Efficacy of tacrolimus in the treatment of children with focal segmental glomerulosclerosis

Mahmoud Kallash, Diego Aviles

New Orleans, LA, USA

Author Affiliations: Pediatric Nephrology Department, Louisiana State University Health Science Center and Children's Hospital of New Orleans, 200 Henry Clay Ave, New Orleans, LA 70118, USA (Kallash M, Aviles D)

Corresponding Author: Mahmoud Kallash, MD, 200 Henry Clay Ave, New Orleans, LA 70118, USA (Tel: 318 658 5750; Fax: 504 896-9240; Email: mkallash@yahoo.com/mkalla@lsuhsc.edu)

doi: 10.1007/s12519-014-0484-y

Background: Focal segmental glomerulosclerosis (FSGS) is the most common glomerular condition leading to end-stage renal disease (ESRD) and the third most common cause of ESRD in pediatric patients.

Methods: This is a retrospective study consisting of 22 pediatric patients with FSGS and heavy proteinuria. After demonstrating steroids resistance, the patients were treated with tacrolimus, targeting a trough level 5-8 ng/mL. The primary outcome is the induction of remission with tacrolimus.

Results: Thirteen patients (59%) achieved remission (complete in 31.8% and partial in 27.2%) and 12 patients showed stable or improved renal function over an average follow-up of 2.9 years (range: 0.5-7 years). There was no significant difference in response rate between African American and Caucasian patients. None of the patients had significant side-effect to tacrolimus and none of the repeat biopsies showed an increase in interstitial fibrosis compared to baseline. The best renal outcome was for patients who achieved complete remission. Partially responsive patients had improved renal function compared with resistant patients.

Conclusion: Tacrolimus is a viable option in the treatment of children with idiopathic steroid resistant FSGS.

Key words: chronic kidney disease; focal segmental glomerulosclerosis (FSGS); proteinuria; tacrolimus

World J Pediatr 2014;10(2):151-154