Mahan J D, Mauer S M, Sibley R K, Vernier R L
J Pediatr. 1984 Oct;105(4):549-57. doi: 10.1016/s0022-3476(84)80418-7.
We analyzed the clinical course, pathologic findings, and results of aggressive medical management and renal transplantation in 41 infants with onset of nephrotic syndrome in the first 3 months of life. All but one infant with congenital onset failed to thrive and had progressive renal insufficiency; 17 were given steroids or cytotoxic drugs or both, without benefit. Severe bacterial infections occurred in 85% of the infants, pyloric stenosis in 12%, gastroesophageal reflux in 8%, and thrombotic events in 10%. All children prior to the era of renal transplantation died before 4 years of age. The last 24 infants received aggressive medical management, which allowed renal transplantation in 17. Two-year patient and graft survival rates were 82% and 71%, respectively. There was no recurrence of the nephrotic syndrome in the children who underwent transplantation. All but one surviving infants has had normal or accelerated growth, although mean height for the group is 3.1 SD below the mean. School and social performance has been normal in 80%. Thus intensive medical therapy combined with renal transplantation offers a very good opportunity for survival with an acceptable quality of life for infants with congenital nephrotic syndrome.
我们分析了41例在出生后前3个月内发病的肾病综合征婴儿的临床病程、病理结果以及积极药物治疗和肾移植的结果。除1例先天性发病的婴儿外，其余婴儿均生长发育不良并伴有进行性肾功能不全；17例婴儿接受了类固醇或细胞毒性药物或两者联合治疗，但均无益处。85%的婴儿发生了严重细菌感染，12%发生了幽门狭窄，8%发生了胃食管反流，10%发生了血栓事件。在肾移植时代之前，所有儿童均在4岁前死亡。最后24例婴儿接受了积极的药物治疗，其中17例接受了肾移植。2年的患者和移植物存活率分别为82%和71%。接受移植的儿童未出现肾病综合征复发。除1例存活婴儿外，其他所有存活婴儿均生长正常或生长加速，尽管该组婴儿的平均身高比平均水平低3.1个标准差。80%的儿童学业和社交表现正常。因此，强化药物治疗联合肾移植为先天性肾病综合征婴儿提供了很好的生存机会和可接受的生活质量。
