BACKGROUND: Tenosynovial giant cell tumour (TGCT) is a locally aggressive neoplasm for which few systemic treatment options exist. This study evaluated the efficacy and safety of vimseltinib, an oral, switch-control, CSF1R inhibitor, in patients with symptomatic TGCT not amenable to surgery.
METHODS: MOTION is a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial done in 35 specialised hospitals in 13 countries. Eligible patients were adults (aged ≥18 years) with a histologically confirmed diagnosis of TGCT for which surgical resection could potentially worsen functional limitation or cause severe morbidity. Patients were randomly assigned (2:1) with interactive response technology to vimseltinib (30 mg orally twice weekly) or placebo, administrated in 28-day cycles for 24 weeks. Patients and site personnel were masked to treatment assignment until week 25, unless progressive disease was confirmed earlier. The primary endpoint was objective response rate by independent radiological review using Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST) at week 25 in the intention-to-treat population. Safety was assessed in all patients who received the study drug. The trial is registered with ClinicalTrials.gov, NCT05059262, and enrolment is complete.
RESULTS: Between Jan 21, 2022, and Feb 21, 2023, 123 patients were randomly assigned (83 to vimseltinib and 40 to placebo). 73 (59%) patients were female and 50 (41%) were male. Nine (11%) of 83 patients assigned to vimseltinib and five (13%) of 40 patients assigned to placebo discontinued treatment before week 25; one patient in the placebo group did not receive any study drug. Objective response rate per RECIST was 40% (33 of 83 patients) in the vimseltinib group vs 0% (none of 40) in the placebo group (difference 40% [95% CI 29-51]; p<0·0001). Most treatment-emergent adverse events (TEAEs) were grade 1 or 2; the only grade 3 or 4 TEAE that occurred in more than 5% of patients receiving vimseltinib was increased blood creatine phosphokinase (eight [10%] of 83). One patient in the vimseltinib group had a treatment-related serious TEAE of subcutaneous abscess. No evidence of cholestatic hepatotoxicity or drug-induced liver injury was noted.
CONCLUSIONS: Vimseltinib produced a significant objective response rate and clinically meaningful functional and symptomatic improvement in patients with TGCT, providing an effective treatment option for these patients.
BACKGROUND: Deciphera Pharmaceuticals.

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Tenosynovial giant cell tumor is a benign neoplasm arising from the synovium of joints, including the temporomandibular joint (TMJ). Despite its benign nature, these tumors may exhibit aggressive behavior. A 57-year-old woman with a swollen, hardened area in the left TMJ was referred to the university´s clinic. The diagnosis of tenosynovial giant cell tumor was made based on the presence of hyperplastic synovial lining containing mononuclear and giant cells, hemorrhagic areas, hemosiderin deposits, and calcification foci in the biopsy. A low condylectomy was performed, and histopathologic analysis of the surgical piece upheld the diagnosis. Due to histopathologic resemblance with other giant cell-rich lesions (giant cell granuloma of the jaws, brown tumor of hyperparathyroidism, and non-ossifying fibroma) for which signature mutations are known, mutational analysis of KRAS, FGFR1, and TRPV4 genes was conducted. The results revealed wild-type sequences for all the mutations tested, thereby supporting the diagnosis of tenosynovial giant cell tumor.

BACKGROUND: Magnetic Resonance-guided Focused Ultrasound (MRgFUS) treatment for certain anatomy locations can be extremely challenging due to patient positioning and potential motion. This present study describes the treatment of a recurrent tenosynovial giant cell tumor of the plantar forefoot using the ExAblate 2100 system in combination with patient immobilization device.
METHODS: Prior to the treatment, several patient immobilization devices were investigated. Vacuum cushions were selected and tested for safety and compatibility with the treatment task and the MR environment.
RESULTS: During the treatment, one vacuum cushion immobilized the patient\'s right leg in knee flexion and allowed the bottom of the foot to be securely positioned on the treatment window. Another vacuum cushion supported the patient upper body extended outside the scanner bore. 19 sonications were successfully executed. The treatment was judged to be successful. No immediate complications were observed.
CONCLUSIONS: MRgFUS treatment of a recurrent tenosynovial giant cell tumor of the right plantar forefoot was successful with the use of patient immobilization vacuum cushions.
CONCLUSIONS: The immobilization system could be utilized to aid future MRgFUS treatment of lesions in challenging anatomic locations. Various sizes of the vacuum cushions are available to potentially better accommodate other body parts and treatment configurations.

Aim: Real-world treatment patterns in tenosynovial giant cell tumor (TGCT) patients remain unknown. Pexidartinib is the only US FDA-approved treatment for TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Objective: To characterize drug utilization and treatment patterns in TGCT patients. Methods: In a retrospective observational study using IQVIA\'s linked prescription and medical claims databases (2018-2021), TGCT patients were stratified by their earliest systemic therapy claim (pexidartinib [N = 82] or non-FDA-approved systemic therapy [N = 263]). Results: TGCT patients treated with pexidartinib versus non-FDA-approved systemic therapies were predominantly female (61 vs 50.6%) and their median age was 47 and 54 years, respectively. Pexidartinib-treated patients had the highest 12-month probability of remaining on treatment (54%); 34.1% of pexidartinib users had dose reduction after their first claim. Conclusion: This study provides new insights into the unmet need, utilization and treatment patterns of systemic therapies for the treatment of TGCT patients.
Treatment patterns in patients with tenosynovial giant cell tumors in the USAThis database study is the first investigation of how drugs are used to treat patients with tenosynovial giant cell tumor (TGCT) in the real world. We researched adult TGCT patients from IQVIA\'s prescription and medical claims databases who started treatment with pexidartinib (N = 82) or other non-US FDA-approved systemic therapies (N = 263). The patients included in this analysis were mostly women (61.0 and 50.6%) and their median age was 47 and 54 years for pexidartinib and other non-FDA-approved systemic therapies, respectively. The patients treated with pexidartinib were most likely to remain on treatment (54.0%) at the end of the first year. Most patients (79.3%) started pexidartinib treatment at a total daily dose of 800 mg/day, as per the product label. Only 34.1% of patients had reduced medication dose during follow-up. Of note, this study found that TGCT patients were treated with other systemic therapies which remain unproven to be safe and effective in medical studies of TGCT. Given the unmet need, and with pexidartinib being the only approved systemic treatment in USA, there is an opportunity for the larger population of adult TGCT patients to benefit from its use. Further research is needed to identify barriers for access to pexidartinib and treatment of TGCT patients.

OBJECTIVE: A tenosynovial giant cell tumor (TGCT) is a proliferative lesion of the synovial membrane of the joints, tendon sheaths and/or bursae. There are two described subtypes, including the localized and diffuse forms. A TGCT can also be intraarticular or extraarticular. An intraarticular localized tenosynovial giant cell tumor (L-TGCT) of the knee is characterized by nodular hyperplasic synovial tissue that can remain asymptomatic for a long time, but as the mass grows, it may cause mechanical symptoms that may require surgical treatment. The aim of our study is to present a rare case of an L-TGCT of the knee joint treated with an arthroscopic excision.
METHODS: We describe the case of a 17-year-old female with pain, swelling and knee locking in the absence of trauma. The magnetic resonance imaging (MRI) displayed a well-circumscribed small mass in the anterior medial compartment, adherent to the infrapatellar fat pad. The lesion presented the typical MRI characteristics of an intraarticular localized TGCT. The patient was treated with an arthroscopic mass removal and partial synovectomy. The gross pathology showed an ovoid nodule that was covered by a fibrous capsule; a histopathology examination confirmed the diagnosis. The patient was able to return to normal daily activities one month after surgery; at the three-year follow-up, she was free of symptoms with no evidence of disease on the MRI.
CONCLUSIONS: In patients with a small-dimension L-TGCT in the anterior compartment of the knee that presents an MRI pattern and causes mechanical symptoms, an arthroscopic en-bloc excision can be performed that results in good outcomes and a rapid return to preinjury levels.

BACKGROUND: Diffuse-type tenosynovial giant cell tumor (D-TGCT) is a mono-articular, soft-tissue tumor. Although it can behave locally aggressively, D-TGCT is a non-malignant disease. This is the first study describing the natural course of D-TGCT and evaluating active surveillance as possible treatment strategy.
METHODS: This retrospective, multicenter study included therapy naïve patients with D-TGCT from eight sarcoma centers worldwide between 2000 and 2019. Patients initially managed by active surveillance following their first consultation were eligible. Data regarding the radiological and clinical course and subsequent treatments were collected.
RESULTS: Sixty-one patients with primary D-TGCT were initially managed by active surveillance. Fifty-nine patients had an MRI performed around first consultation: D-TGCT was located intra-articular in most patients (n = 56; 95 %) and extra-articular in 14 cases (24 %). At baseline, osteoarthritis was observed in 13 patients (22 %) on MRI. Most of the patients\' reported symptoms: pain (n = 43; 70 %), swelling (n = 33; 54 %). Eight patients (13 %) were asymptomatic. Follow-up data were available for 58 patients; the median follow-up was 28 months. Twenty-one patients (36 %) had radiological progression after 21 months (median). Eight of 45 patients (18 %) without osteoarthritis at baseline developed osteoarthritis during follow-up. Thirty-seven patients (64 %) did not clinically deteriorate during follow-up. Finally, eighteen patients (31 %) required a subsequent treatment.
CONCLUSIONS: Active surveillance can be considered adequate for selected therapy naïve D-TGCT patients. Although follow-up data was limited, almost two-thirds of the patients remained progression-free, and 69 % did not need treatment during the follow-up period. However, one-fifth of patients developed secondary osteoarthritis. Prospective studies on active surveillance are warranted.

OBJECTIVE: The recurrence rate following the excision of tenosynovial giant cell tumors (TSGCT) of the hand is very high. Intraoperative application of a surgical microscope has been reported. However, to date, there are no reports of medium-term outcomes related to this technique. This study aimed to evaluate the medium-term outcomes of tumor excision using surgical microscope for TSGCT of the hand.
METHODS: A total of 27 patients, who underwent an initial surgery for histologically-confirmed TSGCT of the hand, between 2008 and 2020, were included and evaluated. The mean follow-up time postoperatively was 6.8 years. Tumor recurrence and preoperative tumor characteristics were assessed.
RESULTS: All tumors were adherent to tendons, tendon sheaths, neurovascular structures or periarticular ligaments and capsules. Bony lesions were observed in 11 tumors. The surgical microscope was used in 13 tumors. Recurrences were observed in three tumors (overall recurrence rate: 11%). Tumor characteristics were similar in both groups, but the recurrence rate in the group treated using the surgical microscope was 0%, whereas the recurrence rate in the group treated without the surgical microscope was 21%. Re-operations using the surgical microscope for recurrent tumors were performed, without recurrence postoperatively.
CONCLUSIONS: Among patients with TSGCT of the hand treated with tumor excision using the surgical microscope, the postoperative recurrence rate was 0%. Based on the results of this study, the surgical microscope might be used for excision of TSGCTs of the hand.

A middle-aged woman presented to our hospital with a chief complaint of a mass on the left shoulder for 1 year. The initial lump was small with no pain or tenderness, and the patient had not sought medical attention for numbness in the left shoulder. Clinical examination showed a mass on the left shoulder measuring 11 × 8 × 3 cm approximately with no apparent skin damage or ecchymosis. No limitations in left shoulder joint movements were observed, and the patient exhibited normal movement of the left elbow joint, wrist joint, and metacarpophalangeal joint. Moreover, the left radial artery was palpable.

Tenosynovial giant cell tumor is a benign condition that originates from synovial cells within joints, tendon sheaths, or bursae and may present either in localized (benign) or diffuse (locally aggressive) forms. Currently, the primary treatment approach is surgical, yielding satisfactory results with low recurrence rates in the localized forms, whereas the diffuse type displays high recurrence rates. In parallel, clinical trials are underway to explore pharmaceutical treatment options for the advanced diffuse type. This article aims at consolidating current knowledge about diagnosis and management of this rare tumor, additionally proposing a brief overview of novel therapeutic approaches.
La tumeur à cellules géantes ténosynoviale, bénigne, prend son origine dans les cellules synoviales des articulations, des gaines tendineuses ou des bourses et se présente dans une forme soit localisée (bénigne), soit diffuse (localement agressive). Le traitement principal est chirurgical, offrant des résultats satisfaisants à long terme, avec un faible risque de récidive dans la forme localisée, alors que le taux de récidives est élevé dans la forme diffuse. Parallèlement, des essais cliniques sont en cours pour explorer des options de traitement systémique pour les formes diffuses sévères. Cet article rappelle les connaissances actuelles pour le diagnostic et la prise en charge de cette tumeur rare. De plus, nous proposons un aperçu succinct des nouvelles approches thérapeutiques.