Do pilocarpine drops help dry mouth in palliative care patients: A protocol for an aggregated series of n-of-1 trials

Background: It is estimated that 39,000 Australians die from malignant disease yearly. Of these, 60% to 88% of advanced cancer patients suffer xerostomia, the subjective feeling of mouth dryness. Xerostomia has significant physical, social and psychological consequences which compromise function and quality of life. Pilocarpine is one treatment for xerostomia. Most studies have shown some variation in individual response to pilocarpine, in terms of dose used, and timing and extent of response.We will determine a population estimate of the efficacy of pilocarpine drops (6 mg) three times daily compared to placebo in relieving dry mouth in palliative care (PC) patients. A secondary aim is to assess individual patients' response to pilocarpine and provide reports detailing individual response to patients and their treating clinician. Methods/Design. Aggregated n-of-1 trials (3 cycle, double blind, placebo-controlled crossover trials using standardized measures of effect). Individual trials will identify which patients respond to the medication. To produce a population estimate of a treatment effect, the results of all cycles will be aggregated. Discussion. Managing dry mouth with treatment supported by the best possible evidence will improve functional status of patients, and improve quality of life for patients and carers. Using n-of-1 trials will accelerate the rate of accumulation of high-grade evidence to support clinical therapies used in PC. Trial registration. Australia and New Zealand Clinical Trial Registry Number: 12610000840088. © 2013 Nikles et al.; licensee BioMed Central Ltd

Single-patient multiple crossover studies to determine the effectiveness of paracetamol in relieving pain suffered by patients with advanced cancer taking regular opioids: A pilot study

Paracetamol is a useful adjunct when used in combination with “weak opioids” for chronic pain in palliative care patients with advanced cancer, however it is not certain that there is continuing benefit when used in conjunction with “strong” opioids. N-of-1 trials allowed individual treatment decisions to be made for each participant: there was no added benefit for any of the participants, although no conclusion about the added benefit of paracetamol above regular opioids was possible for the group, due to insufficient numbers recruited. Paracetamol may not provide added benefit above regular opioids; this should assessed on a case by case basis to justify the extra tablet load