Limited and discordant data are available on
cyclosporine A (CsA) treatment for
proteinuria in
Alport syndrome (AS). To address this lack of consistent data, we have studied 15 AS patients (14 males; mean age 15.3 +/- 6.0 years) treated with CsA. Patient selection criteria included a urinary
protein/
creatinine ratio > or =1 mg/mg and a
creatinine clearance >40 ml/min/1.73 m(2). CsA treatment was started at an initial dose of 5 mg/kg/day and subsequently adjusted to reach target C2 levels of 500 ng/ml. Renal function,
proteinuria, and blood pressure were monitored. Blood pressure was treated to avoid the administration of
angiotensin converting enzyme or
angiotensin receptor blockers for the first 2 years of
therapy. The average follow-up was 3.5 years. Five patients had
chronic renal failure at the beginning of treatment, of whom three and one reached
end-stage renal failure within 1 and 3 years, respectively. In the remaining 11 patients, the glomerular filtration rate declined by 11 +/- 6% within 6 months, but remained stable thereafter.
Proteinuria decreased by 63 +/- 21% from baseline, but returned nearly to baseline after 2.5 years of follow-up. Based on these results, we suggest that CsA is effective in reducing
proteinuria in patients with
Alport syndrome but that this effect is temporary. Our data do not support the use of CsA
therapy for proteinuric patients with AS, particularly if they have
chronic renal failure.