Therapeutic interventions for disease progression in Huntington's disease
Editorial Group: Cochrane Movement Disorders Group
Published Online: 8 JUL 2009
Assessed as up-to-date: 22 DEC 2007
Copyright © 2009 The Cochrane Collaboration. Published by John Wiley & Sons, Ltd.
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.
- Publication Status: New
- Published Online: 8 JUL 2009
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.
Evaluate the effectiveness of therapeutic interventions aimed at modifying disease progression in HD.
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.
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.
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.
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
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.
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
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。
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(ethyleicosapentanoic 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。
此翻譯計畫由臺灣國家衛生研究院(National Health Research Institutes, Taiwan)統籌。
Intervencije za odgađanje progresije Huntingtonove bolesti
Huntingtonova bolest (HB) je autosomno dominantna neurodegenerativna bolest za koju trenutno nema dostupnog lijeka. U ovom je Cochrane sustavnom pregledu analizirana učinkovitost intervencija usmjerenih na promjenu napredovanja (progresije) bolesti te ocijenjena metodološka kvaliteta odgovarajućih kliničkih ispitivanja. U sustavni je pregled uključeno osam istraživanja koja su imala ukupno 1366 sudionika. Rezultati pokazuju da ni jedna intervencija nije pokazala učinak na promjenu progresije bolesti u HB-u.
Prevela: Katarina Vučić
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