A combined low-density lipoprotein apheresis and prednisone therapy for steroid-resistant primary focal segmental glomerulosclerosis in children.
BACKGROUND
Treatment of steroid-resistant (SR) primary focal segmental glomerulosclerosis (FSGS) remains a major challenge in nephrology. A prospective study was conducted to clarify the therapeutic role of low-density lipoprotein apheresis (LDL-A) in 11 nephrotic children with SR and cyclosporine A (CsA)-resistant primary FSGS.


METHODS
Based on entry criteria, all 11 eligible patients had biopsy-proven primary FSGS presenting with nephrotic syndrome (NS) and were resistant to steroid and conventional-dose CsA therapy. LDL-A was performed twice a week for 3 weeks (first course), then weekly for 6 weeks (second course). Beginning from the second LDL-A course, a dosage of 1 mg/kg/d of prednisone was administered for 6 weeks, then tapered.


RESULTS
Seven patients experienced remission of NS, 5 of whom achieved complete remission within 4 weeks after initiating prednisone therapy with LDL-A. These 5 patients maintained normal renal function during follow-up (median, 4.4 years). Of 2 patients with partial remission, 1 patient maintained stable renal function during follow-up (4.5 years), whereas the other patient showed a gradual decline in renal function and progressed to end-stage renal failure (ESRF) 7.8 years after LDL-A therapy. Four patients who were considered to experience treatment failure had persistent NS and progressed to ESRF in 1.3 years (median) after LDL-A therapy. Complete remission (n = 5) was associated with significantly more highly selective proteinuria compared with treatment failure (n = 4).


CONCLUSIONS
This study suggests that combined LDL-A and prednisone therapy can be a valuable addition to therapeutic options for treating patients with SR-FSGS. The role of LDL-A in treating these patients deserves to be assessed further in larger randomized controlled trials.
Hattori
Motoshi
M
Department of Pediatric Nephrology, Tokyo Women's Medical University, School of Medicine, Tokyo, Japan. hattori@kc.twmu.ac.jp
Chikamoto
Hiroko
H
Akioka
Yuko
Y
Nakakura
Hyogo
H
Ogino
Daisuke
D
Matsunaga
Akira
A
Fukazawa
Akira
A
Miyakawa
Sanpei
S
Khono
Miyuki
M
Kawaguchi
Hiroshi
H
Ito
Katsumi
K
D000293:Adolescent; D001781:Blood Component Removal; D002482:Cellulose; D002648:Child; D003131:Combined Modality Therapy; D016572:Cyclosporine / Q000494:pharmacology / Q000627:therapeutic use; D016264:Dextran Sulfate; D004351:Drug Resistance; D005260:Female; D005500:Follow-Up Studies; D005923:Glomerulosclerosis, Focal Segmental / Q000097:blood / Q000150:complications / Q000188:drug therapy / Q000628:therapy*; D006801:Humans; D006949:Hyperlipidemias / Q000188:drug therapy / Q000209:etiology / Q000628:therapy; D008077:Lipoproteins, LDL / Q000097:blood*; D008297:Male; D009404:Nephrotic Syndrome / Q000097:blood / Q000188:drug therapy / Q000209:etiology / Q000628:therapy; D011241:Prednisone / Q000627:therapeutic use*; D011446:Prospective Studies; D011507:Proteinuria / Q000188:drug therapy / Q000209:etiology / Q000628:therapy; D012074:Remission Induction; D016896:Treatment Outcome
D008077:Lipoproteins, LDL; D016572:Cyclosporine; D002482:Cellulose; D016264:Dextran Sulfate; D011241:Prednisone