No

Anticystine Agent; Urinary Tract Product

Orphan drug: Management of nephropathic cystinosis

C

Clinical effects on the fetus: Use only when the potential benefits outweigh the potential hazards to the fetus; in animal studies, cysteamine reduced the fertility of rats and offspring survival at very large doses

Breast-feeding/lactation: It is unknown whether cysteamine is excreted in breast milk; discontinue nursing or discontinue drug during lactation

Hypersensitivity to cysteamine or penicillamine

Withhold cysteamine if a mild rash develops; restart at a lower dose and titrate to therapeutic dose; adjust cysteamine dose if CNS symptoms due to the drug develop, rather than the disease; adjust cysteamine dose downward if severe GI symptoms develop (most common during initiation of therapy)

>5%:

Central nervous system: Fever, lethargy (11%)

Dermatologic: Rash (7%)

Gastrointestinal: Vomiting (35%), anorexia (31%), diarrhea (16%)

<5%:

Cardiovascular: Hypertension

Central nervous system: Somnolence, encephalopathy, headache, seizures, ataxia, confusion, dizziness, jitteriness, nervousness, impaired cognition, emotional changes, hallucinations, nightmares

Dermatologic: Urticaria

Endocrine & metabolic: Dehydration

Gastrointestinal: Bad breath, abdominal pain, dyspepsia, constipation, gastroenteritis, duodenitis, duodenal ulceration

Hematologic: Anemia, leukopenia

Hepatic: Abnormal LFTs

Neuromuscular & skeletal: Tremor, hyperkinesia

Otic: Decreased hearing

Symptoms may include vomiting, reduction of motor activity, GI or renal hemorrhage

Treatment is generally supportive; hemodialysis may be appropriate

Reacts with cystine in the lysosome to convert it to cysteine and to a cysteine-cysteamine mixed disulfide, both of which can then exit the lysosome in patients with cystinosis, an inherited defect of lysosomal transport

Initiate therapy with 1/4 to 1/8 of maintenance dose; titrate slowly upward over 4-6 weeks

Children >12 years and Adults (>110 lbs): 2 g/day in 4 divided doses; dosage may be increased to 1.95 g/m²/day if cystine levels are <1 nmol/1/2 cystine/mg protein, although intolerance and incidence of adverse events may be increased

Blood counts and LFTs during therapy; monitor leukocyte cystine measurements every 3 months to determine adequate dosage and compliance (measure 5-6 hours after administration); monitor more frequently when switching salt forms

Leukocyte cystine: <1 nmol/1/2 cystine/mg protein

Sedation is common; may cause confusion, nervousness, impaired cognition, and hallucinations

Concurrent use with psychotropic may produce additive sedation

No information available to require special precautions

No effects or complications reported

Take as directed. Maintain adequate hydration (2-3 L/day of fluids unless instructed to restrict fluid intake). It may be necessary to include other medication in treatment regimen. Periodic blood tests will need to be performed. You may experience dizziness, confusion, or lethargy; use caution with tasks that require alertness until response to drug is known. Report fever, gastric disturbances, or rash. Pregnancy/breast-feeding precautions: Inform prescriber if you are or intend to be pregnant. Consult prescriber if breast-feeding.

Capsule, as bitartrate: 50 mg, 150 mg