Abstract
BackgroundTenosynovial giant cell tumor (TGCT) is a rare, locally aggressive neoplasm arising from the synovium of joints, bursae, and tendon sheaths affecting small and large joints. It represents a wide spectrum ranging from minimally symptomatic to massively debilitating. Most findings to date are mainly from small, retrospective case series, and thus the morbidity and actual impact of this rare disease remain to be elucidated. This study prospectively explores the management of TGCT in tertiary sarcoma centers.MethodsThe TGCT Observational Platform Project registry was a multinational, multicenter, prospective observational study involving 12 tertiary sarcoma centers in 7 European countries, and 2 US sites. This study enrolled for 2 years all consecutive ≥ 18 years old patients, with histologically diagnosed primary or recurrent cases of diffuse-type TGCT. Patient demographic and clinical characteristics were collected at baseline and every 6 months for 24 months. Quality of life questionnaires (PROMIS-PF and EQ-5D) were also administered at the same time-points. Here we report baseline patient characteristics.Results166 patients were enrolled between November 2016 and March 2019. Baseline characteristics were: mean age 44 years (mean age at disease onset: 39 years), 139/166 (83.7%) had prior treatment, 71/166 patients (42.8%) had ≥ 1 recurrence after treatment of their primary tumor, 76/136 (55.9%) visited a medical specialist ≥ 5 times, 66/116 (56.9%) missed work in the 24 months prior to baseline, and 17/166 (11.6%) changed employment status or retired prematurely due to disease burden. Prior treatment consisted of surgery (i.e., arthroscopic, open synovectomy) (128/166; 77.1%) and systemic treatments (52/166; 31.3%) with imatinib (19/52; 36.5%) or pexidartinib (27/52; 51.9%). Treatment strategies at baseline visits consisted mainly of watchful waiting (81/166; 48.8%), surgery (41/166; 24.7%), or targeted systemic therapy (37/166; 22.3%). Patients indicated for treatment reported more impairment compared to patients indicated for watchful waiting: worst stiffness NRS 5.16/3.44, worst pain NRS 6.13/5.03, PROMIS-PF 39.48/43.85, and EQ-5D VAS 66.54/71.85.ConclusionThis study confirms that diffuse-type TGCT can highly impact quality of life. A prospective observational registry in rare disease is feasible and can be a tool to collect curated-population reflective data in orphan diseases.Name of registry: Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project (TOPP).Trial registration number: NCT02948088.Date of registration: 10 October 2016.URL of Trial registry record: https://clinicaltrials.gov/ct2/show/NCT02948088?term=NCT02948088&draw=2.
Highlights
Tenosynovial giant cell tumor (TGCT) is a rare, locally aggressive mesenchymal neoplasm arising from the synovium of joints, bursae, and tendon sheaths and affects both small and large joints [1]
Muscle sarcomas are indicated to present a broad range of apparent diffusion coefficient (ADC), dependent on diffusion-weighted imaging (DWI) which is crucial regarding tumor composition, and distinguishing between malignant and benign lesions [10]
The institutional review board or ethics board provided approval in each center, and written informed consent was obtained from each patient who participated in this study
Summary
Tenosynovial giant cell tumor (TGCT) is a rare, locally aggressive mesenchymal neoplasm arising from the synovium of joints, bursae, and tendon sheaths and affects both small and large joints [1]. From the molecular point of view, both subtypes usually share the presence of a fusion involving the colony stimulating factor (CSF) gene, which drives tumor growth [3, 4] Both subtypes share a common pathophysiology, they represent a wide spectrum of clinical entities, making TGCT behavior complex and hard to predict [5]. Tenosynovial giant cell tumor (TGCT) is a rare, locally aggressive neoplasm arising from the synovium of joints, bursae, and tendon sheaths affecting small and large joints. It represents a wide spectrum ranging from minimally symptomatic to massively debilitating. This study prospectively explores the management of TGCT in tertiary sarcoma centers
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