Jaiswal Shikha, Hishikar Rajesh, Khandwal Onkar, Agarwal Manju, Joshi Usha, Halwai Ajay, Maheshwari Basant, Sheohare Raka
Assistant Professor, Department of Pharmacology, PT JNM Medical College , Raipur, Chhattisgarh, India .
Professor and Head, Department of Pharmacology, PT JNM Medical college , Raipur, Chhattisgarh, India .
J Clin Diagn Res. 2017 Feb;11(2):FC01-FC03. doi: 10.7860/JCDR/2017/22650.9395. Epub 2017 Feb 1.
Thalassaemia Major patients require frequent blood transfusion leading to iron overload. Excessive iron gets deposited in vital organs and leads to dysfunction of the heart, liver, anterior pituitary, pancreas, and joints. Our body has limited mechanism to excrete iron, so patients with iron overload and its complications need safe and effective iron chelation therapy.
To assess the efficacy of Deferasirox (DFX) as an iron chelator, with specific reference to reduction in serum ferritin level.
This is a prospective; observational study done in 45 multitransfused Thalassaemia Major Children receiving DFX therapy at registered Thalassaemia society Raipur Chhattisgarh. DFX was given in an initial dose of 20 mg/kg/day and according to response increased to a maximum of 40 mg/kg/day. Serum ferritin level was estimated at time of registration and at every three monthly intervals (four times during study period). The primary end point of the study was change in serum ferritin level after 12 months of DFX therapy.
The mean serum ferritin before DFX therapy of all cases was 3727.02 ng/mL. After 12 months of mean dose of 38 mg/kg/day of DFX, the mean decline in serum ferritin was 1207.11 ng/mL (drop by 32.38%, p-value <0.001).
DFX monotherapy has a good safety profile and effectively chelates total body iron in Thalassaemia major patients.
重型地中海贫血患者需要频繁输血，这会导致铁过载。过量的铁沉积在重要器官中，导致心脏、肝脏、垂体前叶、胰腺和关节功能障碍。我们的身体排泄铁的机制有限，因此铁过载及其并发症患者需要安全有效的铁螯合疗法。
评估地拉罗司（DFX）作为铁螯合剂的疗效，特别提及血清铁蛋白水平的降低情况。
这是一项前瞻性观察性研究，在印度恰蒂斯加尔邦赖布尔注册的地中海贫血协会接受DFX治疗的45例多次输血的重型地中海贫血儿童中进行。DFX的初始剂量为20mg/kg/天，并根据反应增加至最大40mg/kg/天。在登记时以及每三个月（研究期间共四次）测定血清铁蛋白水平。该研究的主要终点是DFX治疗12个月后血清铁蛋白水平的变化。
所有病例在DFX治疗前的平均血清铁蛋白为3727.02ng/mL。在平均剂量为38mg/kg/天的DFX治疗12个月后，血清铁蛋白的平均下降量为1207.11ng/mL（下降32.38%，p值<0.001）。
DFX单一疗法具有良好的安全性，可有效螯合重型地中海贫血患者体内的总铁。
