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Single centre retrospective study of the safety and efficacy of mycophenolate mofetil (MMF) in children and adolescents with nephrotic syndrome
  1. A Hassan,
  2. S Waller
  1. Department of Paediatric Nephrology, Evelina Children's Hospital, St. Thomas' Hospital, London, UK

Abstract

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.

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