Generic Name and Formulations:
Cysteamine bitartrate 25mg, 75mg; del-rel caps.
Indications for PROCYSBI:
Management of nephropathic cystinosis.
Adults and Children:
<2yrs: not established. Initiate immediately after diagnosis. Take on an empty stomach. Swallow whole or sprinkle contents of cap on 4oz of food (eg, applesauce or berry jelly) or mix in 4oz of liquids (eg, orange or apple juice); also, may administer via feeding tube after mixing with 4oz of applesauce. ≥2yrs (cysteamine-naive): initiate at ⅙ to ¼ of the maintenance dose; increase gradually over 4–6 weeks. Maintenance: 1.3g/m2/day, in 2 divided doses given every 12hrs. Max 1.95g/m2/day (if >50kg: max 1000mg every 12hrs). Goal of therapy: to maintain WBC cystine level <1nmol ½ cystine/mg protein. Switching from immediate-release cysteamine: give previous total daily dose of immediate-release caps. Dose titration, measuring WBC cystine levels: see full labeling.
Monitor for development of skin or bone lesions and interrupt dosing if occur; may restart at a lower dose. Discontinue if severe skin rash develops. Consider lowering the dose if severe GI symptoms develop. Evaluate and monitor for CNS symptoms (eg, somnolence, encephalopathy, seizures); interrupt or adjust dose if severe, persist or progress. Monitor WBCs and alkaline phosphatase levels. Monitor for signs/symptoms of benign intracranial hypertension; permanently discontinue if confirmed. Pregnancy. Nursing mothers: not recommended.
Avoid alcohol. Concomitant drugs that increase gastric pH (eg, PPIs); monitor WBC cystine levels. Separate dosing of carbonate- or bicarbonate-containing products by at least 1hr before or 1hr after. May give concomitant Vit.D, thyroid hormone, or other electrolyte/mineral replacements for Fanconi syndrome.
Vomiting, nausea, abdominal pain, breath or skin odor, diarrhea, fatigue, rash, headache; Ehlers-Danlos-like syndrome, GI ulceration/bleeding, leukopenia, elevated alkaline phosphatase, pseudotumor cerebri, papilledema.
Caps 25mg—60; 75mg—250