Pediatric Nephrology

, Volume 20, Issue 1, pp 5–9

The clinical trial imperative

Editorial Commentary

Abstract

As clinical judgment has joined forces with evidence-based medicine, nephrologists critically assess the validity of the data available to support therapeutic decisions for children with kidney disease. Unfortunately, a paucity of data often prevents the practice of evidence-based medicine. Three scenarios illustrating the status of evidence and clinical decision making are provided. A review of the pediatric nephrology literature supports a call for general support of ongoing clinical trials and encourages the creation of additional feasible clinical trials to improve the practice of medicine.

Keywords

Nephrology Research Evidence 

References

  1. 1.
    Lowrie EG, Laird NM, Parker TF, Sargent JA (1981) Effect of the hemodialysis prescription of patient morbidity: report from the National Cooperative Dialysis Study. N Engl J Med 305:1176–1181PubMedGoogle Scholar
  2. 2.
    Gotch FA, Sargent JA (1985) A mechanistic analysis of the National Cooperative Dialysis Study (NCDS). Kidney Int 28:526–534PubMedGoogle Scholar
  3. 3.
    Hakim RM, Breyer J, Ismail N, Schulman G (1994) Effects of dose of dialysis on morbidity and mortality. Am J Kidney Dis 23:661–669PubMedGoogle Scholar
  4. 4.
    Collins AJ, Ma JZ, Umen A, Keshaviah P (1994) Urea index and other predictors of hemodialysis patient survival. Am J Kidney Dis 23:272–282PubMedGoogle Scholar
  5. 5.
    Held PJ, Port FK, Wolfe RA, Stannard DC, Carroll CE, Daugirdas JT, Bloembergen WE, Greer JW, Hakim RM (1996) The dose of hemodialysis and patient mortality. Kidney Int 50:550–556PubMedGoogle Scholar
  6. 6.
    Bloembergen WE, Stannard DC, Port FK, Wolfe RA, Pugh JA, Jones CA, Greer JW, Golper TA, Held PJ (1996) Relationship of dose of hemodialysis and cause-specific mortality. Kidney Int 50:557–565PubMedGoogle Scholar
  7. 7.
    National Kidney Foundation (1997) NKF-DOQI clinical practice guidelines for hemodialysis adequacy. Am J Kidney Dis 30:S15–S66Google Scholar
  8. 8.
    National Kidney Foundation—Dialysis Outcomes Quality Initiative (1997) NKF-DOQI clinical practice guidelines for the treatment of anemia of chronic renal failure. Am J Kidney Dis 30:S192–S240PubMedGoogle Scholar
  9. 9.
    Eknoyan G, Beck GJ, Cheung AK, Daugirdas JT, Greene T, Kusek JW, Allon M, Bailey J, Delmez JA, Depner TA, Dwyer JT, Levey AS, Levin NW, Milford E, Ornt DB, Rocco MV, Schulman G, Schwab SJ, Teehan BP, Toto R (2002) Effect of dialysis dose and membrane flux in maintenance hemodialysis. N Engl J Med 347:2010–2019CrossRefPubMedGoogle Scholar
  10. 10.
    National Institutes of Health (2003) U.S. Renal Data System, USRDS 2003 Annual Data Report: atlas of end-stage renal disease in the United States.Google Scholar
  11. 11.
    International Study of Kidney Disease in Children (1981) The primary nephrotic syndrome in children. Identification of patients with minimal change nephrotic syndrome from initial response to prednisone. J Pediatr 98:561–564PubMedGoogle Scholar
  12. 12.
    Pei Y, Cattran D, Delmore T, Katz A, Lang A, Rance P (1987) Evidence suggesting under-treatment in adults with idiopathic focal segmental glomerulosclerosis. Regional Glomerulonephritis Registry Study. Am J Med 82:938–944CrossRefPubMedGoogle Scholar
  13. 13.
    Rydel JJ, Korbet SM, Borok RZ, Schwartz MM (1995) Focal segmental glomerular sclerosis in adults: presentation, course, and response to treatment. Am J Kidney Dis 25:534–542PubMedGoogle Scholar
  14. 14.
    Korbet SM (1999) Clinical picture and outcome of primary focal segmental glomerulosclerosis. Nephrol Dial Transplant 14 [Suppl] 3:68–73Google Scholar
  15. 15.
    Wall CA, Troyanov S, Cattran DC (2003) Is partial remission a useful outcome in idiopathic focal segmental glomerulosclerosis? J Am Soc Nephrol 14:527AGoogle Scholar
  16. 16.
    Gipson DS, Huang K, Ferris ME, Massengill S, Presler T, Thomas DB (2003) The NY FSGS pathology classification: a pediatric perspective from the Glomerular Disease Collaborative Network. J Am Soc Nephrol 14:285–286Google Scholar
  17. 17.
    Ingulli E, Tejani A (1991) Racial differences in the incidence and renal outcome of idiopathic focal segemental glomerulosclerosis in children. Pediatr Nephrol 5:393–397PubMedGoogle Scholar
  18. 18.
    Ponticelli C, Rizzoni G, Edefonti A, Altieri P, Rivolta E, Rinaldi S, Ghio L, Lusvarghi E, Gusmano R, Locatelli F, Pasquali S, Castellani A, Casa-Alberighi OD (1993) A randomized trial of cyclosporine in steroid-resistant idiopathic nephrotic syndrome. Kidney Int 43:1377–1384PubMedGoogle Scholar
  19. 19.
    Lieberman KV, Tejani A (1996) A randomized double-blind placebo-controlled trial of cyclosporine in steroid-resistant idiopathic focal segmental glomerulosclerosis in children. J Am Soc Nephrol 7:56–63PubMedGoogle Scholar
  20. 20.
    Cattran DC, Appel GB, Hebert LA, Hunsicker LG, Pohl MA, Hoy WE, Maxwell DR, Kunis CL (1999) A randomized trial of cyclosporine in patients with steroid-resistant focal segmental glomerulosclerosis. North America Nephrotic Syndrome Study Group. Kidney Int 56:2220–2226CrossRefPubMedGoogle Scholar
  21. 21.
    Mendoza SA, Reznik VM, Griswold WR, Krensky AM, Yorgin PD, Tune BM (1990) Treatment of steroid-resistant focal segmental glomerulosclerosis with pulse methylprednisolone and alkylating agents. Pediatr Nephrol 4:303–307PubMedGoogle Scholar
  22. 22.
    Tarshish P, Tobin JN, Bernstein J, Edelmann CM (1996) Cyclophosphamide does not benefit patients with focal segmental glomerulosclerosis. A report of the International Study of Kidney Disease in Children. Pediatr Nephrol 10:590–593CrossRefPubMedGoogle Scholar
  23. 23.
    Briggs WA, Choi MJ, Scheel Jr PJ (1998) Successful mycophenolate mofetil treatment of glomerular disease. Am J Kidney Dis 31:213–217PubMedGoogle Scholar
  24. 24.
    Tahzib M, Frank R, Gauthier B, Valderrama E, Trachtman H (1999) Vitamin E treatment of focal segmental glomerulosclerosis: results of an open-label study. Pediatr Nephrol 13:649–652CrossRefPubMedGoogle Scholar
  25. 25.
    Weber S, Gribouval O, Esquivel EL, Moriniere V, Tete MJ, Legendre C, Niaudet P, Antignac C (2004) NPHS2 mutation analysis shows genetic heterogeneityof steroid-resistant nephrotic syndrome and low post-transplant recurrence. Kidney Int 66:571–579CrossRefPubMedGoogle Scholar
  26. 26.
    Kaplan JM, Kim SH, North KN, Rennke H, Correia LA, Tong HQ, Mathis BJ, Rodriguez-Perez JC, Allen PG, Beggs AH, Pollak MR. (2000) Mutations in ACTN4, encoding alpha-actinin-4, cause familial focal segmental glomerulosclerosis. Nat Genet 24:251–256CrossRefPubMedGoogle Scholar
  27. 27.
    Lahdenkari AT, Kestila M, Holmberg C, Koskimies O, Jalanko H (2004) Nephrin gene ( NPHS1) in patients with minimal change nephrotic syndrome (MCNS). Kidney Int 65:1856–1863CrossRefPubMedGoogle Scholar
  28. 28.
    Kaltenis P, Schumacher V, Jankauskiene A, Laurinavicius A, Royer-Pokora B (2004) Slow progressive FSGS associated with an F392L WT1 mutation. Pediatr Nephrol 19:353–356CrossRefPubMedGoogle Scholar
  29. 29.
    Ruf RG, Lichtenberger A, Karle SM, Haas JP, Anacleto FE, Schultheiss M, Zalewski I, Imm A, Ruf EM, Mucha B, Bagga A, Neuhaus T, Fuchshuber A, Bakkaloglu A, Hildebrandt F, Arbeitsgemeinschaft Fur Padiatrische Nephrologie Study Group (2004) Patients with mutations in NPHS2 (podocin) do not respond to standard steroid treatment of nephrotic syndrome. J Am Soc Nephrol 15:722–732PubMedGoogle Scholar

Copyright information

© IPNA 2004

Authors and Affiliations

  1. 1.Department of MedicineUniversity of North CarolinaChapel HillUSA
  2. 2.Department of PediatricsUniversity of North CarolinaChapel HillUSA
  3. 3.Department of Pediatrics, Schneider Children’s HospitalLong Island Jewish Medical CenterLong IslandUSA
  4. 4.Division of Nephrology and HypertensionUniversity of North CarolinaChapel HillUSA

Personalised recommendations