Wolfe L, Olivieri N, Sallan D, Colan S, Rose V, Propper R, Freedman M H, Nathan D G
N Engl J Med. 1985 Jun 20;312(25):1600-3. doi: 10.1056/NEJM198506203122503.
We examined the efficacy of long-term subcutaneous deferoxamine therapy in the prevention of iron-related cardiac disease in patients with thalassemia major who began treatment after the age of 10 years. Of 36 such patients without preexisting cardiac disease, 19 did not comply with the program of chelation therapy. Over the course of treatment (1977 to 1983) serum ferritin and aspartate aminotransferase levels fell in the compliant group, from mean values (+/- S.D.) of 4765 +/- 2610 to 2950 +/- 1850 ng per milliliter and 58.1 +/- 22 IU to 30 +/- 20 IU per liter, respectively (P less than 0.05), but rose in the noncompliant group, from 5000 +/- 2316 to 6040 +/- 2550 ng per milliliter and 56.6 +/- 20 to 90 +/- 35 IU per liter, respectively. Only one patient in the compliant group acquired cardiac disease and died of fulminant congestive heart failure. In contrast, 12 noncompliant patients acquired cardiac disease, and 7 died. In addition, the mean age of the compliant population (18.9 +/- 4.5 years) now approaches the mean age of acquisition of cardiac disease in the noncompliant group (19 +/- 4.3). These data demonstrate that compliance with treatment with deferoxamine may protect patients from cardiac disease induced by iron overload.
我们研究了长期皮下注射去铁胺疗法对10岁后开始治疗的重型地中海贫血患者预防铁相关心脏疾病的疗效。在36例无既往心脏病的此类患者中，19例未遵守螯合治疗方案。在治疗过程中（1977年至1983年），依从组的血清铁蛋白和天冬氨酸转氨酶水平下降，分别从平均值（±标准差）每毫升4765±2610纳克降至2950±1850纳克，每升58.1±22国际单位降至30±20国际单位（P<0.05），而不依从组则上升，分别从每毫升5000±2316纳克升至6040±2550纳克，每升56.6±20国际单位升至90±35国际单位。依从组中只有1例患者患心脏病并死于暴发性充血性心力衰竭。相比之下，12例不依从患者患心脏病，7例死亡。此外，依从组的平均年龄（18.9±4.5岁）现在接近不依从组患心脏病的平均年龄（19±4.3岁）。这些数据表明，遵守去铁胺治疗可能使患者免受铁过载引起的心脏病。
