Drugs for the treatment of patients with thalassemia.

Treatment for thalassemia depends on which type you have and how severe it is.

Mild thalassemia
is the most common form and does not require treatment.
Moderate thalassemia
(thalassemia intermedia and more severe hemoglobin H disease) may be treated with:(thalassemia intermedia and more severe hemoglobin H disease) may be treated with:

  • Folic acid (a vitamin that your body needs to produce new red blood cells). People with thalassemia often lack enough folic acid in their diets to keep a good supply of red blood cells.
  • Blood transfusions. You may need a blood transfusion when your body is under stress, such as during an infection. If you have moderate thalassemia, do not take medicines that increase the amount of iron in the body, which can damage organs. These medicines include:
  • Iron supplements or multivitamins that contain iron.
  • Vitamin C, which can increase the amount of iron that your body absorbs from food

Severe thalassemia
is often treated with:

  • Regular blood transfusions. A child with severe thalassemia typically starts to have symptoms when he or she is 6 months old and will need blood transfusions every 4 to 6 weeks. The transfusions often lead to increased iron levels, which also may require treatment.
  • Folic acid, which your body needs to produce red blood cells. A person with thalassemia needs folic acid supplements because red blood cells are destroyed faster than normal. As a result, the body often uses up its stores of this vitamin.

Indications for Starting Iron Chelation

  • The need for iron chelation should be discussed from an early stage when it is clear that a child is going to need long-term regular transfusion. This should be included in any discussions with the parents concerning the risks and benefits of transfusion.
  • Iron chelation should be started when:

  •  10 or more blood transfusions have been given
  • The serum ferritin is consistently more than 1000 μg/l
  • The child has been receiving regular transfusions for more than one year
  • It is planned to continue the transfusions for at least a further 12 months
  • Ideally chelation should not be started before the age of 2 years

Deferiprone
Brand Names Kelfer® or GPO L-1
Why do I need this medicine? Deferiprone is used to remove excess iron in your body. Iron may build up in your body after repeated blood transfusions. Too much iron in your body can damage your vital organs  such as the heart and the liver.
How do I take this medicine? Deferiprone comes as a tablet to take by mouth. It is usually taken three times a day. Take Deferiprone exactly as directed by your doctor. Do not take more or less than instructed by your doctor. Do not stop Deferiprone unless your doctor has advised you to do so. You may take Deferiprone with or without food. If you experience nausea or vomiting. you may take it with food. Your doctor may adjust your dose of deferiprone every 2 to 3 months depending on your laboratory test results.
What should I do if I have forgotten to take this medicine? - Take the missed dose as soon as you remember. If it is almost time for your next dose skip the missed dose and return to your normal dosing schedule.
- DO NOT double a dose under any circumstances. If you accidentally take more Deferiprone than instructed alert your doctor immediately.
- Remember to take Deferiprone exactly as directed. If you often forget to take your medicine let your doctor and pharmacist know.
When should I not use this medicine? - Do not take Deferiprone together with other similar medicines that remove excess iron from your body such as desferrioxamine.
- Alert your doctor if you have low white blood cell counts or any blood disorders. Alert your doctor if you are taking any medicine which lowers your white blood cell counts.
- Do not take Deferiprone if you are pregnant or breastfeeding.
What should I take note of while taking this medicine? Inform your doctor if you have liver or kidney disease an infection of the liver called hepatitis or HIV infection (commonly known as AIDS).For as long as you are taking Deferiprone you will need to have regular blood tests to monitor your body's response to the medicine. Your doctor will advise you about how often you need to have blood tests.
What side effects could I experience? - Common side effects of Deferiprone include nausea vomiting diarrhoea stomach pain joint pain and an increased appetite.
- Deferiprone will cause your urine to turn a reddish brown in colour.
- Inform your doctor immediately if you experience fever sore throat or flu-like symptoms.
- Some side effects can be serious. If you experience any of these symptoms or those listed in the IMPORTANT WARNING section call your doctor immediately or get emergency medical treatment: reddish purple spots or rash especially on the lower half of the body hives swelling around the eyes fast or pounding heartbeat dizziness lightheadedness fainting
Can I take this with other medicines? - Alert your doctor if you are taking vitamin C (ascorbic acid) or any antacids which contain aluminium salts.
- Always inform your doctor and pharmacist if you are taking any other medicines including herbal tonics supplements and medicines that you buy without a prescription.
How should I store this medicine? Keep this medication in the container it came in tightly closed and out of reach of children. Store it at room temperature and away from excess heat and moisture (not in the bathroom). Throw away any medication that is outdated or no longer needed. Talk to your pharmacist about the proper disposal of your medication.
Deferoxamine
Brand Names  Desferal®
Why do I need this medicine? Desferrioxamine is used to remove excess iron in your body. Iron may build up in your body after repeated blood transfusions. Too much iron in your body can damage your vital organs such as the heart and the liver. Desferrioxamine may also be used to remove excess aluminium in the body in people who have severe kidney disease.
How do I take this medicine? Desferrioxamine is given as an injection.
It may be injected by yourself or by your doctor.
If you are injecting Desferrioxamine by yourself your doctor or nurse will teach you how to do the injection.
Inject Desferrioxamine exactly as directed by your doctor. Do not inject more or less than instructed by your doctor. Follow your doctor's instructions exactly.
Desferrioxamine may be injected by
    – intramuscular injection (injection into a muscle)
    – slow intravenous infusion (injection into a vein)
    – slow subcutaneous infusion (injection beneath the skin) using an infusion pump.
Desferrioxamine should be dissolved in the solvent provided by your doctor or pharmacist before use.The reconstituted solution should be clear and pale yellow in colour. Do not use if the solution appears cloudy or discoloured. The reconstituted solution should be stored at room temperature.Throw away the used syringe as instructed by your doctor. Do not dispose in the normal household waste.
What should I do if I have forgotten to take this medicine? Take the missed dose as soon as you remember. DO NOT double a dose under any circumstances. If you accidentally inject more Desferrioxamine than instructed alert your doctor immediately. If you often forget to inject your medicine let your doctor and pharmacist know.
When should I not use this medicine? Alert your doctor if you have kidney disease. Alert your doctor if you are pregnant or breastfeeding. Do not breastfeed while you are being treated with Desferrioxamine. Desferrioxamine is not recommended for use in children younger than 3 years old.
What should I take note of while taking this medicine? Inform your doctor if you have heart disease hyperparathyroidism or diabetes. For as long as you are taking Desferrioxamine you will need to have regular blood and urine tests to check your iron level. Your doctor will advise you about how often you need to have these tests. As long-term use of Desferrioxamine may affect your vision and hearing you will also need to go for eyesight and hearing tests from time to time. Children who are treated with Desferrioxamine will need to have regular checks to monitor their growth and body weight.
What side effects could I experience? Common side effects of Desferrioxamine include pain irritation and redness at the injection site muscle and joint pain fever rash headache and nausea. As Desferrioxamine may impair your vision or hearing do not drive or take part in any activity in which you need to be alert. Desferrioxamine will cause your urine to turn reddish-brown in colour. This is normal; do not be alarmed. Alert your doctor quickly if you experience any of these side effects:
- difficulty breathing
- unexplained diarrhoea stomach pain fever or sore throat
- changes or loss of hearing or vision
- rapid heartbeat
- chest pain
Can I take this with other medicines? Alert your doctor if you are taking any other medicines especially those listed here:
    – vitamin C (ascorbic acid) – prochlorperazine (a medicine to treat nausea and vomiting)
    – erythropoietin injection (an injection for treating anaemia)
    – methyldopa (a blood pressure medicine)
Always inform your doctor and pharmacist if you are taking any other medicines including herbal tonics supplements and medicines that you buy without a prescription
Are there any restrictions on the type of food? Avoid alcohol.
Avoid excessive consumption of beverages or foods containing vitamin C as it can interfere with your treatment with Desferrioxamine.
How should I store this medicine? Store below in a cool dry place away from the reach of children. The reconstituted solution should be stored at room temperature. Medicines must not be used past the expiry date.
Deferasirox
Brand Names Exjade®
Why do I need this medicine? Deferasirox is used to remove excess iron in your body. Iron may build up in your body after repeated blood transfusions. Too much iron in your body can damage your vital organs such as the heart and the liver.
How do I take this medicine? Take Deferasirox exactly as directed by your doctor. Do not take more or less than instructed by your doctor. Do not stop this medicine unless your doctor has advised you to do so.
Take Deferasirox on an empty stomach 30 minutes before food. Take it at the same time everyday.
Disperse the tablet in a glass of water orange or apple juice. Stir well. Drink the entire contents immediately. If there is any residue left in the glass add a small amount of water and drink the entire content. If you use juice first disperse the tablets in about 2 tablespoons of water before diluting with juice. DO NOT swallow chew or crush the tablet. Do not disperse the tablet in milk or fizzy drinks.
What should I do if I have forgotten to take this medicine? Take the missed dose as soon as you remember. If it is almost time for your next dose skip the missed dose and return to your normal dosing schedule. DO NOT double a dose under any circumstances.
Remember to take Deferasirox exactly as directed. If you often forget to take your medicine let your doctor and pharmacist know.
When should I not use this medicine? Do not take Deferasirox together with other similar medicines that remove excess iron from your body such as desferroxamine.  Alert your doctor if you suffer from kidney disease.
What should I take note of while taking this medicine? Inform your doctor if you have liver disease low white blood cell counts or other blood disorders. Alert your doctor if you are pregnant or breastfeeding. For as long as you are being treated with Deferasirox you will need to have regular blood tests to check your body's response to the medicine. Your doctor will advise you about how often you need to have blood tests.
What side effects could I experience? Common side effects of Deferasirox include fever headache nausea vomiting diarrhoea stomach pain cough sore throat rash and dizziness. If you feel dizzy do not drive or take part in any activity in which you need to be alert. Alert your doctor quickly if you experience any of these side effects:
    – changes in vision or hearing
    – weight gain of more than 5 pounds (2.3 kg) a week
    – swelling in the legs or arms
    – reduced urine output
    – unusual bleeding or bruising
    – dark urine
    – yellowing of the skin or eyes
    – shortness of breath
    – unusual tiredness
Can I take this with other medicines? Alert your doctor if you are taking any other medicines especially those listed here:
    – antacids that contain aluminium
    – medicines for fits (epilepsy) such as lamotrigine carbamazepine or valproic acid
    – antidepressants
    – lithium (a medicine for treating mental illness)
Always inform your doctor and pharmacist if you are taking any other medicines including herbal tonics supplements and medicines that you buy without a prescription.
Are there any restrictions on the type of food I can take? Avoid alcohol.
How should I store this medicine? Store in a cool dry place away from the reach of children.
Medicines must not be used past the expiry date.
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