Successful long-term iron chelation with deferasirox depends on structured laboratory and clinical monitoring. The schedule below summarises mainstream recommendations - always follow the locally approved prescribing information and individualise based on patient factors.
Baseline Assessments (Before Initiation) #
- Serum creatinine (twice, on different days), creatinine clearance, urinalysis (proteinuria)
- Liver function tests (ALT, AST, alkaline phosphatase, bilirubin)
- Complete blood count
- Serum ferritin (baseline; combined with transfusion-burden assessment)
- LIC by MRI R2 / T2* (where available)
- Cardiac T2* in patients with prolonged transfusion history or thalassemia >= age 8-10
- Audiology and ophthalmology baseline
- Pregnancy test in women of childbearing potential
- Body weight (for mg/kg dose calculation)
During the First Month (Initiation Phase) #
- Serum creatinine and electrolytes weekly for 4 weeks
- Serum ALT/AST monthly
- Body weight, blood pressure at each clinic visit
- Symptom screen for GI, skin, hypersensitivity reactions
Routine Long-Term Monitoring #
| Parameter | Frequency | Action Threshold |
|---|---|---|
| Serum creatinine | Monthly | Persistent rise > 33% above baseline (or above ULN) - dose reduction or discontinuation per label |
| Liver function tests | Monthly | ALT/AST > 5x ULN - interrupt and investigate |
| Serum ferritin | Monthly | Trend-driven dose adjustment; target individualised to patient |
| Complete blood count | Monthly (more often in MDS / cytopenia risk) | Worsening cytopenia warrants reassessment |
| Urinalysis (proteinuria) | Monthly | New / worsening proteinuria - investigate tubulopathy |
| Body weight | Each visit | Recalculate mg/kg dose |
| LIC (MRI R2/T2*) | Every 6-12 months | Dose titration based on LIC trend |
| Cardiac T2* | Annually (selected patients) | T2* < 20 ms - intensify chelation |
| Audiology | Annually | New hearing loss - review chelation |
| Ophthalmology (lens) | Annually | New cataract - review |
| Endocrine screening (thalassemia) | Annually | Growth, puberty, thyroid, glycaemia |
Triggers for Immediate Review / Discontinuation #
- Severe rash, blisters, mucosal involvement (suspected SJS / TEN / DRESS) - discontinue immediately, do not re-introduce
- Acute renal failure or persistent creatinine rises despite dose reduction
- Hepatic failure or persistent ALT/AST > 5x ULN
- GI hemorrhage
- Severe cytopenia in MDS patients
- Hypersensitivity / anaphylaxis
Dose Titration Principles #
- Increase by 5-10 mg/kg/day every 3-6 months when ferritin / LIC remains above target
- Decrease by 5-10 mg/kg/day when ferritin falls below ~500 ng/mL or LIC < 3 mg Fe/g dw
- Interrupt therapy if serum ferritin < 500 ng/mL on consecutive measurements (per local labelling)