The Journal of Pediatrics
Volume 148, Issue 6 , Pages 718-719 , June 2006

A deeper look into cysteamine absorption for the treatment of cystinosis

  • Robert Kleta, MD, PhD

      Affiliations

    • Corresponding Author InformationReprint requests: Dr Robert Kleta, FASN, SHBG, MGB, NHGRI, NIH, Building 10, Room 10C103C, MSC 1851, 10 Center Drive, Bethesda, MD 20892

References 

  1. Gahl WA , Thoene JG , Schneider A . Cystinosis . N Engl J Med . 2002;347:111–121
  2. Kleta R , Bernardini I , Ueda M , et al.   Long-term follow-up of well-treated nephropathic cystinosis patients . J Pediatr . 2004;145:555–560
  3. Kleta R , Kaskel F , Dohil R , et al.  NIH Office of Rare Diseases   First NIH/Office of Rare Diseases Conference on Cystinosis (past, present, and future) . Pediatr Nephrol . 2005;20:452–454
  4. Sonies BC , Almajid P , Kleta R , Bernardini I , Gahl WA . Swallowing dysfunction in 101 patients with nephropathic cystinosis (benefit of long-term cysteamine therapy) . Medicine (Baltimore) . 2005;84:137–146
  5. Ueda M , O’Brien K , Rosing DR , et al.   Coronary artery and other vascular calcifications in cystinosis patients after kidney transplantation . Clin J Am Soc Nephrol . 2006; In press
  6. Kleta R , Gahl WA . Pharmacological treatment of nephropathic cystinosis with cysteamine . Expert Opin Pharmacother . 2004;5:2255–2262
  7. Schneider JA . Treatment of cystinosis (simple in principle, difficult in practice) . J Pediatr . 2004;145:436–438
  8. Gahl WA , Kuehl EM , Iwata F , Lindblad A , Kaiser-Kupfer MI . Corneal crystals in nephropathic cystinosis (natural history and treatment with cysteamine eyedrops) . Mol Genet Metab . 2000;71:100–120
  9. Dohil R , Fidler M , Barshop B , et al.   Understanding intestinal cysteamine bitartrate absorption . J Pediatr . 2006;148:764–769
  10. Dohil R , Newbury RO , Sellers ZM , Deutsch R , Schneider JA . The evaluation and treatment of gastrointestinal disease in children with cystinosis receiving cysteamine . J Pediatr . 2003;143:224–230
  11. Dohil R , Fidler M , Barshop B , et al.   Esomeprazole therapy for gastric acid hypersecretion in children with cystinosis . Pediatr Nephrol . 2005;20:1786–1793

 Supported in part by the Intramural Research Program of the National Human Genome Research Institute, National Institutes of Health.

PII: S0022-3476(06)00267-8

doi: 10.1016/j.jpeds.2006.03.048

The Journal of Pediatrics
Volume 148, Issue 6 , Pages 718-719 , June 2006

Article Tools