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Idiopathic nephrotic syndrome is steroid-sensitive in more than 90% of cases in children. However 60% of cases are steroid dependent and required treatment with immunosuppressive agent. Cyclophosphamide and ciclosporin are used for long time to reduce steroid dependency, but duration of these treatments should be restricted because of gonadotoxicity for cyclophosphamide and nephrotoxicity for ciclosporin.
Mycophenolate mofetil appears as an alternative treatment without gonadotoxicity and nephrotoxicity. However, contrary to cyclophosphamide, mycophenolate mofetil does not seem to have a residual action so that treatment must be maintained during months or years.
The aim of the study is to compare efficacy of cyclophosphamide and mycophenolate mofetil in steroid dependent nephrotic syndrome in children.
Aim of the study: Comparison of efficacy of cyclophosphamide 148mg/kg in 12 weeks and mycophenolate mofetil 1200mg/m² during 18 months, in children with steroid dependent nephrotic syndrome.
The 70 patients will be recruited in the 26 centres of paediatric nephrology in France, included and randomized at the time of a relapse. They will receive the same steroid treatment in the 2 arms.
The primary point will be occurrence of a relapse during the 24 months of follow-up. Detection of relapse will be done by using dipsticks and confirm by biological dosages (albuminemia and proteinuria/CREATININURIA ratio). Clinical and biological check up will be done every 3 months during all the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Mycophenolate mofetil | Experimental | Mycophenolate mofetil |
|
| Cyclophosphamide | Active Comparator | Cumulative dose of 148mg/kg of cyclophosphamide in 84 days (2mg/kg/day during 12 weeks) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cyclophosphamide | Drug | 2mg/kg/day during 12 weeks (cumulative dose 148mg/kg) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Relapse of nephrotic syndrome (defined by occurrence of proteinuria ≥ 0,25 g/mmol of CREATININURIA (or ≥ 2g/g) with hypoalbuminemia ≤ 30g/L AND/OR dipsticks >2+ during 3 days and proteinuria/CREATININURIA ratio ≥ 0,25 g/mmol) during 2 years. | Months 1, 3, 6, 9, 12, 15, 18, 21, 24 |
| Measure | Description | Time Frame |
|---|---|---|
| In case of relapse, steroid threshold dose to maintain a remission compare to those before inclusion in the study | Months 1, 3, 6, 9, 12, 15, 18, 21, 24 | |
| Cumulative steroid dose received during the years before and under treatment | Months 1, 3, 6, 9, 12, 15, 18, 21, 24 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Véronique BAUDOUIN, MD | Assistance Publique - Hôpitaux de Paris | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Robert Debre Hospital, AP-HP | Paris | Paris | 75019 | France |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39513526 | Derived | Larkins NG, Hahn D, Liu ID, Willis NS, Craig JC, Hodson EM. Non-corticosteroid immunosuppressive medications for steroid-sensitive nephrotic syndrome in children. Cochrane Database Syst Rev. 2024 Nov 8;11(11):CD002290. doi: 10.1002/14651858.CD002290.pub6. |
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| ID | Term |
|---|---|
| D009402 | Nephrosis, Lipoid |
| ID | Term |
|---|---|
| D009401 | Nephrosis |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
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| ID | Term |
|---|---|
| D003520 | Cyclophosphamide |
| D009173 | Mycophenolic Acid |
| ID | Term |
|---|---|
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
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| Mycophenolate mofetil | Drug | 1200mg/m²/jour in two divided doses during 18 months |
|
|
| Comparison of growth data, during the year before and under treatment | Months 1, 3, 6, 9, 12, 15, 18, 21, 24 |
| Pharmacokinetics measurement of MPA and relation with efficacy in case of treatment with MMF | One month after beginning MMF |
| D005261 |
| Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D005227 | Fatty Acids |
| D008055 | Lipids |