Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial

罗米普洛斯蒂姆 医学 血小板减少性紫癜 安慰剂 临床终点 内科学 血小板生成素 血小板生成素 随机对照试验 临床试验 胃肠病学 血小板 巨核细胞 遗传学 替代医学 干细胞 病理 造血 生物
作者
David J. Kuter,James B. Bussel,Roger M. Lyons,Vinod Pullarkat,Terry Gernsheimer,Francis M. Senecal,Louis M. Aledort,James N. George,Craig M. Kessler,Miguel Á. Sanz,Howard A. Liebman,Frank T. Slovick,J. Th. M. de Wolf,Emmanuelle Bourgeois,Troy H. Guthrie,Adrian C. Newland,Jeffrey S. Wasser,Solomon I. Hamburg,Carlos Grande,François Lefrère
出处
期刊:The Lancet [Elsevier BV]
卷期号:371 (9610): 395-403 被引量:844
标识
DOI:10.1016/s0140-6736(08)60203-2
摘要

Chronic immune thrombocytopenic purpura (ITP) is characterised by accelerated platelet destruction and decreased platelet production. Short-term administration of the thrombopoiesis-stimulating protein, romiplostim, has been shown to increase platelet counts in most patients with chronic ITP. We assessed the long-term administration of romiplostim in splenectomised and non-splenectomised patients with ITP.In two parallel trials, 63 splenectomised and 62 non-splenectomised patients with ITP and a mean of three platelet counts 30x10(9)/L or less were randomly assigned 2:1 to subcutaneous injections of romiplostim (n=42 in splenectomised study and n=41 in non-splenectomised study) or placebo (n=21 in both studies) every week for 24 weeks. Doses of study drug were adjusted to maintain platelet counts of 50x10(9)/L to 200x10(9)/L. The primary objectives were to assess the efficacy of romiplostim as measured by a durable platelet response (platelet count > or =50x10(9)/L during 6 or more of the last 8 weeks of treatment) and treatment safety. Analysis was per protocol. These studies are registered with ClinicalTrials.gov, numbers NCT00102323 and NCT00102336.A durable platelet response was achieved by 16 of 42 splenectomised patients given romplostim versus none of 21 given placebo (difference in proportion of patients responding 38% [95% CI 23.4-52.8], p=0.0013), and by 25 of 41 non-splenectomised patients given romplostim versus one of 21 given placebo (56% [38.7-73.7], p<0.0001). The overall platelet response rate (either durable or transient platelet response) was noted in 88% (36/41) of non-splenectomised and 79% (33/42) of splenectomised patients given romiplostim compared with 14% (three of 21) of non-splenectomised and no splenectomised patients given placebo (p<0.0001). Patients given romiplostim achieved platelet counts of 50x10(9)/L or more on a mean of 13.8 (SE 0.9) weeks (mean 12.3 [1.2] weeks in splenectomised group vs 15.2 [1.2] weeks in non-splenectomised group) compared with 0.8 (0.4) weeks for those given placebo (0.2 [0.1] weeks vs 1.3 [0.8] weeks). 87% (20/23) of patients given romiplostim (12/12 splenectomised and eight of 11 non-splenectomised patients) reduced or discontinued concurrent therapy compared with 38% (six of 16) of those given placebo (one of six splenectomised and five of ten non-splenectomised patients). Adverse events were much the same in patients given romiplostim and placebo. No antibodies against romiplostim or thrombopoietin were detected.Romiplostim was well tolerated, and increased and maintained platelet counts in splenectomised and non-splenectomised patients with ITP. Many patients were able to reduce or discontinue other ITP medications. Stimulation of platelet production by romiplostim may provide a new therapeutic option for patients with ITP.
最长约 10秒,即可获得该文献文件

科研通智能强力驱动
Strongly Powered by AbleSci AI
更新
PDF的下载单位、IP信息已删除 (2025-6-4)

科研通是完全免费的文献互助平台,具备全网最快的应助速度,最高的求助完成率。 对每一个文献求助,科研通都将尽心尽力,给求助人一个满意的交代。
实时播报
ahh完成签到 ,获得积分10
1秒前
辛勤安梦完成签到,获得积分10
2秒前
Akjan完成签到,获得积分10
5秒前
查查make完成签到,获得积分10
9秒前
Jasper应助大橙子采纳,获得10
10秒前
GUO发布了新的文献求助30
11秒前
三石完成签到 ,获得积分10
11秒前
跳跃的白云完成签到 ,获得积分10
12秒前
酷酷亦寒完成签到 ,获得积分10
14秒前
量子星尘发布了新的文献求助10
16秒前
Blaseaka完成签到 ,获得积分10
17秒前
Owen应助科研通管家采纳,获得10
17秒前
深情安青应助科研通管家采纳,获得10
17秒前
17秒前
汉堡包应助科研通管家采纳,获得10
18秒前
小二郎应助科研通管家采纳,获得30
18秒前
爆米花应助科研通管家采纳,获得10
18秒前
沉静的浩然完成签到,获得积分10
19秒前
开朗的绮山完成签到,获得积分10
19秒前
19秒前
老迟到的土豆完成签到 ,获得积分10
22秒前
单薄的日记本完成签到,获得积分10
22秒前
大橙子发布了新的文献求助10
23秒前
舒适的天奇完成签到 ,获得积分10
24秒前
25秒前
27秒前
都是小儿卡通书完成签到,获得积分10
28秒前
29秒前
陶醉的又夏完成签到 ,获得积分10
29秒前
lily完成签到 ,获得积分10
32秒前
33秒前
子苓完成签到 ,获得积分10
35秒前
Jun完成签到 ,获得积分10
35秒前
phil完成签到,获得积分10
36秒前
祁乐安发布了新的文献求助20
36秒前
如初完成签到,获得积分10
37秒前
zzuwxj完成签到,获得积分10
40秒前
糊涂的语兰完成签到,获得积分10
44秒前
多余完成签到,获得积分10
45秒前
喝酸奶不舔盖完成签到 ,获得积分10
45秒前
高分求助中
【提示信息,请勿应助】关于scihub 10000
Les Mantodea de Guyane: Insecta, Polyneoptera [The Mantids of French Guiana] 3000
徐淮辽南地区新元古代叠层石及生物地层 3000
The Mother of All Tableaux: Order, Equivalence, and Geometry in the Large-scale Structure of Optimality Theory 3000
Handbook of Industrial Diamonds.Vol2 1100
Global Eyelash Assessment scale (GEA) 1000
Picture Books with Same-sex Parented Families: Unintentional Censorship 550
热门求助领域 (近24小时)
化学 材料科学 医学 生物 工程类 有机化学 生物化学 物理 内科学 纳米技术 计算机科学 化学工程 复合材料 遗传学 基因 物理化学 催化作用 冶金 细胞生物学 免疫学
热门帖子
关注 科研通微信公众号,转发送积分 4038157
求助须知:如何正确求助?哪些是违规求助? 3575869
关于积分的说明 11373842
捐赠科研通 3305650
什么是DOI,文献DOI怎么找? 1819255
邀请新用户注册赠送积分活动 892655
科研通“疑难数据库(出版商)”最低求助积分说明 815022