Tenosynovial giant cell tumor

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Elizabeth J. Davis, MD
Vanderbilt University
Nashville, TN

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Note: these are regimens tested in subtype-specific populations, please see the main soft tissue sarcoma page for other regimens.

1 regimens on this page
1 variants on this page


All lines of therapy

Pexidartinib monotherapy

Regimen

Study Years of enrollment Evidence Comparator Comparative Efficacy
Tap et al. 2019 (ENLIVEN) 2015-2016 Phase 3 (E-RT-esc) Placebo1 Superior ORR

1Patents assigned to placebo were offered open-label pexidartinib after week 24.

Targeted therapy

  • Pexidartinib (Turalio) as follows:
    • Cycle 1: 400 mg PO every morning and 600 mg PO every evening
    • Cycle 2 onwards: 400 mg PO twice per day

14-day cycles

References

  1. ENLIVEN: Tap WD, Gelderblom H, Palmerini E, Desai J, Bauer S, Blay JY, Alcindor T, Ganjoo K, Martín-Broto J, Ryan CW, Thomas DM, Peterfy C, Healey JH, van de Sande M, Gelhorn HL, Shuster DE, Wang Q, Yver A, Hsu HH, Lin PS, Tong-Starksen S, Stacchiotti S, Wagner AJ; ENLIVEN investigators. Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial. Lancet. 2019 Aug 10;394(10197):478-487. Epub 2019 Jun 19. link to original article link to PMC article contains dosing details in manuscript PubMed NCT02371369