Chronic iron overload management is a long game. This post distils a practical framework around three principles: quantify the burden, modulate the dose, monitor the outcome.
1. Quantify the Burden #
- Baseline: serum ferritin, LIC (MRI R2/T2*), cardiac T2*, renal and hepatic function, endocrine screen
- Quantify iron-loading rate: transfusion-unit tracking
- Define individual patient target (typically ferritin 500-1000 ng/mL, LIC < 7 mg Fe/g dw)
2. Modulate the Dose #
- Start at 20 mg/kg/day (dispersible deferasirox; adjust for formulation)
- Titrate by 5-10 mg/kg/day every 3-6 months based on ferritin trend
- Reduce or interrupt when below target thresholds
- Balance iron-removal against tolerability (renal, hepatic, GI)
3. Monitor the Outcome #
| Parameter | Cadence |
|---|---|
| Serum ferritin | Monthly |
| Renal function | Weekly x 4, then monthly |
| Liver function | Monthly |
| LIC MRI | 6-12 monthly |
| Cardiac T2* | Annually |
| Audiology / ophthalmology / endocrine | Annually |
4. Multidisciplinary Follow-Up #
Haematology, cardiology, endocrinology, radiology (MRI specialist), pharmacy and psychosocial support - ideally coordinated through a thalassemia / hematology centre.
5. Adherence #
Adherence is the single most important predictor of long-term outcome - emphasise once-daily routine, dispersible-tablet preparation method, empty-stomach administration, and patient-led symptom reporting.
DEFRATAJ - five strengths for fine-grained dosing. See monitoring guidelines.