Dhamija Mayank, Mahajan Amita, Kalra Manas, Virmani Anju
Department of Pediatric Hematology and Oncology, Rajiv Gandhi Cancer Institute and Research Centre, Rohini, New Delhi, India.
Indian J Med Paediatr Oncol. 2013 Jan;34(1):16-20. doi: 10.4103/0971-5851.113407.
To evaluate the efficacy and safety of the oral iron chelator deferasirox in treating transfusional hemosiderosis in a cohort of Indian children with thalassemia major with high iron load.
The first 50 children (age 2-18 yrs) with thalassemia major to commence deferasirox at our center were enrolled and followed up for a period of 36 months between April 2008 and March 2011. The dose of deferasirox was determined by their baseline serum ferritin and was adjusted to a maximum of 40 mg/kg/day depending on response. Ferritin levels, SGOT, SGPT, serum creatinine and urine albumin were regularly monitored.
Of the 50 patients, 76% documented a significant decline in serum ferritin (P<0.05). Seven (14%) patients had a stable ferritin whilst 5 patients (10%) documented an increase over the study period. The mean serum ferritin at baseline, 12, 24 and 36 months was 4354, 3260, 3290 and 3042, respectively (P<0.05). The median serum ferritin at the same time points was 3555, 2810, 2079 and 2271, respectively (P<0.05). No severe toxicity was seen.
Deferasirox, when given in doses ≥30 mg/kg, was found to be an effective and safe drug in reducing transfusional hemosiderosis. Thirty five (70%) needed dose escalation upto 40 mg/kg/day. Fifteen (30%), however did not achieve a negative iron balance despite maximally permissible doses.
评估口服铁螯合剂地拉罗司治疗印度重型地中海贫血高铁负荷患儿输血性含铁血黄素沉着症的疗效和安全性。
选取2008年4月至2011年3月期间在本中心开始使用地拉罗司的首批50例重型地中海贫血患儿（年龄2 - 18岁），进行为期36个月的随访。地拉罗司的剂量根据其基线血清铁蛋白确定，并根据反应调整至最大40mg/kg/天。定期监测铁蛋白水平、谷草转氨酶、谷丙转氨酶、血清肌酐和尿白蛋白。
50例患者中，76%的患者血清铁蛋白显著下降（P<0.05）。7例（14%）患者铁蛋白稳定，5例（10%）患者在研究期间铁蛋白升高。基线、12个月、24个月和36个月时的平均血清铁蛋白分别为4354、3260、3290和3042（P<0.05）。相同时间点的血清铁蛋白中位数分别为3555、2810、2079和2271（P<0.05）。未观察到严重毒性。
发现剂量≥30mg/kg的地拉罗司是一种有效且安全的药物，可减少输血性含铁血黄素沉着症。35例（70%）患者需要将剂量增至40mg/kg/天。然而，15例（30%）患者尽管使用了最大允许剂量，仍未实现负铁平衡。
