Intervention Review

Therapeutic interventions for disease progression in Huntington's disease

  1. Tiago Mestre1,*,
  2. Joaquim Ferreira2,
  3. Miguel M Coelho2,
  4. Mário Rosa1,
  5. Cristina Sampaio2

Editorial Group: Cochrane Movement Disorders Group

Published Online: 8 JUL 2009

Assessed as up-to-date: 22 DEC 2007

DOI: 10.1002/14651858.CD006455.pub2

How to Cite

Mestre T, Ferreira J, Coelho MM, Rosa M, Sampaio C. Therapeutic interventions for disease progression in Huntington's disease. Cochrane Database of Systematic Reviews 2009, Issue 3. Art. No.: CD006455. DOI: 10.1002/14651858.CD006455.pub2.

Author Information

  1. 1

    Institute of Molecular Medicine, Neurological Clinical Research Unit, Lisboa, Portugal

  2. 2

    Faculdade de Medicina de Lisboa, Laboratório de Farmacologia Clínica e Terapêutica, Lisboa, Portugal

*Tiago Mestre, Neurological Clinical Research Unit, Institute of Molecular Medicine, Hospital de Santa Maria, Av. Prof. Egas Moniz, Lisboa, 1649-028, Portugal. tmestre@gmail.com.

Publication History

  1. Publication Status: New
  2. Published Online: 8 JUL 2009

SEARCH

 

Abstract

  1. Top of page
  2. Abstract
  3. Plain language summary
  4. 摘要

Background

Huntington's disease (HD) is an autosomal dominant neurodegenerative disease with an average onset between the fourth and fifth decade of life; it leads to death 15 to 20 years after the onset of symptoms. Although several drugs seem effective in controlling the incapacitating manifestations of HD, no specific therapy is known. The present review aims at analysing the best available data on therapeutic interventions investigated with the goal of modifying the progression of the disease as measured in terms of survival, disability or progression of HD core symptoms.

Objectives

Evaluate the effectiveness of therapeutic interventions aimed at modifying disease progression in HD.

Search methods

The search strategy developed for the Movement Disorders Group was undertaken. The Cochrane Controlled Trials Register, Medline, EMBASE and Clinical Trials Database of the United States National Institute of Health were thoroughly searched until December 2007.

Selection criteria

All randomised, double-blinded, placebo-controlled clinical trials of therapeutics investigated with the goal of modifying disease progression in HD were included. Participants should have genetically confirmed diagnosis of HD or compatible symptoms and a family history. Trials had a follow-up duration of more than three months and at least ten participants. All pharmacological and non-pharmacological interventions were included.

Data collection and analysis

Two reviewers independently assessed the eligibility of identified trials. The methodological quality was assessed and eligible data were registered onto standardised forms. An intention-to-treat analysis was conducted, when feasible. If data were not available in the original publication, the principal investigator of the trial was contacted for further information. A meta-analysis was to be conducted when possible; otherwise, a descriptive summary of the results was provided. The software Revman 5.0.15 was used for statistical analysis.

Main results

Eight trials were included involving a total of 1366 HD patients. The duration of the studies ranged between 30 and 144 weeks (median: 52 weeks). The following interventions were selected: vitamin E, Idebenone, Baclofen, Lamotrigine, creatine, coenzyme Q10 + Remacemide, ethyl-eicosapentanoic acid and Riluzole. No trials produced positive results for the selected efficacy outcome measures. A descriptive summary of the trials is provided. The selected interventions were found to be generally safe and well tolerated.

Authors' conclusions

Only pharmacological interventions were included and none proved to be effective as a disease-modifying therapy for HD. Further trials with greater methodological quality should be conducted using more sensitive biological markers. Pre-symptomatic mutation carriers should be included in future studies.

 

Plain language summary

  1. Top of page
  2. Abstract
  3. Plain language summary
  4. 摘要

Interventions to delay progression of Huntington's disease

Huntington´s disease (HD) is an autosomal dominant neurodegenerative disease for which no cure is currently available. We proposed to assess the effectiveness of interventions aimed at modifying disease progression and evaluate the methodological quality of the corresponding clinical trials. We selected eight trials comprising a total of 1366 participants. The results show that no intervention demonstrated an effect in modifying disease progression in HD.

 

摘要

  1. Top of page
  2. Abstract
  3. Plain language summary
  4. 摘要

背景

關於亨丁頓舞蹈症(Huntington's disease)惡化之治療

亨丁頓舞蹈症是一種體顯性遺傳的神經退化性疾病。約在40至50歲之間發作。發病後15至20年死亡。儘管有些藥物似乎能控制亨丁頓舞蹈症造成的失能狀態,特定療法仍屬未知。本篇研究乃藉由評估生存率、失能狀況、及亨丁頓舞蹈症核心症狀惡化情形,探討關於介入治療以達成改善疾病惡化。

目標

評估治療改善亨丁頓舞蹈症惡化的效果。

搜尋策略

以下資料庫皆搜尋至2007年12月:Cochrane Controlled Trials Register, Medline, EMBASE and Clinical Trials Database of the United States National Institute of Health

選擇標準

我們涵括所有關於治療改善亨丁頓舞蹈症惡化的隨機雙盲安慰劑對照臨床試驗。試驗者必須藉由基因測試確立亨丁頓舞蹈症之診斷,或是具有相符合的症狀,及家族史。試驗達三個月的追蹤且至少十人。所有藥物及非藥物的治療均被收錄。

資料收集與分析

兩名研究者確認試驗的資格。也評估試驗方式的品質。合格的資料被收錄標準格式內。若無法從原始文獻取得數據,則會連絡文獻作者以取得資訊。僅可能將這些資料進行統合分析,否則就使用敘述性摘要。使用Rev Man 5.0.15軟體為統基分析工具。

主要結論

8份試驗,共1366位亨丁頓舞蹈症病患。研究時間介於30周至144周,平均為52周。治療方式計有維他命E(vitamin E), 艾地本(Idebenone), 貝可芬(Baclofen), 樂命達(Lamotrigine), 肌酸(creatine), 輔脢Q10(coenzyme Q10) + Remacemide, 乙基二十碳五酸(ethyleicosapentanoic acid)和Riluzole。這些試驗提供敘述性的摘要。這些治療整體而言安全且無不適症狀。

作者結論

試驗僅有藥物治療方式,且無法證明對於改善亨丁頓舞蹈症的效果。亙進一步的研究必須使用更敏感的生物標記及建立較佳的研究方式。尚未發病而具有基因變異的人也必須納入研究。

翻譯人

本摘要由新光醫院彭馨婷翻譯。

此翻譯計畫由臺灣國家衛生研究院(National Health Research Institutes, Taiwan)統籌。

總結

亨丁頓舞蹈症是一種體顯性遺傳的神經退化性疾病,目前無法治癒。我們評估治療改善亨丁頓舞蹈症惡化的效果,及相關臨床研究的品質。我們選取8份試驗,共1366位亨丁頓舞蹈症病患。研究結果沒有發現對於改善亨丁頓舞蹈症的效果。