曲恩汀和d-青霉胺治疗Wilson病的临床结果和安全性比较分析：系统回顾和荟萃分析

Rare Pub Date : 2025-01-01 DOI:10.1016/j.rare.2025.100077

Hafiz Muhammad Ehsan Arshad, Muhammad Zain Raza, Musab Maqsood, Muhammad Omais, Muhammad Hashim Faisal, Ali Ahmad Nadeem

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引用次数: 0

摘要

威尔逊病（WD）是一种罕见的铜代谢紊乱，需要终生治疗，通常使用d -青霉胺或曲entine。本综述比较了它们的临床有效性和安全性。方法检索pubmed、Cochrane、Clinicaltrials.gov和WHO-ICTRP以及其他来源。报告任何年龄或分期的WD患者接受曲恩汀或d -青霉胺治疗的队列和随机对照试验被纳入。Mantel-Haenszel方法以及固定或随机效应模型用于分析二分类结果。结果共纳入26个队列和1个随机对照试验。使用曲恩汀治疗失败的几率更大(OR = 4.09；95 % ci: 2.34 - -7.15;p & lt; 0.00001);(2)曲恩汀组不良事件较少(OR = 0.34；95 % ci: 0.14 - -0.80; = 0.01页);(3)曲恩汀组因不良事件而中断治疗的患者较少(OR = 0.30；95 % ci: 0.21 - -0.43;p & lt; 0.00001);(4)症状加重无显著差异(OR = 1.68；95 % ci: 0.88 - -3.20; = 0.012页)。症状加重亚组分析显示，神经系统加重差异无统计学意义(OR = 1.33；95 % ci: 0.44 - -3.97;p = 0.61)，使用曲恩汀治疗肝脏恶化的几率更高(OR = 2.45；95 % ci: 1.17 - -5.12; = 0.02页)。与d -青霉胺相比，曲伦汀具有相似的血清铜参数和稍低的尿铜排泄率。曲恩汀影响凝血功能，但无临床相关性，两种治疗对妊娠结局的影响相似。结论曲恩汀治疗与d -青霉胺治疗相比，不良事件和停药发生率明显降低，但治疗失败和神经系统恶化发生率较高。然而，证据的低质量和间接性可能降低了结果的有效性。需要直接比较两种治疗的临床结果的研究来建立更有力的证据。

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Comparative analysis of clinical outcomes and safety profile of trientine and d-penicillamine in the management of Wilson’s disease: A systematic review and meta-analysis

Introduction

Wilson disease (WD) is a rare metabolic disorder of copper metabolism, requiring life-long therapy, usually with D-penicillamine or trientine. This review compares their clinical effectiveness and safety.

Methodology

PubMed, Cochrane, Clinicaltrials.gov and WHO-ICTRP, along with other sources were searched. The cohorts and RCTs reporting WD patients of any age or stage, receiving either trientine or D-penicillamine, were included. The Mantel-Haenszel method along with either a fixed- or random-effects model was used for analysing dichotomous outcomes.

Results

Twenty-six cohorts and one RCT were included. The odds of having (1) treatment failures were greater with trientine (OR = 4.09; 95 %-CI:2.34–7.15; p < 0.00001); (2) adverse events were lesser with trientine (OR = 0.34; 95 %-CI:0.14–0.80; p = 0.01); (3) treatment discontinuation due to adverse events were lesser with trientine (OR = 0.30; 95 %-CI:0.21–0.43; p < 0.00001); and (4) symptomatic worsening were not significantly different (OR = 1.68; 95 %-CI:0.88–3.20; p = 0.012). Subgroup analysis within symptomatic worsening showed non-significant difference for neurological worsening (OR = 1.33; 95 %-CI:0.44–3.97; p = 0.61) and greater odds with trientine for hepatic worsening (OR = 2.45; 95 %-CI:1.17–5.12; p = 0.02). Trientine had similar serum copper parameters and slightly lower urinary copper-excretion rates compared to D-penicillamine. Trientine affected coagulation profiles but with no clinical association, and both treatments had similar effects on pregnancy outcomes.

Conclusion

Trientine therapy had significantly lower incidences of adverse events and treatment discontinuations, but higher incidences of treatment failure and neurological worsening compared to D-penicillamine. However, the low quality and indirectness of the evidence may have lowered the validity of the results. Studies directly comparing the clinical outcomes of both treatments are needed to establish more robust evidence.

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