Hassan Akhila V, Sinha Manish D, Waller Simon
Department of Paediatric Nephrology , Evelina Children's Hospital , Guys & St Thomas NHS Foundation Trust , London , UK.
Clin Kidney J. 2013 Aug;6(4):384-9. doi: 10.1093/ckj/sft071.
The aim of the study was to investigate the efficacy and side effect profile of mycophenolate mofetil (MMF) therapy in children with nephrotic syndrome (NS).
A retrospective case note review was performed on all patients with NS who were commenced on MMF between 1 January 2000 and 31 December 2009 and were followed up for a minimum of 1 year.
The sample size was 73 patients. The duration of follow-up was for a median of 3.2 years, interquartile range (IQR) (1.7-4.7 years). The median age at diagnosis was 3.2 years, IQR (2.3-5.7 years). The median age of MMF commencement was 11 years, IQR (7.9-13.6 years). There were more boys (67%) than girls. The majority were Caucasian (77%), with 18% Asian 4%, Black Africans and 1% other ethnicities. At initial diagnosis, 61 (84%) were steroid sensitive, 9 (12%) steroid resistant, 3 (4%) steroid dependent (SD). Forty-five (74%) of the 61 steroid-sensitive patients became SD, 4 (7%) of them became steroid resistant, 1 (1%) remained steroid-sensitive and 11 (18%) became frequent relapsers. As to the previous use of second-line immunosuppressants, none were used in 5 (7%) patients, one agent in 17 (23%), two in 27 (37%) and three or more agents were used in 23 (32%) patients. MMF was effective in 45 (62%) patients. Of these, 38 (52%) of them were in remission for >2 years; and in 7 (10%) MMF worked for 1 to 2 years (MMF therapy electively stopped/ongoing). MMF therapy allowed 27 (37%) patients to wean steroids completely and 8 (11%) to achieve complete steroid and immunosuppressant withdrawal. A further 8 (11%) had steroids partially weaned. MMF failures were seen in 13 (18%) within the first year and 5 (7%) in the second year. MMF was stopped due to side effects in 4 (6%) and non-compliance in 4 (6%). The majority of patients had no side effects [51 (70%)]. Seven (9%) had gastrointestinal side effects (diarrhoea/abdominal pain); 5 (7%) had immunological side effects (leucopenia/infections); 3 (4%) had both immunological and gastrointestinal side effects; and 2 (3%) suffered arthralgia.
MMF is well tolerated and effective as a second-line agent in treating steroid-sensitive NS. The drug permitted prolonged remission and steroid weaning or other second-line agent withdrawal in a majority of cases.
本研究旨在调查霉酚酸酯（MMF）治疗儿童肾病综合征（NS）的疗效和副作用情况。
对2000年1月1日至2009年12月31日期间开始使用MMF且随访至少1年的所有NS患者进行回顾性病历审查。
样本量为73例患者。随访时间中位数为3.2年，四分位间距（IQR）为（1.7 - 4.7年）。诊断时的年龄中位数为3.2岁，IQR为（2.3 - 5.7年）。开始使用MMF的年龄中位数为11岁，IQR为（7.9 - 13.6年）。男孩（67%）多于女孩。大多数为白种人（77%），18%为亚洲人，4%为非洲黑人，1%为其他种族。初诊时，61例（84%）对类固醇敏感，9例（12%）类固醇抵抗，3例（4%）类固醇依赖（SD）。61例类固醇敏感患者中，45例（74%）变为SD，4例（7%）变为类固醇抵抗，1例（1%）仍对类固醇敏感，11例（18%）成为频繁复发者。至于之前使用二线免疫抑制剂的情况，5例（7%）患者未使用，17例（23%）使用一种药物，27例（37%）使用两种，23例（32%）患者使用三种或更多药物。MMF对45例（62%）患者有效。其中，38例（52%）缓解超过2年；7例（10%）MMF起效1至2年（MMF治疗选择性停药/仍在进行）。MMF治疗使27例（37%）患者完全停用类固醇，8例（11%）实现类固醇和免疫抑制剂完全停用。另有8例（11%）部分停用类固醇。第一年有13例（18%）MMF治疗失败，第二年有5例（7%）。4例（6%）因副作用停用MMF，4例（6%）因不依从停用。大多数患者无副作用[51例（70%）]。7例（9%）有胃肠道副作用（腹泻/腹痛）；5例（7%）有免疫相关副作用（白细胞减少/感染）；3例（4%）有免疫和胃肠道副作用；2例（3%）有关节痛。
MMF作为二线药物治疗类固醇敏感型NS耐受性良好且有效。在大多数情况下，该药物可实现长期缓解以及停用类固醇或其他二线药物。
