艾曲波帕治疗难治性获得性纯红细胞再生障碍疗效研究

Eltrombopag for the treatment of refractory acquired pure red cell aplasia

目的探讨艾曲波帕治疗难治性获得性纯红细胞再生障碍(pure red cell aplasia,PRCA)疗效。方法对2018年3月至2019年5月间,南京医科大学第一附属医院江苏省人民医院收治的3例难治性获得性成人PRCA患者采用艾曲波帕(75mg/d)治疗,评估疗效及耐受性。结果3例患者的红细胞计数(P=0.039)、血红蛋白浓度(P=0.018)、网织红细胞百分比(P=0.046)较治疗前显著升高;血小板计数均较治疗前升高(P=0.024),2例患者治疗后白细胞计数及中性粒细胞绝对计数较前升高,1例较前下降,但仍维持在正常范围(P=0.924;P=0.565)。1例总胆红素(TBIL)、直接胆红素(DBIL)升高,1例丙氨酸氨基转移酶(ALT)、天冬氨酸氨基转移酶(AST)、血肌酐(Scr)升高,1例心悸,经对症处理后好转。结论艾曲波帕对于难治性获得性PRCA患者,有一定疗效,且耐受性良好,值得进一步探索。

Objective To investigate the efficacy of eltrombopag in the treatment of refractory acquired pure red cell aplasia(PRCA). Methods Three patients with refractory acquired PRCA treated in the First Affiliated Hospital of Nanjing Medical University/Jiangsu Province Hospital from March 2018 to May 2019 were treated with eltrombopag(75 mg/d). The clinical data were collected for evaluating efficacy and tolerance. Results The erythrocyte count(P=0.039), hemoglobin concentration(P=0.018) and reticulocyte percentage(P=0.046) in 3 patients were significantly higher than those before treatment. The platelet count was higher than that before treatment(P=0.024). The leukocyte count and absolute neutrophil count increased in 2 patients, and decreased in 1 patient, but still remained in the normal range(P=0.924;P =0.565). Total bilirubin(TBIL) and direct bilirubin(DBIL) increased in 1 case;alanine aminotransferase(ALT), aspartate aminotransferase(AST) and serum creatinine(Scr) increased in 1 case;palpitation occurred in 1 case. All the side effects were alleviated after symptomatic treatment. Conclusion Eltrombopag has certain efficacy and good tolerance in the treatment of refractory acquired PRCA, which is worthy of further exploration.