Trial reportInternational journal of clinical oncology2025
Efficacy and safety of mycophenolate mofetil in patients with high-grade locally advanced or metastatic osteosarcoma (ESMMO): a multicenter, phase II clinical trial.
Trial report in International journal of clinical oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
1 citing paper in PubMed.
- IMP metabolic mechanisms and IMPDH targeting strategies in tumor metabolic reprogramming and therapy (Review).International journal of molecular medicine · 2026Review
Corrections and comments
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Authors and funding
17 authors.
Funding
Abstract
backgroundOsteosarcoma is a prevalent and aggressive bone malignancy primarily affecting young adults. Despite advances in standard treatment, the 5-year survival rate remains low, highlighting the need for new chemotherapy options. Mycophenolate mofetil, an immunosuppressant used post-transplant, has shown promising preclinical anticancer effects in osteosarcoma, warranting further investigation for potential repurpose in humans.
methodsWe conducted this proof-of-concept phase II clinical trial to assess the efficacy and safety of mycophenolate mofetil in high-grade locally advanced or metastatic osteosarcoma. Patients received 3-5 g/day mycophenolate mofetil for four 28-day cycles or until disease progression or unacceptable toxicity. The primary endpoint was progression-free survival (PFS) at 16 weeks. Secondary endpoints included overall survival (OS), overall response rate, safety, pharmacokinetics, pain score, and quality of life.
resultsFifteen patients were enrolled. Due to intolerable toxicity at higher doses, most patients received 3 g/day of mycophenolate mofetil. At 16 weeks, the PFS rate was 40%, with only one patient (6.7%) achieving stable disease. Median PFS and OS were 70 and 301 days, respectively. The most common grade 3-4 adverse events were anemia and fatigue, each occurring in 20% of patients. Pharmacokinetic analysis revealed high interindividual variability.
conclusionAlthough mycophenolate mofetil at 3 g/day was well tolerated, it did not meet expectations for anticancer efficacy in patients with high-grade locally advanced or metastatic osteosarcoma. This study should be considered a negative trial. Further research is needed to explore the therapeutic role of mycophenolate mofetil through alternative strategies or identify more effective interventions for osteosarcoma.
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