A Real-world Study on Patients of Unresectable Giant Cell Tumor of Bone

Study Overview
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Brief Summary
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This is a retrospective observational real-world study, which evaluates the efficacy and safety of denosumab and non-denosumab therapies in the treatment of Chinese populations of surgically unsalvageable or severe post-surgery morbidity associated giant cell tumor of bone (GCTB), collectively referred to as unresectable GCTB, during 2013-2021 in three medical centers, serving as the external control for a single arm phase Ib/II trial on JMT103 treatment of GCTB. 301 patients were enrolled and divided into 2 groups according to their actual previous exposures. Group 1 (n=135) was denosumab group. Group 2 (n=166) included two types of exposures other than denosumab: other anti-GCTB drug therapies, or no therapy on GCTB patients. The dosage, route, frequency and other administration methods was collected according to the actual previous treatment records. The primary outcome measure was the tumor response rate [radiographic tumor response (CR/PR evaluated by ICDs or EORTC criteria) within 12 weeks, or at least 90% reduction of osteoclast like giant cells compared with baseline]. The key secondary endpoint was the tumor response rate [radiographic tumor response (CR/PR evaluated by ICDS or EORTC criteria), or at least 90% reduction of osteoclast like giant cells compared with baseline]. Other secondary Outcome Measures include: proportion of patients whose tumors was surgically resectable; median duration of tumor response (DOR), disease control rate (DCR), and time to disease progression (TTP); and types and proportion of key adverse reactions.

Official Title
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A Real-world Study on Patients of Surgically Unsalvageable or Severe Post-surgery Morbidity Associated Giant Cell Tumor of Bone

Conditions
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Giant Cell Tumor of Bone

Intervention / Treatment
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* Drug: Denosumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 1. Both genders, aged 18 years or above; 2. Patients pathologically diagnosed of giant cell tumor of bone (GCTB), and are surgically unsalvageable, or salvageable but surgery was associated with severe morbidity based on physician's judgement; 3. For patients received denosumab or other anti-GCTB medications, one of the two is required: imaging data (including X-ray, CT, MRI or PET-CT) prior to and at least once followed to administration, or pathological data on surgically resected GCTB; for patients with no medication, imaging data or pathological data on surgically resected GCTB is required. Exclusion Criteria: 1. No access to definite treatment information; 2. Bone metabolic diseases diagnosed before treatment: hypo-/ hyperparathyroidism, hypo-/hyperthyroidism, hypopituitarism, hyperprolactinemia, Cushing syndrome, acromegaly, Paget disease, etc; 3. Complication of malignant tumor receiving anti-tumor therapy.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Denosumab group<br>patients who had received denosumab | Drug: Denosumab<br>* Denosumab, subcutaneous injection, specific usage to see the medical records. Non-denosumab, specific usage to see the medical records.<br>* Other names: Non-denosumab;|
| Non-denosumab group<br>patients who had received anti-GCTB drug therapy other than denosumab, or who did not receive any anti-GCTB medication | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Tumor response rate | Radiographic tumor response (CR/PR evaluated by ICDs or EORTC criteria) within 12 weeks, or at least 90% reduction of osteoclast like giant cells compared with baseline | 12 weeks (radiology), or through study completion, an average of 1 year (histopathology) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Tumor response rate (Key secondary endpoint) | Radiographic tumor response (CR/PR evaluated by ICDs or EORTC criteria), or at least 90% reduction of osteoclast like giant cells compared with baseline | through study completion, an average of 1 year (radiology and histopathology) | 
| Proportion of patients that is surgically resectable |  | through study completion, an average of 1 year | 
| Median duration of tumor response (DOR) |  | through study completion, an average of 1 year | 
| Disease control rate (DCR) |  | through study completion, an average of 1 year | 
| Time to disease progression (TTP) |  | through study completion, an average of 1 year | 
| Types and proportion of key adverse reactions |  | through study completion, an average of 1 year |