TY - JOUR
T1 - Denosumab in Giant Cell Rich Tumors of Bone
T2 - An Open-Label Multicenter Phase II Study
AU - Lipplaa, Astrid
AU - Schreuder, Willem H.
AU - Pichardo, Sarina E.C.
AU - Gelderblom, Hans
N1 - Funding Information: This work was supported by Amgen (ISS 20159990). Funding Information: This study was sponsored by Leiden University Medical Center. The Medical Ethics Committee Leiden The Hague Delft provided Institutional Review Board approval. Publisher Copyright: © The Author(s) 2023. Published by Oxford University Press.
PY - 2023/11
Y1 - 2023/11
N2 - Background: Since giant cell tumors of bone (GCTB) and other giant cell rich tumors of bone (GCRTB) share the histological presence of osteoclastic giant cells and expression of RANK/RANKL, we hypothesized that GCRTB will respond similarly to denosumab as GCTB. The primary objective of this study was to determine the efficacy of denosumab in patients with GCRTB that have recurred or require morbid surgery. Methods: In this open-label, multicenter, phase II trial, patients with GCRTB were included (June 2018-March 2020). Recruitment was stopped because of low accrual. Patients received denosumab (120 mg) subcutaneously (SC) on day 1 of every 4-week cycle with a loading dose of 120 mg SC on days 8 and 15. Results: Three patients were enrolled. One withdrew consent before start of study. The remaining patients had central giant cell granuloma of the jawbone (CGCG). Median treatment duration was 15 cycles (range 12-18). In both subjects, improvement in ossification of lesions was seen. Median follow-up was 28.5 months (range 20-37). One patient developed a recurrence for which surgery was performed. Conclusion: Due to critical emerging real-world data of denosumab in GCRTBs, the study was prematurely stopped and not supportive of use of denosumab for this indication. (ClinicalTrials.gov Identifier: NCT03605199).
AB - Background: Since giant cell tumors of bone (GCTB) and other giant cell rich tumors of bone (GCRTB) share the histological presence of osteoclastic giant cells and expression of RANK/RANKL, we hypothesized that GCRTB will respond similarly to denosumab as GCTB. The primary objective of this study was to determine the efficacy of denosumab in patients with GCRTB that have recurred or require morbid surgery. Methods: In this open-label, multicenter, phase II trial, patients with GCRTB were included (June 2018-March 2020). Recruitment was stopped because of low accrual. Patients received denosumab (120 mg) subcutaneously (SC) on day 1 of every 4-week cycle with a loading dose of 120 mg SC on days 8 and 15. Results: Three patients were enrolled. One withdrew consent before start of study. The remaining patients had central giant cell granuloma of the jawbone (CGCG). Median treatment duration was 15 cycles (range 12-18). In both subjects, improvement in ossification of lesions was seen. Median follow-up was 28.5 months (range 20-37). One patient developed a recurrence for which surgery was performed. Conclusion: Due to critical emerging real-world data of denosumab in GCRTBs, the study was prematurely stopped and not supportive of use of denosumab for this indication. (ClinicalTrials.gov Identifier: NCT03605199).
KW - aneurysmal bone cyst
KW - denosumab
KW - giant cell granuloma
KW - giant cell rich tumor of bone
KW - systemic therapy
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U2 - https://doi.org/10.1093/oncolo/oyad196
DO - https://doi.org/10.1093/oncolo/oyad196
M3 - Article
C2 - 37449658
SN - 1083-7159
VL - 28
SP - 1005-1006, E1099-E1104
JO - oncologist
JF - oncologist
IS - 11
ER -