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Drug Profile

Pexidartinib for the treatment of adult symptomatic patients with tenosynovial giant cell tumors

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Pages 571-576 | Received 27 Mar 2020, Accepted 15 May 2020, Published online: 01 Jun 2020

References

  • Martin RC, Osborne DL, Edwards MJ, et al. Giant cell tumour of tendon sheath, tenosynovial giant cell tumour and pigmented villonodular synovitis: defining the presentation, surgical therapy and recurrence. Oncol Rep. 2000;7:413–419.
  • Mendenhall WM, Mendenhall CM, Reith JD, et al. Pigmented villonodular synovitis. Am J Clin Oncol. 2006;29:548–550.
  • De Saint Aubain Somerhausen N, van de Rijn M, et al. Tenosynovial giant cell tumour: localized type and diffuse type. In: Fletcher CDM, Hogendoorn PCW, Mertens F, editors. World Health Organization classification of tumours of soft tissue and bone. 4th ed. Lyon: International Agency for Research on Cancer; 2013. p100–103.
  • Yoon HJ, Cho YA, Lee JI, et al. Malignant pigmented villonodular synovitis of the temporomandibular joint with lung metastasis: a case report and review of the literature. Oral Surg Oral Med Oral Pathol Oral Radiol Endod. 2011;111:e30–6.
  • Righi A, Gambarotti M, Sbaraglia M, et al. Metastasizing tenosynovial giant cell tumour, diffuse type/pigmented villonodular synovitis. Clin Sarcoma Res. 2015;5:5–15.
  • West RB, Rubin BP, Miller MA, et al. A landscape effect in tenosynovial giant-cell tumour from activation of CSF1 expression by a translocation in a minority of tumour cells. Proc Natl Acad Sci USA. 2006;103:690–695.
  • Cupp JS, Miller MA, Montgomery KD, et al. Translocation and expression of CSF1 in pigmented villonodular synovitis, tenosynovial giant cell tumour, rheumatoid arthritis and other reactive synovitis. Am J Surg Pathol. 2007;31:970–976.
  • Tsuda Y, Hirata M, Katayama K, et al. Massively parallel sequencing of tenosynovial giant cell tumors reveals novel CSF1 fusion transcripts and novel somatic CBL mutations. Int J Cancer. 2019 Dec 15;145(12):3276–3284.
  • Brahmi M, Alberti L, Tirode F, et al. Complete response to CSF1R inhibitor in a translocation variant of teno-synovial giant cell tumor without genomic alteration of the CSF1 gene. Ann Oncol. 2018 Jun 1;29(6):1488–1489.
  • Xie G, Jiang N, Liang C, et al. Pigmented villonodular synovitis: a retrospective multicenter study of 237 cases. PLoS One. 2015;10:e0121451.
  • Palmerini E, Staals EL, Maki RG, et al. Tenosynovial giant cell tumour/pigmented villonodular synovitis: outcome of 294 patients before the era of kinase inhibitors. Eur J Cancer. 2015;51:210–217.
  • Dewar AL, Cambareri AC, Zannettino ACW, et al. Macrophage colony-stimulating factor receptor c-FSM is a novel target of imatinib. Blood. 2005;105:3127–3132.
  • Blay JY, El Sayadi H, Thiesse P, et al. Complete response to imatinib in relapsing pigmented villonodular sinoviti/tenosynovial giant cell tumour (PVNS/TGCT). Ann Oncol. 2008;19:821–822.
  • Cassier PA, Gelderblom H, Stacchiotti S, et al. Efficacy of imatinib mesylate for the treatment of locally advanced and/or metastatic tenosynovial giant cell tumour/pigmented villonodular synovitis. Cancer. 2012;118:1649–1655.
  • Verspoor FGM, Mastboom MJL, Hannink G, et al. Long-term efficacy of imatinib mesylate in patients with advanced tenosynovial giant cell tumor. Sci Rep. 2019 Oct 10;9(1):14551.
  • Brownlow N, Russel AE, Saravanapavan H, et al. Comparison of nilotinib and imatinib inhibition of FMS receptor signalling macrophage production and osteoclastogenesis. Leukemia. 2008;22:649–652.
  • Gelderblom H, Cropet C, Chevreau C, et al. Nilotinib in locally advanced pigmented villonodular synovitis: a multicentre, open-label, single-arm, phase 2 trial. Lancet Oncol. 2018 May;19(5):639–648.
  • Stacchiotti S, Crippa F, Messina A, et al. Response to imatinib in villonodular pigmented synovitis (PVNS) resistant to nilotinib. Clin Sarcoma Res. 2013 May 13;3(1):8.
  • Cassier PA, Italiano A, Gomez-Roca CA, et al. CSF1R inhibition with emactuzumab in locally advanced diffuse-type tenosynovial giant cell tumours of the soft tissue: a dose-escalation and dose-expansion phase I study. Lancet Oncol. 2015;16:949–956.
  • Sankhala KK, Blay JY, Ganjoo KN, et al. A phase I/II dose escalation and expansion study of cabiralizumab (FPA-008), an anti-CSF1R antibody, in tenosynovial giant cell tumor (TGCT, diffuse pigmented villonodular synovitis D-PVNS). J Clin Oncol. 2017;35(suppl; abstr 11078). DOI:10.1200/JCO.2017.35.15_suppl.11078.
  • Walter M, Lucet IS, Patel O, et al. The 2.7 A crystal structure of the autoinhibited human c-Fms kinase domain. J Mol Biol. 2007;367:839–847.
  • Tap WD, Wainberg ZA, Anthony SP, et al. Structure-guided blockade of CSF1R kinase in tenosynovial giant cell tumour. N Engl J Med. 2015;373:428–437.
  • Ao JY, Zhu XD, Chai ZT, et al. Colony-stimulating factor 1 receptor blockade inhibits tumour growth by altering the polarization of tumour-associated macrophages in hepatocellular carcinoma. Mol Cancer Ther. 2017;16(8):1544–1554.
  • Vandell AG, Duchin KL, Desai M, et al. Effect of dose strength, food and pH modifiers in the pharmacokinetics of the multikinase inhibitor pexidartinib. Clin Pharmacol Ther. 2018;103(Suppl S1):S54.
  • Yan D, Kowal J, Akkari L, et al. Inhibition of colony stimulating factor-1 receptor abrogates microenvironment-mediated therapeutic resistance in gliomas. Oncogene. 2017;36(43):6049–6058.
  • Butowski N, Colman H, De Groot JF, et al. Orally administered colony stimulating factor 1 receptor inhibitor PLX3397 in recurrent glioblastoma: an Ivy foundation early phase clinical trials consortium phase II study. Neuro Oncol. 2016;18(4):557–564.
  • Colman H, Raizer JJ, Walbert T, et al. Phase 1b/2 study of pexidartinib (PEX) in combination with radiation therapy (XRT) and temozolomide (TMZ) in newly diagnosed glioblastoma. J Clin Oncol. 2018;36(Suppl 15):2015.
  • Ostergaard M, Peterfy C, Conghan P, et al. OMERACT rheumatoid arthritis magnetic resonance imaging studies: core set of MRI acquisitions, joint pathology definitions and the OMERACT RA-MRI scoring system. J Rheumatol. 2003;30:1385–1386.
  • Peterfy CG, Guermazi A, Zaim S, et al. Whole-Organ Magnetic Resonance Imaging Score (WORMS) of the knee in osteoarthritis. Osteoarthritis Cartilage. 2004;12:177–190.
  • Tap WD, Gelderblom H, Palmerini E, et al. Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial. Lancet. 2019 Aug 10;394(10197):478–487.
  • Gelderblom H, Tap WD, Palmerini E, et al. Pexidartinib for advanced tenosynovial giant cell tumour (TGCT): long-term efficacy and safety from the phase 3 ENLIVEN and phase 1 PLX108-01 (TGCT cohort) studies. Abs n. 11042. J Clin Oncol. 2019;37(Suppl15):11042.
  • Healeay JH, Gelderblom H, Wagner AJ et al. Pexidartinib for locally advanced tenosynovial giant cell tumour: overall long-term pooled efficacy and safety with characterization of hepatic adverse reactions from ENLIVEN and other studies. Abstract 3208650 CTOS 2019 Annual meeting, Tokyo, Japan 2019.
  • Cannarile MA, Weisser M, Jacob W, et al. Colony-stimulating factor 1 receptor (CSF1R) inhibitors in cancer therapy. J Immunother Cancer. 2017;5:53.
  • Radi ZA, Koza-Taylor PH, Bell RR, et al. Increased serum enzyme levels associated with Kupffer cell reduction with no signs of hepatic or skeletal muscle injury. Am J Pathol. 2011;179:240–247.
  • Lamb YN. Pexidartinib: first approval. Drugs. 2019 Nov;79(16):1805–1812.

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