Tenosynovial giant cell tumor (TGCT) is a rare neoplasm predominantly occurring in young and middle-aged adults, characterized by high recurrence and high disability rates. Surgery represents the first-line treatment at present. Although surgical resection can eradicate lesions, repeated surgical interventions constitute the major disease-related burden. Particularly for patients with diffuse-type TGCT (D-TGCT), postoperative recurrence rates can exceed 50%.
Pimicotinib is a highly selective colony-stimulating factor 1 receptor (CSF1R) inhibitor. The phase III MANEUVER trial has verified its prominent tumor shrinkage effect and symptomatic improvement in patients with unresectable, symptomatic TGCT, with an objective response rate (ORR) of 54%. In addition, pimicotinib demonstrates a favorable safety profile; most adverse events are mild in severity, mainly including pruritus, edema, fatigue and elevated creatine kinase, without severe hepatotoxicity.
Neoadjuvant therapy followed by surgery falls within the scope of multimodal treatment, which is mostly applied to recurrent and refractory cases rather than the standard upfront regimen for treatment-naive patients. Clinical case reports and real-world observations have preliminarily validated the feasibility and clinical value of sequential systemic targeted therapy followed by surgical resection. To date, systematic and standardized clinical data regarding neoadjuvant strategies remain scarce, especially randomized controlled evidence comparing neoadjuvant targeted therapy plus surgery versus primary upfront surgery. This study aims to compare the efficacy and safety of neoadjuvant pimicotinib combined with surgery versus upfront primary surgery in the management of D-TGCT, so as to generate evidence-based rationale for developing more effective and safe therapeutic regimens for D-TGCT.
Male or female subjects aged ≥18 years
Histologically confirmed tenosynovial giant cell tumor (TGCT)
Resectable diffuse tenosynovial giant cell tumor (D-TGCT) were determined by multidisciplinary team (MDT) discussion.
Measurable disease as defined by Response Evaluation Criteria In Solid Tumors (RECIST) v1.1 with at least one lesion of ≥2 cm
Prior treatment with highly selective Colony-Stimulating Factor 1 (CSF1)/ Colony-Stimulating Factor 1 Receptor (CSF1R) inhibitors before randomization
Presence of another malignancy requiring active treatment, in the investigator's judgment, which may interfere with study participation or results
Known metastatic TGCT
Severe concomitant arthropathy, severe illness, or uncontrolled infection in the affected joint
Parallel
Pimicotinib
DrugPimicotinib 50 mg orally once daily for 12 consecutive weeks.
Tumor resection surgery
Procedure/SurgeryOpen surgery will be performed based on patient and tumor characteristics discussed at the multidisciplinary team (MDT) meeting. The specific surgical plan will be determined by the surgeon based on clinical judgment. Surgical procedures will follow national guidelines.
Event-free survival
Events are defined as disease progression precluding planned surgery, local recurrence, distant metastasis, treatment discontinuation due to adverse events, or death from any cause.
Local recurrence rate
Surgical complication rate
Change in range of motion
Change in Brief Pain Inventory (BPI)
Score range: 0-10; Higher scores mean more severe pain and greater life interference; lower scores mean pain relief.
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