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口服螯合疗法(地拉罗司)对巴基斯坦重型β地中海贫血多次输血患儿的疗效及不良反应

Efficacy and adverse effects of oral chelating therapy (deferasirox) in multi-transfused Pakistani children with β-thalassemia major.

作者信息

Ejaz Muzamil Shabana, Baloch Shagufta, Arif Fehmina

机构信息

Dr. Muzamil Shabana Ejaz, Associate Professor, Department of Pediatrics Dow University of Health Sciences Civil Hospital Karachi, Karachi - Pakistan.

Dr. Shagufta Baloch, FCPS Trainee, Department of Pediatrics Dow University of Health Sciences Civil Hospital Karachi, Karachi - Pakistan.

出版信息

Pak J Med Sci. 2015;31(3):621-5. doi: 10.12669/pjms.313.6972.

DOI:10.12669/pjms.313.6972
PMID:26150856
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4485283/
Abstract

OBJECTIVE

To determine the efficacy and adverse effects of oral chelation therapy (deferasirox) in multi-transfused β-thalassemia major patients visiting pediatric thalassemia clinic in Civil Hospital Karachi.

METHODS

This prospective study was conducted at pediatric thalassemia clinic of Civil Hospital Karachi. Hundred multi-transfused β-thalassemia patients registered in the clinic for oral iron chelation therapy were included in the study. Information regarding clinical and laboratory parameters including abdominal pain, jaundice, serum ferritin, creatinine and serum transaminase levels were recorded on a Performa and data was analyzed through SPSS 16.

RESULTS

Hundred patients were stratified into two age groups, 54% were below and 46% were above nine year. Majority were males, 62% and 38% were females. Abdominal pain 41%, nausea 31%, vomiting 15%, jaundice 15% and elevated serum creatinine 11.5% were frequently observed clinical adverse effects in this study. Serum glutamic pyruvic transaminase (SGPT) level was statistically significant compared with initial visit and six months after optimal chelation therapy (p=0.030). Although Serum ferritin was decreased but it was not statistically significant (p=0.929).

CONCLUSION

Deferasirox is an effective oral chelation agent for β-thalassemia major patients. Most common adverse effects of the drug are abdominal pain, nausea, vomiting, and elevation of liver enzymes.

摘要

目的

确定口服螯合疗法(地拉罗司)对在卡拉奇市民医院儿科地中海贫血门诊就诊的多次输血的重型β地中海贫血患者的疗效和不良反应。

方法

这项前瞻性研究在卡拉奇市民医院儿科地中海贫血门诊进行。纳入了100名在该门诊登记接受口服铁螯合疗法的多次输血的β地中海贫血患者。在一份表格上记录了包括腹痛、黄疸、血清铁蛋白、肌酐和血清转氨酶水平在内的临床和实验室参数信息,并通过SPSS 16进行数据分析。

结果

100名患者被分为两个年龄组,54%年龄在9岁以下,46%年龄在9岁以上。大多数为男性,占62%,女性占38%。本研究中经常观察到的临床不良反应包括腹痛41%、恶心31%、呕吐15%、黄疸15%和血清肌酐升高11.5%。与初次就诊及最佳螯合治疗6个月后相比,血清谷丙转氨酶(SGPT)水平有统计学意义(p = 0.030)。虽然血清铁蛋白有所下降,但无统计学意义(p = 0.929)。

结论

地拉罗司是一种对重型β地中海贫血患者有效的口服螯合剂。该药物最常见的不良反应是腹痛、恶心、呕吐和肝酶升高。

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