一名麦库恩-阿尔布莱特综合征儿科患者接受布罗苏单抗治疗后纤维组织增生症得到改善：病例报告。

Q4 Medicine JBJS case connector Pub Date : 2024-09-20 eCollection Date: 2024-07-01 DOI:10.2106/JBJS.CC.24.00279

Kenta Sawamura, Takashi Hamajima, Hiroshi Kitoh

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引用次数: 0

摘要

病例：布罗索单抗是一种新型药物，用于治疗遗传性成纤维细胞生长因子 23（FGF23）相关疾病。我们报告了一例患有麦库恩-阿尔布莱特综合征（McCune-Albright Syndrome，MAS）的 11 岁女孩的病例，她因过多的 FGF23 和纤维发育不良（FD）的多发性骨病变而导致低磷血症。Burosumab疗法不仅明显改善了生化指标，还改善了FD病变的影像学外观和临床症状：这是第一份证明布罗苏单抗能有效改善 MAS 患者 FD 病变的报告。

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Improvement of Fibrous Dysplasia After Burosumab Therapy in a Pediatric Patient with McCune-Albright Syndrome: A Case Report.

Case: Burosumab is a novel drug developed to treat hereditary fibroblast growth factor 23 (FGF23)-related disorders. We report the case of an 11-year-old girl with McCune-Albright syndrome (MAS) who sustained hypophosphatemia due to excess FGF23 and multiple bone lesions of fibrous dysplasia (FD). Burosumab therapy markedly improved not only the biochemical parameters but also the radiographic appearance of the FD lesions and clinical symptoms.

Conclusion: This is the first report to demonstrate that burosumab is effective in improving FD lesions in a patient with MAS.

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来源期刊

JBJS case connector Medicine-Medicine (all)

CiteScore

0.60

自引率

0.00%

发文量

319

期刊介绍： JBJS Case Connector helps improve patient care by providing the medical community with a journal that harnesses technology to provide information tools for discovery and reporting of unusual musculoskeletal problems, findings, treatment, and outcomes. Co-edited by Thomas W. Bauer, MD, PhD, and Ronald W. Lindsey, MD, JBJS Case Connector assists orthopaedic surgeons in the search for precedents, connections, and trends in their efforts to improve patient care. Using this unique journal, surgeons can find the commonalities between cases, benefit from the experience of their peers, and filter case information by many important variables in order to provide the best possible care for orthopaedic patients. This cross-referenced online journal includes thousands of orthopaedic case reports. It compiles symptoms, conditions, and demographic details to empower surgeons to find cases similar to theirs. Surgeons can mine the database to reveal emerging trends and identify patterns, distinguishing between truly rare cases and repeated, related single instances of a larger problem. The JBJS Case Connector Image Quiz feature provides interactive quizzes based on images from content published by JBJS and includes a discussion area for further exploration of ideas and concepts. The JBJS Image Quiz app for iPad and iPhone is available in the App Store. Contributions to JBJS Case Connector are welcomed from anywhere in the world and are considered on their merits. Articles must be written in English and should be submitted as outlined in the Instructions to Authors. All authors must abide by the JBJS ethics policies and all submissions to JBJS Case Connector are covered by the JBJS embargo policy.