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

• Published in: Clinical nephrology Vol. 79; no. 6; p. 432
• Main Authors: Bock, Margret E, Cohn, Richard A, Ali, Farah N
• Format: Journal Article
• Language: English
• Published: Germany 01-06-2013
• Subjects: Adolescent; Child; Child, Preschool; Cohort Studies; Dose-Response Relationship, Drug; Female; Glomerular Filtration Rate - drug effects; Humans; Immunosuppressive Agents - administration & dosage; Infant; Male; Nephrotic Syndrome - drug therapy; Remission Induction; Retrospective Studies; Tacrolimus - administration & dosage; Time Factors; Treatment Outcome
• ISSN: 0301-0430
• DOI: 10.5414/CN107610

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.
Data from patients treated at our center from 1999 to 2009 were analyzed.
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.
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.