Impact of the National Institutes of Health Focal Segmental Glomerulosclerosis (NIH FSGS) clinical trial on the treatment of steroid-resistant FSGS

Nephrology Dialysis Transplantation

Volumn 28, Issue 3, 2013, Pages 527-534

  Canetta, Pietro A ^a   Radhakrishnan, Jai ^a  

^a Columbia University College of Physicians and Surgeons ^*  (United States)

Author keywords

Cyclosporine; Focal segmental glomerulosclerosis; Glomerular disease; Mycophenolate; Randomized clinical trials

Indexed keywords

CYCLOSPORIN A; DEXAMETHASONE; GLUCOCORTICOID; METHYLPREDNISOLONE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PLACEBO; PREDNISOLONE; PREDNISONE; STEROID; TACROLIMUS;

ADOLESCENT; ADULT; BONE DISEASE; CARDIOVASCULAR DISEASE; CHILD; CONTROLLED STUDY; CREATININE CLEARANCE; CUSHING SYNDROME; DRUG FATALITY; DRUG MEGADOSE; DRUG PULSE THERAPY; DRUG RESPONSE; FEMALE; FOCAL GLOMERULOSCLEROSIS; GASTROINTESTINAL SYMPTOM; GLOMERULUS FILTRATION RATE; HUMAN; HYPERGLYCEMIA; IMMUNOSUPPRESSIVE TREATMENT; INFECTION; KIDNEY FAILURE; LOW DRUG DOSE; MAJOR CLINICAL STUDY; MALE; MENTAL INSTABILITY; MULTICENTER STUDY; NATIONAL HEALTH ORGANIZATION; NEPHROTOXICITY; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEINURIA; RANDOMIZED CONTROLLED TRIAL; RELAPSE; REMISSION; REVIEW; SCHOOL CHILD; STRIA;

CLINICAL TRIALS AS TOPIC; DRUG RESISTANCE, NEOPLASM; GLOMERULOSCLEROSIS, FOCAL SEGMENTAL; HUMANS; NATIONAL INSTITUTES OF HEALTH (U.S.); STEROIDS; UNITED STATES;

EID: 84874818258     PISSN: 09310509     EISSN: 14602385     Source Type: Journal    
DOI: 10.1093/ndt/gfs563     Document Type: Review

References (47)

1. Strippoli GF, Craig JC, Schena FP. The number, quality, and coverage of randomized controlled trials in nephrology. J Am Soc Nephrol 2004; 15: 411-419
2. Samuels JA, Molony DA. Randomized controlled trials in nephrology: state of the evidence and critiquing the evidence. Adv Chronic Kidney Dis 2012; 19: 40-46
3. Kovesdy CP, Kalantar-Zadeh K. Observational studies versus randomized controlled trials: avenues to causal inference in nephrology. Adv Chronic Kidney Dis 2012; 19: 11-18
4. Leaf DE, Appel GB, Radhakrishnan J. Glomerular disease: why is there a dearth of high quality clinical trials? Kidney Int 2010; 78: 337-342
5. Deegens JK, Wetzels JF. Immunosuppressive treatment of focal segmental glomerulosclerosis: lessons from a randomized controlled trial. Kidney Int 2011; 80: 798-801
6. Chun MJ, Korbet SM, Schwartz MM et al. Focal segmental glomerulosclerosis in nephrotic adults: presentation, prognosis, and response to therapy of the histologic variants. J Am Soc Nephrol 2004; 15: 2169-2177
7. Meyrier A. Mechanisms of disease: focal segmental glomerulosclerosis. Nat Clin Pract Nephrol 2005; 1: 44-54
8. D'Agati VD, Kaskel FJ, Falk RJ. Focal segmental glomerulosclerosis. N Engl J Med 2011; 365: 2398-2411
9. Rydel JJ, Korbet SM, Borok RZ et al. Focal segmental glomerular sclerosis in adults: presentation, course, and response to treatment. Am J Kidney Dis 1995; 25: 534-542
10. Korbet SM, Schwartz MM, Lewis EJ. Primary focal segmental glomerulosclerosis: clinical course and response to therapy. Am J Kidney Dis 1994; 23: 773-783
11. Kitiyakara C, Eggers P, Kopp JB. Twenty-one-year trend in ESRD due to focal segmental glomerulosclerosis in the United States. Am J Kidney Dis 2004; 44: 815-825
12. Gipson DS, Trachtman H, Kaskel FJ et al. Clinical trial of focal segmental glomerulosclerosis in children and young adults. Kidney Int 2011; 80: 868-878
13. Velosa JA, Donadio JV, Jr, Holley KE. Focal sclerosing glomerulonephropathy: a clinicopathologic study. Mayo Clin Proc 1975; 50: 121-133
14. Cameron JS, Turner DR, Ogg CS et al. The long-term prognosis of patients with focal segmental glomerulosclerosis. Clin Nephrol 1978; 10: 213-218
15. Troyanov S, Wall CA, Miller JA et al. Focal and segmental glomerulosclerosis: definition and relevance of a partial remission. J Am Soc Nephrol 2005; 16: 1061-1068
16. Banfi G, Moriggi M, Sabadini E et al. The impact of prolonged immunosuppression on the outcome of idiopathic focal-segmental glomerulosclerosis with nephrotic syndrome in adults. A collaborative retrospective study. Clin Nephrol 1991; 36: 53-59
17. Cattran DC, Rao P. Long-term outcome in children and adults with classic focal segmental glomerulosclerosis. Am J Kidney Dis 1998; 32: 72-79
18. Chapter 6: Idiopathic focal segmental glomerulosclerosis in adults. Kidney Int 2012; 2: 181-185
19. Stirling CM, Mathieson P, Boulton-Jones JM et al. Treatment and outcome of adult patients with primary focal segmental glomerulosclerosis in five UK renal units. QJM 2005; 98: 443-449
20. The primary nephrotic syndrome in children. Identification of patients with minimal change nephrotic syndrome from initial response to prednisone. A report of the International Study of Kidney Disease in children. J Pediatr 1981; 98: 561-564
21. Ponticelli C, Villa M, Banfi G et al. Can prolonged treatment improve the prognosis in adults with focal segmental glomerulosclerosis? Am J Kidney Dis 1999; 34: 618-625
22. Deegens JK, Assmann KJ, Steenbergen EJ et al. Idiopathic focal segmental glomerulosclerosis: a favourable prognosis in untreated patients? Neth J Med 2005; 63: 393-398
23. Pei Y, Cattran D, Delmore T et al. Evidence suggesting undertreatment in adults with idiopathic focal segmental glomerulosclerosis. Regional Glomerulonephritis Registry Study. Am J Med 1987; 82: 938-944
24. Adhikari M, Bhimma R, Coovadia HM. Focal segmental glomerulosclerosis in children from KwaZulu/Natal, South Africa. Clin Nephrol 2001; 55: 16-24
25. Ponticelli C, Rizzoni G, Edefonti A et al. A randomized trial of cyclosporine in steroid-resistant idiopathic nephrotic syndrome. Kidney Int 1993; 43: 1377-1384
26. Cattran DC, Appel GB, Hebert LA et al. A randomized trial of cyclosporine in patients with steroid-resistant focal segmental glomerulosclerosis. North America Nephrotic Syndrome Study Group. Kidney Int 1999; 56: 2220-2226
27. Melocoton TL, Kamil ES, Cohen AH et al. Long-term cyclosporine A treatment of steroid-resistant and steroid-dependent nephrotic syndrome. Am J Kidney Dis 1991; 18: 583-588
28. Ittel TH, Clasen W, Fuhs M et al. Long-term cyclosporine A treatment in adults with minimal change nephrotic syndrome or focal segmental glomerulosclerosis. Clin Nephrol 1995; 44: 156-162
29. Ghiggeri GM, Catarsi P, Scolari F et al. Cyclosporine in patients with steroid-resistant nephrotic syndrome: an open-label, nonrandomized, retrospective study. Clin Ther 2004; 26: 1411-1418
30. Heering P, Braun N, Mullejans R et al. Cyclosporine A and chlorambucil in the treatment of idiopathic focal segmental glomerulosclerosis. Am J Kidney Dis 2004; 43: 10-18
31. Lieberman KV, Tejani A. A randomized double-blind placebocontrolled trial of cyclosporine in steroid-resistant idiopathic focal segmental glomerulosclerosis in children. J Am Soc Nephrol 1996; 7: 56-63
32. Plank C, Kalb V, Hinkes B et al. Cyclosporin A is superior to cyclophosphamide in children with steroid-resistant nephrotic syndrome-a randomized controlled multicentre trial by the Arbeitsgemeinschaft fur Padiatrische Nephrologie. Pediatr Nephrol 2008; 23: 1483-1493
33. Braun N, Schmutzler F, Lange C et al. Immunosuppressive treatment for focal segmental glomerulosclerosis in adults. Cochrane Database Syst Rev 2008, Issue 3. Art No.: CD003233
34. Matalon A, Valeri A, Appel GB. Treatment of focal segmental glomerulosclerosis. Semin Nephrol 2000; 20: 309-317
35. Imbasciati E, Cagnoli L, Case N et al. Controlled study of treatment of steroids and chlorambucil, in alternate months, for membranous nephropathy and focal glomerulosclerosis. Preliminary evaluation of the results. Minerva Nefrol 1980; 27: 571-575
36. Li X, Li H, Ye H et al. Tacrolimus therapy in adults with steroid- and cyclophosphamide-resistant nephrotic syndrome and normal or mildly reduced GFR. Am J Kidney Dis 2009; 54: 51-58
37. Duncan N, Dhaygude A, Owen J et al. Treatment of focal and segmental glomerulosclerosis in adults with tacrolimus monotherapy. Nephrol Dial Transplant 2004; 19: 3062-3067
38. Segarra A, Vila J, Pou L et al. Combined therapy of tacrolimus and corticosteroids in cyclosporin-resistant or -dependent idiopathic focal glomerulosclerosis: a preliminary uncontrolled study with prospective follow-up. Nephrol Dial Transplant 2002; 17: 655-662
39. Choudhry S, Bagga A, Hari P et al. Efficacy and safety of tacrolimus versus cyclosporine in children with steroid-resistant nephrotic syndrome: a randomized controlled trial. Am J Kidney Dis 2009; 53: 760-69
40. Choi MJ, Eustace JA, Gimenez LF et al. Mycophenolate mofetil treatment for primary glomerular diseases. Kidney Int 2002; 61: 1098-1114
41. Montane B, Abitbol C, Chandar J et al. Novel therapy of focal glomerulosclerosis with mycophenolate and angiotensin blockade. Pediatr Nephrol 2003; 18: 772-777
42. Cattran DC, Wang MM, Appel G et al. Mycophenolate mofetil in the treatment of focal segmental glomerulosclerosis. Clin Nephrol 2004; 62: 405-411
43. Li Z, Duan C, He J et al. Mycophenolate mofetil therapy for children with steroid-resistant nephrotic syndrome. Pediatr Nephrol 2010; 25: 883-888
44. Senthil Nayagam L, Ganguli A, Rathi M et al. Mycophenolate mofetil or standard therapy for membranous nephropathy and focal segmental glomerulosclerosis: a pilot study. Nephrol Dial Transplant 2008; 23: 1926-1930
45. Gipson DS, Trachtman H, Kaskel FJ et al. Clinical trials treating focal segmental glomerulosclerosis should measure patient quality of life. Kidney Int 2011; 79: 678-685
46. Tune BM, Kirpekar R, Sibley RK et al. Intravenous methylprednisolone and oral alkylating agent therapy of prednisone-resistant pediatric focal segmental glomerulosclerosis: a long-term follow-up. Clin Nephrol 1995; 43: 84-88
47. Rovin BH, McKinley AM, Birmingham DJ. Can we personalize treatment for kidney diseases? Clin J Am Soc Nephrol 2009; 4: 1670-1676