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比较不同螯合疗法治疗重型β地中海贫血患者的疗效。

COMPARISON OF DIFFERENT TREATMENT MODALITIES OF CHELATION THERAPY IN BETA-THALASSEMIA MAJOR PATIENTS.

机构信息

Shaikh Zayed Hospital Lahore-Pakistan.

Punjab institute of cardiology Lahore-Pakistan.

出版信息

J Ayub Med Coll Abbottabad. 2024 Jan-Mar;36(1):35-39. doi: 10.55519/JAMC-01-12436.

Abstract

BACKGROUND

Thalassemia has a high prevalence and carrier rate of 8-10% in Pakistan, repeated blood transfusions lead to iron deposition in organs. In this Prospective study, we have compared the efficacy of three chelation regimens being used in our country. It has been conducted at PBTS, Fatmid Foundation and Children Hospital Lahore.

METHODS

60 thalassemia major patients, were divided into 3 groups according to their mode of chelation. Patients in group I were on oral iron chelator deferiprone, 7 days per week. Thalassemics in group II were on parenteral iron chelator deferoxamine given subcutaneously for 4 days a week, and group III patients were on combination therapy, deferiprone for 5 days & deferoxamine given twice weekly. The assessment of chelation was done by measurement of serum ferritin and 24-hour urinary iron excretion at the start of the study and then after six months of follow-up. To assess the hepatic iron, hepatic MRIs were also performed.

RESULTS

Ferritin levels were maximally decreased in group II, followed by group III, with no significant reduction in group I. However, a statistically significant difference in mean urinary iron excretion (increased) was seen in group III. The hepatic iron was very high in all three groups as shown by the hepatic MRI.

CONCLUSIONS

Combination chelation therapy is the most effective chelation therapy in iron-overloaded patients. It helps to improve compliance and increases urinary iron excretion. Patients on DFX had the lowest degree of hepatic siderosis even though it was considerably higher than the normal population.

摘要

背景

在巴基斯坦,地中海贫血的患病率和携带率高达 8-10%,反复输血会导致铁在器官中沉积。在这项前瞻性研究中,我们比较了三种螯合方案在我国的疗效。该研究在 PBTS、Fatmid 基金会和拉合尔儿童医院进行。

方法

将 60 名重型地中海贫血患者根据螯合方式分为 3 组。I 组患者接受口服铁螯合剂地拉罗司,每周 7 天。II 组地中海贫血患者接受每周 4 天皮下注射的静脉铁螯合剂去铁胺。III 组患者接受联合治疗,地拉罗司 5 天,去铁胺每周 2 次。在研究开始时和随访 6 个月后,通过测量血清铁蛋白和 24 小时尿铁排泄量来评估螯合作用。为了评估肝铁,还进行了肝脏 MRI。

结果

II 组铁蛋白水平降低最多,其次是 III 组,I 组无明显降低。然而,III 组的平均尿铁排泄量(增加)有统计学显著差异。三组患者的肝脏铁含量均非常高,如肝脏 MRI 所示。

结论

联合螯合疗法是治疗铁过载患者最有效的螯合疗法。它有助于提高依从性并增加尿铁排泄。尽管 DFX 组的患者肝脏铁沉着程度较低,但仍明显高于正常人群。

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