Zachariah Mathew, Tony Surekha, Bashir Wafa, Al Rawas Abdulhakim, Wali Yasser, Pathare Anil
Department of Child Health Sultan Qaboos University Hospital, Muscat, Oman.
Pediatr Hematol Oncol. 2013 Mar;30(2):104-12. doi: 10.3109/08880018.2012.762568. Epub 2013 Jan 30.
Iron overload is mainly responsible for the morbidity and mortality in patients with beta thalassemia major (TM). Our aim was to compare treatment outcomes with oral iron chelators, deferiprone (DFP), and deferasirox (DFX) in the first two decades on therapy. Seventy patients with TM (mean age ± SD, 7.9 ± 4.2; range 1.5-17 years) attending the pediatric day care unit for regular transfusional support were enrolled in this cross-sectional cohort study. The patients were treated either with DFP at the dose of 75-100 mg/kg/d in three divided doses after food or DFX at the dose of 25-40 mg/kg/d as single dose before food. Mean serum ferritin (±SD) was lower in patients below 10 years (n = 44) at 1283 (±600) ng/mL when compared with patients ≥10 years (n = 19) at 1546 (±589) ng/mL. There was no significant difference in mean serum ferritin (±SD) level in patients receiving DFP (1360 ± 589) versus DFX (1260 ± 641) in this cohort, P > 0.05. 67% of the patients had Vitamin D deficiency (<50 umol/L). Our results show comparable efficacy of DFP and DFX with regards to iron chelation as estimated by serial serum ferritin levels; however, MRI T2* values were higher in the DFP-treated patients compared to DFX treatment.
铁过载是重型β地中海贫血(TM)患者发病和死亡的主要原因。我们的目的是比较口服铁螯合剂去铁酮(DFP)和地拉罗司(DFX)在前二十年治疗中的疗效。本横断面队列研究纳入了70例因定期输血支持而在儿科日间护理病房就诊的TM患者(平均年龄±标准差,7.9±4.2岁;范围1.5 - 17岁)。患者分别接受以下治疗:DFP,剂量为75 - 100mg/kg/d,分三次餐后服用;或DFX,剂量为25 - 40mg/kg/d,空腹单次服用。10岁以下患者(n = 44)的平均血清铁蛋白(±标准差)为1283(±600)ng/mL,低于10岁及以上患者(n = 19)的1546(±589)ng/mL。在该队列中,接受DFP治疗的患者(1360±589)与接受DFX治疗的患者(1260±641)的平均血清铁蛋白(±标准差)水平无显著差异,P>0.05。67%的患者存在维生素D缺乏(<50μmol/L)。我们的结果表明,根据系列血清铁蛋白水平评估,DFP和DFX在铁螯合方面具有相当的疗效;然而,与DFX治疗相比,DFP治疗患者的MRI T2*值更高。