rhTPO 北京上市英文.ppt

Safety and efficacy of rh-TPO, rh-TPO as a maintenance therapy? waiting for multicenter clinical trail rh TPO in ITP managementDiscussion Studies on two kinds of TPO receptor agonists were reported: Romiplastin（雷米斯汀） Eltrombopag（艾曲泊帕） rh TPO TPO receptor agonists in ITP management: -- maintenance treatment (ASH 2010, abstract # 67,68.) 292 adult ITP patients were involved, with median administration time of 78 weeks. 94.5% of the patients achieved a PC≥50×109/L. More than 50% had PC≥ 50×109/L on 90% of all visits. Romiplostim Long-term Treatment （Kuter et al. ASH2010，Abstract# 68） 299 adult ITP patients were involved, with an median administration time of 100 weeks. 87% of patients achieved a PC≥50×109/L. Median PC increased to PC≥50×109/L by week2 and remained consistently through week164. The incidence of any bleeding symptoms declined from 56% at baseline to 16% and 20% at week52 and week104 respectively. （Saleh et al. ASH2010，Abstract# 67） Eltrombopag Long-term treatment * * * 不论是致命性出血还是非致命性出血，60岁以上的人发生出血的机率高于40-60岁或是40岁以下的人。 * 慢性ITP患者生活质量低于癌症患者，与糖尿病患者相当。 * * * * * * * * * * * * * * * * * * * * * Response criteria Complete response (CR)： A platelet count ≥ 100 ×109/L measured on two occasions 7 days apart and the absence of bleeding. Response (R)： A platelet count ≥ 30 × 109/L and a greater than two fold increase in platelet count from baseline measured on two occasions 7 days apart and the absence of bleeding. No response (NR)： A platelet count 30 × 109/L or a less than two fold increase in platelet count from baseline or the presence of bleeding. Platelet count must be measured on two occasions more than a day apart. Agenda Background Consensus Guideline Pathophysiology Emerging Treatment Options Pathogenesis of ITP Lost of tolerance to self platelet antigens: Autoantibody-mediated platelet clearance CTL-mediated platelet lysis Dysmegakaryocytopoiesis Lost of tolerance to self platelet antigens Oligoclonal GP-specific T-lymp