Pharmacotherapeutic review and update of idiopathic nephrotic syndrome in children

Aim of the review The therapeutic management of pediatric idiopathic nephrotic syndrome is still a challenge due to the large number of potentially effective pharmacological alternatives and the insufficient scientific evidence available. A bibliographic review was performed in order to identify the available pharmacological alternatives, as well as their place in therapy, and to analyze whether the treatment algorithm developed by the pediatric nephrology department of our hospital is consistent with the evidence published to date. Method A literature search was carried out on MEDLINE, through PubMed, using the medical subject heading (MeSH) nephrotic syndrome. The search was limited to review papers, meta-analyses, clinical practice guidelines, and randomized controlled trials performed on pediatric populations up to September 2009. Results The treatment algorithm established in our hospital is consistent with the evidence available. Prednisone constitutes the first line treatment with evidence level Ia. When corticosteroids do not achieve remission, there are other therapeutic options that are not clearly positioned yet and further studies that provide more information on their comparative efficacy and safety are needed. These alternative therapeutic options are cyclosporine, mycophenolate mofetil, levamisol, cyclophosphamide and methylprednisolone, as independent strategies or as part of “Mendoza Protocol”, tacrolimus and rituximab. Their sequence of introduction in the therapeutic scheme of NS is classified as evidence level IV and grade D recommendation. Conclusion The wide range of options available for the pharmacotherapeutic management of NS and the lack of evidence about the comparative efficacy and safety of the different therapeutic strategies, make its positioning rather difficult. Therefore each hospital needs to draw up protocols based not only on the small amount of evidence available, but also on the authorized indications, availability of the drugs, clinical experience, associated costs, and patient preferences with regard to the duration of treatment, incidence and type of adverse effects. Development of new randomized controlled trials should be encouraged and setting up national plans for the treatment of this pathology might be a good approach for this problem.