Olsen Eva H N, McCain Arlene S, Merricks Elizabeth P, Fischer Thomas H, Dillon Ivy M, Raymer Robin A, Bellinger Dwight A, Fahs Scot A, Montgomery Robert R, Keith James C, Schaub Robert G, Nichols Timothy C
Department of Pathology and Laboratory Medicine, University of North Carolina School of Medicine, Chapel Hill, USA.
Blood. 2003 Jul 15;102(2):436-41. doi: 10.1182/blood-2003-01-0290. Epub 2003 Mar 20.
Recombinant human interleukin-11 (rhIL-11), a glycoprotein 130 (gp130)-signaling cytokine approved for treatment of thrombocytopenia, also raises von Willebrand factor (VWF) and factor VIII (FVIII) by an unknown mechanism. Desmopressin (1-deamino-8-d-arginine vasopressin [DDAVP]) releases stored VWF and FVIII and is used for treatment of VWF and FVIII deficiencies. To compare the effect of these 2 agents, heterozygous von Willebrand disease (VWD) and normal dogs were treated with either rhIL-11 (50 microg/kg/d subcutaneously x 7 days) or DDAVP (5 microg/kg/d intravenously x 7 days). The rhIL-11 produced a gradual and sustained elevation of VWF and FVIII levels in both heterozygous VWD and normal dogs while DDAVP produced a rapid and unsustained increase. Importantly, rhIL-11 treatment produced a 2.5- to 11-fold increase in VWF mRNA in normal canine heart, aorta, and spleen but not in homozygous VWD dogs, thus identifying a mechanism for elevation of plasma VWF in vivo. Moreover, dogs pretreated with rhIL-11 retain a DDAVP-releasable pool of VWF and FVIII, suggesting that rhIL-11 does not significantly alter trafficking of these proteins to or from storage pools. The half-life of infused VWF is unchanged by rhIL-11 in homozygous VWD dogs. These results show that rhIL-11 and DDAVP raise plasma VWF by different mechanisms. Treatment with rhIL-11 with or without DDAVP may provide an alternative to plasma-derived products for some VWD and hemophilia A patients if it is shown safe in clinical trials.
重组人白细胞介素-11(rhIL-11)是一种已被批准用于治疗血小板减少症的糖蛋白130(gp130)信号细胞因子,它还通过未知机制提高血管性血友病因子(VWF)和因子VIII(FVIII)的水平。去氨加压素(1-去氨基-8-D-精氨酸加压素[DDAVP])可释放储存的VWF和FVIII,用于治疗VWF和FVIII缺乏症。为比较这两种药物的效果,对杂合子血管性血友病(VWD)犬和正常犬分别给予rhIL-11(50μg/kg/d皮下注射,共7天)或DDAVP(5μg/kg/d静脉注射,共7天)治疗。rhIL-11使杂合子VWD犬和正常犬的VWF和FVIII水平逐渐且持续升高,而DDAVP则使其快速但非持续性升高。重要的是,rhIL-11治疗使正常犬心脏、主动脉和脾脏中的VWF mRNA增加了2.5至11倍,但纯合子VWD犬未出现这种情况,从而确定了体内血浆VWF升高的机制。此外,用rhIL-11预处理的犬保留了DDAVP可释放的VWF和FVIII池,这表明rhIL-11不会显著改变这些蛋白质进出储存池的运输。在纯合子VWD犬中,rhIL-11不会改变输注VWF的半衰期。这些结果表明,rhIL-11和DDAVP通过不同机制提高血浆VWF水平。如果在临床试验中证明安全,对于一些VWD和甲型血友病患者,使用rhIL-11联合或不联合DDAVP治疗可能是血浆衍生产品的一种替代选择。