Treatment of childhood nephrotic syndrome with long-term, low-dose tacrolimus

Margret E. Bock*, Richard A Cohn, Farah N. Ali

*Corresponding author for this work

Research output: Contribution to journalArticle

7 Citations (Scopus)

Abstract

Aim: Children with steroidresistant (SR) and steroid-dependent (SD) nephrotic syndrome (NS) pose a treatment challenge. Literature on the use of tacrolimus (TAC), a calcineurin inhibitor, for maintenance treatment of NS is sparse. We aimed to evaluate the efficacy and safety of low-dose, long-term TAC for inducing and sustaining remission in children with SD/SR NS. Methods: Data from patients treated at our center from 1999 to 2009 were analyzed. Results: 40 patients with NS were treated with TAC for 3-80-month periods (median 25.2 months). Diagnoses included focal segmental glomerulosclerosis (FSGS) (60%), IgM nephropathy (15%), minimal change disease (20%) and membrano-proliferative glomerulonephritis (MPGN) (5%). 58% of patients had been previously treated with alternate agents. After 1, 2, and 3 years on TAC, complete remission was achieved in 26%, 48%, and 29% of patients; complete or partial remission was achieved in 85%, 100%, and 86%, respectively (p < 0.05). Median time to remission was 41 days (range: 10-270 days). FSGS and SR diseases were associated with lower likelihood of remission (p < 0.05). Remission was equally likely in both treatment naïve patients and those who had received prior second-line agents. Conclusion: Our results demonstrate that TAC treatment for children with SR/SD NS is associated with high rates of sustained remission, even when prior second-line agents failed.

Original languageEnglish (US)
Pages (from-to)432-438
Number of pages7
JournalClinical Nephrology
Volume79
Issue number6
DOIs
StatePublished - Aug 1 2013

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Nephrotic Syndrome
Tacrolimus
Focal Segmental Glomerulosclerosis
Steroids
Lipoid Nephrosis
Therapeutics
Glomerulonephritis
Immunoglobulin M
Safety

Keywords

  • 506
  • FK
  • Focal segmental glomerulosclerosis
  • Nephrotic syndrome
  • Pediatric
  • Steroid resistant

ASJC Scopus subject areas

  • Nephrology

Cite this

Bock, Margret E. ; Cohn, Richard A ; Ali, Farah N. / Treatment of childhood nephrotic syndrome with long-term, low-dose tacrolimus. In: Clinical Nephrology. 2013 ; Vol. 79, No. 6. pp. 432-438.
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abstract = "Aim: Children with steroidresistant (SR) and steroid-dependent (SD) nephrotic syndrome (NS) pose a treatment challenge. Literature on the use of tacrolimus (TAC), a calcineurin inhibitor, for maintenance treatment of NS is sparse. We aimed to evaluate the efficacy and safety of low-dose, long-term TAC for inducing and sustaining remission in children with SD/SR NS. Methods: Data from patients treated at our center from 1999 to 2009 were analyzed. Results: 40 patients with NS were treated with TAC for 3-80-month periods (median 25.2 months). Diagnoses included focal segmental glomerulosclerosis (FSGS) (60{\%}), IgM nephropathy (15{\%}), minimal change disease (20{\%}) and membrano-proliferative glomerulonephritis (MPGN) (5{\%}). 58{\%} of patients had been previously treated with alternate agents. After 1, 2, and 3 years on TAC, complete remission was achieved in 26{\%}, 48{\%}, and 29{\%} of patients; complete or partial remission was achieved in 85{\%}, 100{\%}, and 86{\%}, respectively (p < 0.05). Median time to remission was 41 days (range: 10-270 days). FSGS and SR diseases were associated with lower likelihood of remission (p < 0.05). Remission was equally likely in both treatment na{\"i}ve patients and those who had received prior second-line agents. Conclusion: Our results demonstrate that TAC treatment for children with SR/SD NS is associated with high rates of sustained remission, even when prior second-line agents failed.",
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Treatment of childhood nephrotic syndrome with long-term, low-dose tacrolimus. / Bock, Margret E.; Cohn, Richard A; Ali, Farah N.

In: Clinical Nephrology, Vol. 79, No. 6, 01.08.2013, p. 432-438.

Research output: Contribution to journalArticle

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AU - Ali, Farah N.

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N2 - Aim: Children with steroidresistant (SR) and steroid-dependent (SD) nephrotic syndrome (NS) pose a treatment challenge. Literature on the use of tacrolimus (TAC), a calcineurin inhibitor, for maintenance treatment of NS is sparse. We aimed to evaluate the efficacy and safety of low-dose, long-term TAC for inducing and sustaining remission in children with SD/SR NS. Methods: Data from patients treated at our center from 1999 to 2009 were analyzed. Results: 40 patients with NS were treated with TAC for 3-80-month periods (median 25.2 months). Diagnoses included focal segmental glomerulosclerosis (FSGS) (60%), IgM nephropathy (15%), minimal change disease (20%) and membrano-proliferative glomerulonephritis (MPGN) (5%). 58% of patients had been previously treated with alternate agents. After 1, 2, and 3 years on TAC, complete remission was achieved in 26%, 48%, and 29% of patients; complete or partial remission was achieved in 85%, 100%, and 86%, respectively (p < 0.05). Median time to remission was 41 days (range: 10-270 days). FSGS and SR diseases were associated with lower likelihood of remission (p < 0.05). Remission was equally likely in both treatment naïve patients and those who had received prior second-line agents. Conclusion: Our results demonstrate that TAC treatment for children with SR/SD NS is associated with high rates of sustained remission, even when prior second-line agents failed.

AB - Aim: Children with steroidresistant (SR) and steroid-dependent (SD) nephrotic syndrome (NS) pose a treatment challenge. Literature on the use of tacrolimus (TAC), a calcineurin inhibitor, for maintenance treatment of NS is sparse. We aimed to evaluate the efficacy and safety of low-dose, long-term TAC for inducing and sustaining remission in children with SD/SR NS. Methods: Data from patients treated at our center from 1999 to 2009 were analyzed. Results: 40 patients with NS were treated with TAC for 3-80-month periods (median 25.2 months). Diagnoses included focal segmental glomerulosclerosis (FSGS) (60%), IgM nephropathy (15%), minimal change disease (20%) and membrano-proliferative glomerulonephritis (MPGN) (5%). 58% of patients had been previously treated with alternate agents. After 1, 2, and 3 years on TAC, complete remission was achieved in 26%, 48%, and 29% of patients; complete or partial remission was achieved in 85%, 100%, and 86%, respectively (p < 0.05). Median time to remission was 41 days (range: 10-270 days). FSGS and SR diseases were associated with lower likelihood of remission (p < 0.05). Remission was equally likely in both treatment naïve patients and those who had received prior second-line agents. Conclusion: Our results demonstrate that TAC treatment for children with SR/SD NS is associated with high rates of sustained remission, even when prior second-line agents failed.

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