Indication:
Nephropathic Cystinosis
Cystinosis ( 469)
Cystinosis is rare disorder characterrized by the introsomal accumulation of free cystine in
body tissue. This results in apperarance of cystine crystals in cornea, conjuctivitis, bone marrow,
lymph nodes, leukocytes and internal organs.
These clinical forms have been identified - an infantile ( nephropathic ) form leading to the Fancomi
syndrome and renal insufficiency in the first decade, juvenile ( intermediate ) form in which the
renal disease becomes manifest during the second decade, and an adult ( benign ) form
charaterized by deposition of cystine in the cornea but not in the kidney.
Adverse Reaction:
Most common adverse reactions-
Anorexia 31% diarrhea 16% fever 22% lethargy 11% rash 7% vomiting 35%
Less common-
CNS- abnormal thinking, ataxia, confusion, decreased hearing, depression, dizziness,
emotional lability, encephalopathy, hallucinations, headache, hyperkinesia,
jitteriness, nervousness, nghtmares, seizures, somlonence, tremor.
GI- abdominal pain, bad breath, constipation, duodenitis, dyspepsia, gastroenteritis,
GI ulceration, and bleeding, nausea
GU- interstitial nephritis, renal failure,
Miscellaneous- abnormal liver function, dehydration, hypertension, leukopenia, urticaria
Contra-Indications:
Special precautions-
Rash- if a skin rash develops, withhold cysteamine until rash clears.
Cysteamine can be restarted at a lower dose.
CNS effects- CNS symptoms such as seizures, lethargy, somnolence, depression, and
encepholoapthy have been associated with cysteamine.
If CNS symptoms develop carefully evaluate the patient and adjust the dose as neccesary.
Patients should not engage in hazardous activites until the effects of cysteamine and mental
performance are known.
GI -GI ulceration and bleeding have been reported in patients receiving cysteamine tartarate.
Remiain alert for signs of ulceration and bleeding and inform patients/guardians, about the signs
and symptoms and what steps to take if they occur.
GI symptoms include, nausea, vomiting, and abdominal pain sometimes severe, have been
associated with cysteamine. if these develop therapy may have to be interrupted and dose
adjusted.
Hazardous tasks- cysteamine may cause some people to become drowsy or less alert than they
normally are. Make sure you know how you or your child ( the patient ) reacts to the medicine
before doing anything that could be dangerous.
Pregnancy- use cysteamime bitartrate during pregnancy only if required.
Lacation- decide whether to discontinue breast feeding or the drug taking into account the
importance of the drug to the mother.
Children - the safety and efficacy of cysteamine for cystinotic children have not been
established
Monitoring- monitor blood counts and liver function studies
Dosages/ Overdosage Etc:
Indication-
Nephropathic Cystinosis
Dosage-
Start new patients on1/4 to 1/6 of the maintenance dose of cysteamine. Initiate promptly once the
diagnosis is confirmed ( ie. increased white cell cystine).
Maintenance dose-
Children up to 12 years of age- is 1.2m2/day of the free base given in 4 divided doses.
Intact cystine should not be dministered to children younger than approximately 6 years of age,
because of the risk of aspiration.
cystamine capsule may be administered to children younger than approximately 6years of age
by sprinkling the capsule contents over food.
Patients oldrer than 12 years of age or over 110 Lbs. -
should receive 2g/day in 4 divided doses. This dosage should be reached after 4 to 6 weeks
of incremental dosage increases.
Other Information:
For Availability/supplies
Contact -
1.Indian Drug Manufacturers Association (IDMA)
Phone- 022- 24944624/ 24974308
Fax- 022- 24950723
Email- idma@vsnl.com
Website: www.idma-assn.org
2.Bulk Drug Manufacturers Association (India)(BDMA)
Phone - 040-23703910/ 23706718
Fax- 040-23704804
Email- info@bdmai.org
Website: www.info@bdmai.org
Patient Information:
Cysteamine - Systemic
1. Inform patients not to increase or decrease these medications without thier doctors
approval.
2. There are reports of unexpected deaths in children with cystinosis. Some these
children were receiving cysteamine/phosphocysteamine treatment for their cystinosis
3.Inform patients to not give children capsules to children approximately 6 years of age
because they may not in a position to swallow and they may choke.
4. For children younger than 6 years of age, the capsulemay be broken and contents
sprinkled on food .
5. Inform patients that it is important to take these supplements exactly as directed.
6. Regular blood tests to measure the amount of cystine inside white blood cells
are necessary to detremine the correct dose of cysteamine bitartrate
7. Ulcers and bleeding in the digestive tract have occurred while taking this medicine.
Explain to the patient and the guardian the warning signs of thes adverse reactions
8, Explain to the patients that their doctor may want to do certain tests to find out if any
unwanted effects are occuring. These tests are important because adverse
reactions including ulcers or bleeding in the digestive tract occur.
9.Allergies-
Tell your doctor if you are have ever had any unusual or allergic reaction to
cysteamine or penicillamine. Also tell your healthcare care professional if you are
allergic to any other substances such as foods. preservatives or dyes.
10.Pregnancy-
Cysteamine has not been studied in pregnant women. However,studies in animals
have shown that cysteamine causes a decrease in fertility and a decrease in
survival of their offspring. Before taking this medicine make sure that your doctor
knows if you are pregnant or if you may become pregnant
11. Breast-feeding-
It is known whether cysteamine passes into breast milk. Since cysteamine
has been reported to cause health problems in nursing animals,it may be
necessary for you to stop taking this medicine or to stop breast-feeding
during treatment.
12.Children-
This medicine has been tested in a limited number of children and in
effective doses has been shown to cause differnt side effects or
problems than it does in adults.
13.Older adults-
Many medicines have not been studied specifically in older people. Therefore,
it may not be known whether they work exactly the same way than they do in
younger adults, or if they cause different side effects or problems in older people.
There is no specific information comparing use of cysteamine in the elderly with use
in other age groups
14. Other medical problems-
The presence of other medical problems may affect the use of cysteamine.
Make sure you tell your doctor if you have any other medical problems
especially-
Blood problems (or history of) or
Convulsions (seizures) or
Liver diseases - cysteamine may make these conditions worse
Pharmacology/ Pharmacokinetics:
Pharmacology-
Cystamine is cysteine -depleting agents that lowers the cystine content of cells in patients with
cystosis, an inherent defect of lysomal transport.
Cystamine is aminithiol that participates within lysosomes in a thiol -disulfide interchange reaction
converting cystine into cystine and cysteine-cystamine mixed disulfide , both of which can exit
into lyosome in patients with cystinosis
Pregnancy and lactation:
Pregnancy-
Use cysteamime bitartrate during pregnancy only if required.
Lacation-
Decide whether to discontinue breast feeding or the drug taking into account the
importance of the drug to the mother.
Children -
The safety and efficacy of cysteamine for cystinotic children have not been
established
Manufacturer Details