Effectiveness and Safety of Romiplostim Among Patients with Newly Diagnosed, Persistent and Chronic Immune Thrombocytopenia in European Clinical Practice.
Effectiveness and Safety of Romiplostim Among Patients with Newly Diagnosed, Persistent and Chronic Immune Thrombocytopenia in European Clinical Practice.
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DOI：
10.1007/s12325-021-01727-5
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发表时间：
2021-05
影响因子：
3.8
通讯作者：
McGrath LJ
中科院分区：
文献类型：
--
作者：
Snell Taylor SJ;Nielson CM;Breskin A;Saul B;Yu Y;Alam N;Eisen M;Hippenmeyer J;Janssens A;Kozak T;Papadaki HA;Selleslag D;Viallard JF;Feistritzer C;Kaiafa G;Kelsh M;Kilpatrick K;Brookhart MA;McGrath LJ
Romiplostim自2009年以来已在欧洲获批用于治疗慢性原发性免疫性血小板减少症（ITP）患者。使用来自7个欧洲国家的真实世界数据，我们根据ITP的持续时间测量了罗米司亭开始治疗后24周内的有效性和安全性结局：小于3个月（“新诊断”），3-12个月（“持续性”）和超过12个月（“慢性”）。 纳入了2009年至2012年期间接受≥ 1次romiplostim给药的ITP成人患者。终点包括持久血小板应答、中位血小板计数、补救治疗、出血和不良事件。我们使用截尾加权估计的逆概率来估计每个结果的累积风险。在romiplostim开始治疗时，有64例新诊断的ITP患者，50例持续性ITP患者和226例慢性ITP患者。24周时的持久血小板应答率范围为32% [置信区间（CI）：18-46%]（新诊断患者）至53%（CI 37-68%）（持续患者）。随访期间的中位血小板计数范围为慢性患者的88（CI 80-96）× 109/L至新诊断患者的131（CI 102-160）× 109/L。无论ITP持续时间如何，超过一半的患者停用伴随ITP药物。观察到的不良事件很少。尽管仅批准用于慢性患者，但在欧洲临床实践中管理的患者中，romiplostim治疗效果的估计值相似，无论romiplostim开始治疗时的ITP持续时间如何。在线版本包含补充材料，可通过10.1007/s12325-021-01727-5获得。
Romiplostim has been approved in Europe since 2009 to treat patients with chronic primary immune thrombocytopenia (ITP). Using real-world data from seven European countries, we measured the effectiveness and safety outcomes within 24 weeks following romiplostim initiation by duration of ITP: less than 3 months (“newly diagnosed”), 3–12 months (“persistent”), and more than 12 months (“chronic”). Adults with ITP and ≥ 1 romiplostim administration between 2009 and 2012 were included. Endpoints included durable platelet response, median platelet count, rescue therapy, bleeding and adverse events. We used inverse probability of censoring weighted estimators to estimate cumulative risk of each outcome. There were 64 newly diagnosed, 50 persistent, and 226 chronic ITP patients at romiplostim initiation. Durable platelet response at 24 weeks ranged from 32% [confidence interval (CI): 18–46%] in newly diagnosed patients to 53% (CI 37–68%) in persistent patients. Median platelet count during follow-up ranged from 88 (CI 80–96) × 109/L in chronic patients to 131 (CI 102–160) × 109/L in newly diagnosed patients. Regardless of ITP duration, over half of patients discontinued concomitant ITP medications. Few adverse events were observed. Although only approved for chronic patients, estimates of the romiplostim treatment effect were similar across patients being managed in European clinical practice, regardless of ITP duration at romiplostim initiation. The online version contains supplementary material available at 10.1007/s12325-021-01727-5.
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影响因子：
6.5
作者：
Kuter, David J.;Newland, Adrian;Eisen, Melissa
通讯作者：
Eisen, Melissa
影响因子：
4.5
作者：
AALEN, O
通讯作者：
AALEN, O
DOI：
10.1016/j.sapharm.2005.12.001
发表时间：
2006-03-01
期刊：
Research in social & administrative pharmacy : RSAP
影响因子：
--
作者：
Lobo, Francis S;Wagner, Samuel;Schommer, Jon C
通讯作者：
Schommer, Jon C
影响因子：
2.9
作者：
Heddle, NM;Cook, RJ;Rebulla, P
通讯作者：
Rebulla, P
影响因子：
0.6
作者：
Mihaylov, Georgi;Skopec, Barbara;Cernelc, Peter
通讯作者：
Cernelc, Peter