Deferasirox

Iron Overload

Drug Status

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Government Approvals

US(FDA), UK(BNF)

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WHO Essential Medicine

YES

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Known Teratogen

NO

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Pharmaceutical Class

Iron Chelator

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Controlled Drug Substance

NO

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Drug Ingredient

Deferasirox

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Summary

  • Deferasirox is used to treat chronic iron overload in patients requiring frequent blood transfusions, such as those with thalassemia, sickle cell disease, or myelodysplastic syndromes. It's also prescribed for non-transfusion-dependent thalassemia patients aged 10 years and above to reduce iron buildup.

  • Deferasirox binds to excess iron in your blood, forming a compound that is excreted in feces. This prevents iron buildup in major organs like the liver, heart, and pancreas, reducing the risk of iron-related complications. It helps protect your body from long-term damage.

  • For adults, the usual starting dose of Deferasirox is 20 mg per kilogram of body weight per day. It's taken once a day on an empty stomach. Don't chew the tablet; mix it with water, orange juice, or apple juice instead. Children under 6 years old and people with kidney or liver problems may need a lower dose.

  • Common side effects of Deferasirox include alterations in appetite, mental health effects such as anxiety or mood swings, insomnia, headaches, gastrointestinal side effects like nausea, vomiting, and diarrhea, weight gain, decrease in libido, dizziness, difficulties with concentration and thinking, and fatigue.

  • Deferasirox should not be taken if you're breastfeeding, pregnant, or allergic to it. It can interact negatively with some medicines, vitamins, and supplements. Serious side effects can include allergic reactions, bone marrow problems, severe skin reactions, hearing and vision problems. If you experience any of these, seek immediate medical attention.

Indications and Purpose

How does Deferasirox work?

Deferasirox binds to excess iron in the blood, forming a compound that is excreted in feces. This prevents iron buildup in major organs, particularly the liver, heart, and pancreas, reducing the risk of iron-related complications. By lowering serum ferritin and liver iron concentration, it helps protect the body from long-term damage. Continuous use is necessary to maintain safe iron levels

How does one know if Deferasirox is working?

Deferasirox's effectiveness is tracked by checking blood ferritin levels and how well the patient is doing. The dose is changed based on kidney function (eGFR). eGFR is a measure of how well your kidneys filter blood. If your eGFR is below 40 mL/min/1.73 m² (meaning your kidneys aren't working very well), treatment stops. If it's between 40 and 60 mL/min/1.73 m², the starting dose is halved. If your eGFR drops by more than a third from your starting level, the daily dose is lowered by either 3.5mg/kg or 5mg/kg of your body weight, and your kidney function is checked again within a week. (mg/kg means milligrams per kilogram of body weight).

Is Deferasirox effective?

Deferasirox starts removing iron immediately, but visible effects may take weeks to months. Most patients show a decrease in iron levels after 3 to 6 months of treatment. Regular blood tests help track progress and confirm the medication’s effectiveness. Symptoms related to iron overload, such as fatigue and skin discoloration, may improve over time

What is Deferasirox?

Deferasirox tablets are used to lower high iron levels in the blood. You take it once a day on an empty stomach. Don't chew the tablet; mix it with water, orange juice, or apple juice instead. The main ingredient is deferasirox. Other ingredients, which don't have a medical effect, include crospovidone (a substance that helps the tablet break down) and sodium starch glycolate (another tablet-helper). Important: Serious allergic reactions can happen during the first month of taking this medicine. See a doctor immediately if you experience any unusual symptoms like rash, swelling, or difficulty breathing.

What is Deferasirox used for?

Deferasirox is used to treat chronic iron overload in patients who require frequent blood transfusions, such as those with thalassemia, sickle cell disease, or myelodysplastic syndromes. It is also prescribed for non–transfusion-dependent thalassemia (NTDT) patients aged 10+ years to reduce iron buildup. Excess iron can cause severe organ damage, especially to the heart, liver, and pancreas. This medication helps prevent these complications

Directions for Use

For how long do I take Deferasirox?

Deferasirox is taken long-term until iron levels return to a safe range, as determined by blood tests. The duration depends on the ferritin levels and liver iron concentration (LIC) in the body. In NTDT patients, therapy may be stopped once LIC drops below 3 mg Fe/g dry weight. Regular monitoring is essential to determine when to continue, adjust, or stop treatment

How do I take Deferasirox?

Deferasirox can be taken on an empty stomach or with a small meal like a turkey sandwich or an English muffin with jelly and milk. If you miss a dose, take it later that day, at least two hours after a meal and half an hour before your next meal, unless it's almost time for your next dose or you can't take it on an empty stomach. There are no other special diet rules mentioned for taking deferasirox.

How long does it take for Deferasirox to start working?

Deferasirox starts removing iron immediately, but visible effects may take weeks to months. Most patients show a decrease in iron levels after 3 to 6 months of treatment. Regular blood tests help track progress and confirm the medication’s effectiveness. Symptoms related to iron overload, such as fatigue and skin discoloration, may improve over time

How should I store Deferasirox?

Deferasirox tablets should be stored at room temperature (ideally 77°F or 25°C, but acceptable between 59°F/15°C and 86°F/30°C). Keep them in a dry place away from moisture, in the original tightly sealed container. This protects the medication from damage and keeps it safe for use. Children should not have access to the medication. When you have leftover deferasirox, don't flush it down the toilet. Instead, take it to a medicine take-back program for safe disposal. This prevents environmental contamination and ensures proper waste management.

What is the usual dose of Deferasirox?

Deferasirox is a medication used to treat iron overload. For adults, the usual starting dose is 20 mg per kilogram of body weight, per day (mg/kg/day). A kilogram is about 2.2 pounds. Children under 6 years old need a lower dose because their bodies don't process the medicine as well as adults'. They get about half the effect of an adult dose. People with kidney or liver problems may need a lower dose, determined by their doctor based on their individual health and iron levels. It's crucial to use the lowest effective dose to prevent serious side effects. The doctor will carefully monitor the patient.

Warnings and Precautions

Can Deferasirox be taken safely while breastfeeding?

This medication, deferasirox, might pass into breast milk. We don't know for sure how much or what effects it might have on a baby. Because it could seriously harm a nursing infant, you and your doctor need to decide: continue breastfeeding or take deferasirox. You shouldn't do both. If you choose breastfeeding, you'll stop taking deferasirox. Your doctor can help you make the best decision for you and your baby. 

Can Deferasirox be taken safely while pregnant?

Deferasirox is not recommended during pregnancy, as it may harm the fetus. Animal studies show evidence of birth defects and fetal toxicity, and human data is limited. Women taking this medication should use non-hormonal contraception to prevent pregnancy. If pregnancy occurs, the risks should be discussed with a doctor immediately

Can I take Deferasirox with other prescription drugs?

Deferasirox shouldn't be taken with aluminum-containing antacids (antacids that neutralize stomach acid). It can also weaken the effects of some other medicines. These include drugs broken down by the body's CYP3A4 enzyme (like cyclosporine, simvastatin, and birth control pills), drugs that boost UGT enzymes (which help the body process medications – like rifampicin, phenytoin, phenobarbital, and ritonavir), and bile acid sequestrants (medicines that bind to bile acids in the gut, like cholestyramine, colesevelam, and colestipol). If you take deferasirox with UGT boosters or bile acid sequestrants, your doctor might need to increase your deferasirox dose. This is because these other drugs can reduce the amount of deferasirox your body absorbs, making it less effective. Your doctor will check your ferritin levels (a blood test measuring iron stores) and how you're responding to treatment to decide on the right dose.

Can I take Deferasirox with vitamins or supplements?

Deferasirox can interact with some vitamins and supplements. Avoid taking it with aluminum-containing antacids (medicines that reduce stomach acid) – wait at least two hours between taking them. Melatonin (a sleep aid) and caffeine (a stimulant) may also interact negatively with deferasirox. It's very important to keep a complete list of all your medicines, vitamins, and supplements and share it with your doctor or pharmacist. This helps them to avoid harmful interactions.

Is Deferasirox safe for the elderly?

Elderly patients have a higher risk of kidney and liver complications, so deferasirox should be used cautiously. Frequent monitoring is necessary to detect any signs of organ toxicity or bleeding issues. Dose adjustments may be required based on kidney function and overall health status. Doctors will assess individual risks before prescribing the medication

Is it safe to drink alcohol while taking Deferasirox?

Drinking alcohol while taking deferasirox is not recommended as it may increase the risk of liver damage and other side effects. It's best to limit or avoid alcohol and discuss any concerns with your doctor.

Is it safe to exercise while taking Deferasirox?

Exercising while on deferasirox is generally safe unless you experience severe fatigue or other side effects that limit physical activity. Always consult your healthcare provider before starting any new exercise regimen while on this medication.

Who should avoid taking Deferasirox?

Patients with severe kidney or liver disease should not take deferasirox, as it may worsen their condition. Those with low platelet counts (<50,000/mm³) or active gastric ulcers or bleeding disorders should use it with caution. Individuals allergic to deferasirox or its components should avoid it. Doctors will evaluate potential risks before prescribing the medication