Abstract
CSF1R tyrosine kinase inhibitors (TKI) and antibodies yield response rates and tumor control in patients with diffuse type tenosynovial giant cell tumors (dTGCT). The long term management of patients with dTGCT treated with TKI is however not known. We conducted a retrospective single center study on the 39 patients with advanced and/or inoperable dTGCT referred to the Centre Leon Berard for a medical treatment. The clinical characteristics and treatments of patients who had received at least one line of CSF1R TKI or Ab was collected from the electronic patient records and analyzed, after this study was approved by the Institutional Review Board of the Centre Leon Berard. Statistics were conducted using SPSS 23.0. Thirty-nine patients received at least one line of TKI among the 101 patients with histologically confirmed dTGCT refered to this center. Imatinib, nilotinib, pexidartinib, emactuzumab were the most frequently used agents. First line treatment was given for a median duration of 7 months. With a median follow-up from the initiation of TKI of 30 months, the progression-free rate at 30 months is 56% for the 39 patients. 15 patients had recurrent disease after first line CSF1R inhibitor: 12 (80%) received a 2nd line treatment for a median duration of 6 months and a median time to progression (TTP) of 12 months. Six patients had afterwards a recurrent disease and 5 (83%) received a 3rd line treatment for a median duration of 5 months and a median TTP of 9 months. Progression-free rate at 30 months was observed in 3 of 12 (25%) after line 2 and 1 of 5 (20%) after line 3. None of the patients refered died with a median follow-up of 67 months. CSF1R TKI or Ab provide prolonged tumor control and symptom relief for a majority of patients with inoperable or relapsing dTGCT, in first and subsequent lines. Multiple lines are required for close to 50% of patients with relapsing dTGCT.
Highlights
Diffuse type tenosynovial giant cell tumors (TCGT) is a rare locally aggressive connective tissue tumor of the joints, affecting mostly young adults, with a predominance on the knee and ankle [1,2,3,4]. diffuse type tenosynovial giant cell tumors (dTGCT) frequently present at [1;2] translocation encoding for a fusion gene CSF1/ COL6A3 whose protein product plays a key role in tumor growth [5,6,7]
The clinical characteristics and treatments of patients who had received at least one line of CSF1R tyrosine kinase inhibitors (TKI) or Ab was collected from the electronic patient records and analyzed, after this study was approved by the Institutional Review Board of the Centre Leon Berard
Thirty-nine patients received at least one line of TKI among the 101 patients with histologically confirmed dTGCT refered to this center
Summary
Diffuse type tenosynovial giant cell tumors (TCGT) is a rare locally aggressive connective tissue tumor of the joints, affecting mostly young adults, with a predominance on the knee and ankle [1,2,3,4]. dTGCT frequently present at [1;2] translocation encoding for a fusion gene CSF1/ COL6A3 whose protein product plays a key role in tumor growth [5,6,7]. Diffuse type tenosynovial giant cell tumors (TCGT) is a rare locally aggressive connective tissue tumor of the joints, affecting mostly young adults, with a predominance on the knee and ankle [1,2,3,4]. Before CSF1R antagonists, either tyrosine kinase inhibitors (TKI) or antibodies, the medical treatments for relapsing and inoperable tumors had limited efficacy [1,2,3]. CSF1R antagonists have been reported to yield volumetric response and symptom relief in patients with inoperable diffuse type tenosynovial giant cell tumors (TCGT) [9,10,11,12,13,14,15,16]. The clinical efficacy of tyrosine kinase inhibitors blocking CSF1R (imatinib, nilotinib, pexidartinib) and antibodies against CSF1R (emactuzumab, cabiralizumab) has been confirmed in several retrospective clinical studies for imatinib [10,11], as well as prospective clinical trials, with emactuzumab [12], nilotinib [13], pexidatinib [14,15] and cabiralizumab [16]
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