The DOI matches its Crossref record; 13 of the 13 references it cites were found in a registry or an index.
Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial
Open accessAbstract
Background: Tenosynovial giant cell tumor (TGCT), a rare, locally aggressive neoplasm, overexpresses colony-stimulating factor 1 (CSF1). Surgery is standard with no approved systemic therapy. This phase 3 randomized, double-blind study evaluated pexidartinib, a CSF1 receptor inhibitor, in patients with TGCT. Methods: In part 1 of this two-part study, 120 patients with symptomatic, advanced TGCT for whom surgery was not recommended were randomized (1:1) to pexidartinib (n=61) or placebo (n=59). Part 2 was open-label pexidartinib for all patients. Primary endpoint was centrally reviewed overall response rate (week 25) by RECIST, version 1.1. Secondary endpoints included range of motion, response rate by tumor volume score (TVS), patient-reported outcomes (PROs), and response duration. Emergence of mixed and cholestatic hepatotoxicity caused the Data Monitoring Committee to stop enrollment six patients short of target. Findings: The overall response rate was higher for pexidartinib versus placebo at week 25 by RECIST (39 vs 0%; p<0·0001). At 22 months median follow-up, the best overall response with pexidartinib had increased to 53% by RECIST. Hair color changes, liver enzyme increase, fatigue, and dysgeusia were the most frequent pexidartinib-associated adverse events. Three patients had transaminases ≥3×ULN with total bilirubin and alkaline phosphatase ≥2×ULN indicative of mixed and cholestatic hepatotoxicity, one lasting 7 months and confirmed by biopsy. Interpretation: Pexidartinib is the first systemic therapy to demonstrate a robust tumor response in TGCT with improved patient symptoms and functional outcomes; mixed and cholestatic hepatotoxicity is an identified risk.
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Colony-stimulating factor 1 receptor (CSF1R) inhibitors in cancer therapy
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Structure-guided blockade of CSF1R kinase in tenosynovial giant-cell tumor
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Higher incidence rates than previously known in tenosynovial giant cell tumors
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Diffuse-type tenosynovial giant cell tumour: Current treatment concepts and future perspectives
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Self-citationPatient-reported Symptoms of Tenosynovial Giant Cell Tumors
Gelhorn, H. L., Tong-Starksen, S., McQuarrie, K., Vernon, C., Hanlon, J. T., Maclaine, G. D. H., Lenderking, W. R., Ye, X., Speck, R. M., Lackman, R. D., et al. (2016) · Clinical Therapeutics · doi:10.1016/j.clinthera.2016.03.008 · Cited by 122
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Self-citationHarnessing cytokines and chemokines for cancer therapy
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Immunotherapy: Reshape the Tumor Immune Microenvironment
LV, B., Wang, Y., Ma, D., Cheng, W., Liu, J., Yong, T., Chen, H., & Wang, C. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.844142 · Cited by 482
Tissue macrophages: origin, heterogenity, biological functions, diseases and therapeutic targets
Guan, F., Wang, R., Yi, Z., Luo, P., Liu, W., Xie, Y., Liu, Z., Xia, Z., Zhang, H., & Cheng, Q. (2025) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-025-02124-y · Cited by 371
Tumor microenvironment signaling and therapeutics in cancer progression
Goenka, A., Khan, F., Verma, B., Sinha, P., Dmello, C. C., Jogalekar, M. P., Gangadaran, P., & Ahn, B. (2023) · Cancer Communications · doi:10.1002/cac2.12416 · Cited by 323
Metadata
- Authors
- Tap, W. D., Gelderblom, H., Palmerini, E., Desai, J., Bauer, S., Blay, J.-Y., Alcindor, T., Ganjoo, K., Martín-Broto, J., Ryan, C. W., Thomas, D. M., Peterfy, C., Healey, J. H., van de Sande, M., Gelhorn, H. L., Shuster, D. E., Wang, Q., Yver, A., Hsu, H. H., Lin, P. S., Tong-Starksen, S., Stacchiotti, S., & Wagner, A. J.
- Venue
- The Lancet
- Publisher
- Elsevier BV
- Published
- 2019-08
- Volume
- 394
- Issue
- 10197
- Pages
- 478-487
- ISSN
- 0099-5355 (ISSN-L)
- 0140-6736
- OpenAlex
- W2951817901
- Cited by
- 482
- References
- 13
- FWCI
- 34.89
- Type
- journal article
- Language
- en
- Open access
- green
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- Tap, W. D., Gelderblom, H., Palmerini, E., Desai, J., Bauer, S., Blay, J.-Y., Alcindor, T., Ganjoo, K., Martín-Broto, J., Ryan, C. W., Thomas, D. M., Peterfy, C., Healey, J. H., van de Sande, M., Gelhorn, H. L., Shuster, D. E., Wang, Q., Yver, A., Hsu, H. H., . . . Wagner, A. J. (2019). Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial. The Lancet. https://doi.org/10.1016/s0140-6736(19)30764-0
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