Response to cyclosporine in steroid-resistant nephrotic Syndrome: discontinuation is possible
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Response to cyclosporine in steroid-resistant nephrotic Syndrome: discontinuation is possible. / Klaassen, Ilka Alena; Özgören, Bünyamin; Sadowski, Carolin E; Möller, Kristina; Van Husen, Michel; Lehnhardt, Anja; Timmermann, Kirsten; Freudenberg, Folke; Helmchen, Udo; Oh, Jun; Kemper, Markus Josef.
In: PEDIATR NEPHROL, Vol. 30, No. 9, 09.2015, p. 1477-1483.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Response to cyclosporine in steroid-resistant nephrotic Syndrome: discontinuation is possible
AU - Klaassen, Ilka Alena
AU - Özgören, Bünyamin
AU - Sadowski, Carolin E
AU - Möller, Kristina
AU - Van Husen, Michel
AU - Lehnhardt, Anja
AU - Timmermann, Kirsten
AU - Freudenberg, Folke
AU - Helmchen, Udo
AU - Oh, Jun
AU - Kemper, Markus Josef
PY - 2015/9
Y1 - 2015/9
N2 - BACKGROUND: Steroid-resistant nephrotic syndrome (SRNS) is still regarded as a serious disease although treatment with cyclosporine (CSA) has improved outcome. However, the duration of treatment in responders is unclear, and treatment of patients with genetic causes is a matter of debate.METHODS: Thirty-six patients with SRNS were studied retrospectively. Median age at presentation was 3.2 (range, 0.06-15.0) and median follow-up 15.5 years (range, 1.8-27.7), respectively; 23 (64 %) had focal segmental glomerulosclerosis (FSGS) on biopsy. In 33/36 patients (92 %), genetic testing was performed for at least three most common genes known to be mutated in SRNS.RESULTS: Nineteen patients (53 %), especially those with minimal change nephrotic syndrome (MCNS) at initial biopsy (p < 0.002), entered complete remission with CSA monotherapy, including one patient with compound heterozygous NPHS1 and dominant ACTN4 mutation, respectively. Ten patients entered partial remission (28 %, all FSGS), including two with NPHS2 mutations. Seven patients (six FSGS, one MCNS) did not respond to treatment. In 15 of 19 responders to CSA, treatment was stopped after a median of 3.1 years (range, 0.5-14) and no further relapses occurred in 11/15 (73 %) patients with median follow-up of 9.7 years.CONCLUSIONS: CSA monotherapy is effective in SRNS. Discontinuation of CSA is possible in many patients with complete remission.
AB - BACKGROUND: Steroid-resistant nephrotic syndrome (SRNS) is still regarded as a serious disease although treatment with cyclosporine (CSA) has improved outcome. However, the duration of treatment in responders is unclear, and treatment of patients with genetic causes is a matter of debate.METHODS: Thirty-six patients with SRNS were studied retrospectively. Median age at presentation was 3.2 (range, 0.06-15.0) and median follow-up 15.5 years (range, 1.8-27.7), respectively; 23 (64 %) had focal segmental glomerulosclerosis (FSGS) on biopsy. In 33/36 patients (92 %), genetic testing was performed for at least three most common genes known to be mutated in SRNS.RESULTS: Nineteen patients (53 %), especially those with minimal change nephrotic syndrome (MCNS) at initial biopsy (p < 0.002), entered complete remission with CSA monotherapy, including one patient with compound heterozygous NPHS1 and dominant ACTN4 mutation, respectively. Ten patients entered partial remission (28 %, all FSGS), including two with NPHS2 mutations. Seven patients (six FSGS, one MCNS) did not respond to treatment. In 15 of 19 responders to CSA, treatment was stopped after a median of 3.1 years (range, 0.5-14) and no further relapses occurred in 11/15 (73 %) patients with median follow-up of 9.7 years.CONCLUSIONS: CSA monotherapy is effective in SRNS. Discontinuation of CSA is possible in many patients with complete remission.
U2 - 10.1007/s00467-015-3109-3
DO - 10.1007/s00467-015-3109-3
M3 - SCORING: Journal article
C2 - 25903641
VL - 30
SP - 1477
EP - 1483
JO - PEDIATR NEPHROL
JF - PEDIATR NEPHROL
SN - 0931-041X
IS - 9
ER -
