The first oral tpo-ra drug for the treatment of primary immune thrombocytopenia in China

Recently, Novartis tumor (China) announced that its non peptide oral thrombopoietin receptor agonist (tpo-ra) etrapa ethanolamine tablet is on the market in China and will be used to treat thrombocytopenia in patients with chronic idiopathic immune thrombocytopenia (ITP) after glucocorticoids, immunoglobulins or splenectomy As the optimal scheme of ITP second-line treatment, the drug can not only rapidly increase the number of platelets, but also use convenient oral way, which will help to improve the compliance of patients "ITP" is a platelet killer hidden around people Compared with leukemia, the public has relatively less knowledge of primary immune thrombocytopenia (ITP) In summer, nosebleeds or gums bleed from the fire, and purple spots appear on the skin after slight collision, which is a common small bleeding problem in life However, if bleeding is accompanied by obvious weakness, attention should be paid to it, which may be the symptoms of ITP ITP, formerly known as idiopathic thrombocytopenic purpura, is caused by the fact that platelets of "blood clotting guard" in human body can not reach the normal number due to some acquired autoimmune reasons, resulting in bleeding and non coagulation of patients In daily life, once the number of platelets in patients decreases, spontaneous bleeding and subcutaneous purpura will occur frequently, and even sudden internal and intracranial hemorrhage will occur in serious cases, which is life-threatening On the other hand, ITP patients are accompanied by severe fatigue and low spirit all the year round, which greatly affects the quality of life of patients, and even can not work and study normally Thrombocytopenia is the key to the existing treatment problems It is known that the current treatment of ITP can be divided into first-line and second-line treatment Glucocorticoid is usually used in the first-line treatment Although it is effective in a short time, there are many side effects, such as osteoporosis, necrosis of the femoral head, hypertension, diabetes, acute gastric mucosal disease and other adverse reactions It is self-evident that the harm of teenagers' long-term use of hormone is that becoming "little fat" is not only small, but also may affect their growth and development In addition, more than half of the patients who received hormone therapy were ineffective or relapsed Therefore, second-line treatment is particularly important for the long-term treatment of ITP patients Yang renchi, Professor of thrombus hemostasis diagnosis and treatment center of Hematology hospital, Institute of Hematology, Chinese Academy of Medical Sciences, pointed out that the existing second-line treatment schemes are not perfect and each has its own risks Among them, the overall effective rate of immunosuppressive therapy is not high, and the risk of infection is high; while the effective rate of splenectomy is high, but patients have the risk of short-term and long-term complications In addition, many patients in the second-line treatment of poor results, in order to ensure the low risk period of platelets, platelet transfusion will be carried out directly, not only in the safety of blood sources, but also in the case of local blood tension in various provinces and cities, many treatment is not convenient Innovative research and development help ITP patients usher in the era of rapid upgrading Is there a second-line treatment drug that can not only rapidly improve platelets, reduce the risk of treatment, but also improve the quality of life of patients? With the launch of etrapopa ethanolamine, it is possible to do so First of all, unlike the mechanism of first-line glucocorticoid therapy, which mainly reduces platelet clearance, etrapacetamide tablet is not limited to "throttling", but more helpful to "open source" It interacts selectively with the transmembrane domain of TPO receptor and increases platelet production, which makes the platelet level of ITP patients rise rapidly to a safe level Second, the drug does not compete with endogenous TPO, does not induce TPO antibody, and can achieve stable and predictable platelet count rise A number of studies have shown that about 30% of ITP patients can achieve long-term remission after drug withdrawal Third, as the only oral TPO receptor agonist on the market in China, the drug changes the treatment mode that patients need to be injected regularly, which helps to improve treatment compliance and improve the quality of life of patients Professor Hu Yu, Affiliated Union Hospital of Tongji Medical College, Huazhong University of science and technology, pointed out: "as a second-line treatment recommended drug, etrap ethanolamine tablet will be listed in China to improve the therapeutic effect of ITP in China However, marketing is the first step All experts are concerned about how to enable more people to use innovative drugs for ITP treatment as soon as possible Therefore, it is hoped that the drug can enter the medical insurance as soon as possible, help more and more ITP patients get good curative effect and better treatment experience in the future, get out of the disease haze as soon as possible, and return to a happy life "