ArticleClinical cancer research : an official journal of the American Association for Cancer Research2023
Regorafenib plus Vincristine and Irinotecan in Pediatric Patients with Recurrent/Refractory Solid Tumors: An Innovative Therapy for Children with Cancer Study.
Article in Clinical cancer research : an official journal of the American Association for Cancer Research, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02085148 (A Multi-center, Open-label, Non-randomized, Phase I Dose Escalation Study of Regorafenib), which is not on this map. Cited by 19 papers, 4 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
A Multi-center, Open-label, Non-randomized, Phase I Dose Escalation Study of Regorafenib (BAY 73-4506) in Pediatric Subjects With Solid Malignant Tumors That Are Recurrent or Refractory to Standard Therapy
Who cites it
19 citing papers in PubMed, 4 syntheses or guidelines pooled it, 29 citations in OpenAlex.
- Relapsed rhabdomyosarcoma: treatment recommendations from the European pediatric soft tissue sarcoma study group (EpSSG).British journal of cancer · 2025Guideline
- Tyrosine kinase inhibitors in Ewing's sarcoma: a systematic review.BMC cancer · 2025Pooled it
- Efficacy and safety of regorafenib in the treatment of bone sarcomas: systematic review and meta-analysis.BMC cancer · 2025Pooled it
- Efficacy and safety of anlotinib for the treatment of advanced bone and soft tissue sarcomas: a systematic review and meta-analysis.Frontiers in oncology · 2025Pooled it
- Tumor microenvironment-rearranging Nanoassemblies overcome angiogenesis-immunotherapy resistance to potentiate osteosarcoma treatment.Materials today. Bio · 2026Article
- NUDT1 drives osteosarcoma progression and serves as a robust predictor of clinical outcomes.Cancer cell international · 2026Article
- Targeted therapies in pediatric oncology: A start.Molecular therapy. Oncology · 2026Review
- Exploring drug sensitivity guided individualized treatment in pediatric spindle cell/sclerosing rhabdomyosarcoma.Frontiers in pharmacology · 2026Article
- Gemcitabine-docetaxel therapy in pediatric patients with relapsed or refractory sarcoma: a single-center experience.Frontiers in pediatrics · 2026Article
- Advances in Targeting Growth Factor Signalling in Neuroblastoma and Overcoming Drug Resistance.Cells · 2025Review
- Towards directed therapy for fusion-positive rhabdomyosarcoma.Pharmacology & therapeutics · 2025Review
- Nano-drug codelivery system of vincristine and gemcitabine loaded hyaluronan-altered hollow mesoporous silica nanoparticles: investigation of in vitro drug release and anticancer activity in pancreatic cancer cells.Naunyn-Schmiedeberg's archives of pharmacology · 2025Article
- Review
- Ewing Sarcoma, Desmoplastic Small Round Cell Tumor, and Other Round Cell Sarcomas.Hematology/oncology clinics of North America · 2025Review
- Proteomic Characterization of Liver Cancer Cells Treated with Clinical Targeted Drugs for Hepatocellular Carcinoma.Biomedicines · 2025Article
- A dual readout embryonic zebrafish xenograft model of rhabdomyosarcoma to assess clinically relevant multi-receptor tyrosine kinase inhibitors.Frontiers in oncology · 2025Article
- Druggable upregulated proteins in EWS-FLI-driven Ewing sarcoma as emerging new therapeutic targets.American journal of translational research · 2025Review
- Developmental origins shape the paediatric cancer genome.Nature reviews. Cancer · 2024Review
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors at 12 institutions in 6 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeThis phase Ib study defined the safety, MTD, and recommended phase II dose (RP2D) of regorafenib combined with vincristine and irinotecan (VI). Secondary objectives were evaluation of antitumor activity and pharmacokinetics (PK) of regorafenib and irinotecan. PATIENTS AND
methodsPatients aged 6 months to <18 years with relapsed/refractory solid malignancies [≥50% with rhabdomyosarcoma (RMS)] received regorafenib (starting dose 72 mg/m2/day) concomitantly or sequentially with vincristine 1.5 mg/m2 on days 1 and 8, and irinotecan 50 mg/m2 on days 1-5 (21-day cycle). Adverse events (AE) and tumor response were assessed. PK (regorafenib and irinotecan) were evaluated using a population PK model.
resultsWe enrolled 21 patients [median age, 10 years; 12, RMS; 5, Ewing sarcoma (EWS)]. The MTD/RP2D of regorafenib in the sequential schedule was 82 mg/m2. The concomitant dosing schedule was discontinued because of dose-limiting toxicities in 2 of 2 patients treated. Most common grade 3/4 (>30% of patients) AEs were neutropenia, anemia, thrombocytopenia, and leukopenia. The overall response rate was 48% and disease control rate [complete response (CR)/partial response/stable disease/non-CR/non-progressive disease] was 86%. Median progression-free survival was 7.0 months [95% confidence interval (CI), 2.9-14.8] and median overall survival was 8.7 months (95% CI, 5.5-16.3). When combined with VI, regorafenib PK was similar to single-agent PK in children and adults (treated with regorafenib 160 mg/day).
conclusionsRegorafenib can be combined sequentially with standard dose VI in pediatric patients with relapsed/refractory solid tumors with appropriate dose modifications. Clinical activity was observed in patients with RMS and EWS (ClinicalTrials.gov NCT02085148).
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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 Socratic graph.