Département de chirurgie infantile, HFME, 59, boulevard Pinel, 69677 Bron, France.
IHOP, 1, place Professeur Joseph-Renaut, 69008 Lyon, France.
Orthop Traumatol Surg Res. 2019 Oct;105(6):1181-1185. doi: 10.1016/j.otsr.2019.04.028. Epub 2019 Jul 26.
Aneurysmal bone cyst (ABC) is a benign tumour whose progression involves the RANK/RANKL signalling pathway. Surgery is the reference standard treatment but carries risks that vary with the site of the tumour. Denosumab is a human monoclonal IgG2 antibody that targets the RANK/RANKL pathway and may therefore hold promise for inhibiting ABC progression. The objective of this study was to evaluate denosumab use in paediatric patients (younger than 18 years) with ABC and to describe the clinical and radiological outcomes, as well as the side effect profile.
Denosumab is a viable option in children with ABC refractory to standard treatments.
We retrospectively reviewed the medical files of paediatric patients given denosumab to treat ABC in any of 32 centres affiliated with the French Paediatric Cancer Society (Société Française du Cancer de l'Enfant, SFCE) and French Sarcoma Group (Groupe Sarcome Français, GSF-GETO). We identified 5 patients treated between March 2015 and June 2018. Median age was 8 years (range, 7-17 years). Pain was a symptom in all 5 patients and neurological deficits were present in 3 patients. Surgery was performed in 4 patients, either before (n=3) or after (n=1) denosumab therapy; the remaining patient had no surgery. Denosumab was given as monthly injections in a dosage of 70mg/m for a median of 12 months (range, 4-23 months). The clinical outcomes and changes in computed tomography and/or magnetic resonance imaging findings were evaluated.
Abnormalities in calcium and phosphate levels secondary to the ABC occurred in 2 patients. At median of 24 months (range, 0-28 months) after denosumab initiation, all 5 patients were free of pain, and the neurological deficits in 3 patients had improved. Central remineralisation and cortical reconstitution were demonstrated consistently by the imaging studies.
Denosumab is a viable treatment option in selected paediatric patients with inoperable ABC. The immediate adverse effect profile is acceptable. A larger study with a longer follow-up would be welcome to further assess the contribution of denosumab to the treatment of ABC.
IV.
动脉瘤样骨囊肿（ABC）是一种良性肿瘤，其进展涉及 RANK/RANKL 信号通路。手术是参考标准治疗方法，但存在风险，且风险因肿瘤部位而异。地舒单抗是一种靶向 RANK/RANKL 通路的人源化 IgG2 单克隆抗体，因此可能有望抑制 ABC 的进展。本研究的目的是评估地舒单抗在患有 ABC 的儿科患者（<18 岁）中的应用，并描述其临床和影像学结果以及不良事件谱。
地舒单抗是对标准治疗无效的 ABC 患儿的可行选择。
我们回顾性分析了在法国儿科癌症协会（Société Française du Cancer de l'Enfant，SFCE）和法国肉瘤组（Groupe Sarcome Français，GSF-GETO）的 32 个中心接受地舒单抗治疗 ABC 的儿科患者的病历。我们共确定了 5 例于 2015 年 3 月至 2018 年 6 月期间接受治疗的患者。中位年龄为 8 岁（范围：7-17 岁）。5 例患者均有疼痛症状，3 例患者有神经功能缺损。4 例患者接受了手术，3 例患者在接受地舒单抗治疗前（n=3），1 例患者在接受地舒单抗治疗后（n=1）接受了手术；另 1 例患者未接受手术。地舒单抗的剂量为 70mg/m2，每月一次，中位治疗时间为 12 个月（范围：4-23 个月）。评估了临床结局和计算机断层扫描和/或磁共振成像结果的变化。
2 例患者因 ABC 导致钙和磷水平异常。在开始使用地舒单抗后中位 24 个月（范围：0-28 个月），所有 5 例患者均无疼痛，3 例患者的神经功能缺损均有改善。影像学研究始终显示出中央再矿化和皮质重建。
地舒单抗是一种可行的治疗选择，可用于治疗某些无法手术的 ABC 的儿科患者。即刻不良事件谱是可以接受的。更大规模、随访时间更长的研究将有助于进一步评估地舒单抗对 ABC 治疗的贡献。
IV。
