Positive effect of methylphenidate for cancer-related cognitive impairment: a pilot randomized trial.

CONTEXT: Breast cancer and its treatments can cause cancer-related cognitive impairment (CRCI). Methylphenidate (MPH) may help improve cognitive function. OBJECTIVES: We evaluated the feasibility of using MPH to manage CRCI in women after breast cancer treatment in a pilot randomized, double-blind, placebo-controlled trial. METHODS: Women who completed treatment for breast cancer ≥6 months prior and had documented cognitive dysfunction at trial entry were randomized to MPH or placebo (10 mg/day for 14 days, increased to 20 mg/day for an additional 14 days if tolerated). Patient-reported outcomes, including the Functional Assessment of Cancer Therapy - Cognitive Function (FACT-Cog) and neuropsychological tests were administered on Days 0 (baseline), 15, and 29. RESULTS: Twenty women (mean age=50.2) were enrolled and randomized (MPH: n=9; placebo: n=11). All completed the study. Most had received chemotherapy (80%) in addition to surgery and radiotherapy and were taking hormonal therapy during study participation. Self-reported side effects were mild to moderate and transient. A linear mixed-effects model suggested greater improvement in FACT-Cog Perceived Cognitive Impairments scores in the MPH group compared to the placebo group on Day 29 (mean difference=10.1, p=0.02). No between-group differences were observed in neuropsychological test scores. MPH did not worsen physical, psychological, or sleep-related symptoms compared to placebo. CONCLUSION: This pilot study provides preliminary evidence that MPH may have clinically relevant effects on perceived cognitive dysfunction in women after breast cancer treatment and supports evaluation in a larger phase III trial.