Treatments for iron overload caused by hemochromatosis
| Treatment . | Usual route of treatment . | Advantages . | Principal route/form of iron elimination . | Compliance with treatment . | Disadvantages . | Adverse effects . |
|---|---|---|---|---|---|---|
| Phlebotomy | Venipuncture | Much experience; effective on the part of the clinician, widely available, safe, inexpensive; reversal of cirrhosis in some cases; may improve left ventricular diastolic function | Blood as hemoglobin (1 mL of erythrocytes = 1 mg of Fe) | Excellent for iron depletion; good for maintenance | Requires repeated visits to health-care facility; requires normal erythropoiesis; some patients report intolerance | Transient hypovolemia; fatigue; increases iron absorption; iron deficiency if monitoring inadequate or inappropriate |
| Erythrocytapheresis | Venipuncture | Rapid, safe; may be preferred for patients with severe iron overload | Blood as hemoglobin (1 mL of erythrocytes = 1 mg of Fe) | Excellent in selected patients | Limited clinical experience; requires special apparatus and facility, limited availability; expensive | Transient hypovolemia; fatigue; increases iron absorption; citrate reaction; iron deficiency if monitoring inadequate or inappropriate |
| Deferoxamine (DFO) chelation | Subcutaneous infusion | Much clinical experience in iron overload patients without hemochromatosis; widely available; consider its use in patients intolerant of phlebotomy | Urine as chelate; daily iron excretion variable | Fair | Few reports of use in hemochromatosis, mostly to achieve iron depletion; inadequate chelation of cardiac iron in some cases; expensive | Infusion site reactions; hearing, vision, growth, skeletal abnormalities; zinc deficiency; Yersinia infection |
| Deferasirox (DFX) chelation | Oral | Good chelation of hepatic iron; consider its use in patients with inadequate venous access or intolerant of phlebotomy | Stool as chelate; daily iron excretion variable | Fair | Few reports of use in hemochromatosis to achieve iron depletion; no clear benefit for patients with iron-induced cardiomyopathy; expensive | Toxicity often dose dependent; gastrointestinal symptoms; transaminase elevations; elevation of serum creatinine; rash; rare hearing, vision abnormalities; severe (sometimes fatal) liver, kidney, or marrow toxicity |
| Treatment . | Usual route of treatment . | Advantages . | Principal route/form of iron elimination . | Compliance with treatment . | Disadvantages . | Adverse effects . |
|---|---|---|---|---|---|---|
| Phlebotomy | Venipuncture | Much experience; effective on the part of the clinician, widely available, safe, inexpensive; reversal of cirrhosis in some cases; may improve left ventricular diastolic function | Blood as hemoglobin (1 mL of erythrocytes = 1 mg of Fe) | Excellent for iron depletion; good for maintenance | Requires repeated visits to health-care facility; requires normal erythropoiesis; some patients report intolerance | Transient hypovolemia; fatigue; increases iron absorption; iron deficiency if monitoring inadequate or inappropriate |
| Erythrocytapheresis | Venipuncture | Rapid, safe; may be preferred for patients with severe iron overload | Blood as hemoglobin (1 mL of erythrocytes = 1 mg of Fe) | Excellent in selected patients | Limited clinical experience; requires special apparatus and facility, limited availability; expensive | Transient hypovolemia; fatigue; increases iron absorption; citrate reaction; iron deficiency if monitoring inadequate or inappropriate |
| Deferoxamine (DFO) chelation | Subcutaneous infusion | Much clinical experience in iron overload patients without hemochromatosis; widely available; consider its use in patients intolerant of phlebotomy | Urine as chelate; daily iron excretion variable | Fair | Few reports of use in hemochromatosis, mostly to achieve iron depletion; inadequate chelation of cardiac iron in some cases; expensive | Infusion site reactions; hearing, vision, growth, skeletal abnormalities; zinc deficiency; Yersinia infection |
| Deferasirox (DFX) chelation | Oral | Good chelation of hepatic iron; consider its use in patients with inadequate venous access or intolerant of phlebotomy | Stool as chelate; daily iron excretion variable | Fair | Few reports of use in hemochromatosis to achieve iron depletion; no clear benefit for patients with iron-induced cardiomyopathy; expensive | Toxicity often dose dependent; gastrointestinal symptoms; transaminase elevations; elevation of serum creatinine; rash; rare hearing, vision abnormalities; severe (sometimes fatal) liver, kidney, or marrow toxicity |
It is not feasible to estimate net iron loss or gain attributable to diet or medications in individual patients using routine clinical techniques. Some patients with juvenile-onset hemochromatosis, severe iron overload, and iron-induced cardiomyopathy may benefit from combined treatment with phlebotomy and DFO or DFX.