Abstract | BACKGROUND: METHODS: Patients with prion disease were recruited via the UK national referral system and were offered a choice between quinacrine (300 mg daily), no quinacrine, or randomisation to immediate quinacrine or deferred quinacrine in an open-label, patient-preference trial. The primary endpoints were death and serious adverse events possibly or probably related to the study drug. This study is registered, ISRCTN 06722585. FINDINGS: 107 patients with prion disease (45 sporadic, two iatrogenic, 18 variant, and 42 inherited) were enrolled, 23 in a pilot study and 84 in the main study. Only two patients chose randomisation; 40 took quinacrine during follow-up (37 who chose it at enrollment). Choice of treatment was associated with disease severity, with those least and most severely affected more likely to choose not to receive quinacrine. 78 (73%) patients died: one randomly assigned to deferred treatment, 26 of 38 who chose immediate quinacrine, and 51 of 68 who chose no quinacrine. Although adjusted mortality was lower in those who chose to take quinacrine than in those who did not, this was due to confounding with disease severity, and there was no difference in mortality between groups after adjustment. Four of 40 patients who took quinacrine had a transient response on neurological rating scales. Only two of 14 reported serious adverse events were judged quinacrine-related. INTERPRETATION:
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Authors | John Collinge, Michele Gorham, Fleur Hudson, Angus Kennedy, Geraldine Keogh, Suvankar Pal, Martin Rossor, Peter Rudge, Durre Siddique, Moira Spyer, Dafydd Thomas, Sarah Walker, Tom Webb, Steve Wroe, Janet Darbyshire |
Journal | The Lancet. Neurology
(Lancet Neurol)
Vol. 8
Issue 4
Pg. 334-44
(Apr 2009)
ISSN: 1474-4422 [Print] England |
PMID | 19278902
(Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)
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Chemical References |
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Topics |
- Adolescent
- Adult
- Age Factors
- Aged
- Aged, 80 and over
- Female
- Follow-Up Studies
- Humans
- Kaplan-Meier Estimate
- Male
- Middle Aged
- Neuropsychological Tests
- Prion Diseases
(drug therapy, mortality)
- Quinacrine
(administration & dosage, therapeutic use)
- Risk Factors
- Severity of Illness Index
- Sex Factors
- Young Adult
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