PROLONGED SURVIVAL IN PATIENTS WITH BETA-THALASSEMIA MAJOR TREATED WITH DEFEROXAMINE
PROLONGED SURVIVAL IN PATIENTS WITH BETA-THALASSEMIA MAJOR TREATED WITH DEFEROXAMINE
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DOI：
10.1016/s0022-3476(05)83374-8
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发表时间：
1991-04-01
影响因子：
5.1
通讯作者：
HILGARTNER, MW
中科院分区：
文献类型：
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作者：
EHLERS, KH;GIARDINA, PJ;HILGARTNER, MW
为了确定重度β-地中海贫血患者的生存期是否通过使用大量输血和去铁胺螯合治疗而延长，我们采用Kaplan-Meier乘积限法分析了寿命。第1组患者（n = 71）在1960年至1976年期间接受低输血方案（输血前血红蛋白水平7 - 8 gm/dl）和无螯合作用的受试者的估计中位生存年龄为17.4岁，而第2组受试者（n = 80）为31.0岁，在1976年至1978年之间开始大量输血（输血前血红蛋白水平为10.5至11.5 gm/dl）和去铁胺螯合（每天20至60 mg/kg）（p < 0.0001）。对于70例接受过量输血和去铁胺治疗的患者，我们有数据来计算输注铁的总毫克数与去铁胺累积克数的比值。死亡的24名患者的总铁负荷> 1.05 gm/kg;他们的比率超过31。这些患者的特点是对螯合治疗的依从性差或治疗开始较晚，死亡前无法接受足够的去铁胺。除1例外，所有患者死亡前均出现需要治疗的心律失常，所有患者均出现心力衰竭。在41名同样铁负荷的幸存者中，33人的比率< 31;只有3人心律失常，5人心力衰竭。我们的结论是，去铁胺治疗，当使用量成比例的铁负荷，延迟心脏并发症和延长寿命。
To determine whether survival of patients with beta-thalassemia major has been prolonged by management that utilizes hypertransfusion and chelation with deferoxamine, we analyzed longevity by the Kaplan-Meier product-limit method. Group 1 patients (n = 71) followed between 1960 and 1976 with a low-transfusion regimen (pretransfusion hemoglobin level 7 to 8 gm/dl) and no chelation had an estimated median age of survival of 17.4 years, whereas it was 31.0 years for group 2 subjects (n = 80), who began hypertransfusion between 1976 and 1978 (pretransfusion hemoglobin level 10.5 to 11.5 gm/dl) and chelation with deferoxamine (20 to 60 mg/kg per day) (p < 0.0001). For 70 patients who were treated with hypertransfusion and deferoxamine, we had data to calculate the ratio of total milligrams of transfusional iron to cumulative grams of deferoxamine. The 24 patients who died had a total iron burden of > 1.05 gm/kg; the ratio for them exceeded 31. These patients were characterized by poor compliance with chelation or by late start of therapy, with inability to receive enough deferoxamine before death. Death was preceded by arrhythmia requiring therapy in all but one, and by cardiac failure in all. Of 41 similarly iron-loaded survivors, 33 had a ratio of < 31; only three had an arrhythmia, and five had cardiac failure. We conclude that treatment with deferoxamine, when used in amounts proportional to iron burden, delayed cardiac complications and improved longevity.