Initial treatment of steroid-sensitive idiopathic nephrotic syndrome in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial (INTENT study)
Introduction Idiopathic nephrotic syndrome is the most common glomerular disease in childhood with an incidence of 1.8 cases per 100 000 children in Germany. The treatment of the first episode implies two aspects: induction of remission and sustainment of remission. The recent Kidney Disease Improvi...
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BMJ Publishing Group
2018-10-01
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| author | Burkhard Toenshoff Martin Konrad Steffen Luntz Anja Sander Peter F Hoyer Rasmus Ehren Marcus R Benz Jorg Doetsch Alexander Fichtner Jutta Gellermann Dieter Haffner Britta Höcker Bärbel Kästner Markus J Kemper Uwe Querfeld Lutz T Weber |
| author_facet | Burkhard Toenshoff Martin Konrad Steffen Luntz Anja Sander Peter F Hoyer Rasmus Ehren Marcus R Benz Jorg Doetsch Alexander Fichtner Jutta Gellermann Dieter Haffner Britta Höcker Bärbel Kästner Markus J Kemper Uwe Querfeld Lutz T Weber |
| author_sort | Burkhard Toenshoff |
| collection | DOAJ |
| description | Introduction Idiopathic nephrotic syndrome is the most common glomerular disease in childhood with an incidence of 1.8 cases per 100 000 children in Germany. The treatment of the first episode implies two aspects: induction of remission and sustainment of remission. The recent Kidney Disease Improving Global Outcomes, American Academy of Pediatrics and German guidelines for the initial treatment of the first episode of a nephrotic syndrome recommend a 12-week course of prednisone. Despite being effective, this treatment is associated with pronounced glucocorticoid-associated toxicity due to high-dose prednisone administration over a prolonged period of time. The aim of the INTENT study (Initial treatment of steroid-sensitive idiopathic nephrotic syndrom in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial) is to show that an alternative treatment regimen with mycophenolic acid is not inferior regarding sustainment of remission, but with lower toxicity compared with treatment with glucocorticoids only.Methods and design The study is designed as an open, randomised, controlled, multicentre trial. 340 children with a first episode of steroid-sensitive nephrotic syndrome and who achieved remission by a standard prednisone regimen will be enrolled in the trial and randomised to one of two treatment arms. The standard care group will be treated with prednisone for a total of 12 weeks; in the experimental group the treatment is switched to mycophenolate mofetil, also for a total of 12 weeks in treatment duration. The primary endpoint is the occurrence of a treated relapse within 24 months after completion of initial treatment.Ethics and dissemination Ethics approval for this trial was granted by the ethics committee of the Medical Faculty of the University of Heidelberg (AFmu-554/2014). The study results will be published in accordance with the Consolidated Standards of Reporting Trials statement and the Standard Protocol Items: Recommendations for Interventional Trials guidelines. Our findings will be submitted to major international paediatric nephrology and general paediatric conferences and submitted for publication in a peer-reviewed, open-access journal.Trial registration number DRKS0006547; EudraCT2014-001991-76; Pre-result.Date of registration 30 October 2014; 24 February 2017. |
| format | Article |
| id | doaj-art-cf528ab16d87474b82cad7b4c9076b51 |
| institution | Kabale University |
| issn | 2044-6055 |
| language | English |
| publishDate | 2018-10-01 |
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| spelling | doaj-art-cf528ab16d87474b82cad7b4c9076b512025-02-03T23:05:11ZengBMJ Publishing GroupBMJ Open2044-60552018-10-0181010.1136/bmjopen-2018-024882Initial treatment of steroid-sensitive idiopathic nephrotic syndrome in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial (INTENT study)Burkhard Toenshoff0Martin Konrad1Steffen Luntz2Anja Sander3Peter F Hoyer4Rasmus Ehren5Marcus R Benz6Jorg Doetsch7Alexander Fichtner8Jutta Gellermann9Dieter Haffner10Britta Höcker11Bärbel Kästner12Markus J Kemper13Uwe Querfeld14Lutz T Weber152 Department of Pediatrics I, University Children’s Hospital Heidelberg, Heidelberg, Germany6 Department of General Pediatrics, University Hospital Münster, Münster, Germany5 Coordination Centre for Clinical Trials (KKS), University of Heidelberg, Heidelberg, Baden-Württemberg, GermanyInstitut of Medical Biometry, Heidelberg University Hospital, Heidelberg, Germany5 Department of Pediatrics, University Children’s Hospital Essen, University Hospital Essen, Essen, Germany1 Department of Pediatrics, University Children’s Hospital Köln, University Hospital Köln, Köln, Germany1 Department of Pediatrics, University Children’s Hospital Köln, University Hospital Köln, Köln, Germany1 Department of Pediatrics, University Children’s Hospital Köln, University Hospital Köln, Köln, Germany2 Department of Pediatrics I, University Children’s Hospital Heidelberg, Heidelberg, Germany3 Department of Pediatrics, University Children’s Hospital Berlin, University Hospital Berlin Charité, Berlin, Germany4 Department of Pediatrics, University Children’s Hospital Hannover, University Hospital Hannover, Hannover, Germany2 Department of Pediatrics I, University Children’s Hospital Heidelberg, Heidelberg, Germany6 KKS (Coordination Center for Clinical Trials), University Hospital of Heidelberg, Heidelberg, Germany7 Department of Pediatrics, Asklepios Klinik Nord – Heidberg, Hamburg, Germany3 Department of Pediatrics, University Children’s Hospital Berlin, University Hospital Berlin Charité, Berlin, Germany1 Department of Pediatrics, University Children’s Hospital Köln, University Hospital Köln, Köln, GermanyIntroduction Idiopathic nephrotic syndrome is the most common glomerular disease in childhood with an incidence of 1.8 cases per 100 000 children in Germany. The treatment of the first episode implies two aspects: induction of remission and sustainment of remission. The recent Kidney Disease Improving Global Outcomes, American Academy of Pediatrics and German guidelines for the initial treatment of the first episode of a nephrotic syndrome recommend a 12-week course of prednisone. Despite being effective, this treatment is associated with pronounced glucocorticoid-associated toxicity due to high-dose prednisone administration over a prolonged period of time. The aim of the INTENT study (Initial treatment of steroid-sensitive idiopathic nephrotic syndrom in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial) is to show that an alternative treatment regimen with mycophenolic acid is not inferior regarding sustainment of remission, but with lower toxicity compared with treatment with glucocorticoids only.Methods and design The study is designed as an open, randomised, controlled, multicentre trial. 340 children with a first episode of steroid-sensitive nephrotic syndrome and who achieved remission by a standard prednisone regimen will be enrolled in the trial and randomised to one of two treatment arms. The standard care group will be treated with prednisone for a total of 12 weeks; in the experimental group the treatment is switched to mycophenolate mofetil, also for a total of 12 weeks in treatment duration. The primary endpoint is the occurrence of a treated relapse within 24 months after completion of initial treatment.Ethics and dissemination Ethics approval for this trial was granted by the ethics committee of the Medical Faculty of the University of Heidelberg (AFmu-554/2014). The study results will be published in accordance with the Consolidated Standards of Reporting Trials statement and the Standard Protocol Items: Recommendations for Interventional Trials guidelines. Our findings will be submitted to major international paediatric nephrology and general paediatric conferences and submitted for publication in a peer-reviewed, open-access journal.Trial registration number DRKS0006547; EudraCT2014-001991-76; Pre-result.Date of registration 30 October 2014; 24 February 2017.https://bmjopen.bmj.com/content/8/10/e024882.full |
| spellingShingle | Burkhard Toenshoff Martin Konrad Steffen Luntz Anja Sander Peter F Hoyer Rasmus Ehren Marcus R Benz Jorg Doetsch Alexander Fichtner Jutta Gellermann Dieter Haffner Britta Höcker Bärbel Kästner Markus J Kemper Uwe Querfeld Lutz T Weber Initial treatment of steroid-sensitive idiopathic nephrotic syndrome in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial (INTENT study) BMJ Open |
| title | Initial treatment of steroid-sensitive idiopathic nephrotic syndrome in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial (INTENT study) |
| title_full | Initial treatment of steroid-sensitive idiopathic nephrotic syndrome in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial (INTENT study) |
| title_fullStr | Initial treatment of steroid-sensitive idiopathic nephrotic syndrome in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial (INTENT study) |
| title_full_unstemmed | Initial treatment of steroid-sensitive idiopathic nephrotic syndrome in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial (INTENT study) |
| title_short | Initial treatment of steroid-sensitive idiopathic nephrotic syndrome in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial (INTENT study) |
| title_sort | initial treatment of steroid sensitive idiopathic nephrotic syndrome in children with mycophenolate mofetil versus prednisone protocol for a randomised controlled multicentre trial intent study |
| url | https://bmjopen.bmj.com/content/8/10/e024882.full |
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