Desferal (Deferoxamine)
Iron chelating agent for the treatment of chronic iron overload and acute iron poisoning
Desferal (deferoxamine mesilate) is an iron chelating agent used to treat chronic iron overload caused by repeated blood transfusions in conditions such as thalassemia major, sickle cell disease, and myelodysplastic syndromes. It works by binding excess iron in the body and allowing it to be excreted through urine and stool. Desferal is also the first-line treatment for acute iron poisoning. It is administered by injection or infusion and requires a prescription.
Quick Facts
Key Takeaways
- Desferal is the gold standard iron chelator with over 50 years of clinical experience in managing transfusional iron overload.
- It must be administered by slow subcutaneous infusion (typically 8–12 hours overnight using a portable pump) for optimal effectiveness.
- Regular monitoring of hearing and vision is essential, as high-frequency hearing loss and retinal changes can occur, especially at high doses.
- Vitamin C supplementation (max 200 mg/day) may enhance iron excretion but should only be started after the first month of chelation therapy.
- Treatment should begin when serum ferritin exceeds 1000 ng/mL or after approximately 10–20 blood transfusions to prevent organ damage from iron overload.
What Is Desferal and What Is It Used For?
Desferal contains the active substance deferoxamine mesilate, a naturally derived iron-chelating agent originally isolated from the bacterium Streptomyces pilosus. It belongs to a class of medications known as iron chelating agents, which work by binding to excess iron in the bloodstream and tissues, forming a stable complex called ferrioxamine that can then be safely excreted from the body through the urine and feces.
Iron is an essential mineral for many bodily functions, including oxygen transport, energy production, and DNA synthesis. However, the human body has no natural mechanism to actively excrete excess iron. When patients require regular blood transfusions — as is the case in thalassemia major, sickle cell disease, myelodysplastic syndromes, and other chronic anemias — the iron contained in transfused red blood cells accumulates progressively in vital organs. Without chelation therapy, this iron overload leads to serious and potentially fatal damage to the heart, liver, and endocrine organs.
Desferal has been used clinically since the 1960s and remains one of the most extensively studied iron chelators in medicine. It is listed on the World Health Organization (WHO) Model List of Essential Medicines, reflecting its importance in global healthcare. The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have both approved it for the treatment of chronic iron overload and acute iron poisoning.
Primary Indications
Desferal is approved for the following clinical indications:
- Chronic transfusional iron overload: The primary indication, most commonly in patients with thalassemia major who receive regular blood transfusions. Also used in sickle cell disease, myelodysplastic syndromes, Diamond-Blackfan anemia, and other transfusion-dependent anemias.
- Acute iron poisoning: Desferal is the treatment of choice for significant iron ingestion, particularly in children. It is administered intravenously in the emergency setting to rapidly bind and remove absorbed iron.
- Diagnosis of iron overload: The deferoxamine challenge test (also known as the DFO test) can be used to assess the degree of iron overload by measuring urinary iron excretion after a test dose.
- Aluminium overload in dialysis patients: In patients with chronic kidney disease on dialysis, Desferal can be used to treat aluminium accumulation, which can cause bone disease and encephalopathy.
How Desferal Works
Deferoxamine has a remarkably high and specific affinity for trivalent iron (Fe3+). Each molecule of deferoxamine binds one atom of iron in a 1:1 ratio, forming a hexadentate (six-point) complex known as ferrioxamine. This complex is highly stable, water-soluble, and non-toxic. Once formed, ferrioxamine is excreted primarily through the kidneys (producing the characteristic reddish-brown discolouration of urine) and, to a lesser extent, through the bile into the feces.
Importantly, deferoxamine preferentially chelates free or loosely bound iron — known as the labile iron pool — rather than iron that is already incorporated into essential proteins such as hemoglobin, myoglobin, or transferrin. It also chelates iron stored as ferritin and hemosiderin in tissues. This selectivity means that Desferal removes harmful excess iron while preserving the iron needed for normal physiological functions, though dose-related adverse effects can occur if the ratio of drug to available iron is too high.
Iron overload, if left untreated, can cause irreversible organ damage. Cardiac iron overload remains the leading cause of death in patients with thalassemia major who are inadequately chelated. Desferal has been shown to significantly reduce cardiac iron burden and improve survival in these patients when used consistently and at appropriate doses.
What Should You Know Before Taking Desferal?
Desferal is a potent medication that requires careful medical supervision. Before starting treatment, your healthcare provider will conduct a thorough evaluation including blood tests for serum ferritin, liver and kidney function, and baseline assessments of hearing and vision. Understanding the contraindications, warnings, and precautions associated with Desferal is essential for safe and effective use.
Contraindications
Desferal should not be used in the following situations:
- Hypersensitivity: Known allergy to deferoxamine mesilate or any of the excipients in the formulation. Anaphylactic reactions, though rare, have been reported.
- Severe renal impairment: Since ferrioxamine is primarily excreted through the kidneys, severe renal insufficiency (particularly anuria) is a contraindication because the chelated iron complex cannot be adequately eliminated.
In patients with moderate renal impairment, Desferal may still be used but requires careful dose adjustment and close monitoring of kidney function. Dialysis can be used to remove ferrioxamine in patients with renal failure if chelation therapy is urgently needed.
Warnings and Precautions
Several important warnings and precautions should be considered during Desferal therapy:
- Auditory toxicity: High-frequency sensorineural hearing loss and tinnitus have been reported, particularly at higher doses or when the therapeutic index (ratio of Desferal dose to serum ferritin) is unfavorable. Audiometric testing should be performed before treatment and at regular intervals (every 3–6 months). If hearing changes are detected, the dose should be reduced or treatment temporarily stopped.
- Ocular toxicity: Visual disturbances including decreased visual acuity, blurred vision, impaired colour vision, night blindness, visual field defects, and retinal pigmentary changes (retinopathy) have been reported. Ophthalmological examinations including slit-lamp examination and fundoscopy should be performed at baseline and regularly during treatment.
- Growth retardation: In children, particularly those starting chelation therapy before age 3, high doses of Desferal relative to body weight and low serum ferritin levels have been associated with growth retardation and skeletal abnormalities including metaphyseal changes. Body height and weight should be monitored every 3 months in paediatric patients.
- Infection susceptibility: Desferal may increase susceptibility to certain infections, particularly with Yersinia enterocolitica and Yersinia pseudotuberculosis. This is because these bacteria require iron for growth, and the ferrioxamine complex can act as a siderophore (iron carrier) for these organisms. If a patient develops unexplained fever, abdominal pain, or diarrhoea, Desferal should be temporarily discontinued until the infection is excluded or treated. Mucormycosis (a serious fungal infection) has also been reported in Desferal-treated patients, particularly in those who are dialysis-dependent or have diabetic ketoacidosis.
- Respiratory distress: High intravenous doses of Desferal (particularly above 15 mg/kg/hour) have been associated with acute respiratory distress syndrome (ARDS). The recommended maximum infusion rate should not be exceeded.
- Neurological complications: Rare cases of neurological disturbances including dizziness, neuropathy, and paraesthesia have been reported. Worsening of aluminium-related encephalopathy has been observed in dialysis patients treated with Desferal.
The Desferal dose should be adjusted to maintain the therapeutic index (TI): the mean daily dose of Desferal (mg/kg) divided by the serum ferritin (μg/L) should generally remain below 0.025. Exceeding this ratio, especially when ferritin levels are low, significantly increases the risk of auditory and visual toxicity. Regular monitoring of serum ferritin is essential to guide dose adjustments.
Pregnancy and Breastfeeding
Desferal should generally be avoided during pregnancy, especially during the first trimester. Animal studies have demonstrated embryotoxic and teratogenic effects at high doses, including skeletal malformations. However, there are limited data on the use of deferoxamine in pregnant women. In clinical practice, the decision to use Desferal during pregnancy must weigh the potential risks to the fetus against the risks of untreated iron overload, which can be life-threatening for the mother, particularly if cardiac iron overload is present.
If chelation therapy is deemed necessary during pregnancy, treatment should be conducted under close specialist supervision with careful monitoring of both mother and fetus. Some expert guidelines suggest that deferoxamine may be considered in the second and third trimesters when the benefits clearly outweigh the risks.
It is not known whether deferoxamine or its metabolites are excreted in human breast milk. Because of the potential for adverse effects in the nursing infant, breastfeeding is not recommended during treatment with Desferal. Women should discuss alternative feeding options with their healthcare provider.
How Does Desferal Interact with Other Drugs?
Although deferoxamine has relatively few direct pharmacokinetic drug interactions, several clinically important interactions must be considered. The concurrent use of certain medications can alter the safety or efficacy of Desferal therapy, and healthcare providers should review all medications before initiating treatment.
Major Interactions
| Drug | Interaction | Clinical Recommendation |
|---|---|---|
| Vitamin C (Ascorbic acid) | High-dose vitamin C increases iron absorption and mobilises tissue iron, potentially worsening iron-mediated cardiac toxicity. Vitamin C can impair cardiac function in iron-overloaded patients. | Limit to max 200 mg/day. Start only after 1 month of Desferal therapy. Avoid in patients with cardiac dysfunction. Monitor cardiac function. |
| Prochlorperazine | Concurrent use has been associated with temporary impairment of consciousness and prolonged coma in rare cases. | Avoid concurrent use. Use alternative antiemetics if needed. |
| Other iron chelators (deferasirox, deferiprone) | Combined chelation therapy may enhance iron removal but increases risk of over-chelation and toxicity. Safety of combinations not fully established in all populations. | Combination therapy should only be used under specialist supervision with close monitoring of serum ferritin, renal function, and blood counts. |
Minor Interactions
| Drug / Agent | Interaction | Clinical Recommendation |
|---|---|---|
| Gallium-67 (imaging agent) | Deferoxamine binds gallium-67, potentially causing falsely decreased uptake on scintigraphy and inaccurate imaging results. | Discontinue Desferal 48 hours before gallium-67 imaging. |
| Erythropoiesis-stimulating agents (EPO) | Theoretically, Desferal could reduce iron availability for erythropoiesis, potentially blunting the response to EPO. | Monitor haemoglobin response and iron parameters. Adjust chelation schedule if needed. |
| Aluminium-containing antacids | Deferoxamine also chelates aluminium. In dialysis patients, this can cause a transient increase in serum aluminium before excretion. | Monitor serum aluminium levels in dialysis patients. Consider timing of administration. |
Patients should inform their healthcare provider about all medications they are taking, including over-the-counter supplements and herbal remedies. In particular, iron-containing supplements or multivitamins should not be taken concurrently with chelation therapy, as this would be counterproductive to the treatment goal.
What Is the Correct Dosage of Desferal?
Desferal is supplied as a powder for reconstitution and must be dissolved in sterile water for injection before use. The reconstituted solution can then be administered subcutaneously, intramuscularly, or intravenously depending on the clinical situation. The dosage is highly individualised and should be guided by serum ferritin levels, liver iron concentration, and the therapeutic index.
Adults
Chronic Iron Overload — Subcutaneous Infusion (Preferred)
Dose: 20–60 mg/kg/day
Administration: Slow subcutaneous infusion over 8–12 hours using a portable infusion pump, typically given overnight 5–7 nights per week.
Reconstitution: Dissolve 500 mg vial in 5 mL sterile water for injection. The resulting solution (95 mg/mL) can be further diluted as needed.
The average daily dose is typically 20–40 mg/kg. Doses should not normally exceed 50 mg/kg except in patients with very high iron burdens where the benefit outweighs the risk. The therapeutic index (mean daily dose in mg/kg divided by serum ferritin in μg/L) should generally remain below 0.025.
Chronic Iron Overload — Intramuscular Injection
Dose: 0.5–1 g/day (500–1000 mg)
Administration: Deep intramuscular injection. Less effective than subcutaneous infusion and generally reserved for patients who cannot tolerate subcutaneous infusion.
Chronic Iron Overload — Intravenous Infusion
Dose: Up to 2 g per transfusion unit
Administration: Slow intravenous infusion administered alongside blood transfusion (in a separate line). The infusion rate should not exceed 15 mg/kg/hour. This route can provide additional chelation on transfusion days.
Acute Iron Poisoning
Dose: 15 mg/kg/hour by continuous intravenous infusion
Maximum dose: 80 mg/kg in 24 hours
Administration: Continuous IV infusion until the patient is clinically stable and serum iron levels have normalised. The characteristic "vin rosé" (pinkish-red) discolouration of urine indicates that iron is being chelated and excreted.
Children
Chelation therapy in children should be initiated with caution. Desferal treatment is generally started once the child has received approximately 10–20 blood transfusions or when serum ferritin levels exceed 1000 ng/mL. Special considerations for paediatric dosing include:
Children over 3 Years
Dose: 20–40 mg/kg/day
Administration: Slow subcutaneous infusion over 8–12 hours, 5–7 nights per week. The dose should be carefully adjusted to maintain the therapeutic index below 0.025.
Important: Growth and body height should be monitored every 3 months. High doses relative to body weight (above 40 mg/kg/day) should be avoided as they increase the risk of growth retardation and skeletal changes.
Children under 3 Years
Recommendation: Desferal should generally not be started in children under 3 years of age due to the significant risk of growth retardation. If chelation is urgently required, doses should be kept as low as possible (not exceeding 20 mg/kg/day) with close monitoring of growth parameters.
Elderly
No specific dose adjustments are recommended for elderly patients based on age alone. However, elderly patients are more likely to have reduced renal function, and the dose should be adjusted accordingly based on creatinine clearance. Regular monitoring of renal function, hearing, and vision is particularly important in this population, as age-related changes may increase susceptibility to adverse effects.
Missed Dose
If you miss a dose of Desferal, administer it as soon as you remember. If it is close to the time of your next scheduled dose, skip the missed dose and return to your regular dosing schedule. Do not double the dose to make up for a missed one. Consistent adherence to the chelation regimen is crucial for maintaining effective iron removal and preventing organ damage. Studies have shown that poor compliance with Desferal infusions is one of the strongest predictors of morbidity and mortality in thalassemia patients.
Overdose
Acute overdose of deferoxamine may cause hypotension, tachycardia, and gastrointestinal disturbances. In severe cases, acute visual loss (including blindness), acute renal failure, hepatic failure, and ARDS have been reported. Treatment is primarily supportive, as there is no specific antidote. Haemodialysis or peritoneal dialysis may help remove ferrioxamine in patients with renal impairment. If overdose is suspected, Desferal should be discontinued immediately and the patient should receive appropriate supportive care.
Intravenous infusion rates exceeding 15 mg/kg/hour have been associated with hypotension, urticaria, and respiratory distress. In acute iron poisoning, the total dose should not exceed 80 mg/kg in any 24-hour period. Emergency medical services should be contacted immediately in case of overdose.
What Are the Side Effects of Desferal?
Like all medicines, Desferal can cause side effects, although not everybody gets them. The frequency and severity of side effects depend on the dose, duration of treatment, and individual patient factors. Side effects are more likely to occur when the dose is high relative to the degree of iron overload (i.e., when the therapeutic index is unfavourable). Below is a comprehensive overview of side effects organised by frequency.
Very Common
Affects more than 1 in 10 patients
- Injection site reactions: pain, swelling, induration, redness, itching, and eschar/crust formation at the infusion site
- Arthralgia (joint pain) and myalgia (muscle pain)
- Nausea
- Headache
- Urticaria (hives)
- Reddish-brown discolouration of urine (harmless, indicates iron excretion)
Common
Affects 1 in 10 to 1 in 100 patients
- Fever (pyrexia)
- Abdominal pain
- Vomiting
- Diarrhoea
- Growth retardation and bone changes (in children, especially under age 3)
- Asthma-like symptoms and bronchospasm
Uncommon
Affects 1 in 100 to 1 in 1,000 patients
- High-frequency sensorineural hearing loss and tinnitus
- Visual disturbances: decreased visual acuity, blurred vision, loss of colour vision, night blindness, visual field defects, cataracts, optic neuritis
- Retinopathy (retinal pigmentary degeneration)
- Peripheral sensory neuropathy (numbness, tingling)
- Dizziness
- Hypotension (especially with rapid IV infusion)
Rare
Affects fewer than 1 in 1,000 patients
- Anaphylactic/anaphylactoid reactions
- Acute respiratory distress syndrome (ARDS) — particularly with high IV doses
- Yersinia infections (enterocolitis, septicaemia)
- Mucormycosis (opportunistic fungal infection)
- Renal impairment, including acute renal failure
- Hepatic dysfunction
- Seizures (very rare)
- Leg cramps
- Blood disorders: thrombocytopenia (low platelets), leucopenia (low white blood cells)
The auditory and ocular side effects deserve special attention because they can be irreversible if not detected early. The risk is dose-dependent and is greatest when the therapeutic index exceeds 0.025. These side effects are generally reversible if detected promptly and the dose is reduced or treatment is temporarily discontinued. However, delayed recognition can lead to permanent damage.
Injection site reactions are the most commonly reported side effects and can be minimised by rotating injection sites, using appropriate needle length, and ensuring proper infusion technique. Application of topical corticosteroid cream or addition of small amounts of hydrocortisone to the infusion solution may help reduce local reactions.
Contact your healthcare provider immediately if you experience any changes in vision or hearing, unexplained fever or abdominal pain (which may indicate infection), signs of an allergic reaction (difficulty breathing, facial swelling), or significant skin reactions at injection sites. Do not stop treatment without medical advice, as untreated iron overload can be dangerous.
How Should You Store Desferal?
Proper storage of Desferal is essential to maintain its stability and effectiveness. The medication should be stored according to the following guidelines to ensure that it remains safe for use.
- Unopened vials: Store below 25°C (77°F). Do not freeze. Keep the vials in the original carton to protect from light.
- Reconstituted solution: After dissolving the powder in sterile water for injection, the resulting clear to slightly yellowish solution should ideally be used immediately. If not used immediately, it may be stored at 2–8°C (refrigerator) for up to 24 hours. At room temperature (below 25°C), the reconstituted solution should be used within 3 hours.
- Visual inspection: Before use, visually inspect the reconstituted solution for particulate matter and discolouration. The solution should be clear and free of visible particles. Do not use if the solution appears cloudy, discoloured, or contains particles.
- Shelf life: Check the expiry date on the packaging. Do not use Desferal after the expiry date stated on the label.
- Disposal: Any unused reconstituted solution should be discarded. Do not save reconstituted solution for later use. Dispose of unused medication and needles according to local guidelines for pharmaceutical waste.
Keep Desferal out of the reach and sight of children. The infusion pump and supplies should also be stored in a clean, dry location. Patients who self-administer at home should be trained in proper reconstitution technique, storage procedures, and safe disposal of sharps and pharmaceutical waste.
What Does Desferal Contain?
Desferal has a straightforward formulation designed for parenteral (injectable) use. Understanding the composition is important for patients with known sensitivities or allergies.
Active Ingredient
Each vial contains 500 mg deferoxamine mesilate (also written as desferrioxamine mesylate or deferoxamine mesylate). Deferoxamine mesilate is the mesylate salt form of deferoxamine, which improves the stability and solubility of the compound. The molecular formula is C25H48N6O8·CH4O3S, with a molecular weight of approximately 656.79 g/mol.
Excipients
Desferal vials typically contain no additional excipients. The vial contains only the lyophilised (freeze-dried) deferoxamine mesilate powder. For reconstitution, sterile water for injection is used as the solvent (not included in the vial). This minimalist formulation reduces the risk of excipient-related adverse reactions.
Physical Appearance
Desferal is supplied as a white to off-white, sterile, lyophilised powder in glass vials sealed with a rubber stopper and aluminium cap. After reconstitution with sterile water for injection, it forms a clear, colourless to slightly yellowish solution suitable for subcutaneous, intramuscular, or intravenous administration.
The reconstituted solution has a pH of approximately 3.5–5.5. When mixed with normal saline (0.9% sodium chloride) or glucose solutions for intravenous infusion, compatibility should be verified according to the product labelling, as turbidity or precipitation may occur with certain diluents.
Frequently Asked Questions About Desferal
Desferal (deferoxamine) is used to treat chronic iron overload caused by repeated blood transfusions, most commonly in patients with thalassemia major, sickle cell disease, and myelodysplastic syndromes. It is also used for acute iron poisoning, particularly in children, and to diagnose iron or aluminium overload. Deferoxamine works by binding excess iron in the body, forming a complex that is safely excreted through the kidneys and bile.
Desferal is supplied as a powder that must be dissolved in sterile water before use. It is most commonly administered as a slow subcutaneous infusion over 8–12 hours using a portable infusion pump, typically overnight. It can also be given intramuscularly or intravenously in hospital settings. The subcutaneous route is preferred for chronic therapy because it provides sustained drug levels and maximises iron excretion.
The most common side effects of Desferal include injection site reactions such as pain, swelling, and redness. Other frequent side effects include nausea, headache, joint and muscle pain, and fever. Less common but important side effects include visual disturbances (blurred vision, decreased visual acuity), hearing impairment (high-frequency hearing loss), and growth retardation in children treated before age 3. Regular monitoring of vision and hearing is recommended during treatment.
Desferal should generally be avoided during pregnancy, especially in the first trimester, as animal studies have shown potential for skeletal abnormalities. However, the decision must weigh the risk of untreated iron overload against potential fetal harm. If treatment is necessary during pregnancy, it should be under close specialist supervision. Breastfeeding is not recommended during Desferal treatment as it is unknown whether deferoxamine passes into breast milk.
Desferal treatment for chronic iron overload is typically long-term and may continue for years or even a lifetime, as long as the patient continues to receive blood transfusions. Treatment usually begins after 10–20 blood transfusions or when serum ferritin levels exceed 1000 ng/mL. The duration and intensity of treatment are guided by regular monitoring of serum ferritin and liver iron concentration. Consistent adherence to the infusion regimen is critical for preventing iron-related organ damage.
Desferal (deferoxamine) must be given by injection or infusion, while newer iron chelators such as deferasirox (Exjade/Jadenu) and deferiprone (Ferriprox) can be taken orally. Desferal has the longest track record and remains the gold standard for efficacy, particularly in cardiac iron removal. Oral chelators offer better convenience and adherence but may have different side effect profiles. The choice depends on patient factors including age, iron burden, organ involvement, and ability to comply with the infusion regimen.
References
This article is based on the following peer-reviewed sources and clinical guidelines. All medical information has been verified against international standards and represents evidence level 1A where applicable.
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- European Medicines Agency (EMA). Desferal — Summary of Product Characteristics (SmPC). European Medicines Agency; 2024.
- U.S. Food and Drug Administration (FDA). Desferal (deferoxamine mesylate for injection) — Prescribing Information. Novartis Pharmaceuticals Corporation; 2023.
- Cappellini MD, Cohen A, Porter J, Taher A, Viprakasit V (eds). Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT), 4th Edition. Thalassaemia International Federation; 2021.
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- British National Formulary (BNF). Deferoxamine mesilate. National Institute for Health and Care Excellence (NICE); 2025.
- Kontoghiorghes GJ, Kolnagou A, Demetriou T, et al. New era in the treatment of iron overload diseases with chelation protocols and genotyping and phenotyping of iron metabolism genes. International Journal of Molecular Sciences. 2023;24(16):12540.
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