Table 1.
Systemic therapies for giant-cell tumor of bone (GCTB)
| Therapy | Type of study | Patients, n | Outcome |
|---|---|---|---|
| Sunitinib28 | Phase II | 1 | 68+ weeks on study SD |
| Bisphosphonates21 | Retrospective | 25 | Confounded by use of CT, XRT, IFNα, Emb In vitro inhibition of GCTB-derived osteoclast resorption |
| Bisphosphonates29 | Retrospective, case-controlled, after curettage | 44 | One of 24 developed recurrence in the bisphosphonate group; six of 20 in the control group |
| Zoledronic acid30 | Phase II, after intralesional curettage | 24 | 82% RFS at 60 months with zoledronic acid |
| Calcitonin | Retrospective, calcitonin after curettage | 25 | 52% had local recurrences |
| Denosumab23,26,31 | Phase I | 37 | 20 of 20 patients with histologic response |
| Ten of 15 with radiologic response | |||
| Five of 27 had grade 3–5 AEs | |||
| Phase II | 282 | 163 of 169 had no disease progression in cohort 1 (not a surgical candidate) | |
| 72% had objective tumor response | |||
| Three patients with ONJ | |||
| 9% SAEs reported | |||
| Phase II | 271 | Most patients had improvement in pain within 2 months |
Abbreviations: SD, stable disease; CT, computed tomography; XRT, radiation therapy; IFN, interferon; Emb, embolization; RFS, recurrence-free survival; AEs, adverse events; ONJ, osteonecrosis of the jaw; SAEs, serious AEs.