Complications of β-thalassemia major in north America
Complications of β-thalassemia major in north America
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DOI：
10.1182/blood-2003-09-3167
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发表时间：
2004-07-01
期刊：
影响因子：
20.3
通讯作者：
Cohen, AR
中科院分区：
文献类型：
--
作者：
Cunningham, MJ;Macklin, EA;Cohen, AR
在过去的40年中，重型β地中海贫血患者的治疗有了显着改善;然而，这些患者目前的临床状况仍然很差。我们在美国国立卫生研究院资助的地中海贫血临床研究网络的登记处对342例患者进行了横断面研究。在35%的受试患者中存在丙型肝炎暴露的证据，与年龄相关，自发病毒清除率为33%。铁蛋白水平范围为147至11010 ng/mL（中位数为1696 ng/mL）。中位肝脏铁含量为7.8 mg/g干重，23%的患者的值为15 mg/g干重或更高。没有15岁或以下的患者，5%的16至24岁的患者患有需要药物治疗的心脏病。10%的人活组织检查有肝硬化。内分泌并发症在成人中很常见。74例（22%）患者近期植入了中心静脉输液器（CVAD）。在38例患者的80次菌血症发作中，90%可归因于CVAD。在接受去铁胺螯合治疗的330例患者中，224例（68%）报告无并发症。我们的结论是，丙型肝炎，铁相关的器官功能障碍，铁螯合治疗的并发症是强烈的年龄依赖于北美β地中海贫血患者。(C)2004年，美国血液学会。
Treatment of patients with beta-thalassemia major has improved dramatically during the past 40 years; however, the current clinical status of these patients remains poorly characterized. We performed a cross-sectional study of 342 patients in the Registry of the National Institutes of Health-sponsored Thalassemia Clinical Research Network. Evidence of hepatitis C exposure was present in 35% of tested patients, was associated with age, and had a rate of spontaneous viral clearance of 33%. Ferritin levels ranged from 147 to 11010 ng/mL (median, 1696 ng/mL). Median hepatic iron content was 7.8 mg/g dry weight and 23% of patients had values of 15 mg/g dry weight or higher. No patients 15 years or younger and 5% of patients aged 16 to 24 years had heart disease requiring medication. Ten percent had cirrhosis on biopsy. Endocrinologic complications were common among adults. Seventy-four (22%) patients had recent implantable central venous access devices (CVADs) placed. Among 80 episodes of bacteremia in 38 patients, 90% were attributable to the CVAD. Among 330 patients who had received deferoxamine chelation therapy, 224 (68%) reported no complications. We conclude that hepatitis C, iron-related organ dysfunction, and complications of iron chelation therapy are strongly age-dependent in North American patients with beta-thalassemia. (C) 2004 by The American Society of Hematology.