Serum ferritin level as a predictor of impaired growth and puberty in thalassemia major patients
Serum ferritin level as a predictor of impaired growth and puberty in thalassemia major patients
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DOI：
10.1111/j.1600-0609.2004.00371.x
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发表时间：
2005-02-01
影响因子：
3.1
通讯作者：
Tamary, H
中科院分区：
文献类型：
--
作者：
Shalitin, S;Carmi, D;Tamary, H
目的：既往研究表明，重型地中海贫血患者在青春期前开始去铁胺（DFO）治疗可以预防铁引起的生长障碍和青春期发育障碍。然而，螯合的早期启动也与 DFO 毒性有关。这项回顾性研究的目的是确定我们的重型地中海贫血患者内分泌并发症和 DFO 骨毒性的患病率，并将其与铁螯合程度相关联。方法：对 39 名重型地中海贫血患者进行了中位随访 16.3 年（范围 2-28）。使用重复的年度测量来计算研究期间的个体平均血清铁蛋白水平。通过手腕和脊柱 X 光片评估骨 DFO 毒性；通过人体测量和青春期阶段的内分泌功能障碍；以及由于缺乏促性腺激素释放激素的黄体生成激素反应而导致的性腺功能减退症。结果：螯合疗法在中位年龄 4.9 岁开始。研究期间的平均血清铁蛋白水平为 2698 +/- 1444 ng/mL。 59% 达到青春期的患者出现性腺功能减退症，36% 达到最终身高的患者出现身材矮小。青春期平均铁蛋白水平 2500 ng/mL 是性腺功能减退症的临界值，青春期前铁蛋白水平 3000 ng/mL 是最终身材矮小的临界值。达到最终身高的患者均未出现 DFO 骨毒性迹象。结论：青春期血清铁蛋白水平高是性腺功能减退症的危险因素，生命头十年血清铁蛋白水平高预示着最终身材矮小。通过较早开始 DFO 或联合使用 DFO 和去铁酮来改善螯合是否会导致更好的生长和性发育而不产生 DFO 毒性，仍有待确定。
Objective: Previous studies suggested that in patients with thalassemia major, initiating deferoxamine (DFO) therapy before puberty can prevent iron-induced failure of growth and puberty. However, early initiation of chelation has also been associated with DFO toxicity. The aim of this retrospective study was to determine the prevalence rates of endocrine complications and DFO bone toxicity in our thalassemia major patients and to correlate them with the degree of iron chelation. Methods: Thirty-nine patients with thalassemia major were followed for a median of 16.3 yr (range 2-28). Individual mean serum ferritin level during the study period was calculated using repeated annual measurements. Bone DFO toxicity was assessed by wrist and spine radiographs; endocrine dysfunction by anthropometric measurements and pubertal stage; and hypogonadotropic hypogonadism by lack of luteinizing hormone response to gonadotropin-releasing hormone. Results: Chelation therapy was initiated at median age 4.9 yr. Mean serum ferritin level during the study period was 2698 +/- 1444 ng/mL. Hypogonadism was noted in 59% of the patients who reached pubertal age, and short stature was found in 36% of patients who reached final height. Mean ferritin level of 2500 ng/mL during puberty was the cut-off for hypogonadism, and ferritin level of 3000 ng/mL during prepuberty was the cut-off for final short stature. None of the patients who attained final height had signs of DFO bone toxicity. Conclusions: High serum ferritin levels during puberty are a risk factor for hypogonadism, and high serum ferritin levels during the first decade of life predict final short stature. It remains to be determined whether improving chelation by earlier initiation of DFO or by the combined use of DFO and deferiprone will lead to better growth and sexual development without DFO toxicity.