PDF(Pubmed)
Abstract：
UNASSIGNED: Eltrombopag (EPAG), a thrombopoietin receptor agonist, was approved for the treatment of severe aplastic anemia (SAA) combined with immunosuppressive therapy (IST). However, EPAG contains a typical biphenyl structure, which causes liver function damage.
UNASSIGNED: Twenty patients with SAA who were intolerant or refractory to EPAG were enrolled in a multicenter prospective registry of the Chinese Eastern Collaboration Group of Anemia (ChiCTR2100045895) from October 2020 to June 2023.
UNASSIGNED: Eight patients who were ineffective to EPAG, six with kidney impairment, and nine with abnormal liver function (two with concomitant liver and kidney impairment) were converted to avatrombopag (AVA) therapy with the median duration of AVA treatment was 6 (3-24) months. 17 cases (85%) achieved trilineage hematological response (HR): complete remission (CR) in 3 cases (15%), good partial remission (GPR) in 4 cases (20%), partial remission (PR) in 10 cases (50%), and no response (NR) in 3 cases (15%). The median time to response was 1.7 (0.5-6.9) months, with 16 cases (94%) achieving response within six months and 17 cases (100%) within 12 months. 9 cases (50%) achieved transfusion independence. AVA converted treatment was associated with higher neutrophil counts (0.8×109/L vs 2.2×109/L, p=0.0003), platelet counts (11×109/L vs 39×109/L, p=0.0008), hemoglobin count (59g/L vs 98g/L, p=0.0002), red cell count (1.06×1012/L vs 2.97×1012/L, p=0.001), and absolute reticulocyte count (31.99 ×109/L vs 67.05×109/L p=0.0004) were all significantly elevated compared with the pre-treatment level. After the conversion to AVA therapy, liver and kidney function indexes were maintained within the normal range, no AVA related grade 2 or higher adverse events occurred, and no thrombotic events occurred.
UNASSIGNED: The conversion to AVA was an optimal choice for patients with SAA who were EPAG intolerant or refractory.
UNASSIGNED: http://www.chictr.org.cn/showproj.html?proj=125480, identifier ChiCTR2100045895.
UNASSIGNED: Twenty patients with SAA who were intolerant or refractory to EPAG were enrolled in a multicenter prospective registry of the Chinese Eastern Collaboration Group of Anemia (ChiCTR2100045895) from October 2020 to June 2023.
UNASSIGNED: Eight patients who were ineffective to EPAG, six with kidney impairment, and nine with abnormal liver function (two with concomitant liver and kidney impairment) were converted to avatrombopag (AVA) therapy with the median duration of AVA treatment was 6 (3-24) months. 17 cases (85%) achieved trilineage hematological response (HR): complete remission (CR) in 3 cases (15%), good partial remission (GPR) in 4 cases (20%), partial remission (PR) in 10 cases (50%), and no response (NR) in 3 cases (15%). The median time to response was 1.7 (0.5-6.9) months, with 16 cases (94%) achieving response within six months and 17 cases (100%) within 12 months. 9 cases (50%) achieved transfusion independence. AVA converted treatment was associated with higher neutrophil counts (0.8×109/L vs 2.2×109/L, p=0.0003), platelet counts (11×109/L vs 39×109/L, p=0.0008), hemoglobin count (59g/L vs 98g/L, p=0.0002), red cell count (1.06×1012/L vs 2.97×1012/L, p=0.001), and absolute reticulocyte count (31.99 ×109/L vs 67.05×109/L p=0.0004) were all significantly elevated compared with the pre-treatment level. After the conversion to AVA therapy, liver and kidney function indexes were maintained within the normal range, no AVA related grade 2 or higher adverse events occurred, and no thrombotic events occurred.
UNASSIGNED: The conversion to AVA was an optimal choice for patients with SAA who were EPAG intolerant or refractory.
UNASSIGNED: http://www.chictr.org.cn/showproj.html?proj=125480, identifier ChiCTR2100045895.
摘要：
■Eltrombopag（EPAG），血小板生成素受体激动剂,已被批准用于治疗重型再生障碍性贫血（SAA）联合免疫抑制治疗（IST）。然而,EPAG含有典型的联苯结构，导致肝功能受损.
从2020年10月至2023年6月，20名对EPAG不耐受或难治性的SAA患者被纳入中国东部贫血协作组（ChiCTR2100045895）的多中心前瞻性注册。
■8名对EPAG无效的患者，六个患有肾脏损伤，9例肝功能异常(2例合并肝肾损害)转用avatrombopag(AVA)治疗,AVA治疗的中位持续时间为6(3-24)个月.17例（85％）达到三系血液学反应（HR）：完全缓解（CR）3例（15％），良好的部分缓解（GPR）4例（20％），部分缓解(PR)10例(50%),3例(15%)无反应(NR)。中位反应时间为1.7（0.5-6.9）个月，其中16例（94%）在6个月内获得缓解，17例（100%）在12个月内获得缓解。9例(50%)取得输血独立。AVA转化治疗与较高的中性粒细胞计数相关（0.8×109/Lvs2.2×109/L，p=0.0003),血小板计数（11×109/Lvs39×109/L，p=0.0008),血红蛋白计数（59g/Lvs98g/L，p=0.0002),红细胞计数（1.06×1012/Lvs2.97×1012/L，p=0.001),与治疗前相比，网织红细胞绝对计数（31.99×109/Lvs67.05×109/Lp=0.0004）均显着升高。转换为AVA治疗后，肝肾功能指标均维持在正常范围内,没有发生AVA相关的2级或更高的不良事件，并且没有发生血栓性事件.
■对于EPAG不耐受或难治性的SAA患者，转换为AVA是最佳选择。
■http://www.chictr.org.cn/showproj.html?proj=125480，标识符ChiCTR2100045895。
从2020年10月至2023年6月，20名对EPAG不耐受或难治性的SAA患者被纳入中国东部贫血协作组（ChiCTR2100045895）的多中心前瞻性注册。
■8名对EPAG无效的患者，六个患有肾脏损伤，9例肝功能异常(2例合并肝肾损害)转用avatrombopag(AVA)治疗,AVA治疗的中位持续时间为6(3-24)个月.17例（85％）达到三系血液学反应（HR）：完全缓解（CR）3例（15％），良好的部分缓解（GPR）4例（20％），部分缓解(PR)10例(50%),3例(15%)无反应(NR)。中位反应时间为1.7（0.5-6.9）个月，其中16例（94%）在6个月内获得缓解，17例（100%）在12个月内获得缓解。9例(50%)取得输血独立。AVA转化治疗与较高的中性粒细胞计数相关（0.8×109/Lvs2.2×109/L，p=0.0003),血小板计数（11×109/Lvs39×109/L，p=0.0008),血红蛋白计数（59g/Lvs98g/L，p=0.0002),红细胞计数（1.06×1012/Lvs2.97×1012/L，p=0.001),与治疗前相比，网织红细胞绝对计数（31.99×109/Lvs67.05×109/Lp=0.0004）均显着升高。转换为AVA治疗后，肝肾功能指标均维持在正常范围内,没有发生AVA相关的2级或更高的不良事件，并且没有发生血栓性事件.
■对于EPAG不耐受或难治性的SAA患者，转换为AVA是最佳选择。
■http://www.chictr.org.cn/showproj.html?proj=125480，标识符ChiCTR2100045895。
