Trial reportBritish journal of cancer2023
Regorafenib in patients with advanced Ewing sarcoma: results of a non-comparative, randomised, double-blind, placebo-controlled, multicentre Phase II study.
Trial report in British journal of cancer, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 4 of them syntheses that pooled it.
What it found
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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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 4 syntheses or guidelines pooled it, 28 citations in OpenAlex.
- Efficacy and safety comparison of small molecule anti-angiogenic drugs in the treatment of bone and soft tissue sarcomas : a network meta-analysis.BMC cancer · 2025Pooled it
- Tyrosine kinase inhibitors in Ewing's sarcoma: a systematic review.BMC cancer · 2025Pooled it
- Clinical practice guidelines for the treatment of Ewing sarcoma (Spanish Sarcoma Research Group-GEIS).Clinical & translational oncology : official publication of the Federation of Spanish Oncology Societies and of the National Cancer Institute of Mexico · 2025Guideline
- Efficacy and safety of regorafenib in the treatment of bone sarcomas: systematic review and meta-analysis.BMC cancer · 2025Pooled it
- Trabectedin and low-dose irinotecan to target EWS::FLI1 in Ewing sarcoma: a phase 1/2 trial.Nature medicine · 2026Trial
- Metastatic Ewing Sarcoma, Patterns of Care and Outcomes of Patients in a Real-Life National Setting Over a Decade.Cancer medicine · 2026Observational
- Sarcomas in Adolescents and Young Adults.Current oncology reports · 2026Review
- Multi-Targeted TKIs in Patients with Advanced Ewing Sarcoma: A Systematic Review and Single-Arm Meta-Analysis.Cancers · 2026Review
- Targeting developmental vulnerabilities in childhood sarcomas.Cancer metastasis reviews · 2025Review
- KC1036, a multi-kinase inhibitor with anti-angiogenic activity, can effectively suppress the tumor growth of Ewing sarcoma.Angiogenesis · 2025Article
- Small round cell sarcoma tumoroid biobank reveals CIC::DUX4 sarcoma vulnerability to MCL-1 inhibition.Nature communications · 2025Article
- Gene Fusions as Potential Therapeutic Targets in Soft Tissue Sarcomas.Biomolecules · 2025Review
- Decoding the immune landscape in Ewing sarcoma pathogenesis: The role of tumor infiltrating immune cells and immune milieu.Journal of bone oncology · 2025Review
- Review
- Clinical characterization and therapeutic targeting of fusion genes in oncology.Future oncology (London, England) · 2025Review
- Druggable upregulated proteins in EWS-FLI-driven Ewing sarcoma as emerging new therapeutic targets.American journal of translational research · 2025Review
- UK guidelines for the management of bone sarcomas.British journal of cancer · 2025Review
- A Systematic Review and Meta-Analysis of Regorafenib's Effectiveness and Safety in the Treatment of Bone Sarcoma.The archives of bone and joint surgery · 2025Review
- Role of tumor-derived exosomes and immune cells in osteosarcoma progression and targeted therapy.Frontiers in immunology · 2025Review
- Primary and Metastatic Pancreatic Ewing Sarcomas: A Case Report and Review of the Literature.Diagnostics (Basel, Switzerland) · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
20 authors at 15 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe REGOBONE multi-cohort study explored the efficacy and safety of regorafenib for patients with advanced bone sarcomas; this report details the Ewing sarcoma (ES) cohort.
methodsPatients with relapsed ES progressing despite prior standard therapy, were randomised (2:1) to receive regorafenib or placebo. Patients on placebo could crossover to receive regorafenib after centrally confirmed progression. The primary endpoint was the progression-free rate at 8 weeks. With one-sided α of 0.05, and 80% power, at least 14/24 progression-free patients at 8 weeks were needed for success.
resultsFrom September 2014 to November 2019, 41 patients were accrued. 36 patients were evaluable for efficacy: 23 on regorafenib and 13 on placebo. Thirteen patients (56%; one-sided 95% CI [37.5%-[)) were progression-free at 8 weeks on regorafenib vs. 1 (7.7%; 95% CI [0.4%-[) on placebo. Median PFS was 11.4 weeks on regorafenib, and 3.9 weeks on placebo. Ten placebo patients crossed over to receive regorafenib after progression. The most common grade ≥3 regorafenib-related adverse events were pain (22%), asthenia (17%), thrombocytopenia (13%) and diarrhoea (13%).
conclusionAlthough the primary endpoint was not met statistically in this randomised cohort, there is evidence to suggest that regorafenib might modestly delay tumour progression in relapsed ES after failure of prior chemotherapy.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.