OBJECTIVES: To perform a cost-effectiveness analysis (CEA) of cysteamine in the treatment of infantile cystinosis vs. control group consisting of patients who had been given only conservative and symptomatic treatment (CaST). METHODS: Markov model was developed in TreeAge Pro 2009. The model evaluated the costs and health outcomes of cysteamine treatment at a dose of 1.30 g/m2/day compared with the use of CaST. The model distinguished two populations, depending on the time of initiation of the treatment (before the age of two – P1 and after the age of two – P2, which implies worse prognosis for the time of occurrence of end stage renal disease (ESRD)). The CEA was conducted from both a common payer perspective (a patient and a public payer) and a public payer perspective. The time horizon of the analysis covered the period from the age of one or four (depending on the start of cysteamine therapy) to fifty (currently, the oldest living patients with cystinosis reach the fifth decade of life). The main measures of the outcomes in the CEA were life-years gained (LYG) and life-years gained to the onset of ESRD. RESULTS: From the common payer perspective the cost per LYG was PLN 95,337 and PLN 192,272, respectively for the population P1 and P2. Cost of LYG to the onset of ESRD was PLN 33,317 and PLN 64,163, respectively for populations P1 and P2. The results obtained from the public payer perspective did not differ significantly from the results obtained from the common payer perspective. CONCLUSIONS: Cysteamine treatment of patients with cystinosis vs. therapy involving only CaST is more expensive, however produces better health outcomes. Regarding the acceptability threshold in Poland cysteamine therapy can be considered a cost-effective technology compared with CaST in patients who began treatment before the age of two.