Combined chelation therapy with daily oral deferiprone and twice-weekly subcutaneous infusion of desferrioxamine in children with β-thalassemia: 3-year experience.
Study Goal
The researchers aimed to evaluate the safety and efficacy of combined oral deferiprone (DFP) and subcutaneous desferrioxamine (DFO) therapy in reducing ferritin levels in transfusion-dependent β-thalassemia children.
Results Summary
The combination therapy significantly reduced median ferritin levels from 2,954.6 to 936.6 ng/ml in responders, with adverse events including elevated alanine aminotransferase (n = 5) and other minor side effects. Nonresponders had higher median age and longer transfusion duration.
Population
Transfusion-dependent β-thalassemia children (ages 3-18 years), including β-thalassemia major and β-thalassemia hemoglobin E patients.
Effective Dosage
50-100 mg/kg/day oral DFP combined with 40 mg/kg/dose s.c. DFO twice weekly.
Duration
Up to 36 months (28 patients completed follow-up).
Interactions
None mentioned
| Intervention | Direction | Endpoint | Population | Dosage | Impact | Claim # |
|---|---|---|---|---|---|---|
combined treatment with oral and subcutaneous iron chelators (50-100 mg/kg/day of oral deferiprone (DFP) combined with 40 mg/kg/dose s.c. desferrioxamine (DFO) twice weekly) | no change | chelation therapy | transfusion-dependent β-thalassemia children | - | is a safe and effective alternative to chelation monotherapy | #1 |
combined treatment with oral and subcutaneous iron chelators (50-100 mg/kg/day of oral deferiprone (DFP) combined with 40 mg/kg/dose s.c. desferrioxamine (DFO) twice weekly) | decrease | median ferritin levels | responders (among transfusion-dependent β-thalassemia children) | from 2,954.6 to 936.6 ng/ml | exhibited a significant fall | #2 |
combined treatment with oral and subcutaneous iron chelators (50-100 mg/kg/day of oral deferiprone (DFP) combined with 40 mg/kg/dose s.c. desferrioxamine (DFO) twice weekly) | decrease | serum ferritin | responders (among transfusion-dependent β-thalassemia children) | 6 months | Time to a significant decrease | #3 |
100 mg/kg/day of DFP | decrease | ferritin | nonresponders (among transfusion-dependent β-thalassemia children) | <15% | ferritin declined <15% | #4 |
combined treatment with oral and subcutaneous iron chelators | increase | adverse events | 13 patients (among transfusion-dependent β-thalassemia children) | 16 episodes | occurred | #5 |
OBJECTIVE: To study the efficacy of combined treatment with oral and subcutaneous iron chelators. MATERIAL AND METHODS: 50-100 mg/kg/day of oral deferiprone (DFP) combined with 40 mg/kg/dose s.c. desferrioxamine (DFO) twice weekly were given to transfusion-dependent β-thalassemia children. RESULTS: Enrolled patients (9 with β-thalassemia major and 33 with β-thalassemia hemoglobin E), ranging from 3 to 18 years in age, were divided into 3 groups; group 1 ferritin ≥1,000-2,500 ng/ml (n = 10), group 2 ferritin >2,500-4,000 ng/ml (n = 23) and group 3 ferritin >4,000 ng/ml (n = 9). Of the 42 patients, 28 reached the 36-month follow-up. Ten patients whose ferritin declined <15% while receiving 100 mg/kg/day of DFP were considered nonresponders. The median age and previous transfusion duration before enrollment were significantly higher in nonresponders than responders (p = 0.04 and 0.003, respectively). The responders exhibited a significant fall in median ferritin levels from 2,954.6 to 936.6 ng/ml (p < 0.001). Time to a significant decrease in serum ferritin among responders was 6 months. In 13 patients, 16 episodes of adverse events occurred: hemophagocytosis with cytopenia (n = 1), neutropenia (n = 2), thrombocytopenia (n = 2), elevated alanine aminotransferase (n = 5), elevated serum creatinine (n = 1), proteinuria (n = 1) and gastrointestinal discomfort (n = 4). CONCLUSION: Combination therapy with daily oral DFP and subcutaneous DFO twice weekly is a safe and effective alternative to chelation monotherapy in β-thalassemia children.