EFFICACY OF DEFEROXAMINE IN PREVENTING COMPLICATIONS OF IRON OVERLOAD IN PATIENTS WITH THALASSEMIA MAJOR
EFFICACY OF DEFEROXAMINE IN PREVENTING COMPLICATIONS OF IRON OVERLOAD IN PATIENTS WITH THALASSEMIA MAJOR
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DOI：
10.1056/nejm199409013310902
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发表时间：
1994-09-01
影响因子：
158.5
通讯作者：
HARRIS, JW
中科院分区：
文献类型：
--
作者：
BRITTENHAM, GM;GRIFFITH, PM;HARRIS, JW
背景资料。为了确定去铁胺是否能预防重型地中海贫血患者输血铁负荷过高的并发症，我们对59例患者(女性30例，男性29例，年龄7~31岁)进行了为期4~10年或直至死亡的定期评估。在每一次随访中，我们都进行了详细的临床和实验室评估，并使用非侵入性磁性设备测量了肝脏铁的储存量。通过肝脏铁储存的磁性测量评估的体内铁负荷与累积输血铁负荷与累积去铁胺使用量的比率密切相关(R=0.89，P<；0.001)(以每公斤体重中铁的毫米摩尔表示，相对于每公斤去铁胺克，转换为自然对数)。比率(输血铁负荷与去铁胺使用量)的自然对数每增加一个单位，与糖耐量受损(相对风险19.3；95%可信区间4.8至77.4)、糖尿病(相对风险9.2；95%可信区间1.8至47.7)、心脏病(相对风险9.9；95%可信区间1.9至51.2)和死亡(相对风险12.6；95%可信区间2.4至65.4)的风险增加相关。在研究期间，所有9例死亡都发生在23名较晚开始螯合治疗的患者中，他们使用的去铁胺较少，与他们的输血铁负荷有关(P<；0.001)。早期使用与输血铁负荷成比例的去铁胺可以减少体内的铁负荷，并有助于预防糖尿病、心脏病和重型地中海贫血患者的早期死亡。
Background. To determine whether deferoxamine prevents the complications of transfusional iron overload in thalassemia major, we evaluated 59 patients (30 were female and 29 male; age range, 7 to 31 years) periodically for 4 to 10 years or until death.Methods. At each follow-up visit, we performed a detailed clinical and laboratory evaluation and measured hepatic iron stores with a noninvasive magnetic device.Results. The body iron burden as assessed by magnetic measurement of hepatic iron stores was closely correlated (R = 0.89, P < 0.001) with the ratio of cumulative transfusional iron load to cumulative deferoxamine use (expressed in millimoles of iron per kilogram of body weight, in relation to grams of deferoxamine per kilogram, transformed into the natural logarithm). Each increase of one unit in the natural logarithm of the ratio (transfusional iron load to deferoxamine use) was associated with an increased risk of impaired glucose tolerance (relative risk, 19.3; 95 percent confidence interval, 4.8 to 77.4), diabetes mellitus (relative risk, 9.2; 95 percent confidence interval, 1.8 to 47.7), cardiac disease (relative risk, 9.9; 95 percent confidence interval, 1.9 to 51.2), and death (relative risk, 12.6; 95 percent confidence interval, 2.4 to 65.4). All nine deaths during the study occurred among the 23 patients who had begun chelation therapy later and used less deferoxamine in relation to their transfusional iron load (P < 0.001).Conclusions. The early use of deferoxamine in an amount proportional to the transfusional iron load reduces the body iron burden and helps protect against diabetes mellitus, cardiac disease, and early death in patients with thalassemia major.