Using Single Patient Trials to determine the effectiveness of psychostimulants in fatigue in advanced cancer patients [ 2007 - 2012 ]

Research Grant

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Researchers: Prof Geoffrey Mitchell (Principal investigator) ,  A/Pr Michael Yelland Dr Jane Nikles Dr Rohan Vora Prof David Currow
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Brief description The lack of good evidence in palliative care (PC) is widely acknowledged but research in PC is difficult. Methodological barriers include: difficulties in recruitment, high rates of attrition, problems with maintaining distinct and sustainable intervention strategies, poorly chosen outcomes and opposition to randomization. Organizational barriers include: lack of research infrastructure, few trained clinical researchers, prioritisation of clinical responsibilities and funding difficulties. The hierarchy of evidence rates RCTs as the gold standard. An alternative is the n-of-1 trial: a randomized, double-blind cross-over comparison of active drug with placebo or another drug. The patient is in effect their own control. N-of-1 trials provide an objective means of testing the effectiveness of medicines in individual patients, providing evidence stronger than RCT evidence for the efficacy of that drug in that particular individual. If multiple n-of-1 trials are conducted, the resultant data amounts to RCT evidence for that treatment in a population. We propose n-of-1 trials as a workable option for researching the benefit of drugs and other therapies in PC patients. If successful, this model could be accepted internationally as the gold standard for research in this difficult population group. This would be a world first and of great national and international significance. In advanced cancer, the prevalence of fatigue is very high at 60-90% and can be related to the treatment or the disease itself. The impact of fatigue on function (physical, mental, social and spiritual) and hence quality of life (QOL) is very significant for many palliative patients as well as their families-carers. The role of pyschostimulants in the management of fatigue in patients with advanced cancer and life limiting disease needs to be defined. We will conduct n-of-1 trials of psychostimulants (i.e. methylphenidate) for fatigue in a group of 40 patients, recruited from 5 sites around Australia through a national clinical trial network recently set up for palliative care research. Managing fatigue with treatment supported by the best possible evidence for individual patients and producing any improvement in fatigue will improve patients� functional status, and will greatly improve QOL for patients and carers.

Funding Amount $AUD 162,563.00

Funding Scheme NHMRC Strategic Awards

Notes Palliative Care Research

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