静脉注射免疫球蛋白或大剂量甲泼尼龙，联合或不联合口服泼尼松，用于未治疗的成人重度自身免疫性血小板减少性紫癜：一项随机、多中心试验。

Intravenous immunoglobulin or high-dose methylprednisolone, with or without oral prednisone, for adults with untreated severe autoimmune thrombocytopenic purpura: a randomised, multicentre trial.

作者信息

Godeau Bertrand, Chevret Sylvie, Varet Bruno, Lefrère François, Zini Jean Marc, Bassompierre François, Chèze Stéphane, Legouffe Eric, Hulin Cyrille, Grange Marie José, Fain Olivier, Bierling Philippe

机构信息

Laboratoire d'Immunologie Leucoplaquettaire, EFS Ile de France, France.

出版信息

Lancet. 2002 Jan 5;359(9300):23-9. doi: 10.1016/S0140-6736(02)07275-6.

DOI:10.1016/S0140-6736(02)07275-6

PMID:11809183

Abstract

BACKGROUND

Treatment of adults with autoimmune thrombocytopenic purpura (AITP) is based more on individual experience than on results of controlled studies. We compared intravenous immunoglobulin with high-dose methylprednisolone in untreated adults with severe AITP and assessed efficacy of subsequent oral steroids compared with placebo. Primary outcome was number of days with platelet count greater than 50 x 10(9)/L within the first 21 days.

METHODS

We did a randomised multicentre trial based on a 232 design. 122 adults with severe AITP (platelet count < or =20 x 10(9)/L) were randomly assigned to receive either intravenous immunoglobulin or high-dose methylprednisolone on days 1-3 (randomisation A), and then to receive either oral prednisone or placebo (randomisation B) on days 4-21. Analysis was by intention to treat.

FINDINGS

Six patients were excluded from the analysis. The number of days on which platelet counts were above 50 x 10(9)/L was 18 in 56 patients receiving intravenous immunoglobulin and 14 in 60 receiving high-dose methylprednisolone (p=0.02). Percentage of patients who had platelet counts over 50 x 10(9)/L on days 2 and 5 was 7% and 79%, respectively, in the intravenous immunoglobulin group compared with 2% and 60%, respectively, in the high-dose methylprednisolone group (p=0.04). During the second treatment period, prednisone was more effective than placebo for all short-term endpoints. Patients who received intravenous immunoglobulin and prednisone had platelet count greater than 50 x 10(9)/L for 18.5 days (p=0.005), and those treated with high-dose methylprednisolone and prednisone had this count for 17.5 days.

INTERPRETATION

Intravenous immunoglobulin and oral prednisone seems to be more effective than high-dose methylprednisolone and oral prednisone in adults with severe AITP, although the latter treatment is effective and well tolerated.

摘要

背景

成人自身免疫性血小板减少性紫癜（AITP）的治疗更多基于个人经验而非对照研究结果。我们比较了静脉注射免疫球蛋白与大剂量甲泼尼龙对未经治疗的重度AITP成人患者的疗效，并评估了后续口服类固醇与安慰剂相比的疗效。主要结局是在前21天内血小板计数大于50×10⁹/L的天数。

方法

我们进行了一项基于2×3设计的随机多中心试验。122例重度AITP成人患者（血小板计数≤20×10⁹/L）被随机分配在第1 - 3天接受静脉注射免疫球蛋白或大剂量甲泼尼龙（随机分组A），然后在第4 - 21天接受口服泼尼松或安慰剂（随机分组B）。分析采用意向性治疗。

结果

6例患者被排除在分析之外。56例接受静脉注射免疫球蛋白的患者血小板计数高于50×10⁹/L的天数为18天，60例接受大剂量甲泼尼龙的患者为14天（p = 0.02）。静脉注射免疫球蛋白组在第2天和第5天血小板计数超过50×10⁹/L的患者百分比分别为7%和79%，大剂量甲泼尼龙组分别为2%和60%（p = 0.04）。在第二个治疗期，泼尼松在所有短期终点方面比安慰剂更有效。接受静脉注射免疫球蛋白和泼尼松的患者血小板计数大于50×10⁹/L的天数为18.5天（p = 0.005），接受大剂量甲泼尼龙和泼尼松治疗的患者为17.5天。