Aims: GCTB is a primary osteolytic tumour characterised by local invasiveness and limited non-surgical treatment options. Osteoclast-like giant cells expressing surface RANK, and stromal cells expressing RANK ligand, are features of GCTB. We report sequential FDG-PET as a sensitive early indicator of GCTB response to denosumab from two phase 2 trials.
Methods: Adult or skeletally mature GCTB patients (N=360) received subcutaneous denosumab 120mg on days 1, 8 and 15 and Q4W thereafter. In some centres, combination FDG-PET and CT images were obtained sequentially as part of routine care. All evaluable scans were reviewed by central independent treatment-blinded reviewers.
Results: Paired pre- and post-treatment FDG-PET scans from 26 patients (144 evaluable scans) showed marked reductions in FDG-PET avidity and reductions in standardised uptake values (SUV) early in response to denosumab at the first post-treatment scan (median [Q1, Q3] 68.5 [50, 84] days from first dose). Results for all lesions (n=26 patients; 144 evaluable scans), lung (n=5; 49 evaluable scans), axial skeleton (n=11; 49 evaluable scans) and appendicular skeleton lesions (n=11; 46 evaluable scans) were: 9.8, 6.5, 9.2, and 10.0 for baseline median SUVmax; 2.7, 1.8, 2.7, 4.0 for best on-study median SUVmax; 1.8, 1.4, 1.7, and 2.1 for mean EORTC Standard Uptake; -64%, -64%, -64% and -66% for best SUVmax response (mean % change from baseline). All paired samples showed a best EORTC response of >25% reduction in SUV uptake following initiation of denosumab treatment. Sequential FDG-PET imaging studies revealed a sustained and progressive SUVmax reduction in GCTB lesions with continued denosumab treatment.
Conclusions: This is the largest GCTB FDG-PET imaging data set from an interventional clinical trial reported to date. FDG-PET is a sensitive early indicator of tumour response to denosumab, preceding both cytoreduction (RECIST) and new bone formation/calcification (HU density). Most importantly, denosumab-induced response was sustained for GCTB patients.