Abstract
Since there is no specific therapy for cystinosis and since AA decreases the free-cystine content of cystinotic fibroblasts (Science 86:1040, 1974), a randomized double-blind trial of high dose (200 mg/Kg/day) AA was undertaken. An unblinded committee monitored the data every 4 months. At entry the AA and placebo (PL) groups were comparable in all major clinical parameters. There were 52 patient years for the AA group and 50 patient years for the PL group. Of the 11 patients who died, began chronic dialysis or were transplanted, 8 had received AA. Neither treatment affected the WBC cystine content. Patients receiving AA had a greater mean rise in serum creatinine (Cr). This was especially true in patients whose initial Cr was ≥1.0 & <3.5. Although a small benificial effect of AA could not be excluded statistically, it was more likely that AA hastened the progression of renal failure. In view of this adverse risk-benefit ratio the study was terminiated.
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Schneider, J., Schlesselman, J., Mendoza, S. et al. 562 FAILURE OF ASCORBIC ACID (AA) THFRAPY IN NEPHROPATHY CYSTINOSIS. Pediatr Res 12 (Suppl 4), 457 (1978). https://doi.org/10.1203/00006450-197804001-00567
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DOI: https://doi.org/10.1203/00006450-197804001-00567