近日，美国FDA批准Shionogi的Mulpleta（lusutrombopag）用于治疗慢性肝病引起的成人血小板减少症。这是美国FDA今年批准的第26款新药。
批准日期：2018年8月1日 公司：Shionogi
MULPLETA（艾曲波帕 lusutrombopag）片剂,用于口服
美国最初批准：2018年
作用机制
Lusutrombopag是一种口服生物可利用的小分子TPO受体激动剂，与巨核细胞上表达的人TPO受体的跨膜结构域相互作用，诱导巨噬细胞祖细胞从造血干细胞和巨核细胞成熟中增殖和分化。
适应症和用法
MULPLETA是一种血小板生成素受体激动剂，用于治疗预定接受手术的慢性肝病患者的血小板减少症。
剂量和给药
在预定程序开始前8-14天开始MULPLETA给药。
患者应在最后一次给药后2-8天进行手术。
推荐用量：每日口服3毫克，每日一次，不含食物，持续7天。
剂量形式和强度
片剂：3mg。
禁忌症
没有。
警告和注意事项
血栓形成/血栓栓塞并发症：MULPLETA是血小板生成素（TPO）受体激动剂，TPO受体激动剂与慢性肝病患者的血栓形成和血栓栓塞并发症有关。监测血小板计数和血栓栓塞事件，并立即进行治疗。
不良反应
最常见的不良反应（3％）：头痛。
要报告可疑的不良反应，请致电1-800-849-9707联系SMART公司，或致电1-800-FDA-1088或www.fda.gov/medwatch联系FDA。
用于特定人群
哺乳期：治疗期间不建议母乳喂养。
包装提供/存储和处理
MULPLETA以含有7片-NDC 59630-551-07的儿童抗性泡罩包装中的3mg lusutrombopag片剂提供。
将MULPLETA存放在20°C至25°C（68°F至77°F）的原始包装中; 允许偏移15°C至30°C（59°F至86°F）[见USP受控室温]。

Mulpleta Approved for Thrombocytopenia in Patients With Chronic Liver Disease
The Food and Drug Administration (FDA) has approved Mulpleta (lusutrombopag; Shionogi) for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.
The approval was based on data from 2 randomized, double-blind, placebo-controlled trials (L-PLUS 1 [N=97] and L-PLUS 2 [N=215]) involving patients with chronic liver disease who were undergoing an invasive procedure and had a platelet count <50 x 109/L. Patients were randomized to Mulpleta 3mg or placebo once daily for 7 days. In L‐PLUS 1, the major efficacy outcome was the proportion of patients who required no platelet transfusion prior to the primary invasive procedure, while in L‐PLUS 2, it was the proportion of patients who required no platelet transfusion prior to the primary invasive procedure and no rescue therapy for bleeding from randomization through 7 days after the primary invasive procedure. In both trials, responders were defined as patients who had a platelet count of ≥50 x 109/L with an increase of ≥20 x 109/L from baseline.
In L-PLUS 1, 78% of patients (38/49) receiving Mulpleta required no platelet transfusion prior to the invasive procedure, compared with 13% (6/48) who received placebo (treatment difference: 64%; P <.0001). Seventy-six percent of Mulpleta-treated patients were considered responders vs 6% of patients in the placebo arm (treatment difference: 68%; P <.0001).
In L-PLUS 2, 65% (70/108) of patients who received Mulpleta required no platelet transfusion prior to the invasive procedure or rescue therapy for bleeding through 7 days after the procedure, compared with 29% (31/107) receiving placebo (treatment difference: 37%; P <.0001). Sixty-five percent of Mulpleta-treated patients were considered responders vs 13% of patients in the placebo group (treatment difference: 52%; P <.0001).
The most common adverse reaction associated with therapy in clinical trials was headache.
Mulpleta, a thrombopoietin receptor agonist, will be supplied in 3mg strength tablets in blister packs containing 7 tablets.
https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/210923s000lbl.pdf
https://www.shionogi.com/pdf/pi/wp-content/themes/pdfs/mulpleta.pdf