Introduction Methylphenidate is a psychostimulant that has been used to relieve depressive symptoms in advanced cancer patients. No studies compare its efficacy against placebo in this group of patients.

Objective To explore the efficacy of methylphenidate compared with placebo in the relief of depressive symptoms in advanced cancer patients.

Material and methods A multicentre, double-blind, randomised placebo-controlled clinical trial was undertaken comparing the efficacy of methylphenidate and placebo in depressive symptoms. Advanced stage cancer patients were eligible if they scored at least two points on the Two Question Screening Survey for depression. A reduction of at least two points on the Edmonton Symptom Assessment Scale for depression (0–10) was considered as a response.

Results Sixty-nine patients were included (methylphenidate: n=31, placebo: n=38); median daily dose of methylphenidate was 25 mg. Fifty-eight patients (84%) who completed the first week of treatment were considered suitable for evaluation. In the intention to treat analysis, there were 14/31 (45%) responses with methylphenidate and 10/38 (26%) responses with placebo (difference: 19%; 95% CI: 4% to 39%; p=0.10). With the Hospital Anxiety and Depression Scale, 11/19 (58%) patients with methylphenidate and 10/24 (42%) with placebo improved from a score compatible with depression in the first 7 days (difference 16%; 95% CI 13% to 42%; p=0.29). The proportion of patients indicating adverse effects was similar for both cohorts (p=0.99).

Conclusion Compared with the placebo, methylphenidate demonstrated a positive trend in the incidence of response for depressive symptoms in advanced cancer patients.