OBJECTIVE: Cystinosis causes renal and other organ failure. Treatment with 6-hourly cysteamine bitartrate (Cystagon, Mylan, Morgantown, West Virginia) reduces intracellular cystine and the rate of organ deterioration. A recent study showed that an enteric-release cysteamine required less frequent daily dosing. This report describes the long-term use of enteric-coated (EC) cysteamine bitartrate (Cystagon) in children with cystinosis. STUDY DESIGN: After a pharmacokinetic and pharmacodynamic study of EC-cysteamine in children with cystinosis, 5 patients remained on twice-daily treatment. White blood cell cystine levels were measured 12 hours after ingestion every 4 to 8 weeks. These levels were then compared with the patient's previous 6-h post-dose levels taken while on regular cysteamine bitartrate before entering the study. Blood chemistry was also measured. RESULTS: Five children with cystinosis (mean age, 9 years; range, 8 to 17 years) who previously took cysteamine bitartrate (mean dose, 47 mg/kg body wt), received EC-cysteamine for 10 to 27 months (mean dose, 25 mg/kg body wt) and had mean white blood cell cystine levels of 0.77 and 0.71 nmol half-cystine/mg protein, respectively. During the study period, patients maintained adequate growth and there was no significant deterioration in renal or thyroid function. Two children were required to restart acid suppression after 6 months on EC-cysteamine therapy. CONCLUSIONS: Long-term, twice-daily EC-cysteamine, given at approximately 60% of the previous daily dose of cysteamine bitartrate, was effective at maintaining white blood cell cystine levels within a satisfactory range. There was no significant deterioration in renal or thyroid function.
OBJECTIVE:Cystinosis causes renal and other organ failure. Treatment with 6-hourly cysteamine bitartrate (Cystagon, Mylan, Morgantown, West Virginia) reduces intracellular cystine and the rate of organ deterioration. A recent study showed that an enteric-release cysteamine required less frequent daily dosing. This report describes the long-term use of enteric-coated (EC) cysteamine bitartrate (Cystagon) in children with cystinosis. STUDY DESIGN: After a pharmacokinetic and pharmacodynamic study of EC-cysteamine in children with cystinosis, 5 patients remained on twice-daily treatment. White blood cell cystine levels were measured 12 hours after ingestion every 4 to 8 weeks. These levels were then compared with the patient's previous 6-h post-dose levels taken while on regular cysteamine bitartrate before entering the study. Blood chemistry was also measured. RESULTS: Five children with cystinosis (mean age, 9 years; range, 8 to 17 years) who previously took cysteamine bitartrate (mean dose, 47 mg/kg body wt), received EC-cysteamine for 10 to 27 months (mean dose, 25 mg/kg body wt) and had mean white blood cell cystine levels of 0.77 and 0.71 nmol half-cystine/mg protein, respectively. During the study period, patients maintained adequate growth and there was no significant deterioration in renal or thyroid function. Two children were required to restart acid suppression after 6 months on EC-cysteamine therapy. CONCLUSIONS: Long-term, twice-daily EC-cysteamine, given at approximately 60% of the previous daily dose of cysteamine bitartrate, was effective at maintaining white blood cell cystine levels within a satisfactory range. There was no significant deterioration in renal or thyroid function.
Authors: Daryl M Okamura; Nadia M Bahrami; Shuyu Ren; Katie Pasichnyk; Juliana M Williams; Jon A Gangoiti; Jesus M Lopez-Guisa; Ikuyo Yamaguchi; Bruce A Barshop; Jeremy S Duffield; Allison A Eddy Journal: J Am Soc Nephrol Date: 2013-09-05 Impact factor: 10.121
Authors: Craig B Langman; Larry A Greenbaum; Minnie Sarwal; Paul Grimm; Patrick Niaudet; Georges Deschênes; Elisabeth Cornelissen; Denis Morin; Pierre Cochat; Debora Matossian; Segolene Gaillard; Mary Jo Bagger; Patrice Rioux Journal: Clin J Am Soc Nephrol Date: 2012-05-03 Impact factor: 8.237
Authors: Doris A Trauner; Jennifer Williams; Angela O Ballantyne; Amy M Spilkin; Jennifer Crowhurst; John Hesselink Journal: Pediatr Nephrol Date: 2010-07-22 Impact factor: 3.714
Authors: Zygmunt Warzecha; Dagmara Ceranowicz; Artur Dembiński; Piotr Ceranowicz; Jakub Cieszkowski; Atsukazu Kuwahara; Ikuo Kato; Marcin Dembiński; Peter C Konturek Journal: Med Sci Monit Date: 2012-05
Authors: Martijn J Wilmer; Joost P Schoeber; Lambertus P van den Heuvel; Elena N Levtchenko Journal: Pediatr Nephrol Date: 2010-08-24 Impact factor: 3.714
Authors: Toshio Fujisawa; Benjamin Rubin; Akiko Suzuki; Prabhudas S Patel; William A Gahl; Bharat H Joshi; Raj K Puri Journal: PLoS One Date: 2012-04-20 Impact factor: 3.240