Abstract
Background: Iron chelation with continuous subcutaneous deferoxamine injections is painful and expensive, in particular due to the cost of infusion pumps. Oral chelators are not widely available in Algeria. This prospective study was designed to evaluate the safety and efficacy of bolus subcutaneous deferoxamine (DFO) and phlebotomy in patients with beta-thalassemia major (TM) or intermedia (TI), or sickle cell disease (SCD), treated with hydroxyurea.
Methods: 31 patients were enrolled (22 TM, 5 TI, 4 SCD). Mean hydroxyurea dosage was 17 mg/kg/d [15–24]. Phlebotomy was performed when hemoglobin was >8 g/dL and ferritin >1000 ng/ml; 13 patients met these criteria (5 TM, 4 TI, 4 SCD). One TI patient was not compliant; the 12 remaining patients underwent phlebotomy removing 3 to 7 ml/kg, every 2 to 4 weeks as tolerated. Three mild hypotension episodes were noted. Bolus subcutaneous deferoxamine was given to the 25 patients with adequate health insurance. Mean deferoxamine dosage was 30±6 mg/kg/d, in two daily boluses. Skin nodules developed in 6 patients and a subcutaneous abscess in 1 patient. Blood transfusions were given when hemoglobin was <6g/dL.
Results:
. | phlebotomy only . | DFO only . | Phlebotomy+DFO . |
---|---|---|---|
N patients | 3 TM, 3 TI | 17 TM, 2 TI | 2 TM, 4 SCD |
Median age (yrs) | 16.5 [7–50] | 15 [6–21] | 20.5 [15–24] |
Median F.U. (mos) | 27 [12–38] | 20 [12–28] | 18 [13–23] |
Median N erythrocyte concentrates transfused during F.U. | 0 [0–1] | 6 [0–19] | 0 |
Median baseline ferritin (ng/ml) | 2800 | 4130 | 3760 |
Median last ferritin (ng/ml) | 884 | 1510 | 1665 |
. | phlebotomy only . | DFO only . | Phlebotomy+DFO . |
---|---|---|---|
N patients | 3 TM, 3 TI | 17 TM, 2 TI | 2 TM, 4 SCD |
Median age (yrs) | 16.5 [7–50] | 15 [6–21] | 20.5 [15–24] |
Median F.U. (mos) | 27 [12–38] | 20 [12–28] | 18 [13–23] |
Median N erythrocyte concentrates transfused during F.U. | 0 [0–1] | 6 [0–19] | 0 |
Median baseline ferritin (ng/ml) | 2800 | 4130 | 3760 |
Median last ferritin (ng/ml) | 884 | 1510 | 1665 |
Conclusions: Combination of phlebotomy and bolus subcutaneous deferoxamine very effectively reduced iron overload in patients with TM, TI and SCD. Phlebotomies could be performed in 12 patients thanks to hydroxyurea treatment which had increased their Hb level above 8 g/dL. Such combined therapy can be proposed in countries which can not afford infusion pumps or oral chelators.
Author notes
Disclosure: No relevant conflicts of interest to declare.