Alymara Vasiliki, Bourantas Dimitrios, Chaidos Aristeidis, Bouranta Paraskevi, Gouva Maria, Vassou Amalia, Tzouvara Evagelia, Bourantas Konstantinos L
Hematology Unit, University of Ioannina Medical School, Ioannina, Greece.
Hematol J. 2004;5(6):475-9. doi: 10.1038/sj.thj.6200550.
The purpose of our study was to evaluate the effectiveness and safety of combined therapy with deferoxamine (DFO) and deferiprone (DFP) in patients with beta-thalassemia major and increased serum ferritin.
Our study was performed in 36 patients with beta-thalassemia major. DFP was administered orally in a total daily dose of 60 mg/kg for 6 days per week and DFO was administered subcutaneously in a total daily dose of 40-50 mg/kg for 4-6 days per week. The efficacy of combined treatment was assessed by measurements of serum ferritin and 24-h urine iron excretion levels.
Out of the 36 patients, 11 discontinued DFO after a mean of 4 months; however, 25 patients, who continued to receive the combined therapy showed a very satisfactory compliance. After a mean of 13.5 months, their mean serum ferritin levels reduced from 2637 + 1292 to 1580 + 1024 ng/ml (P = 0.002) and their mean urinary iron excretion elevated from 0.41 + 0.27 to 0.76 +0.49 mg/24h (P = 0.003). The observed side effects were gastrointestinal disorders,elevations in liver enzymes, mild neutropenia, joint symptoms, taste disorders, dizziness and fatigue.
The results of this study show that combined iron-chelation therapy with DFO and DFP results in satisfactory reduction of serum ferritin with no significant toxicity.
我们研究的目的是评估去铁胺（DFO）和去铁酮（DFP）联合治疗对重型β地中海贫血且血清铁蛋白升高患者的有效性和安全性。
我们对36例重型β地中海贫血患者进行了研究。DFP口服给药，每日总剂量为60mg/kg，每周服用6天；DFO皮下给药，每日总剂量为40 - 50mg/kg，每周给药4 - 6天。通过测量血清铁蛋白和24小时尿铁排泄水平来评估联合治疗的疗效。
36例患者中，11例在平均4个月后停用DFO；然而，25例继续接受联合治疗的患者依从性非常令人满意。平均13.5个月后，他们的平均血清铁蛋白水平从2637 + 1292降至1580 + 1024ng/ml（P = 0.002），平均尿铁排泄量从0.41 + 0.27升高至0.76 + 0.49mg/24小时（P = 0.003）。观察到的副作用有胃肠道疾病、肝酶升高、轻度中性粒细胞减少、关节症状、味觉障碍、头晕和疲劳。
本研究结果表明，DFO和DFP联合铁螯合疗法可使血清铁蛋白得到满意降低，且无明显毒性。
