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Denosumab: A Potential Treatment Option for Aneurysmal Bone Cysts

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Peking University People's Hospital

关键词

抽象

The efficacy of traditional therapeutic approaches for aneurysmal bone cysts (ABC), such as surgery, embolization, sclerotherapy and radiotherapy, are often compromised for lesions in axial skeletons and adolescents complicated with pathological fracture. Therefore, denosumab, a new drug that has been successfully used in giant cell tumor of bone but has seldom used in ABC, was used to treat ABC in this trial.

描述

Patients with primary ABC treated with perioperative denosumab in the Musculoskeletal Tumor Center of Peking University People's Hospital between January 2014 and December 2016 will be reported in this trial.Approved by the Ethics Committee of Peking University People's Hospital and with the full informed consent from the patients or their families, all patients received surgery and treatment with denosumab perioperatively. Denosumab 120 mg was given subcutaneously (abdomen wall or upper arms) every 4 weeks (Q4W), with a loading dose on both Day 8 and Day 15 of the first cycle. Meanwhile, all patients were suggested to take 800mg oral calcium supplement every day and avoid any dental or oral surgery (tooth extraction, tooth filling, etc.). Patients were followed once a month since the date of surgery. On follow-up visits, patient-reported adverse reactions, X-ray image of jawbone, serum calcium, alkaline phosphatase, renal and hepatic function, hemoglobin, white blood cell, and platelet count were collected, and histological examinations, radiological examinations (such as X-ray, computed tomography (CT), magnetic resonance image (MRI) and positron emission tomography-computed tomography (PET-CT) and postoperative functional status evaluations by MSTS(Musculoskeletal tumor society) score were conducted.

日期

最后验证: 12/31/2017
首次提交: 01/09/2018
提交的预估入学人数: 01/28/2018
首次发布: 01/29/2018
上次提交的更新: 01/28/2018
最近更新发布: 01/29/2018
实际学习开始日期: 12/31/2013
预计主要完成日期: 12/30/2016
预计完成日期: 12/30/2017

状况或疾病

Bone Cyst Aneurysmal
Pathological Fracture
Recurrent Disease
Refractory Tumor

干预/治疗

Drug: denosumab (Xgeva) treatment

相 1/相 2

手臂组

干预/治疗
Experimental: denosumab (Xgeva) treatment
Patients with aneurismal bone cysts received perioperative denosumab(Xgeva).
Drug: denosumab (Xgeva) treatment
Denosumab (Xgeva) 120 mg was given subcutaneously (abdomen wall or upper arms) every 4 weeks (Q4W), with a loading dose on both Day 8 and Day15 of the first cycle, perioperatively.

资格标准

有资格学习的年龄 12 Years 至 12 Years
有资格学习的性别All
接受健康志愿者
标准

Inclusion Criteria:

1. pathologically diagnosed with primary aneurysmal bone cyst

2. lesions in axial skeletons (i.e. spine, pelvis) which are difficult to reach a satisfactory surgical margin, and surgery can lead to very high incidence of complications.

3. pathological fracture

4. recurrence cases

Exclusion Criteria:

1. metastases or multi-focal lesions

2. malignancy

结果

主要结果指标

1. Objective Response Rate (ORR) [at least 1-year]

the number of participats (complete response+ partial response according to RECIST 1.1)/ total participants number.

2. local control rate [at least 1-year]

CT or MR proof that the tumor relapse in the primary site by at least two radiologist.

次要成果指标

1. Number of participants with abnormal laboratory values and/or adverse events that are related to the use of Denosumab(Xgeva) [at least 1-year]

include patient-reported adverse reactions, X-ray image of jawbone, serum calcium, alkaline phosphatase, renal and hepatic function, hemoglobin, white blood cell, and platelet count were collected in follow-up every 3-month.

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