在发达国家,死亡病例较罕见并且有完善的记录,主要原因是感染和血栓并发症。[1]Gordillo R, Spitzer A. The nephrotic syndrome. Pediatr Rev. 2009;30:94-104.http://www.ncbi.nlm.nih.gov/pubmed/19255123?tool=bestpractice.com[2]Eddy AA, Symons JM. Nephrotic syndrome in childhood. Lancet. 2003;362:629-639.http://www.ncbi.nlm.nih.gov/pubmed/12944064?tool=bestpractice.com[36]International Study of Kidney Disease in Children. Minimal change nephrotic syndrome in children: deaths during the first 5 to 15 years' observation. Report of the International Study of Kidney Disease in Children. Pediatrics. 1984;73:497-501.http://www.ncbi.nlm.nih.gov/pubmed/6709428?tool=bestpractice.com在发展中国家和地区,尽管受治疗设施的限制,但半数肾病综合征 (NS) 患者受益于皮质类固醇,然而皮质类固醇的耐药性被证明导致高病死率。[37]Bhimma R, Adhikari M, Asharam K, et al. The spectrum of chronic kidney disease (stages 2-5) in KwaZulu-Natal, South Africa. Pediatr Nephrol. 2008;23:1841-1846.http://www.ncbi.nlm.nih.gov/pubmed/18548285?tool=bestpractice.com在这些国家中,资源匮乏、晚期转诊和肾替代治疗的高成本造成各种病因导致的(包括 NS 和 MCD)慢性肾脏疾病的预后不良。[37]Bhimma R, Adhikari M, Asharam K, et al. The spectrum of chronic kidney disease (stages 2-5) in KwaZulu-Natal, South Africa. Pediatr Nephrol. 2008;23:1841-1846.http://www.ncbi.nlm.nih.gov/pubmed/18548285?tool=bestpractice.com
皮质类固醇治疗后蛋白尿的缓解比肾组织病变缓解在长期预后方面有更大的价值。
过渡到成年
童年 MCD 在成年的复发率相对较高,但肾功能可能正常并且总体发病率较低。[38]Ruth EM, Kemper MJ, Leumann EP, et al. Children with corticosteroid-sensitive nephrotic syndrome come of age: long-term outcome. J Pediatr. 2005;147:202-207.http://www.ncbi.nlm.nih.gov/pubmed/16126050?tool=bestpractice.com建议所有年龄接近成年的儿童患者由成人肾病医师诊治。
即使在成人中,MCD 也具有良好的预后,因为 90% 的患者对口服类固醇有效(根据多项儿童大型前瞻性随机试验、儿童和成人中广泛的观察研究以及少量的成人小样本随机对照试验的有益成果报告)。[39]Pais P, Avner ED. Nephrotic syndrome. In: Kliegman RM, Stanton BF, St. Geme JW III, Schor NF, Behrman RE, eds. Nelson Textbook of Pediatrics. 19th ed. Philadelphia, PA: Saunders Elsevier; 2011.[40]Nachman PH, Jennette JC, Falk RJ. Primary glomerular disease. In: Brenner BM, ed. Brenner and Rector's The Kidney. 8th ed. Philadelphia, Pa: Saunders Elsevier; 2007:30.[41]Appel GB. Glomerular disorders and nephrotic syndromes. In: Goldman L, Ausiello D, eds. Cecil Medicine. 23rd ed. Philadelphia, PA: Saunders Elsevier; 2007:122.然而,如同在儿童中所见,多数进入缓解期的成人会复发。少数 MCD 成人患者最后会发展成为局灶性节段性肾小球硬化症,这可能最终导致肾衰竭。[40]Nachman PH, Jennette JC, Falk RJ. Primary glomerular disease. In: Brenner BM, ed. Brenner and Rector's The Kidney. 8th ed. Philadelphia, Pa: Saunders Elsevier; 2007:30.此外,MCD 成人患者往往起效更慢,25% 以上有效患者需要 3 至 4 个月或更长时间才能完全缓解。[39]Pais P, Avner ED. Nephrotic syndrome. In: Kliegman RM, Stanton BF, St. Geme JW III, Schor NF, Behrman RE, eds. Nelson Textbook of Pediatrics. 19th ed. Philadelphia, PA: Saunders Elsevier; 2011.[40]Nachman PH, Jennette JC, Falk RJ. Primary glomerular disease. In: Brenner BM, ed. Brenner and Rector's The Kidney. 8th ed. Philadelphia, Pa: Saunders Elsevier; 2007:30.[41]Appel GB. Glomerular disorders and nephrotic syndromes. In: Goldman L, Ausiello D, eds. Cecil Medicine. 23rd ed. Philadelphia, PA: Saunders Elsevier; 2007:122.由于使用二线药物(例如环磷酰胺)可能产生毒性和副作用,利妥昔单抗已成功用于皮质类甾醇依赖性 MCD 患者的替代治疗,可以成功地停止类固醇治疗。[42]Hoxha E, Stahl RA, Harendza S. Rituximab in adult patients with immunosuppressive-dependent minimal change disease. Clin Nephrol. 2011;76:151-158.http://www.ncbi.nlm.nih.gov/pubmed/21762648?tool=bestpractice.com