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成骨不全症:从自身免疫和免疫治疗角度进行探索

Osteogenesis imperfecta: exploring an autoimmune and immunotherapy perspective.

作者信息

Goddard Jackson F, Mehrotra Shikhar, Mehrotra Meenal

机构信息

Department of Surgery, Medical University of South Carolina, Charleston, SC 29425, United States.

出版信息

JBMR Plus. 2025 Apr 9;9(6):ziaf053. doi: 10.1093/jbmrpl/ziaf053. eCollection 2025 Jun.

Abstract

Osteogenesis imperfecta (OI), also called brittle bone disease, is a genetic osteodysplasia characterized by a defect in type 1 collagen. Often diagnosed in infancy or early childhood, young patients are affected by frequent fractures. Osteogenesis imperfecta was first named almost 200 yr ago, yet there are still no FDA-approved treatments for OI, and existing treatments target only the skeletal defects of the disease. In this review, we briefly examine current treatments and ongoing clinical trials. Then, by analyzing OI with an osteoimmunological perspective, we have compiled evidence that OI has an autoimmune component. This autoimmune component of OI remains unconsidered, even though an immunology-based therapy has shown promise in treating OI. Acknowledging an autoimmune component of OI is critical to understanding its mechanisms and allowing for the development of more efficacious treatments and novel immunotherapies. Considering the existing literature and the growing impact of immunotherapeutic therapies in cancer and other autoimmune diseases, we believe it may be time to rethink the immune aspects of this genetic disorder and develop novel immunomodulating strategies to improve the quality of life for OI patients.

摘要

成骨不全症(OI),也称为脆骨病,是一种遗传性骨发育异常,其特征是I型胶原蛋白存在缺陷。通常在婴儿期或幼儿期被诊断出来,年轻患者经常受到骨折的影响。成骨不全症在近200年前首次被命名,但目前仍没有美国食品药品监督管理局(FDA)批准的针对成骨不全症的治疗方法,现有的治疗方法仅针对该疾病的骨骼缺陷。在本综述中,我们简要研究了当前的治疗方法和正在进行的临床试验。然后,通过从骨免疫学角度分析成骨不全症,我们收集了证据表明成骨不全症存在自身免疫成分。尽管基于免疫学的疗法在治疗成骨不全症方面已显示出前景,但成骨不全症的这种自身免疫成分仍未得到考虑。认识到成骨不全症的自身免疫成分对于理解其发病机制以及开发更有效的治疗方法和新型免疫疗法至关重要。考虑到现有文献以及免疫疗法在癌症和其他自身免疫性疾病中日益增加的影响,我们认为可能是时候重新思考这种遗传性疾病的免疫方面,并开发新的免疫调节策略来改善成骨不全症患者的生活质量了。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0b8c/12063996/54134ac17a96/ziaf053f1.jpg

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