Management of Iron Chelating Therapy in Thalassemia
| Timepoint . | Assessment . | Comment . | Results . | Treatment Recommendations . |
|---|---|---|---|---|
| At start of therapy | Liver biopsy under U/S guidance with quantitative liver iron, histology, PCR for hepatitis C RNA | Should be obtained after approximately 1 yr of regular transfusion | HIC < 3.2 mg/g dry weight | |
| HIC ≥ 3.2 mg/g dry weight | Defer chelation; reassess HIC in 6 mo | |||
| Initiate DFO at 25 mg/kg/night × 5 nights/wk | ||||
| Radiographs of cartilage in wrists, knees, thoracolumbosacral spine; bone age | Should be reviewed by pediatric radiologist and endocrinologist with previous experience in toxicity of DFO | |||
| Standing and sitting heights | ||||
| Serum ferritin, Fe, and TIBC | ||||
| Serum ALT | ||||
| Hepatitis screen | ||||
| WBC ascorbate concentration | ||||
| If WBC ascorbate low, administer vitamin C PO 100 mg/night during DFO infusion | ||||
| Yearly, before age 5 yr | Liver Bx under U/S guidance; assessments as above | HIC < 3.2 mg/g dry weight | Discontinue DFO; reassess HIC in 6 mo | |
| HIC ≥ 3.2 but <7 mg/g dry weight | Continue DFO at 25 mg/kg/night × 5 nights/wk | |||
| HIC ≥ 7 mg/g dry weight | Increase DFO to 35 mg/kg/night × 6-7 nights/wk | |||
| Radiographs as above | Same as above | If severe spinal or metaphyseal changes present, reduce DFO to 25 mg/kg/night × 4 nights/wk even if HIC ≥ 7 mg/g dry weight. Reassess in 6 mo | ||
| Standing and sitting heights | ||||
| Serum ferritin, serum iron, and TIBC | ||||
| Serum ALT | ||||
| Hepatitis screen | ||||
| WBC ascorbate concentration | ||||
| If WBC ascorbate low, administer vitamin C PO 100 mg/night during DFO infusion | ||||
| Q18 mo, from age 5-10 yr | Liver Bx under U/S guidance with quantitative HIC, histology, PCR for hepatitis C RNA | HIC < 3.2 mg/g dry weight | ||
| HIC ≥ 3.2 but < 7 mg/g dry weight | Discontinue DFO; reassess HIC in 6 mo | |||
| Maintain DFO at 40 mg/kg/night × 5 nights/wk | ||||
| HIC ≥ 7 but < 15 mg/g dry weight | Maintain DFO at 40 mg/kg/night × 6-7 nights/wk | |||
| HIC ≥ 15 mg/g dry weight | Maintain DFO at 40-50 mg/kg/night × 7 nights/wk | |||
| Radiographs as above | Same as above | If abnormal, reassess HIC promptly | Titrate DFO as above | |
| Standing and sitting heights | ||||
| Serum ferritin, Fe, and TIBC | ||||
| Serum ALT | ||||
| Hepatitis screen | ||||
| WBC ascorbate concentration | ||||
| If WBC ascorbate low, administer vitamin C PO 100 mg/night during DFO infusion | ||||
| Timepoint | Assessment | Comment | Results | Treatment Recommendations |
| Timepoint . | Assessment . | Comment . | Results . | Treatment Recommendations . |
|---|---|---|---|---|
| At start of therapy | Liver biopsy under U/S guidance with quantitative liver iron, histology, PCR for hepatitis C RNA | Should be obtained after approximately 1 yr of regular transfusion | HIC < 3.2 mg/g dry weight | |
| HIC ≥ 3.2 mg/g dry weight | Defer chelation; reassess HIC in 6 mo | |||
| Initiate DFO at 25 mg/kg/night × 5 nights/wk | ||||
| Radiographs of cartilage in wrists, knees, thoracolumbosacral spine; bone age | Should be reviewed by pediatric radiologist and endocrinologist with previous experience in toxicity of DFO | |||
| Standing and sitting heights | ||||
| Serum ferritin, Fe, and TIBC | ||||
| Serum ALT | ||||
| Hepatitis screen | ||||
| WBC ascorbate concentration | ||||
| If WBC ascorbate low, administer vitamin C PO 100 mg/night during DFO infusion | ||||
| Yearly, before age 5 yr | Liver Bx under U/S guidance; assessments as above | HIC < 3.2 mg/g dry weight | Discontinue DFO; reassess HIC in 6 mo | |
| HIC ≥ 3.2 but <7 mg/g dry weight | Continue DFO at 25 mg/kg/night × 5 nights/wk | |||
| HIC ≥ 7 mg/g dry weight | Increase DFO to 35 mg/kg/night × 6-7 nights/wk | |||
| Radiographs as above | Same as above | If severe spinal or metaphyseal changes present, reduce DFO to 25 mg/kg/night × 4 nights/wk even if HIC ≥ 7 mg/g dry weight. Reassess in 6 mo | ||
| Standing and sitting heights | ||||
| Serum ferritin, serum iron, and TIBC | ||||
| Serum ALT | ||||
| Hepatitis screen | ||||
| WBC ascorbate concentration | ||||
| If WBC ascorbate low, administer vitamin C PO 100 mg/night during DFO infusion | ||||
| Q18 mo, from age 5-10 yr | Liver Bx under U/S guidance with quantitative HIC, histology, PCR for hepatitis C RNA | HIC < 3.2 mg/g dry weight | ||
| HIC ≥ 3.2 but < 7 mg/g dry weight | Discontinue DFO; reassess HIC in 6 mo | |||
| Maintain DFO at 40 mg/kg/night × 5 nights/wk | ||||
| HIC ≥ 7 but < 15 mg/g dry weight | Maintain DFO at 40 mg/kg/night × 6-7 nights/wk | |||
| HIC ≥ 15 mg/g dry weight | Maintain DFO at 40-50 mg/kg/night × 7 nights/wk | |||
| Radiographs as above | Same as above | If abnormal, reassess HIC promptly | Titrate DFO as above | |
| Standing and sitting heights | ||||
| Serum ferritin, Fe, and TIBC | ||||
| Serum ALT | ||||
| Hepatitis screen | ||||
| WBC ascorbate concentration | ||||
| If WBC ascorbate low, administer vitamin C PO 100 mg/night during DFO infusion | ||||
| Timepoint | Assessment | Comment | Results | Treatment Recommendations |
| Q18 mo, after 10 yr | Liver Bx under U/S guidance; assessments as above | HIC < 3.2 mg/g dry weight | Discontinue DFO; reassess HIC in 6 mo | |
| HIC ≥ 3.2 but < 7 mg/g dry weight | Maintain DFO at 40 mg/kg/night × 5 nights/wk | |||
| HIC ≥ 7 but < 15 mg/g dry weight | Maintain DFO at 40 mg/kg/night × 6-7 nights/wk | |||
| HIC ≥ 15 mg/g dry weight | Maintain DFO at 50 mg/kg/night × 7 nights/wk | |||
| Radiographs as above | Same as above | If abnormal, reassess HIC promptly | Titrate DFO as above | |
| Standing and sitting heights | ||||
| Serum ferritin, Fe, and TIBC | ||||
| Serum ALT | ||||
| Hepatitis screen | ||||
| WBC ascorbate concentration | ||||
| If WBC ascorbate low, administer vitamin C PO 100 mg/night during DFO infusion |
| Q18 mo, after 10 yr | Liver Bx under U/S guidance; assessments as above | HIC < 3.2 mg/g dry weight | Discontinue DFO; reassess HIC in 6 mo | |
| HIC ≥ 3.2 but < 7 mg/g dry weight | Maintain DFO at 40 mg/kg/night × 5 nights/wk | |||
| HIC ≥ 7 but < 15 mg/g dry weight | Maintain DFO at 40 mg/kg/night × 6-7 nights/wk | |||
| HIC ≥ 15 mg/g dry weight | Maintain DFO at 50 mg/kg/night × 7 nights/wk | |||
| Radiographs as above | Same as above | If abnormal, reassess HIC promptly | Titrate DFO as above | |
| Standing and sitting heights | ||||
| Serum ferritin, Fe, and TIBC | ||||
| Serum ALT | ||||
| Hepatitis screen | ||||
| WBC ascorbate concentration | ||||
| If WBC ascorbate low, administer vitamin C PO 100 mg/night during DFO infusion |
Abbreviations: PCR, polymerase chain reaction; WBC, white blood cell; PO, orally; Q, every; HIC, hepatic iron concentration; DFO, deferoxamine; U/S, ultrasound; TIBC, total iron binding capacity; ALT, alanine aminotransferase; BX, biopsy.