Nephropathic cystinosis

Active Ingredient: Mercaptamine

Indication for Mercaptamine

Population group: only infants (40 days - 1 year old) , children (1 year - 12 years old) , adolescents (12 years - 18 years old)
Therapeutic intent: Curative procedure

Mercaptamine is indicated for the treatment of proven nephropathic cystinosis. Cysteamine reduces cystine accumulation in some cells (e.g. leukocytes, muscle and liver cells) of nephropathic cystinosis patients and, when treatment is started early, it delays the development of renal failure.

For this indication, competent medicine agencies globally authorize below treatments:

For patients up to age 12 years 1.30 g/m²/day of the free base divided 4 times daily and for patients over age 12 and 50 kg weight 2 g/day, divided 4 times daily

For:

Dosage regimens

Regimen A: In case that patient age in years is < 12, oral, 1.3 grams mercaptamine per square meter of body surface, divided daily, 4 doses in total.

Regimen B: In case that patient age in years is ≥ 12 and patient weight is ≥ 50 kg, oral, 2 grams mercaptamine, divided daily, 4 doses in total.

Detailed description

Cysteamine treatment should be initiated under the supervision of a physician experienced in the treatment of cystinosis.

The goal of therapy is to keep leucocyte cystine levels below 1 nmol hemicystine/mg protein. White blood cell (WBC) cystine levels should therefore be monitored to adjust the dose. The WBC levels should be measured 5 to 6 hours after dosing and should be checked frequently when initiating therapy (e.g. monthly) and every 3-4 months when on a stable dose.

  • For children up to age 12 years, cysteamine dosing should be on the basis of body surface area (g/m²/day). The recommended dose is 1.30 g/m²/day of the free base divided four times daily.
  • For patients over age 12 and over 50 kg weight, the recommended cysteamine dose is 2 g/day, divided four times daily.

Starting doses should be ¼ to 1/6 of the expected maintenance dose, increased gradually over 4-6 weeks to avoid intolerance. The dose should be raised if there is adequate tolerance and the leucocyte cystine level remains >1 nmol hemicystine/mg protein. The maximum dose of cysteamine used in clinical trials was 1.95 g/m²/day.

The use of doses higher than 1.95 g/m²/day is not recommended.

Digestive tolerance of cysteamine is improved when the medicinal product is taken just after or with food.

Active ingredient

Mercaptamine

Mercaptamine reduces corneal cystine crystal accumulation acting as a cystine-depleting agent by converting cystine to cysteine and cysteine-cysteamine mixed disulfides.

Read more about Mercaptamine

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