在确定青少年系统性硬化症治疗方案之前需要考虑的问题:托珠单抗单药治疗有效安全成功的案例报告

IF 1.9 Q2 ORTHOPEDICS
M. Funauchi, K. Kinoshita
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引用次数: 2

摘要

青少年系统性硬化症(SSc)是一种罕见的疾病,它会导致多种疾病,包括生长迟缓和学习障碍,以及多器官纤维化和微血管疾病导致的生活质量受损。最近,免疫抑制剂如环磷酰胺和霉酚酸酯以及生物制剂在成年SSc患者中的疗效已被报道。然而,由于其罕见性和某些患者皮肤硬化症可能自限性,对于青少年SSc的治疗尚未达成共识。在这里,我们报告了一例13年发病的SSc,除了皮肤硬化,间质性肺病和可能的心肌纤维化外,还伴有生长迟缓和学习障碍,该病例成功地使用tocilizumab单药治疗,没有明显的不良反应。由于需要对患者的成长和教育以及标准治疗进行仔细的个案管理,因此此类病例的记录对于评估青少年SSc的有效和安全治疗非常重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Problems to Consider Before Determining the Regimen of the Treatment for Juvenile Systemic Sclerosis Treatment: A Case Report Where Tocilizumab Monotherapy Succeeded Efficiently and Safely
Juvenile systemic sclerosis (SSc) is a rare condition that results in various disorders, including growth retardation and learning disabilities in addition to impaired quality of life due to fibrosis and microvascular disorders in multiple organs. Recently, efficacies of immunosuppressants such as cyclophosphamide and mycophenolate mofetil, as well as biological agents, have been reported in adult patients with SSc. However, there has been no consensus in the treatment of juvenile SSc due to its rarity and the fact that skin sclerosis may be self-limiting in some patients. Here, we present a case of 13-year-onset SSc with growth retardation and learning disabilities, in addition to skin sclerosis, interstitial lung disease, and possible myocardial fibrosis that was successfully treated with tocilizumab monotherapy without remarkable adverse reactions. As careful case-by-case management of patient’s growth and education along with standard treatment is needed, the documentation of such case is important for the evaluation of the efficient and safe therapy for juvenile SSc.
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来源期刊
CiteScore
4.40
自引率
0.00%
发文量
14
审稿时长
8 weeks
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