BJH:免疫性血小板减少症成人在接受艾曲波帕或罗米司亭治疗后改用阿凡波帕:一项多中心美国研究

2022-02-23 MedSci原创 MedSci原创

阿凡波帕在使用罗米司亭或艾曲波帕治疗后有效,即使在对先前 TPO-RA 反应不足的患者中也具有高反应率。

患有免疫性血小板减少症 (ITP) 的患者可能对一种血小板生成素受体激动剂 (TPO-RA) 有反应,但对另一种没有反应。描述从罗米司亭或艾曲波帕转换为阿凡波帕(一种较新的口服 TPO-RA)的患者结果的可用数据有限。

图:每个患者在转换前(罗米司亭或艾曲波帕治疗期间)与转换为阿凡波帕后的中位血小板计数。对于每个患者,血小板计数的中位数是在接受该药物时测量的最近三次血小板计数的中位数。(A)所有患者(N-=44)。(B)患者因罗米司亭或艾曲波帕无效而改变(N=14)。1例患者血小板计数为585×109/l,在保留图中均省略。缩写:AVA、血小板、PLT、PLT、血小板计数、ROMI、

研究人员对美国四个三级 ITP 转诊中心从艾曲波帕或罗米普洛汀转为阿凡波帕的 ITP 成人进行了一项回顾性观察研究。纳入了 44 名患者,平均 ITP 持续时间为 8.3 年,中位数(范围)为 4 次之前的 ITP 治疗。在阿凡波帕上,41/44 名患者 (93%) 达到血小板反应 (≥50 × 109 /l),38/44 名患者 (86%) 达到完全反应 (≥100 × 109 /l)。

缩写:PC,血小板计数。

在所有患者中,艾曲波帕或罗米司亭的中位血小板计数为 44 × 109 /l vs 阿凡波帕的 113 × 109 /l (p < 0.0001);在因 罗米司亭/艾曲波帕无效而改用罗米司亭/艾曲波帕的患者中,罗米司亭/艾曲波帕为 28 × 109 /l,而阿凡波帕为 88 × 109 /l (p = 0.025)。57% 的患者在转换前同时接受 ITP 药物治疗后在转换后停药,其中 63% 的患者接受慢性皮质类固醇治疗。

在经过大量预处理的慢性 ITP 人群中,阿凡波帕在使用罗米司亭或艾曲波帕治疗后有效,即使在对先前 TPO-RA 反应不足的患者中也具有高反应率。这些发现强调了当先前的TPO-RA不能提供足够的有效性、便利性或耐受性时,在TPO-RAs之间切换的潜在价值。需要对更大的患者群体进行进一步的研究,以证实本研究的发现,并扩大对特定患者亚群体的观察。

 

原始出处:

Al-Samkari, HPJiang, DGernsheimer, TLiebman, HLee, SWojdyla, MVredenburg, MCuker, AAdults with immune thrombocytopenia who switched to avatrombopag following prior treatment with eltrombopag or romiplostim: A multicentre US studyBr J Haematol2022001– 8.

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Blood Cancer J:艾曲波帕联合或不联合他克莫司治疗复发/难治性获得性再生障碍性贫血的疗效比较

该研究发现EPAG+他克莫司的临床疗效优于EPAG单药治疗,且两组的安全性和克隆进化率具有可比性。此外,事后分析表明,EPAG+他克莫司在年轻和复发患者中的优势更为突出。

Am J Hematol;骨髓衰竭患者长期使用艾曲波帕治疗会导致铁耗竭

艾曲波帕 (EPAG) 已被批准用于治疗再生障碍性贫血和免疫性血小板减少症,部分患者需要长期治疗。由于多价阳离子螯合,长期治疗会导致一直以来被低估的铁耗竭。

NEJM:免疫抑制疗法加用艾曲波帕治疗重度再生障碍性贫血

在标准免疫抑制治疗中加入艾曲波帕可提高既往未治疗过的重度再生障碍性贫血患者的血液学缓解率、缓解速度和缓解深度

AJH:艾曲波帕治疗单倍体相同外周血干细胞移植后持续性血小板减少症患者

持续性血小板减少症(PT)是异基因造血干细胞移植(allo-HSCT)后常见的并发症,对生存率产生显著的负面影响。已发表的数据表明,PT的机制可归因于血小板生成受损、外周破坏加速或两种之间的相互作用。

Blood Cells Mol Dis:艾曲波帕作为原发性免疫性血小板减少症患者二线治疗的有效性和安全性

研究证实了艾曲波帕作为二线治疗慢性ITP患者的有效性和安全性

Leukemia:艾曲波帕eltrombopag在骨髓增生异常综合征的PDX模型中的临床前评估

骨髓增生异常综合征(MDS)是一种克隆性造血干细胞疾病,其特征是骨髓(BM)发育不良、血细胞的减少、复发性基因组畸变以及疾病发展为急性髓系白血病的易感性。