Diffuse-type tenosynovial giant cell tumour: Current treatment concepts and future perspectives

Staals, Eric L.; Ferrari, Stefano; Donati, Davide María; Palmerini, Emanuela

doi:10.1016/j.ejca.2016.04.022

Cited by 100 publications

(112 citation statements)

References 39 publications

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“…9 Individual CSF1 (a-b) and adiponectin (c-d) plasma levels by time. C, cycle; CSF1, colony-stimulating factor 1; D, day disease is associated with pain, swelling, limitation of motion, and hemorrhagic joint effusions, which affect quality of life and are clinically important from the patient's perspective [13]. In other studies, treatment with pexidartinib in TGCT patients resulted in an ORR of 52% (12/23) (phase I) [1] and 39% (24/61) (phase III) [14].…”

Section: Discussionmentioning

confidence: 99%

A phase I study of pexidartinib, a colony-stimulating factor 1 receptor inhibitor, in Asian patients with advanced solid tumors

et al. 2019

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Background Pexidartinib, a novel, orally administered small-molecule tyrosine kinase inhibitor, has strong selectivity against colonystimulating factor 1 receptor. This phase I, nonrandomized, open-label multiple-dose study evaluated pexidartinib safety and efficacy in Asian patients with symptomatic, advanced solid tumors. Materials and Methods Patients received pexidartinib: cohort 1, 600 mg/d; cohort 2, 1000 mg/d for 2 weeks, then 800 mg/d. Primary objectives assessed pexidartinib safety and tolerability, and determined the recommended phase 2 dose; secondary objectives evaluated efficacy and pharmacokinetic profile. Results All 11 patients (6 males, 5 females; median age 64, range 23-82; cohort 1 n = 3; cohort 2 n = 8) experienced at least one treatment-emergent adverse event; 5 experienced at least one grade ≥ 3 adverse event, most commonly (18%) for each of the following: increased aspartate aminotransferase, blood alkaline phosphatase, gamma-glutamyl transferase, and anemia. Recommended phase 2 dose was 1000 mg/d for 2 weeks and 800 mg/d thereafter. Pexidartinib exposure, area under the plasma concentration-time curve from zero to 8 h (AUC 0-8h ), and maximum observed plasma concentration (C max ) increased on days 1 and 15 with increasing pexidartinib doses, and time at C max (T max ) was consistent throughout all doses. Pexidartinib exposure and plasma levels of adiponectin and colony-stimulating factor 1 increased following multiple daily pexidartinib administrations. One patient (13%) with tenosynovial giant cell tumor showed objective tumor response. Conclusions This was the first study to evaluate pexidartinib in Asian patients with advanced solid tumors. Pexidartinib was safe and tolerable in this population at the recommended phase 2 dose previously determined for Western patients (funded by Daiichi Sankyo; clinicaltrials.gov number, NCT02734433).

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Section: Discussionmentioning

confidence: 99%

A phase I study of pexidartinib, a colony-stimulating factor 1 receptor inhibitor, in Asian patients with advanced solid tumors

et al. 2019

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“…Due to the rapid relapse after discontinuation and severe side effects of targeted therapy, these treatments are only recommended for patients with surgical unresectable disease or multiple recurrences that produce clinical and functional impairment [12]. Other systemic treatments are individually available in trial settings [15]. …”

Section: Discussionmentioning

confidence: 99%

Cryosurgery as Additional Treatment in Tenosynovial Giant Cell Tumors

Verspoor

Scholte

Geest

et al. 2016

Sarcoma

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Introduction. Tenosynovial giant cell tumors (TGCT) emerge from the synovium and can behave aggressively. Surgical resection is the standard treatment. However, up to half of the patients with diffuse type show recurrences. Several additional treatments have been applied to reduce recurrences; none of these treatments was proven to be superior to surgical resection solely. This article describes the results of additional cryosurgery to surgical resection. Materials and Methods. We retrospectively evaluated 141 TGCT patients, between 1999 and 2007. Twelve patients had additional cryosurgery. The knee (n = 8), hip (n = 2), ankle (n = 1), and elbow (n = 1) were affected. Primary outcome variables were treatment indications, recurrences, and complications. Results. Indications for additional cryosurgery were extended disease, bone involvement, and locations that are difficult to surgically get disease-free such as cruciate ligaments. Five patients had recurrent disease, all of which had prior treatments. None of the primary treated patients had recurrent disease. One patient had a deep infection. Discussion. Cryosurgery may serve as an additional treatment for diffuse TCGT in selected cases. However, because of the small number of patients and the heterogeneous group we could not prove an advantage of additional cryosurgery over surgical resection only. Cryosurgery should be considered for further evaluation in a prospective study. If there is any effect it would be helpful, especially in patients with multiple TGCT recurrences.

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“…Yttrium synovectomy has been used in the adjuvant setting in diffuse intraarticular disease [166]. Due to a translocation involving the macrophage colony-stimulating factor ( M - CSF or CSF1 ) gene seen in a proportion of cells, imatinib has demonstrated activity in its treatment [167].…”

Section: Borderline Tumoursmentioning

confidence: 99%

UK guidelines for the management of soft tissue sarcomas

Dangoor¹,

et al. 2016

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Soft tissue sarcomas (STS) are rare tumours arising in mesenchymal tissues, and can occur almost anywhere in the body. Their rarity, and the heterogeneity of subtype and location means that developing evidence-based guidelines is complicated by the limitations of the data available. However, this makes it more important that STS are managed by teams, expert in such cases, to ensure consistent and optimal treatment, as well as recruitment to clinical trials, and the ongoing accumulation of further data and knowledge. The development of appropriate guidance, by an experienced panel referring to the evidence available, is therefore a useful foundation on which to build progress in the field. These guidelines are an update of the previous version published in 2010 (Grimer et al. in Sarcoma 2010:506182, 2010). The original guidelines were drawn up following a consensus meeting of UK sarcoma specialists convened under the auspices of the British Sarcoma Group (BSG) and were intended to provide a framework for the multidisciplinary care of patients with soft tissue sarcomas. This current version has been updated and amended with reference to other European and US guidance. There are specific recommendations for the management of selected subtypes of disease including retroperitoneal and uterine sarcomas, as well as aggressive fibromatosis (desmoid tumours) and other borderline tumours commonly managed by sarcoma services. An important aim in sarcoma management is early diagnosis and prompt referral. In the UK, any patient with a suspected soft tissue sarcoma should be referred to one of the specialist regional soft tissues sarcoma services, to be managed by a specialist sarcoma multidisciplinary team. Once the diagnosis has been confirmed using appropriate imaging, plus a biopsy, the main modality of management is usually surgical excision performed by a specialist surgeon. In tumours at higher risk of recurrence or metastasis pre- or post-operative radiotherapy should be considered. Systemic anti-cancer therapy (SACT) may be utilized in some cases where the histological subtype is considered more sensitive to systemic treatment. Regular follow-up is recommended to assess local control, development of metastatic disease, and any late-effects of treatment. For local recurrence, and more rarely in selected cases of metastatic disease, surgical resection would be considered. Treatment for metastases may include radiotherapy, or systemic therapy guided by the sarcoma subtype. In some cases, symptom control and palliative care support alone will be appropriate.

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Diffuse-type tenosynovial giant cell tumour: Current treatment concepts and future perspectives

Cited by 100 publications

References 39 publications

A phase I study of pexidartinib, a colony-stimulating factor 1 receptor inhibitor, in Asian patients with advanced solid tumors

A phase I study of pexidartinib, a colony-stimulating factor 1 receptor inhibitor, in Asian patients with advanced solid tumors

Cryosurgery as Additional Treatment in Tenosynovial Giant Cell Tumors

UK guidelines for the management of soft tissue sarcomas

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