Mycophenolate mofetil in steroid-dependent idiopathic nephrotic syndrome.
Prospective studies have established the mycophenolate mofetil (MMF) efficiency in childhood idiopathic nephrotic syndrome (INS) but reports on the long-term outcome are lacking. Moreover, the search for factors influencing its efficiency would be useful to define its place among the other treatments.



We performed a monocentric retrospective study including 96 children with steroid-dependent INS followed for 4.7 years (median) (IQ 3-6) after the onset of MMF treatment. The characteristics of responder patients (n = 74), as defined by a 50 % decrease of relapse rate and/or a 60 % decrease of steroid dose, and of non-responder patients (n = 22) were compared by univariate analysis and multivariate logistic regression.



Withdrawal of prednisone was achieved in 48/96 patients after a median duration of 18.1 months (IQ 7.8-30.0) of MMF. Only 26/48 patients did not relapse under MMF alone. After MMF was stopped in these patients, only six remained in remission without any treatment at last follow-up. Responders had a shorter time to remission at the first flare (9.5 vs. 15 days, p = 0.02), a shorter disease duration prior to the onset of MMF (22.2 vs. 94.5 months, p = 0.001), and were younger at the MMF initiation (6.7 vs. 10.1 years, p = 0.02) than non-responder patients. The age of MMF initiation was an independent factor associated with efficiency (OR = 0.80, 95 % CI [0.69, 0.93], p < 0.01).



MMF is more efficient in young patients treated early in the disease course. Nevertheless, MMF has no remnant effect while nearly all patients relapsed after withdrawal of the drug.
Department of Pediatric Nephrology, Hôpital Robert Debré, APHP, 48 Boulevard Sérurier, 75935, Paris, Cedex 19, France. laurene.dehoux@aphp.fr.
Laurène
L
Dehoux
Department of Pediatric Nephrology, Hôpital Robert Debré, APHP, 48 Boulevard Sérurier, 75935, Paris, Cedex 19, France.
Julien
J
Hogan
Department of Pediatric Nephrology, Hôpital Robert Debré, APHP, 48 Boulevard Sérurier, 75935, Paris, Cedex 19, France.
Claire
C
Dossier
Department of Pediatric Nephrology, Hôpital Robert Debré, APHP, 48 Boulevard Sérurier, 75935, Paris, Cedex 19, France.
Marc
M
Fila
Department of Pediatric Nephrology, Hôpital Robert Debré, APHP, 48 Boulevard Sérurier, 75935, Paris, Cedex 19, France.
Olivier
O
Niel
Department of Pediatric Nephrology, Hôpital Robert Debré, APHP, 48 Boulevard Sérurier, 75935, Paris, Cedex 19, France.
Anne
A
Maisin
Department of Pediatric Nephrology, Hôpital Robert Debré, APHP, 48 Boulevard Sérurier, 75935, Paris, Cedex 19, France.
Marie Alice
MA
Macher
Department of Pediatric Nephrology, Hôpital Robert Debré, APHP, 48 Boulevard Sérurier, 75935, Paris, Cedex 19, France.
Thérésa
T
Kwon
Department of Pediatric Nephrology, Hôpital Robert Debré, APHP, 48 Boulevard Sérurier, 75935, Paris, Cedex 19, France.
Véronique
V
Baudouin
Department of Pediatric Nephrology, Hôpital Robert Debré, APHP, 48 Boulevard Sérurier, 75935, Paris, Cedex 19, France.
Georges
G
Deschênes
D000367:Age Factors; D000894:Anti-Inflammatory Agents, Non-Steroidal; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D004359:Drug Therapy, Combination / Q000379:methods; D005260:Female; D005500:Follow-Up Studies; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D009173:Mycophenolic Acid / Q000627:therapeutic use*; D009404:Nephrotic Syndrome / Q000188:drug therapy* / Q000453:epidemiology; D011241:Prednisone / Q000627:therapeutic use*; D012008:Recurrence; D012074:Remission Induction / Q000379:methods; D012189:Retrospective Studies; D013997:Time Factors; D016896:Treatment Outcome; D028761:Withholding Treatment
D000894:Anti-Inflammatory Agents, Non-Steroidal; D005938:Glucocorticoids; D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid; D011241:Prednisone
Child; Minimal change disease; Prednisone; Proteinuria; Relapse