Aim To investigate the efficacy and side effect profile of mycophenolate mofetil (MMF) therapy in children and adolescents with nephrotic syndrome.
Methods A retrospective case note review was performed on all patients with nephrotic syndrome who were commenced on MMF between 1/1/2000 to 31/12/2009 and followed up for a minimum of 1 year.
Results Sample size was 73 patients. Duration of follow up was for a median of 3.2 years, interquartile range (IQR) (1.7-4.7 years). Median age at diagnosis was 3.2 years, IQR (2.3-5.7 years). Median age of MMF commencement was 11 years, IQR (7.9- 13.6 years). There were more boys 49 (67%) than girls. Majority of patients were Caucasians 55 (75%), the remainder were Asian 13 (18%), Black African 3 (4%) and other ethnicities 2 (3%).
At initial diagnosis, 61 (84%) patients were steroid sensitive, 9 (12%) steroid resistant, 3 (4%) steroid dependent. Forty-five (74%) of the 61 steroid sensitive patients later became steroid dependent, 4 (7%) of them became steroid resistant, 1 (1%) remained steroid sensitive and 11 (18%) became frequent relapsers. Previous use of second line immunosuppressants: none 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, in 7 (10%) patients MMF worked for 1-2 years (MMF therapy electively stopped / ongoing); and 38 (52%) of them were in remission for >2 years. 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%) patients within first year and 5 (7%) in the second year. MMF was stopped due to side effects in 4 (5.5%) patients and non-compliance in 4 (5.5%).
Majority of patients had no side effects 51 (70%). Seven (9%) patients 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 nephrotic syndrome. The drug permitted prolonged remission and steroid weaning or other second line agent withdrawal in a majority of cases.