Methylphenidate for Hematologic Cancer-Related Fatigue: A Randomized Clinical Trial
In brief
Methylphenidate improved fatigue scores 5 points more than placebo in blood cancers
In a randomized crossover trial of 152 adults with hematologic cancer, fatigue scores improved by 8.9 points during six weeks of methylphenidate, compared with 3.9 points on placebo. Side effects were mostly mild but more common with methylphenidate, and the results may not apply to patients receiving recent chemotherapy or other psychotropic drugs.
- Journal
- Journal of clinical oncology : official journal of the American Society of Clinical Oncology (Q1)
- Published
- 30 October 2026
- Study design
- Randomized controlled trial
- Evidence level
- Level 1, High (CEBM 1b)
- Authors
- Henrik Frederiksen, Henrik Gregersen, Sören Möller, Dennis Lund Hansen, Jens-Jakob Kjer Møller, Mette Borg Clausen, et al.
- PMID
- 42962158
- DOI
- 10.1200/JCO-26-00578
Why clinicians should know about it
- Picked for Hematology (top studies of the week, 1 November 2026): Randomized trial of fatigue treatment in blood cancers
Abstract
PURPOSE: Patients with hematologic cancer rate fatigue as their most frequent and severe unmet issue. Methylphenidate (MTP) has been reported to alleviate cancer-related fatigue (CRF) in some studies, but data from hematologic cancers are lacking. We investigated MTP for hematologic CRF. METHODS: Using a double-blinded, placebo-controlled, two-period, crossover design, 152 adult fatigued patients with hematologic cancer were randomly assigned to 6 weeks of either MTP or placebo, with a treatment-free week before the crossover. Eligibility required a Visual Analog Scale fatigue score ≥4 (range, 0-10), hemoglobin ≥8.0 g/dL, no chemotherapy within 8 weeks, and no concomitant psychopharmacologic treatment. The primary end point was the between-group difference in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) score from baseline to end of each period. RESULTS: The median age was 59.0 years (range, 21-83), 44% were women, the performance status was 0 in 57.3%, and 11% had received high-dose therapy. FACIT-F improved 8.9 points (95% CIs, 7.3 to 10.4) after MTP and 3.9 points (95% CI, 2.3 to 5.4) after placebo. The between-group difference of 5.0 points (95% CI, 2.8 to 7.2) was in favor of MTP. Reduced fatigue ratings in favor of MTP were also observed using other tools. Toxicity was mild, but more adverse events were reported during the MTP (N = 73; 57%) versus placebo (N = 52; 40%) periods. CONCLUSION: MTP treatment alleviated fatigue and may be considered as a pharmacologic fatigue intervention in patients with hematologic cancer.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.