Palifermin for the reduction of acute GVHD: a randomized, double-blind, placebo-controlled trial
Palifermin for the reduction of acute GVHD: a randomized, double-blind, placebo-controlled trial
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DOI：
10.1038/bmt.2011.261
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发表时间：
2012-10-01
影响因子：
4.8
通讯作者：
Waller, E. K.
中科院分区：
文献类型：
--
作者：
Jagasia, M. H.;Abonour, R.;Waller, E. K.
这项前瞻性、随机、双盲、安慰剂对照研究评价了帕利弗明降低骨髓消融术和allo-SCT后重度（3-4级）急性GVHD发生率的疗效。接受allo-SCT治疗恶性血液病的成人在预处理前连续三天接受安慰剂或帕利弗明60 μ g/kg每日一次，在预处理后接受180 mg/kg单次给药，但通常在allo-SCT前1或2天。受试者在第1、3、6和11天接受MTX（加CYA或他克莫司）。每周评价一次急性GVHD，每天评价口腔粘膜炎。受试者被随机分配至安慰剂组（n=78）或帕利佛明组（n=77）。大约一半的受试者（48%安慰剂组，51%帕利弗明组）接受了TBI预处理。治疗组间的主要疗效终点（3-4级急性GVHD的受试者发生率）相似（安慰剂组17%，帕利弗明组16%）。3-4级口腔粘膜炎（安慰剂组为73%，帕利佛明组为81%）和其他次要疗效终点在治疗组之间相似。最常报告的治疗相关不良事件为皮肤/皮下注射。皮疹、瘙痒和红斑等事件。这项骨髓消融术和allo-SCT后急性GVHD的探索性研究未提供帕利佛明给药方案治疗效果的证据。
This prospective, randomized, double-blind, placebo-controlled study evaluated the efficacy of palifermin to reduce the incidence of severe (grade 3-4) acute GVHD after myeloablation and allo-SCT. Adults who received allo-SCT for hematologic malignancies received placebo or palifermin 60 mu g/kg daily on three consecutive days before conditioning and a single dose of 180 mg/kg after conditioning, but often 1 or 2 days before allo-SCT. Subjects received MTX (plus CYA or tacrolimus) on days 1, 3, 6 and 11. Acute GVHD was evaluated once weekly and oral mucositis was evaluated daily. Subjects were randomly assigned to placebo (n=78) or palifermin (n=77). Conditioning included TBI in approximately half of the subjects (48% placebo, 51% palifermin). The primary efficacy end point, subject incidence of grade 3-4 acute GVHD, was similar between treatment groups (17% placebo, 16% palifermin). Grade 3-4 oral mucositis (73% placebo, 81% palifermin) and other secondary efficacy end points were similar between treatment groups. The most commonly reported treatment-related adverse events were skin/s.c. events such as rash, pruritus, and erythema. This exploratory study of acute GVHD after myeloablation and allo-SCT did not provide evidence of a treatment effect with this dosing regimen of palifermin.