布罗索尤单抗治疗皮肤骨骼低磷血症综合征。
Burosumab for the treatment of cutaneous-skeletal hypophosphatemia syndrome.
作者信息
Abebe Lillian, Phung Kim, Robinson Marie-Eve, Waldner Richelle, Carsen Sasha, Smit Kevin, Tice Andrew, Lazier Joanna, Armour Christine, Page Marika, Dover Saunya, Rauch Frank, Koujok Khaldoun, Ward Leanne M
机构信息
The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
Department of Pediatrics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
出版信息
Bone Rep. 2023 Nov 11;20:101725. doi: 10.1016/j.bonr.2023.101725. eCollection 2024 Mar.
Cutaneous-skeletal hypophosphatemia syndrome (CSHS) is a rare bone disorder featuring fibroblast growth factor-23 (FGF23)-mediated hypophosphatemic rickets. We report a 2-year, 10-month-old girl with CSHS treated with burosumab, a novel human monoclonal antibody targeting FGF23. This approach was associated with rickets healing, improvement in growth and lower limb deformity, and clinically significant benefit to her functional mobility and motor development. This case report provides evidence for the effective use of FGF23-neutralizing antibody therapy beyond the classic FGF23-mediated disorders of X-linked hypophosphatemia and tumor-induced osteomalacia.
皮肤骨骼低磷血症综合征（CSHS）是一种罕见的骨骼疾病，其特征为成纤维细胞生长因子23（FGF23）介导的低磷性佝偻病。我们报告了一名2岁10个月大患有CSHS的女孩，使用布罗索尤单抗进行治疗，布罗索尤单抗是一种新型的靶向FGF23的人单克隆抗体。这种治疗方法使佝偻病愈合，生长及下肢畸形得到改善，对其功能活动能力和运动发育产生了具有临床意义的益处。本病例报告为FGF23中和抗体疗法在经典的FGF23介导的X连锁低磷血症和肿瘤诱导的骨软化症之外的有效应用提供了证据。
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