Ongoing Trials
1) Mycophenolate in FSGS: The Glomerular Center is participating in the NIH-supported study of steroid resistant FSGS. Patients will be randomized to a mycophenolate mofetil (cellcept) arm and a cyclosporin arm. Enrollment of new patients is ongoing. 

2) Genetics of FSGS: Studies of the genetic forms of FSGS in collaboration with Dr. Martin Pollak of the Brigham and Womens Hospital are also ongoing. 

Completed Studies and Trials
North American Trial of Cyclosporine - This was a randomized, double blind, multicenter trial of cyclosporine vs. placebo in 50 patients with steroid resistant FSGS. The results showed greater partial and complete remissions with cyclosporine as well as better preservation of long-term renal function in the cyclosporine group.
(Cattran D, Appel GB, Hebert L, Hunsicker L, Pohl M, Hoy,Maxwell P, Kunis C. for the N. American collaborative Study Group. A randomized contolled trial of cyclosporine in steroid resistant FSGS.Kidney Int 56: 2220-2226, 1999.) 

Collapsing pattern of FSGS with Pamidronate: in patients receiving the drug pamidronate to prevent bone resorption during therapy for malignancies.
(Markowitz G, Appel GB, Fine P, Fenves AZ, Loon NR, JagannathS, Kuhn JA, Dratch A, D'Agati V. Collapsing FSGS following treatment with high dose pamidronate. J Amer Soc Neph.12:1164-1172, 2001.)

Plasmapheresis in FSGS: A review of data of patients at three major NY transplant centers shows the outcome of plasmapheresis in the treatment of recurrent FSGS in the transplanted kidney.
(Matalon A, Markowitz GS, Joseph RE, Cohen DJ, Saal SD, Kaplan B, D'Agati V, Appel GB. Plasmapheresis Treatment of Recurrent FSGS in Adult Transplant Recipients. Clin Neph 56:271-278, 2001.)

The Genetics of FSGS: A prior study in collaboration with Dr. Martin Pollak of Harvard University  examined the genetic abnormalities of glomerular epithelial cell proteins in Caucasians and African Americans with FSGS and  identifed a specific mutation (R229Q) that may define a subgroup of FSGS patients unresponsive to corticosteroids. This is clinically very important and may help guide therapy.
(Tsukaguchi H, Sudhakar A,  Appel GB,  and Pollak M et al . NPHS2 mutations in late-onset focal segmental glomerulosclerosis: R229Q is a common disease-associated allele. J Clin Invest 2002; 110: 1659–1666)

Other FSGS papers supported in part by The Glomerular Center
  • Stokes MB, Markowitz GS, Lin J, Valeri AM, D'Agati VD. Glomerular tip lesion: a distinct entity within the minimal change disease/focal segmental glomerulosclerosis spectrum. Kidney International  May;65(5):1690-70 .2004
  • Cattran D, Wang MM, Appel GB, Matalon A, Briggs. Mycophenolate mofetil in the treatment of focal segmental glomerulosclerosis.Clin Nephrol. Dec;62(6):405-11 2004.  This study treated 18 patients with FSGS with mycophenolate mofetil (cellcept) leading to improvement in proteinuria in approximately one half of patients.It provides support for the future NIH trial of FSGS using MMF.
  • Markowitz GS., Schwimmer J., Stokes MB, Nasr S, Seigle R, Valeri A D'Agati V. C1q nephropathy: A variant of focal segmental glomerulosclerosis.
    Kidney International 64 (4), 1232-1240 2003
  • Schwimmer JA, Markowitz GS, Valeri A, Appel GB. Collapsing glomerulopathy.Seminars in  Nephrology.  Mar; 23(2):209-18. 2003.
    This study reviews all current knowledge about the pathogenesis, etiology and course of this serious pattern of FSGS
  • Kambahm N, Markowitz GS, Valeri AM, Lin J, D'Agati V. Obesity-related glomerulopathy: An emerging epidemic. Kidney Int. 59: 1498-1509, 2001. 
  • Matalon A., Valeri A, Appel GB. Treatment of Focal Segmental Glomerulosclerosis. in Seminars in Nephrology 20:309-317, 2000.