Intravenous delivery of cysteamine for the treatment of cystinosis: association with hepatotoxicity.
Nephropathic cystinosis is a lysosomal storage disorder, which, if untreated, results in renal failure by age 10 years. Oral cysteamine has been shown to preserve renal function in these patients. In this study, a 2-year-old girl with nephropathic cystinosis and severe gastrointestinal dysmotility was treated with intravenous (i.v.) administration of cysteamine hydrochloride (HCl). This is only the second report of long-term i.v. cysteamine therapy for nephropathic cystinosis. Unlike the treatment in the previous case, however, treatment in our patient was limited by liver toxicity.
Division of Pediatric Nephrology, MMC 491, University of Minnesota Children's Hospital, 420 Delaware St SE, Minneapolis, MN, 55455, USA. bende014@umn.edu
Michael R
MR
Bendel-Stenzel
Julia
J
Steinke
Ranjan
R
Dohil
Youngki
Y
Kim
D056486:Chemical and Drug Induced Liver Injury / Q000175:diagnosis / Q000209:etiology; D003131:Combined Modality Therapy; D003543:Cysteamine / Q000009:adverse effects / Q000627:therapeutic use*; D003554:Cystinosis / Q000150:complications / Q000503:physiopathology / Q000628:therapy*; D004359:Drug Therapy, Combination; D005260:Female; D005767:Gastrointestinal Diseases / Q000150:complications / Q000473:pathology / Q000503:physiopathology; D005769:Gastrointestinal Motility / Q000187:drug effects / Q000502:physiology; D006801:Humans; D007037:Hypothyroidism / Q000150:complications; D007223:Infant; D007275:Injections, Intravenous; D016030:Kidney Transplantation; D011837:Radiation-Protective Agents / Q000009:adverse effects / Q000627:therapeutic use*; D051437:Renal Insufficiency / Q000209:etiology / Q000601:surgery
D011837:Radiation-Protective Agents; D003543:Cysteamine