[1]郭伟红,李强,朱梅.遗传性肾性尿崩症的药物治疗[J].国际内分泌代谢杂志,2016,36(03):184-186.[doi:10.3760/cma.j.issn.1673-4157.2016.03.10]
 Guo Weihong*,Li Qiang,Zhu Mei..Drug treatment of congenital nephrogenic diabetes insipidus[J].International Journal of Endocrinology and Metabolism,2016,36(03):184-186.[doi:10.3760/cma.j.issn.1673-4157.2016.03.10]
点击复制

遗传性肾性尿崩症的药物治疗()
分享到:

《国际内分泌代谢杂志》[ISSN:1673-4157/CN:12-1383/R]

卷:
36
期数:
2016年03期
页码:
184-186
栏目:
综述
出版日期:
2016-05-20

文章信息/Info

Title:
Drug treatment of congenital nephrogenic diabetes insipidus
作者:
郭伟红李强朱梅
300052 天津医科大学总医院内分泌代谢病科(郭伟红, 朱梅); 300010 天津海河医院普外科(李强)
Author(s):
Guo Weihong* Li Qiang Zhu Mei.
*Department of Endocrinology, The General Hospital, Tianjin Medical University, Tianjin 300052, China
关键词:
遗传性肾性尿崩症 精氨酸加压素受体2 水通道蛋白-2
Keywords:
Congenital nephrogenic diabetes insipidus Arginine vasopressin receptor 2 Auqaporin-2
DOI:
10.3760/cma.j.issn.1673-4157.2016.03.10
摘要:
遗传性肾性尿崩症由精氨酸加压素受体2(AVPR2)或水通道蛋白-2(AQP2)基因突变引起。 目前临床治疗主要是改善多饮、多尿的症状。随着对发病机制研究的不断深入,新型药物如AVPR2激动剂和拮抗剂、磷酸二酯酶抑制剂、他汀类、前列腺素E受体激动剂等,通过改善突变的AVPR2/AQP2在细胞内的合成、修饰、表达等达到治疗的目的。
Abstract:
Congenital nephrogenic diabetes insipidus is caused by mutations of Arginine vasopressin receptor 2(AVPR2)/Auqaporin-2(AQP2). The current clinical treatment is aimed at improving symptoms of polydipsia, polyuria. With the deepening research on the pathogenesis, AVPR2 agonists and antagonists, phosphodiesterase antagonists, statins, prostaglandin E receptor agonists can improve mutational AVPR2/AQP2 in synthesis, modification, expression in cells, so achieve the goal of treatment.

参考文献/References:

[1] Wesche D, Deen PM, Knoers NV. Congenital nephrogenic diabetes insipidus: the current state of affairs[J]. Pediatr Nephrol, 2012, 27(12):2183-2204. DOI: 10.1007/s00467-012-2118-8.
[2] Verkman AS, Anderson MO, Papadopoulos MC. Aquaporins: important but elusive drug targets[J]. Nat Rev Drug Discov,2014,13(4):259-277. DOI: 10.1038/nrd4226.
[3] 廖二元. 内分泌代谢病学[M]. 第3版, 北京:人民卫生出版社, 2012: 1036-1038.
[4] Boussemart T, Nsota J, Martin-Coignard D, et al. Nephrogenic diabetes insipidus: treat with caution.Pediatr Nephrol, 2009,24(9):1761-1763. DOI: 10.1007/s00467-009-1187-9.
[5] Robben JH, Sze M, Knoers NV, et al. Functional rescue of vasopressinV2 receptor mutants in MDCK cells by pharmacochaperones: relevance to therapy of nephrogenic diabetes insipidus[J]. Am J Physiol Renal Physiol, 2007,292(1): F253-F260.
[6] Rochdi MD, Vargas GA, Carpentier E, et al. Functional characterization of vasopressin type 2 receptor substitutions(R137H/C/L)leading to nephrogenic diabetes insipidus and nephrogenic syndrome of inappropriate antidiuresis:implications for treatments [J]. Mol Pharmacol,2010,77(5):836-845.DOI: 10.1124/mol.109.061804.
[7] Benier V, Morello JP, Zarruk A, et al. Pharmacologic chaperones as a potential treatment for X-linked nephrogenic giabetes insipidus[J]. J Am Soc Nephrol, 2006,17(1):232-243.
[8] Los EL, Deen PM, Robben JH. Potential of nonpeptide(ant)agonists to rescue vasopressin V2 receptor mutants for the treatment of X-linked nephrogenic diabetes insipidus[J]. J Neuroendocrinol, 2010,22(5):393-399.DOI: 10.1111/j.1365-2826.2010.01983.x.
[9] Robben JH, Kortenoeven ML, Sze M, et al. Intracellular activation of vasopressin V2 receptor mutants in nephrogenic diabetes insipidus by nonpeptide agonists[J]. Proc Natl Acad Sci U S A,2009,106(29):12195-12200. DOI: 10.1073/pnas.0900130106.
[10] Jean-Alphonse F, Perkovska S, Frantz MC, et al. Biased agonist pharmacochaperones of the AVP V2 receptor may treat congenital nephrogenic diabetes insipidus[J]. J Am Soc Nephrol, 2009, 20(10):2190-2203. DOI: 10.1681/ASN.2008121289.
[11] Erdélyi LS, Balla A, Patócs A, et al. Altered agonist sensitivity of a mutant V2 receptor suggests a novel therapeutic strategy for nephrogenic diabetes insipidus[J]. Mol Endocrinol, 2014, 28(5):634-643. DOI: 10.1210/me.2013-1424.
[12] Bouley R, Hasler U, Lu HA, et al. Bypassing vasopressin receptor signaling pathways in nephrogenic diabetes insipidus[J]. Semin Nephrol, 2008, 28(3):266-278. DOI: 10.1016/j.semnephrol.
[13] Sanches TR, Volpini RA, Massola Shimizu MH, et al. Sildenafil reduces polyuria in rats with lithium-induced NDI[J].Am J Physiol Renal Physiol, 2012, 302(1): F216-F225. DOI: 10.1152/ajprenal.00439.2010.
[14] Bouley R, Lu HA, Nunes P, et al. Calcitonin has a vasopressin-like effect on aquaporin-2 trafficking and urinary concentration[J]. J Am Soc Nephrol, 2011, 22(1):59-72. DOI: 10.1681/ASN.2009121267.
[15] Li W, Zhang Y, Bouley R, et al. Simvastatin enhances aquaporin-2 surface expression and urinary concentration in vasopressin-deficient Brattleboro rats through modulation of Rho GTPase[J]. Am J Physiol Renal Physiol, 2011, 301(2): F309-F318.DOI: 10.1152/ajprenal.00001.2011.
[16] Bonfrate L,Procino G,Wang DQ,et al. A novel therapeutic effect ofstatinsonnephrogenic diabetes insipidus[J]. J Cell Mol Med,2015,19(2):265-282. DOI: 10.1111/jcmm.12422.
[17] Procino G, Barbieri C, Carmosino M, et al. Fluvastatin modulates renal water reabsorption in vivo through increased AQP2 availability at the apical plasma membrane of collecting duct cells[J]. Pflugers Arch, 2011, 462(5):753-766. DOI: 10.1007/s00424-011-1007-5.
[18] Procino G,Milano S,Carmosino M, et al. Combination of secretin and fluvastatin ameliorates the polyuria associated with X-linked nephrogenic diabetes insipidus inmice[J]. Kidney Int, 2014, 86(1):127-138. DOI: 10.1038/ki.2014.10.
[19] Li JH, Chou CL, Li B, et al. A selective EP4 PGE2 receptor agonist alleviates disease in a new mouse model of X-linked nephrogenic diabetes insipidus[J].J Clin Invest, 2009,119(10):3115-3126. DOI: 10.1172/JCI39680.
[20] Olesen ET, Rutzler MR, Moeller HB, et al. Vasopressin-independent targeting of aquaporin-2 by selective E-prostanoid receptor agonists alleviates nephrogenic diabetes insipidus[J]. Proc Natl Acad Sci U S A, 2011,108(31):12949-12954. DOI: 10.1073/pnas.1104691108.
[21] Gao M, Cao R, Du S, et al. Disruption of prostaglandin E2 receptor EP4 impairs urinary concentration via decreasing aquaporin 2 in renal collecting ducts. Proc Natl Acad Sci U S A, 2015,112(27):8397-8402. DOI: 10.1073/pnas.1509565112.
[22] Yang B, Zhao D, Verkman AS. Hsp90 inhibitor partially corrects nephrogenic diabetes insipidus in a conditional knock-in mouse model of aquaporin-2 mutation[J]. FASEB J, 2009, 23(2):503-512. DOI: 10.1096/fj.08-118422.
[23] Taiyab A, Sreedhar AS, Rao ChM. Hsp90 inhibitors, GA and 17AAG, lead to ER stress-induced apoptosis in rat histiocytoma[J]. Biochem Pharmacol, 2009, 78(2):142-152.DOI:10.1016/j.bcp.2009.04.001.

更新日期/Last Update: 2016-05-20