In a UK trial reported in the Journal of Clinical Oncology, Stone et al found that the stimulant methylphenidate did not significantly improve fatigue vs placebo in patients with advanced cancers receiving palliative care.
Study Details
In the double-blind multicenter study, 159 patients with advanced incurable cancers and fatigue of > 3/10 on a numeric rating scale were randomly assigned between June 2018 and April 2023 to receive methylphenidate (n = 82) or placebo (n = 79) beginning at 5 mg twice daily with individualized dose titration once per week over 6 weeks up to a maximum of 20 mg three times daily. Exclusion criteria included: hypertension; psychiatric, cardiovascular, cerebrovascular, renal, liver, or blood disorders; substance dependency; and epilepsy.
The trial ended at 10 weeks. The primary outcome measure was the difference in Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F) scores between groups at 6 ± 2 weeks.
Key Findings
After 6 ± 2 weeks, FACIT-F scores were increased (improved) by 1.97 points (95% confidence interval [CI] = –0.95 to 4.90 points, P = .186) with methylphenidate vs placebo.
At 10 weeks, the FACIT-F score in the methylphenidate group was nominally higher vs the placebo group (difference = 2.20 points, 95% CI = 0.39–4.01 points); however, the difference did not reach the minimal clinically important difference of five points.
At 6 ± 2 weeks, no differences between groups were observed in quality-of-life or symptom domains except for depression scores; these were nominally reduced (improved) in the methylphenidate group vs the placebo group (difference = –1.35, 95% CI = –2.41 to –0.30).
There were no differences in serious adverse events between the two groups (25 occurred in each group).
The investigators concluded, “After 6 ± 2 weeks of treatment, methylphenidate was not superior to placebo for treating fatigue in advanced cancer. Methylphenidate was safe and well tolerated.”
Patrick Charles Stone, MD, MA, MRCP, of Marie Curie Palliative Care Research Department, Division of Psychiatry, University College London, is the corresponding author for the Journal of Clinical Oncology article.
Disclosure: The study was supported by the UK National Institute for Health Research (NIHR). For full disclosures of the study authors, visit ascopubs.org.
