Testing pilocarpine drops for dry mouth in advanced cancer using n-of-1 trials: A feasibility study

Nikles, Jane, Mitchell, Geoffrey K., Hardy, Janet, Agar, Meera, Senior, Hugh Edgar John, Carmont, Sue-Ann, Schluter, Philip J., Good, Phillip, Vora, Rohan and Currow, David (2015) Testing pilocarpine drops for dry mouth in advanced cancer using n-of-1 trials: A feasibility study. Palliative Medicine, 29 10: 967-974. doi:10.1177/0269216315585473

Author Nikles, Jane
Mitchell, Geoffrey K.
Hardy, Janet
Agar, Meera
Senior, Hugh Edgar John
Carmont, Sue-Ann
Schluter, Philip J.
Good, Phillip
Vora, Rohan
Currow, David
Title Testing pilocarpine drops for dry mouth in advanced cancer using n-of-1 trials: A feasibility study
Journal name Palliative Medicine   Check publisher's open access policy
ISSN 0269-2163
Publication date 2015-05-22
Year available 2015
Sub-type Article (original research)
DOI 10.1177/0269216315585473
Open Access Status Not Open Access
Volume 29
Issue 10
Start page 967
End page 974
Total pages 8
Place of publication London, United Kingdom
Publisher Sage Publications
Collection year 2016
Language eng
Formatted abstract
Background: Dry mouth is a common and troublesome symptom in palliative care. Pilocarpine is a cholinergic agent that promotes salivation.

Aim: This study aimed to test the feasibility of using n-of-1 trials to test pilocarpine drops compared to placebo, for patients of palliative care units with advanced cancer, who experienced dry mouth.

Design: This was an N-of-1 study, in which each participant was offered three cycles of pilocarpine drops 4% (6 mg tds) (3 days) and placebo drops (3 days) in random order.

Setting/participants: Participants were patients of specialist palliative care services with advanced cancer assessed as having a dry mouth, defined as having a score of ⩾3 on an 11-point self-rated xerostomia numerical rating scale, from any cause. Patients self-completed a diary using validated symptom and quality-of-life scores. The randomisation order was unmasked at the end of each person’s trial by a clinician independent of the trial to allow a treatment decisions for individual patients to be made.

Results: Nine patients completed at least 1 cycle; 33 cycles of data were completed in total, comprising 438 doses of pilocarpine. Four patients completed the trial: two responded and two did not. Most withdrawals related to deteriorating condition, unacceptable toxicity, non-compliance with study procedures or withdrawal of consent. Many issues contributed to slow recruitment and high withdrawal rate.

Conclusion: The formulation of pilocarpine drops proved unacceptable to most participants. More work is required to determine an appropriate formulation, dose and method of delivery and then a retest of pilocarpine drops for this symptom.
Keyword Pilocarpine
Palliative care
Feasibility studies
Clinical trial
Crossover trials
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ

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Created: Thu, 24 Sep 2015, 14:54:08 EST by Natalie Cowley on behalf of School of Nursing, Midwifery and Social Work