ESPN/ERA-EDTA Registry, Department of Medical Informatics, Academic Medical Center, Amsterdam, The Netherlands.
Clin J Am Soc Nephrol. 2012 Mar;7(3):458-65. doi: 10.2215/CJN.07430711. Epub 2012 Jan 5.
Primary hyperoxaluria (PH) as a cause of ESRD in children is believed to have poor outcomes. Data on management and outcomes of these children remain scarce.
DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS: This study included patients aged <19 years who started renal replacement therapy (RRT) between 1979 and 2009 from 31 countries providing data to a large European registry.
Of 9247 incident patients receiving RRT, 100 patients had PH. PH children were significantly younger than non-PH children at the start of RRT. The median age at RRT of PH children decreased from 9.8 years in 1979-1989 to 1.5 years in 2000-2009. Survival was 86%, 79%, and 76% among PH patients at 1, 3, and 5 years after the start of RRT, compared with 97%, 94%, and 92% in non-PH patients, resulting in a three-fold increased risk of death over non-PH patients. PH and non-PH patient survival improved over time. Sixty-eight PH children received a first kidney (n=13) or liver-kidney transplantation (n=55). Although the comparison was hampered by the lower number of kidney transplantations primarily derived from the earlier era of RRT, kidney graft survival in PH patients was 82%, 79%, and 76% at 1, 3, and 5 years for liver-kidney transplantation and 46%, 28%, and 14% at 1, 3, and 5 years for kidney transplantation alone, compared with 95%, 90%, and 85% in non-PH patients.
The outcomes of PH children with ESRD are still poorer than in non-PH children but have substantially improved over time.
原发性高草酸尿症（PH）是儿童终末期肾病（ESRD）的一个病因，其预后被认为较差。关于这些儿童的管理和结局的数据仍然很少。
设计、设置、参与者和测量：本研究纳入了 1979 年至 2009 年期间来自 31 个国家的年龄＜19 岁开始接受肾脏替代治疗（RRT）的患者，这些国家的数据均纳入一个大型欧洲登记处。
在接受 RRT 的 9247 例首发患者中，有 100 例为 PH。与非-PH 儿童相比，PH 儿童开始 RRT 时年龄明显较小。PH 儿童 RRT 的中位年龄从 1979-1989 年的 9.8 岁降至 2000-2009 年的 1.5 岁。PH 患者在 RRT 开始后 1、3 和 5 年的生存率分别为 86%、79%和 76%，而非-PH 患者的生存率分别为 97%、94%和 92%，导致 PH 患者的死亡风险增加了三倍。PH 和非-PH 患者的生存率随时间推移而提高。68 例 PH 患儿接受了首次肾（n=13）或肝肾联合移植（n=55）。尽管由于 RRT 早期肾移植数量较少，比较受到限制，但 PH 患者的肾移植存活率分别为肝肾联合移植后 1、3 和 5 年的 82%、79%和 76%，以及单独肾移植后 1、3 和 5 年的 46%、28%和 14%，而非-PH 患者分别为 95%、90%和 85%。
PH 合并 ESRD 儿童的结局仍较非-PH 儿童差，但随时间推移已显著改善。
