RESUMO
Immune thrombocytopenia (ITP) is an immune-mediated acquired hemorrhagic autoimmune disease. At present, the first-line therapeutic drugs for ITP include glucocorticoids and intravenous immunoglobulins. However, about 1/3 of the patients had no response to the first-line treatment, or relapsed after dose reduction or withdrawal of glucocorticoids. In recent years, with the gradual deepening of the understanding on the pathogenesis of ITP, the drugs targeting different pathogenesis continually emerge, including immunomodulators, demethylating agents, spleen tyrosine kinase (SYK) inhibitors and neonatal Fc receptor (FcRn) antagonist. However, most of these drugs are in clinical trials. This review summarized briefly the recent advances in the treatment of glucocorticoids resistance and relapsed ITP, so as to provide reference for the clinical treatments.
Assuntos
Recém-Nascido , Humanos , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Glucocorticoides/uso terapêutico , Trombocitopenia , Imunoglobulinas Intravenosas/uso terapêutico , Fatores Imunológicos/uso terapêuticoRESUMO
Abstract Hematopoietic stem cells (HSCs), as a kind of adult stem cell, is first used in clinical practice. It has been widely used in hematopoietic stem cell transplantation and has broad application prospects in the field of gene therapy. However, the shortage of HSC is still the main bottleneck restricting the clinical application of HSC transplantation, especially cord blood HSC transplantation. With the continuous improvement of the concept of HSC and the in-depth study of the molecular mechanism regulating HSC self-renewal and differentiation, it have been explored that a variety of strategies for ex vivo expansion of HSC, such as adding small molecule compounds in vitro culture system, simulating bone marrow microenvironment, regulating HSC metabolism and biomaterial-based amplification methods. In this review, recent advances in research of ex vivo expansion strategy of HSC are summarized and discussed.