A comparison of immediate release and delayed release cysteamine in 17 patients with nephropathic cystinosis

Abstract Background Nephropathic cystinosis is a rare and severe metabolic disease leading to an accumulation of cystine in lysosomes which especially harms kidney function. A lifelong therapy with the aminothiol cysteamine can delay development end-stage renal necessity transplantation. The purpose our study was compare effectiveness immediate-release delayed-release on levels as well assessing onset adverse effects. Methods We retrospectively analysed 17 patients after single dose (Cystagon®, Mylan Pharmaceuticals, Canonsburg, PA Recordati Pharma GmbH) (Procysbi®; Horizon USA Chiesi Farmaceutici S.p.A., Parma, Italy) respectively. Data were collected during period three years context optimizing individual treatment regimens. DR-cysteamine reduced 70% equivalent IR-cysteamine. efficacy both formulas depleting white blood cells’ their side effects compared. Results Immediate (IR)- (DR) effectively decreased intracellular under target value 0.5 nmol cystine/mg protein, while fewer occurred DR-cysteamine. Mean maximum reached 60 min IR-cysteamine 180 Conclusion effective less reported. Our data show that should be dosed higher than order decrease over extended time. Moreover, suggest increasing dosing scheme Procysbi® times daily, prevent rapid achieve steadier decline levels. Due more convenient scheme, might ameliorate adherence improve patients’ quality life.