A Phase 2 Study of Allogeneic Mesenchymal Stromal Cells for Luminal Crohn's Disease Refractory to Biologic Therapy
A Phase 2 Study of Allogeneic Mesenchymal Stromal Cells for Luminal Crohn's Disease Refractory to Biologic Therapy
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DOI：
10.1016/j.cgh.2013.06.021
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发表时间：
2014-01-01
影响因子：
12.6
通讯作者：
Herrmann, Richard P.
中科院分区：
文献类型：
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作者：
Forbes, Geoffrey M.;Sturm, Marian J.;Herrmann, Richard P.
背景与目的：外周血干细胞移植已经成功地治疗了少数克罗恩病(CD)患者，但需要事先进行骨髓调理。间充质基质细胞(MSCs)逃避免疫识别，因此在给药前不需要进行骨髓调理。我们研究了同种异体骨髓间充质干细胞在腔内CD患者中的疗效。方法：我们的第二阶段开放标签多中心研究包括16名英夫利昔单抗或阿达单抗难治、经内窥镜证实的活动性腔内CD的患者(21-55岁；6名男性)。受试者每周静脉注射异基因间充质干细胞(2×10(6)个/kg体重)，连续4周。主要终点是首次应用MSC 42天后的临床反应(CDAI>；100分下降)；次要终点是临床缓解(CDAI；
BACKGROUND & AIMS: Transplantation of peripheral blood stem cells has been successful therapy for small numbers of patients with Crohn's disease (CD), but requires prior myeloconditioning. Mesenchymal stromal cells (MSCs) escape immune recognition, so myeloconditioning is not required before their administration. We investigated the efficacy of allogeneic MSCs in patients with luminal CD. METHODS: Our phase 2, open-label, multicenter study included 16 patients (21-55 y old; 6 men) with infliximab-or adalimumab-refractory, endoscopically confirmed, active luminal CD (CD activity index [CDAI], >250). Subjects were given intravenous infusions of allogeneic MSCs (2 x 10(6) cells/kg body weight) weekly for 4 weeks. The primary end point was clinical response (decrease in CDAI >100 points) 42 days after the first MSC administration; secondary end points were clinical remission (CDAI,