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Use of neoadjuvant pexidartinib with limb salvage surgery for diffuse tenosynovial giant cell tumor: A case report

Published:November 16, 2022DOI:https://doi.org/10.1016/j.jos.2022.10.016
      Tenosynovial giant cell tumor is a neoplasm of synovium in which dysregulation of the colony-stimulating factor 1 (CSF1) axis attracts immune cells, particularly macrophages, leading to the formation of a mass [
      • Giustini N.
      • Bernthal N.M.
      • Bukata S.V.
      • Singh A.S.
      Tenosynovial giant cell tumor: case report of a patient effectively treated with pexidartinib (PLX3397) and review of the literature.
      ]. Clinically and radiographically, TGCT is divided into localized type – characterized by a well-circumscribed nodular appearance comprising 90% of cases – and diffuse type, characterized by an expansive villous proliferation within joints that can extend extra-articularly. Diffuse TGCT (dTGCT) is locally aggressive and frustrating to manage. Surgery is the mainstay of treatment but, while curative for localized disease, it is associated with failures above 50% for diffuse cases [
      • Mastboom M.J.L.
      • Palmerini E.
      • Verspoor F.G.M.
      • Rueten-Budde A.J.
      • Stacchiotti S.
      • Staals E.L.
      • et al.
      Surgical outcomes of patients with diffuse-type tenosynovial giant-cell tumours: an international, retrospective, cohort study.
      ]. Local recurrence of disease has been shown to be a risk factor for subsequent failures of operative treatment, and repeated surgical interventions contribute to overall morbidity, joint stiffness, and impaired quality of life [
      • Palmerini E.
      • Staals E.L.
      • Maki R.G.
      • Pengo S.
      • Cioffi A.
      • Gambarotti M.
      • et al.
      Tenosynovial giant cell tumour/pigmented villonodular synovitis: outcome of 294 patients before the era of kinase inhibitors.
      ,
      • Mastboom M.J.L.
      • Verspoor F.G.M.
      • Gelderblom H.
      • van de Sande M.A.J.
      Limb amputation after multiple treatments of tenosynovial giant cell tumour: series of 4 Dutch cases.
      ]. Because of this, multiple adjuvant therapies – including external beam radiotherapy, isotopic synoviorthesis, and cryotherapy – have been utilized attempting to limit local recurrences, but each have limited success and associated risks along with complicating future joint arthroplasty procedures [
      • Palmerini E.
      • Staals E.L.
      • Maki R.G.
      • Pengo S.
      • Cioffi A.
      • Gambarotti M.
      • et al.
      Tenosynovial giant cell tumour/pigmented villonodular synovitis: outcome of 294 patients before the era of kinase inhibitors.
      ,
      • Ottaviani S.
      • Ayral X.
      • Dougados M.
      • Gossec L.
      Pigmented villonodular synovitis: a retrospective single-center study of 122 cases and review of the literature.
      ].

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