Overview
The drug Cysteamine is approved by the United States Food and Drug Administration (FDA) and can be used in the treatment of nephropathic cystinosis. It is a condition that occurs by birth leading to complications associated with kidneys. Symptomatic treatment along with administration of Cysteamine is done. The drug works by the formation of various bi-products. The drug is available in 25 dosages and 75 dosages that are supplied in a bottle. Care should be taken to store the bottle. Administration of the medication is done starting from a lower dose and then increasing the dosage. Individuals suffering from Ehlers-danlos syndrome (a group of conditions affecting connective tissues such as skin, blood vessels, and joints), or the occurrence of symptoms such as skin rashes, ulcers, and bleeding in the stomach should be cautious during the administration of the drug.
How Does the Drug Cysteamine Work?
The drug works by reducing the build-up of cystine crystals by forming bi-products like cysteine, and cysteine-cysteamine. Interchange reactions with lysosomes occur, leading to the exit of cysteine crystals from lysosomes in patients diagnosed with cystinosis.
Available Doses and Dosage Forms:
Recommended Dosage: 25 milligrams (mg) to 75 milligrams of delayed-release capsule
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25 mg Delayed Release Capsule: Capsule with an opaque cap of light blue color imprinted with a raptor logo and light blue body with 25 mg imprint and supplied in a bottle containing 60 capsules.
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75 mg Delayed Release Capsule: Capsule cap of dark blue color with raptor logo and light blue body imprinted with 75 mg and supplied in a bottle containing 250 capsules.
Warnings:
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Ehlers-Danlos Syndrome: The dosage should be reduced when bony or skin lesions tend to occur.
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Skin rash: Erythema multiforme (skin lesion that can be identified with the help of papules occurring as a hypersensitivity reaction) or toxic epidermal necrolysis are skin rashes of a severe nature that can occur due to the administration of Cysteamine. The drug should be discontinued if they occur.
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Gastrointestinal Ulcers and Bleeding: Gastrointestinal symptoms are monitored, and the dose is decreased if symptoms become severe.
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Elevated Levels of Alkaline Phosphatase Levels or Leukopenia: The dose should be stopped or decreased until the levels of alkaline phosphatase and white blood cells get back to normal.
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Benign Intracranial Hypertension: Look out for the signs of the condition and reduce the dose accordingly. The drug should be discontinued upon confirmation of the diagnosis.
For Patients:
What Is Nephropathic Cystinosis?
It is a disorder leading to a condition of severe renal disease along with extra renal complications. This is caused due to the mutation of the gene CTNS. This condition was first reported due to the presence of cystine crystals in the spleen and liver.
Following are the types of cystinosis:
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Infantile nephropathic cystinosis.
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Juvenile nephropathic cystinosis.
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Non-nephropathic cystinosis.
How Is Nephropathic Cystinosis Diagnosed?
Clinical conditions determining nephropathic cystinosis are as follows:
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Polyuria.
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Polydipsia.
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Failure to thrive.
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Metabolic acidosis.
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Electrolytes loss.
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Fructose intolerance.
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Genetic testing confirms the diagnosis.
Treatment
It is treated by depletion therapy, in which the progression of the renal failure is slowed down along with decreasing the extra renal complications.
Systemic Depletion Therapy: Depletion of cystine takes place, generating molecules like cysteine and cysteine-cysteamine
Learn More About the Drug:
Before Starting:
When and Why to Take the Drug Cysteamine?
The drug is taken for the treatment of a condition called nephropathic cystinosis. A condition that can lead to severe kidney disease. It is taken twice daily. It can be taken along with food.
Things to Inform the Doctor Before Taking the Drug Cysteamine:
The doctor should be informed of the following:
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Presence of a habit of drinking alcohol.
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The presence of bone-related problems is skin issues such as skin rash.
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A medical history of stomach or intestinal problems like bleeding or ulcers.
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History of lack of energy, seizures, depression, unusual sleepiness, or any changes in the ability to think properly.
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The presence of the liver is a blood problem.
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Presence of any medical conditions under treatment or have been diagnosed.
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Pregnant or planning to become pregnant. As it is not known of the harmful effects on the child, the condition should be brought in notice of the physician to avoid any potential risks.
What Are the Side Effects of the Drug Cysteamine?
Serious side effects can occur during the treatment with this drug, such as listed below:
1. Skin Joint and Bone Problems: Treatment of people with increased doses of the drug may lead to the development of abnormal changes in their skin and bone changes such as stretch marks, fractures, bony injuries, and bony deformities leading to pain in the joints can occur. Minute changes in the skin have to be noticed or observed while taking the drug. Any skin problem or changes should be brought in notice to the physician immediately.
2. Skin Rash: The most commonly occurring side effect is a skin rash which can also turn out to be severe. Decreasing the dose of the drug can help in decreasing the growth of the rash. The drug can also be stopped upon the advice of the physician when the rash is severe.
3. Stomach or Intestinal Problems: Bleeding or answers in the stomach can develop when the individual is taking medications that contain cysteamine bitartrate.
4. Central Nervous System Symptoms: Depression, seizures, and sleepiness can occur under treatment with this drug. This medication can affect the functioning of the brain leading to encephalopathy. Development of any symptom should be told to the doctor right away.
5. Benign Intracranial Hypertension: Immediate-release formulation of the truck can sometimes lead to pseudotumor cerebri. It is a condition in which there is increased pressure of fluid around the brain. Examination to detect the condition and the problem is treated at the earliest.
Common side effects:
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Vomiting.
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Nausea.
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Diarrhea.
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Tiredness.
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Headache.
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Abdominal pain.
How to Use the Drug Cysteamine?
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The drug is to be taken two times each day at a frequency of 12 hours.
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At first, the drug can be administered at a lower dose, upon which the dose can be increased helping in avoiding the side effects.
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The capsule is to be taken on the whole.
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The capsule or capsule contents should not be crushed or chewed.
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The medication can be taken with any fruit juice except grape juice.
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Avoid consuming food two hours prior and 30 minutes at least after taking medication to increase absorption. The amount of food can be limited to half a cup, nearly four ounces if the individual is not able to stop the food before taking the medication. The capsule is to be taken in a consistent manner considering the intake of food. High-fat food can be avoided.
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Alcohol should be avoided during the treatment period with Cysteamine.
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When the patient is under treatment with medications containing carbonate or bicarbonate, administer Cysteamine an hour before and an hour after.
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What should be avoided while taking the drug Cysteamine?
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Avoid driving or operating any machinery until the effects of the drug are known. The drug can make an individual sleepy or can decrease alertness than normal.
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Avoid drinking alcohol. As alcohol can change the effect and mechanism of the drug and can also increase the amount of the drug in your blood, causing serious side effects it is advised not to drink alcohol when and the treatment with the drug Cysteamine.
Missed Dose:
Take the missed dose as soon as possible up to eight hours after the actual time of the missed dose This can be skipped if the duration for the next dose is less than four hours. The next dose can be taken regularly at the scheduled time. Care should be taken not to administer two doses at once to compensate, as it can result in toxicity.
Drug Overdose:
Overdosing can occur due to accidental administration of the drug or wanted administration of the drug to harm themselves. Nausea and vomiting were reported when a second dose of the drug was taken 30 minutes after the first dose. Vomiting and dehydration can be expected due to overdosing, and administration of fluids along with proper care and follow-up of the patient helps in recovering from overdosing.
Drug Storage:
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The drug is stored at 20 to 25 degrees Celsius, and the permitted excursion is between 15 and 30-degree centigrade. It should be protected from moisture and light.
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Re-package or subdivision of the drug is not advised. The medication is dispensed in original packaging. The oxygen absorber or desiccant should not be removed from the container. The container should be tightly closed and placed in a dry area.
Avoid Self-Medication:
The drug should not be used upon the suggestion of another individual already using the drug. Cysteamine should be used only under a proper prescription by the physician.
For Doctors:
Indication:
The drug Cysteamine is indicated in patients suffering from nephropathic cystinosis.
Dosing:
25 milligrams and 75 milligrams dosages are available.
The patient is administered the minimum dose at first, after which it is increased.
What Are the Pharmacological Aspects of Cysteamine?
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Mechanism of Action:
The drug is an aminothiol that participates in a reaction that converts cystine to cysteine and a mixed disulphate which can exit the lysosomes in patients suffering from nephropathic cystinosis, thereby decreasing the crystal cysteine content.
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Pharmacodynamics:
Persons suffering from nephropathic cystinosis have concentrations of WBC system that are less than required. The administration of single doors of the drug Cysteamine can increase the concentration of WBC 15 to the peak within 3.5 hours and can return to the normal concentration after 12 hours.
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Absorption:
Absorption for the delayed-release formulation is 188 minutes and that of immediate-release Cysteamine is 73 minutes. When food was administered 30 minutes after taking the drug, the C max was decreased by 34% and by 32% when the individual had taken a meal 2 hours after the dose.
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Elimination:
The concentration of Cysteamine tends to decrease in the blood continuously for 30 minutes after each dose and the concentration of WBC cysteine increases accordingly.
Plasma clearance is similar between immediate-release formulation and delayed-release formulation.
Clinical Toxicity:
Carcinogenesis, Mutagenesis, and Impairment of Fertility: The drug was proven non-mutagenic and was reported to show no effect on reproductive or fertility performance. At an oral dosage of 375 milligrams, the fertility rate was altered along with changes in the survival of the offspring.
Contraindications:
The drug is not indicated in individuals with hypersensitivity reactions to the drug and Penicillins.
What Are the Drug Interactions of Cysteamine?
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Drugs Increasing Gastric PH: Proton pump inhibitors and medications containing carbonates and bicarbonate are the drugs that increase the pH of the stomach. These can in turn alter the pharmacokinetics of the drug as Cysteamine is released prematurely, increasing the WBC cystine concentration.
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Use of Drugs With Alcohol: Consumption of alcohol along with the drug Cysteamine can increase the rate of release of the drug and adversely change the properties along with the safety and effectiveness of the drug. Alcohol consumption is avoided when the individual is under treatment with Cysteamine.
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Medications Used in Managing Fanconi Syndrome: Administration of the drug Cysteamine with other mineral and electrolyte replacements which are necessary for the management of Fanconi syndrome along with thyroid hormone and vitamin D does not pose any negative effects.
Other Specifications:
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Pregnancy: Pregnant women are advised about the potential risk to the child when the woman is under treatment with the drug Cysteamine. The risk of miscarriages or major birth defects occurred nearly in two to four percent of pregnancies.
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Lactation: No reports were present informing the accumulation of the drug in human milk. No effects on breastfed infants or milk production effects were also reported. As it was identified in the milk of rats, the potential risk of the presence of the drug in human milk is informed prior.
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Pediatric Use: Treatment of pediatric patients of age 2 years or older for nephropathic cystinosis is safe and effective using the drug Cysteamine.
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Geriatric Use: No studies have been conducted to test the safety of this drug in geriatric patients.
