RESUMO
BACKGROUND: 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). METHODS: 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. RESULTS: 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. CONCLUSIONS: 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.
