Milliner D S, Eickholt J T, Bergstralh E J, Wilson D M, Smith L H
Department of Internal Medicine, Mayo Clinic, Rochester, MN 55905.
N Engl J Med. 1994 Dec 8;331(23):1553-8. doi: 10.1056/NEJM199412083312304.
The prognosis for patients with primary hyperoxaluria has been ominous, with the expectation of renal failure, poor results with transplantation, and early death.
We studied the long-term effects of orthophosphate and pyridoxine therapy in 25 patients with primary hyperoxaluria who were treated for an average of 10 years (range, 0.3 to 26). Their mean age at the start of treatment was 12 years (median, 6; range, 0.5 to 32). We also studied the effect of orthophosphate and pyridoxine on urinary supersaturation with calcium oxalate, crystal inhibition using a seeded growth system, and crystal formation using scanning electron microscopy in 12 patients during three-day stays in the clinical research center.
The mean (+/- SD) glomerular filtration rate at the start of treatment was 91 +/- 26 ml per minute per 1.73 m2. The median decline in glomerular filtration rates was 1.4 ml per minute per 1.73 m2 of body-surface area per year. The actuarial survival free of end-stage renal disease was 96, 89, 74, and 74 percent of 5, 10, 15, and 20 years, respectively. Treatment with orthophosphate and pyridoxine reduced urinary supersaturation with calcium oxalate from 8.3 +/- 3.0 to 2.1 +/- 1.7 kJ per mole at 38 degrees C (P < 0.001), increased the inhibition of calcium oxalate formation from 63 +/- 11 to 108 +/- 10 inhibitor units per 24 hours (P < 0.001), and improved the crystalluria score from 2.6 +/- 0.3 to 0.6 +/- 0.1 (P < 0.001).
Treatment of patients with primary hyperoxaluria with orthophosphate and pyridoxine decreases urinary calcium oxalate crystallization and appears to preserve renal function.
原发性高草酸尿症患者的预后一直不容乐观，预期会出现肾衰竭、移植效果不佳及过早死亡。
我们研究了正磷酸盐和吡哆醇疗法对25例原发性高草酸尿症患者的长期影响，这些患者平均接受治疗10年（范围为0.3至26年）。治疗开始时他们的平均年龄为12岁（中位数为6岁；范围为0.5至32岁）。我们还在临床研究中心对12例患者进行了为期三天的观察，研究了正磷酸盐和吡哆醇对草酸钙尿饱和度、使用晶种生长系统的晶体抑制作用以及使用扫描电子显微镜观察的晶体形成的影响。
治疗开始时平均（±标准差）肾小球滤过率为每分钟每1.73平方米91±26毫升。肾小球滤过率的中位数下降为每年每1.73平方米体表面积1.4毫升每分钟。无终末期肾病的精算生存率在5年、10年、15年和20年时分别为96%、89%、74%和74%。正磷酸盐和吡哆醇治疗使38摄氏度时草酸钙尿饱和度从8.3±3.0千焦每摩尔降至2.1±1.7千焦每摩尔（P<0.001），使草酸钙形成的抑制作用从每24小时63±11抑制剂单位增加到108±10抑制剂单位（P<0.001），并使结晶尿评分从2.6±0.3提高到0.6±0.1（P<0.001）。
用正磷酸盐和吡哆醇治疗原发性高草酸尿症患者可减少草酸钙尿结晶，并似乎能保留肾功能。
