleeding spots or petechiae and ecchymosis, intracranial hemorrhage. Four cases were treated with immunoglobulin and/or steroid hormone therapy (one of them received cross-matched platelets transfusion), while the symptoms of the other two cases improved spontaneously. Five cases recovered and were discharged from the hospital, while one case had not recovered but the family members requested to be discharged forwardly. Four cases were hospitalized within two weeks, but two cases were hospitalized for more than two weeks due to other diseases or factors (e.g., neonatal sepsis, neonatal enteritis, congenital heart disease, neonatal asphyxia, etc.). CONCLUSION FNAIT is characterized by decreased platelet count, with or without bleeding symptoms, and may occur in the first and following pregnancy. FNAIT can recover spontaneously or have a good prognosis after treatment. However, the complication with other diseases or factors may affect the prognosis.
Adult ; Aged ; Antigens, Human Platelet ; Data Analysis ; Female ; Hemorrhage ; Humans ; Infant, Newborn ; Middle Aged ; Platelet Count ; Platelet Transfusion/adverse effects* ; Pregnancy ; Retrospective Studies ; Thrombocytopenia, Neonatal Alloimmune/therapy*