Department of Pediatrics, Chang Gung Memorial Hospital, Chang Gung University College of Medicine, Taoyuan, Taiwan.
Transfusion. 2011 May;51(5):949-54. doi: 10.1111/j.1537-2995.2010.02939.x. Epub 2010 Nov 15.
Deferasirox (DFRA) is a new approved oral iron chelator. Its advantages are that it is convenient and better tolerated and adhered to due to "once-daily" oral dosage. However, its use in the field is limited and it is yet to be subjected to postmarketing surveillance.
A 18.75-year-old male with β-thalassemia major received oral DFRA therapy due to transfusional iron overload for 27 months. He had received iron chelation therapy with deferoxamine injection together with oral deferiprone. However, his compliance was poor (very high routine serum ferritin level, ranging from 1059 to 6030 ng/mL). After 25 months of DFRA therapy, the serum ferritin level declined from 4097 to 1343 ng/mL. He experienced five hospital admissions including coma, Fanconi syndrome, hepatic dysfunction, and thrombocytopenia after using DFRA as oral iron chelator. After we discontinued DFRA, he recovered fully without hepatic dysfunction, thrombocytopenia, proteinuria, glucosuria, and hypophosphatemia.
Our case illustrates the potential risks of DFRA-induced renal toxicity, hepatic dysfunction, and thrombocytopenia. Meticulous monitoring of kidney, liver, and hematopoietic function is mandatory for patients undergoing treatment with DFRA. Further investigation of the potential risk and adverse effects of long-term DFRA use is necessary.
地拉罗司（DFRA）是一种新批准的口服铁螯合剂。它的优点是由于“每日一次”的口服剂量，使用方便且耐受性和顺应性更好。然而，其在临床上的应用受到限制，尚未进行上市后监测。
一名 18.75 岁男性，患有重型β地中海贫血，因输血引起的铁过载接受了 27 个月的口服 DFRA 治疗。他曾接受过注射用去铁胺联合口服去铁酮的铁螯合治疗，但他的依从性很差（常规血清铁蛋白水平非常高，范围为 1059 至 6030ng/mL）。在接受 DFRA 治疗 25 个月后，血清铁蛋白水平从 4097 降至 1343ng/mL。在使用 DFRA 作为口服铁螯合剂后，他经历了五次住院治疗，包括昏迷、范可尼综合征、肝功能障碍和血小板减少症。停用 DFRA 后，他完全康复，没有肝功能障碍、血小板减少症、蛋白尿、糖尿和低磷血症。
我们的病例说明了 DFRA 引起的肾毒性、肝功能障碍和血小板减少症的潜在风险。接受 DFRA 治疗的患者必须密切监测肾脏、肝脏和造血功能。需要进一步调查长期使用 DFRA 的潜在风险和不良反应。
