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.
Bendel-Stenzel
Michael R
MR
Division of Pediatric Nephrology, MMC 491, University of Minnesota Children's Hospital, 420 Delaware St SE, Minneapolis, MN, 55455, USA. bende014@umn.edu
Steinke
Julia
J
Dohil
Ranjan
R
Kim
Youngki
Y
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