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)