Clinical Trial: Steroid Treatment of Idiopathic Nephrotic Syndrome
Study Status: Completed
Recruit Status: Unknown status
Study Type: Observational
Official Title: Steroid Treatment of Childhood Idiopathic Nephrotic Syndrome: Epidemiology, Therapeutic Adequacy, Medium and Long Term Outcomes. A Prospective Observational Cohort Study.
Brief Summary:
Idiopathic nephrotic syndrome (INS) is the most frequent glomerular disease in childhood. Currently, all children with INS are treated at onset with steroids. The optimal duration and dosage of steroid therapy is debated. For each patient, the challenge is to minimise potential side effects of steroids, while achieving a good clinical response.
The aim of our study is to assess the benefits and potential adverse effects of a prolonged initial corticosteroid regimen, for the treatment of the initial episode. The results will be compared with data obtained retrospectively. In addition genetic studies will be undertaken with the aim of evaluating pharmacodynamics of steroid treatment with the ultimate goal to individualise treatment in single patients.
- Study group: children aged 6 months - 18 years, diagnosed with an initial episode of idiopathic nephrotic syndrome
- Control group: data of children with a onset INS between January 2007 and December 2009 from the same area of the study group and treated with a short steroid regimen will be retrospectively analysed and compared
Detailed Summary:
Background Idiopathic nephrotic syndrome (INS) is the most frequent glomerular disease in childhood, with an incidence of 2-4 cases per 100,000 children. Currently, all children with INS are treated at onset with steroids. Approximately 80% of cases respond to oral corticosteroid therapy, but 75-80% of these children will experience relapses which can recur over many years. The optimal duration and dosage of steroid therapy at onset and during subsequent relapses is debated. For each patient, the challenge is to minimise potential side effects of steroid therapy (hypertension, bone disease, Cushing syndrome, obesity, growth retardation, cataracts and a variety of psychological, social and behavioural disturbances) while achieving a good clinical response. This is particularly difficult at the beginning of the illness, because of the lack of reliable indicators that allow the identification of patients who will respond poorly or will experience frequent relapses. In 2006 the Cochrane Collaboration published the first systematic review of corticosteroid therapy for childhood idiopathic nephrotic syndrome. They concluded that children with the first episode of steroid sensitive INS should be treated for a minimum of three months, to achieve a reduction in the subsequent relapse rate.
The fine border between the optimal dose of steroids and the collateral effects can not be overlooked given the results of some recent studies (relative to patients with chronic inflammatory bowel disease or children with acute lymphoblastic leukemia subject to prolonged cycles of steroid therapy) based on the pharmacogenetic evaluation of the diverse individual responses to steroids. Clinical data supports the concept that the collateral effects of steroids are not exclusively dose related, but can depend on constitutional hypersensitivity or resistance, at times specific for a particular corti
Sponsor: Azienda Ospedaliera Universitaria di Bologna Policlinico S. Orsola Malpighi
Current Primary Outcome:
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Azienda Ospedaliera Universitaria di Bologna Policlinico S. Orsola Malpighi
Dates:
Date Received: June 30, 2011
Date Started: July 2011
Date Completion: July 2015
Last Updated: March 7, 2012
Last Verified: March 2012
